{"id":"https://openalex.org/W4403158181","doi":"https://doi.org/10.1002/pbc.31351","title":"CHIEF: A retrospective self\u2010control study of children with severe hemophilia A without inhibitors comparing emicizumab to FVIII prophylaxis","display_name":"CHIEF: A retrospective self\u2010control study of children with severe hemophilia A without inhibitors comparing emicizumab to FVIII prophylaxis","publication_year":2024,"publication_date":"2024-10-04","ids":{"openalex":"https://openalex.org/W4403158181","doi":"https://doi.org/10.1002/pbc.31351","pmid":"https://pubmed.ncbi.nlm.nih.gov/39367598"},"language":"en","primary_location":{"id":"doi:10.1002/pbc.31351","doi":"https://doi.org/10.1002/pbc.31351","is_oa":true,"landing_page_url":"https://doi.org/10.1002/pbc.31351","pdf_url":"https://onlinelibrary.wiley.com/doi/pdfdirect/10.1002/pbc.31351","source":{"id":"https://openalex.org/S119714264","display_name":"Pediatric Blood & Cancer","issn_l":"1545-5009","issn":["1545-5009","1545-5017"],"is_oa":false,"is_in_doaj":false,"is_core":true,"listed_in":["anvur-scientific","cwts-core","dongbi-c","doyens","jufo-1","ki-jl-1","medline","norway-1","poland-100","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310320595","host_organization_name":"Wiley","host_organization_lineage":["https://openalex.org/P4310320595"],"host_organization_lineage_names":["Wiley"],"type":"journal"},"license":"cc-by-nc","license_id":"https://openalex.org/licenses/cc-by-nc","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Pediatric Blood &amp; Cancer","raw_type":"journal-article"},"volume":null,"type":"article","indexed_in":["crossref","pubmed"],"open_access":{"is_oa":true,"oa_status":"hybrid","oa_url":"https://onlinelibrary.wiley.com/doi/pdfdirect/10.1002/pbc.31351","any_repository_has_fulltext":false},"authorships":[{"author_position":"first","author":{"id":"https://openalex.org/A5033588580","display_name":"Daniel Mashiach","orcid":"https://orcid.org/0000-0002-4025-7412","observed_orcids":["https://orcid.org/0000-0002-4025-7412"]},"institutions":[{"id":"https://openalex.org/I1336910626","display_name":"Children's Hospital of Los Angeles","ror":"https://ror.org/00412ts95","country_code":"US","type":"funder","lineage":["https://openalex.org/I1336910626"]},{"id":"https://openalex.org/I93393672","display_name":"Western University of Health Sciences","ror":"https://ror.org/05167c961","country_code":"US","type":"education","lineage":["https://openalex.org/I93393672"]}],"countries":["US"],"is_corresponding":true,"raw_author_name":"Daniel Mashiach","raw_affiliation_strings":["Hemostasis and Thrombosis Center Children's Hospital Los Angeles Los Angeles California USA","Western University of Health Sciences, College of Osteopathic Medicine of the Pacific Pomona California USA","Hemostasis and Thrombosis Center, Children's Hospital Los Angeles, Los Angeles, California, USA","Western University of Health Sciences, College of Osteopathic Medicine of the Pacific, Pomona, California, USA"],"raw_orcid":"https://orcid.org/0000-0002-4025-7412","affiliations":[{"raw_affiliation_string":"Hemostasis and Thrombosis Center Children's Hospital Los Angeles Los Angeles California USA","institution_ids":["https://openalex.org/I1336910626"]},{"raw_affiliation_string":"Western University of Health Sciences, College of Osteopathic Medicine of the Pacific Pomona California USA","institution_ids":["https://openalex.org/I93393672"]},{"raw_affiliation_string":"Hemostasis and Thrombosis Center, Children's Hospital Los Angeles, Los Angeles, California, USA","institution_ids":["https://openalex.org/I1336910626"]},{"raw_affiliation_string":"Western University of Health Sciences, College of Osteopathic Medicine of the Pacific, Pomona, California, USA","institution_ids":["https://openalex.org/I93393672"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5113419785","display_name":"Patrice Mead","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I1336910626","display_name":"Children's Hospital of Los