R. Arranz et al., CLINICAL-RESULTS IN 50 MULTIPLY TRANSFUSED PATIENTS WITH SEVERE APLASTIC-ANEMIA TREATED WITH BONE-MARROW TRANSPLANTATION OR IMMUNOSUPPRESSIVE THERAPY, Bone marrow transplantation, 13(4), 1994, pp. 383-387
Fifty patients with aplastic anemia (AA) were treated with BMT or immu
nosuppressive therapy (IST). Twenty-one patients underwent BMT using c
yclophosphamide (CY) and 7 Gy total lymphoid irradiation (TLI) and cyc
losporin A (CsA) plus methotrexate (MTX). Actuarial survival is 71% at
5.3 gears with an incidence of graft failure of 0% and of acute GVHD
of 38.9%, Univariate analysis of variables influencing survival showed
a trend for a poorer outcome in patients who received > 30 transfusio
ns prior to BMT and in male recipients from female donors. Twenty-nine
patients > 40 years of age or without matched siblings received antit
hymocyte/antilymphacyte globulin (ATG/ ALG). Response rate to the firs
t course of treatment was 46.4%. Subsequent courses of IST rescued 33%
of patients who relapsed or had not responded. Actuarial survival is
62% at 8.6 years. In our experience both treatment strategies have giv
en encouraging results although overall morbidity is higher in the IST
group because 25% of patients are therapy or transfusion-dependent. T
he role of irradiation in the conditioning regimen of BMT patients, re
cently challenged, is discussed.