{"id":"https://openalex.org/W2090134286","doi":"https://doi.org/10.1016/j.bbmt.2004.12.286","title":"Combined home enzyme replacement therapy and unrelated cord blood transplantation for Hurler\u2019s syndrome (MPSI) in a pediatric HSCT center","display_name":"Combined home enzyme replacement therapy and unrelated cord blood transplantation for Hurler\u2019s syndrome (MPSI) in a pediatric HSCT center","publication_year":2005,"publication_date":"2005-01-29","ids":{"openalex":"https://openalex.org/W2090134286","doi":"https://doi.org/10.1016/j.bbmt.2004.12.286","mag":"2090134286"},"language":"en","primary_location":{"id":"doi:10.1016/j.bbmt.2004.12.286","doi":"https://doi.org/10.1016/j.bbmt.2004.12.286","is_oa":true,"landing_page_url":"https://doi.org/10.1016/j.bbmt.2004.12.286","pdf_url":"http://www.astctjournal.org/article/S1083879104009383/pdf","source":{"id":"https://openalex.org/S59987174","display_name":"Transplantation and Cellular Therapy","issn_l":"1083-8791","issn":["1083-8791","1523-6536","2666-6367"],"is_oa":false,"is_in_doaj":false,"is_core":true,"listed_in":["anvur-scientific","cwts-core","dongbi-b","doyens","jufo-1","ki-jl-1","medline","norway-1","poland-100","poland-20","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310320990","host_organization_name":"Elsevier BV","host_organization_lineage":["https://openalex.org/P4310320990"],"host_organization_lineage_names":["Elsevier BV"],"type":"journal"},"license":"cc-by-nc-nd","license_id":"https://openalex.org/licenses/cc-by-nc-nd","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Biology of Blood and Marrow Transplantation","raw_type":"journal-article"},"volume":null,"type":"article","is_traditional":true,"indexed_in":["crossref"],"open_access":{"is_oa":true,"oa_status":"hybrid","oa_url":"http://www.astctjournal.org/article/S1083879104009383/pdf","any_repository_has_fulltext":false},"authorships":[{"author_position":"first","author":{"id":"https://openalex.org/A5113681482","display_name":"Marie Rodriguez","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I145608581","display_name":"University of Miami","ror":"https://ror.org/02dgjyy92","country_code":"US","type":"education","lineage":["https://openalex.org/I145608581"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"M. Rodriguez","raw_affiliation_strings":["University of Miami School of Medicine, Miami, FL"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"University of Miami School of Medicine, Miami, FL","institution_ids":["https://openalex.org/I145608581"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5024439402","display_name":"I. Khimji","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I2802864460","display_name":"Jackson Memorial Hospital","ror":"https://ror.org/02y070a55","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I2802864460","https://openalex.org/I4210089954"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"I. Khimji","raw_affiliation_strings":["Jackson Memorial Hospital, Miami, FL","Jackson Memorial Hospital, Miami, FL#TAB#"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Jackson Memorial Hospital, Miami, FL","institution_ids":["https://openalex.org/I2802864460"]},{"raw_affiliation_string":"Jackson Memorial Hospital, Miami, FL#TAB#","institution_ids":["https://openalex.org/I2802864460"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5061296999","display_name":"S. Rhodd","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I2802864460","display_name":"Jackson Memorial Hospital","ror":"https://ror.org/02y070a55","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I2802864460","https://openalex.org/I4210089954"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"S. Rhodd","raw_affiliation_strings":["Jackson Memorial