Bone marrow transplantation for severe aplastic anemia: the Barcelona Hospital Clinic experience.
OBJECTIVE
The outcome of patients with severe aplastic anemia (SAA) has improved considerably over the last decades. Bone marrow transplantation (BMT) is the treatment of choice in young patients who have an HLA-identical sibling donor. This study analyzes the outcome and factors related to survival in patients with SAA receiving BMT in our institution.


METHODS
Between March 1978 and December 1996, 49 consecutive patients received an HLA-identical sibling marrow transplant for SAA. Median age was 21 years (range, 4 to 47) and 15 (31%) were women. Median interval from diagnosis to transplant was 2.6 months (range, 0.5 to 159). Between 1978 and 1982 all patients were conditioned with cyclophosphamide (CY) alone and received methotrexate (MTX) until day 102 as graft-versus-host disease (GvHD) prophylaxis. From 1983 most patients received CY and thoraco-abdominal irradiation (TAI) as the conditioning regimen and cyclosporin A (CSA) as GvHD prophylaxis.


RESULTS
Survival probability at 10 years was 55 +/- 7% with a median follow-up for the surviving patients of 8.5 years. The incidences of graft failure, grade II to IV acute GvHD, and chronic GvHD were 21%, 39.5% and 31%, respectively. In multivariate analysis three factors adversely influenced survival: a) age > or = 30 years (p = 0.05); b) > or = 10 transfusion units pre-BMT (p = 0.008); and c) use of long course MTX for GvHD prophylaxis (p = 0.01). One case of squamous-cell carcinoma occurred in a TAI-treated patient 13 years post-transplantation.


CONCLUSIONS
BMT is effective in young patients with SAA who have an HLA-identical sibling donor, particularly if minimally transfused pre-transplant. The introduction of TAI and CSA to our preparative regimen has led to a remarkably increased survival.
Hernández-Boluda
J C
JC
Department of Hematology, Postgraduate School of Hematology Farreras Valenti, Hospital Clínic, University of Barcelona, Spain.
Marín
P
P
Carreras
E
E
Aguilar
J L
JL
Grañena
A
A
Rozman
C
C
Montserrat
E
E
D000293:Adolescent; D000328:Adult; D000741:Anemia, Aplastic / Q000401:mortality / Q000628:therapy*; D016026:Bone Marrow Transplantation / Q000009:adverse effects; D002294:Carcinoma, Squamous Cell; D002648:Child; D002675:Child, Preschool; D005260:Female; D005500:Follow-Up Studies; D006084:Graft Rejection / Q000453:epidemiology; D006086:Graft vs Host Disease / Q000209:etiology / Q000401:mortality; D006680:HLA Antigens / Q000276:immunology; D006801:Humans; D015994:Incidence; D008297:Male; D008875:Middle Aged; D009679:Nuclear Family; D013030:Spain / Q000453:epidemiology; D016019:Survival Analysis; D019172:Transplantation Conditioning; D014184:Transplantation, Homologous / Q000009:adverse effects; D016896:Treatment Outcome
D006680:HLA Antigens