title: Outcomes of patients with non-melanoma solid tumours receiving self-funded pembrolizumab at Chris O'Brien Lifehouse.
abstract: BACKGROUND
  Immunotherapy agents show anti-cancer activity in several solid cancers. Efficacy in non-melanoma solid tumours for non-approved indications is unknown.
  
  
  OBJECTIVE
  To evaluate patient and disease characteristics, rate and duration of response, and toxicity of self-funded pembrolizumab in patients with non-melanoma solid cancers.
  
  
  METHODS
  Retrospective review describing outcomes and toxicity of self-funded pembrolizumab in patients with non-melanoma solid cancers treated at Chris O'Brien Lifehouse.
  
  
  RESULTS
  From April 2015 to December 2015, 21 patients received or were planned to receive self-funded pembrolizumab. The median age was 50 years (16-76), 28 and 10% had an Eastern Cooperative Oncology Group performance status of 2, and 3-4 respectively. Sixty-two percent received at least two to four lines of prior drug treatment. Median follow-up was 3.0 months (range, 0.4-9.6). Fourteen (67%) patients requested pembrolizumab. Pembrolizumab was clinician offered for 7 (33%) patients. Patients who requested pembrolizumab had worse outcomes. Three patients died before receiving pembrolizumab. Of the 18 patients that received at least one dose, a partial response was observed in 3 (17%). Progressive disease occurred in 83%. Four patients received only one cycle of pembrolizumab and died after a median of 27 days (range 13-43). Immune-related adverse events of any grade occurred in 33%. No grade 3-4 events were observed.
  
  
  CONCLUSIONS
  Pembrolizumab was well tolerated. Meaningful responses were observed in 17% of treated patients. Response continues after 5-6.5 months follow-up in 11% and >8 months of follow-up for the other responding patient. Financial impact to the patient can be substantial. Outcomes for 33% were poor with three patients dying prior to receiving therapy and four dying within weeks of receiving one dose. This highlights issues regarding the careful selection of patients, futility of anti-cancer therapy at the end-of-life and patients' perceived benefit of receiving this therapy.
authors.affiliation: Department of Medical Oncology, Chris O'Brien Lifehouse, Sydney, New South Wales, Australia.
authors.forename: A J
authors.initials: AJ
authors.lastname: Lomax
authors.affiliation: Department of Medical Oncology, Chris O'Brien Lifehouse, Sydney, New South Wales, Australia.|Department of Medical Oncology, Royal Prince Alfred Hospital, Sydney, New South Wales, Australia.|Sydney Medical School, University of Sydney, Sydney, New South Wales, Australia.
authors.forename: J
authors.initials: J
authors.lastname: Beith
authors.affiliation: Department of Medical Oncology, Chris O'Brien Lifehouse, Sydney, New South Wales, Australia.|Department of Medical Oncology, Royal Prince Alfred Hospital, Sydney, New South Wales, Australia.|Sydney Medical School, University of Sydney, Sydney, New South Wales, Australia.
authors.forename: V
authors.initials: V
authors.lastname: Bhadri
authors.affiliation: Department of Medical Oncology, Chris O'Brien Lifehouse, Sydney, New South Wales, Australia.|Department of Medical Oncology, Royal Prince Alfred Hospital, Sydney, New South Wales, Australia.|Sydney Medical School, University of Sydney, Sydney, New South Wales, Australia.
authors.forename: M
authors.initials: M
authors.lastname: Boyer
authors.affiliation: Department of Medical Oncology, Chris O'Brien Lifehouse, Sydney, New South Wales, Australia.|Department of Medical Oncology, Royal Prince Alfred Hospital, Sydney, New South Wales, Australia.|Sydney Medical School, University of Sydney, Sydney, New South Wales, Australia.
authors.forename: P
authors.initials: P
authors.lastname: Grimison
authors.affiliation: Department of Medical Oncology, Chris O'Brien Lifehouse, Sydney, New South Wales, Australia.|Department of Medical Oncology, Royal Prince Alfred Hospital, Sydney, New South Wales, Australia.|Sydney Medical School, University of Sydney, Sydney, New South Wales, Australia.
authors.forename: L G
authors.initials: LG
authors.lastname: Horvath
authors.affiliation: Department of Medical Oncology, Chris O'Brien Lifehouse, Sydney, New South Wales, Australia.|Department of Medical Oncology, Royal Prince Alfred Hospital, Sydney, New South Wales, Australia.|Sydney Medical School, University of Sydney, Sydney, New South Wales, Australia.
authors.forename: S
authors.initials: S
authors.lastname: Kao
authors.affiliation: Department of Medical Oncology, Chris O'Brien Lifehouse, Sydney, New South Wales, Australia.|Department of Medical Oncology, Royal Prince Alfred Hospital, Sydney, New South Wales, Australia.|Sydney Medical School, University of Sydney, Sydney, New South Wales, Australia.
authors.forename: M
authors.initials: M
authors.lastname: Tattersall
authors.affiliation: Department of Medical Oncology, Chris O'Brien Lifehouse, Sydney, New South Wales, Australia.|Kinghorn Cancer Centre, Sydney, New South Wales, Australia.
authors.forename: D
authors.initials: D
authors.lastname: Thomas
authors.affiliation: Department of Medical Oncology, Chris O'Brien Lifehouse, Sydney, New South Wales, Australia.|Department of Medical Oncology, Royal Prince Alfred Hospital, Sydney, New South Wales, Australia.|Sydney Medical School, University of Sydney, Sydney, New South Wales, Australia.
authors.forename: C
authors.initials: C
authors.lastname: McNeil
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D000911:Antibodies, Monoclonal / Q000009:adverse effects / Q000627:therapeutic use*; D061067:Antibodies, Monoclonal, Humanized / Q000009:adverse effects / Q000627:therapeutic use*; D000970:Antineoplastic Agents / Q000009:adverse effects / Q000627:therapeutic use*; D004305:Dose-Response Relationship, Drug; D005260:Female; D005382:Financing, Personal; D006801:Humans; D008297:Male; D008875:Middle Aged; D018579:Patient Selection; D012189:Retrospective Studies; D012878:Skin Neoplasms / Q000188:drug therapy* / Q000191:economics / Q000401:mortality; D015996:Survival Rate; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D000911:Antibodies, Monoclonal; D061067:Antibodies, Monoclonal, Humanized; D000970:Antineoplastic Agents; C582435:pembrolizumab
keywords: advanced cancer; end-of-life; futility; immunotherapy; pembrolizumab