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Conversano, Ester, Anna Maria Chiara Galimberti et Martina Bevacqua. « Sindrome nefrosica : raccomandazioni terapeutiche oltre le linee guida SINePe ». Medico e Bambino 39, no 9 (9 novembre 2020) : 591–95. http://dx.doi.org/10.53126/meb39591.
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Da Rin Della Mora, Roberta. « Le cadute pediatriche in ospedale : una indagine italiana ». PROFESSIONI INFERMIERISTICHE, no 1 (2013) : 48–54. http://dx.doi.org/10.7429/pi.2013.661048.
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Tiozzo, Emanuela, Valentina Biagioli, Matilde Brancaccio, Riccardo Ricci, Anna Marchetti, Immacolata Dall’Oglio, Orsola Gawronski et al. « Using an App to monitor postoperative pain at home in pediatric patients ». Journal of Child Health Care 25, no 2 (11 février 2021) : 212–24. http://dx.doi.org/10.1177/1367493520919313.
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A prospective comparative study was conducted in 487 pediatric patients (69% male, mean age = 6.4 ± 4.0) to evaluate (a) the incidence, intensity, and characteristics of pain in pediatric patients at home during the first 24 hours and 5 days after surgery and (b) the factors associated with higher pain intensity, including the impact of an application (App) compared to the paper-and-pencil approach. Postoperative pain was assessed by patients or their parents at home using the ‘Bambino Gesù’ Children’s Hospital (Ospedale Pediatrico Bambino Gesù, OPBG) tool for participants aged 4–17 years or the Faces, Legs, Arms, Cry, and Consolability scale for participants less than four years old. Participants were assigned to two groups: those who used the paper-and-pencil version of the pain scale and those who used the App. Overall, 209 of the 472 (44%) participants reported pain during the first 24 hours, and 92 of the 420 (22%) reported pain between one and five days after surgery. Higher pain intensity scores were associated with being in the App group, directly assessing own pain, and using the OPBG tool. The App was effective in facilitating pain assessment. Health professionals could empower pediatric patients and their parents in assessing pain at home through a dedicated App.
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Ozbay, I., C. Kucur, F. E. Koçak, B. Savran et F. Oghan. « ACTA OTORHINOLARYNGOLOGICA ITALICA ». Acta Otorhinolaryngologica Italica 36, no 5 (octobre 2016) : 381–85. http://dx.doi.org/10.14639/0392-100x-897.
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Lobiettivo del presente studio è stato determinare se i livelli plasmatici dei prodotti avanzati di ossidazione proteica (AOPP) rappresentino dei marker di stress ossidativo nei pazienti pediatrici affetti da tonsillite cronica. Per lo studio sono stati arruolati, presso i Dipartimenti di Otorinolaringoiatria e Chirurgia pediatrica dellOspedale Universitario di Dumlupinar, trenta bambini sani e trenta affetti da tonsillite cronica. Il gruppo dei pazienti affetti da malattia è stato sottoposto a un prelievo ematico preoperatorio e ad una biopsia intraoperatoria del tessuto tonsillare. Il gruppo dei pazienti sani è stato sottoposto unicamente al prelievo ematico. I livelli plasmatici e tissutali degli AOPP sono quindi stati misurati mediante spettrofotometria. I livelli sierici degli AOPP sono risultati essere più elevati nel gruppo dei pazienti affetti da tonsillite cronica (13,1 ± 3,3 ng/ml) rispetto al gruppo di controllo (11,6 ± 2,3 ng/ml; P < 0,05). Il livello tissutale medio degli AOPP nei pazienti malati è risultato essere superiore a quello plasmatico medio sia nel gruppo dei pazienti sani che in quello dei pazienti malati (41,9 ± 13,5 ng/mg; P < 0,05). I livelli plasmatici e tissutali degli AOPP sono risultati quindi essere più elevati nei pazienti malati rispetto al gruppo di controllo. Gli AOPP potrebbero quindi rappresentare una nuova classe di molecole pro-infiammatorie coinvolte nello stress ossidativo nella tonsillite cronica e potrebbero avere un ruolo come marker di stress ossidativo nei pazienti pediatrici affetti da tale patologia.
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Neri, Enrica, Francesca Agostini, Michela Poggiali, Marcello Stella, Augusto Biasini et Fiorella Neri. « Care e follow up : ospedale e università ». QUADERNI ACP 28, no 3 (2021) : 131–33. http://dx.doi.org/10.53141/qacp.2021.131-133.
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The collaboration between Bufalini Hospital’s NICU (Neonatal Intensive Care Unit), Cesena, and the Developmental Psychodynamics Laboratory of Bologna’s Psychology Department, Cesena branch, has led to a shared reflection regarding both the Care of preterm births under 1500 grams, from 3 to 24 months of corrected age, and of a multidisciplinary neuro and psychodevelopmental follow up procedure . The studies, published in international journals, have led to the formulation of new research hypotheses and clinical reflection.
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Bovo, R., P. Trevisi, E. Zanoletti, D. Cazzador, T. Volo, E. Emanuelli et A. Martini. « New trends in rehabilitation of children with ENT disorders ». Acta Otorhinolaryngologica Italica 37, no 05 (octobre 2017) : 355–67. http://dx.doi.org/10.14639/0392-100x-1426.
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Negli ultimi 20 anni il miglioramento dellassistenza neonatale ha determinato un progressivo aumento dei bambini che sopravvivono in presenza di gravi malformazioni o patologie congenite. Questi bambini richiedono una riabilitazione prolungata, talora multidisciplinare e complessa. Purtroppo, unorganizzazione adeguata alla riabilitazione della disfagia, dei disturbi della comunicazione e della respirazione non è sempre disponibile, non è sempre coordinata in equipe multidisciplinari che operino sia negli ospedali che sul territorio e non è facile mantenere tutte le figure professionali coinvolte al passo con le sempre più rapide innovazioni. Scopo del presente lavoro è presentare un aggiornamento su alcuni aspetti tuttora controversi della riabilitazione in età pediatrica.
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Kahn, L. « The "Ospedale degli Innocenti" and the "Bambino" of the American Academy of Pediatrics ». PEDIATRICS 110, no 1 (1 juillet 2002) : 175–80. http://dx.doi.org/10.1542/peds.110.1.175.
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Monteu, Francesca, Gabriella D'Alonzo et Raffaele Nuzzi. « Pediatric Pseudotumor Cerebri : Epidemiological Features. » Open Ophthalmology Journal 14, no 1 (14 septembre 2020) : 47–58. http://dx.doi.org/10.2174/1874364102014010047.
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Objective: The purpose was to study the epidemiological and clinical features of Pediatric Pseudotumor Cerebri; we especially evaluated age, sex, sexual maturity, weight, symptoms, duration of the episode, visual acuity, correlation with systemic diseases or drugs. Methods: We investigated a population of 40 children (80 eyes), aged between 2 and 18 years, that referred to our Centre (Città della Salute e della Scienza di Torino - presidio Ospedale Regina Margherita) from 2000 to 2018. All the patients were monitored with visual field exam, fundus exam and Frisen classification, pupillary reflexes, best corrected visual acuity, orthoptic exam. Then, the demographic characteristics, BMI (body max index) and weight changes, the correlation with systemic diseases or drugs, the Relative Afferent Pupillary Defect (RAPD), the VI cranial nerve deficit, the time required for the diagnosis, the modality of onset, the correlation between diagnosis delay and best corrected visual acuity, the resolution time and the probability of recurrence were studied. Results: We observed 2 age peaks in our population, one consisting of adolescents (aged between 11 and 14 years) and one consisting of younger children aged between 4 and 7 years, with a statistically significant correlation between age and BMI. The deficit of the VI cranial nerve and the diplopia were resolved within the first month, despite the longer time of resolution of the papilledema. Headache was the main onset symptom, but after reclassifying onset symptoms per weight classes, in overweight children, we found an increased variability of symptoms (dizziness, postural instability, photophobia, nausea, vomit, strabism and diplopia, visual fogging, drowsiness, irritability). Moreover, we found only one totally asymptomatic case, during a routine visit, in a 6 years old overweight child with Down syndrome. Conclusion: The Pediatric Pseudotumor Cerebri is, even nowadays, a little known pathology. To provide a correct diagnostic assessment, it will be imperative to recollect the epidemiological features of this disease.
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Castignani, Anna Maria. « Genitori, infermieri e medici in ospedale. Il gruppo in oncoematologia pediatrica : un modello di riflessione e di intervento ». INTERAZIONI, no 2 (novembre 2018) : 77–93. http://dx.doi.org/10.3280/int2018-002007.
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Gawronski, Orsola, et Susanna Padrini. « Parents' appraisal of children's pain : a grounded theory ; Mayo Clinic/Ospedale Pediatrico Bambino Gesù joint nursing research project ». Paediatrics and Child Health 19 (décembre 2009) : S182—S184. http://dx.doi.org/10.1016/j.paed.2009.08.008.
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Tonizzi, Elisabetta M. « Una discussione su un libro controverso : Rockefeller d'Italia di Paride Rugafiori. Gerolamo Gaslini. Affari, politica e filantropia ». ITALIA CONTEMPORANEA, no 259 (novembre 2010) : 314–19. http://dx.doi.org/10.3280/ic2010-259007.
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L'industriale Gerolamo Gaslini (1877-1964), attivo nel campo degli oli vegetali e dell'industria alimentare, ebbe grande successo e solida reputazione imprenditoriale. Nel 1938 costruě a Genova l'Istituto Giannina Gaslini, ospedale pediatrico rinomato a livello internazionale, intitolato alla figlia minore morta prematuramente. Per finanziarlo e gestirlo, nel 1950 donň tutto il suo ingente patrimonio alla Fondazione Gerolamo Gaslini. Quest'ultima ha promosso la ricerca sul suo benefattore, conclusa nel 2006. Senza spiegazioni, la fondazione ha poi rifiutato di pubblicarla. Con tre anni di ritardo, l'autore, Paride Rugafiori, ha deciso, per difendere l'autonomia della ricerca scientifica, di pubblicare il libro, basato su una corposa mole di fonti primarie. Questa documentazione ha permesso di quantificare esattamente l'entitŕ dell'evasione fiscale delle imprese di Gaslini. Al dibattito partecipano tre discussant: due storici, M. Elisabetta Tonizzi e Fabio Levi, e un filosofo, Luca Bagetto, che propongono chiavi di lettura diversificate in base alle loro competenze. Nella sua risposta l'autore, oltre a commentare con precisione i loro rilievi critici, sottolinea l'impianto multidisciplinare del volume.
