Avapritinib as maintenance after chemotherapy for refractory systemic mastocytosis

Background: Diagnosis and treatment of systemic mastocytosis remain a challenge for clinicians. Disease symptoms vary, and close cooperation with other specialists is highly required. Despite the wider access to novel targeting molecules, there is still a place for conventional chemotherapy. Case report: A reported patient had advanced systemic mastocytosis, which failed the multikinase inhibitor — midostaurin, but responded promptly to 2-CdA with cytarabine. Conclusions: Avapritinib — a selective and potent KIT inhibitor was found to be effective as response maintenance.