Vol 12, No 2 (2021)
Case report
Published online: 2021-11-30

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The use of midostaurin in aggressive systemic mastocytosis (ASM) with the c-KIT D816V mutation within The Emergency Access to Drug Technologies Programme

Alan Majeranowski12, Andrzej Mital2
Hematology in Clinical Practice 2021;12(2):84-88.


Aggressive systemic mastocytosis (ASM) is a very rare subtype of systemic mastocytosis (SM) and, together with systemic mastocytosis with an associated hematologic neoplasm (SM-AHN) and mast cell leukemia (MCL), it belongs to advanced systemic mastocytosis (AdvSM). Different strategies of systemic treatment are used in patients with AdvSM. The protocols base on the cladribine, interferon, allogeneic hematopoietic stem cell transplantation, polychemotherapy or the midostaurin — the only approved drug therapy in AdvSM. Options available in Poland — cladribine and interferon — are characterized by a non-lasting effect and, unfortunately, midostaurin still remains unreimbursed for patients. On October 5, 2018, The Polish Agency for Health Technology Assessment and Tariff System approved the midostaurin to be financed within The Emergency Access to Drug Technologies Programme in ASM with the c-KIT D816V mutation. Based on a case report, the following manuscript describes authors’ experiences with the use of midostaurin. The aim of the article is to present an overview of the current status of midostaurin in the treatment of ASM and to summarize the results of clinical trials, focusing on its effectiveness and safety. Authors, hereby, report a case of a patient diagnosed in 2000 with cutaneous mastocytosis (CM), that developed a transformation to ASM in 2015. The following lines of treatment were used: cladribine, pegylated interferon alpha, dasatinib. Significant side effects were observed after each line, without obtaining a lasting response to treatment. In July 2020, patient began a therapy with midostaurin. Afterwards, a normalization of peripheral blood count parameters together with reduction of serum tryptase level were observed, without noticing any adverse effects.

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Hematology in Clinical Practice