Evidence map›Paper›PMID 39644334›Full record

SynthesisAnnals of hematology2024

Patient-reported outcome domains in multiple myeloma randomized controlled trials and association with survival outcomes.

Darshi Shah, Francesco Sparano, Catherine Luo, Daniela Krepper, Johannes M Giesinger, Thomas Baldi, Eilidh Duncan, Madeline Pe, Rajshekhar Chakraborty, Fabio Efficace

Abstract readSystematic Review
PubMed Publisher
In one paragraph

Synthesis in Annals of hematology, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing papers in PubMed
–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

3 citing papers in PubMed.

  1. Article
  2. Article
  3. Review
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

10 authors.

Darshi ShahRenaissance School of Medicine at Stony, Brook University, Stony Brook, NY, 11790, USA.
Francesco SparanoItalian Group for Adult Hematologic Diseases (GIMEMA), Data Center and Health Outcomes Research Unit, Rome, Italy.
Catherine LuoRenaissance School of Medicine at Stony, Brook University, Stony Brook, NY, 11790, USA.
Daniela KrepperUniversity Hospital of Psychiatry II, Medical University of Innsbruck, Innsbruck, Austria.
Johannes M GiesingerUniversity Hospital of Psychiatry II, Medical University of Innsbruck, Innsbruck, Austria.
Thomas BaldiItalian Group for Adult Hematologic Diseases (GIMEMA), Data Center and Health Outcomes Research Unit, Rome, Italy.
Eilidh DuncanMyeloma Patients Europe (MPE), Brussels, Belgium.
Madeline PeQuality of Life Department, European Organization for Research and Treatment of Cancer (EORTC) Headquarters, Brussels, Belgium.
Rajshekhar Chakraborty *Multiple Myeloma and Amyloidosis Program, Columbia University Irving Medical Center, New York, NY, USA. rc3360@cumc.columbia.edu.
Fabio Efficace *Italian Group for Adult Hematologic Diseases (GIMEMA), Data Center and Health Outcomes Research Unit, Rome, Italy.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Patient-reported outcomes (PROs) are crucial endpoints in multiple myeloma (MM) randomized controlled trials (RCTs), yet there is significant variability in their methodology and reporting. Our study aimed to (a) identify the most commonly pre-specified PRO domains in MM RCTs and those most responsive to modern therapies, and (b) examine the association between PROs and progression-free survival (PFS)/overall survival (OS). We performed a systematic review of MM RCTs that used EORTC QLQ-C30 and published between 01/2014-06/2023. The association between PFS/OS and PRO was explored using Fisher's exact test or Pearson's Chi-squared test. Thirty-five RCTs were identified, with PROs as secondary or exploratory endpoints in all studies. About one-third of RCTs (n=11, 31.4%) pre-specified at least one EORTC QLQ-C30 domain, with the most common domains being Global health status/Quality of life (GHS/QoL) (n = 10, 90.9%), Physical Functioning (n = 6, 54.5%), Fatigue (n = 6, 54.5%), and Pain (n = 6, 54.5%). A statistically significant and/or clinically meaningful difference in at least one EORTC QLQ-C30 domain between arms was seen in 23/35 trials (65.7%), with the most common domains showing improvement being GHS/QoL (12/23 trials), Pain (11/23 trials), Fatigue (9/23 trials), and Physical Functioning (9/23 trials). PRO was noted to be concordant with PFS in 19/33 (57.6%) trials (p = 0.398), and with OS in 22/31 (71%) trials (p = 0.018). Our study identified key PRO domains that can be potentially used as primary endpoint in MM RCTs. Additionally, significant association between PROs and OS highlight the importance of integrating PROs to better capture treatment efficacy.

Indexed as

Multiple MyelomaPatient Reported Outcome MeasuresQuality of LifeRandomized Controlled Trials as TopicHumansProgression-Free SurvivalSurvival RateMultiple MyelomaPatient-Reported OutcomesRandomized Controlled Trials

Identifiers

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Registered trials

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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.