Videos

In an interview with Pharmacy Times, Claudia R. Morris, MD, lead investigator and author of the phase 3 Sickle Cell Disease Treatment With Arginine Therapy (STArT) trial and a pediatric emergency medicine physician at Children’s Healthcare of Atlanta, discussed how treatment timing and patient heterogeneity may inform the design of future sickle cell pain trials.

In an interview with Pharmacy Times, Claudia R. Morris, MD, lead investigator and author of the phase 3 STArT trial and a pediatric emergency medicine physician at Children’s Healthcare of Atlanta, discussed how patient history and institutional pain-management practices may influence time to crisis resolution and how care teams, including pharmacists, can help standardize treatment.

The panelists examined dosing considerations for avapritinib, starting at 25 mg with titration to 50 mg in indolent systemic mastocytosis (SM) compared with 200 mg in advanced disease, as well as midostaurin's 100 mg twice-daily dosing in advanced SM, emphasizing patient counseling that dose adjustments reflect tolerability rather than treatment failure.

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.

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.