Patient-reported outcomes: important data in early trials of novel therapies

Abstract

Multiple myeloma is an incurable disease that has nonetheless been the focus of substantial treatment advances over the past decade, one of which is ciltacabtagene autoleucel (cilta-cel), a chimeric antigen receptor (CAR) T-cell therapy approved by the US Food and Drug Administration (FDA) in March, 2022, for patients with relapsed or refractory multiple myeloma who have not responded following several lines of therapy. FDA approval of cilta-cel was based on the CARTITUDE-1 phase 1b–2 trial, which reported an overall response rate of 97%, complete response rate of 67%, and median duration of response that was not reached at a median of 12·7 months of follow-up.