{"id":"https://openalex.org/W2902495987","doi":"https://doi.org/10.1182/asheducation-2018.1.361","title":"Beyond transfusion therapy: new therapies in thalassemia including drugs, alternate donor transplant, and gene therapy","display_name":"Beyond transfusion therapy: new therapies in thalassemia including drugs, alternate donor transplant, and gene therapy","publication_year":2018,"publication_date":"2018-11-30","ids":{"openalex":"https://openalex.org/W2902495987","doi":"https://doi.org/10.1182/asheducation-2018.1.361","mag":"2902495987","pmid":"https://pubmed.ncbi.nlm.nih.gov/30504333","pmcid":"https://www.ncbi.nlm.nih.gov/pmc/articles/6245990"},"language":"en","primary_location":{"id":"doi:10.1182/asheducation-2018.1.361","doi":"https://doi.org/10.1182/asheducation-2018.1.361","is_oa":true,"landing_page_url":"https://doi.org/10.1182/asheducation-2018.1.361","pdf_url":"https://ashpublications.org/hematology/article-pdf/2018/1/361/1254816/hem01850.pdf","source":{"id":"https://openalex.org/S4210206817","display_name":"Hematology","issn_l":"1520-4383","issn":["1520-4383","1520-4391"],"is_oa":false,"is_in_doaj":false,"is_core":true,"listed_in":["cwts-core","dongbi-d","doyens","ki-jl-1","medline","norway-1","poland-100","russia-white-list-2"],"host_organization":"https://openalex.org/P4310320264","host_organization_name":"American Society of Hematology","host_organization_lineage":["https://openalex.org/P4310320264"],"host_organization_lineage_names":["American Society of Hematology"],"type":"journal"},"license":null,"license_id":null,"version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Hematology","raw_type":"journal-article"},"volume":null,"type":"review","indexed_in":["crossref","pubmed"],"open_access":{"is_oa":true,"oa_status":"bronze","oa_url":"https://ashpublications.org/hematology/article-pdf/2018/1/361/1254816/hem01850.pdf","any_repository_has_fulltext":true},"authorships":[{"author_position":"first","author":{"id":"https://openalex.org/A5049244658","display_name":"John B. Porter","orcid":"https://orcid.org/0000-0003-3000-9359","observed_orcids":["https://orcid.org/0000-0003-3000-9359"]},"institutions":[{"id":"https://openalex.org/I45129253","display_name":"University College London","ror":"https://ror.org/02jx3x895","country_code":"GB","type":"education","lineage":["https://openalex.org/I124357947","https://openalex.org/I45129253"]}],"countries":["GB"],"is_corresponding":true,"raw_author_name":"John Porter","raw_affiliation_strings":["University College London, London, United Kingdom"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"University College London, London, United Kingdom","institution_ids":["https://openalex.org/I45129253"]}]}],"institutions":[],"countries_distinct_count":1,"institutions_distinct_count":1,"corresponding_author_ids":["https://openalex.org/A5049244658"],"corresponding_institution_ids":["https://openalex.org/I45129253"],"apc_list":null,"apc_paid":null,"fwci":0.7249,"has_fulltext":true,"cited_by_count":40,"citation_normalized_percentile":{"value":0.60900901,"is_in_top_1_percent":false,"is_in_top_10_percent":false},"cited_by_percentile_year":{"min":88,"max":99},"biblio":{"volume":"2018","issue":"1","first_page":"361","last_page":"370"},"is_retracted":false,"is_paratext":false,"is_xpac":false,"primary_topic":{"id":"https://openalex.org/T10554","display_name":"Hemoglobinopathies and Related Disorders","score":0.9955999851226807,"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":0.9955999851226807,"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":0.0017999999690800905,"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/T11280","display_name":"Hemophilia Treatment and Research","score":0.0003000000142492354,"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/beta-thalassemia","display_name":"beta-thalassemia","score":0.878000020980835},{"id":"https://openalex.org/keywords/transfusion-dependent-thalassemia","display_name":"transfusion-dependent thalassemia","score":0.9449999928474426},{"id":"https://openalex.org/keywords/curative-treatment","display_name":"curative treatment","score":0.597000002861023},{"id":"https://openalex.org/keywords/gene-therapy","display_name":"gene therapy","score":0.7319999933242798},{"id":"https://openalex.org/keywords/haploidentical-hematopoietic-stem-cell-transplantation","display_name":"haploidentical hematopoietic stem cell transplantation","score":0.921999990940094},{"id":"https://openalex.org/keywords/matched-related-donor","display_name":"matched