{"id":"https://openalex.org/W4252974875","doi":"https://doi.org/10.3410/f.727123129.793544797","title":"Faculty Opinions recommendation of Safety and efficacy of BAY 94-9027, a prolonged-half-life factor VIII.","display_name":"Faculty Opinions recommendation of Safety and efficacy of BAY 94-9027, a prolonged-half-life factor VIII.","publication_year":2018,"publication_date":"2018-04-13","ids":{"openalex":"https://openalex.org/W4252974875","doi":"https://doi.org/10.3410/f.727123129.793544797"},"language":"en","primary_location":{"id":"doi:10.3410/f.727123129.793544797","doi":"https://doi.org/10.3410/f.727123129.793544797","is_oa":false,"landing_page_url":"https://doi.org/10.3410/f.727123129.793544797","pdf_url":null,"source":{"id":"https://openalex.org/S4363604054","display_name":"Faculty Opinions \u2013 Post-Publication Peer Review of the Biomedical Literature","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":null,"host_organization_name":null,"host_organization_lineage":[],"host_organization_lineage_names":[],"type":"repository"},"license":null,"license_id":null,"version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Faculty Opinions \u2013 Post-Publication Peer Review of the Biomedical Literature","raw_type":"dataset"},"volume":null,"type":"peer-review","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/A5083658192","display_name":"Valder R. Arruda","orcid":"https://orcid.org/0000-0002-8100-5481","observed_orcids":["https://orcid.org/0000-0002-8100-5481"]},"institutions":[{"id":"https://openalex.org/I79576946","display_name":"University of Pennsylvania","ror":"https://ror.org/00b30xv10","country_code":"US","type":"education","lineage":["https://openalex.org/I79576946"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Valder Arruda","raw_affiliation_strings":["Gene Therapy and Vaccines Faculty, University of Pennsylvania, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Gene Therapy and Vaccines Faculty, University of Pennsylvania, USA","institution_ids":["https://openalex.org/I79576946"]}]},{"author_position":"last","author":{"id":"https://openalex.org/A5113997051","display_name":"Ben J. Samelson-Jones","orcid":"https://orcid.org/0000-0001-6772-4140","observed_orcids":["https://orcid.org/0000-0001-6772-4140"]},"institutions":[{"id":"https://openalex.org/I1335321130","display_name":"Children's Hospital of Philadelphia","ror":"https://ror.org/01z7r7q48","country_code":"US","type":"funder","lineage":["https://openalex.org/I1335321130"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Ben Samelson-Jones","raw_affiliation_strings":["Department of Pediatrics, Division of Hematology, The Children's Hospital of Philadelphia, USA"],"raw_orcid":"https://orcid.org/0000-0001-6772-4140","affiliations":[{"raw_affiliation_string":"Department of Pediatrics, Division of Hematology, The Children's Hospital of Philadelphia, USA","institution_ids":["https://openalex.org/I1335321130"]}]}],"institutions":[],"countries_distinct_count":1,"institutions_distinct_count":2,"corresponding_author_ids":[],"corresponding_institution_ids":[],"apc_list":null,"apc_paid":null,"fwci":null,"has_fulltext":false,"cited_by_count":0,"citation_normalized_percentile":null,"cited_by_percentile_year":null,"biblio":{"volume":null,"issue":null,"first_page":null,"last_page":null},"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/extended-half-life-factor-viii","display_name":"extended half-life factor VIII","score":0.7789999842643738},{"id":"https://openalex.org/keywords/hemophilia-a","display_name":"hemophilia A","score":0.8960000276565552},{"id":"https://openalex.org/keywords/polyethylene-glycol-conjugation","display_name":"polyethylene glycol conjugation","score":0.7979999780654907},{"id":"https://openalex.org/keywords/b-domain-deletion","display_name":"B-domain deletion","score":0.7860000133514404},{"id":"https://openalex.org/keywords/prophylaxis","display_name":"prophylaxis","score":0.8970000147819519},{"id":"https://openalex.org/keywords/on-demand-treatment","display_name":"on-demand