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.
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. ;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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