related donor","score":0.5049999952316284},{"id":"https://openalex.org/keywords/luspatercept","display_name":"luspatercept","score":0.9359999895095825},{"id":"https://openalex.org/keywords/transfusion-independence","display_name":"transfusion independence","score":0.6639999747276306},{"id":"https://openalex.org/keywords/chelation-therapy","display_name":"chelation therapy","score":0.7950000166893005},{"id":"https://openalex.org/keywords/hepcidin","display_name":"hepcidin","score":0.8880000114440918},{"id":"https://openalex.org/keywords/iron-metabolism","display_name":"iron metabolism","score":0.9750000238418579},{"id":"https://openalex.org/keywords/erythropoiesis","display_name":"erythropoiesis","score":0.9039999842643738},{"id":"https://openalex.org/keywords/health-care-costs","display_name":"health care costs","score":0.6710000038146973}],"concepts":[{"id":"https://openalex.org/C71924100","wikidata":"https://www.wikidata.org/wiki/Q11190","display_name":"Medicine","level":0,"score":0.7898802161216736},{"id":"https://openalex.org/C2777799968","wikidata":"https://www.wikidata.org/wiki/Q185137","display_name":"Thalassemia","level":2,"score":0.68862384557724},{"id":"https://openalex.org/C133925201","wikidata":"https://www.wikidata.org/wiki/Q188419","display_name":"Life expectancy","level":3,"score":0.6312986612319946},{"id":"https://openalex.org/C2779951463","wikidata":"https://www.wikidata.org/wiki/Q7268788","display_name":"Quality of life (healthcare)","level":2,"score":0.5537756085395813},{"id":"https://openalex.org/C177713679","wikidata":"https://www.wikidata.org/wiki/Q679690","display_name":"Intensive care medicine","level":1,"score":0.5106909871101379},{"id":"https://openalex.org/C111599444","wikidata":"https://www.wikidata.org/wiki/Q213901","display_name":"Genetic enhancement","level":3,"score":0.49880552291870117},{"id":"https://openalex.org/C2777218474","wikidata":"https://www.wikidata.org/wiki/Q1069061","display_name":"Chelation therapy","level":3,"score":0.4512443244457245},{"id":"https://openalex.org/C2780844101","wikidata":"https://www.wikidata.org/wiki/Q2380264","display_name":"Ineffective erythropoiesis","level":4,"score":0.4184425473213196},{"id":"https://openalex.org/C2779703530","wikidata":"https://www.wikidata.org/wiki/Q837174","display_name":"Erythropoiesis","level":3,"score":0.3958505690097809},{"id":"https://openalex.org/C143998085","wikidata":"https://www.wikidata.org/wiki/Q162555","display_name":"Oncology","level":1,"score":0.32195448875427246},{"id":"https://openalex.org/C2778248108","wikidata":"https://www.wikidata.org/wiki/Q5445","display_name":"Anemia","level":2,"score":0.31964191794395447},{"id":"https://openalex.org/C126322002","wikidata":"https://www.wikidata.org/wiki/Q11180","display_name":"Internal medicine","level":1,"score":0.3125275671482086},{"id":"https://openalex.org/C104317684","wikidata":"https://www.wikidata.org/wiki/Q7187","display_name":"Gene","level":2,"score":0.0897853672504425},{"id":"https://openalex.org/C159110408","wikidata":"https://www.wikidata.org/wiki/Q121176","display_name":"Nursing","level":1,"score":0.0},{"id":"https://openalex.org/C2908647359","wikidata":"https://www.wikidata.org/wiki/Q2625603","display_name":"Population","level":2,"score":0.0},{"id":"https://openalex.org/C99454951","wikidata":"https://www.wikidata.org/wiki/Q932068","display_name":"Environmental health","level":1,"score":0.0},{"id":"https://openalex.org/C185592680","wikidata":"https://www.wikidata.org/wiki/Q2329","display_name":"Chemistry","level":0,"score":0.0},{"id":"https://openalex.org/C55493867","wikidata":"https://www.wikidata.org/wiki/Q7094","display_name":"Biochemistry","level":1,"score":0.0}],"mesh":[{"descriptor_ui":"D006801","descriptor_name":"Humans","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D013789","descriptor_name":"Thalassemia","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":false},{"descriptor_ui":"D013789","descriptor_name":"Thalassemia","qualifier_ui":"Q000378","qualifier_name":"metabolism","is_major_topic":false},{"descriptor_ui":"D013789","descriptor_name":"Thalassemia","qualifier_ui":"Q000628","qualifier_name":"therapy","is_major_topic":true},{"descriptor_ui":"D015316","descriptor_name":"Genetic Therapy","qualifier_ui":"Q000379","qualifier_name":"methods","is_major_topic":true},{"descriptor_ui":"D033581","descriptor_name":"Stem Cell