As reported in the Journal of Clinical Oncology by Wagner et al, final analysis of the phase II AMPECT trial has shown a median duration of response of more than 3 years in patients with advanced malignant perivascular epithelioid cell tumors treated with nab-sirolimus.
The primary analysis of the study supported the November 2021 approval of nab-sirolimus in this setting. At the time of the primary analysis, median duration of response had not been reached.
Study Details
In the study, 31 evaluable patients received nab-sirolimus at 100 mg/m2 on days 1 and 8 in 21-day cycles until disease progression or unacceptable toxicity. The current analysis reports findings at 3 years after the primary analysis.
Key Findings
The mean treatment duration for all patients was 14.5 months (range = 0.3–65.2 months), with three patients still receiving nab-sirolimus at final analysis.
At study completion, confirmed objective response on independent radiologist review was observed in 12 patients (38.7%, 95% confidence interval [CI] = 21.8%–57.8%). Median duration of response was 39.7 months (95% CI = 6.5 months to not reached).
Median progression-free survival remained the same as in the primary analysis, at 10.6 months (95% CI = 5.5–41.2 months). Median overall survival was 53.1 months (95% CI = 22.2 months to not reached).
Overall, the most common treatment-related adverse events of any grade were stomatitis (82.4%), fatigue (61.8%), and rash (61.8%). No new or unexpected adverse events and no grade ≥ 4 treatment-related adverse events were reported.
The investigators concluded, “These results highlight the long-term clinical benefit of nab-sirolimus in patients with advanced malignant perivascular epithelioid cell tumors, with a duration of response of > 3 years.”
Andrew J. Wagner, MD, PhD, of Dana-Farber Cancer Institute and Harvard Medical School, is the corresponding author for the Journal of Clinical Oncology article.
Disclosure: The study was supported by Aadi Bioscience and by a U.S. Food and Drug Administration Office of Orphan Products Development grant. For full disclosures of the study authors, visit ascopubs.org.
