Growth and endocrine function in patients with Hurler syndrome after hematopoietic stem cell transplantation

Bone Marrow Transplantation

Volumn 41, Issue 12, 2008, Pages 1005-1011

  Polgreen, L E ^a   Tolar, J ^a   Plog, M ^a   Himes, J H ^b   Orchard, P J ^a   Whitley, C B ^a   Miller, B S ^a   Petryk, A ^a,b  

^a UNIVERSITY OF MINNESOTA MEDICAL SCHOOL   (United States)

^b UNIVERSITY OF MINNESOTA   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ARGININE; BUSULFAN; CLONIDINE; CYCLOPHOSPHAMIDE; FLUDARABINE; LARONIDASE; LEUPRORELIN; SOMATOMEDIN C; THYMOCYTE ANTIBODY;

ADRENARCHE; AGE; ARTICLE; BODY HEIGHT; CLINICAL FEATURE; COHORT ANALYSIS; CONTROLLED STUDY; CORRELATION ANALYSIS; ENDOCRINE FUNCTION; FEMALE; FOLLOW UP; GROWTH; GROWTH DISORDER; GROWTH HORMONE DEFICIENCY; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; HURLER SYNDROME; HYPOTHYROIDISM; INFANT; MAJOR CLINICAL STUDY; MALE; MUCOPOLYSACCHARIDOSIS; OUTCOME ASSESSMENT; PRECOCIOUS PUBERTY; PRESCHOOL CHILD; PREVALENCE; PRIORITY JOURNAL; REPEATED DRUG DOSE; SCORING SYSTEM; SHORT STATURE; WHOLE BODY RADIATION;

ADOLESCENT; ADOLESCENT DEVELOPMENT; CHILD; CHILD DEVELOPMENT; CHILD, PRESCHOOL; FEMALE; FOLLOW-UP STUDIES; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; INFANT; MALE; MUCOPOLYSACCHARIDOSIS I; RETROSPECTIVE STUDIES; TRANSPLANTATION CONDITIONING; WHOLE-BODY IRRADIATION;

EID: 47849115960     PISSN: 02683369     EISSN: 14765365     Source Type: Journal    
DOI: 10.1038/bmt.2008.20     Document Type: Article

References (39)

