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Volumn 12, Issue , 2017, Pages 62-68

Early hematopoietic stem cell transplantation in a patient with severe mucopolysaccharidosis II: A 7 years follow-up

Author keywords

Hematopoietic stem cell transplantation; Mucopolysaccharidosis; Neurocognition

Indexed keywords

AMPICILLIN; BUSULFAN; CEFOTAXIME; CEFTRIAXONE; CORTICOSTEROID; CYCLOPHOSPHAMIDE; CYCLOSPORIN; GENTAMICIN; GLYCOSAMINOGLYCAN; IDURONATE 2 SULFATASE; THYMOCYTE ANTIBODY;

EID: 85020240310     PISSN: None     EISSN: 22144269     Source Type: Journal    
DOI: 10.1016/j.ymgmr.2017.05.010     Document Type: Article
Times cited : (38)

References (32)
  • 1
    • 0000869162 scopus 로고    scopus 로고
    • The Mucopolysaccharidoses
    • C.R. Scriver A.L. Beaudet W.S. Sly D. Valle 8th ed McGrow-Hill New York
    • Neufeld, E.F., Muenzer, J., The Mucopolysaccharidoses. Scriver, C.R., Beaudet, A.L., Sly, W.S., Valle, D., (eds.) The Metabolic and Molecular Bases of Inherited Disease, 8th ed, 2001, McGrow-Hill, New York, 3421–3452.
    • (2001) The Metabolic and Molecular Bases of Inherited Disease , pp. 3421-3452
    • Neufeld, E.F.1    Muenzer, J.2
  • 2
    • 79955493662 scopus 로고    scopus 로고
    • Natural progression of neurological disease in mucopolisaccharidosis type II
    • Holt, J.B., Poe, M.D., Escolar, M.L., Natural progression of neurological disease in mucopolisaccharidosis type II. Pediatrics 127 (2011), e1258–e1265.
    • (2011) Pediatrics , vol.127 , pp. e1258-e1265
    • Holt, J.B.1    Poe, M.D.2    Escolar, M.L.3
  • 3
    • 84857061064 scopus 로고    scopus 로고
    • The role of enzyme replacement therapy in severe Hunter syndrome – an expert panel consensus
    • Muenzer, J., Bodamer, O., Burton, B., Clarke, L., The role of enzyme replacement therapy in severe Hunter syndrome – an expert panel consensus. Eur. J. Pediatr. 171 (2012), 181–188.
    • (2012) Eur. J. Pediatr. , vol.171 , pp. 181-188
    • Muenzer, J.1    Bodamer, O.2    Burton, B.3    Clarke, L.4
  • 4
    • 84885998729 scopus 로고    scopus 로고
    • Blood-brain barrier molecular Trojan horse enables imaging of brain uptake of radioiodinated recombinant protein in the rhesus monkey
    • Boado, R.J., Hui, E.K., Lu, J.Z., Sumbria, R.K., Pardridge, W.M., Blood-brain barrier molecular Trojan horse enables imaging of brain uptake of radioiodinated recombinant protein in the rhesus monkey. Bioconjug. Chem. 24 (2013), 1741–1749.
    • (2013) Bioconjug. Chem. , vol.24 , pp. 1741-1749
    • Boado, R.J.1    Hui, E.K.2    Lu, J.Z.3    Sumbria, R.K.4    Pardridge, W.M.5
  • 5
    • 84894631502 scopus 로고    scopus 로고
    • Long-term experience with enzyme replacement therapy (ERT) in MPS II patients with a severe phenotype: an international case
    • Lampe, C., Bosserhoff, A.K., Burton, B.K., Giugliani, R., Long-term experience with enzyme replacement therapy (ERT) in MPS II patients with a severe phenotype: an international case. J. Inherit. Metab. Dis. 37 (2014), 823–829.