Hospital, Miami, FL","Jackson Memorial Hospital, Miami, FL#TAB#"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Jackson Memorial Hospital, Miami, FL","institution_ids":["https://openalex.org/I2802864460"]},{"raw_affiliation_string":"Jackson Memorial Hospital, Miami, FL#TAB#","institution_ids":["https://openalex.org/I2802864460"]}]},{"author_position":"middle","author":{"id":null,"display_name":"C. Lapage","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I2802864460","display_name":"Jackson Memorial Hospital","ror":"https://ror.org/02y070a55","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I2802864460","https://openalex.org/I4210089954"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"C. Lapage","raw_affiliation_strings":["Jackson Memorial Hospital, Miami, FL","Jackson Memorial Hospital, Miami, FL#TAB#"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Jackson Memorial Hospital, Miami, FL","institution_ids":["https://openalex.org/I2802864460"]},{"raw_affiliation_string":"Jackson Memorial Hospital, Miami, FL#TAB#","institution_ids":["https://openalex.org/I2802864460"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5035080055","display_name":"L. Zalesky","orcid":null,"observed_orcids":[]},"institutions":[],"countries":[],"is_corresponding":false,"raw_author_name":"L. Zalesky","raw_affiliation_strings":["Accredo Therapeutics, Fort Lauderdale, FL"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Accredo Therapeutics, Fort Lauderdale, FL","institution_ids":[]}]},{"author_position":"middle","author":{"id":null,"display_name":"P. Jimenez","orcid":null,"observed_orcids":[]},"institutions":[],"countries":[],"is_corresponding":false,"raw_author_name":"P. Jimenez","raw_affiliation_strings":["Accredo Therapeutics, Fort Lauderdale, FL"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Accredo Therapeutics, Fort Lauderdale, FL","institution_ids":[]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5027107930","display_name":"Deborah Barbouth","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I145608581","display_name":"University of Miami","ror":"https://ror.org/02dgjyy92","country_code":"US","type":"education","lineage":["https://openalex.org/I145608581"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"D. Barbouth","raw_affiliation_strings":["University of Miami School of Medicine, Miami, FL"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"University of Miami School of Medicine, Miami, FL","institution_ids":["https://openalex.org/I145608581"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5036810359","display_name":"Louis J. Elsas","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I145608581","display_name":"University of Miami","ror":"https://ror.org/02dgjyy92","country_code":"US","type":"education","lineage":["https://openalex.org/I145608581"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"L. Elsas","raw_affiliation_strings":["University of Miami School of Medicine, Miami, FL"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"University of Miami School of Medicine, Miami, FL","institution_ids":["https://openalex.org/I145608581"]}]},{"author_position":"last","author":{"id":"https://openalex.org/A5057831353","display_name":"Gary Kleiner","orcid":"https://orcid.org/0000-0003-2066-9476","observed_orcids":["https://orcid.org/0000-0003-2066-9476"]},"institutions":[{"id":"https://openalex.org/I145608581","display_name":"University of Miami","ror":"https://ror.org/02dgjyy92","country_code":"US","type":"education","lineage":["https://openalex.org/I145608581"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"G. Kleiner","raw_affiliation_strings":["University of Miami School of Medicine, Miami, FL"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"University