Angeles","ror":"https://ror.org/00412ts95","country_code":"US","type":"funder","lineage":["https://openalex.org/I1336910626"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Patrice Mead","raw_affiliation_strings":["Hemostasis and Thrombosis Center Children's Hospital Los Angeles Los Angeles California USA","Hemostasis and Thrombosis Center, Children's Hospital Los Angeles, Los Angeles, California, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Hemostasis and Thrombosis Center Children's Hospital Los Angeles Los Angeles California USA","institution_ids":["https://openalex.org/I1336910626"]},{"raw_affiliation_string":"Hemostasis and Thrombosis Center, Children's Hospital Los Angeles, Los Angeles, California, USA","institution_ids":["https://openalex.org/I1336910626"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5107769648","display_name":"Kendall Carneiro","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I1336910626","display_name":"Children's Hospital of Los Angeles","ror":"https://ror.org/00412ts95","country_code":"US","type":"funder","lineage":["https://openalex.org/I1336910626"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Kendall Carneiro","raw_affiliation_strings":["Hemostasis and Thrombosis Center Children's Hospital Los Angeles Los Angeles California USA","Hemostasis and Thrombosis Center, Children's Hospital Los Angeles, Los Angeles, California, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Hemostasis and Thrombosis Center Children's Hospital Los Angeles Los Angeles California USA","institution_ids":["https://openalex.org/I1336910626"]},{"raw_affiliation_string":"Hemostasis and Thrombosis Center, Children's Hospital Los Angeles, Los Angeles, California, USA","institution_ids":["https://openalex.org/I1336910626"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5060869564","display_name":"Jemily Malvar","orcid":"https://orcid.org/0000-0002-5481-6780","observed_orcids":["https://orcid.org/0000-0002-5481-6780"]},"institutions":[{"id":"https://openalex.org/I1336910626","display_name":"Children's Hospital of Los Angeles","ror":"https://ror.org/00412ts95","country_code":"US","type":"funder","lineage":["https://openalex.org/I1336910626"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Jemily Malvar","raw_affiliation_strings":["Cancer and Blood Disease Institute Children's Hospital Los Angeles Los Angeles California USA","Cancer and Blood Disease Institute, Children's Hospital Los Angeles, Los Angeles, California, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Cancer and Blood Disease Institute Children's Hospital Los Angeles Los Angeles California USA","institution_ids":["https://openalex.org/I1336910626"]},{"raw_affiliation_string":"Cancer and Blood Disease Institute, Children's Hospital Los Angeles, Los Angeles, California, USA","institution_ids":["https://openalex.org/I1336910626"]}]},{"author_position":"middle","author":{"id":null,"display_name":"Susan Knight","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I1336910626","display_name":"Children's Hospital of Los Angeles","ror":"https://ror.org/00412ts95","country_code":"US","type":"funder","lineage":["https://openalex.org/I1336910626"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Susan Knight","raw_affiliation_strings":["Hemostasis and Thrombosis Center Children's Hospital Los Angeles Los Angeles California USA","Hemostasis and Thrombosis Center, Children's Hospital Los Angeles, Los Angeles, California, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Hemostasis and Thrombosis Center Children's Hospital Los Angeles Los Angeles California USA","institution_ids":["https://openalex.org/I1336910626"]},{"raw_affiliation_string":"Hemostasis and Thrombosis Center, Children's Hospital Los Angeles, Los Angeles, California, USA","institution_ids":["https://openalex.org/I1336910626"]}]},{"author_position":"last","author":{"id":"https://openalex.org/A5004338432","display_name":"Guy A. Young","orcid":"https://orcid.org/0000-0001-6013-1254","observed_orcids":["https://orcid.org/0000-0001-6013-1254"]},"institutions":[{"id":"https://openalex.org/I1174212","display_name":"University