A systematic review and meta-analysis published in the Journal of the American Academy of Dermatology suggests that avapritinib consistently improves the skin manifestations of systemic mastocytosis (SM) in individuals with both indolent and advanced forms of the rare disease.
Cutaneous (skin) involvement is common with SM, occurring in about 80% of people with the condition. It can manifest as itching, flushing, hives and other symptoms that can significantly affect quality of life. Improving these symptoms is an important treatment goal for both indolent systemic mastocytosisMastocytosis Rare disease caused by the buildup of mast cells. Cutaneous mastocytosis primarily affects the skin and is more common in children, while systemic mastocytosis affects internal organs and is more common in adults. (ISM) and advanced systemic mastocytosis (AdvSMAdvanced SM In these subtypes of SM, mast cells begin to damage organs. Advanced SM includes the subtypes aggressive SM, mast cell leukemia and SM with an associated hematological neoplasm.).
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Avapritinib is a targeted therapy designed to block the activity of KIT D816V, which is responsible for about 95% of ISM cases. The drug is approved by the U.S. Food and Drug Administration to treat both indolent and advanced forms of SM.
To evaluate the available evidence, researchers conducted a systematic review of studies published through August 2025. The review included four studies involving a total of 259 patients. Two studies enrolled individuals with ISM, while the other two focused on AdvSM. Three of the studies reported demographic data, with a median patient age of 55. Just over two-thirds of participants were women.
Across three of the included studies, pooled analysis found that 87.2% of participants experienced improvement in cutaneous symptoms after treatment with avapritinib.
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Researchers highlighted results from two clinical trials. In the EXPLORER trial, patients receiving avapritinib reported significant improvements in overall symptoms, including those related to skin involvement. The treatment was associated with a 10.9-point reduction in total symptom score.
Findings from the PIONEER trial showed that avapritinib significantly reduced the severity of skin spots, improved skin symptom scores and decreased the overall surface area of skin lesions.
“In conclusion, avapritinib demonstrated consistent efficacy with improvement in skin manifestations across both ISM and AdvSM,” the authors wrote.
