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Journal articles on the topic 'Surrogate endpoint, Validation, Leukemia'

Author: Grafiati

Published: 9 March 2023

Last updated: 31 July 2025

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1

Valsecchi, Maria Grazia, Meenakshi Devidas, Ausiliatrice Lucenti, et al. "Evaluation of Minimal Residual Disease As a Surrogate Endpoint for Event Free Survival in Childhood B-Lineage Acute Lymphoblastic Leukemia." Blood 128, no. 22 (2016): 759. http://dx.doi.org/10.1182/blood.v128.22.759.759.

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Abstract Introduction The need to optimize clinical evaluation of new drugs stimulates researchers and regulatory bodies to consider novel endpoints that facilitate assessment of the efficacy of a new drug earlier in time than do traditional endpoints. To be a useful marker of efficacy, an early endpoint must be an accurate surrogate for the true clinical endpoint. Minimal residual disease (MRD) is a strong prognostic factor for Event Free Survival (EFS) in children with newly diagnosed acute lymphoblastic leukemia (ALL) and is used routinely to assess treatment response and stratify treatment

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2

Wang, Lin, Tim Disher, Fei Fei Liu, et al. "Evaluating CR as a surrogate endpoint for PFS in R/R chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL): A meta-analysis of randomized controlled trials (RCT)." Journal of Clinical Oncology 42, no. 16_suppl (2024): 7046. http://dx.doi.org/10.1200/jco.2024.42.16_suppl.7046.

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7046 Background: CLL/SLL is the most common leukemia in adults. Achieving a CR by International Workshop on CLL 2018 criteria indicates complete eradication of CLL/SLL in all disease compartments. Patients with R/R CLL/SLL who achieved CR tend to have delayed disease progression/death compared with patients who did not achieve CR. This is the first report to evaluate CR as a surrogate endpoint for PFS in R/R CLL/SLL using aggregate RCT data. Methods: A systematic literature review (SLR) identified published RCTs in R/R CLL/SLL from inception to 10/2023, reporting nonzero CR rates (CRR) and PFS

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Gonneau, Christele, Marc Brugarolas, Lotje Van Haecht, et al. "Validation and Implementation of Flow Cytometry Based Minimal Residual Disease (MRD) Assay for Chronic Lymphocytic Leukemia (CLL) Clinical Studies." Blood 144, Supplement 1 (2024): 6761. https://doi.org/10.1182/blood-2024-206125.

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Background: With the advances made in novel anti-cancer therapies, Chronic Lymphocytic Leukemia (CLL) patient remission rates have significantly improved over the years. Minimal or measurable residual disease (MRD) is emerging as an independent predictor of progression-free survival (PFS) and overall survival (OS) in several heme indications and has been proposed as a potential surrogate endpoint for long term CLL survival in clinical trials. While next generation sequencing (NGS) methods are emerging for establishing MRD measurements in CLL, flow cytometry remains as an option of choice for m

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Hjelmgren, Jonas, Kristoffer Nilsson, and Gunnar Birgegård. "JAK2 V617F as a Marker for Long-Term Disease Progression and Mortality in Polycythemia Vera and its Role in Economic Modeling." Journal of Health Economics and Outcomes Research 7, no. 1 (2020): 61–70. http://dx.doi.org/10.36469/jheor.2020.13083.

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Background: In order to facilitate sound economic evaluations of novel treatments, health-economic models of polycythemia vera (PV) must combine effects on surrogate endpoints in trials with disease progression (DP) and mortality in long-term cohort data. Objective: We validate an economic model for PV that uses Janus Kinase 2 (JAK2) burden as a surrogate endpoint to predict DP (thrombosis, myelofibrosis, and acute leukemia) and overall survival (OS) based on progression-specific mortality. Methods: Long-term observational studies that include information about baseline JAK2 burden were identi

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Yin, Jun, Geoffrey L. Uy, Betsy Laplant, et al. "Event-Free Survival As a Surrogate Endpoint for Overall Survival in Previously Untreated Acute Myeloid Leukemia: An Individual Patient-Level Analysis of Multiple Randomized Trials (Alliance A151614)." Blood 132, Supplement 1 (2018): 1386. http://dx.doi.org/10.1182/blood-2018-99-113902.

