Official websites use. Share sensitive information only on official, secure websites. RP is part of the advisory board of GSK. All authors read and approved the final version of this manuscript. The original contributions presented in the study are included in the article and its Online Supplementary Appendix. Further inquiries can be directed to the corresponding author. This article is distributed under the terms of the Creative Commons Attribution Noncommercial License by-nc 4. We sought to assess the efficacy and safety of BM in a real-world setting in patients who benefited from an early access program.
We conducted an observational, retrospective, multicenter study. Eligibility criteria were treatment of relapsed or refractory multiple myeloma RRMM in monotherapy in adult patients who have received at least three lines of therapy previously, including at least one immunomodulatory agent IMiD , a proteasome inhibitor PI and an anti-CD38 monoclonal antibody, and whose disease progressed during the last treatment period.
The primary end-point of the study is to assess the overall survival OS. Between November and December , patients were treated with BM; 97 were eligible for the efficacy evaluation and for safety. The median age was 66 range, 37β82 years. High-risk cytogenetics were identified in Fifty-five The median number of prior lines of treatment was five range, 3β The median number of BM cycles administered was three range, 1β The overall response rate at best response was The median OS was 9.
The median duration of response was 9 months range, 4. Treatment was delayed for 55 The occurrence of keratopathy was Overall, our data are concordant with the results from DREAMM-2 in terms of efficacy and safety on a non-biased population. Effective and safe novel therapies are needed for the treatment of patients with relapsed or refractory multiple myeloma RRMM. The course of the disease consists in multiple relapses, as MM is still not curable.
Even though the range of options has widened in recent years, especially with the emergence of immunotherapy, RRMM still has a poor prognosis, and, therefore, new drugs with innovative mechanisms are necessary. After a median follow-up of 6 months, the median progression-free survival PFS was 2. Regarding tolerance, the main toxicity was corneal toxicity, with one quarter of patients exhibiting grade 3 or 4 disorders.
