Severe aplastic anemia: a prospective study of the effect of early marrow transplantation on acute mortality.
A prospective randomized trial of therapy for severe aplastic anemia was designed to compare early bone marrow transplantation with conventional treatments. All patients with a sibling matched at the major histocompatibility region were transplanted. Transplantation was performed with 17-100 (median 33) days of original diagnosis. Conventional treatments included transfusion support with or without androgens. Twenty-four of 36 patients intered on the transplant arm are alive after 4-20 (median 9) mo with full marrow reconstitution. Only two are limited by chronic graft-versus-host disease. In contrast only 12 of 31 conventionally treated patients are alive. Six of these survivors have improved, five incompletely. The 19 nontransplant deaths have occurred within 1-11 (median 3) mo of diagnosis. Compared to nontransplant regimens, early transplantation more effectively restores normal marrow function and decreases the acute mortality of severe marrow aplasia (p = 0.006). Pending longer follow-up, early marrow transplantation appears to be the most effective available treatment for severe aplastic anemia.
D000728:Androgens / Q000627:therapeutic use; D000741:Anemia, Aplastic / Q000401:mortality / Q000473:pathology / Q000628:therapy*; D000818:Animals; D001803:Blood Transfusion; D001854:Bone Marrow Cells; D016026:Bone Marrow Transplantation; D006801:Humans; D008297:Male; D013997:Time Factors; D014184:Transplantation, Homologous
D000728:Androgens