of Southern California","ror":"https://ror.org/03taz7m60","country_code":"US","type":"education","lineage":["https://openalex.org/I1174212"]},{"id":"https://openalex.org/I1336910626","display_name":"Children's Hospital of Los Angeles","ror":"https://ror.org/00412ts95","country_code":"US","type":"funder","lineage":["https://openalex.org/I1336910626"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Guy Young","raw_affiliation_strings":["Cancer and Blood Disease Institute Children's Hospital Los Angeles Los Angeles California USA","Department of Pediatrics University of Southern California, Keck School of Medicine Los Angeles California USA","Hemostasis and Thrombosis Center Children's Hospital Los Angeles Los Angeles California USA","Cancer and Blood Disease Institute, Children's Hospital Los Angeles, Los Angeles, California, USA","Department of Pediatrics, University of Southern California, Keck School of Medicine, Los Angeles, California, USA","Hemostasis and Thrombosis Center, Children's Hospital Los Angeles, Los Angeles, California, USA"],"raw_orcid":"https://orcid.org/0000-0001-6013-1254","affiliations":[{"raw_affiliation_string":"Cancer and Blood Disease Institute Children's Hospital Los Angeles Los Angeles California USA","institution_ids":["https://openalex.org/I1336910626"]},{"raw_affiliation_string":"Department of Pediatrics University of Southern California, Keck School of Medicine Los Angeles California USA","institution_ids":["https://openalex.org/I1174212"]},{"raw_affiliation_string":"Hemostasis and Thrombosis Center Children's Hospital Los Angeles Los Angeles California USA","institution_ids":["https://openalex.org/I1336910626"]},{"raw_affiliation_string":"Cancer and Blood Disease Institute, Children's Hospital Los Angeles, Los Angeles, California, USA","institution_ids":["https://openalex.org/I1336910626"]},{"raw_affiliation_string":"Department of Pediatrics, University of Southern California, Keck School of Medicine, Los Angeles, California, USA","institution_ids":["https://openalex.org/I1174212"]},{"raw_affiliation_string":"Hemostasis and Thrombosis Center, Children's Hospital Los Angeles, Los Angeles, California, USA","institution_ids":["https://openalex.org/I1336910626"]}]}],"institutions":[],"countries_distinct_count":1,"institutions_distinct_count":3,"corresponding_author_ids":["https://openalex.org/A5033588580"],"corresponding_institution_ids":["https://openalex.org/I1336910626","https://openalex.org/I93393672"],"apc_list":{"value":4550,"currency":"USD","value_usd":4550},"apc_paid":{"value":4550,"currency":"USD","value_usd":4550},"fwci":0.8035,"has_fulltext":true,"cited_by_count":3,"citation_normalized_percentile":{"value":0.73956743,"is_in_top_1_percent":false,"is_in_top_10_percent":false},"cited_by_percentile_year":{"min":96,"max":97},"biblio":{"volume":"71","issue":"12","first_page":"e31351","last_page":"e31351"},"is_retracted":false,"is_paratext":false,"is_xpac":false,"primary_topic":{"id":"https://openalex.org/T11280","display_name":"Hemophilia Treatment and Research","score":1.0,"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"}},"topics":[{"id":"https://openalex.org/T11280","display_name":"Hemophilia Treatment and Research","score":1.0,"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/T10746","display_name":"Platelet Disorders and Treatments","score":0.0,"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/T11917","display_name":"Coagulation, Bradykinin, Polyphosphates, and Angioedema","score":0.0,"subfield":{"id":"https://openalex.org/subfields/2716","display_name":"Genetics"},"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/hemophilia-a","display_name":"hemophilia A","score":0.9539999961853027},{"id":"https://openalex.org/keywords/emicizumab","display_name":"emicizumab","score":0.9330000281333923},{"id":"https://openalex.org/keywords/factor-viii-prophylaxis","display_name":"factor VIII