    • (2014) J. Inherit. Metab. Dis. , vol.37 , pp. 823-829
    • Lampe, C.1    Bosserhoff, A.K.2    Burton, B.K.3    Giugliani, R.4
  • 6
    • 84926200256 scopus 로고    scopus 로고
    • Long-term outcome of Hurler syndrome patients after hematopoietic cell transplantation: an international multicenter study
    • Aldenhoven, M., Wynn, R.F., Orchard, P.J., O'Meara, A., Long-term outcome of Hurler syndrome patients after hematopoietic cell transplantation: an international multicenter study. Blood 125 (2015), 2164–2172.
    • (2015) Blood , vol.125 , pp. 2164-2172
    • Aldenhoven, M.1    Wynn, R.F.2    Orchard, P.J.3    O'Meara, A.4
  • 7
    • 84959544523 scopus 로고    scopus 로고
    • Enzyme replacement therapy prior to hematopoietic stem cell transplantation in mucopolysaccharidosis type I: 10 year combined experience of 2 centers
    • Gosh, A., Miller, W., Orchard, P.J., Jones, S.A., Enzyme replacement therapy prior to hematopoietic stem cell transplantation in mucopolysaccharidosis type I: 10 year combined experience of 2 centers. Mol. Genet. Metab. 117 (2016), 373–377.
    • (2016) Mol. Genet. Metab. , vol.117 , pp. 373-377
    • Gosh, A.1    Miller, W.2    Orchard, P.J.3    Jones, S.A.4
  • 8
    • 84912535118 scopus 로고    scopus 로고
    • Early treatment is associated with improved cognition in Hurler syndrome
    • Poe, M.D., Chagnon, S.L., Escolar, M.L., Early treatment is associated with improved cognition in Hurler syndrome. Ann. Neurol. 76 (2014), 747–753.
    • (2014) Ann. Neurol. , vol.76 , pp. 747-753
    • Poe, M.D.1    Chagnon, S.L.2    Escolar, M.L.3
  • 9
    • 81155157525 scopus 로고    scopus 로고
    • Enzyme replacement therapy and/or hematopoietic stem cell transplantation at diagnosis in patients with mucopolysaccharidosis type I: results of a European consensus procedure
    • de Ru, M.H., Boelens, J.J., Das, A.M., Jones, S.A., Enzyme replacement therapy and/or hematopoietic stem cell transplantation at diagnosis in patients with mucopolysaccharidosis type I: results of a European consensus procedure. Orphanet J. Rare Dis., 6, 2011, 55.
    • (2011) Orphanet J. Rare Dis. , vol.6 , pp. 55
    • de Ru, M.H.1    Boelens, J.J.2    Das, A.M.3    Jones, S.A.4
  • 10
    • 34447121276 scopus 로고    scopus 로고
    • Enzyme replacement therapy in patients who have mucopolysaccharidosis I and are younger than 5 years: results of a multinational study of recombinant human alpha-L-iduronidase (laronidase)
    • Wraith, J.E., Beck, M., Lane, R., van der Ploeg, A., Enzyme replacement therapy in patients who have mucopolysaccharidosis I and are younger than 5 years: results of a multinational study of recombinant human alpha-L-iduronidase (laronidase). Pediatrics 120 (2007), e37–e46.
    • (2007) Pediatrics , vol.120 , pp. e37-e46
    • Wraith, J.E.1    Beck, M.2    Lane, R.3    van der Ploeg, A.4
  • 11
    • 0037295890 scopus 로고    scopus 로고
    • Hematopoietic cell transplantation for inherited metabolic diseases: an overview of outcomes and practice guidelines
    • Peters, C., Sterward, C.G., Hematopoietic cell transplantation for inherited metabolic diseases: an overview of outcomes and practice guidelines. Bone Marrow Transplant. 31 (2003), 229–239.
    • (2003) Bone Marrow Transplant. , vol.31 , pp. 229-239
    • Peters, C.1    Sterward, C.G.2
  • 12
    • 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., Szabolcs, P., 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 112 (2008), 2979–2989.
