메뉴 건너뛰기




Volumn 19, Issue 1 SUPPL., 2013, Pages

Multi-Institutional Assessments of Transplantation for Metabolic Disorders

Author keywords

[No Author keywords available]

Indexed keywords

ADRENOLEUKODYSTROPHY; ALLOGENEIC STEM CELL TRANSPLANTATION; ARTICLE; ENZYME ACTIVITY; ENZYME REPLACEMENT; EVENT FREE SURVIVAL; GRAFT FAILURE; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; HURLER SYNDROME; METABOLIC DISORDER; NUCLEAR MAGNETIC RESONANCE IMAGING; OUTCOME ASSESSMENT; SURVIVAL RATE; SURVIVAL TIME; TRANSPLANTATION;

EID: 84871875870     PISSN: 10838791     EISSN: 15236536     Source Type: Journal    
DOI: 10.1016/j.bbmt.2012.10.026     Document Type: Article
Times cited : (6)

References (32)
  • 1
    • 0014352329 scopus 로고
    • Hurler and Hunter syndromes: mutual correction of the defect in cultured fibroblasts
    • Fratantoni J.C., Hall C.W., Neufeld E.F. Hurler and Hunter syndromes: mutual correction of the defect in cultured fibroblasts. Science 1968, 162:570-572.
    • (1968) Science , vol.162 , pp. 570-572
    • Fratantoni, J.C.1    Hall, C.W.2    Neufeld, E.F.3
  • 2
    • 0019466750 scopus 로고
    • Reversal of clinical features of Hurler's disease and biochemical improvement after treatment by bone-marrow transplantation
    • Hobbs J.R., Hugh-Jones K., Barrett A.J., et al. Reversal of clinical features of Hurler's disease and biochemical improvement after treatment by bone-marrow transplantation. Lancet 1981, 2:709-712.
    • (1981) Lancet , vol.2 , pp. 709-712
    • Hobbs, J.R.1    Hugh-Jones, K.2    Barrett, A.J.3
  • 3
    • 0035718871 scopus 로고    scopus 로고
    • Enzyme replacement therapy in feline mucopolysaccharidosis I
    • Kakkis E.D., Schuchman E., He X., et al. Enzyme replacement therapy in feline mucopolysaccharidosis I. Mol Genet Metab 2001, 72:199-208.
    • (2001) Mol Genet Metab , vol.72 , pp. 199-208
    • Kakkis, E.D.1    Schuchman, E.2    He, X.3
  • 4
    • 17144399564 scopus 로고    scopus 로고
    • The first 5 years of clinical experience with laronidase enzyme replacement therapy for mucopolysaccharidosis I
    • Wraith J.E. The first 5 years of clinical experience with laronidase enzyme replacement therapy for mucopolysaccharidosis I. Expert Opin Pharmacother 2005, 6:489-506.
    • (2005) Expert Opin Pharmacother , vol.6 , pp. 489-506
    • Wraith, J.E.1
  • 5
    • 34547147899 scopus 로고    scopus 로고
    • Outcomes of hematopoietic stem cell transplantation for Hurler's syndrome in Europe: a risk factor analysis for graft failure
    • Boelens J.J., Wynn R.F., 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, 40:225-233.
    • (2007) Bone Marrow Transplant , vol.40 , pp. 225-233
    • Boelens, J.J.1    Wynn, R.F.2    O'Meara, A.3
  • 6
    • 31344470473 scopus 로고    scopus 로고
    • Results of the cord blood transplantation study (COBLT): outcomes of unrelated donor umbilical cord blood transplantation in pediatric patients with lysosomal and peroxisomal storage diseases
    • Martin P.L., Carter S.L., Kernan N.A., et al. Results of the cord blood transplantation study (COBLT): outcomes of unrelated donor umbilical cord blood transplantation in pediatric patients with lysosomal and peroxisomal storage diseases. Biol Blood Marrow Transplant 2006, 12:184-194.
    • (2006) Biol Blood Marrow Transplant , vol.12 , pp. 184-194
    • Martin, P.L.1    Carter, S.L.2    Kernan, N.A.3
  • 7
    • 53449089638 scopus 로고    scopus 로고
