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Albumin-bound sirolimus shrank tumours in about four in ten patients with malignant PEComa, with responses lasting years and best results in tumours with TSC2 mutations, and became the first approved treatment for the disease.
Phase 2 study of 34 patients with malignant PEComa treated with intravenous nab-sirolimus weekly for two of every three weeks.
Objective response by independent review was 39 percent with a median duration of response not reached (over 2.5 years), median progression-free survival 10.6 months; 89 percent of TSC2-mutant tumours responded. Stomatitis, myelosuppression and hyperglycaemia were manageable.
Nab-sirolimus is the approved first-line treatment for advanced malignant PEComa, and TSC1/TSC2 testing helps predict response.
Shares Andrew J. Wagner, Perivascular epithelioid cell tumour (PEComa), Journal of Clinical Oncology.
Shares AMPECT, Sirolimus protein-bound particles.
Shares Sirolimus protein-bound particles, Perivascular epithelioid cell tumour (PEComa).
Shares AMPECT, Sirolimus protein-bound particles, Perivascular epithelioid cell tumour (PEComa).
Shares Sirolimus protein-bound particles, Perivascular epithelioid cell tumour (PEComa).