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Volumn 97, Issue 8, 2008, Pages 1108-1112

Outcome in six children with mucopolysaccharidosis type IH, hurler syndrome, after haematopoietic stem cell transplantation (HSCT)

Author keywords

Bone marrow transplantation; Developmental outcome; Mucopolysaccharidosis

Indexed keywords

ARTICLE; BONE MALFORMATION; CARPAL TUNNEL SYNDROME; CHILD; CHIMERA; CLINICAL ARTICLE; COGNITION; ECHOCARDIOGRAPHY; ENZYME REPLACEMENT; EYE EXAMINATION; FOLLOW UP; GROWTH RETARDATION; HEARING TEST; HEART VALVE; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HIP SURGERY; HUMAN; HURLER SYNDROME; INFANT; LABORATORY TEST; NEUROPHYSIOLOGY; PRESCHOOL CHILD; PRIORITY JOURNAL; RETRANSPLANTATION; SPINE SURGERY; SWEDEN; COGNITIVE DEFECT; EARLY DIAGNOSIS; FEMALE; GENETICS; GENOTYPE; HYPERMETROPIA; MALE; METHODOLOGY; POINT MUTATION; TIME;

EID: 63849138912     PISSN: 08035253     EISSN: 16512227     Source Type: Journal    
DOI: 10.1111/j.1651-2227.2008.00811.x     Document Type: Article
Times cited : (42)

