ONE COURSE VERSUS 2 COURSES OF ANTITHYMOCYTE GLOBULIN FOR THE TREATMENT OF SEVERE APLASTIC-ANEMIA IN CHILDREN

Purpose: The aim of the therapeutic trials was to optimize the treatme nt of severe aplastic anemia (SAA) and moderate aplastic anemia in chi ldren who lack a suitable bone marrow donor, using immunosuppressive t herapy in the most effective combination and dose. Patients and Method s: Two sequential therapeutic trials for the treatment of severe and m oderate aplastic anemia in children were conducted by 10 institutions. The treatment protocols included antithymocyte globulin (ATG), predni sone, and cyclosporine A (CSA); patients entered on the first protocol , 0190 (ATG x 2), were given two courses of ATG, and those enrolled on the second protocol, 0190B (ATG x 1), were given only one course of A TG. Ten patients were evaluable on ATG x 2. All patients had SAA; thre e had hepatitis-induced severe aplastic anemia (HI-SAA). Twelve patien ts were evaluable on ATG x 1; all had SAA, one of whom had HI-SAA. Res ults: Seven of 10 patients on ATG x 2 responded, and eight of 12 patie nts treated on ATG x 1 responded. Conclusion: Treatment with immunosup pressive therapy using ATG, CSA, and prednisone was very well tolerate d The response rates in both protocols were similar, and results compa re favorably with those of previous therapeutic trials, suggesting tha t a second course of ATG is not necessary.