{"id":"https://openalex.org/W4283396263","doi":"https://doi.org/10.1097/01.hs9.0000849036.11558.e5","title":"P1545: DURABILITY OF HEMOGLOBIN RESPONSE AND REDUCTION IN TRANSFUSION BURDEN IS MAINTAINED OVER TIME IN PATIENTS WITH PYRUVATE KINASE DEFICIENCY TREATED WITH MITAPIVAT IN A LONG-TERM EXTENSION STUDY","display_name":"P1545: DURABILITY OF HEMOGLOBIN RESPONSE AND REDUCTION IN TRANSFUSION BURDEN IS MAINTAINED OVER TIME IN PATIENTS WITH PYRUVATE KINASE DEFICIENCY TREATED WITH MITAPIVAT IN A LONG-TERM EXTENSION STUDY","publication_year":2022,"publication_date":"2022-06-01","ids":{"openalex":"https://openalex.org/W4283396263","doi":"https://doi.org/10.1097/01.hs9.0000849036.11558.e5"},"language":"en","primary_location":{"id":"doi:10.1097/01.hs9.0000849036.11558.e5","doi":"https://doi.org/10.1097/01.hs9.0000849036.11558.e5","is_oa":true,"landing_page_url":"https://doi.org/10.1097/01.hs9.0000849036.11558.e5","pdf_url":null,"source":{"id":"https://openalex.org/S4210211592","display_name":"HemaSphere","issn_l":"2572-9241","issn":["2572-9241"],"is_oa":true,"is_in_doaj":true,"is_core":true,"listed_in":["cwts-core","doaj","dongbi-b","doyens","jufo-1","ki-jl-2","norway-1","oarl","poland-20","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310318547","host_organization_name":"Wolters Kluwer","host_organization_lineage":["https://openalex.org/P4310318547"],"host_organization_lineage_names":["Wolters Kluwer"],"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":"HemaSphere","raw_type":"journal-article"},"volume":null,"type":"article","indexed_in":["crossref","doaj"],"open_access":{"is_oa":true,"oa_status":"gold","oa_url":"https://doi.org/10.1097/01.hs9.0000849036.11558.e5","any_repository_has_fulltext":true},"authorships":[{"author_position":"first","author":{"id":"https://openalex.org/A5027937488","display_name":"Rachael F. Grace","orcid":"https://orcid.org/0000-0001-7302-0449","observed_orcids":["https://orcid.org/0000-0001-7302-0449"]},"institutions":[{"id":"https://openalex.org/I4210166317","display_name":"Dana-Farber/Boston Children's Cancer and Blood Disorders Center","ror":"https://ror.org/05k11pb55","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I1288882113","https://openalex.org/I4210117453","https://openalex.org/I4210166317"]}],"countries":["US"],"is_corresponding":true,"raw_author_name":"R. F. Grace","raw_affiliation_strings":["Dana-Farber/Boston Children\u2019s Cancer and Blood Disorders Center, Boston, MA, United States of America"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Dana-Farber/Boston Children\u2019s Cancer and Blood Disorders Center, Boston, MA, United States of America","institution_ids":["https://openalex.org/I4210166317"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5018221952","display_name":"A. Glenth\u00f8j","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I2802567020","display_name":"Copenhagen University Hospital","ror":"https://ror.org/05bpbnx46","country_code":"DK","type":"healthcare","lineage":["https://openalex.org/I2802567020"]},{"id":"https://openalex.org/I2802709434","display_name":"Rigshospitalet","ror":"https://ror.org/03mchdq19","country_code":"DK","type":"healthcare","lineage":["https://openalex.org/I2802567020","https://openalex.org/I2802709434","https://openalex.org/I4210145385"]}],"countries":["DK"],"is_corresponding":false,"raw_author_name":"A. Glenth\u00f8j","raw_affiliation_strings":["Department of Haematology, Copenhagen University Hospital, Rigshospitalet, Copenhagen, Denmark"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Haematology, Copenhagen University Hospital, Rigshospitalet, Copenhagen, Denmark","institution_ids":["https://openalex.org/I2802567020","https://openalex.org/I2802709434"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5034535532","display_name":"Wilma