prophylaxis","score":0.8889999985694885},{"id":"https://openalex.org/keywords/clotting-factor-concentrates","display_name":"clotting factor concentrates","score":0.8320000171661377},{"id":"https://openalex.org/keywords/annualized-bleeding-rate","display_name":"annualized bleeding rate","score":0.9570000171661377},{"id":"https://openalex.org/keywords/hemarthrosis","display_name":"hemarthrosis","score":0.9039999842643738},{"id":"https://openalex.org/keywords/children","display_name":"children","score":0.5540000200271606},{"id":"https://openalex.org/keywords/treatment-costs","display_name":"treatment costs","score":0.48500001430511475},{"id":"https://openalex.org/keywords/factor-viii-inhibitors","display_name":"factor VIII inhibitors","score":0.5799999833106995}],"concepts":[{"id":"https://openalex.org/C71924100","wikidata":"https://www.wikidata.org/wiki/Q11190","display_name":"Medicine","level":0,"score":0.9316651821136475},{"id":"https://openalex.org/C2992139802","wikidata":"https://www.wikidata.org/wiki/Q179217","display_name":"Clotting factor","level":2,"score":0.49231112003326416},{"id":"https://openalex.org/C187212893","wikidata":"https://www.wikidata.org/wiki/Q123028","display_name":"Pediatrics","level":1,"score":0.47693198919296265},{"id":"https://openalex.org/C126322002","wikidata":"https://www.wikidata.org/wiki/Q11180","display_name":"Internal medicine","level":1,"score":0.40220800042152405}],"mesh":[{"descriptor_ui":"D002648","descriptor_name":"Child","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D002648","descriptor_name":"Child","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D002648","descriptor_name":"Child","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D002648","descriptor_name":"Child","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D002648","descriptor_name":"Child","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D002648","descriptor_name":"Child","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D002675","descriptor_name":"Child, Preschool","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D002675","descriptor_name":"Child, Preschool","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D002675","descriptor_name":"Child, Preschool","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D002675","descriptor_name":"Child, Preschool","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D002675","descriptor_name":"Child, Preschool","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D002675","descriptor_name":"Child, Preschool","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D005169","descriptor_name":"Factor VIII","qualifier_ui":"Q000627","qualifier_name":"therapeutic use","is_major_topic":true},{"descriptor_ui":"D005169","descriptor_name":"Factor VIII","qualifier_ui":"Q000627","qualifier_name":"therapeutic use","is_major_topic":true},{"descriptor_ui":"D005169","descriptor_name":"Factor VIII","qualifier_ui":"Q000627","qualifier_name":"therapeutic use","is_major_topic":true},{"descriptor_ui":"D005169","descriptor_name":"Factor VIII","qualifier_ui":"Q000627","qualifier_name":"therapeutic use","is_major_topic":true},{"descriptor_ui":"D005169","descriptor_name":"Factor VIII","qualifier_ui":"Q000627","qualifier_name":"therapeutic use","is_major_topic":true},{"descriptor_ui":"D005169","descriptor_name":"Factor VIII","qualifier_ui":"Q000627","qualifier_name":"therapeutic use","is_major_topic":true},{"descriptor_ui":"D005260","descriptor_name":"Female","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D005260","descriptor_name":"Female","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D005260","descriptor_name":"Female","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D005260","descriptor_name":"Female","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D005260","descriptor_name":"Female","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D005260","descriptor_name":"Female","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D005500","descriptor_name":"Follow-Up