Gilteritinib: A Review in Relapsed or Refractory FLT3-Mutated Acute Myeloid Leukaemia

Gilteritinib (Xospata®), a next-generation tyrosine kinase inhibitor (TKI), is approved in several countries/regions worldwide for the treatment of relapsed or refractory acute myeloid leukaemia (AML) in adults with FMS-like tyrosine kinase 3 (FLT3) mutations. In this patient population, oral gilteritinib significantly improved overall survival (OS) and the response rate for complete remission with full or partial haematological recovery compared with salvage chemotherapy in the phase III ADMIRAL trial. In an integrated safety analysis of patients with relapsed or refractory AML, the most commonly reported grade ≥ 3 treatment-related adverse events (AEs) in gilteritinib recipients included anaemia, febrile neutropenia and thrombocytopenia. Clinically relevant AEs of special interest (AESIs) with gilteritinib therapy included differentiation syndrome, posterior reversible encephalopathy syndrome, QT interval prolongation and pancreatitis. AEs, including AESIs, were generally manageable with dose reduction, interruption or discontinuation. All patients of reproductive potential should use contraception during gilteritinib treatment due to the risk of embryo-foetal toxicity. Given its convenient oral regimen, along with the poor prognosis and paucity of treatment options for adults with relapsed or refractory FLT3-mutated AML, gilteritinib represents a valuable first-line targeted monotherapy in these patients.