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Blood, 15 July 2004, Vol. 104, No. 2, pp. 579-585.
Prepublished online as a Blood First Edition Paper on March 23, 2004; DOI 10.1182/blood-2004-01-0338.
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TRANSPLANTATION
A decision analysis of allogeneic bone marrow transplantation for the myelodysplastic syndromes: delayed transplantation for low-risk myelodysplasia is associated with improved outcome
Corey S. Cutler,
Stephanie J. Lee,
Peter Greenberg,
H. Joachim Deeg,
Waleska S. Pérez,
Claudio Anasetti,
Brian J. Bolwell,
Mitchell S. Cairo,
Robert Peter Gale,
John P. Klein,
Hillard M. Lazarus,
Jane L. Liesveld,
Philip L. McCarthy,
Gustavo A. Milone,
J. Douglas Rizzo,
Kirk R. Schultz,
Michael E. Trigg,
Armand Keating,
Daniel J. Weisdorf,
Joseph H. Antin, and
Mary M. Horowitz
From the International Bone Marrow Transplant Registry (IBMTR), Health Policy Institute, Medical College of Wisconsin, Milwaukee; Dana-Farber Cancer Institute, Boston, MA; Stanford University Medical Center, Stanford, CA; VA Palo Alto Health Care System, Palo Alto, CA; Fred Hutchinson Cancer Research Center, Seattle, WA; Cleveland Clinic Foundation, Cleveland, OH; Case Western Reserve University, Cleveland, OH; Columbia University, New York, NY; Center for Advanced Studies in Leukemia, Los Angeles, CA; University of Rochester Medical Center, NY; Roswell Park Cancer Institute, Buffalo, NY; Fundaleu, Buenos Aires, Argentina; British Columbia's Children's Hospital, Vancouver, BC, Canada; Alfred I. duPont Hospital for Children, Wilmington, DE; Princess Margaret Hospital, Toronto, ON, Canada; and University of Minnesota, Minneapolis.
Bone marrow transplantation (BMT) can cure myelodysplastic syndrome (MDS), although transplantation carries significant risks of morbidity and mortality. Because the optimal timing of HLA-matched BMT for MDS is unknown, we constructed a Markov model to examine 3 transplantation strategies for newly diagnosed MDS: transplantation at diagnosis, transplantation at leukemic progression, and transplantation at an interval from diagnosis but prior to leukemic progression. Analyses using individual patient risk-assessment data from transplantation and nontransplantation registries were performed for all 4 International Prognostic Scoring System (IPSS) risk groups with adjustments for quality of life (QoL). For low and intermediate-1 IPSS groups, delayed transplantation maximized overall survival. Transplantation prior to leukemic transformation was associated with a greater number of life years than transplantation at the time of leukemic progression. In a cohort of patients under the age of 40 years, an even more marked survival advantage for delayed transplantation was noted. For intermediate-2 and high IPSS groups, transplantation at diagnosis maximized overall survival. No changes in the optimal transplantation strategies were noted when QoL adjustments were incorporated. For low- and intermediate-1-risk MDS, delayed BMT is associated with maximal life expectancy, whereas immediate transplantation for intermediate-2- and high-risk disease is associated with maximal life expectancy. (Blood. 2004;104:579-585)

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