Barcellini","orcid":"https://orcid.org/0000-0003-1428-9944","observed_orcids":["https://orcid.org/0000-0003-1428-9944"]},"institutions":[{"id":"https://openalex.org/I2803066834","display_name":"Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico","ror":"https://ror.org/016zn0y21","country_code":"IT","type":"healthcare","lineage":["https://openalex.org/I189158943","https://openalex.org/I2803066834","https://openalex.org/I4210153126"]}],"countries":["IT"],"is_corresponding":false,"raw_author_name":"W. Barcellini","raw_affiliation_strings":["Fondazione IRCCS Ca\u2019 Granda Ospedale Maggiore Policlinico, Milan, Italy"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Fondazione IRCCS Ca\u2019 Granda Ospedale Maggiore Policlinico, Milan, Italy","institution_ids":["https://openalex.org/I2803066834"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5012381772","display_name":"Madeleine M. Verhovsek","orcid":"https://orcid.org/0000-0002-5022-9765","observed_orcids":["https://orcid.org/0000-0002-5022-9765"]},"institutions":[{"id":"https://openalex.org/I98251732","display_name":"McMaster University","ror":"https://ror.org/02fa3aq29","country_code":"CA","type":"education","lineage":["https://openalex.org/I98251732"]}],"countries":["CA"],"is_corresponding":false,"raw_author_name":"M. Verhovsek","raw_affiliation_strings":["McMaster University, Hamilton, ON, Canada"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"McMaster University, Hamilton, ON, Canada","institution_ids":["https://openalex.org/I98251732"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5088721255","display_name":"J. A. Rothman","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":false,"raw_author_name":"J. A. Rothman","raw_affiliation_strings":["Duke University Medical Center, Durham, NC, United States of America"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Duke University Medical Center, Durham, NC, United States of America","institution_ids":["https://openalex.org/I4210126298"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5108449829","display_name":"M. Morado Arias","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I2800562746","display_name":"Hospital Universitario La Paz","ror":"https://ror.org/01s1q0w69","country_code":"ES","type":"healthcare","lineage":["https://openalex.org/I2800562746"]}],"countries":["ES"],"is_corresponding":false,"raw_author_name":"M. Morado Arias","raw_affiliation_strings":["Hematology Department, Hospital Universitario La Paz, Madrid, Spain"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Hematology Department, Hospital Universitario La Paz, Madrid, Spain","institution_ids":["https://openalex.org/I2800562746"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5110532280","display_name":"D. Mark Layton","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I153355300","display_name":"Imperial College Healthcare NHS Trust","ror":"https://ror.org/056ffv270","country_code":"GB","type":"healthcare","lineage":["https://openalex.org/I153355300"]},{"id":"https://openalex.org/I2801748203","display_name":"Hammersmith Hospital","ror":"https://ror.org/05jg8yp15","country_code":"GB","type":"healthcare","lineage":["https://openalex.org/I153355300","https://openalex.org/I2801748203"]}],"countries":["GB"],"is_corresponding":false,"raw_author_name":"D. M. Layton","raw_affiliation_strings":["Hammersmith Hospital, Imperial College Healthcare NHS Trust, London, United Kingdom"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Hammersmith Hospital, Imperial College Healthcare NHS Trust, London, United Kingdom","institution_ids":["https://openalex.org/I153355300","https://openalex.org/I2801748203"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5023145973","display_name":"O. Andres","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I25974101","display_name":"University of W\u00fcrzburg","ror":"https://ror.org/00fbnyb24","country_code":"DE","type":"education","lineage":["https://openalex.org/I25974101"]}],"countries":["DE"],"is_corresponding":false,"raw_author_name":"O. Andres","raw_affiliation_strings":["Department of