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Article: Umbilical cord blood transplantation for Maroteaux-Lamy syndrome (mucopolysaccharidosis type VI)

TitleUmbilical cord blood transplantation for Maroteaux-Lamy syndrome (mucopolysaccharidosis type VI)
Authors
Issue Date2000
PublisherNature Publishing Group. The Journal's web site is located at http://www.nature.com/bmt
Citation
Bone Marrow Transplantation, 2000, v. 26 n. 4, p. 455-458 How to Cite?
AbstractSevere Maroteaux-Lamy syndrome (mucopolysaccharidosis type VI) is usually fatal by early adulthood. Bone marrow transplantation is the only form of definitive enzyme replacement therapy available. A 5-year-old boy with Maroteaux-Lamy syndrome has successful recovery of bone marrow and enzymatic functions after umbilical cord blood transplant from his unaffected HLA-identical brother. Busulphan (16 mg/kg) and cyclophosphamide (200 mg/kg) were used as preparative chemotherapy with short methotrexate and long cyclosporin as prophylaxis against graft-versus-host disease (GVHD). A total of 6.08 x 10 7/kg nucleated cells and 2.92 x 10 5/kg CD34 + cells were transplanted with neutrophil engraftment achieved on day 26. There was no evidence of acute and chronic GVHD. Fifteen months after transplant, a normal level of N-acetylgalactosamine-4-sulphatase activity was achieved despite mixed chimerism. There was clinical improvement of hepatosplenomegaly, facial and skin features, joint mobility and resolution of suppurative middle ear effusion. He returned to school and continued to perform well in academic studies. We report here the first successful umbilical cord blood transplant as treatment of Maroteaux-Lamy syndrome.
Persistent Identifierhttp://hdl.handle.net/10722/148189
ISSN
2015 Impact Factor: 3.636
2015 SCImago Journal Rankings: 1.585
ISI Accession Number ID
References

 

DC FieldValueLanguage
dc.contributor.authorLee, Ven_US
dc.contributor.authorLi, CKen_US
dc.contributor.authorShing, MMKen_US
dc.contributor.authorChik, KWen_US
dc.contributor.authorLam, CWen_US
dc.contributor.authorTsang, KSen_US
dc.contributor.authorPong, Hen_US
dc.contributor.authorHuen, KFen_US
dc.contributor.authorYuen, PMPen_US
dc.date.accessioned2012-05-29T06:11:22Z-
dc.date.available2012-05-29T06:11:22Z-
dc.date.issued2000en_US
dc.identifier.citationBone Marrow Transplantation, 2000, v. 26 n. 4, p. 455-458en_US
dc.identifier.issn0268-3369en_US
dc.identifier.urihttp://hdl.handle.net/10722/148189-
dc.description.abstractSevere Maroteaux-Lamy syndrome (mucopolysaccharidosis type VI) is usually fatal by early adulthood. Bone marrow transplantation is the only form of definitive enzyme replacement therapy available. A 5-year-old boy with Maroteaux-Lamy syndrome has successful recovery of bone marrow and enzymatic functions after umbilical cord blood transplant from his unaffected HLA-identical brother. Busulphan (16 mg/kg) and cyclophosphamide (200 mg/kg) were used as preparative chemotherapy with short methotrexate and long cyclosporin as prophylaxis against graft-versus-host disease (GVHD). A total of 6.08 x 10 7/kg nucleated cells and 2.92 x 10 5/kg CD34 + cells were transplanted with neutrophil engraftment achieved on day 26. There was no evidence of acute and chronic GVHD. Fifteen months after transplant, a normal level of N-acetylgalactosamine-4-sulphatase activity was achieved despite mixed chimerism. There was clinical improvement of hepatosplenomegaly, facial and skin features, joint mobility and resolution of suppurative middle ear effusion. He returned to school and continued to perform well in academic studies. We report here the first successful umbilical cord blood transplant as treatment of Maroteaux-Lamy syndrome.en_US
dc.languageengen_US
dc.publisherNature Publishing Group. The Journal's web site is located at http://www.nature.com/bmten_US
dc.relation.ispartofBone Marrow Transplantationen_US
dc.subject.meshAdulten_US
dc.subject.meshAntigens, Cd34 - Blooden_US
dc.subject.meshCells, Cultureden_US
dc.subject.meshChild, Preschoolen_US
dc.subject.meshFamily Healthen_US
dc.subject.meshFemaleen_US
dc.subject.meshFetal Blooden_US
dc.subject.meshFibroblasts - Enzymologyen_US
dc.subject.meshGraft Survivalen_US
dc.subject.meshHematopoietic Stem Cell Transplantation - Methodsen_US
dc.subject.meshHumansen_US
dc.subject.meshLeukocytes - Enzymologyen_US
dc.subject.meshMaleen_US
dc.subject.meshMucopolysaccharidosis Vi - Therapyen_US
dc.subject.meshN-Acetylgalactosamine-4-Sulfatase - Blooden_US
dc.subject.meshNuclear Familyen_US
dc.subject.meshTransplantation Chimera - Blooden_US
dc.titleUmbilical cord blood transplantation for Maroteaux-Lamy syndrome (mucopolysaccharidosis type VI)en_US
dc.typeArticleen_US
dc.identifier.emailLam, CW:ching-wanlam@pathology.hku.hken_US
dc.identifier.authorityLam, CW=rp00260en_US
dc.description.naturelink_to_subscribed_fulltexten_US
dc.identifier.pmid10982295-
dc.identifier.scopuseid_2-s2.0-0033841112en_US
dc.relation.referenceshttp://www.scopus.com/mlt/select.url?eid=2-s2.0-0033841112&selection=ref&src=s&origin=recordpageen_US
dc.identifier.volume26en_US
dc.identifier.issue4en_US
dc.identifier.spage455en_US
dc.identifier.epage458en_US
dc.identifier.isiWOS:000088837200015-
dc.publisher.placeUnited Kingdomen_US

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