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Johnson, Robert V. « Caring for intubated newborns : analgesia and sedation. Collaborative discussions between the Ospedale Pediatrico Bambino Gesú and the Mayo Clinic ». Paediatrics and Child Health 19 (octobre 2009) : S15—S17. http://dx.doi.org/10.1016/j.paed.2009.05.034.
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Carmagnola, Daniela, Marilisa Toma, Dolaji Henin, Mariachiara Perrotta, Laura Gianolio, Alessandra Colombo et Claudia Dellavia. « Dental Emergencies in an Italian Pediatric Hospital during the COVID-19 Pandemic ». Healthcare 10, no 3 (15 mars 2022) : 537. http://dx.doi.org/10.3390/healthcare10030537.
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Emergency rooms (ER) are largely used by patients with oral complaints, who choose the ER over private or public dental offices for oral prevention and treatment. During the COVID-19 pandemic, the activity of most dental facilities was limited, and most hospitals and ERs were dedicated to the treatment of COVID-19 patients. The present study analyzed the number of and reason for visits at the emergency room (ER) of Ospedale dei Bambini “Vittore Buzzi”, the main pediatric hospital in Milano, Italy, between 2019 and 2020, with a particular focus on oral emergencies. In 2019, 25,435 children turned to the ER, compared to 16,750 in 2020. About 10% of the children were eventually admitted to the hospital in both years. The number of admissions for infectious diseases, other than COVID-19, signed an important decrease in 2020, while trauma/injuries decreased slightly in number but increased in proportion. The number and proportion of ER visits for oral complaints decreased in 2020 compared to 2019. Stomatitis was the most frequent condition, followed by traumatic injuries, which decreased in number and percentage between 2019 and 2020. Oral infections and painful caries accounted for about 15% of the cases in both 2019 and 2020. These data highlight the need to promote territorial services for the prevention and treatment of oral health complaints, including dental emergencies.
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Caccianiga, Paolo, Lorenzo Giovanni Mantovani, Marco Baldoni et Gianluigi Caccianiga. « Distribution of Malocclusion Traits in the Pediatric Population of Milan : An Observational Study ». International Journal of Environmental Research and Public Health 19, no 21 (30 octobre 2022) : 14199. http://dx.doi.org/10.3390/ijerph192114199.
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Background: Epidemiological investigations define the prevalence and distribution of the various types of malocclusions, and can help to identify etiological factors and set the most correct orthodontic therapy. Aim: The goal of this study was to verify the prevalence and distribution of various types of malocclusions in the pediatric population. Methods: The study was performed on a sample of 350 patients (ages 5–9) being treated at the ASST Grande Ospedale Metropolitano Niguarda in Milan. A comparison was presented with one similar epidemiological investigation conducted 22 years earlier by the same researchers. The values of the malocclusion indices were reported from the cephalometric analyzes of the patients and were differentiated on the basis of gender and ethnicity. Results: The predominant traits of malocclusion in the general population of the analyzed sample were: skeletal class II (47.43%), hyper-divergence (40.86%), maxillary retrusion (46%), mandibular retrusion (66%), maxillary hypoplasia (50%), mandibular hypoplasia (49.14%), Wits index > 2 mm (22.57%); overjet > 4 mm (31.1%) and overbite > 4 mm (24.86%). Substantial differences were found between Italian patients and patients belonging to different ethnic groups in almost all parameters, and between the male and female genders in some of them. Patients in the 2000 study had a higher prevalence of Class II and hyper-divergence. Conclusions: This epidemiological investigation can suggest different approaches in setting the orthodontic treatment plan based on the ethnic group of the patient taken in charge and encourage more specific and large-scale analytical studies on the subject.
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Wood, John C., Raffaella Origa, Annalisa Agus, Gildo Matta, Thomas D. Coates et Renzo Galanello. « Onset of Cardiac Iron Loading in Pediatric Patients. » Blood 110, no 11 (16 novembre 2007) : 2765. http://dx.doi.org/10.1182/blood.v110.11.2765.2765.
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Abstract Patients with thalassemia major develop life threatening cardiac complications in their teens and twenties from iron overload. Cardiac MRI allows us to diagnosis preclinical cardiac iron deposition, but it is not known at what age T2* screening should be initiated. Historical data and pilot MRI studies suggest that patients must reach a critical transfusional exposure prior to cardiac iron uptake. This study was a two-institution characterization of the prevalence of cardiac iron overload in 77 pediatric patients. Methods: Study was performed at the Ospedale Regionale Microcitemie in Cagliari and the Childrens Hospital Los Angeles (CHLA). Retrospective review of medical records was authorized by the IRB for pediatric patients undergoing cardiac MRI prior to July of 2007. Subjects at both institutions had assessment of cardiac T2* and cardiac function using validated techniques on a 1.5 T General Electric CVi scanner; patients at CHLA also underwent MRI-based liver iron measurements. Complete transfusional iron burden was measured in the Italian patients. Serial MRI data was recorded in 30 patients, but associations between cardiac T2* and age were determined using linear and logistic regression using only results from the first patient MRI scan. Results: Patient ages ranged from 8.0 to 18 years of age at Cagliari (n=36) and 2.5 to 17.9 years of age at CHLA (n=41), reflecting the use of MRI to monitor liver iron in patients < 8 years of age. Patients were moderately iron loaded with hepatic iron concentrations of 12.7 ± 9.8 (CHLA) and ferritin values of 2329 ± 1162 (Cagliari). Median cardiac T2* was 30.2 and ranged from 3.4 to 72.8. Cardiac T2* and its reciprocal were uncorrelated with liver iron (CHLA) and ferritin levels (Cagliari). Figure 1 demonstrates the decrease in cardiac T2* with increasing chronologic age. Serial data are connected by lines. Although linear regression was weakly positive (r2 = 0.06, p = 0.03), the relationship was fundamentally nonlinear. No patient below the age of 9.5 years of age demonstrated an abnormal cardiac T2* (< 20 ms) while 36% of patients between the ages of 15–18 years had detectable cardiac iron. Figure 2 demonstrates the logistic regression curve modeling the prevalence of detectable cardiac iron as a function of age (r2= 0.13, p< 0.002). Chronologic age was highly correlated (r2 = 0.88) with both transfusional iron burden and with duration of transfusion therapy, making it impossible to separate the relative importance of these three variables. Conclusion: Thalassemia major patients did not accumulate cardiac iron until early in their second decade of life. Consequently, cardiac iron monitoring can be safely deferred until children are able undergo MRI examination without sedation. Figure Figure Figure Figure
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Perasso, Giulia, Chiara Allegri et Gloria Camurati. « Child Play Specialist e Child Life Specialist : ruolo, evoluzione storica e benefici per il paziente pediatrico. Una rassegna della letteratura ». PSICOLOGIA DELLA SALUTE, no 3 (octobre 2021) : 57–81. http://dx.doi.org/10.3280/pds2021-003011.
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Il gioco rappresenta un diritto fondamentale del bambino ed assume un ruolo cruciale du-rante l'esperienza dell'ospedalizzazione. Le figure del Child Play Specialist e del Child Life Specialist (riconosciute rispettivamente nel sistema sanitario del Regno Unito e degli Stati Uni-ti) forniscono programmi di gioco che normalizzino l'esperienza di ricovero del bambino e aiutino la familiarizzazione con il contesto medico. La presente rassegna persegue tre principali obiettivi: i. definire il ruolo dello specialista del gioco, la sua formazione, le tecniche implemen-tate, le aree di similitudine e differenza tra CPS e CLS; ii. esaminare l'evoluzione storica di questa professione; iii. indagare gli effetti dell'intervento dello specialista del gioco sulla salute bio-psico-sociale del paziente pediatrico. Di 613 fonti complessive (n=193 da Scopus, n=403 da Pubmed, n=17 da PsycInfo), 17 pubblicazioni sono state incluse, avendo applicato criteri inerenti lingua, tipologia di pubblicazione e pertinenza dei contenuti. Dalle fonti esaminate ri-sulta che: I. CLS e CPS presentano percorsi di training e tecniche simili. Emergono peculiarità di approccio diverse per CPS e CLS; II. Le esperienze pionieristiche di programmi di gioco in ospedale risalgono agli anni '20 con contributi significativi di Plank, Bergmann e Brooks; III. I principali effetti sulla salute infantile documentati sono il potenziamento del coping e la ridu-zione del ricorso a terapie farmacologiche per la gestione del dolore. Si evidenzia la necessità di un consenso internazionale sulla definizione del ruolo dello specialista del gioco, al fine di accrescere la ricerca empirica rispetto a tali professionisti della salute.
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Gatelli, Italo Francesco, Ottavio Vitelli, Gaia Chiesa, Francesca De Rienzo et Stefano Martinelli. « Noninvasive Cardiac Output Monitoring in Newborn with Hypoplastic Left Heart Syndrome ». American Journal of Perinatology 37, S 02 (septembre 2020) : S54—S56. http://dx.doi.org/10.1055/s-0040-1713603.
