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Case report: A case of mast cell leukemia treated with avapritinib: from diagnostic challenge to transplantation

Title: Case report: A case of mast cell leukemia treated with avapritinib: from diagnostic challenge to transplantation
Authors: Faraci, Danilo Giuseppe; Caravelli, Daniela; Schianca, Fabrizio Carnevale; Zaniol, Elena; Enrico, Fiorenza; Boffano, Carlo; Labate, Carmelo; Sterpi, Greta; Fornari, Alessandro; Sangiorgio, Valentina; Crisà, Elena
Source: Frontiers in Oncology ; volume 16 ; ISSN 2234-943X
Publisher Information: Frontiers Media SA
Publication Year: 2026
Collection: Frontiers (Publisher - via CrossRef)
Description: Systemic mastocytosis (SM) is a rare hematological neoplasm characterized by the proliferation and accumulation of clonal mast cells (MCs) in the skin, bone marrow, and other extracutaneous organs. Mast cell leukemia (MCL) is the rarest and more aggressive subtype of SM, with very poor prognosis and abysmal survival. Treatment strategies are poorly defined and non-standardized, ranging from symptomatic management to KIT-targeted tyrosine kinase inhibitors and, in selected cases, allogeneic stem cell transplantation, currently the only potentially curative chance for MCL patients. Here, we report a case of a 66-year-old woman affected by MCL, highlighting clinical features, diagnostic challenges and complexity, and our treatment choices. Furthermore, we review current literature data and emerging therapeutic approaches, emphasizing the importance of greater clinical awareness and comprehensive multidisciplinary approach to correctly diagnose and treat this condition in order to improve patient outcomes in this challenging disease.
Document Type: article in journal/newspaper
Language: unknown
DOI: 10.3389/fonc.2026.1746589
DOI: 10.3389/fonc.2026.1746589/full
Availability: https://doi.org/10.3389/fonc.2026.1746589; https://www.frontiersin.org/articles/10.3389/fonc.2026.1746589/full
Rights: https://creativecommons.org/licenses/by/4.0/
Accession Number: edsbas.3BB975A2
Database: BASE