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Abstract Background: Overall survival (OS) remains the definitive primary efficacy endpoint to evaluate previously untreated acute myeloid leukemia (AML) therapies, but it requires prolonged follow-up. An earlier endpoint assessed post-treatment would expedite clinical trial conduct and accelerate patient access to effective new therapies. Our objective was to formally evaluate event-free survival (EFS) as a surrogate endpoint for OS in untreated AML. Methods: Individual patient data were analyzed from 2,475 patients (pts) from 4 multicenter, randomized controlled phase III trials of active tr

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Schilhabel, Anke, Henrik Knecht, Anton W. Langerak, et al. "Analytical Validation of Patient-Specific PCR-Based MRD Assessment for Use As a Primary Endpoint in CLL Clinical Trials." Blood 126, no. 23 (2015): 2924. http://dx.doi.org/10.1182/blood.v126.23.2924.2924.

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Abstract Introduction. Minimal residual disease (MRD) is an objective measure of disease status defined by the number of leukemic cells in the blood or bone marrow of leukemic patients. In recent clinical studies of chronic lymphocytic leukemia (CLL), undetectable MRD levels (< 1 tumor cell/10,000 leukocytes) have been shown to correlate with prolonged progression free survival (PFS) and overall survival, independent of treatment or known risk factors. MRD assessment has been proposed as an alternative to PFS as a primary endpoint in frontline CLL pivotal studies to evaluate the efficacy of

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7

Dimier, Natalie, Paul Delmar, Carol Ward, et al. "A Model for Predicting Effect of Treatment on Progression-Free Survival Using Minimal Residual Disease As a Surrogate Endpoint in Chronic Lymphocytic Leukemia." Blood 126, no. 23 (2015): 720. http://dx.doi.org/10.1182/blood.v126.23.720.720.

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Abstract Introduction The standard primary endpoint in clinical trials of chronic lymphocytic leukemia (CLL) is progression-free survival (PFS). Given the increasingly long follow up required to detect differences in PFS between treatment arms in the era of more efficient therapeutics, valid surrogate endpoints are urgently needed to reduce clinical trial duration, thereby accelerating drug development, reducing costs and allowing patients (pts) earlier access to novel treatment options. Pts with CLL who achieve levels of minimal residual disease (MRD) of <1 clonal cell/10.000 leukocytes in

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Wang, Xiaofei, Xiaoyi Wang, Lydia Hodgson, et al. "Validation of Progression‐Free Survival as a Surrogate Endpoint for Overall Survival in Malignant Mesothelioma: Analysis of Cancer and Leukemia Group B and North Central Cancer Treatment Group (Alliance) Trials." Oncologist 22, no. 2 (2017): 189–98. http://dx.doi.org/10.1634/theoncologist.2016-0121.

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9

Kaur, Pavinder, Anil Pahuja, Kevin Nguyen, et al. "Best Practices for Validation of Measurable Residual Disease Assessments By Multiparameter Flow Cytometry in Emerging Clinical Trials of Acute Myeloid Leukemia." Blood 136, Supplement 1 (2020): 22–23. http://dx.doi.org/10.1182/blood-2020-137787.

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Background: Measurable Residual Disease (MRD) assessments are gaining increasing acceptance as a prognostic factor for tailoring treatment in hematological malignancies. Acute Myeloid Leukemia (AML) is a heterogeneous disease with high relapse rates and presents a high unmet need for effective treatment options. Measurement of residual disease after therapy reflects a combination of all resistance mechanisms and is currently used for guiding treatment options. Study Design: In this study, we aimed to validate an AML-MRD assay by multiparameter flow cytometry (MFC) methodology. This is a 4-tube

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Bahar, N., L. Mohseninejad, K. McDonald, and T. Wilke. "A META‐ANALYTIC ENDPOINT VALIDATION OF SURROGATES USED IN CLINICAL TRIALS EVALUATING THE EFFICACY OF THERAPIES IN PATIENTS WITH CHRONIC LYMPHOCYTIC LEUKEMIA (CLL)." Hematological Oncology 41, S2 (2023): 743–44. http://dx.doi.org/10.1002/hon.3165_594.

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Bahar, Nasim, Leyla Mohseninejad, Keltie Mcdonald, and Thomas Wilke. "PB1947: A META-ANALYTIC ENDPOINT VALIDATION OF SURROGATES USED IN CLINICAL TRIALS EVALUATING THE EFFICACY OF THERAPIES IN PATIENTS WITH CHRONIC LYMPHOCYTIC LEUKEMIA." HemaSphere 7 (August 2023): e641659a. http://dx.doi.org/10.1097/01.hs9.0000974604.64165.9a.

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12

Xie, Zhiyi, Lisa Chamberlain, Andrew Carson, et al. "Monitoring Minimal Residual Disease in Acute Myeloid Leukemia Using Genomic or cfDNA with MyMRD®, a Targeted NGS Panel." Blood 132, Supplement 1 (2018): 5268. http://dx.doi.org/10.1182/blood-2018-99-118971.

