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

Biology of Blood and Marrow Transplantation

Volumn 15, Issue 5, 2009, Pages 618-625

  Boelens, Jaap Jan ^a   Rocha, Vanderson ^b   Aldenhoven, Mieke ^a   Wynn, Robert ^c   O'Meara, Anne ^d   Michel, Gerard ^e   Ionescu, Irina ^f   Parikh, Suhag ^g   Prasad, Vinod K ^g   Szabolcs, Paul ^g   Escolar, Maria ^h   Gluckman, Eliane ^b   Cavazzana Calvo, Marina ⁱ   Kurtzberg, Joanne ^g  

^a UNIVERSITY MEDICAL CENTER UTRECHT   (Netherlands)

^b SAINT LOUIS HOSPITAL   (France)

^c ROYAL MANCHESTER CHILDREN S HOSPITAL   (United Kingdom)

^d OUR LADY S CHILDREN S HOSPITAL   (Ireland)

^e TIMONE HOSPITAL   (France)

^f EUROCORD   (France)

^g DUKE UNIVERSITY   (United States)

^h UNIVERSITY OF NORTH CAROLINA   (United States)

ⁱ HÔPITAL NECKER ENFANTS MALADES   (France)

Author keywords

(Umbilical) cord blood; Hurler syndrome; Stem cell transplantation

Indexed keywords

ALEMTUZUMAB; BUSULFAN; CD34 ANTIGEN; CYCLOPHOSPHAMIDE; CYCLOSPORIN A; METHOTREXATE; MYCOPHENOLIC ACID 2 MORPHOLINOETHYL ESTER; PREDNISOLONE; T LYMPHOCYTE ANTIBODY; THYMOCYTE ANTIBODY;

ARTICLE; BLOOD CELL; CELL COUNT; CHILD; CORD BLOOD STEM CELL TRANSPLANTATION; ENGRAFTMENT; EVENT FREE SURVIVAL; FEMALE; GRAFT SURVIVAL; GRAFT VERSUS HOST REACTION; HLA SYSTEM; HUMAN; HUMAN CELL; HUNTER SYNDROME; HURLER SYNDROME; INFANT; MAJOR CLINICAL STUDY; MALE; NEUTROPHIL; OVERALL SURVIVAL; RISK ASSESSMENT; RISK FACTOR; SURVIVAL RATE; SURVIVAL TIME; TREATMENT OUTCOME;

AGE FACTORS; ANTILYMPHOCYTE SERUM; BUSULFAN; CELL COUNT; CORD BLOOD STEM CELL TRANSPLANTATION; CYCLOPHOSPHAMIDE; FEMALE; GRAFT SURVIVAL; HISTOCOMPATIBILITY TESTING; HUMANS; INFANT; MALE; MUCOPOLYSACCHARIDOSIS I; RETROSPECTIVE STUDIES; RISK FACTORS; SURVIVAL ANALYSIS; TIME FACTORS; TISSUE DONORS; TRANSPLANTATION CHIMERA; TRANSPLANTATION CONDITIONING; TREATMENT OUTCOME;

EID: 63749102158     PISSN: 10838791     EISSN: 15236536     Source Type: Journal    
DOI: 10.1016/j.bbmt.2009.01.020     Document Type: Article

References (35)