Paediatrics, University of W\u00fcrzburg, W\u00fcrzburg, Germany"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Paediatrics, University of W\u00fcrzburg, W\u00fcrzburg, Germany","institution_ids":["https://openalex.org/I25974101"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5016689216","display_name":"F. Galact\u00e9ros","orcid":"https://orcid.org/0000-0003-2029-8126","observed_orcids":["https://orcid.org/0000-0003-2029-8126","https://orcid.org/0000-0001-8130-1208"]},"institutions":[{"id":"https://openalex.org/I3019246885","display_name":"Centre Hospitalier Universitaire Henri-Mondor","ror":"https://ror.org/04m61mj84","country_code":"FR","type":"healthcare","lineage":["https://openalex.org/I3019246885"]},{"id":"https://openalex.org/I4210130325","display_name":"H\u00f4pitaux Universitaires Henri-Mondor","ror":"https://ror.org/033yb0967","country_code":"FR","type":"healthcare","lineage":["https://openalex.org/I4210097159","https://openalex.org/I4210130325"]}],"countries":["FR"],"is_corresponding":false,"raw_author_name":"F. Galact\u00e9ros","raw_affiliation_strings":["Unit\u00e9 des Maladies G\u00e9n\u00e9tiques du Globule Rouge, CHU Henri Mondor, Creteil, France"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Unit\u00e9 des Maladies G\u00e9n\u00e9tiques du Globule Rouge, CHU Henri Mondor, Creteil, France","institution_ids":["https://openalex.org/I3019246885","https://openalex.org/I4210130325"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5007020077","display_name":"Eduard Johannes van Beers","orcid":"https://orcid.org/0000-0002-3934-7189","observed_orcids":["https://orcid.org/0000-0002-3934-7189"]},"institutions":[{"id":"https://openalex.org/I3018483916","display_name":"University Medical Center Utrecht","ror":"https://ror.org/0575yy874","country_code":"NL","type":"healthcare","lineage":["https://openalex.org/I3018483916"]}],"countries":["NL"],"is_corresponding":false,"raw_author_name":"E. J. van Beers","raw_affiliation_strings":["Van Creveldkliniek, Department of Internal Medicine, University Medical Center Utrecht, Utrecht, Netherlands"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Van Creveldkliniek, Department of Internal Medicine, University Medical Center Utrecht, Utrecht, Netherlands","institution_ids":["https://openalex.org/I3018483916"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5085403315","display_name":"K. Onodera","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210093896","display_name":"Tohoku University Hospital","ror":"https://ror.org/00kcd6x60","country_code":"JP","type":"healthcare","lineage":["https://openalex.org/I4210093896"]}],"countries":["JP"],"is_corresponding":false,"raw_author_name":"K. Onodera","raw_affiliation_strings":["Tohoku University Hospital, Sendai, Japan"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Tohoku University Hospital, Sendai, Japan","institution_ids":["https://openalex.org/I4210093896"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5080150203","display_name":"Vip Viprakasit","orcid":"https://orcid.org/0000-0003-3162-1849","observed_orcids":["https://orcid.org/0000-0003-3162-1849"]},"institutions":[{"id":"https://openalex.org/I1332387789","display_name":"Siriraj Hospital","ror":"https://ror.org/0331zs648","country_code":"TH","type":"healthcare","lineage":["https://openalex.org/I1332387789"]},{"id":"https://openalex.org/I25399158","display_name":"Mahidol University","ror":"https://ror.org/01znkr924","country_code":"TH","type":"education","lineage":["https://openalex.org/I25399158"]}],"countries":["TH"],"is_corresponding":false,"raw_author_name":"V. Viprakasit","raw_affiliation_strings":["Siriraj Hospital, Mahidol University, Bangkok, Thailand"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Siriraj Hospital, Mahidol University, Bangkok, Thailand","institution_ids":["https://openalex.org/I1332387789","https://openalex.org/I25399158"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5063350418","display_name":"Satheesh