Biology of Blood and Marrow Transplantation
Volume 15, Issue 5 , Pages 618-625 , May 2009

Risk Factor Analysis of Outcomes after Unrelated Cord Blood Transplantation in Patients with Hurler Syndrome

  • Jaap Jan Boelens

      Affiliations

    • Department of Immunology/Hematology and BMT, University Medical Center Utrecht, Utrecht, The Netherlands
    • Corresponding Author InformationCorrespondence and reprint requests: JaapJan Boelens, MD, PhD, Department of Immunology/Hematology and BMT, Room KC 03.063.0, University Medical Center Utrecht, Lundlaan 6, 3584 EA, Utrecht, The Netherlands.
    • ,
  • Vanderson Rocha

      Affiliations

    • Department of Hematology/BMT, Eurocord/Hôpital Saint Louis, Paris, France
    • ,
  • Mieke Aldenhoven

      Affiliations

    • Department of Immunology/Hematology and BMT, University Medical Center Utrecht, Utrecht, The Netherlands
    • ,
  • Robert Wynn

      Affiliations

    • Department of Hematology/BMT, Royal Manchester Children's Hospital, Manchester, United Kingdom
    • ,
  • Anne O'Meara

      Affiliations

    • Department of Hematology/Oncology, Our Lady's Hospital for Sick Children, Dublin, Ireland
    • ,
  • Gerard Michel

      Affiliations

    • Hôpital d'Enfants La Timone and the Institut Paoli-Calmettes, Marseille, France
    • ,
  • Irina Ionescu

      Affiliations

    • Eurocord/St. Louis, Paris, France
    • ,
  • Suhag Parikh

      Affiliations

    • Division of Pediatric Blood and Marrow Transplantation, Duke University, Durham, North Carolina
    • ,
  • Vinod K. Prasad

      Affiliations

    • Division of Pediatric Blood and Marrow Transplantation, Duke University, Durham, North Carolina
    • ,
  • Paul Szabolcs

      Affiliations

    • Division of Pediatric Blood and Marrow Transplantation, Duke University, Durham, North Carolina
    • ,
  • Maria Escolar

      Affiliations

    • Centre for Development and Learning, University of North Carolina, Chapel Hill, North Carolina
    • ,
  • Eliane Gluckman

      Affiliations

    • Department of Hematology/BMT, Eurocord/Hôpital Saint Louis, Paris, France
    • ,
  • Marina Cavazzana-Calvo

      Affiliations

    • Department of Biotherapy, Hopital Necker-Enfants Malades, Paris, France
    • ,
  • Joanne Kurtzberg

      Affiliations

    • Division of Pediatric Blood and Marrow Transplantation, Duke University, Durham, North Carolina
    • ,
  • EUROCORD, Inborn error Working Party of EBMT and Duke University

Received 21 October 2008 ,Accepted 29 January 2009.