    • Unrelated donor umbilical cord blood transplantation for inherited metabolic disorders in 159 pediatric patients from a single center: influence of cellular composition of the graft on transplantation outcomes
    • Prasad V.K., Mendizabal A., Parikh S.H., et al. Unrelated donor umbilical cord blood transplantation for inherited metabolic disorders in 159 pediatric patients from a single center: influence of cellular composition of the graft on transplantation outcomes. Blood 2008, 112:2979-2989.
    • (2008) Blood , vol.112 , pp. 2979-2989
    • Prasad, V.K.1    Mendizabal, A.2    Parikh, S.H.3
  • 8
    • 2342535103 scopus 로고    scopus 로고
    • Cord-blood transplants from unrelated donors in patients with Hurler's syndrome
    • Staba S.L., Escolar M.L., Poe M., et al. Cord-blood transplants from unrelated donors in patients with Hurler's syndrome. N Engl J Med 2004, 350:1960-1969.
    • (2004) N Engl J Med , vol.350 , pp. 1960-1969
    • Staba, S.L.1    Escolar, M.L.2    Poe, M.3
  • 10
    • 81155162672 scopus 로고    scopus 로고
    • Outcomes of transplantation using a various cell source in children with hurlers syndrome after myelo-ablative conditioning. An Eurocord-EBMT-CIBMTR collaborative study
    • Boelens J., Aldenhoven M., Purtill D., et al. Outcomes of transplantation using a various cell source in children with hurlers syndrome after myelo-ablative conditioning. An Eurocord-EBMT-CIBMTR collaborative study. Biol Blood Marrow Transplant 2010, 16(Suppl 2):S180-S181.
    • (2010) Biol Blood Marrow Transplant , vol.16 , Issue.SUPPL. 2
    • Boelens, J.1    Aldenhoven, M.2    Purtill, D.3
  • 11
    • 28444488536 scopus 로고    scopus 로고
    • Human umbilical cord blood progenitors: the potential of these hematopoietic cells to become neural
    • Chen N., Hudson J.E., Walczak P., et al. Human umbilical cord blood progenitors: the potential of these hematopoietic cells to become neural. Stem Cells 2005, 23:1560-1570.
    • (2005) Stem Cells , vol.23 , pp. 1560-1570
    • Chen, N.1    Hudson, J.E.2    Walczak, P.3
  • 12
    • 3242755834 scopus 로고    scopus 로고
    • A new human somatic stem cell from placental cord blood with intrinsic pluripotent differentiation potential
    • Kogler G., Sensken S., Airey J.A., et al. A new human somatic stem cell from placental cord blood with intrinsic pluripotent differentiation potential. J Exp Med 2004, 200:123-135.
    • (2004) J Exp Med , vol.200 , pp. 123-135
    • Kogler, G.1    Sensken, S.2    Airey, J.A.3
  • 13
    • 70449719317 scopus 로고    scopus 로고
    • Musculoskeletal manifestations of lysosomal storage disorders
    • Aldenhoven M., Sakkers R.J., Boelens J., et al. Musculoskeletal manifestations of lysosomal storage disorders. Ann Rheum Dis 2009, 68:1659-1665.
    • (2009) Ann Rheum Dis , vol.68 , pp. 1659-1665
    • Aldenhoven, M.1    Sakkers, R.J.2    Boelens, J.3
  • 14
    • 33947673920 scopus 로고    scopus 로고
    • Rheumatologic aspects of lysosomal storage diseases
    • Manger B., Mengel E., Schaefer R.M. Rheumatologic aspects of lysosomal storage diseases. Clin Rheumatol 2007, 26:335-341.
    • (2007) Clin Rheumatol , vol.26 , pp. 335-341
    • Manger, B.1    Mengel, E.2    Schaefer, R.M.3
  • 15
    • 0017265264 scopus 로고
    • Fatty acid abnormality in adrenoleukodystrophy
    • Igarashi M., Schaumburg H.H., Powers J., et al. Fatty acid abnormality in adrenoleukodystrophy. J Neurochem 1976, 26:851-860.
    • (1976) J Neurochem , vol.26 , pp. 851-860
    • Igarashi, M.1    Schaumburg, H.H.2    Powers, J.3
  • 16
    • 0035071054 scopus 로고    scopus 로고
