Frederick R. Appelbaum, MD; Janet Barrall, MD; Rainer Storb, MD; Lloyd D. Fisher, PhD; Gary Schoch; Robert E. Ramberg; Howard Shulman, MD; Claudio Anasetti, MD; Scott I. Bearman, MD; Patrick Beatty, MD, PhD; William I. Bensinger, MD; C. Dean Buckner, MD; Reginald A. Clift; John A. Hansen, MD; Paul Martin, MD, PhD; Finn B. Petersen, MD; Jean E. Sanders, MD; Jack Singer, MD; Patricia Stewart, MD; Keith M. Sullivan, MD; Robert P. Witherspoon, MD; E. Donnall Thomas, MD
Study Objective: To determine the efficacy of allogeneic bone marrow transplantation for severe myelodysplasia, and to identify variables predictive of outcome.
Design: Case series study.
Setting: A referral-based bone marrow transplant center.
Patients: Consecutive series of 59 patients with myelodysplasia or closely related disorders and either life-threatening cytopenia or a progressive increase in marrow blast percentage.
Intervention: Patients were treated with high-dose cyclophosphamide and total body irradiation followed by allogeneic bone marrow transplantation from either an HLA-identical (n = 45) or HLA-partially matched (n = 14) donor.
Measurements and Main Results: The product-limit estimate for disease-free survival 3 years after transplant is 45% (95% CI, 32% to 59%). The commonest causes of death after transplant were disease recurrence, interstitial pneumonia, and graft-versus-host disease, accounting for eight deaths each. In a univariate analysis, younger patients, those with shorter disease duration, and those whose disease was characterized by an abnormal cytogenetic karyotype had better survival and disease-free survival than the group as a whole. In a multivariate analysis, younger age and abnormal karyotype were independent predictors of improved disease-free survival and overall survival. Patients who received transplants when they had fewer blasts in their bone marrow had a decreased chance for disease recurrence when compared with patients with excess blasts.
Conclusions: Bone marrow transplantation offers a potential cure for many patients with myelodysplasia. Best results can be expected in younger patients who receive transplants relatively early in their disease course.
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Appelbaum FR, Barrall J, Storb R, Fisher LD, Schoch G, Ramberg RE, et al. Bone Marrow Transplantation for Patients with Myelodysplasia: Pretreatment Variables and Outcome. Ann Intern Med. 1990;112:590–597. doi: 10.7326/0003-4819-112-8-590
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Published: Ann Intern Med. 1990;112(8):590-597.
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Print ISSN: 0003-4819 | Online ISSN: 1539-3704
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