Background Ipilimumab was approved in 2011 by the US Food and Drug Administration in 2011 for the treatment of unresectable or metastatic (advanced) melanoma, although pivotal data using the approved 3 mg/kg monotherapy q3w × 4 were available only for patients with previously treated disease.
Objective To investigate patient and disease characteristics, survival outcomes, and safety in treatment-naïve patients receiving ipilimumab therapy.
Methods Adult patients with treatment-naïve advanced melanoma who received ≥1 dose of ipilimumab 3 mg/kg during April 2011-Sept 2012, with ≥12 months having elapsed since the start of treatment, were identified from 34 US sites. Personnel from each study site retrospectively abstracted existing data from individual patient medical records, which were collected and validated by an independent research organization.
Results In all, 273 patients were included in the study. The median age of the total study population was 64 years (range, 26-91), and 64.8% were men. At diagnosis, 56.0% were stage IV M1c, and 12.1% had brain metastases. 50 patients had a BRAF mutation, 181 were BRAF wild-type, and BRAF status was not known for 42. 78% of patients received all 4 planned doses of ipilimumab. Median survival from initiation of ipilimumab treatment was 14.5 months (95% confidence index [CI], 12.9-18.7). The overall one-year survival rate was 59.2% (95% CI, 53.0-64.8); and 71.0% and 54.9% for patients with BRAF-mutated and wild-type tumors, respectively. Adverse events of any grade, grade 3, and grade 4 occurred in 164 patients (60.1%), 45 (16.5%), and 8 (2.9%), respectively. The most common grade 3 or 4 adverse events were colitis (4.0%), fatigue (2.9%), and diarrhea (1.5%). Drug-related adverse events were primarily immune-related and occurred in 147 patients (53.8%), including grade 3/4 in 15.7% of patients (13.9% and 1.8%, respectively). No deaths were attributed to ipilimumab.
Conclusions This observational study provides real-world, clinical practice evidence supporting improved survival with the approved ipilimumab 3 mg/kg monotherapy in patients with treatment-naïve advanced melanoma, including prolonged survival in some patients. The safety profile was consistent with that reported in clinical trials.
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