The Use of Biologic Therapeutic Regimens in Cutaneous and Systemic Mastocytosis: A Scoping Review.

Abstract

Background: Mastocytoses, a category of diseases characterized by the clonal proliferation of mast cells and the release of their mediators, can be broadly classified as systemic mastocytosis (SM), cutaneous mastocytosis (CM), or mast cell sarcoma (MCS), with treatment options and prognoses varying based on subtype. Advancements in biologics and genetic testing have increased the variety of therapeutic options for mastocytoses. This paper characterizes the therapeutic landscape for mastocytoses with a focus on biologics and other novel medications.

Methods: A comprehensive literature search of PubMed and Scopus databases was conducted from the inception of the databases to January 31, 2024. Search terms included "[disease state] AND [biologic class]" or "[disease state] AND [biologic name]".

Results: Of the 672 screened articles, 55 met the following inclusion criteria: (1) diagnosis of either CM, SM, or MCS, and (2) treatment with one of the following biologic regimens: tyrosine kinase inhibitor (TKI) (including a JAK/STAT inhibitor (JAK1/2i), or a monoclonal antibody). Treatment led to successful resolution of the disease or reduction of symptoms in 90% of the patients using TKIs (n=698), including all patients (n=4) using the JAK1/2i ruxolitinib alone, and 90.6% (n=125) using monoclonal antibodies.

Conclusion: Successful treatment or symptom improvement of SM, CM, and MCS has been demonstrated through the use of JAK1/2i, TKI, and monoclonal antibodies. More studies are needed to characterize JAK1/2i regimens as clinical trials are lacking.