메뉴 건너뛰기




Volumn 87, Issue 11, 1996, Pages 4894-4902

Outcome of unrelated donor bone marrow transplantation in 40 children with Hurler syndrome

Author keywords

[No Author keywords available]

Indexed keywords

ADULT; ARTICLE; BONE MARROW TRANSPLANTATION; CLINICAL ARTICLE; COGNITION; ENZYME DEFICIENCY; FEMALE; HLA SYSTEM; HUMAN; HURLER SYNDROME; LYSOSOME STORAGE DISEASE; MALE; MUCOPOLYSACCHARIDOSIS; NEUROPSYCHOLOGICAL TEST; PEROXISOME; PRIORITY JOURNAL; SURVIVAL TIME; TREATMENT OUTCOME;

EID: 9344245169     PISSN: 00064971     EISSN: None     Source Type: Journal    
DOI: 10.1182/blood.v87.11.4894.bloodjournal87114894     Document Type: Article
Times cited : (291)

References (57)
  • 1
    • 0000820862 scopus 로고
    • The mucopolysaccharidoses
    • Scriver CR, Beaudet AL, Sly WS, Valle D (eds): New York, NY, McGraw-Hill
    • Neufeld E, Muenzer J: The mucopolysaccharidoses, in Scriver CR, Beaudet AL, Sly WS, Valle D (eds): The Metabolie and Molecular Bases of Inherited Disease, vol 2. New York, NY, McGraw-Hill, 1995, p 2465
    • (1995) The Metabolie and Molecular Bases of Inherited Disease , vol.2 , pp. 2465
    • Neufeld, E.1    Muenzer, J.2
  • 2
    • 0000841497 scopus 로고
    • An electron microscopic and biochemical study of Type II glycogenosis
    • Baudhuin P, Hers HG, Loeb H: An electron microscopic and biochemical study of Type II glycogenosis. Lab Invest 13:1139, 1964
    • (1964) Lab Invest , vol.13 , pp. 1139
    • Baudhuin, P.1    Hers, H.G.2    Loeb, H.3
  • 4
    • 0015101540 scopus 로고
    • Effect of leukocyte transfusion in a child with type II mucopolysaccharidosis
    • Knudson AG, Di Ferrante N, Curtis JE: Effect of leukocyte transfusion in a child with type II mucopolysaccharidosis. Proc Natl Acad Sci USA 68:1738, 1971
    • (1971) Proc Natl Acad Sci USA , vol.68 , pp. 1738
    • Knudson, A.G.1    Di Ferrante, N.2    Curtis, J.E.3
  • 5
    • 0000966871 scopus 로고
    • Central nervous system reconstitution after bone marrow transplantation for lysosomal and peroxisomal storage diseases
    • Rich RR, Fleisher TA, Schwartz BD, Shearer WT, Strober W (eds): St. Louis, MO, Mosby
    • Krivit W, Sung JH, Lockman LA, Shapiro EG: Central nervous system reconstitution after bone marrow transplantation for lysosomal and peroxisomal storage diseases, in Rich RR, Fleisher TA, Schwartz BD, Shearer WT, Strober W (eds): Principles of Clinical Immunology, vol 2. St. Louis, MO, Mosby, 1995, p 1852
    • (1995) Principles of Clinical Immunology , vol.2 , pp. 1852
    • Krivit, W.1    Sung, J.H.2    Lockman, L.A.3    Shapiro, E.G.4
  • 7
    • 0022526817 scopus 로고
    • Bone marrow transplantation for Hurler syndrome: Assessment of metabolic correction
    • Whitley CB, Ramsay NK, Kersey JH, Krivit W: Bone marrow transplantation for Hurler syndrome: assessment of metabolic correction. Birth Defects 22:7, 1986
    • (1986) Birth Defects , vol.22 , pp. 7
    • Whitley, C.B.1    Ramsay, N.K.2    Kersey, J.H.3    Krivit, W.4
  • 10
    • 0022875823 scopus 로고
    • Psychomotor development of children with mucopolysaccharidosis type I-H following bone marrow transplantation
    • Hugh-Jones K: Psychomotor development of children with mucopolysaccharidosis type I-H following bone marrow transplantation. Birth Defects 22:25, 1986
    • (1986) Birth Defects , vol.22 , pp. 25
    • Hugh-Jones, K.1
  • 16
    • 0345692186 scopus 로고
    • A summary of 75 patients with storage disease treated by BMT
    • Hobbs JR (ed): London, UK, COGENT
    • Krivit W: A summary of 75 patients with storage disease treated by BMT, in Hobbs JR (ed): Correction of Certain Genetic Diseases by Transplantation. London, UK, COGENT, 1989, p 179
    • (1989) Correction of Certain Genetic Diseases by Transplantation , pp. 179
    • Krivit, W.1
  • 17
    • 0025101032 scopus 로고
    • Normal intraocular pressure after a bone marrow transplant in glaucoma associated with mucopolysaccharidosis Type I-H