Transplantation","qualifier_ui":"Q000379","qualifier_name":"methods","is_major_topic":true},{"descriptor_ui":"D061349","descriptor_name":"Unrelated Donors","qualifier_ui":null,"qualifier_name":null,"is_major_topic":true},{"descriptor_ui":"D064591","descriptor_name":"Allografts","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D064592","descriptor_name":"Autografts","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false}],"locations_count":3,"locations":[{"id":"doi:10.1182/asheducation-2018.1.361","doi":"https://doi.org/10.1182/asheducation-2018.1.361","is_oa":true,"landing_page_url":"https://doi.org/10.1182/asheducation-2018.1.361","pdf_url":"https://ashpublications.org/hematology/article-pdf/2018/1/361/1254816/hem01850.pdf","source":{"id":"https://openalex.org/S4210206817","display_name":"Hematology","issn_l":"1520-4383","issn":["1520-4383","1520-4391"],"is_oa":false,"is_in_doaj":false,"is_core":true,"listed_in":["cwts-core","dongbi-d","doyens","ki-jl-1","medline","norway-1","poland-100","russia-white-list-2"],"host_organization":"https://openalex.org/P4310320264","host_organization_name":"American Society of Hematology","host_organization_lineage":["https://openalex.org/P4310320264"],"host_organization_lineage_names":["American Society of Hematology"],"type":"journal"},"license":null,"license_id":null,"version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Hematology","raw_type":"journal-article"},{"id":"pmh:oai:pubmedcentral.nih.gov:6245990","doi":null,"is_oa":true,"landing_page_url":"https://www.ncbi.nlm.nih.gov/pmc/articles/6245990","pdf_url":null,"source":{"id":"https://openalex.org/S2764455111","display_name":"PubMed Central","issn_l":null,"issn":null,"is_oa":true,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I1299303238","host_organization_name":"National Institutes of Health","host_organization_lineage":["https://openalex.org/I1299303238"],"host_organization_lineage_names":[],"type":"repository"},"license":null,"license_id":null,"version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Hematology Am Soc Hematol Educ Program","raw_type":"Text"},{"id":"pmid:30504333","doi":null,"is_oa":false,"landing_page_url":"https://pubmed.ncbi.nlm.nih.gov/30504333","pdf_url":null,"source":{"id":"https://openalex.org/S4306525036","display_name":"PubMed","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I1299303238","host_organization_name":"National Institutes of Health","host_organization_lineage":["https://openalex.org/I1299303238"],"host_organization_lineage_names":[],"type":"repository"},"license":null,"license_id":null,"version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Hematology. American Society of Hematology. Education Program","raw_type":"Journal Article"}],"best_oa_location":{"id":"doi:10.1182/asheducation-2018.1.361","doi":"https://doi.org/10.1182/asheducation-2018.1.361","is_oa":true,"landing_page_url":"https://doi.org/10.1182/asheducation-2018.1.361","pdf_url":"https://ashpublications.org/hematology/article-pdf/2018/1/361/1254816/hem01850.pdf","source":{"id":"https://openalex.org/S4210206817","display_name":"Hematology","issn_l":"1520-4383","issn":["1520-4383","1520-4391"],"is_oa":false,"is_in_doaj":false,"is_core":true,"listed_in":["cwts-core","dongbi-d","doyens","ki-jl-1","medline","norway-1","poland-100","russia-white-list-2"],"host_organization":"https://openalex.org/P4310320264","host_organization_name":"American Society of Hematology","host_organization_lineage":["https://openalex.org/P4310320264"],"host_organization_lineage_names":["American Society of Hematology"],"type":"journal"},"license":null,"license_id":null,"version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Hematology","raw_type":"journal-article"},"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":{"pdf":true,"grobid_xml":true},"content_urls":{"pdf":"https://content.openalex.org/works/W2902495987.pdf","grobid_xml":"https://content.openalex.org/works/W2902495987.grobid-xml"},"referenced_works_count":35,"referenced_works":["https://openalex.org/W1974266372","https://openalex.org/W1986677646","https://openalex.org/W1997170085","https://openalex.org/W2002791371","https://openalex.org/W2008973770","https://openalex.org/W2008989169","https://openalex.org/W2011733246","https://openalex.org/W2017468796","https://openalex.org/W2022281178","https://openalex.org/W2028135932","https://openalex.org/W2031292381","https://openalex.org/W2032313248","https://openalex.org/W2045631809","https://openalex.org/W2078107954","https://openalex.org/W2085914612","https