Chonat","orcid":"https://orcid.org/0000-0002-5909-0800","observed_orcids":["https://orcid.org/0000-0002-5909-0800"]},"institutions":[{"id":"https://openalex.org/I150468666","display_name":"Emory University","ror":"https://ror.org/03czfpz43","country_code":"US","type":"education","lineage":["https://openalex.org/I150468666"]},{"id":"https://openalex.org/I4210146983","display_name":"Children's Healthcare of Atlanta","ror":"https://ror.org/050fhx250","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210146983"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"S. Chonat","raw_affiliation_strings":["Aflac Cancer and Blood Disorders Center, Children\u2019s Healthcare of Atlanta and Department of Pediatrics, Emory University, Atlanta, GA, United States of America"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Aflac Cancer and Blood Disorders Center, Children\u2019s Healthcare of Atlanta and Department of Pediatrics, Emory University, Atlanta, GA, United States of America","institution_ids":["https://openalex.org/I150468666","https://openalex.org/I4210146983"]}]},{"author_position":"middle","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/I1289784979","display_name":"University College Hospital","ror":"https://ror.org/00wrevg56","country_code":"GB","type":"healthcare","lineage":["https://openalex.org/I1289784979","https://openalex.org/I1340918713"]}],"countries":["GB"],"is_corresponding":false,"raw_author_name":"J. B. Porter","raw_affiliation_strings":["Haematology Department, University College London Hospitals, London, United Kingdom"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Haematology Department, University College London Hospitals, London, United Kingdom","institution_ids":["https://openalex.org/I1289784979"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5112842486","display_name":"M. P. Judge","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210092565","display_name":"Agios Pharmaceuticals (United States)","ror":"https://ror.org/002x06r10","country_code":"US","type":"company","lineage":["https://openalex.org/I4210092565"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"M. P. Judge","raw_affiliation_strings":["Agios Pharmaceuticals, Inc., Cambridge, MA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Agios Pharmaceuticals, Inc., Cambridge, MA","institution_ids":["https://openalex.org/I4210092565"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5053330274","display_name":"Penelope A. Kosinski","orcid":"https://orcid.org/0000-0002-4465-0924","observed_orcids":["https://orcid.org/0000-0002-4465-0924"]},"institutions":[{"id":"https://openalex.org/I4210092565","display_name":"Agios Pharmaceuticals (United States)","ror":"https://ror.org/002x06r10","country_code":"US","type":"company","lineage":["https://openalex.org/I4210092565"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"P. A. Kosinski","raw_affiliation_strings":["Agios Pharmaceuticals, Inc., Cambridge, MA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Agios Pharmaceuticals, Inc., Cambridge, MA","institution_ids":["https://openalex.org/I4210092565"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5058505998","display_name":"P. Hawkins","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210092565","display_name":"Agios Pharmaceuticals (United States)","ror":"https://ror.org/002x06r10","country_code":"US","type":"company","lineage":["https://openalex.org/I4210092565"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"P. Hawkins","raw_affiliation_strings":["Agios Pharmaceuticals, Inc., Cambridge, MA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Agios Pharmaceuticals, Inc., Cambridge, MA","institution_ids":["https://openalex.org/I4210092565"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5066921649","display_name":"Sarah Gheuens","orcid":"https://orcid.org/0000-0001-8695-4240","observed_orcids":["https://orcid.org/0000-0001-8695-4240"]},"institutions":[{"id":"https://openalex.org/I4210092565","display_name":"Agios