- Dual therapy with ipilimumab and nivolumab was associated with a 42% overall response rate (ORR) among patients with high-grade neuroendocrine cancer.
Why this matters
- Current treatment options for high-grade neuroendocrine carcinomas are limited to aggressive chemotherapy regimens.
- 33 patients with rare neuroendocrine tumors enrolled in the multicohort, prospective, open-label, multicenter phase 2 clinical trial, Dual Anti-CTLA-4 and Anti-PD-1 Blockade in Rare Tumors (DART).
- 18 patients (56%) had high-grade disease, and the most common tumor sites were gastrointestinal (47%; n=15) and lung (19%; n=6). Pancreatic neuroendocrine tumors were studied separately.
- Patients had received a median of 2 prior lines of therapy.
- Patients received ipilimumab (1 mg every 6 weeks) and nivolumab (240 mg every 22 weeks).
- Funding: NIH, Bristol-Myers Squibb.
- ORR was 24% (1 complete response [CR], 7 partial responses [PR]).
- All responses occurred among patients with high-grade disease (ORR, 42%; CR, 5%; PR, 37%).
- 6-month PFS was 30%; median OS was 11 months.
- Most common toxicities included fatigue (30%) and nausea (27%).
- Most common immune-related adverse event was grade 3-4 abnormal liver function; no grade 5 events observed.
- No central pathology review.
- High-grade vs low/intermediate-grade analysis not prespecified.
- Small, single-arm, nonrandomized cohort.