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Objective This study aimed to describe the first two cases of electrical cardiometry applied to newborn with hypoplastic left heart syndrome for hemodynamical assessment in the first days of life before surgical correction and see if this can help decision making process in these patients. Study Design We describe two case series of two full-term newborn with hypoplastic left heart syndrome in the Neonatal Intensive Care Unit, ASST Grande Ospedale Metropolitano Niguarda, between December 2019 and January 2020. Results Case 1 was persistently hemodynamically stable with prostaglandin E1 infusion at 0.01 mcg/kg/min, showing good capillary refill time, good diuresis, no difference between pre- and postductal values of oxygen saturation or blood pressure. Electrical cardiometry monitoring constantly showed cardiac output values higher than 300 mL/kg/min. Case 2 showed poor clinical condition needing prostaglandin E1 infusion up to 0.05 mcg/kg/min, intubation and septostomy associated with low cardiac output around 190 mL/kg/min. Once cardiac output has begun to rise and reached values constantly over 300 mL/kg/min, clinical condition improved with amelioration in oxygen saturation, diuresis, blood pressure, and blood gas analysis values. She was then extubated and finally clinically stable until surgery with minimal infusion of prostaglandin E1 at 0.01 mcg/kg/min. Conclusion This case highlights how hemodynamic information provided by electrical cardiometry can be used to supplement the combined data from all monitors and the clinical situation to guide therapy in these newborns waiting surgery. Key Points
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Merli, Pietro, Daria Pagliara, Tommaso Mina, Valentina Bertaina, Giuseppina Li Pira, Stefania Lazzaro, Simone Biagini et al. « Alphabeta T and B-Cell Depleted HLA-Haploidentical Hematopoietic Stem Cell Transplantation (TBdepl-haploHSCT) in Children with Myelodysplastic Syndromes ». Blood 138, Supplement 1 (5 novembre 2021) : 170. http://dx.doi.org/10.1182/blood-2021-153913.
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Abstract Background: Pediatric myelodysplastic syndromes (MDSs) are a heterogeneous group of clonal disorders, accounting for less than 5% of childhood hematologic malignancies. Usual indications to HSCT are: MDSs with excess of blasts, MDSs secondary to previously administered chemoradiotherapy and RCC associated with monosomy 7, complex karyotype, severe neutropenia, or erythrocyte/platelet transfusion dependence [Locatelli & Strahm, Blood 2018]. We previously demonstrated that TBdepl-haploHSCT is a suitable option for children with acute leukemia, with outcomes comparable to those reported in studies using either an HLA-identical sibling or an unrelated volunteer as donor. Here we present the results of this approach in children with MDSs. Patients and methods: Between February 2013 and February 2021, 23 children with MDSs other than juvenile myelomonocytic leukemia received TBdepl-haploHSCT from an HLA-partially matched relative at Ospedale Pediatrico Bambino Gesù, Rome, Italy or at IRCCS Fondazione Policlinico San Matteo, Pavia, Italy as part of a prospective study (#NCT01810120). All patients were prepared to the allograft using a fully-myeloablative conditioning regimen including a combination of cytotoxic drugs and/or total body irradiation (TBI). Anti-T-lymphocyte globulin (ATLG) was used before transplantation (12 mg/kg total dose, from days -5 to day -3) to modulate bi-directional donor/recipient alloreactivity. Rituximab (200 mg/sqm) was administered on day -1 to prevent post-transplantation EBV-induced lymphoproliferative disorders (PTLD). No patient received any post-transplant pharmacological GvHD prophylaxis. Results: Characteristics of patients enrolled in the study are shown in Table 1 (which reports also donor and graft characteristics). Median follow-up of surviving patients is 4.2 years (range: 0.5 - 8.5 years). Seventeen children were affected by refractory cytopenia of childhood (RCC) (2 cases occurring in the context of inherited bone marrow failure syndromes: one had GATA2 deficiency and the other SAMD9L mutation), while 1 and 5 were affected by MDS with excess of blasts 1 (EB1) and EB2 (one had GATA2 deficiency), respectively. Median time to neutrophil and platelet recovery was 14 (range 10-19) and 11 (range 9-14) days, respectively, with four patients (3 with RCC and 1 with EB2) experiencing primary graft failure, the cumulative incidence of this complication being 17.3% (95% CI 0.3-31.5). All these 4 patients were rescued with a second TBdepl-haploHSCT from the same or the other parent. Cumulative incidence of grade II-III acute GvHD was 11.4% (95% CI 0-25.2). One patient developed skin and gut GvHD after the second TBdepl-haploHSCT, while for all other patients skin was the sole organ involved; no case of grade IV GvHD was observed. One patient developed moderate chronic GvHD [cumulative incidence 5.2% (95% CI 0-14.8)], which completely resolved with low-dose steroids and ruxolitinib. Notably, no patient died for transplant-related complications. Six patients experienced CMV, 2 HHV-6 and 1 adenoviral infection/reactivation; one patient developed lung aspergillosis, which resolved with specific treatment. One patient affected by EB2, not in remission at time of transplant, relapsed 27 months after HSCT, the 5-year cumulative incidence of relapse being 7.1% (95% CI, 0-19.7); she eventually died after failing a second HSCT. The 5-year probability of overall and event-free survival were 92.3% (95% CI 56.6 -98.9) and 76.3% (95% CI 51.3-89.6) (Figure 1A and B), respectively. Five-year disease-free-survival was 90% (95% CI 47.3-98.5). Because of the low number of events, no prognostic factor related to OS and EFS was found. In particular, the MDS variant did not influence the patient's outcome. The median CD3+ cell count on day +30, +90, +180 and +360 were 113, 171, 558 and 1307/mcl, respectively. Conclusions: These data indicate that TBdepl-haploHSCT is a safe and effective transplant option also in children with MDS. Indeed, the low risk of both non-relapse mortality and acute/chronic GvHD makes this approach particularly attractive in the pediatric setting. Moreover, this haplo strategy compares favorably with T-cell replete approaches [Suo et al., 2020]. Figure 1 Figure 1. Disclosures Merli: JAZZ: Consultancy; SOBI: Consultancy. Locatelli: Miltenyl: Honoraria; Novartis: Consultancy, Membership on an entity's Board of Directors or advisory committees; Bellicum: Consultancy, Membership on an entity's Board of Directors or advisory committees; bluebird bio, Inc.: Consultancy; Amgen: Honoraria, Membership on an entity's Board of Directors or advisory committees.
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Simon-Lopez, Ramon, et Susanna Barella. « DAO-2, a New Software Tool for Multiple Differential Diagnoses with Probability. Application for the Screening of Rare Anemias in Sardinia ». Blood 124, no 21 (6 décembre 2014) : 4905. http://dx.doi.org/10.1182/blood.v124.21.4905.4905.
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Abstract The increasing number of laboratory parameters and diagnostic information makes every day more difficult to remember the entire differential Diagnosis list for all of them. The high prevalence or rare anemias in the island of Sardinia (Italy) makes mandatory to screen the general population for the possible presence of hemoglobinopathies. In fact, the prevalence of Beta Thalassemia goes from 9.1 % of the population in the City of Nuoro to 11.7% in the City of Muravera. The prevalence of Alpha Thalassemia genetic mutations is much more frequent, going from 9.1% in the City of Tempio to 39% in the City of Alghero. DAO-2 is a software tool made using C## (C Sharp) and SQL relational databases, that permit to do Multiple Differential Diagnoses that consist in the Differential Diagnosis of more than one symptom at the same time. After, using the probabilistic theory called Bayes Theorem or Rule of Bayes, gives the probability of every possible disease or medical condition. We have checked the utility of DAO-2 as a software tool for the screening of the rare anemias in Sardinia. The Ospedale Pediatrico Microcitemico is a Hospital a Specialized in thalassemia and other rare anemias. We have cheked the utility of DAO-2 in 394 consecutive patients with a known anemia that came to our hospital and also in 195 persons in that we have excluded the presence of anemia or genetic conditions that predispose to a rare anemia (controls). TableDiagnosticnNON BETA (control)195HbH (alfa thalasemia intermedia)129Thalassemia intermedia Splenectomized33Alfa Thalassemia Trait diag.suspected32Beta Thalassemia Trait30Iron Deficiency Anaemia22Thalassemia intermedia (Beta-Thal Interm)18Alfa Thalassemia Heterozygous14Thrombopenia11microdrepanocitosi (Beta-S)8Thalassemia homozygous (young children Intermedia/Major)7Beta Thal Thalassemia major transfused7HbH (Alfa Thalassemia intermedia) transfused6Hereditary Spherocytosis Splenectomized5Blackfan-Diamond Anemia3Dyserithropoietic Anaemia type I (CDA I)3Sickle cell anaemia3Iron Deficiency Anaemia treated3Piruvate-Kinase deficiency3IRIDA iron-refractory iron deficiency anemia3Beta Thalassemia major3Fanconi's Anaemia2Dyserithropoietic Anaemia type II (CDA II)2Hyporegenerative anemia2Anemia of Newborn2Megaloblastic Anemia1Piruvate-Kinase deficiency Splenectomized1Hemoglobinopaty (Taybe) + α- thalassemia1Hemoglobinopaty hyperunstable (Hb Cagliari)1Hemoglobinopaty Koln1Hemoglobinopaty E (Hb E)1Hemoglobinopaty G (Hb G-Copenaghen)1Hemoglobinopaty J (Hb J-SARDEGNA)1HbH (alfa thalasemia intermedia) Splenectomized1Leucopenia & Neutropenia1Metahemoglobinemia (MET-Hb)1Pyropoikilocytosis1Evans's Syndrome1Hereditary Spherocytosis28Delta-Beta Thalassemia (Sardegna)1HbH (alfa thalasemia intermedia) + Beta Thalassemia Trait1Total394 The use of this new software, called DAO-2 makes possible to predict in the majority of the cases the presence of a rare anemia or a heterozygous non clinical carrier of genetic changes that can provoke a rare anemia. It is necessary to do prospective studies to check the efficiency of this software before to use it in the clinical practice. Disclosures No relevant conflicts of interest to declare.
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Lucidi, Vincenzina, Giovanni Ciabattoni, Sergio Bella, Peter J. Barnes et Paolo Montuschi. « Exhaled 8-isoprostane and prostaglandin E2 in patients with stable and unstable cystic fibrosis☆⁎This work was performed at the Catholic University of the Sacred Heart, Rome, Italy, and Ospedale Pediatrico Bambino Gesù, Rome, Italy. This work was funded by the Catholic University of the Sacred Heart. » Free Radical Biology and Medicine 45, no 6 (septembre 2008) : 913–19. http://dx.doi.org/10.1016/j.freeradbiomed.2008.06.026.
