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Blood, 1 May 2005, Vol. 105, No. 9, pp. 3749-3756.
Prepublished online as a Blood First Edition Paper on January 6, 2005; DOI 10.1182/blood-2004-08-3312.
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Submitted August 26, 2004
Accepted December 23, 2004
Allogeneic hematopoietic cell transplantation for infants with acute lymphoblastic leukemia
Jean E Sanders*, Ho Joon Im, Paul A Hoffmeister, Ted A Gooley, Ann E Woolfrey, Paul A Carpenter, Robert G Andrews, Eileen M Bryant, and Frederick R Appelbaum
Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, WA, USA; Department of Pediatrics, University of Washington School of Medicine, Seattle, WA, USA
Department of Pediatrics, Asan Medical Center, University of Ulsan College of Medicine, Seoul, Korea, Republic of
Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, WA, USA
Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, WA, USA; Department of Biostatistics, University of Washington School of Public Health and Community Medicine, Seattle, WA, USA
Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, WA, USA; Department of Medicine, University of Washington School of Medicine, Seattle, WA, USA
* Corresponding author; email: jsanders{at}fhcrc.org.
The role of transplantation in infants with acute lymphoblastic leukemia (ALL) is not defined. We analyzed results of 40 infants diagnosed before age 12 months who received a hematopoietic cell transplant (HCT) between July 1982 and February 2003 in CR1 (n=17), CR2/3 (n=7) or relapse (n=16). Patients were conditioned with cyclophosphamide with total body irradiation (n=39) or busulfan (n=1). Donors were matched related, (n=8), mismatched related (n=16), or unrelated (n=16). Graft-versus-host disease (GVHD) prophylaxis was methotrexate or cyclosporine (7) or methotrexate plus cyclosporine (33). Thirty-nine engrafted, 20 developed acute GVHD, and 7 chronic GVHD. Sixteen relapsed and seven died of other causes. Patients in CR1 had disease-free survival (DFS) of 76% compared to 45% for CR2/CR3 and 8% for relapse (P=0.0001). Of 33 patients with cytogenetic data, 26 (79%) had MLL gene rearrangement. Fourteen of these 26 were in CR1 and 11 survive in remission. Outcome was associated with phase of disease, but having the MLL gene was not a factor predictive of outcome. Late effects included growth and other hormone deficiencies. These data demonstrate that infants with ALL and MLL gene have excellent DFS when transplanted in CR1 and consideration for transplantation in CR1 is warranted.

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