Pharmaceuticals (United States)","ror":"https://ror.org/002x06r10","country_code":"US","type":"company","lineage":["https://openalex.org/I4210092565"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"S. Gheuens","raw_affiliation_strings":["Agios Pharmaceuticals, Inc., Cambridge, MA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Agios Pharmaceuticals, Inc., Cambridge, MA","institution_ids":["https://openalex.org/I4210092565"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5039900033","display_name":"R. Xu","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210092565","display_name":"Agios Pharmaceuticals (United States)","ror":"https://ror.org/002x06r10","country_code":"US","type":"company","lineage":["https://openalex.org/I4210092565"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"R. Xu","raw_affiliation_strings":["Agios Pharmaceuticals, Inc., Cambridge, MA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Agios Pharmaceuticals, Inc., Cambridge, MA","institution_ids":["https://openalex.org/I4210092565"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5033574166","display_name":"B. McGee","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210092565","display_name":"Agios Pharmaceuticals (United States)","ror":"https://ror.org/002x06r10","country_code":"US","type":"company","lineage":["https://openalex.org/I4210092565"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"B. McGee","raw_affiliation_strings":["Agios Pharmaceuticals, Inc., Cambridge, MA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Agios Pharmaceuticals, Inc., Cambridge, MA","institution_ids":["https://openalex.org/I4210092565"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5029504343","display_name":"Vanessa Beynon","orcid":"https://orcid.org/0000-0002-9719-1223","observed_orcids":["https://orcid.org/0000-0002-9719-1223"]},"institutions":[{"id":"https://openalex.org/I4210092565","display_name":"Agios Pharmaceuticals (United States)","ror":"https://ror.org/002x06r10","country_code":"US","type":"company","lineage":["https://openalex.org/I4210092565"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"V. Beynon","raw_affiliation_strings":["Agios Pharmaceuticals, Inc., Cambridge, MA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Agios Pharmaceuticals, Inc., Cambridge, MA","institution_ids":["https://openalex.org/I4210092565"]}]},{"author_position":"last","author":{"id":"https://openalex.org/A5066258828","display_name":"Hanny T. Al-Samkari","orcid":"https://orcid.org/0000-0001-6175-1383","observed_orcids":["https://orcid.org/0000-0001-6175-1383"]},"institutions":[{"id":"https://openalex.org/I136199984","display_name":"Harvard University","ror":"https://ror.org/03vek6s52","country_code":"US","type":"education","lineage":["https://openalex.org/I136199984"]},{"id":"https://openalex.org/I4210087915","display_name":"Massachusetts General Hospital","ror":"https://ror.org/002pd6e78","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210087915","https://openalex.org/I48633490"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"H. Al-Samkari","raw_affiliation_strings":["Division of Hematology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, United States of America"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Division of Hematology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, United States of America","institution_ids":["https://openalex.org/I136199984","https://openalex.org/I4210087915"]}]}],"institutions":[],"countries_distinct_count":11,"institutions_distinct_count":22,"corresponding_author_ids":["https://openalex.org/A5027937488"],"corresponding_institution_ids":["https://openalex.org/I4210166317"],"apc_list":{"value":2480,"currency":"USD","value_usd":2480},"apc_paid":{"value":2480,"currency":"USD","value_usd":2480},"fwci":0.1553,"has_fulltext":false,"cited_by_count":1,"citation_normalized_percentile":{"value":0.42004742,"is_in_top_1_percent":false,"is_in_top_10_percent":false},"cited_by_percentile_year":{"min":88,"max":93},"biblio":{"volume":"6","issue":null,"first_page":"1426","last_page":"1427"},"is_retracted":false,"is_paratext":false,"is_xpac":false,"primary_topic":{"id":"https://openalex.org/T10554","display_name":"Hemoglobinopathies