Evidence map›Paper›PMID 41085804›Full record

SynthesisJournal of patient-reported outcomes2025

Joint modeling of progression-free survival and patient-reported outcomes to evaluate the association between disease progression and symptoms among patients with relapsed/refractory multiple myeloma.

Stefan Knop, Hermann Einsele, Devender Dhanda, Thomas S Marshall, Laurie Eliason, Dylan McLoone, Clyde Caisip, Jenny M H Chen, Doris Boehm, Amol D Dhamane and 3 more

Abstract readMeta-Analysis
In one paragraph

Synthesis in Journal of patient-reported outcomes, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed, 1 pooled it
–field-weighted citation impact
1 · What the graph read from 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.

2 · The registry

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.

3 · Its place in the literature

Who cites it

1 citing paper in PubMed, 1 synthesis or guideline pooled it.

  1. Pooled it
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

13 authors.

Stefan KnopDepartment of Hematology and Oncology, Nuremberg General Hospital, Paracelsus Medical School, Nuremberg, Germany.
Hermann EinseleDepartment of Internal Medicine II, Würzburg University Hospital, Würzburg, Germany.
Devender DhandaBristol Myers Squibb, Lawrenceville, NJ, USA.
Thomas S MarshallBristol Myers Squibb, Lawrenceville, NJ, USA.
Laurie EliasonBristol Myers Squibb, Lawrenceville, NJ, USA.
Dylan McLoonePrecision AQ, Vancouver, BC, Canada.
Clyde CaisipPrecision AQ, Vancouver, BC, Canada.
Jenny M H ChenPrecision AQ, Vancouver, BC, Canada.
Doris BoehmBristol Myers Squibb, Munich, Germany.
Amol D DhamaneBristol Myers Squibb, Lawrenceville, NJ, USA.
Karthik RamasamyOxford University & Oxford University Hospitals NHS Trust, Oxford, Oxfordshire, UK.
Shannon CopePrecision AQ, Vancouver, BC, Canada. shannon.cope@precisionaq.com.ORCID http://orcid.org/0000-0002-1721-9485
Kevin TowlePrecision AQ, Vancouver, BC, Canada.

Funding

Bristol-Myers Squibb Bristol-Myers Squibb
6 · The paper itself

Abstract

backgroundHere we aim to evaluate the relationship between progression-free survival (PFS) and patient-reported symptoms (measured by health-related quality of life scores) among patients with relapsed/refractory multiple myeloma (RRMM). METHODOLOGY: Pain and fatigue were identified as the most common patient-relevant symptoms within RRMM based on a predefined literature review of patient preference/qualitative studies (confirmed by clinical experts). Consequently, the European Organisation for Research and Treatment of Cancer QLQ-C30 pain, QLQ-MY20 disease symptoms (pain in different locations), and QLQ-C30 fatigue domains were selected. Change from baseline scores per symptom domain was jointly modeled with PFS assuming a current slope association structure. For each symptom, we evaluated trial-specific joint models based on individual patient data from 7 RRMM clinical trials. The association between symptoms and PFS was summarized via association-effect hazard ratios (HRs) from the joint models, where a HR > 1 indicates that symptom worsening was associated with an increased hazard of a progression/death event. Meta-analyses were performed to synthesize the joint model HRs from all trials into one summary statistic (meta-HR) per symptom domain.

resultsAcross trials, worsening in pain and fatigue was associated with an increased hazard of progression events (disease progression/death) based on joint-model-association-effect HRs. Specifically, meta-HRs (95% CI) were 1.10 (1.02, 1.19) for QLQ-C30 pain, 1.10 (1.01, 1.20) for QLQ-MY20 disease symptoms, and 1.11 (1.05, 1.17) for QLQ-C30 fatigue.

conclusionsThis study demonstrated that worsening in pain and fatigue was consistently associated with an increased hazard of disease progression or death events across numerous RRMM clinical trials with varying disease severity. This suggests risk of PFS events may align with patient experience in terms of worsening symptom burden.

Indexed as

Multiple MyelomaPatient Reported Outcome MeasuresQuality of LifeDisease ProgressionFatigueHumansMaleProgression-Free SurvivalSymptom BurdenHealth-related quality of lifeJoint modelsPatient-reported symptomsRelapsed/refractory multiple myeloma

Identifiers

PMID41085804
PMCPMC12521705

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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.