Daratumumab monotherapy for relapsed AL amyloidosis: a single center retrospective analysis.

Abstract

PURPOSE: Treatment of relapsed AL amyloidosis remains challenging as patients frequently suffer from severe organ dysfunctions. Daratumumab, a cornerstone drug in multiple myeloma and newly diagnosed AL amyloidosis, has also shown promise in relapsed AL amyloidosis. MATERIALS AND METHODS: To assess the efficacy of daratumumab, we conducted a retrospective study of 40 patients who received daratumumab monotherapy in our prospective amyloidosis cohort. RESULTS: The median age of the cohort was 67&#xa0;years, and 31 patients were classified as stage III/IV by the Mayo 2012 guidelines and 32 patients as stage IIIa/IIIb by the European staging system. The overall hematologic and cardiac response rates were 72.5% and 42.4%, respectively. With median follow-up duration of 26.9&#xa0;months, the estimated median progression-free survival was 21.9&#xa0;months. Deep hematologic (p&#x2009;<&#x2009;0.001) and cardiac response (p&#x2009;=&#x2009;0.001) were associated with prolonged progression free survival. The duration of hematologic/cardiac responses were 26.2&#xa0;months and 27.8&#xa0;months, respectively. The median overall survival was 30.9&#xa0;months. Treatment was well tolerated, as with 3 patients experiencing grade 3-4 hematologic adverse events and 8 experiencing grade 3-4 non-hematologic adverse events. DISCUSSION: Our findings suggest that daratumumab monotherapy provides rapid and durable responses and is generally well tolerated in patients with relapsed AL amyloidosis.