and Related Disorders","score":0.9667999744415283,"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":0.9667999744415283,"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/T12286","display_name":"Erythrocyte Function and Pathophysiology","score":0.00800000037997961,"subfield":{"id":"https://openalex.org/subfields/2737","display_name":"Physiology (medical)"},"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/T11534","display_name":"Erythropoietin and Anemia Treatment","score":0.00559999980032444,"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/pyruvate-kinase-deficiency","display_name":"pyruvate kinase deficiency","score":0.9010000228881836},{"id":"https://openalex.org/keywords/mitapivat","display_name":"mitapivat","score":0.9980000257492065},{"id":"https://openalex.org/keywords/allosteric-activators","display_name":"allosteric activators","score":0.6100000143051147},{"id":"https://openalex.org/keywords/hemoglobin-response","display_name":"hemoglobin response","score":0.7480000257492065},{"id":"https://openalex.org/keywords/transfusion-burden","display_name":"transfusion burden","score":0.9240000247955322},{"id":"https://openalex.org/keywords/long-term-extension-study","display_name":"long-term extension study","score":0.621999979019165},{"id":"https://openalex.org/keywords/hemolysis","display_name":"hemolysis","score":0.6420000195503235},{"id":"https://openalex.org/keywords/red-blood-cell-indices","display_name":"red blood cell indices","score":0.49300000071525574},{"id":"https://openalex.org/keywords/dose-optimization","display_name":"dose optimization","score":0.31200000643730164},{"id":"https://openalex.org/keywords/oral-therapy","display_name":"oral therapy","score":0.3109999895095825},{"id":"https://openalex.org/keywords/sickle-cell-disease","display_name":"sickle cell disease","score":0.5849999785423279},{"id":"https://openalex.org/keywords/thalassemia","display_name":"thalassemia","score":0.6949999928474426},{"id":"https://openalex.org/keywords/chronic-anemia","display_name":"chronic anemia","score":0.3700000047683716},{"id":"https://openalex.org/keywords/hemoglobinopathies","display_name":"hemoglobinopathies","score":0.3569999933242798}],"concepts":[{"id":"https://openalex.org/C71924100","wikidata":"https://www.wikidata.org/wiki/Q11190","display_name":"Medicine","level":0,"score":0.8368926644325256},{"id":"https://openalex.org/C203092338","wikidata":"https://www.wikidata.org/wiki/Q1340863","display_name":"Clinical endpoint","level":3,"score":0.7055978178977966},{"id":"https://openalex.org/C2778917026","wikidata":"https://www.wikidata.org/wiki/Q43041","display_name":"Hemoglobin","level":2,"score":0.5880221128463745},{"id":"https://openalex.org/C2779902561","wikidata":"https://www.wikidata.org/wiki/Q471706","display_name":"Hemolysis","level":2,"score":0.53593909740448},{"id":"https://openalex.org/C2780644154","wikidata":"https://www.wikidata.org/wiki/Q3043149","display_name":"Pyruvate kinase deficiency","level":5,"score":0.5142757296562195},{"id":"https://openalex.org/C27081682","wikidata":"https://www.wikidata.org/wiki/Q269829","display_name":"Placebo","level":3,"score":0.5039903521537781},{"id":"https://openalex.org/C2778248108","wikidata":"https://www.wikidata.org/wiki/Q5445","display_name":"Anemia","level":2,"score":0.46908679604530334},{"id":"https://openalex.org/C2780014101","wikidata":"https://www.wikidata.org/wiki/Q183605","display_name":"Blood transfusion","level":2,"score":0.4317615330219269},{"id":"https://openalex.org/C126322002","wikidata":"https://www.wikidata.org/wiki/Q11180","display_name":"Internal