Modern Management of Indolent Systemic Mastocytosis: Clinical Practice and Emerging Evidence

Introduction to ISM with emphasis on evolving clinical perception, disease heterogeneity, and persistent unmet needs despite conventional symptomatic therapy.

Overview of KIT D816V–driven disease biology in ISM and practical approaches to longitudinal symptom monitoring, including emerging use of the Mastocytosis Control Test.

Role of patient-reported outcomes in trials and practice, criteria for escalation beyond supportive care, and the paradigm shift introduced by selective KIT D816V inhibition, including avapritinib.

Shared decision-making around initiating targeted therapy, key safety considerations for avapritinib, and core efficacy and safety findings from the 6‑month randomized phase of the PIONEER trial.

Use of symptom and quality-of-life tools alongside biomarkers in PIONEER, principles of dose titration from 25 mg to 50 mg, and practical recommendations for longitudinal safety monitoring.

Future directions in ISM, including evolving treatment goals, unanswered questions about osteoporosis, anaphylaxis, resistance, curative strategies, and the need to extend advances to pediatric populations.