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Graziadei, Giovanna, Laura Sainati, Pietro Bonomo, Donatella Venturelli, Nicoletta Masera, Maddalena Casale, Aurora Vassanelli et al. « Transfusion Therapy in a Multi-Ethnic Sickle Cell Population Real-World Practice. a Preliminary Data Analysis of Multicentre Survey ». Blood 132, Supplement 1 (29 novembre 2018) : 2389. http://dx.doi.org/10.1182/blood-2018-99-115938.
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Abstract Introduction. Despite the increasing of number of patients with Sickle Cell Disease (SCD) in Italy, due to multi-ethnic migratory phenomena, a large percentage of Caucasian sickle population is already present in Italy mainly with b-thal/HbS genotype. Red cell transfusion is one effective treatment for both acute and chronic complications of SCD, while hydroxycarbamide (HC) is used to reduce the frequency of painful vaso-occlusive crises (VOCs) and decrease the need for blood transfusion. Through the National Comprehensive Reference Centers for SCD, the Italian Society of Thalassemia and Hemoglobinopathies (SITE), in collaboration with the Society Italian Transfusion Medicine and Immunohematology (SIMTI) and the Italian Association of Hematology and Pediatric Oncology (AIEOP) conducted a national survey to collect information on different therapeutic approaches used for SCD patients. Aim. To assess therapeutic approaches used a large Italian cohort of patients with SCD, accounting for age, genotype and ethnicity. Patients and Methods. Observational Longitudinal Systemic Multicentre Study (https://clinicaltrials.gov/ct2/show/NCT03397017). Data were collected from 2015 to 2018 through a standard web-based application (www.SITE-italia.org) encrypted by the Central Server. All the SCD patients, treated or not treated, were included in order to identify the overall number and all gave written informed consent. The study was approved by Ethics Committee of Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico of Milan, Italy. Results. Thirty-four centers were involved from 14 Italian regions and 1,579 patients were enrolled (802 male and 777 female; median age 23 years - IQR, 25th-75th 10-41 yrs). Genotype, age and ethnicity distribution are shown in Table 1A. As expected, the median age of non-Caucasian patients, mainly HbSS, is significantly lower than Caucasian ones (p<0.001). Out of 1,579, 365 SCD patients (23%) did not receive any therapy. Acute transfusion regimen (ATR), Chronic transfusion regimen (CTR) and HC were given in monotherapy, respectively in 160, 226 and 197 patients, or in succession/combination in 631 (Table 1B), distributed throughout genotypes. The main reasons for ATR were acute anemia (384 events) and VOCs (352), followed by acute chest syndrome (ACS; 170), surgery (82), pregnancy (64), splenic sequestration (26), stroke (9); multi-organ failure (MOFs 6) and priapism (5). For CRT, it was acute anemia (306 events) and prevention of VOCs (371), ACS (107), primary stroke prevention (78) and secondary prevention stroke (55), pain HC-resistent (39) and leg ulcers (12). For 275 patients out of 631 it was possible to follow the timing of therapy switching (Table 1C). Of 275 patients, 67.6% switched from ATR/CRT to HC, 2.9% from HC to CRT and 6.5% stopped every therapy. Out of 275 patients, 104 were treated with overlapping therapeutic regimen. Discussion. The significant difference of age in Caucasian and non-Caucasian patients is probably due to the efficacy of the national prevention program of hemoglobinopathies, because the non-Caucasian patients are prevalently born out of Italy. The transfusional approach is similar in HbSS and b°-thal/HbS and b+-thal/HbS patients regarding both ATR and CTR. HbSC genotype needed less therapies(p <0.001), confirming a less severe clinical pattern. About the combo or sequential therapy, HC was the more frequent chronic therapy used lifelong, mainly in patients with HbSS, because of wide spread of age and transfusional match problems due to different ethnicity. Summary/Conclusion. The transfusional approach is similar in HbSS, b°-thal/HbS and b+-thal/HbS patients with similar indications, prevalently VOCs and anemia. The significant higher age in Caucasian cohort and the consequent long term follow up could be the cause of variable therapeutic approach observed, however Hydroxycarbamide seemed to be the therapy more frequently used and finally suggested to manage chronic manifestations. Figure. Figure. Disclosures Origa: Apopharma: Honoraria; Novartis: Honoraria; Bluebird Bio: Consultancy; Cerus Corporation: Research Funding. Forni:Apopharma: Other: DSM Board; Celgene: Research Funding; Novartis: Other: travel expenses, Research Funding; Shire: Research Funding; Roche: Consultancy.
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Algeri, Mattia, Daria Pagliara, Valentina Bertaina, Giuseppina Li Pira, Elia Girolami, Giovanna Leone, Antonella Meschini et al. « Outcome of Children with Wiskott-Aldrich Syndrome (WAS) Given TCR Alpha-Beta/CD19 Depleted Hematopoietic Stem Cell Transplantation (HSCT) from an HLA-Haploidentical Relative ». Blood 138, Supplement 1 (5 novembre 2021) : 1775. http://dx.doi.org/10.1182/blood-2021-153456.
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Abstract Background: WAS is a rare X-linked recessive disorder, characterized by thrombocytopenia with low platelet volume, recurrent infections, eczema, autoimmunity, vasculitis and increased incidence of malignancies. Patients with classical WAS have chronic morbidities, severely impaired quality of life and a decreased life expectancy. Allogeneic HSCT is the only well-established curative treatment, gene therapy being still an experimental approach. Excellent outcome have been reported in patients transplanted at early age from either matched related and unrelated donors, with 5-year overall survival (OS) exceeding 90%. However, experience with HLA-haploidentical HSCT is limited and has been historically associated with inferior results. Haploidentical HSCT after selective depletion of α/β+ T-cells and CD19+ B-cells (TBdepl-haploHSCT) was shown to be safe and effective in children with multiple types of non-malignant disorders (Merli et al, Blood Adv 2021). To further optimize this approach and accelerate the recovery of adaptive immunity, we conducted a phase I/II trial evaluating the safety and efficacy of post-transplant infusion of a titrated number of donor T-cells transduced with the inducible caspase-9 (iC9) suicide gene (BPX-501, or rivogenlecleucel, cells) in children with either malignant or non-malignant disorders (ClinicalTrials.gov identifier: NCT02065869). We report the outcome of a cohort of 12 children affected by WAS who received a TBdepl-haploHSCT at our Center. Patients and methods: Between 2014 and 2021, 12 patients affected by WAS received a TBdepl-haploHSCT from an HLA-partially matched relative at Ospedale Pediatrico Bambino Gesù in Rome, Italy. Patients and transplant characteristics are depicted in Table 1. All patients received a myeloablative conditioning regimen, combining pharmacokinetic-adjusted busulfan with Thiotepa and Fludarabine. Pre-transplant anti-thymocyte globulins (from day -4 to day -2) were given to modulate bi-directional donor/recipient alloreactivity, while rituximab was administered on day -1 to prevent PTLD. No post-transplant pharmacological GvHD prophylaxis was employed. Eight subjects, enrolled in NCT02065869 trial, additionally received post-transplant infusion of BPX-501 cells (dose: 1x10 6 cells/kg) at a median time of 16 days after HSCT (range 12-20). Results: 11 patients achieved primary donor cell engraftment, while one patient experienced secondary graft failure (GF), likely triggered by CMV reactivation. Median time to neutrophil and platelet recovery was 15 (range 8-33) and 10 days (range 9-16), respectively. The patient with secondary GF was successfully re-transplanted with a second TBdepl-haploHSCT from the same donor. Grade I/II skin acute GvHD (aGvHD) occurred in 3 patients, the cumulative incidence of aGvHD being 25.9% (95% CI 0-47.7). None of these 3 patients required activation of iC9 with rimiducid and no cases of chronic GVHD (cGvHD) were observed. No patient died. With a median follow-up of 58 months (range 1 - 78), the 5-year probability of OS and event-free survival is 100% and 90.7% (95% CI 50.8-98.7), respectively. Considering the successful second allograft, the 5-year disease-free survival is 100%. Platelet recovery is reported in Figure 1A. Two months after the allograft, all evaluable subjects had a platelet count above 50.000/microL (median 202.000, range 53.000-353.000). All subjects but one have full donor chimerism at last follow-up, the remaining patient having stable mixed chimerism (40% of donor cells) without any WAS manifestation. All subjects with a follow-up of at least 6 months are independent of immunoglobulin replacement. Details on reconstitution of lymphocytes subsets are reported in Figure 1B,C,D. Conclusions: TBdepl-haploHSCT after Bu-based conditioning regimen is an highly-effective curative option for children with WAS, being characterized by high-engraftment rate with fast recovery of both neutrophils and platelets, low incidence of aGvHD and no occurrence of cGVHD. Given the prognostic impact of age in determining HSCT outcome in WAS and prompt availability of haploidentical family donors, our data suggest that this approach should be offered without delay to those patients with WAS who lack a matched donor. Infusion of BPX-501 cells contribute to accelerate the recovery of adaptive T-cell immunity, further increasing the safety of the procedure. Figure 1 Figure 1. Disclosures Merli: SOBI: Consultancy; JAZZ: Consultancy. Locatelli: Amgen: Honoraria, Membership on an entity's Board of Directors or advisory committees; Bellicum: Consultancy, Membership on an entity's Board of Directors or advisory committees; bluebird bio, Inc.: Consultancy; Novartis: Consultancy, Membership on an entity's Board of Directors or advisory committees; Miltenyl: Honoraria.
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Merli, Pietro, Daria Pagliara, Mattia Algeri, Federica Galaverna, Giuseppina Li Pira, Valentina Bertaina, Mauro Montanari et al. « Long-Term Outcome of Children with Acute Leukemia (AL) Given Alphabeta T and B-Cell Depleted HLA-Haploidentical Hematopoietic Stem Cell Transplantation (TBdepl-haploHSCT) ». Blood 134, Supplement_1 (13 novembre 2019) : 799. http://dx.doi.org/10.1182/blood-2019-128416.
