Improved Metabolic Correction in Patients with Lysosomal Storage Disease Treated with Hematopoietic Stem Cell Transplant Compared with Enzyme Replacement Therapy

Journal of Pediatrics

Volumn 154, Issue 4, 2009, Pages 609-611

  Wynn, Robert F ^a   Wraith, J Ed ^a   Mercer, Jean ^a   O'Meara, Anne ^b   Tylee, Karen ^a   Thornley, Margaret ^a   Church, Heather J ^a   Bigger, Brian W ^c  

^a ROYAL MANCHESTER CHILDREN S HOSPITAL   (United Kingdom)

^b OUR LADY S CHILDREN S HOSPITAL   (Ireland)

^c UNIVERSITY OF MANCHESTER   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

CHONDROITIN SULFATE; DERMATAN SULFATE; ENZYME;

ARTICLE; CLINICAL ARTICLE; DATA ANALYSIS; ENZYME REPLACEMENT; ENZYME SUBSTRATE; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; LYSOSOME STORAGE DISEASE; PRIORITY JOURNAL;

BIOLOGICAL MARKERS; CHONDROITIN SULFATES; DERMATAN SULFATE; ENZYMES; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; MUCOPOLYSACCHARIDOSIS I; MUCOPOLYSACCHARIDOSIS II; MUCOPOLYSACCHARIDOSIS VI;

EID: 62649094547     PISSN: 00223476     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.jpeds.2008.11.005     Document Type: Article

References (8)

1. Vellodi A. Lysosomal storage disorders. Br J Haematol 128 (2005) 413-431
2. Boelens J.J., Wynn R.F., O'Meara A., Veys P., Bertrand Y., Souillet G., 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
3. Krivit W., Sung J.H., Shapiro E.G., and Lockman L.A. Microglia: the effector cell for reconstitution of the central nervous system following bone marrow transplantation for lysosomal and peroxisomal storage diseases. Cell Transplant 4 (1995) 385-392
4. 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
5. Staba S.L., Escolar M.L., Poe M., Kim Y., Martin P.L., Szabolcs P., et al. Cord-blood transplants from unrelated donors in patients with Hurler's syndrome. N Engl J Med 350 (2004) 1960-1969
6. Wynn RF M.J., Page J., Carr T.F., Jones S.J., and Wraith J.E. Use of enzyme replacement therapy (laronidase) before hematopoietic stem cell transplantation for mucopolysaccharidosis I: experience in 18 patients. J Pediatr (2008) In press
7. Wraith J.E., Clarke L.A., Beck M., Kolodny E.H., Pastores G.M., Muenzer J., et al. Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo-controlled, multinational study of recombinant human alpha-L-iduronidase (laronidase). J Pediatr 144 (2004) 581-588
8. Church H., Tylee K., Cooper A., Thornley M., Mercer J., Wraith E., 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