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FLT3 inhibitors in the treatment of AML

Approximately 10-15% of pediatric patients with acute myeloid leukemia (AML) have mutations in a the FLT3 gene, conferring a poor prognosis. In addition, approximately 80% of infants with acute lymphoblastic leukemia (ALL) have gene rearrangements in the KMT2A gene (KMT2A‐R ALL), often in combination with overexpression of FLT3, which is associated with particularly poor long‐term outcomes. In the Princess Máxima Center we investigate whether inhibition of FLT3 is an effective treatment strategy.
Preclinical and clinical work suggests that targeted inhibition of FLT3 may be a promising strategy for pediatric acute leukemias. This study was the first to evaluate the safety and efficacy of oral midostaurin in children. Single‐agent midostaurin showed limited clinical activity in these heavily pretreated children. Nevertheless, because midostaurin plus chemotherapy demonstrated clinical benefit in adults with FLT3‐mutated AML, and was authorized for use in adult AML, midostaurin will be evaluated in combination with chemotherapy in pediatric patients with FLT3‐mutated AML. This study is currently ongoing. Of interest, more specific FLT3-inhibitors (for example quizartinib) are currently available for testing in children with FLT3-mutated AML, including in the Princess Máxima Center.