1. Bach G, Friedman R, Weissmann B, Neufeld EF. The defect in the Hurler and Scheie syndromes: Deficiency of alpha-L-iduronidase. Proc Natl Acad Sci USA 1972; 69: 2048-2051.
2. Online Mendelian Inheritance in Man, OMIM. McKusick-Nathans Institute for Genetic Medicine, Johns Hopkins University and National Center for Biotechnology Information, National Library of Medicine 2000.
3. Neufeld E, Munezer I. The Metabolic and Molecular Basis of Inherited Disease, 8th edn. McGraw-Hill: New York, 2001, pp 3421-3452.
4. Whitley CB. The Mucopolysaccharidoses. Mosby: St Louis, 1993, pp 367-500.
5. Ashworth JL, Biswas S, Wraith E, Lloyd IC. The ocular features of the mucopolysaccharidoses. Eye 2006; 20: 553-563.
6. Dusing SC, Thorpe D, Rosenberg A, Mercer V, Escolar ML. Gross motor abilities in children with Hurler syndrome. Dev Med Child Neurol 2006; 48: 927-930.
7. Sands MS, Vogler C, Kyle JW, Grubb JH, Levy B, Galvin N et al. Enzyme replacement therapy for murine mucopolysaccharidosis type VII. J Clin Invest 1994; 93: 2324-2331.
8. Russell C, Hendson G, Jevon G, Matlock T, Yu J, Aklujkar M et al. Murine MPS I: Insights into the pathogenesis of Hurler syndrome. Clin Genet 1998; 53: 349-361.
9. Abreu S, Hayden J, Berthold P, Shapiro IM, Decker S, Patterson D et al. Growth plate pathology in feline mucopolysaccharidosis VI. Calcif Tissue Int 1995; 57: 185-190.
10. Silveri CP, Kaplan FS, Fallon MD, Bayever E, August CS. Hurler syndrome with special reference to histologic abnormalities of the growth plate. Clin Orthop Relat Res 1991; 269: 305-311.
11. Oda H, Sasaki Y, Nakatani Y, Maesaka H, Suwa S. Hunter's syndrome. An ultrastructural study of an autopsy case. Acta Pathol Jpn 1988; 38: 1175-1190.
12. Nagashima K, Endo H, Sakakibara K, Konishi Y, Miyachi K, Wey JJ et al Morphological and biochemical studies of a case of mucopolysaccharidosis II (Hunter's syndrome). Acta Pathol Jpn 1976; 26: 115-132.
13. Soper BW, Pung AW, Vogler CA, Grubb JH, Sly WS, Barker JE. Enzyme replacement therapy improves reproductive performance in mucopolysaccharidosis type VII mice but does not prevent postnatal losses. Pediatr Res 1999; 45: 180-186.
14. Toledo SP, Costa VH, Fukui RR, Abelin N. Serum growth hormone levels in Hunter's syndrome. Rev Hosp Clin Fac Med Sao Paulo 1991; 46 9-13.
15. Tylki-Szymanska A, Metera M. Precocious puberty in three boys with Sanfilippo A (mucopolysaccharidosis III A). J Pediatr Endocrinol Metab 1995; 8: 291-293.
16. Grewal SS, Wynn R, Abdenur JE, Burton BK, Gharib M, Haase C et al. Safety and efficacy of enzyme replacement therapy in combination with hematopoietic stem cell transplantation in Hurler syndrome. Genet Med 2005; 7: 143-146.
17. Vellodi A, Young EP, Cooper A, Wraith JE, Winchester B, Meaney C et al. Bone marrow transplantation for mucopolysaccharidosis type I: experience of two British centres. Arch Dis Child 1997; 76: 92-99.
18. Krivit W. Allogeneic stem cell transplantation for the treatment of lysosomal and peroxisomal metabolic diseases. Springer Semin Immunopathol 2004; 26: 119-132.
19. Staba SL, Escolar ML, Poe M, Kim Y, Martin PL, Szabolcs P et al. Cord-blood transplants from unrelated donors in patients with Hurler's syndrome. N Engl J Med 2004; 350: 1960-1969.
20. Cox-Brinkman J, Boelens JJ, Wraith JE, O'Meara A, Veys P, Wijburg FA et al. Haematopoietic cell transplantation (HCT) in combination with enzyme replacement therapy (ERT) in patients with Hurler syndrome. Bone Marrow Transplant 2006; 38: 17-21.
21. Bakker B, Oostdijk W, Bresters D, Walenkamp MJ, Vossen JM, Wit JM. Disturbances of growth and endocrine function after busulphan-based conditioning for haematopoietic stem cell transplantation during infancy and childhood. Bone Marrow Transplant 2004; 33: 1049-1056.
22. Brennan BM, Shalet SM. Endocrine late effects after bone marrow transplant. Br J Haematol 2002; 118: 58-66.
23. Frisk P, Arvidson J, Gustafsson J, Lõnnerholm G. Pubertal development and final height after autologous bone marrow transplantation for acute lymphoblastic leukemia. Bone Marrow Transplantation 2004; 33 205-210.
24. Huma Z, Boulad F, Black P, Heller G, Sklar C. Growth in children after bone marrow transplantation for acute leukemia. Blood 1995; 86 819-824.
25. Legault L, Bonny Y. Endocrine complications of bone marrow transplantation in children. Pediatr Transplant 1999; 3: 60-66.
26. Ranke MB, Schwarze CP, Dopfer R, Klingebiel T, Scheel-Walter HG, Lang P et al. Late effects after stem cell transplantation (SCT) in children - growth and hormones. Bone Marrow Transplant 2005; 35 (Suppl 1): S77-S81.
27. Giorgiani G, Bozzola M, Locatelli F, Picco, P, Zecca M, Cisternino M et al. Role of busulfan and total body irradiation on growth of prepubertal children receiving bone marrow transplantation and results of treatment with recombinant human growth hormone. Blood 1995; 86: 825-831.
28. Shalitin S, Phillip M, Stein J, Goshen Y, Carmi D, Yaniv I. Endocrine dysfunction and parameters of the metabolic syndrome after bone marrow transplantation during childhood and adolescence. Bone Marrow Transplant 2006; 37: 1109-1117.
29. Bakker B, Massa GG, Oostdijk W, Van Weel-Sipman MH, Vossen JM, Wit JM. Pubertal development and growth after total-body irradiation and bone marrow transplantation for haematological malignancies. Eur J Pediatr 2000; 159: 31-37.
30. GH Research Society. Consensus guidelines for the diagnosis and treatment of growth hormone (GH) deficiency in childhood and adolescence: Summary statement of the GH Research Society. J Clin Endocrinol Metab 2000; 85: 3990-3993.
31. Kuczmarski RJ, Ogden CL, Grummer-Strawn LM, Flegal KM, Guo SS, Wei R et al. CDC growth charts: United States. Adv Data 2000; 314 1-27.
32. Lee PA. Normal ages of pubertal events among American males and females. J Adolesc Health Care 1980; 1: 26-29.
33. Dayan CM. Interpretation of thyroid function tests. Lancet 2001; 357: 619-624.
34. Couto-Silva AC, Trivin C, Esperou H, Michon J, Baruchel A, Lemaire P et al. Final height and gonad function after total body irradiation during childhood. Bone Marrow Transplant 2006; 38: 427-432.
35. Krivit W, Peters C, Shapiro EG. Bone marrow transplantation as effective treatment of central nervous system disease in globoid cell leukodystrophy, metachromatic leukodystrophy, adrenoleukodystrophy, mannosidosis, fucosidosis, aspartylglucosaminuria, Hurler, Maroteaux-Lamy, and Sly syndromes, and Gaucher disease type III. Curr Opin Neurol 1999; 12: 167-176.
36. Peters C, Shapiro EG, Anderson J, Henslee-Downey PJ, Klemperer MR, Cowan MJ et al. Hurler syndrome: II. Outcome of HLA-genotypically identical sibling and HLA-haploidentical related donor bone marrow transplantation in fifty-four children. The Storage Disease Collaborative Study Group. Blood 1998; 91: 2601-2608.
37. Field RE, Buchanan JA,. Copplemans MG, Aichroth PM. Bone-marrow transplantation in Hurler's syndrome. Effect on skeletal development. J Bone Joint Surg Br 1994; 76: 975-981.
38. Shankar SM, Bunin NJ, Moshang Jr T. Growth in children undergoing bone marrow transplantation after busulfan and cyclophosphamide conditioning. J Pediatr Hematol Oncol 1996; 18: 362-366.
39. Afify Z, Shaw PJ, Clavano-Harding A, Cowell CT. Growth and endocrine function in children with acute myeloid leukaemia after bone marrow transplantation using busulfan/cyclophosphamide. Bone Marrow Transplant 2000; 25: 1087-1092.