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Treatment of aplastic anaemia with lower‐dose anti‐thymocyte globulin produces similar response rates and survival as per standard dose anti‐thymocyte globulin schedules
Author(s) -
Scott A.,
Morris K.,
Butler J.,
Mills A. K.,
Kennedy G. A.
Publication year - 2016
Publication title -
internal medicine journal
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.596
H-Index - 70
eISSN - 1445-5994
pISSN - 1444-0903
DOI - 10.1111/imj.13175
Subject(s) - medicine , anti thymocyte globulin , gastroenterology , dosing , globulin , eltrombopag , aplastic anemia , transplantation , immunology , bone marrow , antibody , immune thrombocytopenia
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. Aims 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). Conclusion 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.