    • Christiansen SP, Smith TJ, Henslee-Downey PJ: Normal intraocular pressure after a bone marrow transplant in glaucoma associated with mucopolysaccharidosis Type I-H. Am J Ophthalmol 109:230, 1990
    • (1990) Am J Ophthalmol , vol.109 , pp. 230
    • Christiansen, S.P.1    Smith, T.J.2    Henslee-Downey, P.J.3
  • 19
    • 0025906859 scopus 로고
    • Bone marrow transplant in a case of mucopolysaccharidosis I Scheie phenotype: Skin ultrastructure before and after transplantation
    • Navarro C, Dominguez C, Costa M, Onega JJ: Bone marrow transplant in a case of mucopolysaccharidosis I Scheie phenotype: Skin ultrastructure before and after transplantation. Acta Neuropathol 82:33, 1991
    • (1991) Acta Neuropathol , vol.82 , pp. 33
    • Navarro, C.1    Dominguez, C.2    Costa, M.3    Onega, J.J.4
  • 20
    • 0002776718 scopus 로고
    • Bone marrow transplantation for storage diseases
    • Desnick RJ (ed): New York, NY, Churchill-Livingstone
    • Krivit W, Shapiro EG: Bone marrow transplantation for storage diseases, in Desnick RJ (ed): Treatment of Genetic Disease. New York, NY, Churchill-Livingstone, 1991, p 203
    • (1991) Treatment of Genetic Disease , pp. 203
    • Krivit, W.1    Shapiro, E.G.2
  • 21
    • 0026511701 scopus 로고
    • Hypertrophic cardiomyopathy in mucopolysaccharidoses: Regression after bone marrow transplantation
    • Vinallonga X, Sanz N, Balaguer A, Miro L, Ortega JJ, Casaldaliga J: Hypertrophic cardiomyopathy in mucopolysaccharidoses: Regression after bone marrow transplantation. Pediatr Cardiol 13:107, 1992
    • (1992) Pediatr Cardiol , vol.13 , pp. 107
    • Vinallonga, X.1    Sanz, N.2    Balaguer, A.3    Miro, L.4    Ortega, J.J.5    Casaldaliga, J.6
  • 22
    • 0026753902 scopus 로고
    • State of the art review. Bone marrow transplantation treatment for storage diseases
    • Krivit W, Shapiro E, Hoogerbrugge PM, Moser HW: State of the art review. Bone marrow transplantation treatment for storage diseases. Bone Marrow Transplant 10:87, 1992 (suppl 1)
    • (1992) Bone Marrow Transplant , vol.10 , Issue.1 SUPPL. , pp. 87
    • Krivit, W.1    Shapiro, E.2    Hoogerbrugge, P.M.3    Moser, H.W.4
  • 23
    • 0001851796 scopus 로고
    • Marrow transplantation for storage diseases: The plan and need for a control study
    • Forman S, Thomas ED, Blume K (eds): Cambridge, MA, Blackwell
    • Krivit W, Shapiro EG: Marrow transplantation for storage diseases: The plan and need for a control study, in Forman S, Thomas ED, Blume K (eds): Bone Marrow Transplantation. Cambridge, MA, Blackwell, 1993, p 883
    • (1993) Bone Marrow Transplantation , pp. 883
    • Krivit, W.1    Shapiro, E.G.2
  • 27
    • 0029146242 scopus 로고
    • Neuropsychological outcomes of several storage diseases with and without bone marrow transplantation
    • Shapiro EG, Lockman LA, Balthazor M, Krivit W: Neuropsychological outcomes of several storage diseases with and without bone marrow transplantation. J Inherit Metab Dis 18:413, 1995
    • (1995) J Inherit Metab Dis , vol.18 , pp. 413
    • Shapiro, E.G.1    Lockman, L.A.2    Balthazor, M.3    Krivit, W.4
  • 28
    • 0027514178 scopus 로고
    • National Marrow Donor Program: Progress and challenges
    • Champlin R, Coppo P, Howe C: National Marrow Donor Program: Progress and challenges. Bone Marrow Transplant 11:41, 1993 (suppl 1)
    • (1993) Bone Marrow Transplant , vol.11 , Issue.1 SUPPL. , pp. 41
    • Champlin, R.1    Coppo, P.2    Howe, C.3
  • 29
    • 2542631815 scopus 로고
    • High-resolution analysis of the human HLA-DR polymorphism by hybridization with sequence-specific oligonucleotide probes
    • Angelini G, de Preval C, Gorski J, Mach B: High-resolution analysis of the human HLA-DR polymorphism by hybridization with sequence-specific oligonucleotide probes. Proc Natl Acad Sci USA 83:4489, 1986
    • (1986) Proc Natl Acad Sci USA , vol.83 , pp. 4489
    • Angelini, G.1    De Preval, C.2    Gorski, J.3    Mach, B.4
