Treating Advanced Systemic Mastocytosis: Current Options and Emerging Therapies

This episode, titled "Treating Advanced Systemic Mastocytosis: Current Options and Emerging Therapies," featured panelists discussing the following critical questions:

What are the treatment options for patients with advanced systemic mastocytosis?

What is the clinical data to support the use of tyrosine kinase inhibitors in advanced systemic mastocytosis?

Are there any investigational therapies for systemic mastocytosis, both indolent and advanced, that you are excited about?

Led by the moderator, the expert pharmacists examined treatment options for advanced systemic mastocytosis (SM), including higher-dose avapritinib, midostaurin for patients without or with unknown KIT mutation status, and cytoreductive agents such as cladribine and interferon for those not responding to or tolerating targeted therapy. They reviewed supporting clinical data, noting midostaurin's open-label trial results, including a 60% overall response rate and median overall survival of 28.7 months, alongside the EXPLORER and PATHFINDER trials of avapritinib, which showed response rates of 75% but carried a notable risk of intracranial hemorrhage in patients with low platelet counts, cautioning against direct cross-trial comparisons given differing response criteria. The panelists closed by highlighting an active investigational pipeline, including more selective KIT inhibitors, targeted monoclonal antibodies, and early CAR T-cell therapy research for advanced SM.

Throughout the conversation, the experts provided a comprehensive reflection on the field and the factors that may shape how clinicians approach care moving forward.

In the next episode, "Dosing, Monitoring, and Counseling: The Pharmacist's Role in TKI Therapy for Systemic Mastocytosis," panelists continue their discussion on systemic mastocytosis and highlight dosing considerations for avapritinib and midostaurin across indolent and advanced disease, key adverse event monitoring and drug interaction management, and essential patient counseling points for those starting tyrosine kinase inhibitor therapy.