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Abstract Acute myeloid leukemia (AML) is a genetically and phenotypically heterogeneous disorder. Precision therapies for AML have been developed that target specific driver mutations. The efficacies of these therapies are variable, making it critical to determine successful therapies prior to patient relapse. For patients achieving a first complete remission, minimum residual disease (MRD) is an important prognostic factor, as MRD may provide a powerful and timely tool to evaluate therapeutic efficacy. There is a growing demand that new and promising drugs are approved as quickly as possible

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Catania, Gioacchino, Federico Monaco, Massimo Pini, et al. "Prognostic Impact of p190 and p210 Co-Expression at Diagnosis in Chronic Myeloid Leukemia (CML) Patients Treated with Imatinib." Blood 124, no. 21 (2014): 5528. http://dx.doi.org/10.1182/blood.v124.21.5528.5528.

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Abstract Background: Expression of p190 BCR-ABL mRNA is generally considered to be confined to patients with acute lymphoid or more rarely myeloid leukemias, whereas p210 BCR-ABL mRNA is the hallmark of CML. In reality it is not uncommon the presence of p190 m-RNA in p210 CML in chronic phase, due to alternative or missplicing1. Its presence seems to have no impact on prognosis in the pre-TKI era, although it may be expression of genomic instability. Aim: Primary object of this study was to investigate if the co-expression might influence the rate of early outcome surrogate endpoints such as s

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Malek, Sami N., Peter Ouillette, Harry Erba, et al. "Genomic Complexity Identifies Patients with Agressive Chronic Lymphocytic Leukemia." Blood 110, no. 11 (2007): 489. http://dx.doi.org/10.1182/blood.v110.11.489.489.

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Abstract Chronic lymphocytic leukemia (CLL) is the most common leukemia in the western world. CLL has a varied clinical course. Risk assessment and patient counseling relies on clinical parameters and validated biomarkers. Genetic biomarkers, as exemplified in the CLL FISH panel, allow for CLL patient risk stratification. Nonetheless, a subset of patients with apparently favorable lesions (del13q14 or normal FISH results) progresses rapidly and a fraction of patients with unfavorable findings (del17p or del11q) progress at less than predicted rate. Method: We studied 117 previously untreated C

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15

Orgel, Etan, Celia Framson, Rubi Vazquez, David R. Freyer, and Steven D. Mittelman. "Age but Not Adiposity Predicts Asparaginase-Induced Hepatotoxicity during Induction Therapy for Adolescent and Young Adults with Acute Lymphoblastic Leukemia." Blood 132, Supplement 1 (2018): 2662. http://dx.doi.org/10.1182/blood-2018-99-110268.

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Abstract INTRODUCTION: Obesity is associated with increased treatment-related toxicity during therapy for acute lymphoblastic leukemia (ALL). During induction, hepatotoxicity due to L-asparaginase results in significant morbidity and chemotherapy delays/modifications. While hepatotoxicity is more common in older adolescent and young adult (AYA) patients, it cannot be predicted with high accuracy. Obesity defined by the surrogate marker of body mass index (BMI) has been shown to be a risk factor for hepatotoxicity; however, BMI does not accurately reflect body adiposity during ALL therapy due t

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Rawstron, Andy, Claudia Fazi, Neus Villamor, et al. "A Complementary Role of High Throughput Sequencing and Multiparameter Cytometry for Minimal Residual Disease (MRD) Detection in Chronic Lymphocytic Leukemia (CLL):an European Research Initiative (ERIC) Study." Blood 124, no. 21 (2014): 1976. http://dx.doi.org/10.1182/blood.v124.21.1976.1976.

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Abstract BACKGROUND. The detection of minimal residual disease (MRD) at the level of 0.01%/10-4 or above is a strong independent predictor of reduced progression-free (PFS) and overall survival (OS) in patients with CLL treated with chemoimmunotherapy. Although newer agents such as B-cell receptor pathway inhibitors can result in prolonged survival without achieving complete response, there remains a important role for MRD analysis in assessing therapeutic strategies aimed at disease eradication and cure. This is particularly important in front-line trials for fit patients which now require at

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Smith, B. Douglas, Andrew W. Roberts, Gail J. Roboz, et al. "Minimal Residual Disease (MRD) As Exploratory Endpoint in a Phase 1 Study of the Anti-CD123 Mab CSL362 Given As Post-Remission Therapy in Adult Acute Myeloid Leukemia (AML)." Blood 126, no. 23 (2015): 3819. http://dx.doi.org/10.1182/blood.v126.23.3819.3819.

