Enzyme replacement is associated with better cognitive outcomes after transplant in Hurler syndrome

Journal of Pediatrics

Volumn 162, Issue 2, 2013, Pages

  Eisengart, Julie B ^a,b   Rudser, Kyle D ^b   Tolar, Jakub ^a,b   Orchard, Paul J ^a,b   Kivisto, Teresa ^a,b   Ziegler, Richard S ^a,b   Whitley, Chester B ^a,b   Shapiro, Elsa G ^a,b  

^a UNIVERSITY OF MINNESOTA MEDICAL SCHOOL   (United States)

^b UNIVERSITY OF MINNESOTA   (United States)

Author keywords

Central nervous system; CNS; CsA; Cyclosporine; Early Learning Composite; ELC; Enzyme replacement therapy; ERT; HCT; Hematopoietic cell transplantation; Intravenous; IV; MPS; Mucopolysaccharidosis

Indexed keywords

LARONIDASE;

ARTICLE; CHILD; CLINICAL ARTICLE; COGNITION; CONTROLLED STUDY; ENGRAFTMENT; ENZYME REPLACEMENT; FEMALE; FOLLOW UP; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HOSPITALIZATION; HUMAN; HURLER SYNDROME; INFANT; INTELLIGENCE QUOTIENT; LANGUAGE ABILITY; LENGTH OF STAY; MALE; MENTAL DETERIORATION; MOTOR PERFORMANCE; MULLEN SCALE; NEUROPSYCHOLOGICAL TEST; NONVERBAL COMMUNICATION; PRESCHOOL CHILD; PRIORITY JOURNAL; PROBLEM SOLVING; RATING SCALE; SURVIVAL; TREATMENT OUTCOME;

EID: 84872350131     PISSN: 00223476     EISSN: 10976833     Source Type: Journal    
DOI: 10.1016/j.jpeds.2012.07.052     Document Type: Article

References (27)

1. E. Shapiro, L. Lockman, M. Balthazor, and W. Krivit Neuropsychological outcomes of several storage diseases with and without bone marrow transplantation J Inherit Metab Dis 18 1995 413 429
2. J. Boelens, V. Prasad, J. Tolar, R. Wynn, and C. Peters Current international perspectives on hematopoietic stem cell transplantation for inherited metabolic disorders Pediatr Clin North Am 57 2010 123 145
3. J. Russell, and S. Kangarloo Therapeutic drug monitoring of busulfan in transplantation Curr Pharm Design 14 2008 1936 1949
4. C. Peters, M. Balthazor, E. Shapiro, R. King, C. Kollman, and J. Hegland Outcome of unrelated donor bone marrow transplantation in 40 children with Hurler syndrome Blood 87 1996 4894 4902
5. C. Peters, E. Shapiro, J. Anderson, P. Henslee-Downey, M. Klemperer, and M. Cowan Hurler syndrome, II: outcome of HLA-genotypically identical sibling and HLA-haploidentical related donor bone marrow transplantation in fifty-four children Blood 91 1998 2601 2608
6. J. Tolar, S. Grewal, K. Bjoraker, C. Whitley, E. Shapiro, and L. Charnas Combination of enzyme replacement and hematopoietic stem cell transplantation as therapy for Hurler syndrome Bone Marrow Transplant 41 2007 531 535
7. J. Tolar, and P. Orchard alpha-L-iduronidase therapy for mucopolysaccharidosis type I Biologics 2 2008 743 751
8. R. Wynn, J. Mercer, J. Page, T. Carr, S. Jones, and J. Wraith Use of enzyme replacement therapy (Laronidase) before hematopoietic stem cell transplantation for mucopolysaccharidosis I: experience in 18 patients J Pediatr 154 2009 135 139
9. J. Cox-Brinkman, J. Boelens, J. Wraith, A. O'Meara, P. Veys, and F. Wijburg Haematopoietic cell transplantation (HCT) in combination with enzyme replacement therapy (ERT) in patients with Hurler syndrome Bone Marrow Transplant 38 2006 17 21
10. J. Boelens Trends in haematopoietic cell transplantation for inborn errors of metabolism J Inherit Metab Dis 29 2006 413 420
11. P. Orchard, C. Milla, E. Braunlin, T. DeFor, K. Bjoraker, and B. Blazar Pre-transplant risk factors affecting outcome in Hurler syndrome Bone Marrow Transplant 45 2010 1239 1246
12. E. Mullen Mullen Scales of Early Learning 1995 American Guidance Service Circle Pines, MN
13. P. Diggle, P. Heagerty, K.-Y. Liang, and S. Zeger Analysis of longitudinal data 2002 Oxford University Press New York
14. J. Raven, J.C. Raven, and J.H. Court Manual for Raven's progressive matrices and vocabulary scales 1998 Oxford Psychologists Press Oxford, UK
15. S. Zeki, and E. Marg A vision of the brain 1993 Blackwell Oxford, UK
16. E. Neufeld, and J. Muenzer The mucopolysaccharidoses Metab Mol Bases Inherit Dis 8 2001 3421 3452
17. J. Stiles Neural plasticity and cognitive development Devel Neuropsychol 18 2000 237 272
18. M. Ueno Molecular anatomy of the brain endothelial barrier: an overview of the distributional features Curr Med Chem 14 2007 1199 1206
19. J. Thomas, S. Jacobs, J. Kierstein, and J. Hove Outcome after three years of laronidase enzyme replacement therapy in a patient with Hurler syndrome J Inherit Metab Dis 29 2006 762
20. V. Tokic, I. Barisic, N. Huzjak, G. Petkovic, K. Fumic, and E. Paschke Enzyme replacement therapy in two patients with an advanced severe (Hurler) phenotype of mucopolysaccharidosis I Eur J Pediatr 166 2007 727 732
21. W. Krivit, J. Sung, E. Shapiro, and L. Lockman 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
22. B. Ajami, J. Bennett, C. Krieger, W. Tetzlaff, and F. Rossi Local self-renewal can sustain CNS microglia maintenance and function throughout adult life Nat Neurosci 10 2007 1538 1543
23. D. Kennedy, and J. Abkowitz Kinetics of central nervous system microglial and macrophage engraftment: analysis using a transgenic bone marrow transplantation model Blood 90 1997 986 993
24. + monocytes only under defined host conditions Nat Neurosci 10 2007 1544 1553
25. C. Vogler, B. Levy, J. Grubb, N. Galvin, Y. Tan, and E. Kakkis Overcoming the blood-brain barrier with high-dose enzyme replacement therapy in murine mucopolysaccharidosis VII Proc Natl Acad Sci USA 102 2005 14777 14782
26. J. Wraith, M. Beck, R. Lane, A. van der Ploeg, E. Shapiro, and Y. Xue Enzyme replacement therapy in patients who have mucopolysaccharidosis I and are younger than 5 years: results of a multinational study of recombinant human [alpha]L-iduronidase (Laronidase) Pediatrics 120 2007 e37
27. E. Bushnell, and J. Boudreau Motor development and the mind: the potential role of motor abilities as a determinant of aspects of perceptual development Child Dev 64 1993 1005 1021