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Avapritinib in the Treatment of Systemic Mastocytosis: an Update

  • Myeloproliferative Neoplasms (BL Stein, Section Editor)
  • Published:
Current Hematologic Malignancy Reports Aims and scope Submit manuscript

Abstract

Purpose of Review

Patients with systemic mastocytosis, a dangerous and rare myeloid neoplasm, have long had few therapies available to them and, historically, rarely achieved from significant disease control. However, research and translational developments over the last decade have led to promising new options for disease management. In this review, we briefly outline the history of treatment for systemic mastocytosis and subsequently focus on the clinical development and potential applications of avapritinib (previously known as BLU-285), a potent and selective oral inhibitor of the tyrosine kinase most commonly mutated in this condition.

Recent Findings

Phase I data and recent phase II data have demonstrated both safety and efficacy of this agent used as monotherapy, even in patients who have progressed on other targeted therapy. Studies to date have focused on patients with the most aggressive disease, but new trials in indolent mastocytosis are accruing currently.

Summary

Over the next several years, one may anticipate finalized, peer-reviewed, and formally published data for this agent in both advanced systemic and indolent mastocytosis. Evidence from these early studies will also likely highlight where more research is needed.

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  1. Department of Medicine, Medical College of Wisconsin, 9200 W. Wisconsin Ave, Milwaukee, WI, 53226, USA

    Samantha Below & Laura C. Michaelis

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Samantha Below declares no potential conflicts of interest. Laura C. Michaelis reports research funding from Jazz Pharmaceuticals and personal fees for consulting and advisory work from Celgene, Incyte Pharmaceuticals, and Curio Science.

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Below, S., Michaelis, L.C. Avapritinib in the Treatment of Systemic Mastocytosis: an Update. Curr Hematol Malig Rep 16, 464–472 (2021). https://doi.org/10.1007/s11899-021-00650-4

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