Design and Methodological Considerations for Real World Data-Derived Progression-Free Survival in Multiple Myeloma

作者：Jocelyn R. Wang, Jennifer Hayden, Yiyang Yue, Craig S. Meyer, Ryan W. Gan, Youyi Zhang, Benjamin Ackerman, Pranay Mohanty, James Roose, Jennifer L. Lund, Sebastian Schneeweiß, Janick Weberpals, Omar Nadeem, Sikander Ailawadhi, Noopur Raje, Smith Giri, Juned Siddique, Laura Hester, Kelly Reid, Robin Carson, Khaled Sarsour, Ashita S. Batavia · 发表于：Blood · 年份：2024 · DOI：10.1182/blood-2024-199224 · 被引用次数：3 · 研究领域：Multiple Myeloma Research and Treatments

Introduction: Progression-free survival (PFS), a common endpoint used in multiple myeloma (MM) trials, is defined as the time to the earliest occurrence of disease progression or death. Disease progression in MM is determined by the International Myeloma Working Group (IMWG) Uniform Response Criteria based on imaging and biomarkers from blood, urine, and bone marrow biopsy testing. In clinical trials, IMWG-recommended assessments are protocol-based. However, in routine care, they are often performed and recorded differently based on a multitude of factors. Algorithms to ascertain progression events using routinely collected biomarker data are important to classify disease progression and align with trial-defined endpoints. The aim of this study was to construct and evaluate an optimized real-world Progression (rwP) algorithm using real-world data (RWD) and demonstrate how biomarker data are captured and used to derive endpoints in routine practice. Method: A cohort of 270 transplant ineligible newly diagnosed MM (NDMM) patients starting on lenalidomide and dexamethasone (Rd) and meeting select MAIA (NCT02252172) trial eligibility criteria were included from the nationwide Flatiron Health electronic health record-derived deidentified database from 2015 to 2023. A rwP algorithm was developed using key IMWG-recommended biomarkers, including serum protein electrophoresis (SPEP), urine protein electrophoresis (UPEP), and serum free light chains (FLCs). The initial baseline value f...