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Abstract Background AML patients (pts) who achieve a morphologic complete remission (CR) may have MRD in bone marrow (BM) and/or peripheral blood detected as patient-specific leukemia-aberrant immunophenotype by multiparameter flow cytometry (MFC) or as abnormal transcripts of AML gene mutations/translocations by quantitative RT-PCR. Presence of MRDafter induction and/or consolidation therapy identifies pts at high risk of relapse, with increasing MRD levels indicating impendingrelapse in individual pts. Although standardized testing and validated endpoints for MRD determination across institu

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18

Catania, Gioacchino, Nicol Trincheri, Enrico Marco Gottardi, et al. "Prognostic Impact of p190 and p210 Co-Expression in Early Molecular Response in Chronic Myeloid Leukemia (CML) Patients Treated with Tyrosin Kinase Inibitors." Blood 128, no. 22 (2016): 5446. http://dx.doi.org/10.1182/blood.v128.22.5446.5446.

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Abstract Background: Expression of p190 BCR-ABL mRNA is generally considered to be confined to patients with acute lymphoid or more rarely myeloid leukemias, whereas p210 BCR-ABL mRNA is the hallmark of CML. In reality it is not uncommon the presence of p190 m-RNA in p210 CML in chronic phase, due to alternative or missplicing. Its presence seems to have no impact on prognosis in the pre-TKI era, although it may be expression of genomic instability. Aim: Primary object of this study was to investigate if the co-expression might influence the rate of early outcome surrogate endpoints such as su

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19

Ciani, Oriana, Bogdan Grigore, Hedwig Blommestein, et al. "Validity of Surrogate Endpoints and Their Impact on Coverage Recommendations: A Retrospective Analysis across International Health Technology Assessment Agencies." Medical Decision Making 41, no. 4 (2021): 439–52. http://dx.doi.org/10.1177/0272989x21994553.

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Background Surrogate endpoints (i.e., intermediate endpoints intended to predict for patient-centered outcomes) are increasingly common. However, little is known about how surrogate evidence is handled in the context of health technology assessment (HTA). Objectives 1) To map methodologies for the validation of surrogate endpoints and 2) to determine their impact on acceptability of surrogates and coverage decisions made by HTA agencies. Methods We sought HTA reports where evaluation relied on a surrogate from 8 HTA agencies. We extracted data on the methods applied for surrogate validation. W

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Rossi, Davide, Silvia Rasi, Claudio Tripodo, et al. "Host Genetic Background and Risk of Richter Syndrome: The Genotype of LRP4 Is An Independent Predictor of Chronic Lymphocytic Leukemia Transformation to Aggressive Lymphoma." Blood 114, no. 22 (2009): 2340. http://dx.doi.org/10.1182/blood.v114.22.2340.2340.

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Abstract Abstract 2340 Poster Board II-317 Richter syndrome (RS) represents the transformation of chronic lymphocytic leukemia (CLL) to aggressive lymphoma, most commonly diffuse large B-cell lymphoma (DLBCL). Mechanisms and risk factors of CLL transformation to RS are known only in part. This study aimed at exploring the role of the host genetic background in RS transformation and was based on a consecutive series of 331 CLL, of which 21 had transformed to RS (all clonally related to the CLL clone). Twenty eight additional cases of clonally related RS were also collected for validation purpos

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Goulart, Hannah, Dahniel L. Sastow, Nicholas Dreher, et al. "Do Hematologic Parameters Predict Thrombosis or Disease Transformation in Essential Thrombocythemia?" Blood 142, Supplement 1 (2023): 3200. http://dx.doi.org/10.1182/blood-2023-182450.

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Background: Patients with essential thrombocythemia (ET) are burdened with an increased risk of thrombosis and disease transformation, both significant contributors to morbidity and mortality. However, based on the available literature, there are limited tools available to predict the development of these complications. Particularly, the association between hematologic parameters and thrombosis is unclear, making risk stratification and disease management challenging. Utilizing a large retrospective database, we assessed the impact of hematocrit (Hct), red blood cell count (RBC), white blood c

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Green, EM, G. Yothers, and Daniel J. Sargent. "Surrogate endpoint validation: statistical elegance versus clinical relevance." Statistical Methods in Medical Research 17, no. 5 (2008): 477–86. http://dx.doi.org/10.1177/0962280207081863.

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Burzykowski, Tomasz, and Marc Buyse. "Surrogate threshold effect: an alternative measure for meta-analytic surrogate endpoint validation." Pharmaceutical Statistics 5, no. 3 (2006): 173–86. http://dx.doi.org/10.1002/pst.207.