treatment","score":0.7739999890327454},{"id":"https://openalex.org/keywords/annualized-bleeding-rate","display_name":"annualized bleeding rate","score":0.9769999980926514},{"id":"https://openalex.org/keywords/factor-viii-inhibitors","display_name":"factor VIII inhibitors","score":0.9229999780654907},{"id":"https://openalex.org/keywords/treatment-safety","display_name":"treatment safety","score":0.4970000088214874},{"id":"https://openalex.org/keywords/recombinant-factor-viii","display_name":"recombinant factor VIII","score":0.7020000219345093},{"id":"https://openalex.org/keywords/bleeding-prevention","display_name":"bleeding prevention","score":0.47999998927116394},{"id":"https://openalex.org/keywords/treatment-tolerability","display_name":"treatment tolerability","score":0.4449999928474426}],"concepts":[{"id":"https://openalex.org/C71924100","wikidata":"https://www.wikidata.org/wiki/Q11190","display_name":"Medicine","level":0,"score":0.8542932868003845},{"id":"https://openalex.org/C204243189","wikidata":"https://www.wikidata.org/wiki/Q1363085","display_name":"Randomization","level":3,"score":0.5647568106651306},{"id":"https://openalex.org/C141071460","wikidata":"https://www.wikidata.org/wiki/Q40821","display_name":"Surgery","level":1,"score":0.4932476878166199},{"id":"https://openalex.org/C168563851","wikidata":"https://www.wikidata.org/wiki/Q1436668","display_name":"Randomized controlled trial","level":2,"score":0.44514578580856323},{"id":"https://openalex.org/C2776259030","wikidata":"https://www.wikidata.org/wiki/Q769508","display_name":"Bleed","level":2,"score":0.4448864758014679},{"id":"https://openalex.org/C115880899","wikidata":"https://www.wikidata.org/wiki/Q1124247","display_name":"Bay","level":2,"score":0.4191006124019623},{"id":"https://openalex.org/C126322002","wikidata":"https://www.wikidata.org/wiki/Q11180","display_name":"Internal medicine","level":1,"score":0.3314785063266754},{"id":"https://openalex.org/C147176958","wikidata":"https://www.wikidata.org/wiki/Q77590","display_name":"Civil engineering","level":1,"score":0.0},{"id":"https://openalex.org/C127413603","wikidata":"https://www.wikidata.org/wiki/Q11023","display_name":"Engineering","level":0,"score":0.0}],"mesh":[],"locations_count":1,"locations":[{"id":"doi:10.3410/f.727123129.793544797","doi":"https://doi.org/10.3410/f.727123129.793544797","is_oa":false,"landing_page_url":"https://doi.org/10.3410/f.727123129.793544797","pdf_url":null,"source":{"id":"https://openalex.org/S4363604054","display_name":"Faculty Opinions \u2013 Post-Publication Peer Review of the Biomedical Literature","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":null,"host_organization_name":null,"host_organization_lineage":[],"host_organization_lineage_names":[],"type":"repository"},"license":null,"license_id":null,"version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Faculty Opinions \u2013 Post-Publication Peer Review of the Biomedical Literature","raw_type":"dataset"}],"best_oa_location":null,"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/2","score":0.6600000262260437,"display_name":"Zero hunger"}],"study_designs":[],"awards":[],"funders":[],"has_content":{"pdf":false,"grobid_xml":false},"content_urls":null,"referenced_works_count":0,"referenced_works":[],"related_works":["https://openalex.org/W2566584570","https://openalex.org/W3146526119","https://openalex.org/W2913571980","https://openalex.org/W2917616103","https://openalex.org/W2981289366","https://openalex.org/W2924601621","https://openalex.org/W2985586814","https://openalex.org/W3013359875","https://openalex.org/W2059843712","https://openalex.org/W2971032798"],"abstract_inverted_index":{"Essentials":[0],"Recombinant":[1],"factor":[2,57,69,86],"VIII":[3,58,70,87],"BAY":[4,14,30,46,79,104,144,359],"94-9027":[5,15,31,47,80,105,145,360],"conjugates":[6,90],"in":[7,91,112],"a":[8,82,92,157,355],"site-specific":[9,93],"manner":[10,94],"with":[11,20,95,114,132,172,189,348],"polyethylene":[12,96],"glycol.":[13,97],"was":[16,49,236,300,334,370],"given":[17],"to":[18,27,38,61,141,197,285,294,305],"patients":[19,113,188,247,263,292,306,325],"severe":[21,115],"hemophilia":[22,75,116],"A":[23,76],"as":[24],"prophylaxis":[25,107,209,330],"and":[26,42,53,101,108,136,340,369,382,396],"treat":[28],"bleeds.":[29,45],"prevented":[32,361],"bleeds":[33,111,344],"at":[34,153],"dose":[35,280,367],"intervals":[36,154],"up":[37],"every":[39],"7":[40],"days":[41,140],"effectively":[43],"treated":[44,264],"treatment":[48,73,109,296,313,373],"mainly":[50],"well":[51],"tolerated":[52],"no":[54],"patient":[55,353],"developed":[56,354],"inhibitors.":[59],"Click":[60],"hear":[62],"Dr":[63],"Tiede's":[64],"perspective":[65],"on":[66,149,161,252,389,394],"half-life":[67],"extended":[68],"for":[71,106,146,195,213,262,273,291,308,323,372],"the":[72,177],"of":[74,103,110,342,374,380,391],"SUMMARY:":[77],"Background":[78],"is":[81],"B-domain-deleted":[83],"prolonged-half-life":[84],"recombinant":[85],"(FVIII)":[88],"that":[89],"Objective":[98],"Assess":[99],"efficacy":[100,234],"safety":[102],"A.":[117],"Patients/methods":[118],"In":[119],"this":[120],"multinational,":[121],"phase":[122],"2/3,":[123],"partially":[124],"randomized,":[125],"open-label":[126],"trial,":[127],"men":[128],"aged":[129],"12-65":[130],"years":[131],"FVIII":[133,142,356],"<":[134],"1%":[135],"\u2265":[137],"150":[138],"exposure":[139],"received":[143,180],"36":[147],"weeks":[148,216],"demand":[150],"or":[151,203],"prophylactically":[152],"determined":[155],"following":[156],"10-week":[158],"run-in":[159,178],"period":[160],"25":[162],"IU":[163,182,200,206],"kg-1":[164,183,201,207],"body":[165],"weight":[166],"two":[167,184,265,314],"times":[168,185,266,315],"per":[169,186,267,316],"week.":[170],"Patients":[171,222],">":[173],"1":[174,191],"bleed":[175,192],"during":[176],"subsequently":[179],"30-40":[181],"week;":[187],"\u2264":[190,349],"were":[193,220,224,270,346],"eligible":[194,225,272,307],"randomization":[196,218,274,309],"every-5-days":[198,295],"(45-60":[199],")":[202,208],"every-7-days":[204,329],"(60":[205],"(1":[210],":":[211],"1)":[212],"26":[214],"additional":[215],"until":[217,331],"arms":[219],"filled.":[221],"who":[223,269,311,327],"but":[226,310],"not":[227,271],"randomized":[228,293],"continued":[229,312,328],"twice-weekly":[230],"prophylaxis.":[231],"The":[232,242,377],"primary":[233],"outcome":[235],"annualized":[237],"bleeding":[238,362],"rate":[239],"(ABR).":[240],"Results":[241],"intent-to-treat":[243],"population":[244],"included":[245],"132":[246],"(prophylaxis,":[248],"n":[249,254],"=":[250,255,276,298,319],"112;":[251],"demand,":[253],"20).":[256],"Median":[257,289,321],"ABR":[258,290,322],"(quartile":[259],"[Q1;":[260],"Q3])":[261],"week":[268,317],"(n":[275,297,318],"13)":[277],"improved":[278],"after":[279],"increase":[281],"(17.4":[282],"[14.3;":[283],"26.0]":[284],"4.1":[286],"[2.0;":[287],"10.6]).":[288],"43)":[299],"1.9":[301],"(0;":[302],"4.2),":[303],"similar":[304],"11).":[320],"32/43":[324],"(74%)":[326],"study":[332],"end":[333],"0.96":[335],"(0.0;":[336],"4.3).":[337],"Six":[338],"hundred":[339],"thirty-six":[341],"702":[343],"(90.6%)":[345],"controlled":[347],"2":[350],"infusions.":[351],"No":[352],"inhibitor.":[357],"Conclusions":[358],"across":[363],"three":[364],"individually":[365],"tailored":[366],"regimens":[368],"effective":[371],"bleeds.\u00a9":[375],"2016":[376],"Authors.":[378],"Journal":[379],"Thrombosis":[381,395],"Haemostasis":[383],"published":[384],"by":[385],"Wiley":[386],"Periodicals,":[387],"Inc.":[388],"behalf":[390],"International":[392],"Society":[393],"Haemostasis.":[397],"PMID:":[398],"27992112":[399]},"counts_by_year":[],"updated_date":"2026-09-29T14:09:16.559479","created_date":"2022-05-12T00:00:00"}