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Background: TBdepl-haploHSCT is a suitable option for children with AL in need of an allograft, lacking an HLA-compatible donor. We previously published promising results in a cohort of 80 children with AL, given this type of allograft (Locatelli et al., Blood 2017), demonstrating a low incidence of acute and chronic graft-versus-host disease (GvHD) and low non-relapse mortality (NRM), translating into a final outcome comparable to that of patients transplanted from an HLA-compatible donor. We present the long-term follow-up analysis of this study (NCT01810120), now including 134 patients, with a minimum observation time of 100 days after the allograft. Patients and methods: Between October 2010 and April 2019, 134 children with AL in morphological complete remission (CR) received TBdepl-haploHSCT from an HLA-partially matched relative (a parent in 97% of cases) at Ospedale Pediatrico Bambino Gesù in Rome, Italy. All patients were prepared to the allograft using a fully-myeloablative conditioning regimen including a combination of cytotoxic drugs and/or total body irradiation (TBI). Anti-T-lymphocyte globulin (ATLG) was used before transplantation (12 mg/kg total dose, from days -5 to day -3) to modulate bi-directional donor/recipient alloreactivity. Rituximab (200 mg/sqm) was administered on day -1 to prevent post-transplantation EBV-induced lymphoproliferative disorders (PTLD). No patient received any post-transplant GvHD prophylaxis. Results: Characteristics of patients enrolled in the study are shown in Table 1. Median follow-up of surviving patients is 60 months (range: 3 months - 8.7 years). Only 3 patients did not achieve engraftment (all affected by acute myeloid leukemia and who did not receive TBI during conditioning regimen); median time to neutrophil and platelet recovery was 13 (range 9-22) and 11 (range 8-23) days, respectively. Cumulative incidence of grade II-III acute GvHD was 16.5% (95% CI 9.9-22.6). One patient developed gut GvHD, while for all other patients skin was the sole organ involved; no case of grade IV GvHD was recorded. Eight out of the 123 patients at risk developed chronic GvHD, in all cases of limited severity, the cumulative incidence of this complication being 7.6% (95% CI 2.3-12.6). Six patients died for transplant-related complications (2 because of idiopathic pneumonitis and 1 each of disseminated adenovirus infection, cardiac insufficiency, combined CMV/rhinovirus pneumonia and sepsis from Pseudomonas aeruginosa), the 5-year cumulative incidence of NRM being 4.5% (95% CI, 1.8-9.0). Since 25 patients relapsed at a median time of 173 days (range 59-1012) after HSCT, the 5-year cumulative incidence of relapse is 21.1% (95% CI, 14.2-29.1). The 5-year probability of overall and leukemia-free survival (LFS) were 74.6 (95% CI 65.1 -71.9) and 74.4% (95% CI 65.4-81.4) (Figure 1A), respectively. Use of TBI during the preparative regimen, age at transplant above the median value (Figure 1B) and disease status at transplantation (CR1 and CR2, Figure 1C) were associated with better patient's outcome, because of a reduced incidence of relapse (Figure 1D for TBI). All these 3 factors remained statistically significant in multivariable analysis for LFS: hazard ratio (HR) for TBI was 0.16 (95% CI, 0.06- 0.37, p<0.001, HR for age at HSCT was 0.27 (95% CI, 0.11-0.65, p=0.003), while that for disease status was 0.49 (95% CI, 0.26-0.91, p=0.02), respectively. The 5-year GvHD/relapse-free survival was 69.9% (95% CI 60.8-77.3). The median CD3+ cell count on day +30, +90, +180 and +360 were 187, 215, 660 and 1260/mcl, respectively. Conclusions: These data confirm in a larger population and with a longer follow-up that TBdepl-haploHSCT is a safe and effective transplant option, being associated with a low risk of both NRM and acute/chronic GvHD, resulting into a 5-year LFS comparable or even better with that reported in studies using either an HLA-identical sibling or an unrelated volunteer as donor. In particular, the low incidence of chronic GvHD preserves a good quality of life in patients with long life-expectancy. Leukemia recurrence represents the main cause of treatment failure and strategies based either on the use of a titrated number of donor T cells transduced with a safety switch or ex-vivo depleted of the alloreactive component could further improve patient's outcome. Figure 1 Disclosures Merli: Novartis: Honoraria; Sobi: Consultancy; Amgen: Honoraria; Bellicum: Consultancy. Algeri:Miltenyi: Honoraria; Atara Biotherapeutics: Consultancy, Honoraria; Bluebird bio: Consultancy, Honoraria. Locatelli:Amgen: Honoraria, Membership on an entity's Board of Directors or advisory committees; Bellicum: Consultancy, Membership on an entity's Board of Directors or advisory committees; bluebird bio: Consultancy; Miltenyi: Honoraria; Novartis: Consultancy, Membership on an entity's Board of Directors or advisory committees.
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Merli, Pietro, Daria Pagliara, Federica Galaverna, Giuseppina Li Pira, Valentina Bertaina, Stefania Gaspari, Emilia Boccieri et al. « Outcome of Children with Different Non-Malignant Disorders Given Alphabeta T and B-Cell Depleted HLA-Haploidentical Hematopoietic Stem Cell Transplantation (TBdepl-haploHSCT) ». Blood 136, Supplement 1 (5 novembre 2020) : 2–4. http://dx.doi.org/10.1182/blood-2020-142703.
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Background: allogeneic HSCT is the only potentially curative treatment for many non-malignant diseases (NMD), either inherited or acquired. However, many patients lack an HLA-matched donor (familiar (MFD) or unrelated (MUD)) and the outcome of children transplanted from an HLA-haploidentical relative (haplo) was historically inferior to that of transplants from a MFD or a MUD. We previously published promising results in a cohort of 23 children with NMD given this type of allograft (Bertaina et al., Blood 2014), demonstrating a low transplant-related mortality (TRM) and high cure rates. Here, we report the outcome of a large cohort of children affected by NMD who received a TBdepl-haploHSCT at our Center (NCT01810120). Patients and methods: Between February 2011 and June 2020, 80 consecutive patients affected by NMD received TBdepl-haploHSCT from an HLA-partially matched relative at Ospedale Pediatrico Bambino Gesù in Rome, Italy. Patients had many different disorders (see Table for details on patient- and transplant-related characteristics). Median time from diagnosis to transplant for the whole cohort was 12 months (range 1-177), while it was 2.5 months (range 1.3-11.2) for SCID patients. All patients, including children with SCID, received a conditioning regimen, which varied according to the original disease. Pre-transplant anti-thymocyte globulins (from day -4 to day -2) were given to modulate bi-directional donor/recipient alloreactivity, while rituximab (on day -1) was administered to prevent PTLD. Moreover, no post-transplant pharmacological GvHD prophylaxis was given. Results: fifty-eight patients (72.5%) achieved primary donor cell engraftment, while 3 patients experienced secondary graft failure (GF); the cumulative incidence of either primary or secondary GF was 27.8% (95% CI 17.2-37.0). Median time to neutrophil and platelet recovery was 13.5 (range 9-33) and 10 days (range 7-51), respectively. As expected, GF occurred more frequently in children with disorders known to be associated with an increased GF risk (i.e., HLH, thalassemia, SAA or osteopetrosis) (see also Figure 1A). Three children (4%) experiencing GF died because of infectious complications before retransplant. Sixteen of the 22 patients with either primary or secondary GF were successfully retransplanted (2 with a mismatched unrelated cord blood unit, the other having received a second TBdepl-haploHSCT from either the same donor or the other parent). Since 3 other patients died [all because of infectious complications, 2 due to disseminated adenovirus infection and 1 to CMV pneumonia)], TRM is 7.8% (95% CI 1.6-13.7). Eighteen patients experienced acute GVHD of any grade, the cumulative incidence of this complication being 22% (95% CI 13.5-31.8); 10/18 patients developed grade II acute GVHD (no patient developed grade III or IV aGVHD), this resulting into a cumulative incidence of 12.9% (95% CI 6.6-21.4). Only one patient at risk developed mild chronic GVHD. Twenty-two and 7 patients developed clinically-relevant (i.e., with a viral load > 1000 copies/ml and/or requiring specific antiviral-treatment) CMV and adenovirus infection, respectively, at a median time of 4 (range 0-16) and 1 (range 1-4) weeks from HSCT. Time averaged area under the curve (i.e., viral burden under the curve/weeks at risk for infection) for CMV and ADV are reported in Figure 1B. With a median follow-up of 36 months (range 2 - 110), the 5-year probability of overall survival and event-free survival for the entire cohort of patients is 92.1% (95% CI 83.3-96.4) (Figure 1C) and 68.1% (95% CI 56.4-77.2), respectively. Considering the 16/22 given a successful 2nd allograft, the 5-year disease-free survival is 88.4% (95% CI 78.9-93.8). Details on reconstitution of CD3+, CD4+ and CD8+ lymphocytes are reported in Figure 1D. Conclusions: TBdepl-haploHSCT is an effective option for children with different NMD. GF (either primary or secondary) is a challenging problem in a sub-group of patients at risk (i.e., those with HLH, thalassemia, SAA or osteopetrosis): thus, new strategies to overcome this problem are desirable. However, a second transplant is able to rescue most of these patients. Prompt availability of this type of transplant, limiting infectious risk, low incidence of both acute and chronic GvHD preserving a good quality of life in patients makes this strategy an attractive choice in patients with NMD. Figure 1 Disclosures Merli: Bellicum Pharmaceuticals: Consultancy, Membership on an entity's Board of Directors or advisory committees; SOBI: Consultancy, Membership on an entity's Board of Directors or advisory committees; Jazz: Honoraria; Sanofi-Genzyme: Honoraria; Atara Therapeutics: Honoraria. Algeri:BlueBird Bio: Membership on an entity's Board of Directors or advisory committees; Atara Therapeutics: Membership on an entity's Board of Directors or advisory committees. Locatelli:Jazz Pharmaceeutical: Speakers Bureau; Medac: Speakers Bureau; Miltenyi: Speakers Bureau; Bellicum Pharmaceutical: Membership on an entity's Board of Directors or advisory committees; Novartis: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Amgen: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau.
