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.
Original language | English (US) |
---|---|
Pages (from-to) | 3146-3153 |
Number of pages | 8 |
Journal | Leukemia and Lymphoma |
Volume | 61 |
Issue number | 13 |
DOIs | |
State | Published - 2020 |
Keywords
- Acute myeloid leukemia
- FLT3
- idarubicin
- maintenance
- midostaurin
ASJC Scopus subject areas
- Hematology
- Oncology
- Cancer Research