Alpelisib for TEK- and PIK3CA-Related Venous Malformations: A Systematic Review.
Julie Phan, Christopher Tran, Shweta Srinivasa et al.
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In 30 seconds
This systematic review examined alpelisib treatment in 47 patients with molecularly diagnosed TEK- or PIK3CA-related venous malformations. Among evaluable patients, 77% had a partial or complete radiological response, and 98% experienced symptomatic improvement. Reported adverse effects were predominantly mild; hyperglycaemia was the most frequent, occurring in 58% of patients with available safety data. These findings describe outcomes reported across the reviewed patients and do not establish comparative effectiveness.
Key findings
- A partial or complete radiological response was reported in 77% of evaluable patients.
- Symptomatic improvement was reported in 98% of patients.
- Hyperglycaemia occurred in 58% of patients with available safety data; adverse effects were predominantly mild.
Why it matters
The review brings together reported clinical and radiological outcomes for alpelisib in venous malformations with molecular diagnoses. The high proportions with symptom improvement and radiological response, alongside reported adverse effects, may help inform clinical discussions about this targeted therapy while highlighting the need to consider safety monitoring.
What to keep in mind
The abstract does not report a comparator or provide details on study designs, follow-up, or the number of patients evaluable for each outcome; the findings therefore do not establish comparative effectiveness.