Blood Journal
Leading the way in experimental and clinical research in hematology

Outcomes of pediatric bone marrow transplantation for leukemia and myelodysplasia using matched sibling, mismatched related, or matched unrelated donors

  1. Peter J. Shaw1,
  2. Fangyu Kan2,
  3. Kwang Woo Ahn3,
  4. Stephen R. Spellman2,
  5. Mahmoud Aljurf4,
  6. Mouhab Ayas4,
  7. Michael Burke5,
  8. Mitchell S. Cairo6,
  9. Allen R. Chen7,
  10. Stella M. Davies8,
  11. Haydar Frangoul9,
  12. James Gajewski10,
  13. Robert Peter Gale11,
  14. Kamar Godder12,
  15. Gregory A. Hale13,
  16. Martin B.A. Heemskerk14,
  17. John Horan15,
  18. Naynesh Kamani16,
  19. Kimberly A. Kasow17,
  20. Ka Wah Chan18,
  21. Stephanie J. Lee19,
  22. Wing H. Leung20,
  23. Victor A. Lewis21,
  24. David Miklos22,
  25. Machteld Oudshoorn23,
  26. Effie W. Petersdorf19,
  27. Olle Ringdén24,
  28. Jean Sanders19,
  29. Kirk R. Schultz25,
  30. Adriana Seber26,
  31. Michelle Setterholm2,
  32. Donna A. Wall27,
  33. Lolie Yu28, and
  34. Michael A. Pulsipher29
  1. 1Children's Hospital at Westmead, Sydney, Australia;
  2. 2National Marrow Donor Program, Minneapolis, MN;
  3. 3Medical College of Wisconsin, Milwaukee, WI;
  4. 4King Faisal Specialist Hospital & Research Center, Riyadh, Saudi Arabia;
  5. 5University of Minnesota, Minneapolis, MN;
  6. 6Columbia University, New York, NY;
  7. 7Johns Hopkins University, Baltimore, MD;
  8. 8Cincinnati Children's Hospital Medical Center, Cincinnati, OH;
  9. 9Vanderbilt Children's Hospital, Nashville, TN;
  10. 10Oregon Health & Science University, Portland, OR;
  11. 11Celgene Corp, Summit, NJ;
  12. 12Children's Medical Center, Virginia Commonwealth Health Systems, Richmond, VA;
  13. 13All Children's Hospital, St Petersburg, FL;
  14. 14Dutch Transplant Foundation, Leiden, The Netherlands;
  15. 15Emory University, Atlanta, GA;
  16. 16Children's National Medical Center, Washington, DC;
  17. 17The University of North Carolina at Chapel Hill, Chapel Hill, NC;
  18. 18Texas Transplant Institute, San Antonio, TX;
  19. 19Fred Hutchinson Cancer Research Center, Seattle, WA;
  20. 20St Jude Children's Research Hospital, Memphis, TN;
  21. 21Alberta Children's Hospital, Calgary, AB;
  22. 22Stanford University, Stanford, CA;
  23. 23Europdonor Foundation, Leiden, The Netherlands;
  24. 24 Karolinska University Hospital, Stockholm, Sweden;
  25. 25University of British Columbia, Vancouver, BC;
  26. 26Instituto de Oncologia Pediatrica GRAACC Unifesp, São Paulo, Brazil;
  27. 27CancerCare Manitoba, Winnipeg, MB;
  28. 28Children's Hospital/LSUHSC, New Orleans, LA; and
  29. 29The University of Utah, Salt Lake City, UT


Although some trials have allowed matched or single human leukocyte antigen (HLA)–mismatched related donors (mmRDs) along with HLA-matched sibling donors (MSDs) for pediatric bone marrow transplantation in early-stage hematologic malignancies, whether mmRD grafts lead to similar outcomes is not known. We compared patients < 18 years old reported to the Center for International Blood and Marrow Transplant Research with acute myeloid leukemia, acute lymphoblastic leukemia, chronic myeloid leukemia, and myelodysplastic syndrome undergoing allogeneic T-replete, myeloablative bone marrow transplantation between 1993 and 2006. In total, patients receiving bone marrow from 1208 MSDs, 266 8/8 allelic-matched unrelated donors (URDs), and 151 0-1 HLA-antigen mmRDs were studied. Multivariate analysis showed that recipients of MSD transplants had less transplantation-related mortality, acute graft-versus-host disease (GVHD), and chronic GVHD, along with better disease-free and overall survival than the URD and mmRD groups. No differences were observed in transplant-related mortality, acute and chronic GVHD, relapse, disease-free survival, or overall survival between the mmRD and URD groups. These data show that mmRD and 8/8 URD outcomes are similar, whereas MSD outcomes are superior to the other 2 sources. Whether allele level typing could identify mmRD recipients with better outcomes will not be known unless centers alter practice and type mmRD at the allele level.

  • Submitted January 11, 2010.
  • Accepted July 15, 2010.
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