of Miami School of Medicine, Miami, FL","institution_ids":["https://openalex.org/I145608581"]}]}],"institutions":[],"countries_distinct_count":1,"institutions_distinct_count":2,"corresponding_author_ids":[],"corresponding_institution_ids":[],"apc_list":{"value":3650,"currency":"USD","value_usd":3650},"apc_paid":{"value":3650,"currency":"USD","value_usd":3650},"fwci":0.0,"has_fulltext":true,"cited_by_count":0,"citation_normalized_percentile":{"value":0.14335333,"is_in_top_1_percent":false,"is_in_top_10_percent":false},"cited_by_percentile_year":null,"biblio":{"volume":"11","issue":"2","first_page":"97","last_page":"97"},"is_retracted":false,"is_paratext":false,"is_xpac":false,"primary_topic":{"id":"https://openalex.org/T10945","display_name":"Lysosomal Storage Disorders Research","score":0.9994999766349792,"subfield":{"id":"https://openalex.org/subfields/2716","display_name":"Genetics (clinical)"},"field":{"id":"https://openalex.org/fields/27","display_name":"Medicine"},"domain":{"id":"https://openalex.org/domains/4","display_name":"Health Sciences"}},"topics":[{"id":"https://openalex.org/T10945","display_name":"Lysosomal Storage Disorders Research","score":0.9994999766349792,"subfield":{"id":"https://openalex.org/subfields/2716","display_name":"Genetics (clinical)"},"field":{"id":"https://openalex.org/fields/27","display_name":"Medicine"},"domain":{"id":"https://openalex.org/domains/4","display_name":"Health Sciences"}},{"id":"https://openalex.org/T12604","display_name":"Glycogen Storage Diseases and Myoclonus","score":9.999999747378752e-05,"subfield":{"id":"https://openalex.org/subfields/1303","display_name":"Biochemistry"},"field":{"id":"https://openalex.org/fields/13","display_name":"Biochemistry, Genetics and Molecular Biology"},"domain":{"id":"https://openalex.org/domains/1","display_name":"Life Sciences"}},{"id":"https://openalex.org/T10345","display_name":"Hematopoietic Stem Cell Transplantation","score":9.999999747378752e-05,"subfield":{"id":"https://openalex.org/subfields/2720","display_name":"Hematology"},"field":{"id":"https://openalex.org/fields/27","display_name":"Medicine"},"domain":{"id":"https://openalex.org/domains/4","display_name":"Health Sciences"}}],"keywords":[{"id":"https://openalex.org/keywords/hurler-syndrome","display_name":"Hurler syndrome","score":0.8830000162124634},{"id":"https://openalex.org/keywords/mucopolysaccharidosis-type-i","display_name":"mucopolysaccharidosis type I","score":0.9290000200271606},{"id":"https://openalex.org/keywords/enzyme-replacement-therapy","display_name":"enzyme replacement therapy","score":0.8339999914169312},{"id":"https://openalex.org/keywords/unrelated-cord-blood-transplantation","display_name":"unrelated cord blood transplantation","score":0.8790000081062317},{"id":"https://openalex.org/keywords/engraftment","display_name":"engraftment","score":0.7110000252723694},{"id":"https://openalex.org/keywords/pediatric-bone-marrow-transplantation","display_name":"pediatric bone marrow transplantation","score":0.75},{"id":"https://openalex.org/keywords/outpatient-infusion","display_name":"outpatient infusion","score":0.4230000078678131},{"id":"https://openalex.org/keywords/neurodegeneration","display_name":"neurodegeneration","score":0.628000020980835},{"id":"https://openalex.org/keywords/lysosomal-storage","display_name":"lysosomal storage","score":0.652999997138977}],"concepts":[{"id":"https://openalex.org/C71924100","wikidata":"https://www.wikidata.org/wiki/Q11190","display_name":"Medicine","level":0,"score":0.9689850211143494},{"id":"https://openalex.org/C2777636572","wikidata":"https://www.wikidata.org/wiki/Q25379699","display_name":"Hurler syndrome","level":3,"score":0.8181700706481934},{"id":"https://openalex.org/C2779969927","wikidata":"https://www.wikidata.org/wiki/Q916766","display_name":"Enzyme