Midostaurin is NOT Used for Acute Lymphoblastic Leukemia (ALL)
Midostaurin is specifically approved and indicated for FLT3-mutated Acute Myeloid Leukemia (AML), not for Acute Lymphoblastic Leukemia (ALL). These are distinct hematologic malignancies with different molecular profiles and treatment paradigms.
Approved Indication for Midostaurin
Midostaurin is FDA and EMA-approved exclusively for newly diagnosed FLT3-mutated AML in combination with standard induction chemotherapy (cytarabine and anthracycline), not for ALL. 1, 2, 3
The drug targets FLT3 mutations (both ITD and TKD variants) that occur in approximately 30% of AML patients but are not characteristic of ALL. 2, 4
Evidence Base in AML (Not ALL)
The RATIFY trial, which established midostaurin's efficacy, enrolled patients aged 18-59 years with newly diagnosed FLT3-mutated AML, demonstrating median overall survival of 74.7 months versus 25.6 months with placebo (HR 0.78, P=0.009). 2, 4
All clinical guidelines and evidence provided reference midostaurin's use in AML specifically, with no mention of ALL as an indication. 5
Critical Distinction Between AML and ALL
AML and ALL are fundamentally different diseases: AML arises from myeloid precursors and commonly harbors FLT3 mutations, while ALL arises from lymphoid precursors with different molecular drivers (such as BCR-ABL in Philadelphia chromosome-positive ALL). 5
Treatment paradigms differ completely: ALL typically requires different chemotherapy backbones and targeted agents (such as tyrosine kinase inhibitors for BCR-ABL-positive ALL, not FLT3 inhibitors). 5
Common Pitfall to Avoid
Do not confuse AML with ALL based on the shared term "acute leukemia." The distinction is critical for appropriate treatment selection, as using midostaurin in ALL would be off-label and unsupported by evidence. 5, 1
If a patient has ALL, appropriate targeted therapy would depend on the specific molecular subtype (e.g., TKI maintenance for BCR-ABL-positive ALL is recommended, not midostaurin). 5