1. Neufeld E.F., and Muenzer J. The mucopolysaccharidosis. In: Scriver C., Beaudet A., Sly W., and Valle D. (Eds). The Metabolic and Molecular Basis of Inherited Disease (2001), McGraw-Hill, New York 3421-3452
2. Wraith J.E. The first 5 years of clinical experience with laronidase enzyme replacement therapy for mucopolysaccharidosis I. Expert Opin Pharmacother 6 (2005) 489-506
3. Kakkis E.D., Muenzer J., Tiller G.E., et al. Enzyme-replacement therapy in mucopolysaccharidosis I. N Engl J Med 344 (2001) 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., and de Koning T. The clinical outcome of Hurler syndrome after stem cell transplantation. Biol Blood Marrow Transplant 14 (2008) 88-98
7. Boelens J.J., Wynn R.F., 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 40 (2007) 225-233
8. Staba S.L., Escolar M.L., Poe M., et al. Cord-blood transplants from unrelated donors in patients with Hurler's syndrome. N Engl J Med 350 (2004) 1960-1969
9. Martin P.L., Carter S.L., Kernan N.A., 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 12 (2006) 184-194
10. Prasad V.K., Mendizabal A., Parikh S.H., 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 112 (2008) 2979-2989
11. Escolar M.L., Poe M.D., Provenzale J.M., et al. Transplantation of umbilical-cord blood in babies with infantile Krabbe's disease. N Engl J Med 352 (2005) 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 18 (1974) 295-304
13. Shulman H.M., Sullivan K.M., Weiden P.L., et al. Chronic graft-versus-host syndrome in man. A long-term clinicopathologic study of 20 Seattle patients. Am J Med 69 (1980) 204-217
14. Gooley T.A., Lesenring W., Crowley J., and Storer B.E. Estimation of failure probabilities in the presence of competing risks: new representations of old estimators. Stat Med 18 (1999) 695-706
15. Gray R.J. A class of K-sample tests for comparing the cumulative incidence of a competing risk. Ann. Stat 116 (1988) 1141-1154
16. Fine J.P., and Gray R.J. A proportional hazards model for subdistribution of a competing risk. J Am Stat Assoc 94 (1999) 496-509
17. Peters C., Shapiro E.G., 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 91 (1998) 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 133 (1998) 119-125
19. Gluckman E., and Rocha V. Donor selection for unrelated cord blood transplants. Curr Opin Immunol 18 (2006) 565-570
20. Wagner J.E., Barker J.N., DeFor T.E., 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 100 (2002) 1611-1618
21. Eapen M., Rubinstein P., Zhang M.J., et al. Outcomes of transplantation of unrelated donor umbilical cord blood and bone marrow in children with acute leukaemia: a comparison study. Lancet 369 (2007) 1947-1954
22. Baxter M.A., Wynn R.F., Schyma L., et al. Marrow stromal cells from patients affected by MPS I differentially support haematopoietic progenitor cell development. J Inherit Metab Dis 28 (2005) 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 31 (2003) 1105-1117
24. Cox-Brinkman J., Boelens J.J., Wraith J.E., et al. Haematopoietic cell transplantation (HCT) in combination with enzyme replacement therapy (ERT) in patients with Hurler syndrome. Bone Marrow Transplant 38 (2006) 17-21
25. Tolar J., Grewal S.S., Bjoraker K.J., et al. Combination of enzyme replacement and hematopoietic stem cell transplantation as therapy for Hurler syndrome. Bone Marrow Transplant 41 (2008) 531-535
26. Peters C., Balthazor M., Shapiro E.G., et al. Outcome of unrelated donor bone marrow transplantation in 40 children with Hurler syndrome. Blood 87 (1996) 4894-4902
27. Rocha V., and Locatelli F. Searching for alternative hematopoietic stem cell donors for pediatric patients. Bone Marrow Transplant 41 (2008) 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 39 (2007) 207-210
29. Szabolcs P., and Niedzwiecki D. Immune reconstitution in children after unrelated cord blood transplantation. Biol Blood Marrow Transplant 14 (2008) 66-72
30. Gardiner C.M., Meara A.O., and Reen D.J. Differential cytotoxicity of cord blood and bone marrow-derived natural killer cells. Blood 91 (1998) 207-213
31. Kogler G., Sensken S., Airey J.A., et al. A new human somatic stem cell from placental cord blood with intrinsic pluripotent differentiation potential. J Exp Med 200 (2004) 123-135
32. Chen N., Hudson J.E., Walczak P., et al. Human umbilical cord blood progenitors: the potential of these hematopoietic cells to become neural. Stem Cells 23 (2005) 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 59 (2006) 244-249
34. Le Blanc K., and Ringden O. Immunobiology of human mesenchymal stem cells and future use in hematopoietic stem cell transplantation. Biol Blood Marrow Transplant 11 (2005) 321-334
35. Ryan J.M., Barry F.P., Murphy J.M., and Mahon B.P. Mesenchymal stem cells avoid allogeneic rejection. J Inflamm (Lond) 2 (2005) 8