replacement therapy","level":3,"score":0.808826744556427},{"id":"https://openalex.org/C2911091166","wikidata":"https://www.wikidata.org/wiki/Q106419912","display_name":"Transplantation","level":2,"score":0.5478647351264954},{"id":"https://openalex.org/C187212893","wikidata":"https://www.wikidata.org/wiki/Q123028","display_name":"Pediatrics","level":1,"score":0.450268030166626},{"id":"https://openalex.org/C2778264360","wikidata":"https://www.wikidata.org/wiki/Q675010","display_name":"Lysosomal storage disease","level":3,"score":0.4457119107246399},{"id":"https://openalex.org/C2780372218","wikidata":"https://www.wikidata.org/wiki/Q417222","display_name":"Methylprednisolone","level":2,"score":0.4401642084121704},{"id":"https://openalex.org/C2910727661","wikidata":"https://www.wikidata.org/wiki/Q25379699","display_name":"Mucopolysaccharidosis I","level":4,"score":0.4327964782714844},{"id":"https://openalex.org/C141071460","wikidata":"https://www.wikidata.org/wiki/Q40821","display_name":"Surgery","level":1,"score":0.41921716928482056},{"id":"https://openalex.org/C2779134260","wikidata":"https://www.wikidata.org/wiki/Q12136","display_name":"Disease","level":2,"score":0.36797112226486206},{"id":"https://openalex.org/C126322002","wikidata":"https://www.wikidata.org/wiki/Q11180","display_name":"Internal medicine","level":1,"score":0.2816982865333557}],"mesh":[],"locations_count":1,"locations":[{"id":"doi:10.1016/j.bbmt.2004.12.286","doi":"https://doi.org/10.1016/j.bbmt.2004.12.286","is_oa":true,"landing_page_url":"https://doi.org/10.1016/j.bbmt.2004.12.286","pdf_url":"http://www.astctjournal.org/article/S1083879104009383/pdf","source":{"id":"https://openalex.org/S59987174","display_name":"Transplantation and Cellular Therapy","issn_l":"1083-8791","issn":["1083-8791","1523-6536","2666-6367"],"is_oa":false,"is_in_doaj":false,"is_core":true,"listed_in":["anvur-scientific","cwts-core","dongbi-b","doyens","jufo-1","ki-jl-1","medline","norway-1","poland-100","poland-20","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310320990","host_organization_name":"Elsevier BV","host_organization_lineage":["https://openalex.org/P4310320990"],"host_organization_lineage_names":["Elsevier BV"],"type":"journal"},"license":"cc-by-nc-nd","license_id":"https://openalex.org/licenses/cc-by-nc-nd","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Biology of Blood and Marrow Transplantation","raw_type":"journal-article"}],"best_oa_location":{"id":"doi:10.1016/j.bbmt.2004.12.286","doi":"https://doi.org/10.1016/j.bbmt.2004.12.286","is_oa":true,"landing_page_url":"https://doi.org/10.1016/j.bbmt.2004.12.286","pdf_url":"http://www.astctjournal.org/article/S1083879104009383/pdf","source":{"id":"https://openalex.org/S59987174","display_name":"Transplantation and Cellular Therapy","issn_l":"1083-8791","issn":["1083-8791","1523-6536","2666-6367"],"is_oa":false,"is_in_doaj":false,"is_core":true,"listed_in":["anvur-scientific","cwts-core","dongbi-b","doyens","jufo-1","ki-jl-1","medline","norway-1","poland-100","poland-20","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310320990","host_organization_name":"Elsevier BV","host_organization_lineage":["https://openalex.org/P4310320990"],"host_organization_lineage_names":["Elsevier BV"],"type":"journal"},"license":"cc-by-nc-nd","license_id":"https://openalex.org/licenses/cc-by-nc-nd","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Biology of Blood and Marrow Transplantation","raw_type":"journal-article"},"sustainable_development_goals":[{"score":1.0,"id":"https://metadata.un.org/sdg/3","display_name":"Good health and well-being"}],"study_designs":[{"id":"https://openalex.org/study-designs/case-report","display_name":"Case