medicine","level":1,"score":0.4163699448108673},{"id":"https://openalex.org/C141071460","wikidata":"https://www.wikidata.org/wiki/Q40821","display_name":"Surgery","level":1,"score":0.35032913088798523},{"id":"https://openalex.org/C168563851","wikidata":"https://www.wikidata.org/wiki/Q1436668","display_name":"Randomized controlled trial","level":2,"score":0.33497172594070435},{"id":"https://openalex.org/C90924648","wikidata":"https://www.wikidata.org/wiki/Q120569","display_name":"Gastroenterology","level":1,"score":0.3337882161140442},{"id":"https://openalex.org/C2780854706","wikidata":"https://www.wikidata.org/wiki/Q900970","display_name":"Pyruvate kinase","level":4,"score":0.32280591130256653},{"id":"https://openalex.org/C62231903","wikidata":"https://www.wikidata.org/wiki/Q1057","display_name":"Metabolism","level":2,"score":0.07796284556388855},{"id":"https://openalex.org/C142724271","wikidata":"https://www.wikidata.org/wiki/Q7208","display_name":"Pathology","level":1,"score":0.0},{"id":"https://openalex.org/C204787440","wikidata":"https://www.wikidata.org/wiki/Q188504","display_name":"Alternative medicine","level":2,"score":0.0},{"id":"https://openalex.org/C20251656","wikidata":"https://www.wikidata.org/wiki/Q162643","display_name":"Glycolysis","level":3,"score":0.0}],"mesh":[],"locations_count":3,"locations":[{"id":"doi:10.1097/01.hs9.0000849036.11558.e5","doi":"https://doi.org/10.1097/01.hs9.0000849036.11558.e5","is_oa":true,"landing_page_url":"https://doi.org/10.1097/01.hs9.0000849036.11558.e5","pdf_url":null,"source":{"id":"https://openalex.org/S4210211592","display_name":"HemaSphere","issn_l":"2572-9241","issn":["2572-9241"],"is_oa":true,"is_in_doaj":true,"is_core":true,"listed_in":["cwts-core","doaj","dongbi-b","doyens","jufo-1","ki-jl-2","norway-1","oarl","poland-20","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310318547","host_organization_name":"Wolters Kluwer","host_organization_lineage":["https://openalex.org/P4310318547"],"host_organization_lineage_names":["Wolters Kluwer"],"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":"HemaSphere","raw_type":"journal-article"},{"id":"pmh:oai:pubmedcentral.nih.gov:9431292","doi":null,"is_oa":true,"landing_page_url":"https://www.ncbi.nlm.nih.gov/pmc/articles/9431292","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":"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":"Hemasphere","raw_type":"Text"},{"id":"pmh:oai:doaj.org/article:d102fc02d19a4525b0d83f1ccf2a8af3","doi":null,"is_oa":true,"landing_page_url":"https://doaj.org/article/d102fc02d19a4525b0d83f1ccf2a8af3","pdf_url":null,"source":{"id":"https://openalex.org/S4306401280","display_name":"DOAJ (DOAJ: Directory of Open Access Journals)","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":"cc-by-sa","license_id":"https://openalex.org/licenses/cc-by-sa","version":"submittedVersion","is_accepted":false,"is_published":false,"raw_source_name":"HemaSphere, Vol 6, Pp 1426-1427 (2022)","raw_type":"article"}],"best_oa_location":{"id":"doi:10.1097/01.hs9.0000849036.11558.e5","doi":"https://doi.org/10.1097/01.hs9.0000849036.11558.e5","is_oa":true,"landing_page_url":"https://doi.org/10.1097/01.hs9.0000849036.11558.e5","pdf_url":null,"source":{"id":"https://openalex.org/S4210211592","display_name":"HemaSphere","issn_l":"2572-9241","issn":["2572-9241"],"is_oa":true,"is_in_doaj":true,"is_core":true,"listed_in":["cwts-core","doaj","dongbi-b","doyens","jufo-1","ki-jl-2","norway-1","oarl","poland-20","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310318547","host_organization_name":"Wolters Kluwer","host_organization_lineage":["https://openalex.org/P4310318547"],"host_organization_lineage_names":["Wolters Kluwer"],"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":"HemaSphere","raw_type":"journal-article"},"sustainable_development_goals":[{"score":1.0,"display_name":"Good health and