BMC Cancer. 2025 Aug 8;25(1):1288. doi: 10.1186/s12885-025-14724-6.
ABSTRACT
BACKGROUND: There is a need to better reflect the patient experience in multiple myeloma (MM), beyond just clinical progression, with the use of a novel endpoint that combines disease progression with patient-reported outcomes.
METHODS: A systematic literature review (SLR) identified relevant MM symptoms, which were narrowed down by experts using a Delphi panel. A post-hoc analysis of the MagnetisMM-3 trial of elranatamab assessed the impact of change in patient-reported outcomes (PROs) on the risk of disease progression or death. A composite endpoint for symptomatic progression-free survival (SPFS) combined these symptoms and PFS (PFS event plus 10-point worsening in pain, fatigue, or poor mobility within 28 days).
RESULTS: The SLR identified 16 symptoms from 34 publications. Delphi consensus was that drowsiness, fatigue, pain, and poor mobility are linked to disease progression. Three symptoms had a significant association with PFS: A 10-point worsening on the 100-point scales for pain, fatigue, and poor mobility corresponded to a 22%, 20%, and 31% increase in risk of progression, respectively. Median SPFS was not reached (95% CI 12.2- not reached).
CONCLUSIONS: SPFS is the first composite endpoint in MM that combines PFS and clinically relevant PROs. The patient-centric approach in the conception of this endpoint allows for a nuanced and comprehensive understanding of the MM patient experience.
PMID:40781281 | DOI:10.1186/s12885-025-14724-6