Studies","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D005500","descriptor_name":"Follow-Up Studies","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D005500","descriptor_name":"Follow-Up Studies","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D005500","descriptor_name":"Follow-Up Studies","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D005500","descriptor_name":"Follow-Up Studies","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D005500","descriptor_name":"Follow-Up Studies","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D006467","descriptor_name":"Hemophilia A","qualifier_ui":"Q000150","qualifier_name":"complications","is_major_topic":true},{"descriptor_ui":"D006467","descriptor_name":"Hemophilia A","qualifier_ui":"Q000150","qualifier_name":"complications","is_major_topic":true},{"descriptor_ui":"D006467","descriptor_name":"Hemophilia A","qualifier_ui":"Q000150","qualifier_name":"complications","is_major_topic":true},{"descriptor_ui":"D006467","descriptor_name":"Hemophilia A","qualifier_ui":"Q000150","qualifier_name":"complications","is_major_topic":true},{"descriptor_ui":"D006467","descriptor_name":"Hemophilia A","qualifier_ui":"Q000150","qualifier_name":"complications","is_major_topic":true},{"descriptor_ui":"D006467","descriptor_name":"Hemophilia A","qualifier_ui":"Q000150","qualifier_name":"complications","is_major_topic":true},{"descriptor_ui":"D006467","descriptor_name":"Hemophilia A","qualifier_ui":"Q000188","qualifier_name":"drug therapy","is_major_topic":true},{"descriptor_ui":"D006467","descriptor_name":"Hemophilia A","qualifier_ui":"Q000188","qualifier_name":"drug therapy","is_major_topic":true},{"descriptor_ui":"D006467","descriptor_name":"Hemophilia A","qualifier_ui":"Q000188","qualifier_name":"drug therapy","is_major_topic":true},{"descriptor_ui":"D006467","descriptor_name":"Hemophilia A","qualifier_ui":"Q000188","qualifier_name":"drug therapy","is_major_topic":true},{"descriptor_ui":"D006467","descriptor_name":"Hemophilia A","qualifier_ui":"Q000188","qualifier_name":"drug therapy","is_major_topic":true},{"descriptor_ui":"D006467","descriptor_name":"Hemophilia A","qualifier_ui":"Q000188","qualifier_name":"drug therapy","is_major_topic":true},{"descriptor_ui":"D006470","descriptor_name":"Hemorrhage","qualifier_ui":"Q000139","qualifier_name":"chemically induced","is_major_topic":false},{"descriptor_ui":"D006470","descriptor_name":"Hemorrhage","qualifier_ui":"Q000139","qualifier_name":"chemically induced","is_major_topic":false},{"descriptor_ui":"D006470","descriptor_name":"Hemorrhage","qualifier_ui":"Q000139","qualifier_name":"chemically induced","is_major_topic":false},{"descriptor_ui":"D006470","descriptor_name":"Hemorrhage","qualifier_ui":"Q000139","qualifier_name":"chemically induced","is_major_topic":false},{"descriptor_ui":"D006470","descriptor_name":"Hemorrhage","qualifier_ui":"Q000139","qualifier_name":"chemically induced","is_major_topic":false},{"descriptor_ui":"D006470","descriptor_name":"Hemorrhage","qualifier_ui":"Q000139","qualifier_name":"chemically induced","is_major_topic":false},{"descriptor_ui":"D006470","descriptor_name":"Hemorrhage","qualifier_ui":"Q000517","qualifier_name":"prevention & control","is_major_topic":false},{"descriptor_ui":"D006470","descriptor_name":"Hemorrhage","qualifier_ui":"Q000517","qualifier_name":"prevention & control","is_major_topic":false},{"descriptor_ui":"D006470","descriptor_name":"Hemorrhage","qualifier_ui":"Q000517","qualifier_name":"prevention & control","is_major_topic":false},{"descriptor_ui":"D006470","descriptor_name":"Hemorrhage","qualifier_ui":"Q000517","qualifier_name":"prevention & control","is_major_topic":false},{"descriptor_ui":"D006470","descriptor_name":"Hemorrhage","qualifier_ui":"Q000517","qualifier_name":"prevention & control","is_major_topic":false},{"descriptor_ui":"D006470","descriptor_name":"Hemorrhage","qualifier_ui":"Q000517","qualifier_name":"prevention & control","is_major_topic":false},{"descriptor_ui":"D006801","descriptor_name":"Humans","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D006801","descriptor_name":"Humans","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D006801","descriptor_name":"Humans","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D006801","descriptor_name":"Humans","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D006801","descriptor_name":"Humans","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D006801","descriptor_name":"Humans","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D007223","descriptor_name":"Infant","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D007223","descriptor_name":"Infant","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D007223","descriptor_name":"Infant","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D007223","descriptor_name":"Infant","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D007223","descriptor_name":"Infant","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D007223","descriptor_name":"Infant","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D008297","descriptor_name":"Male","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D008297","descriptor_name":"Male","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D008297","descriptor_name":"Male","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D008297","descriptor_name":"Male","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D008297","descriptor_name":"Male","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D008297","descriptor_name":"Male","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D012189","descriptor_name":"Retrospective Studies","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D012189","descriptor_name":"Retrospective Studies","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D012189","descriptor_name":"Retrospective Studies","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D012189","descriptor_name":"Retrospective Studies","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D012189","descriptor_name":"Retrospective Studies","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D012189","descriptor_name":"Retrospective Studies","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D018033","descriptor_name":"Antibodies, Bispecific","qualifier_ui":"Q000627","qualifier_name":"therapeutic use","is_major_topic":true},{"descriptor_ui":"D018033","descriptor_name":"Antibodies, Bispecific","qualifier_ui":"Q000627","qualifier_name":"therapeutic use","is_major_topic":true},{"descriptor_ui":"D018033","descriptor_name":"Antibodies, Bispecific","qualifier_ui":"Q000627","qualifier_name":"therapeutic use","is_major_topic":true},{"descriptor_ui":"D018033","descriptor_name":"Antibodies, Bispecific","qualifier_ui":"Q000627","qualifier_name":"therapeutic use","is_major_topic":true},{"descriptor_ui":"D018033","descriptor_name":"Antibodies, Bispecific","qualifier_ui":"Q000627","qualifier_name":"therapeutic use","is_major_topic":true},{"descriptor_ui":"D018033","descriptor_name":"Antibodies, Bispecific","qualifier_ui":"Q000627","qualifier_name":"therapeutic use","is_major_topic":true},{"descriptor_ui":"D061067","descriptor_name":"Antibodies, Monoclonal, Humanized","qualifier_ui":"Q000008","qualifier_name":"administration & dosage","is_major_topic":true},{"descriptor_ui":"D061067","descriptor_name":"Antibodies, Monoclonal, Humanized","qualifier_ui":"Q000008","qualifier_name":"administration & dosage","is_major_topic":true},{"descriptor_ui":"D061067","descriptor_name":"Antibodies, Monoclonal, Humanized","qualifier_ui":"Q000008","qualifier_name":"administration & dosage","is_major_topic":true},{"descriptor_ui":"D061067","descriptor_name":"Antibodies, Monoclonal, Humanized","qualifier_ui":"Q000008","qualifier_name":"administration & dosage","is_major_topic":true},{"descriptor_ui":"D061067","descriptor_name":"Antibodies, Monoclonal, Humanized","qualifier_ui":"Q000008","qualifier_name":"administration & dosage","is_major_topic":true},{"descriptor_ui":"D061067","descriptor_name":"Antibodies, Monoclonal, Humanized","qualifier_ui":"Q000008","qualifier_name":"administration & dosage","is_major_topic":true},{"descriptor_ui":"D061067","descriptor_name":"Antibodies, Monoclonal, Humanized","qualifier_ui":"Q000627","qualifier_name":"therapeutic use","is_major_topic":true},{"descriptor_ui":"D061067","descriptor_name":"Antibodies, Monoclonal, Humanized","qualifier_ui":"Q000627","qualifier_name":"therapeutic use","is_major_topic":true},{"descriptor_ui":"D061067","descriptor_name":"Antibodies, Monoclonal, Humanized","qualifier_ui":"Q000627","qualifier_name":"therapeutic use","is_major_topic":true},{"descriptor_ui":"D061067","descriptor_name":"Antibodies, Monoclonal, Humanized","qualifier_ui":"Q000627","qualifier_name":"therapeutic use","is_major_topic":true},{"descriptor_ui":"D061067","descriptor_name":"Antibodies, Monoclonal, Humanized","qualifier_ui":"Q000627","qualifier_name":"therapeutic use","is_major_topic":true},{"descriptor_ui":"D061067","descriptor_name":"Antibodies, Monoclonal, Humanized","qualifier_ui":"Q000627","qualifier_name":"therapeutic use","is_major_topic":true}],"locations_count":2,"locations":[{"id":"doi:10.1002/pbc.31351","doi":"https://doi.org/10.1002/pbc.31351","is_oa":true,"landing_page_url":"https://doi.org/10.1002/pbc.31351","pdf_url":"https://onlinelibrary.wiley.com/doi/pdfdirect/10.1002/pbc.31351","source":{"id":"https://openalex.org/S119714264","display_name":"Pediatric Blood & Cancer","issn_l":"1545-5009","issn":["1545-5009","1545-5017"],"is_oa":false,"is_in_doaj":false,"is_core":true,"listed_in":["anvur-scientific","cwts-core","dongbi-c","doyens","jufo-1","ki-jl-1","medline","norway-1","poland-100","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310320595","host_organization_name":"Wiley","host_organization_lineage":["https://openalex.org/P4310320595"],"host_organization_lineage_names":["Wiley"],"type":"journal"},"license":"cc-by-nc","license_id":"https://openalex.org/licenses/cc-by-nc","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Pediatric Blood &amp; Cancer","raw_type":"journal-article"},{"id":"pmid:39367598","doi":null,"is_oa":false,"landing_page_url":"https://pubmed.ncbi.nlm.nih.gov/39367598","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":"Pediatric blood & cancer","raw_type":"Journal Article"}],"best_oa_location":{"id":"doi:10.1002/pbc.31351","doi":"https://doi.org/10.1002/pbc.31351","is_oa":true,"landing_page_url":"https://doi.org/10.1002/pbc.31351","pdf_url":"https://onlinelibrary.wiley.com/doi/pdfdirect/10.1002/pbc.31351","source":{"id":"https://openalex.org/S119714264","display_name":"Pediatric Blood & Cancer","issn_l":"1545-5009","issn":["1545-5009","1545-5017"],"is_oa":false,"is_in_doaj":false,"is_core":true,"listed_in":["anvur-scientific","cwts-core","dongbi-c","doyens","jufo-1","ki-jl-1","medline","norway-1","poland-100","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310320595","host_organization_name":"Wiley","host_organization_lineage":["https://openalex.org/P4310320595"],"host_organization_lineage_names":["Wiley"],"type":"journal"},"license":"cc-by-nc","license_id":"https://openalex.org/licenses/cc-by-nc","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Pediatric Blood &amp; Cancer","raw_type":"journal-article"},"sustainable_development_goals":[{"id":"https://metadata.un.org/sdg/3","score":1.0,"display_name":"Good health and well-being"}],"sustainable_development_goals_aurora":[{"id":"https://metadata.un.org/sdg/17","score":0.49000000953674316,"display_name":"Partnerships for the goals"}],"study_designs":[{"id":"https://openalex.org/study-designs/observational-study","display_name":"Observational