Nab-Sirolimus Yields Long-Lasting Benefits Across PEComa Subgroups

Thomas J. Herzog, MD, Paul & Carolyn Flory professor, University of Cincinnati, deputy director, University of Cincinnati Cancer Center, Cincinnati, OH, discusses the efficacy data seen in a subgroup analysis of the phase 2 AMPECT study (NCT02494570) evaluating nab-Sirolimus (Fyarro; ABI-009) for patients with perivascular epithelioid sarcoma (PEComa).

In the study, an overall response rate (ORR) of 37.5% (95% CI, 15.2%-64.6%) was observed among 16 female patients with malignant PEComa in uterine, ovarian, pelvic, and retroperitoneal sites, regardless of TSC1/TSC2 mutation status. All of the observed responses were confirmed partial responses.

Additional findings showed that the disease control rate (DCR) in the subgroup was 62.5%, and 4 of 16 patients (25%) experienced stable disease for at least 12 weeks. The subgroup was made up of over half of the evaluable study population. Further, the ORR from this analysis was similar to the 38.7% ORR observed in the overall population evaluation.

Transcription:

0:09 | With the overall response rate being the primary end point, the end point was very similar in terms of outcome between the subgroup analysis of the retroperitoneal gynecologic tumors compared with the whole AMPECT trial. [There was a] 37.5% overall response rate with a disease control rate of 63%, and that compared very favorably to 38.7% in the overall AMPECT trial with a disease control rate of 71%.

0:40 | The other important thing to remember, and looking at the spider plot really shows that the best, is the median time to response was extremely fast, about 1.4 months, and the duration of response was a little over 36 months. It is important to remember these responses occur often rapidly, and they are quite durable.

REFERENCE

1. Herzog T, Dockery L, Dickson M, et al. Response to treatment with nab-sirolimus in patients with perivascular epithelioid cell sarcoma (PEComa) of gynecologic or peritoneal origin: subgroup analysis from AMPECT. Presented at: Society of Gynecologic Oncology 2024 Annual Meeting on Women's Cancer, March 16-18, 2024. San Diego, CA.