    • (2008) Blood , vol.112 , pp. 2979-2989
    • Prasad, V.K.1    Mendizabal, A.2    Parikh, S.H.3    Szabolcs, P.4
  • 13
    • 62649094547 scopus 로고    scopus 로고
    • Improved metabolic correction in patients with lysosomal storage disease treated with hematopoietic stem cell transplant compared with enzyme replacement therapy
    • Wynn, R.F., Wraith, J.E., Mercer, J., Improved metabolic correction in patients with lysosomal storage disease treated with hematopoietic stem cell transplant compared with enzyme replacement therapy. J. Pediatr. 154 (2009), 609–611.
    • (2009) J. Pediatr. , vol.154 , pp. 609-611
    • Wynn, R.F.1    Wraith, J.E.2    Mercer, J.3
  • 14
    • 64549141555 scopus 로고    scopus 로고
    • Bone marrow transplantation in children with Hunter syndrome: outcome after 7 to 17 years
    • Guffon, N., Bertrand, Y., Forest, I., Fouilhoux, A., Froissart, R., Bone marrow transplantation in children with Hunter syndrome: outcome after 7 to 17 years. J. Pediatr. 154 (2009), 733–737.
    • (2009) J. Pediatr. , vol.154 , pp. 733-737
    • Guffon, N.1    Bertrand, Y.2    Forest, I.3    Fouilhoux, A.4    Froissart, R.5
  • 15
    • 0032998144 scopus 로고    scopus 로고
    • Long-term follow-up following bone marrow transplantation for Hunter disease
    • Vellodi, A., Young, E., Cooper, A., Lidchi, V., Long-term follow-up following bone marrow transplantation for Hunter disease. J. Inherit. Metab. Dis. 22 (1999), 638–648.
    • (1999) J. Inherit. Metab. Dis. , vol.22 , pp. 638-648
    • Vellodi, A.1    Young, E.2    Cooper, A.3    Lidchi, V.4
  • 16
    • 84992347210 scopus 로고    scopus 로고
    • Allogeneic hematopoietic stem cell transplantation in 34 pediatric cases of mucopolysaccharidosis – a 10-year report from China children transplant group
    • Wang, J., Luan, Z., Jiang, H., Fang, J., Allogeneic hematopoietic stem cell transplantation in 34 pediatric cases of mucopolysaccharidosis – a 10-year report from China children transplant group. Biol. Blood Marrow Transplant. 22 (2016), 2104–2108.
    • (2016) Biol. Blood Marrow Transplant. , vol.22 , pp. 2104-2108
    • Wang, J.1    Luan, Z.2    Jiang, H.3    Fang, J.4
  • 17
    • 84893664730 scopus 로고    scopus 로고
    • Muchopolysaccharidosis type II: identification of 30 novel mutation among Latin American patients
    • Brusius-Facchin, A.C., Schwartz, I.V.D., Zimmer, C., Ribeiro, M.G., Muchopolysaccharidosis type II: identification of 30 novel mutation among Latin American patients. Mol. Genet. Metab. 111 (2014), 133–138.
    • (2014) Mol. Genet. Metab. , vol.111 , pp. 133-138
    • Brusius-Facchin, A.C.1    Schwartz, I.V.D.2    Zimmer, C.3    Ribeiro, M.G.4
  • 18
    • 85009963976 scopus 로고    scopus 로고
    • Clinical, biochemical and molecular characteristics of Filipino patients with mucopolysaccharidosis type II – Hunter syndrome
    • Chiong, M.A.D., Canson, D.M., Abacan, M.A.R., Baluyot, M.M.P., Cordero, C.P., Silao, C.L.T., Clinical, biochemical and molecular characteristics of Filipino patients with mucopolysaccharidosis type II – Hunter syndrome. Orphanet J. Rare Dis., 12, 2017, 7.
