{"id":"https://openalex.org/W2993330168","doi":"https://doi.org/10.1182/blood.v104.11.104.104","title":"Hematopoietic Cell Transplantation for Sickle Cell Disease: Updated Results of the Multicenter Trial.","display_name":"Hematopoietic Cell Transplantation for Sickle Cell Disease: Updated Results of the Multicenter Trial.","publication_year":2004,"publication_date":"2004-11-16","ids":{"openalex":"https://openalex.org/W2993330168","doi":"https://doi.org/10.1182/blood.v104.11.104.104","mag":"2993330168"},"language":"en","primary_location":{"id":"doi:10.1182/blood.v104.11.104.104","doi":"https://doi.org/10.1182/blood.v104.11.104.104","is_oa":false,"landing_page_url":"https://doi.org/10.1182/blood.v104.11.104.104","pdf_url":null,"source":{"id":"https://openalex.org/S200071133","display_name":"Blood","issn_l":"0006-4971","issn":["0006-4971","1528-0020"],"is_oa":false,"is_in_doaj":false,"is_core":true,"listed_in":["anvur-scientific","cwts-core","dongbi-a","doyens","jufo-3","ki-jl-3","medline","norway-2","poland-200","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":null,"license_id":null,"version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Blood","raw_type":"journal-article"},"volume":null,"type":"article","indexed_in":["crossref"],"open_access":{"is_oa":false,"oa_status":"closed","oa_url":null,"any_repository_has_fulltext":false},"authorships":[{"author_position":"first","author":{"id":"https://openalex.org/A5040299600","display_name":"Mark C. Walters","orcid":"https://orcid.org/0000-0002-6515-4559","observed_orcids":["https://orcid.org/0000-0002-6515-4559"]},"institutions":[{"id":"https://openalex.org/I4210126298","display_name":"Duke Medical Center","ror":"https://ror.org/03njmea73","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210126298","https://openalex.org/I4210144876"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Mark C. Walters","raw_affiliation_strings":["Duke University Medical Center, Durham, NC, USA ","Hematology/Oncology, Children\u2019s Hospital & Research Center at Oakland, Oakland, CA, USA ;"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Duke University Medical Center, Durham, NC, USA ","institution_ids":["https://openalex.org/I4210126298"]},{"raw_affiliation_string":"Hematology/Oncology, Children\u2019s Hospital & Research Center at Oakland, Oakland, CA, USA ;","institution_ids":[]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5112079948","display_name":"Melinda Patience","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210126298","display_name":"Duke Medical Center","ror":"https://ror.org/03njmea73","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210126298","https://openalex.org/I4210144876"]}],"countries":["US"],"is_corresponding":true,"raw_author_name":"Melinda Patience","raw_affiliation_strings":["Duke University Medical Center, Durham, NC, USA ","Hematology/Oncology, Children\u2019s Hospital & Research Center at Oakland, Oakland, CA, USA ;"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Duke University Medical Center, Durham, NC, USA ","institution_ids":["https://openalex.org/I4210126298"]},{"raw_affiliation_string":"Hematology/Oncology, Children\u2019s Hospital & Research Center at Oakland, Oakland, CA, USA ;","institution_ids":[]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5103880207","display_name":"Sandie Edwards","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210126298","display_name":"Duke Medical Center","ror":"https://ror.org/03njmea73","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210126298","https://openalex.org/I4210144876"]}],"countries":["US"],"is_corresponding":true,"raw_author_name":"Sandie Edwards","raw_affiliation_strings":["Duke University Medical Center, Durham, NC, USA ","Hematology/Oncology, Children\u2019s Hospital & Research Center at Oakland, Oakland, CA, USA ;"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Duke University Medical Center, Durham, NC, USA ","institution_ids":["https://openalex.org/I4210126298"]},{"raw_affiliation_string":"Hematology/Oncology, Children\u2019s Hospital & Research Center at Oakland, Oakland, CA, USA ;","institution_ids":[]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5073862011","display_name":"Shanda Robertson","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210126298","display_name":"Duke Medical Center","ror":"https://ror.org/03njmea73","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210126298","https://openalex.org/I4210144876"]}],"countries":["US"],"is_corresponding":true,"raw_author_name":"Shanda Robertson","raw_affiliation_strings":["Duke University Medical Center, Durham, NC, USA ","Hematology/Oncology, Children\u2019s Hospital & Research Center at Oakland, Oakland, CA, USA ;"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Duke University Medical Center, Durham, NC, USA ","institution_ids":["https://openalex.org/I4210126298"]},{"raw_affiliation_string":"Hematology/Oncology, Children\u2019s Hospital & Research Center at Oakland, Oakland, CA, USA ;","institution_ids":[]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5087657412","display_name":"Marsha A. McMurray","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210126298","display_name":"Duke Medical Center","ror":"https://ror.org/03njmea73","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210126298","https://openalex.org/I4210144876"]},{"id":"https://openalex.org/I4210153694","display_name":"Rho (United States)","ror":"https://ror.org/049ba1e57","country_code":"US","type":"company","lineage":["https://openalex.org/I4210153694"]}],"countries":["US"],"is_corresponding":true,"raw_author_name":"Marsha McMurray","raw_affiliation_strings":["Duke University Medical Center, Durham, NC, USA ","Rho, Inc., Chapel Hill, NC, USA and"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Duke University Medical Center, Durham, NC, USA ","institution_ids":["https://openalex.org/I4210126298"]},{"raw_affiliation_string":"Rho, Inc., Chapel Hill, NC, USA and","institution_ids":["https://openalex.org/I4210153694"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5111664451","display_name":"Sharyne M Donfield","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210126298","display_name":"Duke Medical Center","ror":"https://ror.org/03njmea73","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210126298","https://openalex.org/I4210144876"]},{"id":"https://openalex.org/I4210153694","display_name":"Rho (United States)","ror":"https://ror.org/049ba1e57","country_code":"US","type":"company","lineage":["https://openalex.org/I4210153694"]}],"countries":["US"],"is_corresponding":true,"raw_author_name":"Sharyne Donfield","raw_affiliation_strings":["Duke University Medical Center, Durham, NC, USA ","Rho, Inc., Chapel Hill, NC, USA and"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Duke University Medical Center, Durham, NC, USA ","institution_ids":["https://openalex.org/I4210126298"]},{"raw_affiliation_string":"Rho, Inc., Chapel Hill, NC, USA and","institution_ids":["https://openalex.org/I4210153694"]}]},{"author_position":"last","author":{"id":"https://openalex.org/A5037247107","display_name":"Keith M. Sullivan","orcid":"https://orcid.org/0000-0002-1379-9216","observed_orcids":["https://orcid.org/0000-0002-1379-9216"]},"institutions":[{"id":"https://openalex.org/I4210126298","display_name":"Duke Medical Center","ror":"https://ror.org/03njmea73","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210126298","https://openalex.org/I4210144876"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Keith M. Sullivan","raw_affiliation_strings":["Duke University Medical Center, Durham, NC, USA "],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Duke University Medical Center, Durham, NC, USA ","institution_ids":["https://openalex.org/I4210126298"]}]}],"institutions":[],"countries_distinct_count":1,"institutions_distinct_count":2,"corresponding_author_ids":["https://openalex.org/A5073862011","https://openalex.org/A5087657412","https://openalex.org/A5103880207","https://openalex.org/A5111664451","https://openalex.org/A5112079948"],"corresponding_institution_ids":["https://openalex.org/I4210126298","https://openalex.org/I4210153694"],"apc_list":{"value":5500,"currency":"USD","value_usd":5500},"apc_paid":null,"fwci":0.5213,"has_fulltext":false,"cited_by_count":6,"citation_normalized_percentile":{"value":0.72601447,"is_in_top_1_percent":false,"is_in_top_10_percent":false},"cited_by_percentile_year":{"min":93,"max":96},"biblio":{"volume":"104","issue":"11","first_page":"104","last_page":"104"},"is_retracted":false,"is_paratext":false,"is_xpac":false,"primary_topic":{"id":"https://openalex.org/T10554","display_name":"Hemoglobinopathies and Related Disorders","score":0.8804000020027161,"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/T10554","display_name":"Hemoglobinopathies and Related Disorders","score":0.8804000020027161,"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/T10345","display_name":"Hematopoietic Stem Cell Transplantation","score":0.1143999993801117,"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"}},{"id":"https://openalex.org/T10693","display_name":"Blood transfusion and management","score":0.0003000000142492354,"subfield":{"id":"https://openalex.org/subfields/2704","display_name":"Biochemistry (medical)"},"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/sickle-cell-disease","display_name":"sickle cell disease","score":1.0},{"id":"https://openalex.org/keywords/bone-marrow-transplantation","display_name":"bone marrow transplantation","score":0.8600000143051147},{"id":"https://openalex.org/keywords/myeloablative-conditioning","display_name":"myeloablative conditioning","score":0.8650000095367432},{"id":"https://openalex.org/keywords/hla-matched-sibling-donor","display_name":"matched sibling donor","score":0.9269999861717224},{"id":"https://openalex.org/keywords/pediatric-patients","display_name":"pediatric patients","score":0.6299999952316284},{"id":"https://openalex.org/keywords/stroke","display_name":"stroke","score":0.6729999780654907},{"id":"https://openalex.org/keywords/acute-chest-syndrome","display_name":"acute chest syndrome","score":0.9179999828338623},{"id":"https://openalex.org/keywords/graft-versus-host-disease","display_name":"graft-versus-host disease","score":0.8489999771118164},{"id":"https://openalex.org/keywords/graft-rejection","display_name":"graft rejection","score":0.8429999947547913},{"id":"https://openalex.org/keywords/lung-function","display_name":"lung function","score":0.722000002861023},{"id":"https://openalex.org/keywords/gonadal-function","display_name":"gonadal function","score":0.843999981880188},{"id":"https://openalex.org/keywords/long-term-follow-up","display_name":"long-term follow-up","score":0.6859999895095825},{"id":"https://openalex.org/keywords/event-free-survival","display_name":"event-free survival","score":0.8320000171661377},{"id":"https://openalex.org/keywords/transplant-related-toxicity","display_name":"transplant toxicity","score":0.45399999618530273},{"id":"https://openalex.org/keywords/central-nervous-system-diseases","display_name":"central nervous system disorders","score":0.5709999799728394},{"id":"https://openalex.org/keywords/testosterone-deficiency","display_name":"androgen deficiency","score":0.41999998688697815}],"concepts":[{"id":"https://openalex.org/C71924100","wikidata":"https://www.wikidata.org/wiki/Q11190","display_name":"Medicine","level":0,"score":0.8617371320724487},{"id":"https://openalex.org/C2780976302","wikidata":"https://www.wikidata.org/wiki/Q4677915","display_name":"Acute chest syndrome","level":4,"score":0.6889394521713257},{"id":"https://openalex.org/C2911091166","wikidata":"https://www.wikidata.org/wiki/Q106419912","display_name":"Transplantation","level":2,"score":0.6029066443443298},{"id":"https://openalex.org/C2780645631","wikidata":"https://www.wikidata.org/wiki/Q671554","display_name":"Stroke (engine)","level":2,"score":0.5608653426170349},{"id":"https://openalex.org/C126322002","wikidata":"https://www.wikidata.org/wiki/Q11180","display_name":"Internal medicine","level":1,"score":0.5043329000473022},{"id":"https://openalex.org/C2779134260","wikidata":"https://www.wikidata.org/wiki/Q12136","display_name":"Disease","level":2,"score":0.49315208196640015},{"id":"https://openalex.org/C2777408962","wikidata":"https://www.wikidata.org/wiki/Q1128050","display_name":"Hematopoietic stem cell transplantation","level":3,"score":0.4769262671470642},{"id":"https://openalex.org/C2775965419","wikidata":"https://www.wikidata.org/wiki/Q6934595","display_name":"Multicenter trial","level":4,"score":0.44085994362831116},{"id":"https://openalex.org/C141071460","wikidata":"https://www.wikidata.org/wiki/Q40821","display_name":"Surgery","level":1,"score":0.41463911533355713},{"id":"https://openalex.org/C2778620579","wikidata":"https://www.wikidata.org/wiki/Q185034","display_name":"Sickle cell anemia","level":3,"score":0.41041308641433716},{"id":"https://openalex.org/C187212893","wikidata":"https://www.wikidata.org/wiki/Q123028","display_name":"Pediatrics","level":1,"score":0.3377259373664856},{"id":"https://openalex.org/C168563851","wikidata":"https://www.wikidata.org/wiki/Q1436668","display_name":"Randomized controlled trial","level":2,"score":0.19569027423858643},{"id":"https://openalex.org/C2992435398","wikidata":"https://www.wikidata.org/wiki/Q6934595","display_name":"Multicenter study","level":3,"score":0.1335013210773468},{"id":"https://openalex.org/C127413603","wikidata":"https://www.wikidata.org/wiki/Q11023","display_name":"Engineering","level":0,"score":0.0},{"id":"https://openalex.org/C78519656","wikidata":"https://www.wikidata.org/wiki/Q101333","display_name":"Mechanical engineering","level":1,"score":0.0}],"mesh":[],"locations_count":1,"locations":[{"id":"doi:10.1182/blood.v104.11.104.104","doi":"https://doi.org/10.1182/blood.v104.11.104.104","is_oa":false,"landing_page_url":"https://doi.org/10.1182/blood.v104.11.104.104","pdf_url":null,"source":{"id":"https://openalex.org/S200071133","display_name":"Blood","issn_l":"0006-4971","issn":["0006-4971","1528-0020"],"is_oa":false,"is_in_doaj":false,"is_core":true,"listed_in":["anvur-scientific","cwts-core","dongbi-a","doyens","jufo-3","ki-jl-3","medline","norway-2","poland-200","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":null,"license_id":null,"version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Blood","raw_type":"journal-article"}],"best_oa_location":null,"sustainable_development_goals":[{"display_name":"Good health and well-being","id":"https://metadata.un.org/sdg/3","score":1.0}],"study_designs":[],"awards":[],"funders":[],"has_content":{"grobid_xml":false,"pdf":false},"content_urls":null,"referenced_works_count":0,"referenced_works":[],"related_works":["https://openalex.org/W1987823938","https://openalex.org/W1508585281","https://openalex.org/W1999484550","https://openalex.org/W2324143840","https://openalex.org/W327766200","https://openalex.org/W7095096009","https://openalex.org/W2909482306","https://openalex.org/W2060745772","https://openalex.org/W2607115608","https://openalex.org/W2552002719"],"abstract_inverted_index":{"Abstract":[0],"Between":[1],"1991":[2],"and":[3,60,103,108,117,136,155,158,202,233,247,286,302,319,341],"2000,":[4],"59":[5],"children":[6],"with":[7,146,168,227,244,249,256,270,360,430],"symptomatic":[8],"sickle":[9,65,432],"cell":[10,66,433],"disease":[11,37,75],"(SCD)":[12],"were":[13,139,204],"enrolled":[14],"in":[15,330,382,414],"a":[16,92,172,262,271,323,347,388,401,425],"multicenter":[17],"investigation":[18],"of":[19,42,64,71,73,89,95,101,115,126,131,185,213,253,258,264,273,276,295,313,335,363,368,371,385,391,396,403,441],"myeloablative":[20],"HLA-identical":[21],"sibling":[22],"bone":[23],"marrow":[24],"transplantation":[25,116],"(BMT).":[26],"Patients":[27],"had":[28,83,148,190