Thesis
Improved surrogacy regressions for clinical trial endpoints in relapse/refractory and newly diagnosed multiple myeloma
Master of Science (M.S.), Drexel University
Dec 2023
DOI:
https://doi.org/10.17918/00001910
Abstract
Use of surrogate endpoints such as objective response rate (ORR) and progression-free survival (PFS) is common in multiple myeloma (MM) risk-benefit modelling for treatment approval, and notably accelerated approval. Yet, there is conflicting evidence supporting their surrogacy between themselves and overall survival (OS). This systematic review characterises all published single arm trials and randomised controlled trials (RCTs) in MM using ORR, PFS, TTP and OS endpoints between 1999 and 2022 (databases provided in supplemental information), and assesses the strength of surrogacy between each relationship, to determine if current data can support improve estimations for survival endpoints. The regressions were evaluated with the coefficient of determination with Pearson's correlation, and the F-test was used in cases of multivariate regressions. The analysis demonstrated that current regressions overpredict survivability endpoints, but there are multivariate models that can accurately predict clinical outcomes with patient characteristics and surrogate endpoints for both RRMM and NDMM.
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Details
- Title
- Improved surrogacy regressions for clinical trial endpoints in relapse/refractory and newly diagnosed multiple myeloma
- Creators
- Rhys Jonathan Butler
- Contributors
- Nicolas J. Alvarez (Advisor)
- Awarding Institution
- Drexel University
- Degree Awarded
- Master of Science (M.S.)
- Publisher
- Drexel University; Philadelphia, Pennsylvania
- Number of pages
- x, 166 pages
- Resource Type
- Thesis
- Language
- English
- Academic Unit
- Chemical (and Biological) Engineering (1970-2026); College of Engineering (1970-2026); Drexel University
- Other Identifier
- 991021819214904721