PMID- 24885479
OWN - NLM
STAT- MEDLINE
DCOM- 20150223
LR  - 20231110
IS  - 1479-5876 (Electronic)
IS  - 1479-5876 (Linking)
VI  - 12
DP  - 2014 May 7
TI  - Clinical experience with ipilimumab 3 mg/kg: real-world efficacy and safety data 
      from an expanded access programme cohort.
PG  - 116
LID - 10.1186/1479-5876-12-116 [doi]
AB  - BACKGROUND: Ipilimumab improves survival in patients with advanced melanoma. The 
      activity and safety of ipilimumab outside of a clinical trial was assessed in an 
      expanded access programme (EAP). METHODS: Ipilimumab was available upon physician 
      request for patients aged 16 or over with pretreated stage III (unresectable)/IV 
      melanoma, for whom no other therapeutic option was available. Patients received 
      ipilimumab 3 mg/kg every 3 weeks for four doses. Patients with stable disease or 
      an objective response to ipilimumab were eligible for retreatment upon disease 
      progression. Tumour assessments were conducted at baseline and week 12. Patients 
      were monitored for adverse events (AEs) within 3 to 4 days of each scheduled 
      visit. RESULTS: Of 855 patients participating in the EAP in Italy, 833 were 
      evaluable for response. Of these, 13% had an objective immune response, and the 
      immune-related disease control rate was 34%. Median progression-free survival and 
      overall survival were 3.7 and 7.2 months, respectively. Efficacy was independent 
      of BRAF and NRAS mutational status. Overall, 33% of patients reported an 
      immune-related AE (irAE). The frequency of irAEs was not associated with response 
      to ipilimumab. CONCLUSIONS: Outside of a clinical trial setting, ipilimumab is a 
      feasible treatment option in patients with pretreated metastatic melanoma, 
      regardless of BRAF and NRAS mutational status. Data from this large cohort of 
      patients support clinical trial evidence that ipilimumab can induce durable 
      disease control and long-term survival in patients who have failed to respond to 
      prior treatment.
FAU - Ascierto, Paolo A
AU  - Ascierto PA
AD  - Istituto Nazionale Tumori Fondazione 'G, Pascale', Napoli, Italy. 
      paolo.ascierto@gmail.com.
FAU - Simeone, Ester
AU  - Simeone E
FAU - Sileni, Vanna Chiarion
AU  - Sileni VC
FAU - Pigozzo, Jacopo
AU  - Pigozzo J
FAU - Maio, Michele
AU  - Maio M
FAU - Altomonte, Maresa
AU  - Altomonte M
FAU - Del Vecchio, Michele
AU  - Del Vecchio M
FAU - Di Guardo, Lorenza
AU  - Di Guardo L
FAU - Marchetti, Paolo
AU  - Marchetti P
FAU - Ridolfi, Ruggero
AU  - Ridolfi R
FAU - Cognetti, Francesco
AU  - Cognetti F
FAU - Testori, Alessandro
AU  - Testori A
FAU - Bernengo, Maria Grazia
AU  - Bernengo MG
FAU - Guida, Michele
AU  - Guida M
FAU - Marconcini, Riccardo
AU  - Marconcini R
FAU - Mandala, Mario
AU  - Mandala M
FAU - Cimminiello, Carolina
AU  - Cimminiello C
FAU - Rinaldi, Gaetana
AU  - Rinaldi G
FAU - Aglietta, Massimo
AU  - Aglietta M
FAU - Queirolo, Paola
AU  - Queirolo P
LA  - eng
PT  - Journal Article
PT  - Research Support, Non-U.S. Gov't
DEP - 20140507
PL  - England
TA  - J Transl Med
JT  - Journal of translational medicine
JID - 101190741
RN  - 0 (Antibodies, Monoclonal)
RN  - 0 (Ipilimumab)
SB  - IM
MH  - Adolescent
MH  - Adult
MH  - Aged
MH  - Aged, 80 and over
MH  - Antibodies, Monoclonal/adverse effects/*therapeutic use
MH  - Cohort Studies
MH  - Female
MH  - Humans
MH  - Ipilimumab
MH  - Male
MH  - Melanoma/*drug therapy
MH  - Middle Aged
MH  - Treatment Outcome
MH  - Young Adult
PMC - PMC4030525
EDAT- 2014/06/03 06:00
MHDA- 2015/02/24 06:00
PMCR- 2014/05/07
CRDT- 2014/06/03 06:00
PHST- 2014/03/24 00:00 [received]
PHST- 2014/03/24 00:00 [accepted]
PHST- 2014/06/03 06:00 [entrez]
PHST- 2014/06/03 06:00 [pubmed]
PHST- 2015/02/24 06:00 [medline]
PHST- 2014/05/07 00:00 [pmc-release]
AID - 1479-5876-12-116 [pii]
AID - 10.1186/1479-5876-12-116 [doi]
PST - epublish
SO  - J Transl Med. 2014 May 7;12:116. doi: 10.1186/1479-5876-12-116.