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Liu, WP, and L. Pacheco. "POSB285 Five Identified Hurdles Associated with Surrogate Endpoint Validation." Value in Health 25, no. 1 (2022): S182. http://dx.doi.org/10.1016/j.jval.2021.11.887.

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Bebu, Ionut, Thomas Mathew, and Brian Agan. "Inference for Surrogate Endpoint Validation in the Binary Case." Journal of Biopharmaceutical Statistics 25, no. 6 (2015): 1272–84. http://dx.doi.org/10.1080/10543406.2015.1008516.

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Komrokji, Rami S., Amy E. DeZern, Katrina Zell, et al. "Validation of International Working Group (IWG) Response Criteria in Higher-Risk Myelodysplastic Syndromes (MDS): A Report on Behalf of the MDS Clinical Research Consortium (MDS CRC)." Blood 126, no. 23 (2015): 909. http://dx.doi.org/10.1182/blood.v126.23.909.909.

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Introduction The primary goal for treatment of higher-risk MDS patients (pts) is to improve overall survival (OS) and delay acute myeloid leukemia (AML) evolution. The IWG 2006 response criteria are used in clinical trials and in clinical practice for assessing efficacy of MDS therapies. These criteria were originally proposed by an international group of experts based on available data and consensus. In an ad hoc landmark analysis of the AZA-001 study using the 2006 IWG criteria, pts who achieved hematological improvement (HI), complete response (CR), marrow CR (mCR), or partial response (PR)

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Wagner, John A. "Overview of Biomarkers and Surrogate Endpoints in Drug Development." Disease Markers 18, no. 2 (2002): 41–46. http://dx.doi.org/10.1155/2002/929274.

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There are numerous factors that recommend the use of biomarkers in drug development including the ability to provide a rational basis for selection of lead compounds, as an aid in determining or refining mechanism of action or pathophysiology, and the ability to work towards qualification and use of a biomarker as a surrogate endpoint. Examples of biomarkers come from many different means of clinical and laboratory measurement. Total cholesterol is an example of a clinically useful biomarker that was successfully qualified for use as a surrogate endpoint. Biomarkers require validation in most

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Trock, Bruce J. "Validation of surrogate endpoint biomarkers in prostate cancer chemoprevention trials." Urology 57, no. 4 (2001): 241–47. http://dx.doi.org/10.1016/s0090-4295(00)00983-3.

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Huang, Guan-Hua, Chin-Chiang Hsieh, Chen-Hsin Chen, and Wei J. Chen. "Statistical validation of endophenotypes using a surrogate endpoint analytic analogue." Genetic Epidemiology 33, no. 6 (2009): 549–58. http://dx.doi.org/10.1002/gepi.20407.

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Candida Fratazzi and Jixiao Niu. "Accelerated orphan drug approval: surrogate endpoints." World Journal of Advanced Pharmaceutical and Medical Research 2, no. 1 (2022): 001–7. http://dx.doi.org/10.53346/wjapmr.2022.2.1.0021.

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Today, orphan drug development is confronted with significant challenges represented by the considerable complexity, diversity of clinical manifestations, and competition in study recruitment. Thus, surrogate endpoints adoption plays a crucial role in rare disease trials by minimizing costs, the number of subjects, and study duration. Surrogate endpoints, to substitute for a direct measure of how patients feel, function, or survive, must be biomarkers that directly correlate with disease clinical manifestations and predict the impact of study drug on the long-term disease progression. Validati

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Ciani, Oriana, Sarah Davis, Paul Tappenden, et al. "VALIDATION OF SURROGATE ENDPOINTS IN ADVANCED SOLID TUMORS: SYSTEMATIC REVIEW OF STATISTICAL METHODS, RESULTS, AND IMPLICATIONS FOR POLICY MAKERS." International Journal of Technology Assessment in Health Care 30, no. 3 (2014): 312–24. http://dx.doi.org/10.1017/s0266462314000300.

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Objectives: Licensing of, and coverage decisions on, new therapies should rely on evidence from patient-relevant endpoints such as overall survival (OS). Nevertheless, evidence from surrogate endpoints may also be useful, as it may not only expedite the regulatory approval of new therapies but also inform coverage decisions. It is, therefore, essential that candidate surrogate endpoints be properly validated. However, there is no consensus on statistical methods for such validation and on how the evidence thus derived should be applied by policy makers.Methods: We review current statistical ap

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Woods, Gail, Marisa Miceli, Monica Grazziutti, et al. "Validation of Serum Aspergillus Galactomannan Index as a Surrogate Endpoint for Outcome of Invasive Aspergillosis: Clinical and Research Implications." Blood 108, no. 11 (2006): 2861. http://dx.doi.org/10.1182/blood.v108.11.2861.2861.

