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Systemic mastocytosis: current status and challenges in 2024
Celalettin Ustun1, Fatma Keklik Karadag2, Michael A Linden3
1Division of Hematology, Oncology and Cellular Therapy, Department of Medicine, Rush University Medical Center, Chicago, IL.
Blood Advances
|January 24, 2025
Summary
Systemic mastocytosis (SM) management has advanced with new classifications and KIT inhibitors improving survival in advanced disease. However, optimal treatment for subsets like SM with associated hematologic neoplasm requires further research.
Area of Science:
- Hematology
- Oncology
- Rare Diseases
Background:
- Systemic mastocytosis (SM) is a rare clonal mast cell disorder.
- Recent decades brought significant advancements in SM biology, prognostication, and management.
- Current diagnostic and prognostic standards rely on WHO and International Consensus Classifications.
Purpose of the Study:
- To provide an updated overview of systemic mastocytosis.
- To cover the latest diagnostic methods and patient classification.
- To review current management strategies based on clinical trials for nonadvanced and advanced SM.
Main Methods:
- Review of recent literature and clinical trials.
- Integration of diagnostic criteria including clinical, morphological, immunophenotypic, and molecular data.
- Classification of SM into nonadvanced (nonAdvSM) and advanced (AdvSM) categories.
Main Results:
- New classifications (WHO, ICC) aid SM diagnosis and prognostication.
- KIT inhibitors like avapritinib show promise in prolonging survival for AdvSM.
- Effective treatments exist for both indolent and AdvSM, but challenges remain.
Conclusions:
- SM diagnosis requires integrated clinical and laboratory findings.
- Advancements in KIT inhibitors offer improved outcomes for AdvSM.
- Further prospective studies are needed for complex subsets like SM-AHN to define optimal treatments.
