Systematic review shows alpelisib improves symptoms in patients with venous malformations, suggesting a beneficial treatment option.
The phosphoinositide 3-kinase alpha inhibitor alpelisib has been increasingly used in the treatment of venous malformations. In this systematic review of 47 patients with a molecular diagnosis for their venous malformations treated with alpelisib, a partial or complete radiological response was achieved by 77% of evaluable patients and symptomatic improvement in 98%. Adverse effects were predominantly mild, with hyperglycaemia the most frequent, occurring in 58% of patients with available safety data. TRIAL REGISTRATION: PROSPERO Registration: CRD420261339549.
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Phan et al. (2026) studied this question.
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