References (22)
  • 1
    • 0000869162 scopus 로고    scopus 로고
    • The Mucopolysaccharidoses
    • CR Schriver, AL Beaudet, D Valle, WS Sly. editors. New York: McGraw-Hill;
    • Neufeld EF, Joseph M. The Mucopolysaccharidoses, in the Metabolic & Molecular Bases of Inherited Disease. In: CR Schriver, AL Beaudet, D Valle, WS Sly. editors. New York: McGraw-Hill; 2001. 3421-52.
    • (2001) Metabolic & Molecular Bases of Inherited Disease , pp. 3421-3452
    • Neufeld, E.F.1    Joseph, M.2
  • 3
    • 0019466750 scopus 로고
    • Reversal of clinical features of Hurler's disease and biochemical improvement after treatment by bone-marrow transplantation
    • Hobbs JR, Hugh-Jones K, Barret AJ, Byrom N, Chambers D, Henry K, et al. Reversal of clinical features of Hurler's disease and biochemical improvement after treatment by bone-marrow transplantation. Lancet 1981; 2: 709-12.
    • (1981) Lancet , vol.2 , pp. 709-712
    • Hobbs, J.R.1    Hugh-Jones, K.2    Barret, A.J.3    Byrom, N.4    Chambers, D.5    Henry, K.6
  • 4
    • 9344245169 scopus 로고    scopus 로고
    • Outcome of unrelated donor bone marrow transplantation in 40 children with Hurler syndrome
    • Peters C, Balthazor M, Shapiro EG, King RJ, Kollman C, Hegland JD, et al. Outcome of unrelated donor bone marrow transplantation in 40 children with Hurler syndrome. Blood 1996; 87: 4894-902.
    • (1996) Blood , vol.87 , pp. 4894-4902
    • Peters, C.1    Balthazor, M.2    Shapiro, E.G.3    King, R.J.4    Kollman, C.5    Hegland, J.D.6
  • 5
    • 33745099961 scopus 로고    scopus 로고
    • Trends in haematopoietic cell transplantation for inborn errors of metabolism
    • Boelens JJ. Trends in haematopoietic cell transplantation for inborn errors of metabolism. J Inherit Metab Dis 2006; 29: 413-20.
    • (2006) J Inherit Metab Dis , vol.29 , pp. 413-420
    • Boelens, J.J.1
  • 6
    • 34547147899 scopus 로고    scopus 로고
    • Outcomes of hematopoietic stem cell transplantation for Hurler's syndrome in Europe: A risk factor analysis for graft failure
    • Epub May 28
    • Boelens JJ, Wynn RF, O'Meara A, et al. Outcomes of hematopoietic stem cell transplantation for Hurler's syndrome in Europe: a risk factor analysis for graft failure. Bone Marrow Transplant 2007; 40: 225-33. Epub 2007 May 28.
    • (2007) Bone Marrow Transplant 2007 , vol.40 , pp. 225-233
    • Boelens, J.J.1    Wynn, R.F.2    O'Meara, A.3
  • 7
    • 20944443759 scopus 로고    scopus 로고
    • Safety and efficacy of enzyme replacement therapy in combination with hematopoietic stem cell transplantation in Hurler syndrome
    • 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: 143-6.
    • (2005) Genet Med , vol.7 , pp. 143-146
    • Grewal, S.S.1    Wynn, R.2    Abdenur, J.E.3    Burton, B.K.4    Gharib, M.5    Haase, C.6
  • 8
    • 35448943425 scopus 로고    scopus 로고
    • Successful treatment of severe heart failure in an infant with Hurler syndrome
    • Epub Sep 4
    • Hirth A, Berg A, Greve G. Successful treatment of severe heart failure in an infant with Hurler syndrome. J Inherit Metab Dis 2007; 30: 820. Epub 2007 Sep 4.
    • (2007) J Inherit Metab Dis 2007 , vol.30 , pp. 820
    • Hirth, A.1    Berg, A.2    Greve, G.3
  • 9
    • 33645046392 scopus 로고    scopus 로고
    • Allogeneic hematopoietic stem cell transplantation for inherited disorders: Experience in a single centre
    • Ringden O, Remberger M, Svahn B-M, Barkholt L, Mattson J, Aschan J, et al. Allogeneic hematopoietic stem cell transplantation for inherited disorders: experience in a single centre. Transplantation 2006; 81: 718-25.
    • (2006) Transplantation , vol.81 , pp. 718-725
    • Ringden, O.1    Remberger, M.2    Svahn, B.-M.3    Barkholt, L.4    Mattson, J.5    Aschan, J.6
  • 10
    • 0032758059 scopus 로고    scopus 로고
    • Effect on cytokine release and graft-versus-host disease of different anti-T cell antibodies during conditioning for unrelated haematopoietic stem cell transplantation
    • Remberger M, Svahn B-M, Hentsche P, Löfgren C, Ringden O. Effect on cytokine release and graft-versus-host disease of different anti-T cell antibodies during conditioning for unrelated haematopoietic stem cell transplantation. Bone Marrow Transplant 1999; 24: 823-30.
