Efficacy and safety of ipilimumab in metastatic melanoma patients surviving more than 2 years following treatment in a phase III trial (MDX010-20).
BACKGROUND
In a phase III trial (ClinicalTrials.gov registration ID: NCT00094653), ipilimumab significantly improved survival versus a vaccine control in pretreated patients with metastatic melanoma. Here, we characterize outcomes of those patients who survived ≥ 2 years.


METHODS
Patients were randomized (3 : 1 : 1) to receive ipilimumab 3 mg/kg + gp100 vaccine, ipilimumab 3 mg/kg + placebo, or gp100 vaccine alone. Baseline demographic data, duration of survival, responses, and safety among patients with ≥ 2 years' survival were analyzed.


RESULTS
Among 676 randomized patients, 474 and 259 patients had at least 2 or 3 years of potential follow-up, respectively, and were eligible for analysis. Among these, 94 (20%) and 42 (16%) survived ≥ 2 and ≥ 3 years, respectively. Survival rates at 2 and 3 years were 25% (24 of 95) and 25% (13 of 53) with ipilimumab alone and 19% (54 of 284) and 15% (24 of 156) with ipilimumab plus gp100. Safety among patients with ≥ 2 years' survival was comparable with the overall study population, with the onset of new ipilimumab-related toxic effect (all grades) reported in 6 of 78 (8%) patients.


CONCLUSIONS
Ipilimumab results in survival of ≥ 2 years in one-fifth of pretreated patients with 2 years potential follow-up in a phase III trial. New onset, low-grade events starting after administration of the last dose were infrequent.


BACKGROUND
NCT00094653.
Division of Hematology/Oncology, Beth Israel Deaconess Medical Center, Boston, USA. Electronic address: dmcdermo@bidmc.harvard.edu.
D
D
McDermott
Department of Immunology, Netherlands Cancer Institute, Amsterdam, The Netherlands.
J
J
Haanen
Oncology Biostatistics, Bristol-Myers Squibb, Wallingford, USA.
T-T
TT
Chen
Department of Medical Oncology, Christie Hospital NHS Trust, Manchester, UK.
P
P
Lorigan
Department of Medical Oncology, Los Angeles Skin Cancer Institute, The Beverly Hills Cancer Center, Beverly Hills, USA.
S
S
O'Day
D000911:Antibodies, Monoclonal / Q000009:adverse effects / Q000627:therapeutic use*; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000009:adverse effects / Q000627:therapeutic use; D060908:CTLA-4 Antigen / Q000276:immunology; D019496:Cancer Vaccines / Q000627:therapeutic use; D005260:Female; D006801:Humans; D000074324:Ipilimumab; D008297:Male; D008545:Melanoma / Q000188:drug therapy* / Q000401:mortality* / Q000556:secondary; D008875:Middle Aged; D009362:Neoplasm Metastasis; D012871:Skin Diseases / Q000188:drug therapy* / Q000401:mortality* / Q000473:pathology; D013534:Survival; D015996:Survival Rate; D016896:Treatment Outcome; D058951:gp100 Melanoma Antigen / Q000008:administration & dosage / Q000276:immunology / Q000627:therapeutic use*
D000911:Antibodies, Monoclonal; D060908:CTLA-4 Antigen; D019496:Cancer Vaccines; D000074324:Ipilimumab; D058951:gp100 Melanoma Antigen
ipilimumab; long-term survivors; melanoma; safety; survival analysis