    • (2017) Orphanet J. Rare Dis. , vol.12 , pp. 7
    • Chiong, M.A.D.1    Canson, D.M.2    Abacan, M.A.R.3    Baluyot, M.M.P.4    Cordero, C.P.5    Silao, C.L.T.6
  • 19
    • 33947578019 scopus 로고    scopus 로고
    • Mucopolysaccharidosis type II: an update on mutation spectrum
    • Froissart, R., da Silva, I.M., Maire, I., Mucopolysaccharidosis type II: an update on mutation spectrum. Acta Paediatr. 96 (2007), 71–77.
    • (2007) Acta Paediatr. , vol.96 , pp. 71-77
    • Froissart, R.1    da Silva, I.M.2    Maire, I.3
  • 20
    • 79956259900 scopus 로고    scopus 로고
    • Placenta analysis of prenatally diagnosed patients reveals early GAG storage in mucopolysaccharidoses II and VI
    • Baldo, G., Matte, U., Artigalas, O., Schwartz, I.V., Placenta analysis of prenatally diagnosed patients reveals early GAG storage in mucopolysaccharidoses II and VI. Mol. Genet. Metab. 103 (2011), 197–198.
    • (2011) Mol. Genet. Metab. , vol.103 , pp. 197-198
    • Baldo, G.1    Matte, U.2    Artigalas, O.3    Schwartz, I.V.4
  • 21
    • 84921663046 scopus 로고    scopus 로고
    • Enzyme replacement therapy in mucopolysaccharidosis II patients under 1 year of age
    • Lampe, C., Atherton, A., Burton, B.K., Descartes, M., Enzyme replacement therapy in mucopolysaccharidosis II patients under 1 year of age. JIMD Rep. 14 (2014), 99–113.
    • (2014) JIMD Rep. , vol.14 , pp. 99-113
    • Lampe, C.1    Atherton, A.2    Burton, B.K.3    Descartes, M.4
  • 22
    • 10744223978 scopus 로고    scopus 로고
    • Outcome of 27 patients with Hurler's syndrome transplanted either from related or unrelated haematopoietic stem cell sources
    • Souillet, G., Guffon, N., Maire, I., Pujol, M., Outcome of 27 patients with Hurler's syndrome transplanted either from related or unrelated haematopoietic stem cell sources. Bone Marrow Transplant. 31 (2003), 1105–1117.
    • (2003) Bone Marrow Transplant. , vol.31 , pp. 1105-1117
    • Souillet, G.1    Guffon, N.2    Maire, I.3    Pujol, M.4
  • 23
    • 85020283963 scopus 로고    scopus 로고
    • Neurophysiological evaluation in cervical myelopathy in patients with mucopolysaccharidosis: the somatosensory and motor-evoked potentials
    • Silva, D.S., Barth, A.L., Esposito, A.C., Costa, A.A.P., Neurophysiological evaluation in cervical myelopathy in patients with mucopolysaccharidosis: the somatosensory and motor-evoked potentials. JIEMS, 2, 2014, S25.
    • (2014) JIEMS , vol.2 , pp. S25
    • Silva, D.S.1    Barth, A.L.2    Esposito, A.C.3    Costa, A.A.P.4
  • 25
    • 84867901875 scopus 로고    scopus 로고
    • Long-term efficacy of hematopoietic stem cell transplantation on brain involvement in patients with mucopolysaccharidosis type II: a nationwide survey in Japan
    • Tanaka, A., Okuyama, T., Suzuki, Y., Sakai, N., Long-term efficacy of hematopoietic stem cell transplantation on brain involvement in patients with mucopolysaccharidosis type II: a nationwide survey in Japan. Mol. Genet. Metab. 107 (2012), 513–520.
    • (2012) Mol. Genet. Metab. , vol.107 , pp. 513-520
    • Tanaka, A.1    Okuyama, T.2    Suzuki, Y.3    Sakai, N.4
  • 26
    • 84928493208 scopus 로고    scopus 로고
    • Longterm outcomes of patients receiving umbilical blood stem cell transplantation for MPS II
    • Escolar, M., Poe, M., Rajan, D., Szabolcs, P., Longterm outcomes of patients receiving umbilical blood stem cell transplantation for MPS II. Mol. Genet. Metab. 108 (2012), S37–S38.
