The following is a summary of “Patient-reported outcome domains in multiple myeloma randomized controlled trials and association with survival outcomes,” published in the December 2024 issue of Hematology by Shah et al.
Patient-reported outcomes (PROs) are vital in multiple myeloma (MM) randomized controlled trials (RCTs) but vary in methodology and reporting.
Researchers conducted a retrospective study on PROs in MM trials.
They reviewed 35 MM RCTs using EORTC QLQ-C30 published from 01/2014 to 06/2023 and analyzed PROs’ association with progression-free survival (PFS)/overall survival (OS) using Fisher’s exact test or Pearson’s Chi-squared test.
The results showed that 35 RCTs included PROs as secondary or exploratory endpoints, with 11 (31.4%) pre-specifying EORTC QLQ-C30 domains. The most common domains were Global health status/Quality of life (GHS/QoL) (10, 90.9%), Physical Functioning (6, 54.5%), Fatigue (6, 54.5%), and Pain (6, 54.5%). Significant differences in at least 1 domain were seen in 23/35 trials (65.7%), with improvements in GHS/QoL (12/23), Pain (11/23), Fatigue (9/23), and Physical Functioning (9/23). PROs were concordant with PFS in 19/33 trials (57.6%, P=0.398) and OS in 22/31 trials (71%, P=0.018).
They identified key PRO domains suitable as primary endpoints in MM RCTs. They found a significant association between PROs and OS, emphasizing their role in capturing treatment efficacy.
Source: link.springer.com/article/10.1007/s00277-024-06129-5