://openalex.org/W2136274459","https://openalex.org/W2141819869","https://openalex.org/W2148659749","https://openalex.org/W2149986474","https://openalex.org/W2151573971","https://openalex.org/W2161085175","https://openalex.org/W2238351347","https://openalex.org/W2315168906","https://openalex.org/W2339807029","https://openalex.org/W2343013666","https://openalex.org/W2401643476","https://openalex.org/W2753758710","https://openalex.org/W2767181888","https://openalex.org/W2768528990","https://openalex.org/W2769949652","https://openalex.org/W2782398165","https://openalex.org/W2793205487","https://openalex.org/W2802087233","https://openalex.org/W2807198924","https://openalex.org/W4285719527"],"related_works":["https://openalex.org/W4417521088","https://openalex.org/W3197016000","https://openalex.org/W2900281431","https://openalex.org/W4200210548","https://openalex.org/W1966661281","https://openalex.org/W7142165594","https://openalex.org/W7155410212","https://openalex.org/W3201030272","https://openalex.org/W4213108639","https://openalex.org/W2010252260"],"abstract_inverted_index":{"Transfusion":[0],"combined":[1],"with":[2,47,106,155,164],"chelation":[3],"therapy":[4,141],"for":[5,131,213],"severe":[6],"\u03b2":[7],"thalassemia":[8,11],"syndromes":[9],"(transfusion-dependent":[10],"[TDT])":[12],"has":[13,93,207],"been":[14,95],"successful":[15],"in":[16,139,220,227,235],"extending":[17],"life":[18,226],"expectancy,":[19],"decreasing":[20,177,184,232],"comorbidities":[21],"and":[22,51,56,118,181,183,215,223,254],"improving":[23,111],"quality":[24,224],"of":[25,64,72,103,114,127,158,173,179,204,225,247,252],"life.":[26],"However,":[27],"this":[28,92],"puts":[29],"lifelong":[30],"demands":[31],"not":[32],"only":[33,97],"on":[34,39],"the":[35,40,70,90,99,112,124,128,202],"patients":[36,145],"but":[37],"also":[38,172],"health":[41,75],"care":[42,76],"systems":[43],"that":[44,143,194],"are":[45,59,171,241],"tasked":[46],"delivering":[48],"long-term":[49],"treatment":[50],"comprehensive":[52],"support.":[53],"Prevention":[54],"programs":[55],"curative":[57,132],"approaches":[58],"therefore":[60],"an":[61,156,191],"important":[62],"part":[63],"overall":[65],"strategy.":[66],"Curative":[67],"treatments":[68,170,240],"alter":[69],"dynamic":[71],"a":[73,85,147,165],"patient's":[74],"costs,":[77],"from":[78],"financial":[79],"commitment":[80],"over":[81],"50":[82],"years,":[83],"into":[84],"potential":[86,125,174],"\"one-off\"":[87],"investment.":[88],"Since":[89],"1980s,":[91],"usually":[94],"available":[96],"to":[98],"30%":[100],"or":[101],"so":[102],"young":[104],"children":[105],"matched":[107,115,148],"sibling":[108],"donors.":[109],"By":[110],"safety":[113],"related":[116],"donors":[117],"haploidentical":[119],"hematopoietic":[120],"stem":[121,162],"cell":[122],"transplants,":[123],"size":[126],"donor":[129,149],"pool":[130],"therapies":[133],"may":[134],"be":[135,151],"increased.":[136],"Recent":[137],"advances":[138],"gene":[140],"demonstrate":[142],"even":[144],"lacking":[146],"can":[150],"rendered":[152],"transfusion":[153,233],"independent":[154],"autograft":[157],"genetically":[159],"modified":[160],"autologous":[161],"cells,":[163],"low":[166],"short-term":[167],"risk.":[168],"Noncurative":[169],"value":[175],"by":[176],"use":[178],"blood":[180],"chelators":[182],"hospital":[185],"visits.":[186],"An":[187],"example":[188],"is":[189],"luspatercept,":[190],"activin-receptor":[192],"trap":[193],"modifies":[195],"transforming":[196],"growth":[197],"factor-\u03b2":[198],"signaling,":[199],"thereby":[200],"increasing":[201],"efficiency":[203],"erythropoiesis.":[205],"This":[206],"entered":[208],"phase":[209],"3":[210],"clinical":[211,243],"trials":[212,244],"TDT":[214],"non-TDT":[216,228],"and,":[217],"usefully":[218],"increases":[219],"both":[221],"Hb":[222],"as":[229,231],"well":[230],"requirements":[234],"TDT.":[236],"Other":[237],"novel":[238],"noncurative":[239],"entering":[242],"such":[245],"improvement":[246],"erythropoiesis":[248],"through":[249],"pharmacological":[250],"manipulation":[251],"hepcidin":[253],"iron":[255],"metabolism.":[256]},"counts_by_year":[{"year":2026,"cited_by_count":4},{"year":2025,"cited_by_count":8},{"year":2024,"cited_by_count":2},{"year":2023,"cited_by_count":5},{"year":2022,"cited_by_count":1},{"year":2021,"cited_by_count":12},{"year":2020,"cited_by_count":3},{"year":2019,"cited_by_count":5}],"updated_date":"2026-11-04T10:54:05.112534","created_date":"2018-12-11T00:00:00"}
