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Prepublished online as a Blood First Edition Paper on February 20, 2003; DOI 10.1182/blood-2002-08-2619.
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Blood, 1 July 2003, Vol. 102, No. 1, pp. 31-35
CLINICAL OBSERVATIONS, INTERVENTIONS, AND THERAPEUTIC TRIALS
HLA-matched related hematopoietic cell transplantation for chronic-phase CML using a targeted busulfan and cyclophosphamide preparative regimen
Jerald P. Radich,
Ted Gooley,
William Bensinger,
Thomas Chauncey,
Reginald Clift,
Mary Flowers,
Paul Martin,
John Slattery,
Debbie Sultan, and
Frederick R. Appelbaum
From the Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, WA; the University of Washington School of Medicine, Seattle, WA; and the Seattle Veterans Affairs Medical Center, Seattle, WA.
Allogeneic blood or marrow transplantation (BMT) is a curative therapy for chronic myeloid leukemia (CML). We have previously reported that the pharmacologic targeting of busulfan combined with cyclophosphamide (TBU/CY) can minimize regimen-related toxicity while preserving antileukemic effects. We report here on 131 consecutive chronic-phase CML patients treated with allogeneic related BMT using a TBU/CY preparative regimen, where the busulfan dose was targeted to achieve a steady-state plasma concentration of at least 900 ng/mL. The median age of the patients was 43 years (range, 14-66 years). Estimates of the probabilities of nonrelapse mortality, relapse, survival, and disease-free survival 3 years after transplantation were 14%, 8%, 86%, and 78%, respectively. Age had no statistically significant effect on survival. Although approximately 60% of the patients developed clinically extensive chronic graft-versus-host disease, the median Karnofsky score at last contact date among survivors was 95%. Of surviving patients, 11% were molecularly positive for the bcr-abl mRNA at last contact, with a median level of bcr-abl transcripts of 4.6 copies/µg RNA. These data suggest that TBU/CY is a very effective preparative regimen for CML in chronic phase, associated with an expected survival at 3 years of approximately 85%, with most patients being in molecular remission. (Blood. 2003;102:31-35)
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