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Midostaurin
Source: Childhood Acute Myeloid Leukemia Treatment (PDQ®)–Health Professional Version, National Cancer Institute.
Source updated: April 15, 2025 · Captured 2026-09-09.
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There is limited experience with midostaurin in pediatric patients with AML.
A phase I/II dose-escalation, single-agent trial in 22 children with refractory or relapsed AML (9 with FLT3 variants) was reported. Seven patients received the initial dose level of 30 mg/m2 given twice daily, and 15 patients received the higher dose level of 60 mg/m2 twice daily, with a median dose duration of 16 days.[30]
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A phase I/II dose-escalation, single-agent trial in 22 children with refractory or relapsed AML (9 with FLT3 variants) was reported. Seven patients received the initial dose level of 30 mg/m2 given twice daily, and 15 patients received the higher dose level of 60 mg/m2 twice daily, with a median dose duration of 16 days.[30]
In patients with AML and FLT3 variants, 55.5% (21.2%–86.3%) had some clinical response at a median time of 14 days (range, 8–22 days), with one patient achieving a CR with incomplete count recovery who was able to proceed to HSCT; this patient was the only long-term survivor in this study.
A phase I/II dose-escalation, single-agent trial in 22 children with refractory or relapsed AML (9 with FLT3 variants) was reported. Seven patients received the initial dose level of 30 mg/m2 given twice daily, and 15 patients received the higher dose level of 60 mg/m2 twice daily, with a median dose duration of 16 days.[30]
Overall, 72.7% of patients experienced treatment-related adverse events, with only one patient experiencing a dose-limiting toxicity (grades 3–4 alanine transaminase elevation).
A phase II trial is under way in Europe, beginning with the 30 mg/m2 twice-daily dosing (NCT03591510).
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