Bone Marrow Transplantation for Patients with Myelodysplasia

Pretreatment Variables and Outcome

  1. Frederick R. Appelbaum, MD;
  2. Janet Barrall, MD;
  3. Rainer Storb, MD;
  4. Lloyd D. Fisher, PhD;
  5. Gary Schoch;
  6. Robert E. Ramberg;
  7. Howard Shulman, MD;
  8. Claudio Anasetti, MD;
  9. Scott I. Bearman, MD;
  10. Patrick Beatty, MD, PhD;
  11. William I. Bensinger, MD;
  12. C. Dean Buckner, MD;
  13. Reginald A. Clift;
  14. John A. Hansen, MD;
  15. Paul Martin, MD, PhD;
  16. Finn B. Petersen, MD;
  17. Jean E. Sanders, MD;
  18. Jack Singer, MD;
  19. Patricia Stewart, MD;
  20. Keith M. Sullivan, MD;
  21. Robert P. Witherspoon, MD; and
  22. E. Donnall Thomas, MD

    Abstract

    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.

    Article and Author Information

    • From the Fred Hutchinson Cancer Research Center and the University of Washington School of Medicine, Seattle, Washington. For current author addresses, see end of text.

    • Grant Support: By grants CA 15704, CA 18221, CA 09515, HL 36444, and CA 18029 from the National Cancer Institute and the National Heart, Lung and Blood Institute, National Institutes of Health, and a Research Career Award AI 02425 from the National Institute of Allergy and Infectious Diseases, National Institutes of Health.

    • Requests for Reprints: Frederick R. Appelbaum, MD, Fred Hutchinson Cancer Research Center, 1124 Columbia Street, Seattle, WA 98104.

    • Current Author Addresses: Drs. Appelbaum, Storb, Fisher, Shulman, Anasetti, Bearman, Beatty, Bensinger, Buckner, Hansen, Martin, Petersen, Sanders, Stewart, Sullivan, Witherspoon, and Thomas and Mr. Schoch, Mr. Ramberg, and Mr. Clift: Fred Hutchinson Cancer Research Center, 1124 Columbia Street, Seattle, WA 98104.

      Dr. Barrall: 1821 23rd Avenue East, Seattle, WA 98112.

      Dr. Singer: Veterans Affairs Medical Center, 1660 South Columbian Way, Seattle, WA 98108.

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    1. Ann Intern Med April 15, 1990 vol. 112 no. 8 590-597
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