ReviewBlood cancer journal2024
Progression-free survival as a surrogate endpoint in myeloma clinical trials: an evolving paradigm.
Review in Blood cancer journal, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 17 papers, 4 of them syntheses that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
17 citing papers in PubMed, 4 syntheses or guidelines pooled it.
- Optimizing oncology drug development: systematic review of 22 years of myeloma randomized controlled trials.Journal of the National Cancer Institute · 2026Pooled it
- Observation or treatment for smoldering multiple myeloma? A systematic review and meta-analysis of randomized controlled studies.Blood cancer journal · 2025Pooled it
- Daratumumab-based quadruplet versus triplet induction regimens in transplant-eligible newly diagnosed multiple myeloma: a systematic review and meta-analysis.Blood cancer journal · 2025Pooled it
- Evaluating Minimal Residual Disease Negativity as a Surrogate Endpoint for Treatment Efficacy in Multiple Myeloma: A Meta-Analysis of Randomized Controlled Trials.American journal of hematology · 2025Pooled it
- Automated Deauville Score computation from baseline [¹⁸F]FDG PET/CT predicts progression-free survival in multiple myeloma: a radiogenomic framework.European journal of nuclear medicine and molecular imaging · 2026Article
- Design Characteristics and Survival Outcomes of Local Trials Supporting the Approval of Targeted Anticancer Drugs in China.Cancer medicine · 2026Article
- Clinical Benefit of Multiple Myeloma Drugs at Regulatory Approval in Brazil, Europe, and the USA: A Retrospective Cohort Study (2003-2024).Clinical drug investigation · 2026Article
- Patients', clinicians' and research's priorities on important outcomes in multiple myeloma: A mixed-methods study.British journal of haematology · 2026Article
- Real-world comparison of progression-free survival and time to next therapy in 761 patients with newly diagnosed multiple myeloma.Blood advances · 2026Article
- Bortezomib-melphalan-prednisone <I>versus</i> lenalidomide-dexamethasone in elderly patients with multiple myeloma: the Real MM phase IV trial.Haematologica · 2026Article
- Age-related disparities in treatment and outcomes for newly diagnosed multiple myeloma: a population-based study.Haematologica · 2025Article
- Article
- Identification of treatment effect modifiers and prognostic factors in newly diagnosed and relapsed or refractory multiple myeloma.Journal of comparative effectiveness research · 2025Article
- Resistance to immunomodulatory drugs in multiple myeloma: the cereblon pathway and beyond.Haematologica · 2025Review
- Evaluating organ preservation strategies versus radical surgery in T2N0 rectal cancer: survival outcomes and tumor size impact.International journal of colorectal disease · 2025Article
- Updated analysis of EMN02 demonstrated overall survival benefit to early ASCT for multiple myeloma.Blood cancer journal · 2025Article
- Busulfan and cyclophosphamide for autologous stem cell transplantation in patients with multiple myeloma after proteasome inhibitor and/or immunomodulatory drug treatment.Scientific reports · 2024Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
16 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Measurement of overall survival (OS) remains the gold standard for interpreting the impact of new therapies for multiple myeloma in phase 3 trials. However, as outcomes have improved, it is increasingly challenging to use OS as the primary endpoint if timely approval of novel agents is to be ensured to enable maximum benefit for patients. Surrogate endpoints of OS, such as progression-free survival (PFS) and response to treatment, have contributed to approval decisions by the Food and Drug Administration (FDA) and European Medicines Agency as endpoints demonstrating clinical benefit, and the FDA has recently supported the use of minimal residual disease (MRD) as an accelerated approval endpoint in multiple myeloma. This review aims to address situations in which the use of PFS as a surrogate endpoint warrants careful interpretation especially for specific subgroups of patients and considers ways to ensure that studies can be designed to account for possible discordance between PFS and OS. The utility of subgroup analyses, including the potential for those not pre-specified, to identify target populations for new agents is also discussed.
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.