  • 30
    • 0018405128 scopus 로고
    • A fluorometric assay using 4-methylumbelliferyl α-L-iduronide for the estimation of α-L-iduronidase activity and the detection of Hurler and Scheie syndromes
    • Hopwood JJ, Muller V, Smithson A, Baggett N: A fluorometric assay using 4-methylumbelliferyl α-L-iduronide for the estimation of α-L-iduronidase activity and the detection of Hurler and Scheie syndromes. Clin Chim Acta 92:257, 1979
    • (1979) Clin Chim Acta , vol.92 , pp. 257
    • Hopwood, J.J.1    Muller, V.2    Smithson, A.3    Baggett, N.4
  • 32
    • 0022408283 scopus 로고
    • Restriction fragment length polymorphisms as markers of engraftment in allogeneic marrow transplantation
    • Blazar BR, Orr HT, Arthur DC, Kersey JH, Filipovich AH: Restriction fragment length polymorphisms as markers of engraftment in allogeneic marrow transplantation. Blood 66:1436, 1985
    • (1985) Blood , vol.66 , pp. 1436
    • Blazar, B.R.1    Orr, H.T.2    Arthur, D.C.3    Kersey, J.H.4    Filipovich, A.H.5
  • 35
    • 0006247743 scopus 로고
    • Childhood dementia: Neuropsychological assessment and treatment of degenerative childhood diseases
    • Tramontana M, Hooper S (eds): New York, NY, Springer-Verlag
    • Shapiro E, Klein K: Childhood dementia: Neuropsychological assessment and treatment of degenerative childhood diseases, in Tramontana M, Hooper S (eds): Advances in Child Neuropsychology, New York, NY, Springer-Verlag, 1993, p 119
    • (1993) Advances in Child Neuropsychology , pp. 119
    • Shapiro, E.1    Klein, K.2
  • 40
    • 0028359859 scopus 로고
    • Minnesota Preschool Affect Rating Scales: Development, reliability, and validity
    • Shapiro EG, McPhee JT, Abbott AA, Sulzbacher SI: Minnesota Preschool Affect Rating Scales: Development, reliability, and validity. J Pediatr Psychol 19:325, 1994
    • (1994) J Pediatr Psychol , vol.19 , pp. 325
    • Shapiro, E.G.1    McPhee, J.T.2    Abbott, A.A.3    Sulzbacher, S.I.4
  • 43
    • 33845382806 scopus 로고
    • Nonparametric estimation from incomplete observations
    • Kaplan E, Meier P: Nonparametric estimation from incomplete observations. J Am Stat Assoc 53:457, 1958
    • (1958) J Am Stat Assoc , vol.53 , pp. 457
    • Kaplan, E.1    Meier, P.2
  • 47
    • 0029143678 scopus 로고
    • Microglia: The effector cell for reconstitution of the central nervous system following bone marrow transplantation for lysosomal and peroxisomal storage diseases
    • Krivit W, Sung JH, Shapiro EG, Lockman LA: Microglia: The effector cell for reconstitution of the central nervous system following bone marrow transplantation for lysosomal and peroxisomal storage diseases. Cell Transplant 4:385, 1995
    • (1995) Cell Transplant , vol.4 , pp. 385
    • Krivit, W.1    Sung, J.H.2    Shapiro, E.G.3    Lockman, L.A.4
  • 52
    • 0020591822 scopus 로고
    • Removal of T cells from bone marrow for transplantation: A monoclonal anti-lymphocyte antibody that fixes human complement
    • Hale G, Bright S, Chumbley G, Hoang T, Metcalf D, Munro AJ, Waldmann H: Removal of T cells from bone marrow for transplantation: A monoclonal anti-lymphocyte antibody that fixes human complement. Blood 62:873, 1983
    • (1983) Blood , vol.62 , pp. 873
    • Hale, G.1    Bright, S.2    Chumbley, G.3    Hoang, T.4    Metcalf, D.5    Munro, A.J.6    Waldmann, H.7
  • 57
    • 9344258233 scopus 로고
    • Unrelated marrow transplantation in patients with Hurler syndrome and leukodystrophy: Results of a phase 1 trial evaluating busulfan, cytoxan and total body irradiation
    • abstr
    • Wagner J, Krivit W: Unrelated marrow transplantation in patients with Hurler syndrome and leukodystrophy: Results of a phase 1 trial evaluating busulfan, cytoxan and total body irradiation. Blood 84:394a, 1994 (abstr, suppl 1)
    • (1994) Blood , vol.84 , Issue.1 SUPPL.
    • Wagner, J.1    Krivit, W.2


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.