title: Clinical experience with ipilimumab 3 mg/kg: real-world efficacy and safety data from an expanded access programme cohort.
abstract: 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.
authors.affiliation: Istituto Nazionale Tumori Fondazione 'G, Pascale', Napoli, Italy. paolo.ascierto@gmail.com.
authors.forename: Paolo A
authors.initials: PA
authors.lastname: Ascierto
authors.forename: Ester
authors.initials: E
authors.lastname: Simeone
authors.forename: Vanna Chiarion
authors.initials: VC
authors.lastname: Sileni
authors.forename: Jacopo
authors.initials: J
authors.lastname: Pigozzo
authors.forename: Michele
authors.initials: M
authors.lastname: Maio
authors.forename: Maresa
authors.initials: M
authors.lastname: Altomonte
authors.forename: Michele
authors.initials: M
authors.lastname: Del Vecchio
authors.forename: Lorenza
authors.initials: L
authors.lastname: Di Guardo
authors.forename: Paolo
authors.initials: P
authors.lastname: Marchetti
authors.forename: Ruggero
authors.initials: R
authors.lastname: Ridolfi
authors.forename: Francesco
authors.initials: F
authors.lastname: Cognetti
authors.forename: Alessandro
authors.initials: A
authors.lastname: Testori
authors.forename: Maria Grazia
authors.initials: MG
authors.lastname: Bernengo
authors.forename: Michele
authors.initials: M
authors.lastname: Guida
authors.forename: Riccardo
authors.initials: R
authors.lastname: Marconcini
authors.forename: Mario
authors.initials: M
authors.lastname: Mandalà
authors.forename: Carolina
authors.initials: C
authors.lastname: Cimminiello
authors.forename: Gaetana
authors.initials: G
authors.lastname: Rinaldi
authors.forename: Massimo
authors.initials: M
authors.lastname: Aglietta
authors.forename: Paola
authors.initials: P
authors.lastname: Queirolo
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D000911:Antibodies, Monoclonal / Q000009:adverse effects / Q000627:therapeutic use*; D015331:Cohort Studies; D005260:Female; D006801:Humans; D000074324:Ipilimumab; D008297:Male; D008545:Melanoma / Q000188:drug therapy*; D008875:Middle Aged; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D000911:Antibodies, Monoclonal; D000074324:Ipilimumab