References 

  1. Neufeld EF, Muenzer J. The mucopolysaccharidosis. In:  Scriver C,  Beaudet A,  Sly W,  Valle D editor. The Metabolic and Molecular Basis of Inherited Disease. New York: McGraw-Hill; 2001;p. 3421–3452
  2. Wraith JE. The first 5 years of clinical experience with laronidase enzyme replacement therapy for mucopolysaccharidosis I. Expert Opin Pharmacother. 2005;6:489–506
  3. Kakkis ED, Muenzer J, Tiller GE, et al. Enzyme-replacement therapy in mucopolysaccharidosis I. N Engl J Med. 2001;344:182–188
  4. EBMT registry. http://www.ebmt.org/4Registry/registry1.html. 2007.
  5. IBMTR registry. http://www.cibmtr.org. 2007.
  6. Aldenhoven M, Boelens J, de Koning T. The clinical outcome of Hurler syndrome after stem cell transplantation. Biol Blood Marrow Transplant. 2008;14:88–98
  7. Boelens JJ, Wynn RF, O'Meara A, et al. Outcomes of hematopoietic stem cell transplantation for Hurler's syndrome in Europe: a risk factor analysis for graft failure. Bone Marrow Transplant. 2007;40:225–233
  8. Staba SL, Escolar ML, Poe M, et al. Cord-blood transplants from unrelated donors in patients with Hurler's syndrome. N Engl J Med. 2004;350:1960–1969
  9. Martin PL, Carter SL, Kernan NA, et al. Results of the cord blood transplantation study (COBLT): outcomes of unrelated donor umbilical cord blood transplantation in pediatric patients with lysosomal and peroxisomal storage diseases. Biol Blood Marrow Transplant. 2006;12:184–194
  10. Prasad VK, Mendizabal A, Parikh SH, et al. Unrelated donor umbilical cord blood transplantation for inherited metabolic disorders in 159 pediatric patients from a single center: influence of cellular composition of the graft on transplant outcomes. Blood. 2008;112:2979–2989
  11. Escolar ML, Poe MD, Provenzale JM, et al. Transplantation of umbilical-cord blood in babies with infantile Krabbe's disease. N Engl J Med. 2005;352:2069–2081
  12. Glucksberg H, Storb R, Fefer A, et al. Clinical manifestations of graft-versus-host disease in human recipients of marrow from HL-A-matched sibling donors. Transplantation. 1974;18:295–304
  13. Shulman HM, Sullivan KM, Weiden PL, et al. Chronic graft-versus-host syndrome in man. A long-term clinicopathologic study of 20 Seattle patients. Am J Med. 1980;69:204–217
  14. Gooley TA, Lesenring W, Crowley J, Storer BE. Estimation of failure probabilities in the presence of competing risks: new representations of old estimators. Stat Med. 1999;18:695–706
  15. Gray RJ. A class of K-sample tests for comparing the cumulative incidence of a competing risk. Ann. Stat. 1988;116:1141–1154
  16. Fine JP, Gray RJ. A proportional hazards model for subdistribution of a competing risk. J Am Stat Assoc. 1999;94:496–509
  17. Peters C, Shapiro EG, Anderson J, et al. Hurler syndrome: II. Outcome of HLA-genotypically identical sibling and HLAhaploidentical related donor bone marrow transplantation in fifty-four children. The Storage Disease Collaborative Study Group. Blood. 1998;91:2601–2608
  18. Guffon N, Souillet G, Maire I, et al. Follow-up of nine patients with Hurler syndrome after bone marrow transplantation. J Pediatr. 1998;133:119–125
  19. Gluckman E, Rocha V. Donor selection for unrelated cord blood transplants. Curr Opin Immunol. 2006;18:565–570
  20. Wagner JE, Barker JN, DeFor TE, et al. Transplantation of unrelated donor umbilical cord blood in 102 patients with malignant and nonmalignant diseases: influence of CD34 cell dose and HLA disparity on treatment-related mortality and survival. Blood. 2002;100:1611–1618
  21. Eapen M, Rubinstein P, Zhang MJ, et al. Outcomes of transplantation of unrelated donor umbilical cord blood and bone marrow in children with acute leukaemia: a comparison study. Lancet. 2007;369:1947–1954
  22. Baxter MA, Wynn RF, Schyma L, et al. Marrow stromal cells from patients affected by MPS I differentially support haematopoietic progenitor cell development. J Inherit Metab Dis. 2005;28:1045–1053
  23. Souillet G, Guffon N, Maire I, et al. Outcome of 27 patients with Hurler's syndrome transplanted from either related or unrelated haematopoietic stem cell sources. Bone Marrow Transplant. 2003;31:1105–1117
  24. Cox-Brinkman J, Boelens JJ, Wraith JE, et al. Haematopoietic cell transplantation (HCT) in combination with enzyme replacement therapy (ERT) in patients with Hurler syndrome. Bone Marrow Transplant. 2006;38:17–21
  25. Tolar J, Grewal SS, Bjoraker KJ, et al. Combination of enzyme replacement and hematopoietic stem cell transplantation as therapy for Hurler syndrome. Bone Marrow Transplant. 2008;41:531–535
  26. Peters C, Balthazor M, Shapiro EG, et al. Outcome of unrelated donor bone marrow transplantation in 40 children with Hurler syndrome. Blood. 1996;87:4894–4902
  27. Rocha V, Locatelli F. Searching for alternative hematopoietic stem cell donors for pediatric patients. Bone Marrow Transplant. 2008;41:207–214
  28. Church H, Tylee K, Cooper A, et al. Biochemical monitoring after haemopoietic stem cell transplant for Hurler syndrome (MPSIH): implications for functional outcome after transplant in metabolic disease. Bone Marrow Transplant. 2007;39:207–210
  29. Szabolcs P, Niedzwiecki D. Immune reconstitution in children after unrelated cord blood transplantation. Biol Blood Marrow Transplant. 2008;14:66–72
  30. Gardiner CM, Meara AO, Reen DJ. Differential cytotoxicity of cord blood and bone marrow-derived natural killer cells. Blood. 1998;91:207–213
  31. Kogler G, Sensken S, Airey JA, et al. A new human somatic stem cell from placental cord blood with intrinsic pluripotent differentiation potential. J Exp Med. 2004;200:123–135
  32. Chen N, Hudson JE, Walczak P, et al. Human umbilical cord blood progenitors: the potential of these hematopoietic cells to become neural. Stem Cells. 2005;23:1560–1570
  33. Meier C, Middelanis J, Wasielewski B, et al. Spastic paresis after perinatal brain damage in rats is reduced by human cord blood mononuclear cells. Pediatr Res. 2006;59:244–249
  34. Le Blanc K, Ringden O. Immunobiology of human mesenchymal stem cells and future use in hematopoietic stem cell transplantation. Biol Blood Marrow Transplant. 2005;11:321–334
  35. Ryan JM, Barry FP, Murphy JM, Mahon BP. Mesenchymal stem cells avoid allogeneic rejection. J Inflamm (Lond). 2005;2:8

 Financial disclosure: See Acknowledgments on page 624.

PII: S1083-8791(09)00072-X

doi: 10.1016/j.bbmt.2009.01.020

Biology of Blood and Marrow Transplantation
Volume 15, Issue 5 , Pages 618-625 , May 2009

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