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Del Bufalo, Francesca, Concetta Quintarelli, Biagio De Angelis, Ignazio Caruana, Matilde Sinibaldi, Luciana Vinti, Pietro Merli et al. « Academic, Phase I/II Trial on T Cells Expressing a Second Generation, CD19-Specific Chimeric Antigen Receptor (CAR) and Inducible Caspase 9 Safety Switch for the Treatment of B-Cell Precursor Acute Lymphoblastic Leukemia (BCP-ALL) and B-Cell Non-Hodgkin Lymphoma (B-NHL) in Children ». Blood 134, Supplement_1 (13 novembre 2019) : 1341. http://dx.doi.org/10.1182/blood-2019-129821.
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Survival rates of children with relapsed/refractory (r/r) BCP-ALL remain unsatisfactory and little progress has been made in the past 2 decades. Similarly, relapse of childhood B-NHL is usually associated with an aggressive disease and poor outcomes. Targeted immunotherapy with T-cells genetically modified to express a CD19-directed CAR showed an unprecedented antitumor efficacy, leading to the recent FDA and EMA approval of two CD19-CAR products for treatment of BCP-ALL and B-NHL. Relevant toxicities have, however, been reported, mainly related to the development of severe Cytokine Release Syndrome (CRS) and/or of neurotoxicity. At Ospedale Pediatrico Bambino Gesù (OPBG) in Rome, we developed a clinical-grade, 2nd generation, CD19-specific CAR construct, including 4.1bb as costimulatory domain and the inducible caspase-9 safety switch (iC9-CD19-CAR), vehiculated by a retroviral vector, to conduct an academic, phase I/II clinical trial in patients (age 1-25 yrs) affected by BCP-ALL or B-NHL. We now report on the results of the phase I and of the first 8 patients treated in the phase II portion of the study, in terms of feasibility, toxicity, maximum tolerated/recommended dose (MTD/RD) and data on response rate and biological correlates. The phase I, dose-escalation portion of the study included 3 dose levels (DL), namely: DL1, 0.5×106; DL2, 1.5×106; DL3, 3.0×106 CAR+ T cells per kg of recipient body weight. In the phase II portion, patients were treated at the RD identified in the phase I, namely 3.0×106 CAR+cells/kg. All patients received a lymphodepleting regimen consisting of fludarabine and cyclophosphamide for 3 days and iC9-CD19-CAR T cells were subsequently administered as single infusion. Patients were monitored for toxicity, expansion and persistence of iC9-CD19-CAR T cells. Seventeen children were enrolled into the trial and received iC9-CD19-CAR T cells between January 2018 and June 2019. Data were analyzed as of July 20, 2019. The characteristics of the patients are detailed in table 1. The designed dose concentration was successfully produced for all the enrolled patients and we did not observe any production failure. The median transduction rate in the drug product was 54% (range 21-73), while the median vector copy number was 3.8 (range 2.8-6.2). During the phase I portion of the study, no dose limiting toxicities (DLTs) have been recorded, defining the MTD as 3.0×106 CAR+ T cells per kg of recipient body weight. The treatment was overall tolerated and all the toxicities were reversible, the most severe being grade 3-4 neutropenia, thrombocytopenia and/or anemia, occurring in 16/17 (94.1%) patients; in 13/16 patients (81.2%) the hematological toxicity developed before the infusion and persisted after the administration of CAR T cells. Cytokine release syndrome (CRS) occurred in 10/17 patients (58.8%) and was overall moderate, reaching grade 3 (Lee criteria) in one patient only. Notably, none of the patients developed neurotoxicity and no activation of the safety switch was required. All patients were assessed for response at 4 weeks from iC9-CD19-CAR T cell infusion and 13/15 (86.7%) patients with ALL achieved complete remission (CR) with negativity of minimal residual disease (MRD), including 2/3 patients receiving the DL1, 9 patients who had failed a previous allogeneic haematopoietic stem-cell transplantation (HSCT) and 6 patients that had previously received blinatumomab, as CD19-directed immunotherapy. The iC9-CD19-CAR T cells expanded in vivo and were detectable by both flow-cytometry and molecular biology in the blood (Fig.1), bone marrow and cerebrospinal fluid of the responders. One CD19-negative relapse 3 months after infusion was recorded, while 3 additional patients relapsed with CD19+ leukemia blasts. Four patients received HSCT while in CR with MRD negativity because of regrowth of normal CD19+ B cells. The 18-month probability of overall survival for the BCP-ALL cohort is 72.2% (Fig.2). One of the 2 B-NHL patients showed a partial response. Our data indicate that iC9-CD19-CAR T cell in an academic setting is feasible, safe and extremely effective in treating highly resistant/relapsed BCP-ALL. In our trial, no major or life-threatening toxicities were observed and, despite the moderate CRS recorded, high rates of CR were achieved, suggesting that the combination of a retroviral platform and 4.1bb as costimulation is able to mediate a potent antitumor effect Disclosures Merli: Amgen: Honoraria; Novartis: Honoraria; Sobi: Consultancy; Bellicum: Consultancy. Algeri:Bluebird bio: Consultancy, Honoraria; Atara Biotherapeutics: Consultancy, Honoraria; Miltenyi: Honoraria. Locatelli:Amgen: Honoraria, Membership on an entity's Board of Directors or advisory committees; Bellicum: Consultancy, Membership on an entity's Board of Directors or advisory committees; bluebird bio: Consultancy; Miltenyi: Honoraria; Novartis: Consultancy, Membership on an entity's Board of Directors or advisory committees.
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Bernaschi, P., A. Portanova, B. Lucignano, S. Chiavelli et D. Menichella. « BATTERIEMIE IN ETÀ PEDIATRICA E NEONATALE : INCIDENZA DELLE INFEZIONI NOSOCOMIALI IN UN OSPEDALE PEDIATRICO NEL BIENNIO 2002-2003. » Microbiologia Medica 19, no 2 (30 juin 2004). http://dx.doi.org/10.4081/mm.2004.3797.
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Portanova, A., P. Bernaschi, M. Argentieri, D. Menichella et T. Langiano. « SORVEGLIANZA DELLE INFEZIONI OSPEDALIERE DA ROTAVIRUS IN UN OSPEDALE PEDIATRICO ». Microbiologia Medica 19, no 2 (30 juin 2004). http://dx.doi.org/10.4081/mm.2004.3976.
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« La prospettiva gruppoanalitica in oncologia pediatrica ». GRUPPI, no 1 (mars 2013) : 97–104. http://dx.doi.org/10.3280/gru2012-001008.
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Quando l'istituzione ospedale si occupa di malattie oncologiche dell'etŕ pediatrica, il tema della sofferenza del corpo e dell'eventualitŕ della morte rischiano di saturare tutti gli spazi mentali del paziente, della sua famiglia e degli operatori. Il linguaggio diventa denso di "fatti concreti" mentre l'elemento emozionale dei soggetti coinvolti, per l'alta densitŕ con cui emerge, assume l'aspetto della "turbolenza". L'istituzione che coltiva la visione gruppale del care, nel conferire alla malattia una lettura complessiva di corpo e mente, offre ai curanti la possibilitŕ di riflettere sui temi critici del lavorare con i pazienti che muoiono, secondo l'assetto della condivisione consapevole.
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Vittori, Alessandro, Giuliano Marchetti, Elisa Francia, Ilaria Mascilini et Sergio G. Picardo. « Organization of a pediatric acupuncture clinic for pain and enuresis during Covid-19 pandemic in a third level Italian Pediatric Hospital : experience of Ospedale Pediatrico Bambino Gesù ». Minerva Pediatrics, juin 2021. http://dx.doi.org/10.23736/s2724-5276.20.06050-8.
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Colagrossi, Luna, Valentino Costabile, Rossana Scutari, Valeria Cento, Luana Coltella, Antonino Reale, Martina Scilipoti et al. « Performance evaluation of a new on-demand molecular test for the rapid identification of severe acute respiratory syndrome coronavirus 2 in pediatric and adult patients ». Frontiers in Microbiology 13 (3 novembre 2022). http://dx.doi.org/10.3389/fmicb.2022.999783.
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The rapid spread of severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) infection has increased the need to identify additional rapid diagnostic tests for an accurate and early diagnosis of infection. Here, we evaluated the diagnostic performance of the cartridge-based reverse transcription polymerase chain reaction (RT-PCR) test STANDARD M10 SARS-CoV-2 (SD Biosensor Inc., Suwon, South Korea), targeting the ORF1ab and E gene of SARS-CoV-2, and which can process up to eight samples in parallel in 60 min. From January 2022 to March 2022, STANDARD™ M10 assay performance was compared with Xpert® Xpress SARS-CoV-2 (Cepheid, Sunnyvale CA) on 616 nasopharyngeal swabs from consecutive pediatric (N = 533) and adult (N = 83) patients presenting at the “Istituto di Ricovero e Cura a Carattere Scientifico” (IRCCS) Ospedale Pediatrico Bambino Gesù, Roma. The overall performance of STANDARD M10 SARS-CoV-2 was remarkably and consistently comparable to the Xpert® Xpress SARS-CoV-2 with an overall agreement of 98% (604/616 concordant results), and negligible differences in time-to-result (60 min vs. 50 min, respectively). When the Xpert® Xpress SARS-CoV-2 results were considered as the reference, STANDARD™ M10 SARS-CoV-2 had 96.5% sensitivity and 98.4% specificity. STANDARD M10 SARS-CoV-2 can thus be safely included in diagnostic pathways because it rapidly and accurately identifies SARS-CoV-2 present in nasopharyngeal swabs.
