Abstract
BACKGROUND:
Ipilimumab is a monoclonal antibody that antagonizes cytotoxic T lymphocyte antigen‐4, a negative regulator of the immune system. The authors report on advanced refractory melanoma patients treated in a compassionate use trial of ipilimumab at the Memorial Sloan‐Kettering Cancer Center.
METHODS:
Patients with advanced refractory melanoma were treated in a compassionate use trial with ipilimumab 10 mg/kg every 3 weeks for 4 doses. Those with evidence of clinical benefit at Week 24 (complete response [CR], partial response [PR], or stable disease [SD]) then received ipilimumab every 12 weeks.
RESULTS:
A total of 53 patients were enrolled, with 51 evaluable. Grade 3/4 immune‐related adverse events were noted in 29% of patients, with the most common immune‐related adverse events being pruritus (43%), rash (37%), and diarrhea (33%). On the basis of immune‐related response criteria, the response rate (CR + PR) was 12% (95% confidence interval [CI], 5%‐25%), whereas 29% had SD (95% CI, 18%‐44%). The median progression‐free survival was 2.6 months (95% CI, 2.3‐5.2 months), whereas the median overall survival (OS) was 7.2 months (95% CI, 4.0‐13.3 months). Patients with an absolute lymphocyte count (ALC) ≥1000/μL after 2 ipilimumab treatments (Week 7) had a significantly improved clinical benefit rate (51% vs 0%; P = .01) and median OS (11.9 vs 1.4 months; P < .001) compared with those with an ALC <1000/μL.
CONCLUSIONS:
The results confirm that ipilimumab is clinically active in patients with advanced refractory melanoma. The ALC after 2 ipilimumab treatments appears to correlate with clinical benefit and OS, and should be prospectively validated. Cancer 2010. © 2010 American Cancer Society.