Report"}],"awards":[],"funders":[],"has_content":{"grobid_xml":true,"pdf":true},"content_urls":{"pdf":"https://content.openalex.org/works/W2090134286.pdf","grobid_xml":"https://content.openalex.org/works/W2090134286.grobid-xml"},"referenced_works_count":0,"referenced_works":[],"related_works":["https://openalex.org/W2138690042","https://openalex.org/W1990892818","https://openalex.org/W2594027811","https://openalex.org/W2132957067","https://openalex.org/W2165282314","https://openalex.org/W1988569924","https://openalex.org/W2015597043","https://openalex.org/W2974599311","https://openalex.org/W2081198924","https://openalex.org/W2216662624"],"abstract_inverted_index":{"Hurler\u2019s":[0],"syndrome":[1],"is":[2,58,253],"caused":[3],"by":[4,144,200],"mutations":[5],"in":[6,13,44,60,81,99,191,224],"the":[7,17,45,52,82,135,140,178,192,217],"alpha":[8],"iduronidase":[9],"gene":[10],"and":[11,32,47,71,86,97,162,204,236],"results":[12],"progressive":[14,251],"deterioration":[15],"of":[16,42,51,74,111,184,210,228],"central":[18],"nervous":[19],"system.":[20],"Allogeneic":[21],"bone":[22,194],"marrow":[23,195],"transplantation":[24,196],"before":[25,126],"age":[26,114],"2":[27],"years":[28],"halts":[29],"disease":[30],"progression":[31],"prolongs":[33],"life.":[34],"Enzyme":[35],"replacement":[36,66],"therapy":[37,67],"(ERT)":[38],"reduces":[39],"lysosomal":[40],"storage":[41],"mucopolysaccharides":[43],"liver":[46],"ameliorates":[48],"extracranial":[49],"manifestations":[50],"disease.":[53],"In":[54],"most":[55],"instances,":[56],"ERT":[57,80,96,138,231,238],"done":[59],"a":[61,100,145,156],"day-hospital":[62],"setting.":[63],"Because":[64],"enzyme":[65],"can":[68],"mitigate":[69],"airway":[70],"pulmonary":[72],"manifestation":[73],"MPS":[75],"I,":[76],"we":[77],"hypothesized":[78],"that":[79,223],"peritransplantation":[83],"period":[84],"(pre-HSCT":[85],"immediately":[87],"post-HSCT)":[88],"would":[89],"decrease":[90],"transplantation-related":[91],"complications.":[92],"We":[93,221],"performed":[94,190,215],"combined":[95,229],"HSCT":[98],"12-month-old":[101],"boy":[102],"with":[103,172,233,242],"MPSI.":[104],"The":[105,153,208],"patient":[106,136,154],"received":[107,123,137,155,180],"his":[108,132,211],"first":[109,226],"dose":[110],"Aldurazyme":[112],"at":[113,139,216],"5":[115],"months,":[116],"shortly":[117],"after":[118],"MPSI":[119],"diagnosis.":[120],"He":[121],"had":[122],"16":[124],"infusions":[125,188,213],"referral":[127],"to":[128,248],"our":[129],"center.":[130],"Throughout":[131],"pretransplantation":[133,235],"workup,":[134],"local":[141,218],"Transplantation":[142,219],"House":[143],"visiting":[146],"nurse.":[147],"There":[148],"were":[149,189,214],"no":[150],"infusion-related":[151],"toxicities.":[152],"5/6":[157],"unrelated":[158],"cord":[159],"blood":[160],"unit":[161,197],"engrafted":[163],"on":[164],"day":[165],"+12.":[166],"Hyperacute":[167],"GVHD":[168],"was":[169],"treated":[170],"successfully":[171],"high-dose":[173],"methylprednisolone":[174],"pulse.":[175],"After":[176],"engraftment,":[177],"child":[179],"8":[181],"additional":[182],"doses":[183],"Aldurazyme.":[185],"Four":[186],"initial":[187],"pediatric":[193,201,205],"following":[198],"in-service":[199],"BMT":[202,206],"nurse":[203],"pharmacist.":[207],"remainder":[209],"4":[212],"House.":[220],"conclude":[222],"this":[225],"case":[227],"outpatient":[230],"infusion":[232],"HSCT,":[234],"posttransplantation":[237],"does":[239],"not":[240],"interfere":[241],"engraftment.":[243],"Further":[244],"studies":[245],"are":[246],"needed":[247],"determine":[249],"whether":[250],"neurodegeneration":[252],"altered.":[254]},"counts_by_year":[],"updated_date":"2026-11-04T10:54:05.112534","created_date":"2016-06-24T00:00:00"}