Predictive factors for cure after immunosuppressive therapy of aplastic anemia

a previous study, we evaluated efficacy of repeated antilymphocyte globulin (ALG) treatment for patients with severe aplastic anemia not responding to an initial ALG treatment or relapsing after initial response to ALG. We no w searched in the same cohort of patients for differences between patients who responded to treatment and remained free of complications and those who relapsed or developed a clonal complication. From 107 patients surviving f or more than 1 year after immunosuppression, 34 remained free from complica tions after the first course of ALG, and 73 presented an event defined as r elapse of aplastic anemia, development of a clonal complication such as par oxysmal nocturnal hemoglobinuria, myelodysplastic syndrome or leukemia, or appearance of a solid tumor. We compared these two groups for survival, cli nical performance and blood counts during follow-up. Survival probability w as 93% for the event-free patients, and 55% for the patients with a complic ation event (p = 0.0003). Event-free patients had a higher incidence of com plete remission (71%), were more often free of immunosuppressive treatment (79%) and independent of transfusions (100%), and had a higher Karnofsky sc ore (91% with a score greater than or equal to 90%) as compared to the grou p with events (29, 37, 67, 48%; p less than or equal to 0.0002). At 1 and 3 years, event-free patients had significantly higher leukocyte and neutroph il counts, as compared to patients with a complication (p < 0.05). However, at 3 and 5 years, event-free patients had borderline higher platelet count s (p = 0.056, p = 0.078) and hemoglobin (p = 0.097, p = 0.061). The coeffic ient of variation as an expression of the variability of the results in eac h group was systematically lower at 3, 5 and 10 years in the group of event -free patients. Despite some differences between the two groups, our data s upport the hypothesis that patients with long-lasting remissions should not be considered as definitively cured of aplastic anemia. Copyright (C) 2000 S. Karger AG, Basel.