Videos

Zahra Mahmoudjafari, PharmD, BCOP, FHOPA, MBA, challenges the assumption that oral agents (belumosudil) are inherently more convenient than intravenous therapy (axatilimab) by highlighting that the intravenous schedule creates structured monitoring touchpoints and that mechanism differences—not route alone—should drive sequencing decisions in heavily pretreated populations.

Brooke Adams, PharmD, BCOP, explores how patient narratives on axatilimab inform treatment decisions, moving beyond National Institutes of Health response criteria to functional outcomes—return to work or family activities, reduction in pain or skin discomfort, improved sleep—and how these improvements may exceed what clinical response rates alone predict.

Zahra Mahmoudjafari, PharmD, BCOP, FHOPA, MBA, translates the CSF1R-targeted mechanism into practical pharmacist workflows: distinct toxicity profiles (periorbital edema, hepatic enzyme elevations), drug interaction review for polypharmacy, and how to communicate the mechanistic rationale to patients and community pharmacy teams.

Led by the moderator, the panelists discussed the rationale for targeting the KIT D816V mutation with tyrosine kinase inhibitors and reviewed six-month and three-year PIONEER trial data showing sustained reductions in symptom burden and improved quality of life with avapritinib in indolent systemic mastocytosis, with minimal treatment discontinuation due to adverse events.

The panelists examined how treatment goals differ between indolent and advanced systemic mastocytosis (SM) — the former focused on symptom control and quality of life, the latter also targeting disease modification to reduce mast cell burden and organ damage — and outlined the pharmacist's role in acute anaphylaxis management, including patient education on epinephrine use and antihistamine dosing.

Led by the moderator, the expert pharmacists examined the broad differential diagnosis for systemic mastocytosis (SM), including non-clonal mast cell activation syndrome, neuroendocrine tumors, myeloid neoplasms, and cutaneous mastocytosis, alongside the evolving landscape of KIT D816V mutation testing — from allele-specific PCR and digital droplet PCR to next-generation and Sanger sequencing — each with distinct sensitivity trade-offs.