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Maiorana, Arianna, Antonella Sabia, Tiziana Corsetti et Carlo Dionisi-Vici. « Safety of vaccines administration in hereditary fructose intolerance ». Orphanet Journal of Rare Diseases 15, no 1 (1 octobre 2020). http://dx.doi.org/10.1186/s13023-020-01552-z.
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Abstract Patients with hereditary fructose intolerance need to follow a life-long fructose dietary and drug restriction to prevent symptoms of intoxication. Concerns about vaccines administration have been manifested overtime, for the risk of a life-threatening acute intoxication. For this reason, at Ospedale Pediatrico Bambino Gesù we performed a deepen research from open sources, datasheets and Pharmaceutical Companies informations from the most common Italian and European vaccines, which are carried out in infancy and childhood. As a safe threshold of 2.4 mg/kg/dose was recently established for oral and parenteral (other than i.v.) route, the manuscript clarifies the safe administration of majority of vaccines in patients with hereditary fructose intolerance.
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Veiceschi, Pierlorenzo, Fabio Pozzi, Francesco Restelli, Tommaso Alfiero, Paolo Castelnuovo et Davide Locatelli. « Endoscopic Endonasal Odontoidectomy Preserving Atlantoaxial Stability : a Pediatric Case ». Journal of Neurological Surgery Part B : Skull Base, 4 mars 2020. http://dx.doi.org/10.1055/s-0039-3402797.
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Abstract Objectives We illustrate endoscopic endonasal odontoidectomy for the Chiari-I malformation respecting craniovertebral junction (CVJ) stability. Design Case report of a 12-year-old girl affected by the Chiari-I malformation. Magnetic resonance imaging (MRI) showed tonsillar herniation, basilar invagination, and dental retroversion, causing angulation and compression of the bulbomedullary junction. Patient underwent endoscopic third ventriculostomy (ETV) with reduction of ventricular size and resolution of gait disturbances, but she complained the Valsalva-induced headaches, hiccup, and dysesthesias in the lower limbs. Endoscopic endonasal odontoidectomy was chosen to decompress the cervicomedullary junction. Setting The research was conducted at University Hospital “Ospedale di Circolo,” Department of Neurosurgery at Varese in Italy. Participants Patients were from neurosurgical and ENT (ear, nose, and throat) skull base team. Main Outcome Measures A bilateral paraseptal approach was performed, using a four-hand technique. After resection of posterior edge of the nasal septum, the choana is entered and a rhinopharynx muscle–mucosal flap is dissected subperiosteal and transposed in oral cavity. The CVJ is exposed and, using neuronavigation and neuromonitoring, odontoidectomy is fulfilled until dura is reached, preserving the anterior arch of C1. Reconstruction is obtained suturing the flap previously harvested. Results Postoperative course was unremarkable and the patient experienced improvement of symptoms. Postoperative MRI documented the appearance of tight cerebrospinal fluid (CSF) film anterior to bulbomedullary junction and in retrotonsillar spaces, opening of the bulbomedullary angle, and slight tonsils reduction. No CVJ instability was occurred with any need of posterior fixation. Conclusion Endoscopic endonasal odontoidectomy is a feasible approach for CVJ malformation. In this case, bulbar decompression was achieved preserving CVJ stability and avoiding posterior fixation.The link to the video can be found at: https://youtu.be/VIobocHfCuc.
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Milani, Gregorio P., Paola Marchisio, Alessia Rocchi, Giuseppe Bertolozzi, Ludovico Furlan, Adriano La Vecchia, Carlo Agostoni et Giorgio Costantino. « Frequency of asymptomatic carriers of SARS-CoV-2 among children and adults after school reopening ». Italian Journal of Pediatrics 47, no 1 (12 mars 2021). http://dx.doi.org/10.1186/s13052-021-01016-5.
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Abstract Background Children often develop an asymptomatic form of Severe Acute Respiratory Syndrome Coronavirus 2 (SARS-CoV-2), but it is debated if children are at higher risk than adults to be asymptomatic carriers of SARS-CoV-2, especially during the school reopening. The main aim of this study was to investigate the frequency of SARS-CoV-2 asymptomatic carriers in children and adults during the reopening of the schools in Milan, Italy. Methods We conducted a cross-sectional study at the pediatric and adult Emergency Department (ED) of the Ca' Granda Ospedale Maggiore Policlinico (Milan) between October 1 and 31, 2020, i.e. 3 weeks after the reopening of schools. Patients admitted to the ED short stay observation and without any sign or symptom consistent with a SARS-CoV-2 were eligible. These patients underwent a nasopharyngeal swab specimen for the detection of SARS-CoV-2. The odds ratio and its 95% confidence interval (CI) was calculated to assess the risk of asymptomatically carrying the SARS-CoV-2 infection in children and adults. Results A total of 69 (27 females, median age 8.7 years) children and 251 (107 females, median age 71 years) adults were enrolled. Pediatric and adult subjects tested positive for SARS-CoV-2 with a similar frequency (1/69 [1.4%] vs 4/251 [1.6%]). Children had an odds ratio to be a carrier of 0.91 (CI 0.02– 9.38) compared to adults. Conclusions The frequency of asymptomatic SARS-CoV-2 carriers was similar among children and adults. Considering the emerging diffusion of new SARS-CoV-2 variants, the asymptomatic spread of SARS-CoV-2 infection among children and adults should be monitored.
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Milani, Gregorio Paolo, Valentina Bollati, Luca Ruggiero, Samantha Bosis, Raffaella Maria Pinzani, Giovanna Lunghi, Federica Rota et al. « Bronchiolitis and SARS-CoV-2 ». Archives of Disease in Childhood, 11 mars 2021, archdischild—2020–321108. http://dx.doi.org/10.1136/archdischild-2020-321108.
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BackgroundIt has been speculated that the SARS-CoV-2 was already widespread in western countries before February 2020.MethodsWe gauged this hypothesis by analysing the nasal swab of infants with either bronchiolitis or a non-infectious disease admitted to the Ospedale Maggiore, Milan (one of the first epicentres of SARS-CoV-2 outbreak in Europe) from November 2019.ResultsThe SARS-CoV-2 RNA was never detected in 218 infants with bronchiolitis (95 females, median age 4.9 months) and 49 infants (22 females, median age 5.6 months) with a non-infectious disease between November 2019 and February 2020. On the contrary, two infants hospitalised for bronchiolitis between March and April 2020 tested positive for SARS-CoV-2.ConclusionsThis study does not support the hypothesis that SARS-CoV-2 was already circulating among infants before the official outbreak of SARS-CoV-2 infection. However, it shows for the first time that SARS-CoV-2 might cause bronchiolitis requiring hospitalisation.
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Brito, Maysa Vasconcelos de, Ana Maria Braga da Silva França, Amanda Alves Fecury, Euzébio de Oliveira, Carla Viana Dendasck et Cláudio Alberto Gellis de Mattos Dias. « Profilo epidemiologico della malaria grave nei neonati e negli adolescenti trattati nel 2016 in un ospedale di riferimento nello Stato di Amapá, Brasile ». Revista Científica Multidisciplinar Núcleo do Conhecimento, 22 juin 2020, 05–23. http://dx.doi.org/10.32749/nucleodoconhecimento.com.br/salute/della-malaria-grave.
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Il presente studio ha preso in considerazione non solo le infezioni da Plasmodium falciparum che è comunemente raccomandato come la causa principale della malaria grave, ma le complicazioni legate al Plasmodium vivax sono state accuratamente descritte. Lo scopo di questo articolo era quello di identificare il profilo epidemiologico, clinico e di laboratorio della malaria grave nei pazienti trattati presso l’ospedale pediatrico e adolescenziale di Macapá – Amapá. È stato effettuato uno studio epidemiologico e descrittivo del carattere retrospettivo. I dati sono stati ottenuti attraverso la consultazione nelle cartelle cliniche dei pazienti per l’anno 2016. La malaria grave è stata considerata 47 casi. Predominavano pazienti maschi con il 63,8% e di età compresa tra i 5 anni e il 59,6% dei casi. Per quanto riguarda la specie, le infezioni da P. vivax si sono distinte con maggiore frequenza con il 72% rispetto a P. falciparum con il 28% dei casi. I mesi con il maggior numero di ricoveri sono stati settembre e novembre, entrambi con il 17%. Le principali manifestazioni cliniche riscontrate sono state: febbre, pallore e tosse. Le condizioni che indicano la gravità che si sono verificate maggiormente: vomito 87%, ittero 23%, dispnea ed età < 1 anno, entrambe con il 21%. Gli esami ematologici hanno mostrato che il 91% dei pazienti aveva globuli rossi al di sotto dei valori di riferimento e il 100% dei casi aveva emoglobina ed ematocrito bassi; trombocitopenia è stata evidenziata nel 72% dei casi. Risultati alterati di dosaggi biochimici di maggiore importanza clinica sono stati: urea, transaminasi, glicimea e proteina C-reattiva. L’identificazione di dati epidemiologici, clinici e di laboratorio sulla malaria grave contribuisce alla diagnosi precoce e al trattamento adeguato della malattia.
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Macchiaiolo, Marina, Filippo M. Panfili, Davide Vecchio, Michaela V. Gonfiantini, Fabiana Cortellessa, Cristina Caciolo, Marcella Zollino et al. « A deep phenotyping experience : up to date in management and diagnosis of Malan syndrome in a single center surveillance report ». Orphanet Journal of Rare Diseases 17, no 1 (18 juin 2022). http://dx.doi.org/10.1186/s13023-022-02384-9.
