Efficacy and safety of ipilimumab in patients with pre-treated, uveal melanoma.
BACKGROUND
Patients with advanced uveal melanoma have a poor prognosis and limited treatment options. Ipilimumab is approved for pre-treated adult patients with advanced melanoma. However, because previous clinical trials with ipilimumab have excluded patients with uveal melanoma, data in this patient population are limited.


METHODS
Pre-treated patients with advanced uveal melanoma received ipilimumab 3 mg/kg through an expanded access programme, every 3 weeks for four doses. Tumour assessments were conducted at baseline and after completion of treatment and patients were monitored throughout for adverse events.


RESULTS
Among 82 assessable patients, 4 (5%) had an immune-related objective response and 24 (29%) had immune-related stable disease lasting ≥3 months for an immune-related disease control rate of 34%. With a median follow-up of 5.6 months, median overall survival (OS) was 6.0 months and median progression-free survival (PFS) was 3.6 months. The 1-year rates of OS and PFS were 31% and 11%, respectively. The safety profile of ipilimumab was similar to that in patients with cutaneous melanoma.


CONCLUSIONS
These data suggest ipilimumab 3 mg/kg is a feasible option in pre-treated patients with metastatic uveal melanoma. Evidence of disease control and a 1-year survival rate of 31% indicate the need for further investigation in randomised, controlled trials to determine the optimal timing and use of ipilimumab in this patient population.
Medical Oncology and Immunotherapy, Department of Oncology, University Hospital of Siena, Tuscan Cancer Institute, Siena.
M
M
Maio
R
R
Danielli
V
V
Chiarion-Sileni
J
J
Pigozzo
G
G
Parmiani
R
R
Ridolfi
F
F
De Rosa
M
M
Del Vecchio
L
L
Di Guardo
P
P
Queirolo
V
V
Picasso
P
P
Marchetti
F
F
De Galitiis
M
M
Mandalà
M
M
Guida
E
E
Simeone
P A
PA
Ascierto
D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D000911:Antibodies, Monoclonal / Q000008:administration & dosage* / Q000009:adverse effects; D018572:Disease-Free Survival; D005260:Female; D006801:Humans; D000074324:Ipilimumab; D008297:Male; D008545:Melanoma / Q000188:drug therapy* / Q000401:mortality / Q000473:pathology; D008875:Middle Aged; D009362:Neoplasm Metastasis; D009367:Neoplasm Staging; D012878:Skin Neoplasms / Q000188:drug therapy* / Q000401:mortality / Q000473:pathology; D016896:Treatment Outcome; D014604:Uveal Neoplasms / Q000188:drug therapy* / Q000401:mortality / Q000473:pathology; D000098943:Uveal Melanoma
D000911:Antibodies, Monoclonal; D000074324:Ipilimumab
efficacy; expanded access programme; ipilimumab; metastatic melanoma; safety; uveal melanoma