title: Treatment of aplastic anaemia with lower-dose anti-thymocyte globulin produces similar response rates and survival as per standard dose anti-thymocyte globulin schedules.
abstract: BACKGROUND
  Aplastic anaemia (AA) is a rare acquired bone marrow failure syndrome resulting from the immune-mediated destruction of haemopoietic stem cells. For adults in whom first-line haemopoietic progenitor cell transplantation is not feasible, combination anti-thymocyte globulin (ATGAM) plus cyclosporine A is standard therapy; however, there are minimal data available regarding the optimal ATGAM dosage in terms of efficacy and survival.
  
  
  OBJECTIVE
  Our institutions have historically used different dosing protocols of ATGAM in the treatment of AA. We aimed to review the outcome of AA patients treated with these protocols and compare them to the published literature.
  
  
  METHODS
  We conducted a retrospective study of 31 adults who received first-line ATGAM for AA and compared response rates and survival between cohorts who received standard (40 mg/kg/day D1-4) versus lower-dose (15 mg/kg/day D1-5) ATGAM schedules.
  
  
  RESULTS
  There were similar rates of response (64 vs 71%, P = 1.0), relapse (33 vs 33%, P = 1.0), transformation (14 vs 24%, P = 0.66) or infection (43 vs 47%, P = 1.0), respectively, between standard and lower-dose cohorts. At a median follow up of 24 months, there was no statistical difference between standard and lower-dose cohorts in either event-free (42.2 vs 64.7%, P = 0.91) or overall survival (73.1 vs 88.2%, P = 0.75).
  
  
  CONCLUSIONS
  Our experience suggests that lower-dose ATGAM at 15 mg/kg/day D1-5 as treatment of AA produces similar responses and outcomes as per standard-dose ATGAM schedules. Prospective trials comparing ATGAM dose schedules in AA are warranted.
authors.affiliation: Department of Cancer Care Services, Royal Brisbane and Women's Hospital, Brisbane, Queensland, Australia. ashleigh.scott@health.qld.gov.au.|Department of Haematology, Pathology Queensland, Brisbane, Queensland, Australia. ashleigh.scott@health.qld.gov.au.|School of Medicine, The University of Queensland, Brisbane, Queensland, Australia. ashleigh.scott@health.qld.gov.au.
authors.forename: A
authors.identifier: http://orcid.org/0000-0001-5574-6713
authors.initials: A
authors.lastname: Scott
authors.affiliation: Department of Cancer Care Services, Royal Brisbane and Women's Hospital, Brisbane, Queensland, Australia.
authors.forename: K
authors.initials: K
authors.lastname: Morris
authors.affiliation: Department of Cancer Care Services, Royal Brisbane and Women's Hospital, Brisbane, Queensland, Australia.
authors.forename: J
authors.initials: J
authors.lastname: Butler
authors.affiliation: Division of Cancer Services, Princess Alexandra Hospital, Brisbane, Queensland, Australia.
authors.forename: A K
authors.initials: AK
authors.lastname: Mills
authors.affiliation: Department of Cancer Care Services, Royal Brisbane and Women's Hospital, Brisbane, Queensland, Australia.|School of Medicine, The University of Queensland, Brisbane, Queensland, Australia.
authors.forename: G A
authors.initials: GA
authors.lastname: Kennedy
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D000741:Anemia, Aplastic / Q000188:drug therapy*; D000961:Antilymphocyte Serum / Q000008:administration & dosage*; D001315:Australia; D016572:Cyclosporine / Q000008:administration & dosage*; D016208:Databases, Factual; D004305:Dose-Response Relationship, Drug; D005260:Female; D006801:Humans; D007166:Immunosuppressive Agents / Q000008:administration & dosage*; D053208:Kaplan-Meier Estimate; D008297:Male; D008875:Middle Aged; D012189:Retrospective Studies; D055815:Young Adult
chemical_list: D000961:Antilymphocyte Serum; D007166:Immunosuppressive Agents; D016572:Cyclosporine
keywords: anti-thymocyte globulin; aplastic anaemia; bone marrow failure