    • (1999) Bone Marrow Transplant , vol.24 , pp. 823-830
    • Remberger, M.1    Svahn, B.-M.2    Hentsche, P.3    Löfgren, C.4    Ringden, O.5
  • 11
    • 0025979090 scopus 로고
    • Prevention of graft-versus-host disease with T cell depletion or cyclosporin and methotrexate. A randomized trial in adult leukemic marrow recipients
    • Ringden O, Pihlstedt P, Markling L, Aschan J, Baryd I, Ljungman P, et al. Prevention of graft-versus-host disease with T cell depletion or cyclosporin and methotrexate. A randomized trial in adult leukemic marrow recipients. Bone Marrow Transplant 1991; 7: 221-6.
    • (1991) Bone Marrow Transplant , vol.7 , pp. 221-226
    • Ringden, O.1    Pihlstedt, P.2    Markling, L.3    Aschan, J.4    Baryd, I.5    Ljungman, P.6
  • 12
    • 0015582539 scopus 로고
    • The quantitative determination of glycosaminoglycans in urine with Alcian Blue & GX
    • Whiteman P. The quantitative determination of glycosaminoglycans in urine with Alcian Blue & GX. Biochem J 1973; 131: 351-7.
    • (1973) Biochem J , vol.131 , pp. 351-357
    • Whiteman, P.1
  • 13
    • 0018405128 scopus 로고
    • A fluoremetric assay using 4-methylumbelliferyl-α-L- iduronide for estimation of α-L-iduronidase activity and the detection of Hurler and Scheies syndromes
    • Hopwood J. A fluoremetric assay using 4-methylumbelliferyl-α-L- iduronide for estimation of α-L-iduronidase activity and the detection of Hurler and Scheies syndromes. Clin Chim Acta 1979; 92: 257-65.
    • (1979) Clin Chim Acta , vol.92 , pp. 257-265
    • Hopwood, J.1
  • 16
    • 35948984160 scopus 로고    scopus 로고
    • Gross motor development of children with Hurler syndrome after umbilical cord blood transplantation
    • Epub Sep 4
    • Dusing SC, Thorpe DE, Poe MD, Rosenberg AE, Mercer VS, Escolar ML. Gross motor development of children with Hurler syndrome after umbilical cord blood transplantation. Phys Ther 2007; 87: 1433-40. Epub 2007 Sep 4.
    • (2007) Phys Ther 2007 , vol.87 , pp. 1433-1440
    • Dusing, S.C.1    Thorpe, D.E.2    Poe, M.D.3    Rosenberg, A.E.4    Mercer, V.S.5    Escolar, M.L.6
  • 18
    • 2342535103 scopus 로고    scopus 로고
    • Staba SL, Esc lar ML, P e M, Kim Y, Martin PL, Szab les P, et al. Cord-blood transplants from unrelated donors in patients with Hurler's syndrome. N Engl J Med 2004; 350: 1960-9.
    • Staba SL, Esc lar ML, P e M, Kim Y, Martin PL, Szab les P, et al. Cord-blood transplants from unrelated donors in patients with Hurler's syndrome. N Engl J Med 2004; 350: 1960-9.
  • 19
    • 0017145225 scopus 로고
    • The heart in the Hurler syndrome. Gross, histologic and ultrastructural observations in five necropsy cases
    • Renteria VG, Ferrans VJ, Roberts WC. The heart in the Hurler syndrome. Gross, histologic and ultrastructural observations in five necropsy cases. Am J Cardiol 1975; 38: 487-501.
    • (1975) Am J Cardiol , vol.38 , pp. 487-501
    • Renteria, V.G.1    Ferrans, V.J.2    Roberts, W.C.3
  • 20
    • 0036345045 scopus 로고    scopus 로고
    • Mohan UR, Hay AA, Cleary MA, Wraith JE, Patel RG. Cardiovascular changes in children with mucopolysaccharide disorders. ACTA Pediatrica 2002; 91: 799-804.
    • Mohan UR, Hay AA, Cleary MA, Wraith JE, Patel RG. Cardiovascular changes in children with mucopolysaccharide disorders. ACTA Pediatrica 2002; 91: 799-804.
  • 21
    • 0141459294 scopus 로고    scopus 로고
    • Usefulness of bone marrow transplantation in the Hurler syndrome
    • Braunlin EA, Stauffer NR, Peters CH. Usefulness of bone marrow transplantation in the Hurler syndrome. Am J Cardiol 2003; 92: 882-6.
    • (2003) Am J Cardiol , vol.92 , pp. 882-886
    • Braunlin, E.A.1    Stauffer, N.R.2    Peters, C.H.3
  • 22
    • 22144442824 scopus 로고    scopus 로고
    • Mixed donor chimerism and low level iduronidase expression may be adequate for neurodevelopmental protection in Hurler syndrome
    • Conway J, Dyack S, Crooks B, Fernandez CV. Mixed donor chimerism and low level iduronidase expression may be adequate for neurodevelopmental protection in Hurler syndrome. J Pediatr 2005; 147: 106-8.
    • (2005) J Pediatr , vol.147 , pp. 106-108
    • Conway, J.1    Dyack, S.2    Crooks, B.3    Fernandez, C.V.4


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.