    • Adrenoleukodystrophy: incidence, new mutation rate, and results of extended family screening
    • Bezman L., Moser A.B., Raymond G.V., et al. Adrenoleukodystrophy: incidence, new mutation rate, and results of extended family screening. Ann Neurol 2001, 49:512-517.
    • (2001) Ann Neurol , vol.49 , pp. 512-517
    • Bezman, L.1    Moser, A.B.2    Raymond, G.V.3
  • 17
    • 28344455215 scopus 로고    scopus 로고
    • X-linked adrenoleukodystrophy: Therapeutic approaches to distinct phenotypes
    • Mahmood A., Dubey P., Moser H.W., Moser A. X-linked adrenoleukodystrophy: Therapeutic approaches to distinct phenotypes. Pediatr Transplant 2005, 9:55-62.
    • (2005) Pediatr Transplant , vol.9 , pp. 55-62
    • Mahmood, A.1    Dubey, P.2    Moser, H.W.3    Moser, A.4
  • 18
    • 33644793371 scopus 로고    scopus 로고
    • Adrenoleukodystrophy: oxidative stress of mice and men
    • Powers J.M., Pei Z., Heinzer A.K., et al. Adrenoleukodystrophy: oxidative stress of mice and men. J Neuropathol Exp Neurol 2005, 64:1067-1079.
    • (2005) J Neuropathol Exp Neurol , vol.64 , pp. 1067-1079
    • Powers, J.M.1    Pei, Z.2    Heinzer, A.K.3
  • 19
    • 0025329012 scopus 로고
    • Reversal of early neurologic and neuroradiologic manifestations of X-linked adrenoleukodystrophy by bone marrow transplantation
    • Aubourg P., Blanche S., Jambaque I., et al. Reversal of early neurologic and neuroradiologic manifestations of X-linked adrenoleukodystrophy by bone marrow transplantation. N Engl J Med 1990, 322:1860-1866.
    • (1990) N Engl J Med , vol.322 , pp. 1860-1866
    • Aubourg, P.1    Blanche, S.2    Jambaque, I.3
  • 20
    • 70449427834 scopus 로고    scopus 로고
    • Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy
    • Cartier N., Hacein-Bey-Abina S., Bartholomae C.C., et al. Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy. Science 2009, 326:818-823.
    • (2009) Science , vol.326 , pp. 818-823
    • Cartier, N.1    Hacein-Bey-Abina, S.2    Bartholomae, C.C.3
  • 21
    • 80051884762 scopus 로고    scopus 로고
    • Outcomes after allogeneic hematopoietic cell transplantation for childhood cerebral adrenoleukodystrophy: the largest single-institution cohort report
    • Miller W.P., Rothman S.M., Nascene D., et al. Outcomes after allogeneic hematopoietic cell transplantation for childhood cerebral adrenoleukodystrophy: the largest single-institution cohort report. Blood 2011, 118:1971-1978.
    • (2011) Blood , vol.118 , pp. 1971-1978
    • Miller, W.P.1    Rothman, S.M.2    Nascene, D.3
  • 22
    • 3242762263 scopus 로고    scopus 로고
    • Cerebral X-linked adrenoleukodystrophy: the international hematopoietic cell transplantation experience from 1982 to 1999
    • Peters C., Charnas L.R., Tan Y., et al. Cerebral X-linked adrenoleukodystrophy: the international hematopoietic cell transplantation experience from 1982 to 1999. Blood 2004, 104:881-888.
    • (2004) Blood , vol.104 , pp. 881-888
    • Peters, C.1    Charnas, L.R.2    Tan, Y.3
  • 23
    • 0041624042 scopus 로고    scopus 로고
    • Analysis of MRI patterns aids prediction of progression in X-linked adrenoleukodystrophy
    • Loes D.J., Fatemi A., Melhem E.R., et al. Analysis of MRI patterns aids prediction of progression in X-linked adrenoleukodystrophy. Neurology 2003, 61:369-374.
    • (2003) Neurology , vol.61 , pp. 369-374
    • Loes, D.J.1    Fatemi, A.2    Melhem, E.R.3
  • 24
    • 0028071898 scopus 로고
    • Childhood cerebral form of adrenoleukodystrophy: short-term effect of bone marrow transplantation on brain MR observations
