TY - JOUR
T1 - Phase II study of ipilimumab in adolescents with unresectable stage III or IV malignant melanoma
AU - Geoerger, Birgit
AU - Bergeron, Christophe
AU - Gore, Lia
AU - Sender, Leonard
AU - Dunkel, Ira J.
AU - Herzog, Cynthia
AU - Brochez, Lieve
AU - Cruz, Ofelia
AU - Nysom, Karsten
AU - Berghorn, Elmer
AU - Simsek, Burcin
AU - Shen, Jun
AU - Pappo, Alberto
N1 - Funding Information:
This research was supported in part through the NIH/NCI Cancer Center Support Grant P30 CA008748 (I.J.D.) and Bristol-Myers Squibb.
Publisher Copyright:
© 2017 Elsevier Ltd
PY - 2017/11
Y1 - 2017/11
N2 - Background Ipilimumab is approved for the treatment of advanced melanoma in adults; however, little information on the efficacy and safety of ipilimumab in younger patients is available. Methods Patients aged 12 to <18 years with previously treated or untreated, unresectable stage III or IV malignant melanoma received ipilimumab 3 or 10 mg/kg every 3 weeks. Primary end-points were 1-year overall survival and safety. Results Over a period of 3.5 years, 12 patients received ipilimumab at either 3 mg/kg (n = 4) or 10 mg/kg (n = 8). The median number of ipilimumab doses was four for 3 mg/kg and three for 10 mg/kg. At 1 year, three of four patients on 3 mg/kg and five of eight patients on 10 mg/kg were alive. Two patients on 10 mg/kg had partial response, and one on 3 mg/kg had stable disease. One patient had durable partial response at 3 years without further treatment, at time of this report. There was one grade 3/4 immune-mediated adverse reaction with 3 mg/kg and five with 10 mg/kg. There were no treatment-related deaths. The study was stopped due to slow accrual. Conclusions At >1 year follow-up, ipilimumab demonstrated activity in melanoma patients aged 12 to <18 years, with a similar safety profile as that seen in adults. Our trial highlights the difficulties of enrolling younger patients with rare diseases in clinical trials for treatments that are approved in adults, suggesting adolescents with cancer types occurring predominantly in adults should be considered for inclusion in adult trials of promising new drugs. Clinical trial registration: NCT01696045.
AB - Background Ipilimumab is approved for the treatment of advanced melanoma in adults; however, little information on the efficacy and safety of ipilimumab in younger patients is available. Methods Patients aged 12 to <18 years with previously treated or untreated, unresectable stage III or IV malignant melanoma received ipilimumab 3 or 10 mg/kg every 3 weeks. Primary end-points were 1-year overall survival and safety. Results Over a period of 3.5 years, 12 patients received ipilimumab at either 3 mg/kg (n = 4) or 10 mg/kg (n = 8). The median number of ipilimumab doses was four for 3 mg/kg and three for 10 mg/kg. At 1 year, three of four patients on 3 mg/kg and five of eight patients on 10 mg/kg were alive. Two patients on 10 mg/kg had partial response, and one on 3 mg/kg had stable disease. One patient had durable partial response at 3 years without further treatment, at time of this report. There was one grade 3/4 immune-mediated adverse reaction with 3 mg/kg and five with 10 mg/kg. There were no treatment-related deaths. The study was stopped due to slow accrual. Conclusions At >1 year follow-up, ipilimumab demonstrated activity in melanoma patients aged 12 to <18 years, with a similar safety profile as that seen in adults. Our trial highlights the difficulties of enrolling younger patients with rare diseases in clinical trials for treatments that are approved in adults, suggesting adolescents with cancer types occurring predominantly in adults should be considered for inclusion in adult trials of promising new drugs. Clinical trial registration: NCT01696045.
KW - Adolescents
KW - Advanced melanoma
KW - Immune-mediated adverse reaction
KW - Ipilimumab
KW - Overall survival
KW - Progression-free survival
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U2 - 10.1016/j.ejca.2017.09.032
DO - 10.1016/j.ejca.2017.09.032
M3 - Article
C2 - 29100190
AN - SCOPUS:85032211648
SN - 0959-8049
VL - 86
SP - 358
EP - 363
JO - European Journal of Cancer
JF - European Journal of Cancer
ER -