CLAUDIO ANASETTI, M. D.; KRISTINE C. DONEY, M.D.; RAINER STORB, M.D.; JOEL D. MEYERS, M.D.; VERNON T. FAREWELL, Ph.D.; C. DEAN BUCKNER, M.D.; FREDERICK R. APPELBAUM, M.D.; KEITH M. SULLIVAN, M.D.; REGINALD A. CLIFT, F.I.M.L.S.; H. JOACHIM DEEG, M.D.; ALEXANDER FEFER, M.D.; PAUL J. MARTIN, M.D.; JACK W. SINGER, M.D.; JEAN E. SANDERS, M.D.; PATRICIA S. STEWART, M.D.; ROBERT P. WITHERSPOON, M.D.; E. DONNALL THOMAS, M.D.
Fifty patients with severe aplastic anemia had no transfusions of blood products until just before marrow transplantation from HLA-identical family members. Of the 50, 42 are still alive 1 to 12 years after transplantation (median, 7 years). By actuarial standards, the 10-year probability of survival is 82%. Of the 42 surviving patients, 37 have Karnofsky performance status scores of 100% and 5 with chronic graft-versus-host disease have scores ranging from 50% to 90% (median, 80%). The 8 deaths were caused by early infection in 1, graft rejection in 1, acute graft-versus-host disease in 3, and chronic graft-versus-host disease in 3. All deaths occurred within two years after transplantation. The incidence of graft failure was 10%. Acute graft-versus-host disease developed in 14 of 44 patients at risk and chronic graft-versus-host disease, in 15 of 41. Risk factors for development of chronic graft-versus-host disease included increased age (p = 0.008) and presence of acute graft-versus-host disease (p = 0.001). The only factor associated with increased risk of death was development of acute graft-versus-host disease (p = 0.05). Results of this study extend our previous finding that patients with severe aplastic anemia who have transplants before the onset of transfusion-induced sensitization have an excellent probability of long-term survival and a normal life.
CLAUDIO ANASETTI, KRISTINE C. DONEY, RAINER STORB, JOEL D. MEYERS, VERNON T. FAREWELL, C. DEAN BUCKNER, et al. Marrow Transplantation for Severe Aplastic Anemia: Long-Term Outcome in Fifty "Untransfused" Patients. Ann Intern Med. 1986;104:461–466. doi: 10.7326/0003-4819-104-4-461
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Published: Ann Intern Med. 1986;104(4):461-466.
Hematology/Oncology, Red Cell Disorders.
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