Updated safety of midostaurin plus chemotherapy in newly diagnosed FLT3 mutation–positive acute myeloid leukemia: the RADIUS-X expanded access program. Issue 13 (9th November 2020)

• Record Type: Journal Article
• Authors: Roboz, Gail J.; Strickland, Stephen A.; Litzow, Mark R.; Dalovisio, Andrew; Perl, Alexander E.; Bonifacio, Gaetano; Haines, Kelly; Barbera, Alysha; Purkayastha, Das; Sweet, Kendra
• Is Part Of: Leukemia & lymphoma. Volume 61:Issue 13(2020)
• Page Start: 3146
• Page End: 3153
• Publication Date: 2020-11-09
• Subjects: Acute myeloid leukemia -- midostaurin -- FLT3 -- idarubicin -- maintenance; Leukemia -- Periodicals; Lymphomas -- Periodicals; 616.99419
• DOI: 10.1080/10428194.2020.1805109
• Languages: English
• ISSNs: 1042-8194

Abstract

Abstract: Approval of midostaurin, a multikinase inhibitor, in combination with chemotherapy for the treatment of adults with newly diagnosed FLT3 mutation–positive acute myeloid leukemia, was based on the phase 3 RATIFY trial results. RADIUS-X (NCT02624570) was an expanded access program providing access to midostaurin during regulatory review and extending the understanding of the safety and tolerability of midostaurin. Patients aged ≥18 years received midostaurin with 1–2 cycles of induction therapy (cytarabine plus daunorubicin or idarubicin) and ≤4 cycles of high-dose cytarabine consolidation chemotherapy or as single-agent maintenance therapy. The study enrolled 103 patients. No new safety events were observed; toxicities were not influenced by age, anthracycline choice, or coadministration of CYP3A4 inhibitors. The most common adverse events (AEs) were febrile neutropenia, nausea, and diarrhea. During maintenance, 46% of patients reported AEs. Midostaurin demonstrated a manageable safety profile and was associated with high transplant and low on-treatment relapse rates.