Primary treatment of acquired aplastic anemia: outcomes with bone marrow transplantation and immunosuppressive therapy. Seattle Bone Marrow Transplant Team.
BACKGROUND
Both immunosuppressive therapy and bone marrow transplantation are accepted treatments for patients with aplastic anemia. Choosing one of these therapies for a given patient depends not only on donor availability but also on such factors as patient age.


OBJECTIVE
To compare survival rates and long-term complications after bone marrow transplantation or immunosuppressive therapy in patients with acquired aplastic anemia and to identify prognostic factors associated with improved survival.


METHODS
Center-based, retrospective analysis.


METHODS
Referral center for patients with aplastic anemia.


METHODS
395 patients with acquired aplastic anemia.


METHODS
Bone marrow transplant from an HLA-identical, related donor or immunosuppressive therapy.


METHODS
Kaplan-Meier survival curves, results of log rank tests, and cumulative incidence curves.


RESULTS
Of 168 bone marrow transplant recipients, 89% had sustained engraftment. Forty-six patients developed grade II to IV acute graft-versus-host disease, and 68 developed chronic graft-versus-host disease that required therapy. Of 227 patients who received immunosuppressive therapy, 44% achieved a complete, partial, or minimal response. Fifty-four percent died or had no response to therapy. Actuarial survival at 15 years was 69% for bone marrow transplant recipients and 38% for patients receiving immunosuppressive therapy (P < 0.001). Improved survival was associated with having bone marrow transplantation as primary therapy, being younger, having no transfusion before transplantation, and having a higher absolute neutrophil count. Disease duration, year of therapy, sex, refractoriness to platelet transfusions, and previous treatment with androgens or corticosteroids did not significantly affect survival.


CONCLUSIONS
Data from this center suggest that bone marrow transplantation may be preferred for younger patients with acquired aplastic anemia who have matched, related donors. Long-term survival is excellent for patients who respond to either form of therapy.
Doney
K
K
Fred Hutchinson Cancer Research Center, Clinical Research Division, Seattle, WA 98104, USA.
Leisenring
W
W
Storb
R
R
Appelbaum
F R
FR
D000206:Actuarial Analysis; D000293:Adolescent; D000328:Adult; D000367:Age Factors; D000741:Anemia, Aplastic / Q000276:immunology / Q000401:mortality / Q000628:therapy*; D016026:Bone Marrow Transplantation / Q000009:adverse effects; D002648:Child; D002675:Child, Preschool; D005260:Female; D006801:Humans; D007166:Immunosuppressive Agents / Q000009:adverse effects / Q000627:therapeutic use*; D007223:Infant; D007958:Leukocyte Count; D008297:Male; D008875:Middle Aged; D009504:Neutrophils; D011379:Prognosis; D012189:Retrospective Studies; D019172:Transplantation Conditioning
D007166:Immunosuppressive Agents