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Abstract Background: Assessing clinical outcome of aspergillosis with conventional clinical and laboratory criteria is difficult. A composite “global outcome response” (clinical, radiologic, pathologic and microbiologic criteria) is frequently used but suffers from poor sensitivity and specificity, and has not been standardized or validated. A reliable, quantitative, non-invasive, and easy to measure laboratory test than can substitute for this composite endpoint, i.e. serve as a surrogate endpoint for aspergillosis outcome is highly desirable. Galactomannan (GM) is an Aspergillus-specific pol

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Santosh Kumar, Rada, and Ganesh Sai Myneni. "SURROGATE ENDPOINT: ALTERNATIVE FOR EARLY ASSESSMENT OF A POTENTIAL TREATMENT EFFECT." Journal of Drug Delivery and Therapeutics 9, no. 4-s (2019): 819–21. http://dx.doi.org/10.22270/jddt.v9i4-s.3371.

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The efficacy of health technologies, medicines and medical devices should be demonstrated in trails that evaluate final patient-relevant outcomes such as survival or morbidity. We provide a summary of the present use of surrogate end points in health care policy, discussing the case for and against their reviewing and adoption validation methods. Although the use of surrogates can be problematic, they can be validated and selected properly, offers important chances for more efficient clinical trials and faster access to new health technologies that benefit health care systems and patients. In

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Hopkinson, D., C. Chadwick, M. Bibi, and A. Bastian. "Review of Surrogate Endpoint Validation Methodologies and Application In Solid Tumour Htas." Value in Health 20, no. 9 (2017): A731. http://dx.doi.org/10.1016/j.jval.2017.08.1994.

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Pullen, Matthew F., Katherine Huppler Hullsiek, Joshua Rhein, et al. "Cerebrospinal Fluid Early Fungicidal Activity as a Surrogate Endpoint for Cryptococcal Meningitis Survival in Clinical Trials." Clinical Infectious Diseases 71, no. 7 (2020): e45-e49. http://dx.doi.org/10.1093/cid/ciaa016.

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Abstract Background In cryptococcal meningitis phase 2 clinical trials, early fungicidal activity (EFA) of Cryptococcus clearance from cerebrospinal fluid (CSF) is used as a surrogate endpoint for all-cause mortality. The Food and Drug Administration allows for using surrogate endpoints for accelerated regulatory approval, but EFA as a surrogate endpoint requires further validation. We examined the relationship between rate of CSF Cryptococcus clearance (EFA) and mortality through 18 weeks. Methods We pooled individual-level CSF data from 3 sequential cryptococcal meningitis clinical trials co

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Miksad, Rebecca A., Vera Zietemann, Raffaella Gothe, et al. "Progression-free survival as a surrogate endpoint in advanced breast cancer." International Journal of Technology Assessment in Health Care 24, no. 04 (2008): 371–83. http://dx.doi.org/10.1017/s0266462308080495.

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Objectives:Progression-free survival (PFS) has not been validated as a surrogate endpoint for overall survival (OS) for anthracycline (A) and taxane-based (T) chemotherapy in advanced breast cancer (ABC). Using trial-level, meta-analytic approaches, we evaluated PFS as a surrogate endpoint.Methods:A literature review identified randomized, controlled A and T trials for ABC. Progression-based endpoints were classified by prospective definitions. Treatment effects were derived as hazard ratios for PFS (HRPFS) and OS (HROS). Kappa statistic assessed overall agreement. A fixed-effects regression m

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Renard, Didier, Helena Geys, Geert Molenberghs, et al. "Validation of a longitudinally measured surrogate marker for a time-to-event endpoint." Journal of Applied Statistics 30, no. 2 (2003): 235–47. http://dx.doi.org/10.1080/0266476022000023776.

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Deng, Yanhong, Yue Cai, Jianwei Zhang, et al. "Validation of neoadjuvant rectal cancer(NAR) score as a surrogate endpoint for disease free survival in Chinese FOWARC study." Journal of Clinical Oncology 37, no. 15_suppl (2019): e15162-e15162. http://dx.doi.org/10.1200/jco.2019.37.15_suppl.e15162.