Study"}],"awards":[],"funders":[],"has_content":{"grobid_xml":true,"pdf":true},"content_urls":{"pdf":"https://content.openalex.org/works/W4403158181.pdf","grobid_xml":"https://content.openalex.org/works/W4403158181.grobid-xml"},"referenced_works_count":22,"referenced_works":["https://openalex.org/W1201749067","https://openalex.org/W1969725427","https://openalex.org/W2020123164","https://openalex.org/W2026502021","https://openalex.org/W2054783695","https://openalex.org/W2168158593","https://openalex.org/W2407876292","https://openalex.org/W2888898681","https://openalex.org/W2919343333","https://openalex.org/W2980900975","https://openalex.org/W2988943167","https://openalex.org/W2990987090","https://openalex.org/W3016733980","https://openalex.org/W3029400735","https://openalex.org/W3046170337","https://openalex.org/W3186150392","https://openalex.org/W4200525245","https://openalex.org/W4304775574","https://openalex.org/W4310093478","https://openalex.org/W4384925496","https://openalex.org/W4390047763","https://openalex.org/W6903533664"],"related_works":["https://openalex.org/W2748952813","https://openalex.org/W3031052312","https://openalex.org/W4389568370","https://openalex.org/W3032375762","https://openalex.org/W1995515455","https://openalex.org/W2080531066","https://openalex.org/W3108674512","https://openalex.org/W1506200166","https://openalex.org/W1489783725","https://openalex.org/W2148612803"],"abstract_inverted_index":{"BACKGROUND:":[0],"Hemophilia":[1],"A":[2],"(HA)":[3],"is":[4,97,249],"an":[5,43,221],"X-linked":[6],"bleeding":[7,45],"disorder":[8],"diagnosed":[9],"by":[10],"a":[11,35,51,115,124,161,252],"deficiency":[12],"in":[13,42,67,78,87,109,220],"factor":[14,23],"VIII":[15],"(FVIII).":[16],"For":[17],"severe":[18],"HA":[19],"(SHA),":[20],"prophylaxis":[21,62,201,213],"clotting":[22],"concentrates":[24],"(CFC)":[25],"has":[26,56],"become":[27,57],"the":[28,58,102,256],"standard":[29],"of":[30,48,73,104,127,134,139,165,199,255,258],"care;":[31],"however,":[32,95,246],"it":[33],"imparts":[34],"high":[36],"treatment":[37],"burden":[38],"and":[39,86,150,154,175,230,237,263],"typically":[40],"results":[41],"annualized":[44],"rate":[46,164],"(ABR)":[47],"2-6.":[49],"Emicizumab,":[50],"subcutaneously":[52],"administered":[53],"FVIII":[54],"substitute,":[55],"de":[59],"facto":[60],"standard-of-care":[61],"for":[63,190,211,228,251],"children":[64,88],"with":[65,93,160],"SHA":[66,111],"many":[68],"countries.":[69],"Previous":[70],"clinical":[71],"trials":[72],"emicizumab":[74,105,142,151,170,180,212,259],"have":[75],"assessed":[76],"ABR":[77,103,147,223],"patients":[79,129,191],"greater":[80],"than":[81,90,131],"12":[82,91,132],"years":[83,92,133],"without":[84],"inhibitors,":[85],"less":[89,130,240],"inhibitors;":[94],"there":[96],"little":[98],"information":[99],"published":[100],"regarding":[101],"compared":[106,206,224,242],"to":[107,207,225,243],"CFC":[108,140,149,200,244],"non-inhibitor":[110],"children.":[112],"METHODS:":[113],"Using":[114],"retrospective":[116],"electronic":[117],"medical":[118],"record":[119],"chart":[120],"review,":[121],"we":[122],"conducted":[123],"self-control":[125],"analysis":[126],"15":[128],"age":[135],"during":[136],"equivalent":[137],"periods":[138],"versus":[141],"prophylaxis.":[143],"RESULTS:":[144],"The":[145,195],"mean":[146,196],"on":[148,260],"was":[152,202,238],"1.79":[153],"1.13":[155],"(p":[156,214],"=":[157,173,183],".092),":[158],"respectively,":[159],"substantially":[162,239],"decreased":[163],"joint":[166,229,261],"bleeds":[167,177],"(CFC":[168,178],"0.94;":[169],"0.33;":[171],"p":[172,182],".001)":[174],"spontaneous":[176,231],"0.79;":[179],"0.23;":[181],".008).":[184],"No":[185],"safety":[186],"events":[187],"were":[188],"recorded":[189],"while":[192],"administering":[193],"emicizumab.":[194],"annual":[197],"cost":[198],"$515,340":[203],"(SD":[204,209],"$199,540),":[205],"$328,410":[208],"$137,230)":[210],"<":[215],".001).":[216],"CONCLUSION:":[217],"Emicizumab":[218],"resulted":[219],"improved":[222],"CFC,":[226],"especially":[227],"bleeds,":[232],"had":[233],"fewer":[234],"administration":[235],"complications,":[236],"expensive":[241],"prophylaxis;":[245],"more":[247],"research":[248],"necessary":[250],"complete":[253],"understanding":[254],"effect":[257],"health":[262],"muscle":[264],"bleeds.":[265]},"counts_by_year":[{"year":2025,"cited_by_count":3}],"updated_date":"2026-10-16T09:59:22.235173","created_date":"2025-10-10T00:00:00"}
