Hematopoietic Cell Transplantation for Mucopolysaccharidosis Patients Is Safe and Effective: Results after Implementation of International Guidelines

Biology of Blood and Marrow Transplantation

Volumn 21, Issue 6, 2015, Pages 1106-1109

  Aldenhoven, Mieke ^a   Jones, Simon A ^b   Bonney, Denise ^c   Borrill, Roisin E ^c   Coussons, Mary ^c   Mercer, Jean ^b   Bierings, Marc B ^a   Versluys, Birgitta ^a   van Hasselt, Peter M ^a   Wijburg, Frits A ^d   van der Ploeg, Ans T ^e   Wynn, Robert F ^c   Boelens, Jaap Jan ^a  

^a UNIVERSITY MEDICAL CENTER UTRECHT   (Netherlands)

^b MANCHESTER UNIVERSITY NHS FOUNDATION TRUST   (United Kingdom)

^c ROYAL MANCHESTER CHILDREN S HOSPITAL   (United Kingdom)

^d UNIVERSITY OF AMSTERDAM   (Netherlands)

^e ERASMUS MEDICAL CENTER   (Netherlands)

Author keywords

Hematopoietic cell transplantation; Hurler syndrome; Mucopolysaccharidosis

Indexed keywords

BUSULFAN; CYCLOPHOSPHAMIDE; CYCLOSPORIN A; FLUDARABINE; METHOTREXATE; PREDNISOLONE; THYMOCYTE ANTIBODY; MYELOABLATIVE AGENT; VIDARABINE;

ACUTE GRAFT VERSUS HOST DISEASE; ADULT; ARTICLE; CELL TRANSPLANTATION; CHILD; DONOR; DRUG EFFICACY; DRUG SAFETY; EVENT FREE SURVIVAL; FEMALE; GRAFT SURVIVAL; HEMATOPOIETIC CELL TRANSPLANTATION; HUMAN; MAJOR CLINICAL STUDY; MALE; MUCOPOLYSACCHARIDOSIS; PRACTICE GUIDELINE; SIBLING; UMBILICAL CORD BLOOD; ACUTE DISEASE; ALLOTRANSPLANTATION; ANALOGS AND DERIVATIVES; CHRONIC DISEASE; CLINICAL TRIAL; CORD BLOOD STEM CELL TRANSPLANTATION; FOLLOW UP; GRAFT VERSUS HOST REACTION; HEMATOPOIETIC STEM CELL TRANSPLANTATION; IMMUNOLOGY; INFANT; MORTALITY; MUCOPOLYSACCHARIDOSES; MULTICENTER STUDY; NEURODEGENERATIVE DISEASES; NEWBORN; PATHOLOGY; PRESCHOOL CHILD; PROCEDURES; PROGNOSIS; RECURRENT DISEASE; SURVIVAL ANALYSIS; TRANSPLANTATION CONDITIONING; UNRELATED DONORS;

ACUTE DISEASE; BUSULFAN; CHILD; CHILD, PRESCHOOL; CHRONIC DISEASE; CORD BLOOD STEM CELL TRANSPLANTATION; CYCLOPHOSPHAMIDE; FEMALE; FOLLOW-UP STUDIES; GRAFT VS HOST DISEASE; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; INFANT; INFANT, NEWBORN; MALE; MUCOPOLYSACCHARIDOSES; MYELOABLATIVE AGONISTS; NEURODEGENERATIVE DISEASES; PRACTICE GUIDELINES AS TOPIC; PROGNOSIS; RECURRENCE; SIBLINGS; SURVIVAL ANALYSIS; TRANSPLANTATION CONDITIONING; TRANSPLANTATION, HOMOLOGOUS; UNRELATED DONORS; VIDARABINE;

EID: 84929052765     PISSN: 10838791     EISSN: 15236536     Source Type: Journal    
DOI: 10.1016/j.bbmt.2015.02.011     Document Type: Article

References (14)

1. Neufeld E.F., Muenzer J. The mucopolysaccharidoses. The metabolic and molecular basis of inherited disease 2001, 3421-3452. McGraw-Hill, New York, NY. C. Scriver, A. Beaudet, W. Sly, D. Valle (Eds.).
2. Boelens J.J., Orchard P.J., Wynn R. Transplantation in inborn errors of metabolism: current considerations and future perspectives. Br J Haematol 2014, 167:293-303.
3. 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 2007, 40:225-233.
4. Bartelink I.H., van Reij E.M., Gerhardt C.E., et al. Fludarabine and exposure-targeted busulfan compares favorably with busulfan/cyclophosphamide-based regimens in pediatric hematopoietic cell transplantation: maintaining efficacy with less toxicity. Biol Blood Marrow Transplant 2014, 20:345-353.
5. 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.
6. 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 1980, 69:204-217.
7. Bearman S.I. The syndrome of hepatic veno-occlusive disease after marrow transplantation. Blood 1995, 85:3005-3020.
8. Boelens J.J., Rocha V., Aldenhoven M., et al. Risk factor analysis of outcomes after unrelated cord blood transplantation in patients with hurler syndrome. Biol Blood Marrow Transplant 2009, 15:618-625. for EUROCORD, Inborn error Working Party of EBMT and Duke University.
9. Boelens J.J., Aldenhoven M., Purtill D., et al. Outcomes of transplantation using various hematopoietic cell sources in children with Hurler syndrome after myeloablative conditioning. Blood 2013, 121:3981-3987.
10. 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 2004, 350:1960-1969.
11. Pai S.Y., Logan B.R., Griffith L.M., et al. Transplantation outcomes for severe combined immunodeficiency, 2000-2009. N Engl J Med 2014, 371:434-446.
12. Aldenhoven M, Wynn RF, Orchard PJ, et al. Long-term clinical outcome of Hurler syndrome patients after hematopoietic cell transplantation: an international multi-center study. Blood. Epub ahead of print.
13. Saif M.A., Bigger B.W., Brookes K.E., et al. Hematopoietic stem cell transplantation improves the high incidence of neutralizing allo-antibodies observed in Hurler's syndrome after pharmacological enzyme replacement therapy. Haematologica 2012, 97:1320-1328.
14. Wynn R.F., Wraith J.E., Mercer J., et al. Improved metabolic correction in patients with lysosomal storage disease treated with hematopoietic stem cell transplant compared with enzyme replacement therapy. J Pediatr 2009, 154:609-611.