    • (2012) Mol. Genet. Metab. , vol.108 , pp. S37-S38
    • Escolar, M.1    Poe, M.2    Rajan, D.3    Szabolcs, P.4
  • 27
    • 0031908164 scopus 로고    scopus 로고
    • Otologic and laryngologic manifestations of mucopolysaccharidosis after bone marrow transplantation
    • Papsin, B.C., Vellodi, A., Bailey, C.M., Ratcliffe, P.C., Leighton, S.E., Otologic and laryngologic manifestations of mucopolysaccharidosis after bone marrow transplantation. Otolaryngol. Head Neck Surg. 118 (1998), 30–36.
    • (1998) Otolaryngol. Head Neck Surg. , vol.118 , pp. 30-36
    • Papsin, B.C.1    Vellodi, A.2    Bailey, C.M.3    Ratcliffe, P.C.4    Leighton, S.E.5
  • 28
    • 84869744327 scopus 로고    scopus 로고
    • Improvements in sensorineural hearing loss after cord blood transplant in patients with mucopolysaccharidosis
    • da Costa, V., O'Grady, G., Jackson, L., Kaylie, D., Raynor, E., Improvements in sensorineural hearing loss after cord blood transplant in patients with mucopolysaccharidosis. Arch. Otolaryngol. Head Neck Surg. 138 (2012), 1071–1076.
    • (2012) Arch. Otolaryngol. Head Neck Surg. , vol.138 , pp. 1071-1076
    • da Costa, V.1    O'Grady, G.2    Jackson, L.3    Kaylie, D.4    Raynor, E.5
  • 29
    • 79960344934 scopus 로고    scopus 로고
    • Pharmacological drugs inducing ototoxicity, vestibular symptoms and tinnitus: a reasoned and updated guide
    • Cianfrone, G., Pentangelo, D., CianfroneF, Mazzei F., Pharmacological drugs inducing ototoxicity, vestibular symptoms and tinnitus: a reasoned and updated guide. Eur. Rev. Med. Pharmacol. Sci. 15 (2011), 601–636.
    • (2011) Eur. Rev. Med. Pharmacol. Sci. , vol.15 , pp. 601-636
    • Cianfrone, G.1    Pentangelo, D.2    CianfroneF, M.F.3
  • 30
    • 84921644517 scopus 로고    scopus 로고
    • Activities of daily living in patients with Hunter syndrome: impact of enzyme replacement therapy and hematopoietic stem cell transplantation
    • Tanjuakio, J., Suzuki, Y., Patel, P., Yasuda, E., Activities of daily living in patients with Hunter syndrome: impact of enzyme replacement therapy and hematopoietic stem cell transplantation. Mol. Genet. Metab. 114 (2015), 161–169.
    • (2015) Mol. Genet. Metab. , vol.114 , pp. 161-169
    • Tanjuakio, J.1    Suzuki, Y.2    Patel, P.3    Yasuda, E.4
  • 31
    • 84929049500 scopus 로고    scopus 로고
    • Cord blood is the optimal graft source for the treatment of pediatric patients with lysosomal storage diseases: clinical outcomes and future directions
    • Aldenhoven, M., Kurtzberger, J., Cord blood is the optimal graft source for the treatment of pediatric patients with lysosomal storage diseases: clinical outcomes and future directions. Cytotherapy 17 (2015), 765–774.
    • (2015) Cytotherapy , vol.17 , pp. 765-774
    • Aldenhoven, M.1    Kurtzberger, J.2
  • 32
    • 84859393724 scopus 로고    scopus 로고
    • Stem cell transplantation in inherited metabolic disorders
    • Wynn, R., Stem cell transplantation in inherited metabolic disorders. Hematology Am. Soc. Hematol. Educ. Program 2011 (2011), 285–291.
    • (2011) Hematology Am. Soc. Hematol. Educ. Program , vol.2011 , pp. 285-291
    • Wynn, R.1


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