,220,231,235,239,261,306,316,338,352,366,378,394,400],"stroke":[29,147,151,174],"(N=30)":[30],"or":[31,48,77,163,207,354,380],"other":[32],"significant":[33],"central":[34],"nervous":[35],"system":[36],"(CNS)":[38],"(N=1),":[39],"recurrent":[40,49],"episodes":[41,51],"acute":[43],"chest":[44],"syndrome":[45],"(ACS)":[46],"(N=20),":[47],"painful":[50],"(N=8)":[52],"before":[53,266,405],"study":[54],"entry.":[55],"Currently,":[56,283],"55":[57,186],"patients":[58,69,82,145,189,359,392,429],"survive,":[59],"50":[61],"survive":[62],"free":[63],"disease.":[67,282,386],"Four":[68],"died":[70,275],"complications":[72,278,370],"graft-versus-host":[74,281],"(GVHD)(N=3)":[76],"intracranial":[78],"hemorrhage":[79],"(N=1).":[80],"Five":[81,238],"graft":[84,169],"rejection":[85,170],"accompanied":[86],"by":[87,280,290],"return":[88],"SCD.":[90],"With":[91],"median":[93],"follow-up":[94,129,218,228],"5.2":[96],"years,":[97],"the":[98,113,123,132,176,214,225,254,439],"Kaplan-Meier":[99],"probabilities":[100],"survival":[102,105],"event-free":[104],"are":[106,292],"93%":[107],"85%,":[109],"respectively.":[110],"To":[111],"investigate":[112],"toxicity":[114,410,440],"to":[118,437],"assess":[119],"its":[120],"impact":[121],"on":[122],"natural":[124],"history":[125,263,272,402],"SCD,":[127],"long-term":[128],"evaluations":[130],"CNS,":[133],"pulmonary":[134,191,241,277],"function,":[135,242],"gonadal":[137,308,409],"function":[138,192,309],"performed.":[140,311],"As":[141],"reported":[142],"previously,":[143],"engrafted":[144],"no":[149],"subsequent":[150],"events":[152],"after":[153,182,200,210,373,416],"BMT,":[154,201],"cerebral":[156],"MRI":[157],"MRA":[159],"exams":[160],"demonstrated":[161],"stable":[162,361],"improved":[164,236],"appearance.":[165],"One":[166,268],"patient":[167,269],"experienced":[171],"second":[173],"when":[175],"Hb":[177],"S":[178],"fraction":[179],"reached":[180],"60%":[181],"BMT.":[183,211,267,374,406,417],"Forty-three":[184],"surviving":[187],"(78%)":[188],"testing":[193,219],"(PFT)":[194],"performed":[195],"at":[196],"least":[197],"1":[198,248],"year":[199],"24":[203,284],"tested":[205],"3":[206],"more":[208],"years":[209,294],"Seven":[212],"12":[215],"who":[216,393],"lacked":[217],"normal":[221,317,324],"baseline":[222],"testing.":[223],"Of":[224,297],"43":[226],"exams,":[229],"30":[230],"stable,":[232],"8":[234],"PFTs.":[237],"worsened":[240],"4":[243,255],"restrictive":[245,259],"changes,":[246],"obstructive":[250],"changes.":[251],"Three":[252],"progression":[257,395],"changes":[260],"ACS":[265,274,404],"caused":[279],"males":[285,300,315],"19":[287],"females":[288,305,337,351,415],"treated":[289],"BMT":[291,423,442],">14":[293],"age.":[296],"these,":[298],"15":[299,314],"(63%)":[301],"14":[303,336],"(74%)":[304],"post-BMT":[307],"studies":[310],"Eleven":[312],"LH":[318],"FSH":[320],"levels,":[321],"however,":[322],"testosterone":[325],"level":[326],"was":[327,411],"observed":[328,412],"only":[329],"4.":[331],"In":[332,357,375,407],"contrast,":[333],"9":[334],"elevated":[339],"gonadotropin":[340],"decreased":[342],"estradiol":[343],"levels":[344],"that":[345,422],"mimicked":[346],"post-menopausal":[348],"state.":[349],"Six":[350],"primary":[353],"secondary":[355],"amenorrhea.":[356],"summary,":[358],"engraftment":[362],"donor":[364],"cells":[365],"cessation":[367],"clinical":[369],"SCD":[372],"addition,":[376,408],"most":[377],"stabilization":[379],"improvement":[381],"sub-clinical":[383],"markers":[384],"However,":[387],"limited":[389],"number":[390],"PFT":[397],"abnormalities":[398],"also":[399],"commonly":[413],"Together,":[418],"these":[419],"data":[420],"confirm":[421],"is":[424],"suitable":[426],"intervention":[427],"for":[428],"severe":[431],"disease,":[434],"however":[435],"efforts":[436],"reduce":[438],"should":[443],"be":[444],"pursued.":[445]},"counts_by_year":[{"year":2013,"cited_by_count":2}],"updated_date":"2026-11-04T10:54:05.112534","created_date":"2019-12-13T00:00:00"}