HETEROGENEITY OF EVENT-FREE SURVIVAL DEFINITION IN RELAPSED OR REFRACTORY DIFFUSE LARGE B-CELL LYMPHOMA
Author(s)
Liva Andersone, MSc1, Marcell Csanádi, MSc, PhD2, György Széles, MD, MSc, PhD2, Joao Duarte Mendes, MSc3, Ahmed Rasmy, MD4, Alla Uhrlandt, BA, MBA, BA, MBA5, Maren Gaudig, MSc3, Carmela Maffucci, PhD3.
1Johnson & Johnson Innovative Medicine, Riga, Latvia, 2Syreon Research Institute, Budapest, Hungary, 3Johnson & Johnson Innovative Medicine, Raritan, NJ, USA, 4Syreon Middle East, Alexandria, Egypt, 5Johnson & Johnson Innovative Medicine, Neuss, Germany.
1Johnson & Johnson Innovative Medicine, Riga, Latvia, 2Syreon Research Institute, Budapest, Hungary, 3Johnson & Johnson Innovative Medicine, Raritan, NJ, USA, 4Syreon Middle East, Alexandria, Egypt, 5Johnson & Johnson Innovative Medicine, Neuss, Germany.
OBJECTIVES: Diffuse large B-cell lymphoma (DLBCL) is an aggressive but potentially curable malignancy; however, treating relapsed/refractory (RR) disease remains challenging. In R/R setting, where curative-intent approaches remain relevant for selected patients, event-free survival (EFS) has emerged as a clinically relevant endpoint. As more trials report EFS, differences in how this endpoint is defined may affect cross-trial interpretation. This study aimed to compare EFS definitions across clinical trials and describe the main sources of variation.
METHODS: Clinical trials in RR DLBCL since 2019 were identified based on a recently conducted systematic literature review covering PubMed, Scopus, Central and grey literature sources. Trial-level EFS definitions were collected from the identified publications then supplemented and cross-checked using ClinicalTrials.gov and study protocol information when available.
RESULTS: Overall, 36 trials were identified and EFS definitions were found in 11. Across definitions, progression and death from any cause were shared events. However, several areas of variation were observed. One was index time, with EFS measurement beginning from randomization, infusion, or first treatment administration. Another area was the role of response, including whether failure to achieve a prespecified response by a defined assessment timepoint was counted as an event, for example stable disease up to day 150, or failure to achieve complete or partial response by week 9. Initiation of subsequent treatment as an event and how that treatment was categorized (i.e. anti-lymphoma or anti-neoplastic) also differed. Whether non-disease events such as patient withdrawal or treatment discontinuation were captured, and whether subsequent stem cell transplantation was explicitly included or excluded were other sources of heterogeneity.
CONCLUSIONS: Our findings describe a high definitional variation across trials without ranking one definition over another. Clearly, EFS was not defined uniformly across trials in RR DLBCL, and these differences should be recognized when interpreting or comparing EFS results across studies.
METHODS: Clinical trials in RR DLBCL since 2019 were identified based on a recently conducted systematic literature review covering PubMed, Scopus, Central and grey literature sources. Trial-level EFS definitions were collected from the identified publications then supplemented and cross-checked using ClinicalTrials.gov and study protocol information when available.
RESULTS: Overall, 36 trials were identified and EFS definitions were found in 11. Across definitions, progression and death from any cause were shared events. However, several areas of variation were observed. One was index time, with EFS measurement beginning from randomization, infusion, or first treatment administration. Another area was the role of response, including whether failure to achieve a prespecified response by a defined assessment timepoint was counted as an event, for example stable disease up to day 150, or failure to achieve complete or partial response by week 9. Initiation of subsequent treatment as an event and how that treatment was categorized (i.e. anti-lymphoma or anti-neoplastic) also differed. Whether non-disease events such as patient withdrawal or treatment discontinuation were captured, and whether subsequent stem cell transplantation was explicitly included or excluded were other sources of heterogeneity.
CONCLUSIONS: Our findings describe a high definitional variation across trials without ranking one definition over another. Clearly, EFS was not defined uniformly across trials in RR DLBCL, and these differences should be recognized when interpreting or comparing EFS results across studies.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
CO121
Topic
Clinical Outcomes, Health Technology Assessment, Methodological & Statistical Research
Topic Subcategory
Clinical Outcomes Assessment, Comparative Effectiveness or Efficacy
Disease
Oncology