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Published on: April 27, 2021
Pharmacotherapy of mast cell disorders
1aDepartment of Respiratory Medicine and Allergy, Karolinska University Hospital, Huddinge bDepartment of Medicine Solna, Immunology and Allergy Unit, Karolinska Institutet and Karolinska University Hospital cMastocytosis Centre Karolinska, Karolinska University Hospital and Karolinska Institutet, Stockholm, Sweden dDepartment of Internal Medicine, Division of Allergy and Clinical Immunology, University of Michigan, Ann Arbor, Michigan, USA.
Purpose Of Review:
Mast cell disorders (MCDs) comprise mastocytosis and disorders referred to as mast cell activation syndrome and are caused by abnormal accumulation and/or activation of mast cells in tissues. Clinical signs and symptoms are protean; therefore, finding suitable treatment options for individual patients entails a challenge for clinicians. The purpose of this manuscript is to review the literature on the available therapeutic interventions in patients with MCD.
Recent Findings:
Pharmacotherapy is mainly directed against the effects of mast cells and their mediators. The current recommendations are exclusively based on expert opinions due to the lack of controlled clinical trials. The targeted therapies aiming at blocking mutant KIT variants and/or downstream signaling pathways are currently being developed and may be considered in severely affected, therapy-refractory patients.
Summary:
There is currently no method for predicting the best available approach to control symptoms in individual patients with MCD. Therefore, a stepwise, individual-based approach in pharmacotherapy options appears to be most successful strategy and is recommended in all patients. The core component of the treatment in most patients is to control symptoms caused by mast cell mediator release, whereas cytoreductive therapies are mainly reserved for patients with advanced/aggressive systemic mastocytosis.
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