A third course of anti‐thymocyte globulin in aplastic anaemia is only beneficial in previous responders

Summary This retrospective study evaluated the outcome of 18 patients with aplastic anaemia treated with a third course of anti‐thymocyte globulin (ATG)‐containing immunosuppressive therapy (IST). Of the 18 patients, seven had responded to one of the previous courses of ATG and 11 were refractory to both the previous courses. Self‐limiting grade ≥3 liver toxicity was observed in three patients. Two patients had to discontinue ATG because of severe systemic side effects. The incidence and manifestations of serum sickness did not appear to be different during the three courses. All of the seven patients that previously responded to one of the courses responded to a third course. In contrast, of 11 patients refractory to the previous courses, only two had a transient partial response. The 3‐yr event‐free survival for the patients who had responded to one of the previous courses of ATG was significantly superior to that of patients refractory to both the previous courses of ATG (83% vs. 0%, P = 0·0001). For aplastic anaemia patients, a third course of ATG‐containing IST is a reasonable option in previous responders. Patients refractory to previous two courses of ATG have a much lower response rate and may be suitable candidates for novel therapeutic options.