Variable disease progression after successful stem cell transplantation: Prospective follow-up investigations in eight patients with Hurler syndrome

Pediatric Transplantation

Volumn 15, Issue 8, 2011, Pages 861-869

  Grigull, Lorenz ^a   Sykora, Karl Walter ^a   Tenger, Andreas ^b   Bertram, Harald ^b   Meyer Marcotty, Max ^c   Hartmann, Hans ^b   Bültmann, Eva ^b   Beilken, Andreas ^a   Živičnjak, Miroslaw ^b   Mynarek, Martin ^a   Osthaus, Alexander W ^b   Schilke, Reinhard ^a   Kollewe, Katja ^a   Lücke, Thomas ^d  

^a HANNOVER MEDICAL SCHOOL   (Germany)

^b Department of Orthopaedic Surgery ^*  (Germany)

^c Department of Plastic Hand and Reconstructive Surgery   (Germany)

^d RUHR UNIVERSITY BOCHUM   (Germany)

Author keywords

follow up; Morbus Hurler; mucopolysaccharidosis; stem cell transplantation

Indexed keywords

FLUDARABINE;

AMBULATORY CARE; ANESTHESIA; CARPAL TUNNEL SYNDROME; CHILD; CLINICAL ARTICLE; CORNEA DISEASE; DENTAL CARE; DEVELOPMENTAL DISORDER; DISEASE COURSE; FEMALE; FOLLOW UP; HAND SURGERY; HEART FAILURE; HEMATOLOGICAL PARAMETERS; HIP DISLOCATION; HIP SURGERY; HUMAN; HURLER SYNDROME; INFANT; MALE; MORTALITY; MUCOPOLYSACCHARIDOSIS; MUSCULOSKELETAL DISEASE; NEUROPSYCHOLOGY; PEDIATRICS; PRESCHOOL CHILD; REVIEW; SCHOOL CHILD; SCOLIOSIS; SPINE MALFORMATION; STEM CELL TRANSPLANTATION; VALVULAR HEART DISEASE;

CHILD, PRESCHOOL; DISEASE PROGRESSION; FEMALE; FOLLOW-UP STUDIES; HUMANS; INFANT; MALE; MUCOPOLYSACCHARIDOSIS I; STEM CELL TRANSPLANTATION; TRANSPLANTATION, HOMOLOGOUS;

EID: 82155197262     PISSN: 13973142     EISSN: 13993046     Source Type: Journal    
DOI: 10.1111/j.1399-3046.2011.01595.x     Document Type: Review

References (21)

1. Beck M,. Variable clinical presentation in lysosomal storage disorders. J Inherit Metab Dis 2001: 24 (Suppl 2): 47-51. (Pubitemid 33061631)
2. Peters C, Steward CG, N ational M arrow D onor P rogram, I nternational B one M arrow T ransplant R egistry, W orking P arty on I nborn E rrors, E uropean B one M arrow T ransplant G roup. Hematopoietic cell transplantation for inherited metabolic diseases: An overview of outcomes and practice guidelines. Bone Marrow Transplant 2003: 31: 229-239. (Pubitemid 36336225)
3. Peters C, Balthazor M, Shapiro EG, et al., Outcome of unrelated donor bone marrow transplantation in 40 children with Hurler syndrome. Blood 1996: 87: 4894-4902. (Pubitemid 26162394)
4. Vellodi A, Young EP, Cooper A, et al., Bone marrow transplantation for mucopolysaccharidosis type I: Experience of two British centres. Arch Dis Child 1997: 76: 92-99. (Pubitemid 27086874)
5. Krivit W, Aubourg P, Shapiro E, et al., Bone marrow transplantation for globoid cell leukodystrophy, adrenoleukodystrophy, metachromatic leukodystrophy, and Hurler syndrome. Curr Opin Hematol 1999: 6: 377-382. (Pubitemid 29503144)
6. Staba SL, Escolar ML, Poe M, et al., Cord-blood transplants from unrelated donors in patients with Hurler's syndrome. N Engl J Med 2004: 350: 1960-1969. (Pubitemid 38580199)
7. Grigull L, Beilken A, Schrappe M, et al., Transplantation of allogeneic CD34-selected stem cells after fludarabine-based conditioning regimen for children with mucopolysaccharidosis 1H (M. Hurler). Bone Marrow Transplant 2005: 35: 265-269. (Pubitemid 40268298)
8. Guffon N, Souillet G, Maire I, et al., Follow-up of nine patients with Hurler syndrome after bone marrow transplantation. J Pediatr 1998: 133: 119-125. (Pubitemid 28323918)
9. Weisstein JS, Delgado E, Steinbach LS, et al., Musculoskeletal manifestations of Hurler syndrome: Long-term follow-up after bone marrow transplantation. J Pediatr Orthop 2004: 24: 97-101. (Pubitemid 38030056)
10. Lücke T, Das AM, Hartmann H, et al., Developmental outcome in five children with Hurler syndrome after stem cell transplantation: A pilot study. Dev Med Child Neurol 2007: 49: 693-696. (Pubitemid 47316792)
11. Braunlin EA, Stauffer N, Peters CH, et al., Usefulness of bone marrow transplantation in the Hurler syndrome. Am J Cardiol 2003: 92: 882-886. (Pubitemid 37163595)
12. Fahnehjelm KT, Törnquist AL, Malm G, et al., Ocular findings in four children with mucopolysaccharidosis I-Hurler (MPS I-H) treated early with haematopoietic stem cell transplantation. Acta Ophthalmol Scand 2006: 84: 781-785. (Pubitemid 44688328)
13. 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 2003: 31: 1105-1117. (Pubitemid 36857368)
14. Malm G, Gustafson B, Berglund G, et al., Outcome in six children with mucopolysaccharidosis type IH, Hurler syndrome, after haematopoietic stem cell transplantation (HSCT). Acta Paediatr 2008: 8: 1108-1112.
15. Peters C, Shapiro EG, Krivit W,. Neuropsychological development in children with Hurler syndrome following hematopoietic stem cell transplantation. Pediatr Transplant 1998: 2: 250-253.
16. Taylor C, Brady P, O'Meara A, et al., Mobility in Hurler syndrome. J Pediatr Orthop 2008: 28: 163-168. (Pubitemid 351498922)
17. Wraith JE, Alani SM,. Carpal tunnel syndrome in the mucopolysaccharidoses and related disorders. Arch Dis Child 1990: 65: 962-963. (Pubitemid 20304934)
18. Muenzer J, Wraith JE, Clarke LA,. International Consensus Panel on Management and Treatment of Mucopolysaccharidosis I. Mucopolysaccharidosis I: Management and treatment guidelines. Pediatrics 2009: 123: 19-29.
19. Soni S, Hente M, Breslin N, et al., Pre-stem cell transplantation enzyme replacement therapy in Hurler syndrome does not lead to significant antibody formation or delayed recovery of the endogenous enzyme post-transplant: A case report. Pediatr Transplant 2007: 11: 563-567. (Pubitemid 47057339)
20. Wynn RF, Mercer J, Page J, et al., Use of enzyme replacement therapy (Laronidase) before hematopoietic stem cell transplantation for mucopolysaccharidosis I: Experience in 18 patients. J Pediatr 2009: 154: 135-139.
21. Munoz-Rojas MV, Vieira T, Costa R, et al., Intrathecal enzyme replacement therapy in a patient with mucopolysaccharidosis type I and symptomatic spinal cord compression. Am J Med Genet 2008: 146A: 2538-2544.