The craniocervical junction following successful haematopoietic stem cell transplantation for mucopolysaccharidosis type I H (Hurler syndrome)

Journal of Inherited Metabolic Disease

Volumn 34, Issue 3, 2011, Pages 755-761

  Miebach, Elke ^a   Church, Heather ^b   Cooper, Alan ^b   Mercer, Jean ^b   Tylee, Karen ^b   Wynn, Robert F ^b   Wraith, J Edmond ^b  

^a JOHANNES GUTENBERG UNIVERSITY   (Germany)

^b ST MARY S HOSPITAL   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

CHONDROITIN SULFATE; DERMATAN SULFATE; GLYCOSAMINOGLYCAN; LEVO IDURONIDASE;

ADOLESCENT; ADULT; ARTICLE; ATLANTOAXIAL JOINT; CERVICAL MYELOPATHY; CHILD; CHIMERA; CHRONIC GRAFT VERSUS HOST DISEASE; CLINICAL ARTICLE; DENS HYPOPLASIA; ENGRAFTMENT; ENZYME ACTIVITY; FEMALE; FOLLOW UP; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; HURLER SYNDROME; MALE; NUCLEAR MAGNETIC RESONANCE IMAGING; ODONTOID DYSPLASIA; PRESCHOOL CHILD; RETROSPECTIVE STUDY; SCHOOL CHILD; SPINAL CORD COMPRESSION; SPINE DISEASE; SPINE INSTABILITY; UNITED KINGDOM;

ADOLESCENT; ADULT; BONE DISEASES, DEVELOPMENTAL; BRAIN; CHILD; CHILD, PRESCHOOL; COHORT STUDIES; FEMALE; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; MAGNETIC RESONANCE IMAGING; MALE; MUCOPOLYSACCHARIDOSIS I; RETROSPECTIVE STUDIES; SKULL; TREATMENT OUTCOME; YOUNG ADULT;

EID: 79959802213     PISSN: 01418955     EISSN: 15732665     Source Type: Journal    
DOI: 10.1007/s10545-011-9309-5     Document Type: Article

References (22)

1. Aldenhoven M, Boelens JJ, de Koning TJ (2008) The clinical outcome of Hurler syndrome after stem cell transplantation. Biol Blood Marrow Transplant 14:485-498 (Pubitemid 351494741)
2. Cokluk C, Aydin K, Rakunt C, Iyigun O, Onder A (2006) The borders of the odontoid process of C2 in adults and in children including the estimation of odontoid/body ratio. Eur Spine J 15:278-282 (Pubitemid 43327660)
3. Cox-Brinkman J, Boelens JJ, Wraith JE et al (2006) Haematopoietic cell transplantation (HCT) in combination with enzyme replacement therapy (ERT) in patients with Hurler syndrome. Bone Marrow Transplant 38:17-21 (Pubitemid 43923084)
4. Elliott S (1988) The odontoid process in children - is it hypoplastic? Clin Radiol 39:391-393
5. Field RE, Buchanan JA, Copplemans MG, Aichroth PM (1994) Bone-marrow transplantation in Hurler's syndrome effect on skeletal development. J Bone Joint Surg Br 76:975-981 (Pubitemid 24368459)
6. Guffon N, Souillet G, Maire I, Straczek J, Guibaud P (1998) Follow-up of nine patients with Hurler syndrome after bone marrow transplantation. J Pediatr 133:119-125 (Pubitemid 28323918)
7. Hite SH, Peters C, Krivit W (2000) Correction of odontoid dysplasia following bone-marrow transplantation and engraftment (in Hurler syndrome MPS 1H). Pediatr Radiol 30:464-470 (Pubitemid 30438676)
8. Khanna G, Van Heest AE, Agel J et al (2007) Analysis of factors affecting development of carpal tunnel syndrome in patients with Hurler syndrome after hematopoietic cell transplantation. Bone Marrow Transplant 39:331-334 (Pubitemid 46403923)
9. Kirks DR (1984) Practical Pediatric Imaging. Little, Brown, Boston
10. Malm G, Gustafsson B, Berglund G et al (2008) Outcome in six children with mucopolysaccharidosis type IH, Hurler syndrome, after haematopeietic stem cell transplantation (HSCT). Acta Paediatr 97:1108-1112
11. McManners T (1983) Odontoid Hypoplasia. Br J Radiol 56:907-910
12. Muenzer J, Wraith JE, Clarke LA (2009) International consensus panel on management and treatment of Mucopolysaccharidosis I: management and treatment guidelines. Pediatrics 123:19-29
13. Neufeld EF, Muenzer J (2001) The mucopolysaccharidoses. In: Scriver CR, Beaudet AL, Sly WS et al (eds) The metabolic and molecular bases of inherited diseases. McGraw-Hill, New York, p 3421-3452
14. Pastores GM, Arn P, Beck M, Clarke JT et al (2007) The MPS I registry: design, methodology, and early findings of a global disease registry for monitoring patients with Mucopolysaccharidosis type I. Mol Genet Metab 91:37-47 (Pubitemid 46550926)
15. Perovic MN, Kopits SE, Thompson RC (1973) Radiological evaluation of the spinal cord in congenital atlanto-axial dislocation. Radiology 109:713-716
16. Peters C, Steward CG, National Marrow Donor Program; International Bone Marrow Transplant Registry: Working Party on Inborn Errors, European Bone Marrow Transplant Group (2003) Hematopoietic cell transplantation for inherited metabolic diseases: an overview of outcomes and practice guidelines. Bone Marrow Transplant 31:229-239
17. Skeletal Dysplasia Group (1989) Instability of the upper cervical spine. Arch Dis Child 64:283-288
18. Smoker WR, Khanna G (2008) Imaging the craniocervical junction. Child Nerv Syst 24:1123-1145
19. Staba SL, Escolar ML, Poe M et al (2004) Cord-blood transplants from unrelated donors in patients with Hurler's syndrome. N Engl J Med 350:1960-1969 (Pubitemid 38580199)
20. Tandon V, Williamson JB, Cowie RA, Wraith JE (1996) Spinal problems in mucopolysaccharidosis I (Hurler syndrome). J Bone Joint Surg Br 78:938-944 (Pubitemid 26392125)
21. Tsukimura T, Tajima Y, Kawashima I et al (2008) Uptake of a recombinant human alpha-L-iduronidase (Iaroidase) by cultured fibroblasts and osteoblasts. Biol Pharm Bull 31:1691-1965
22. Weisstein JS, Delgado E, Steinbach LS, Hart K, Packman S (2004) Musculoskeletal manifestations of Hurler syndrome: long-term follow-up after bone marrow transplantation. J Pediatr Orthop 24:97-101 (Pubitemid 38030056)