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e15162 Background: NAR score is considered as a surrogate endpoint for survival in patients with locally advanced rectal cancer who received neoadjuvant treatment. Here we validate it in Chinese FOWARC study, especially in the chemotherapy alone arm. Methods: NAR score is calculated based on cT, ypT, ypN as reported for patients who received surgery in FOWARC study. NAR score is correlated to DFS with COX regression and Kaplan-Meier analysis. Results: 451 patients had surgery, 147 in FU-RT group, 152 in FOLFOX-RT arm and 152 in FOLFOX arm. NAR score is a significant prognostic factor in COX re

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Wang, Y. C., A. Sandrock, J. R. Richert, L. Meyerson, and X. Miao. "Short-Term Relapse Quantitation as a Fully Surrogate Endpoint for Long-Term Sustained Progression of Disability in RRMS Patients Treated with Natalizumab." Neurology Research International 2011 (2011): 1–6. http://dx.doi.org/10.1155/2011/195831.

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Time to sustained worsening in the expanded disability status scale as the standard for evaluating the accumulation of disability has been used as a measure of clinical efficacy in many relapsing-remitting multiple sclerosis (RRMS) clinical trials. However, this measurement usually requires a large sample and long-term study to demonstrate the treatment effect. Annualized relapse rate or time to first relapse is also widely used as alternative measurements of clinical efficacy. A formal statistical validation of short-term relapse activity as a surrogate endpoint for long-term sustained progre

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Munayco-Guillén, Fernando, Hilbert Cevallos-Alvarado, Susy Bazán-Ruiz, Omar Espinoza-Yovera, and Rafael Pichardo-Rodríguez. "Características y tendencias de los ensayos clínicos sobre tratamientos en leucemias: análisis del Registro Peruano de Ensayos Clínicos (1995-2024)." Revista de la Facultad de Medicina Humana 25, no. 1 (2025): 126–32. https://doi.org/10.25176/rfmh.v2025i1.6825.

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To describe the characteristics and trends of clinical trials (CTs) on leukemia registered in the Peruvian Clinical Trial Registry (REPEC, by its Spanish acronym), a descriptive study was conducted on CTs registered between 1995 and July 2024. The REPEC database was searched using the term “leukemia.” Included were CTs involving patients with acute or chronic leukemias, regardless of disease stage or prior treatment. Variables analyzed included type of leukemia, trial phase, type of product, sponsor, international registration, type of outcome, and methodological characteristics. Out of 2,058

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Sertdemir, Y., and R. Burgut. "Does the decision in a validation process of a surrogate endpoint change with level of significance of treatment effect? A proposal on validation of surrogate endpoints." Contemporary Clinical Trials 30, no. 1 (2009): 8–12. http://dx.doi.org/10.1016/j.cct.2008.08.006.

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Ciani, Oriana, Massimo Piepoli, Neil Smart, et al. "Validation of Exercise Capacity as a Surrogate Endpoint in Exercise-Based Rehabilitation for Heart Failure." JACC: Heart Failure 6, no. 7 (2018): 596–604. http://dx.doi.org/10.1016/j.jchf.2018.03.017.

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Caron, Melissa, Sabrina Hoa, Marie Hudson, Kevin Schwartzman, and Russell Steele. "Pulmonary function tests as outcomes for systemic sclerosis interstitial lung disease." European Respiratory Review 27, no. 148 (2018): 170102. http://dx.doi.org/10.1183/16000617.0102-2017.

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Interstitial lung disease (ILD) is the leading cause of morbidity and mortality in systemic sclerosis (SSc). We performed a systematic review to characterise the use and validation of pulmonary function tests (PFTs) as surrogate markers for systemic sclerosis-associated interstitial lung disease (SSc-ILD) progression.Five electronic databases were searched to identify all relevant studies. Included studies either used at least one PFT measure as a longitudinal outcome for SSc-ILD progression (i.e. outcome studies) and/or reported at least one classical measure of validity for the PFTs in SSc-I

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Enweonye, Igwebuike, and Edith Uzoma Umeh. "Bayesian Meta-Analysis to Validate Correlate of Protection for High Vaccine Efficacy Clinical Trials." European Journal of Information Technologies and Computer Science 2, no. 1 (2022): 19–25. http://dx.doi.org/10.24018/compute.2022.2.1.34.

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In clinical trials, a correlate (surrogate) of protection (CoP) endpoint must be properly validated through rigorous sound methods before it may be approved for use. The validation of surrogate in the context of high vaccine efficacy trials, however, poses great challenge due to sparse data; and conventional methods for statistical validation of surrogate are no longer adequate. Although idea of surrogacy was developed in the context of a single trial, the meta-analytic approach, which allows both individual and trial level surrogacy, has become well accepted. However, the meta-analytic joint

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Simon, Florian, Rudy Ligtvoet, Sandra Robrecht, et al. "Endpoint Surrogacy in Chronic Lymphocytic Leukemia: A Pooled Analysis of the German CLL Study Group." Blood 142, Supplement 1 (2023): 1901. http://dx.doi.org/10.1182/blood-2023-178597.