    • Loes D.J., Stillman A.E., Hite S., et al. Childhood cerebral form of adrenoleukodystrophy: short-term effect of bone marrow transplantation on brain MR observations. AJNR Am J Neuroradiol 1994, 15:1767-1771.
    • (1994) AJNR Am J Neuroradiol , vol.15 , pp. 1767-1771
    • Loes, D.J.1    Stillman, A.E.2    Hite, S.3
  • 25
    • 3142688107 scopus 로고    scopus 로고
    • Evaluation of therapy of X-linked adrenoleukodystrophy
    • Moser H.W., Fatemi A., Zackowski K., et al. Evaluation of therapy of X-linked adrenoleukodystrophy. Neurochem Res 2004, 29:1003-1016.
    • (2004) Neurochem Res , vol.29 , pp. 1003-1016
    • Moser, H.W.1    Fatemi, A.2    Zackowski, K.3
  • 27
    • 79960205823 scopus 로고    scopus 로고
    • Glycosaminoglycan storage in neuroanatomical regions of mucopolysaccharidosis I dogs following intrathecal recombinant human iduronidase
    • Chen A., Vogler C., McEntee M., et al. Glycosaminoglycan storage in neuroanatomical regions of mucopolysaccharidosis I dogs following intrathecal recombinant human iduronidase. APMIS Acta Pathol Microbiol Immunol Scand 2011, 119:513-521.
    • (2011) APMIS Acta Pathol Microbiol Immunol Scand , vol.119 , pp. 513-521
    • Chen, A.1    Vogler, C.2    McEntee, M.3
  • 28
    • 80054687694 scopus 로고    scopus 로고
    • Chitotriosidase as a biomarker of cerebral adrenoleukodystrophy
    • Orchard P.J., Lund T., Miller W., et al. Chitotriosidase as a biomarker of cerebral adrenoleukodystrophy. J Neuroinflamm 2011, 8:144.
    • (2011) J Neuroinflamm , vol.8 , pp. 144
    • Orchard, P.J.1    Lund, T.2    Miller, W.3
  • 29
    • 0034488655 scopus 로고    scopus 로고
    • X-Linked adrenoleukodystrophy: overview and prognosis as a function of age and brain magnetic resonance imaging abnormality. A study involving 372 patients
    • Moser H.W., Loes D.J., Melhem E.R., et al. X-Linked adrenoleukodystrophy: overview and prognosis as a function of age and brain magnetic resonance imaging abnormality. A study involving 372 patients. Neuropediatrics 2000, 31:227-239.
    • (2000) Neuropediatrics , vol.31 , pp. 227-239
    • Moser, H.W.1    Loes, D.J.2    Melhem, E.R.3
  • 30
    • 0035138765 scopus 로고    scopus 로고
    • Evolution of phenotypes in adult male patients with X-linked adrenoleukodystrophy
    • van Geel B.M., Bezman L., Loes D.J., et al. Evolution of phenotypes in adult male patients with X-linked adrenoleukodystrophy. Ann Neurol 2001, 49:186-194.
    • (2001) Ann Neurol , vol.49 , pp. 186-194
    • van Geel, B.M.1    Bezman, L.2    Loes, D.J.3
  • 31
    • 0028124904 scopus 로고
    • Adrenoleukodystrophy: a scoring method for brain MR observations
    • Loes D.J., Hite S., Moser H., et al. Adrenoleukodystrophy: a scoring method for brain MR observations. AJNR Am J Neuroradiol 1994, 15:1761-1766.
    • (1994) AJNR Am J Neuroradiol , vol.15 , pp. 1761-1766
    • Loes, D.J.1    Hite, S.2    Moser, H.3
  • 32
    • 0034038327 scopus 로고    scopus 로고
    • Therapy of X-linked adrenoleukodystrophy: prognosis based upon age and MRI abnormality and plans for placebo-controlled trials
    • Moser H.W., Bezman L., Lu S.E., Raymond G.V. Therapy of X-linked adrenoleukodystrophy: prognosis based upon age and MRI abnormality and plans for placebo-controlled trials. J Inherit Metab Dis 2000, 23:273-277.
    • (2000) J Inherit Metab Dis , vol.23 , pp. 273-277
    • Moser, H.W.1    Bezman, L.2    Lu, S.E.3    Raymond, G.V.4


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.