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Abstract Background Malan syndrome (MALNS) is a recently described ultrarare syndrome lacking guidelines for diagnosis, management and monitoring of evolutive complications. Less than 90 patients are reported in the literature and limited clinical information are available to assure a proper health surveillance. Results A multidisciplinary team with high expertise in MALNS has been launched at the “Ospedale Pediatrico Bambino Gesù”, Rome, Italy. Sixteen Italian MALNS individuals with molecular confirmed clinical diagnosis of MALNS were enrolled in the program. For all patients, 1-year surveillance in a dedicated outpatient Clinic was attained. The expert panel group enrolled 16 patients and performed a deep phenotyping analysis directed to clinically profiling the disorder and performing critical revision of previously reported individuals. Some evolutive complications were also assessed. Previously unappreciated features (e.g., high risk of bone fractures in childhood, neurological/neurovegetative symptoms, noise sensitivity and Chiari malformation type 1) requiring active surveillance were identified. A second case of neoplasm was recorded. No major cardiovascular anomalies were noticed. An accurate clinical description of 9 new MALNS cases was provided. Conclusions Deep phenotyping has provided a more accurate characterization of the main clinical features of MALNS and allows broadening the spectrum of disease. A minimal dataset of clinical evaluations and follow-up timeline has been proposed for proper management of patients affected by this ultrarare disorder.
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Cavallin, Francesco, Serena Calgaro, Martina Borellini, Margherita Magnani, Greta Beltramini, Amir Hussein Abubacar Seni, Bonifacio Rodriguez Cebola, Ana Nicolau Tambo, Giovanni Putoto et Daniele Trevisanuto. « Midwives' Evaluation of a Neonatal Resuscitation in High- and Low-Resource Settings ». Frontiers in Pediatrics 9 (9 mars 2021). http://dx.doi.org/10.3389/fped.2021.644308.
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Aim: To assess midwives' evaluation of a real-life neonatal resuscitation and their opinion on importance of resuscitation interventions.Methods: Multicenter, multi-country study.Setting: Beira Central Hospital (Mozambique) and Azienda Ospedale-Università di Padova (Italy).Subjects: Sixteen Mozambican midwives and 18 Italian midwives.Interventions: Midwives' assessment was evaluated by using a predefined score, which graded each resuscitation intervention (0–2 points) and summed to a total score for each step (initial steps, bag-mask ventilation, and chest compressions). All scores were compared with referral scores given by two expert neonatologists.Results: Both Mozambican and Italian midwives overestimated their performance regarding of initial steps taken during resuscitation, chest compressions, high-oxygen concentrations (p < 0.01), and underestimated the importance of stimulation (p < 0.05). Mozambicans overestimated suctioning (p < 0.001). Participants agreed with experts about the importance of equipment preparation, using a warmer, drying the newborn, removing wet linen and heart rate assessment.Conclusion: Mozambican and Italian midwives overestimated the performance of a real-life neonatal resuscitation, with heterogeneous evaluation of the importance of several aspects of neonatal resuscitation. These findings may be useful for identifying educational goals.
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Tiozzo, Emanuela, Serena Fondi, Valentina Biagioli, Elisa Piccinelli, Francesca Alibrandi, Orsola Gawronski, Immacolata Dall’Oglio et al. « Electronic Assessment and Tracking of Pain at Home : A Prospective Study in Children With Hematologic or Solid Tumors ». Journal of Pediatric Oncology Nursing, 3 décembre 2020, 104345422097544. http://dx.doi.org/10.1177/1043454220975443.
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This study aimed to evaluate the intensity and characteristics of pain, which was assessed at home by children with cancer or their parents using an app for mobile devices, for a 1-month poststudy enrollment. The participants of this observational prospective study were outpatients at the hematology/oncology department of an academic hospital in Italy, aged between 0 and 21 years, and receiving hospital-at-home care. Patients or their parents were asked to assess pain levels for one month at home using the Faces, Legs, Arms, Cry, and Consolability (FLACC) scale for patients aged <4 years and the Ospedale Pediatrico Bambino Gesù (OPBG) tool for patients aged 4 to 21 years. At study conclusion, parents were asked to complete a survey about their satisfaction with the app and to rate their child’s health in general, pain intensity, and frequency in the past 4 weeks. Of the 124 participants using the app, 94 (75.8%) reported pain (≥1) at least once during the 1 month. Of these, 53 (56.4%) at most reported mild pain, 33 (35.1%) moderate pain, and eight (8.5%) severe pain. The abdomen was identified as the most frequent (56.3%) pain site by patients aged 4 to 21 years. The child’s maximum pain intensity during the study period was negatively correlated with the parent’s report of the child’s health during the past month ( r = −.29, p < .01). Most of the participants were satisfied with the app and the information received about app usage. In conclusion, the app facilitated pain assessment and tracking in patients receiving hospital-at-home care.
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Bottino, Ilaria, Maria F. Patria, Gregorio P. Milani, Carlo Agostoni, Paola Marchisio, Mara Lelii, Marco Alberzoni et al. « Can Asymptomatic or Non-Severe SARS-CoV-2 Infection Cause Medium-Term Pulmonary Sequelae in Children ? » Frontiers in Pediatrics 9 (13 mai 2021). http://dx.doi.org/10.3389/fped.2021.621019.
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Pulmonary complications in adults who recovered from severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) have been reported even in minimally symptomatic patients. In this study, lung ultrasound (LUS) findings and pulmonary function of children who recovered from an asymptomatic or mildly symptomatic SARS-CoV-2 infection were evaluated. We prospectively followed up for at least 30 days patients younger than 18 years who recovered from SARS-CoV-2 infection at the Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (Italy). All enrolled patients underwent LUS. Airway resistance measured by the interrupter technique test was assessed in subjects aged 4–6 years, whereas forced spirometry and measurement of diffusing capacity of the lungs for carbon monoxide were performed in subjects older than 6 years. To evaluate a possible correlation between pulmonary alterations and immune response to SARS-CoV-2, two semiquantitative enzyme immune assays were used. We enrolled 16 out of 23 eligible children. The median age of enrolled subjects was 7.5 (0.5–10.5) years, with a male to female ratio of 1.7. No subject presented any abnormality on LUS, airway resistance test, forced spirometry, and diffusing capacity of the lungs for carbon monoxide. On the other hand, all subjects presented Ig G against SARS-CoV-2. In contrast in adults, we did not detect any pulmonary complications in our cohort. These preliminary observations suggest that children with an asymptomatic or mildly symptomatic SARS-CoV-2 infection might be less prone to develop pulmonary complications than adults.
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Ullmann, Nicola, Antonio Di Marco, Fabiana Columbu, Valentina Negro, Maria Beatrice Chiarini Testa, Valentina Panetta, Salvatore Tripodi et al. « A Novel, Portable MESH Nebulizer—An Alternative to Metered Dose Inhaler : Efficacy and Usability in Preschool Wheezers ». Frontiers in Pediatrics 8 (10 décembre 2020). http://dx.doi.org/10.3389/fped.2020.598690.
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Introduction and Objectives: Wheezing episodes are the first causes of doctor's consultation in preschool age. Treatment is usually administered with a metered dose inhaler (MDI) spacer. At variance, many parents and doctors prefer to use a compressor nebulizer, which cannot be easily carried. The study is aimed at testing whether a pocket mesh nebulizer has similar efficacy and acceptability than a standard MDI device.Materials and Methods: The IPAC study was a randomized, controlled, non-inferiority trial (number: 1616/2018, Ospedale Pediatrico Bambino Gesu'—IRCCS). The study had two arms: cases, using MicroAIR U100, and controls, using MDI+spacer device. Both devices were adopted for long-term treatment and for exacerbations. Follow-up was organized with clinical visits and a daily e-diary connected to an application for mobile phone.Results: One hundred patients were enrolled. The frequency of asthmatic symptoms showed a non-inferiority for MicroAIR U100 group vs. MDI. Accordingly, no significant difference was found in the average % of days with cough, wheezing, breathlessness after exercise, days lost at school, and not-programmed visits. Considering only patients with >1 day with symptoms, no significant sdifferences were found in the number of exacerbations nor in the cumulative days with symptoms. The acceptance and usability of both devices have been favorable. However, the MDI+AeroChamber® device showed better acceptability.Conclusions: Our study shows that MicroAIR U-100, a mesh nebulizer, has similar clinical efficacy but lower acceptance and usability than an MDI plus Aerochamber® in delivering therapy in preschool wheezers. Therefore, MicroAIR U-100 might be a valuable second choice, when the delivery of medication with an MDI plus Aerochamber® is not accepted, or wrongly used by the parents.
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Dessi, A., S. Lais, S. Baratta et M. Menconi. « P91 HEMODYNAMICS NURSES ARE EVOLVING WITH INTERVENTIONAL CARDIOLOGY ». European Heart Journal Supplements 24, Supplement_C (1 mai 2022). http://dx.doi.org/10.1093/eurheartj/suac012.088.
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Abstract Subject Matter Hemodynamics deals with issues concerning ‘the bloodstream and the vascular system’ and the figure of nurses pursuing their activity within those settings is constantly growing and evolving. Nurses must keep on training in procedures, new techniques, different procedural approaches and required equipment. Furthermore, this training has to develop besides the fundamentals, i.e. throughout briefing and debriefing sections. In fact, patient care is complex and mechanistic and therefore documented and transversal in order to satisfy clinical needs of the on–going process. In the hemodynamics ward in Massa’s Ospedale del Cuore of the Fondazione Toscana Gabriele Monasterio, diagnostics and interventions performed on patients are consistently developing. A hemodynamic patient leads, affects and characterises the nurse who must be able of taking care of their adult patients and pediatric ones until their prematurity. Objective Highlighting nursing skills in hemodynamics wards and types of training and care of patients of different age groups during procedural approaches. Methods and Materials Shared approval among medical directors of training courses to select for the training plan; mentorship; periodical evaluation of trainee plans; on–the–job nursing training; training courses chaired by experts; simulation of intra–setting emergency; evaluation of panic ranges to detect and it is necessary to act promptly for. Outcome In consideration of creating a nursing team capable of facing scenarios and procedures which can include every life stage of patients, it is paramount that a nurse follows the whole process. The procedural activities include the realization of a job description for basic and specialised skills and its observation. Outcomes and steps outlined to carry out care–giving performances are overseen each year. Lastly, it is necessary to know patients in their pre– and post–procedural stage up until the outcome of care–giving treatments. Conclusions Hemodynamics nurses are able to face changes and clinical solutions development alongside with a cross–disciplinary team.