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Introduction Overall survival (OS) is generally considered the most patient-relevant endpoint in oncology trials, however, its implementation can entail several challenges in the context of chronic lymphocytic leukemia (CLL): 1) The competing survival risk due to comorbidities in the mostly elderly CLL patient population; 2) the high efficacy of targeted therapies warrant longer observation periods or extensive patient recruitment, which can be operationally challenging; 3) the interference with increasingly potent relapse therapies, which can neutralize differences of efficacy of first line r

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Meddi, Elisa, Arianna Savi, Federico Moretti, et al. "Measurable Residual Disease (MRD) as a Surrogate Efficacy-Response Biomarker in AML." International Journal of Molecular Sciences 24, no. 4 (2023): 3062. http://dx.doi.org/10.3390/ijms24043062.

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In acute myeloid leukemia (AML) many patients experience relapse, despite the achievement of morphological complete remission; therefore, conventional morphologic criteria are currently considered inadequate for assessing the quality of the response after treatment. Quantification of measurable residual disease (MRD) has been established as a strong prognostic marker in AML and patients that test MRD negative have lower relapse rates and better survival than those who test positive. Different techniques, varying in their sensitivity and applicability to patients, are available for the measurem

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Funingana, Ionut-Gabriel, Pubudu Piyatissa, Marika Reinius, Cathal McCague, Bristi Basu, and Evis Sala. "Radiomic and Volumetric Measurements as Clinical Trial Endpoints—A Comprehensive Review." Cancers 14, no. 20 (2022): 5076. http://dx.doi.org/10.3390/cancers14205076.

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Clinical trials for oncology drug development have long relied on surrogate outcome biomarkers that assess changes in tumor burden to accelerate drug registration (i.e., Response Evaluation Criteria in Solid Tumors version 1.1 (RECIST v1.1) criteria). Drug-induced reduction in tumor size represents an imperfect surrogate marker for drug activity and yet a radiologically determined objective response rate is a widely used endpoint for Phase 2 trials. With the addition of therapies targeting complex biological systems such as immune system and DNA damage repair pathways, incorporation of integra

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Grigore, Bogdan, Oriana Ciani, Florian Dams, et al. "Surrogate Endpoints in Health Technology Assessment: An International Review of Methodological Guidelines." PharmacoEconomics 38 (November 27, 2020): 1055–70. https://doi.org/10.1007/s40273-020-00935-1.

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In the drive towards faster patient access to treatments, health technology assessment (HTA) agencies are increasingly faced with reliance on evidence from surrogate endpoints, leading to increased decision uncertainty. This study undertook an updated survey of methodological guidance for using surrogate endpoints across international HTA agencies. We reviewed HTA and economic evaluation methods guidance from European, Australian and Canadian HTA agencies. We considered how guidelines addressed the methods for handling surrogate endpoints, including (1) level of evidence, (2) methods of valida

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Bogdan, Grigore, Oriana Ciani, Florian Dams, et al. "Surrogate Endpoints in Health Technology Assessment: An International Review of Methodological Guidelines." Pharmacoeconomics 38 (February 2, 2021): 1055–70. https://doi.org/10.5281/zenodo.4493612.

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In the drive towards faster patient access to treatments, health technology assessment (HTA) agencies are increasingly faced with reliance on evidence from surrogate endpoints, leading to increased decision uncertainty. This study undertook an updated survey of methodological guidance for using surrogate endpoints across international HTA agencies. We reviewed HTA and economic evaluation methods guidance from European, Australian and Canadian HTA agencies. We considered how guidelines addressed the methods for handling surrogate endpoints, including (1) level of evidence, (2) methods of valida

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Pessach, Ilias, Theodoros Spyropoulos, Eleftheria Lamprianidou, and Ioannis Kotsianidis. "MRD Monitoring by Multiparametric Flow Cytometry in AML: Is It Time to Incorporate Immune Parameters?" Cancers 14, no. 17 (2022): 4294. http://dx.doi.org/10.3390/cancers14174294.

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Acute myeloid leukemia (AML) is a heterogeneous group of clonal myeloid disorders characterized by intrinsic molecular variability. Pretreatment cytogenetic and mutational profiles only partially inform prognosis in AML, whereas relapse is driven by residual leukemic clones and mere morphological evaluation is insensitive for relapse prediction. Measurable residual disease (MRD), an independent post-diagnostic prognosticator, has recently been introduced by the European Leukemia Net as a new outcome definition. However, MRD techniques are not yet standardized, thus precluding its use as a surr

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