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Use of midostaurin in mixed phenotype acute leukemia with FLT3 mutation: A case series
Zoë Tremblay1, Anna Wong1, Anne-Sophie Otis1
1Faculty of Pharmacy, Université de Montréal, Montreal, QC, Canada.
Abstract:
Mixed phenotype acute leukemia (MPAL) is a rare type of acute leukemia where blasts present phenotypes from more than one lineage. A poor prognostic has been associated with this disease, and limited data are currently available to guide the choice of therapy. Regarding FLT3-positive MPAL, only one case treated with midostaurin has been published to date. Here, we report the successful use of midostaurin to treat three FLT3-positive MPAL T/myeloid and B/myeloid patients. Midostaurin was successfully added to intensive induction (two patients) and consolidation chemotherapy (three patients) without significant adverse events requiring a dose adjustment or discontinuation. The therapy received resulted in complete remission for two patients and complete remission with an incomplete hematologic recovery for the third. All patients proceeded to HSCT and stayed in remission after an extended follow-up respectively at 28, 31, and 11 months later. These results suggest that the addition of midostaurin during induction and consolidation therapy may represent a treatment option for FLT3-positive MPAL.
Insights
Midostaurin shows promise in treating FLT3-positive mixed phenotype acute leukemia (MPAL). This therapy, combined with chemotherapy, achieved remission in three patients, suggesting a potential new treatment option for this rare leukemia.
Area of Science:
- Hematology
- Oncology
- Molecular Biology
Background:
- Mixed phenotype acute leukemia (MPAL) is a rare hematologic malignancy with poor prognosis.
- Limited therapeutic options exist for MPAL, particularly for FLT3-mutated cases.
- FLT3 mutations are associated with aggressive disease in acute leukemias.
Observation:
- This study reports on three patients with FLT3-positive MPAL (T/myeloid and B/myeloid) treated with midostaurin.
- Midostaurin was integrated into intensive induction and consolidation chemotherapy regimens.
- No significant adverse events necessitated dose adjustment or discontinuation of midostaurin.
Findings:
- Two patients achieved complete remission, and one achieved complete remission with incomplete hematologic recovery.
- All three patients proceeded to hematopoietic stem cell transplantation (HSCT).
- Patients remained in remission at extended follow-up periods (11, 28, and 31 months).
Implications:
- Adding midostaurin to chemotherapy may be a viable treatment strategy for FLT3-positive MPAL.
- This approach warrants further investigation in clinical trials for this challenging leukemia.
- Targeting FLT3 in MPAL could improve patient outcomes and survival rates.
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