well-being","id":"https://metadata.un.org/sdg/3"}],"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/W3211546461","https://openalex.org/W4310163499","https://openalex.org/W4283380923","https://openalex.org/W4283386868","https://openalex.org/W4296932772","https://openalex.org/W2984580366","https://openalex.org/W4389231112","https://openalex.org/W4385856750","https://openalex.org/W2986770844","https://openalex.org/W4310163451"],"abstract_inverted_index":{"Background:":[0],"Mitapivat":[1,516],"(AG-348),":[2],"a":[3,242,305,406,449,468],"first-in-class,":[4],"oral,":[5],"allosteric":[6],"activator":[7],"of":[8,22,177,210,229,298,396,417,446,454,536],"red":[9],"blood":[10],"cell":[11],"(RBC)":[12],"PK":[13,39,81,152,526],"enzyme,":[14],"demonstrated":[15],"improvements":[16,319,328],"in":[17,30,35,91,95,115,130,161,175,203,217,232,262,280,308,315,320,342,360,402,419,432,478,483,505,512,523],"hemoglobin":[18],"(Hb)":[19],"and":[20,24,28,47,128,245,268,283,435,452,519,533],"markers":[21],"hemolysis":[23],"hematopoiesis":[25],"(ACTIVATE,":[26],"NCT03548220),":[27],"reduction":[29,174],"transfusion":[31,89,159,171,189,278,450,469,503,521],"burden":[32,190,522],"(ACTIVATE-T,":[33],"NCT03559699)":[34],"patients":[36],"(pts)":[37],"with":[38,80,151,166,185,301,331,525,538],"deficiency.":[40],"Aims:":[41],"Report":[42],"data":[43],"from":[44,117,288,372,389],"ACTIVATE,":[45,296],"ACTIVATE-T,":[46,506],"their":[48],"long-term":[49,534],"extension":[50],"(LTE)":[51],"study":[52,61,138],"(NCT03853798).":[53],"Methods:":[54],"In":[55,133,295,442,459],"the":[56,131,134,182,208,218,248,263,281,309,350,361,397,420,433,460,481,484,497,513,531],"double-blind,":[57],"placebo":[58],"(PBO)-controlled":[59],"ACTIVATE":[60,316,343,403],"(12-week":[62],"[wk]":[63],"dose-optimization":[64,140],"period":[65,73,141],"[5/20/50":[66,142],"mg":[67,143],"twice":[68],"daily":[69],"[BID]];":[70],"12-wk":[71],"fixed-dose":[72],"[FDP]),":[74],"80":[75],"pts":[76,148,236,254,287,299,311,338,354,365,383,398,411,427,447,455,463,473,524],"(\u226518":[77,149],"years":[78],"[yrs])":[79],"deficiency":[82,153,527],"who":[83,154,206,240,258,290,344,474,495],"were":[84,100,155,164,213,329,355],"not":[85,502,508],"regularly":[86,156],"transfused":[87,157,180],"(\u22644":[88],"episodes":[90,160],"prior":[92,96,162],"yr;":[93],"none":[94],"3":[97],"months":[98],"[mos])":[99],"randomized":[101,312],"1:1":[102],"to":[103,192,215,238,247,256,260,313,325,340,349,375,400,487],"mitapivat":[104,239,261,302,341],"or":[105],"PBO.":[106],"Primary":[107,168],"endpoint":[108,169,196],"was":[109,170,197,501],"Hb":[110,116,230,243,271,306,321,346,358,370,387,407,423,430,438,518],"response":[111,172,231,244,272,279,424,451],"(\u22651.5":[112],"g/dL":[113,369,386],"increase":[114,371,388],"baseline":[118],"[BL]":[119],"sustained":[120,330],"at":[121,124,378,391],"\u22652":[122],"assessments":[123],"Wks":[125],"16,":[126],"20,":[127],"24":[129,193],"FDP).":[132,204],"single-arm,":[135],"open-label":[136],"ACTIVATE-T":[137,212,289,443,479],"(16-wk":[139],"BID];":[144],"24-wk":[145],"FDP),":[146],"27":[147],"yrs)":[150],"(\u22656":[158],"yr)":[163],"treated":[165,300],"mitapivat.":[167,223,326,539],"(\u226533%":[173],"number":[176],"RBC":[178],"units":[179],"during":[181,440],"FDP,":[183],"compared":[184],"pt\u2019s":[186],"individual":[187],"historical":[188],"standardized":[191],"wks).":[194],"Secondary":[195],"achieving":[198],"transfusion-free":[199,284,292,457,476],"status":[200,477,482],"(no":[201],"transfusions":[202,511],"Pts":[205],"completed":[207],"FDP":[209],"ACTIVATE/":[211],"eligible":[214],"continue":[216],"LTE,":[219,282,310,434,461],"where":[220],"all":[221,379,392,436],"received":[222],"The":[224,274],"ACTIVATE/LTE":[225],"analysis":[226,276],"assessed":[227,277],"duration":[228,285],"2":[233,382],"cohorts:":[234],"1)":[235],"assigned":[237,255,339,399],"achieved":[241,291,304,345,429,448,456,475],"continued":[246,332,348],"LTE":[249,264,421,485],"(mitapivat-to-mitapivat":[250],"arm":[251,266],"[M/M]);":[252],"2)":[253],"PBO":[257,314,401],"switched":[259],"(PBO-to-mitapivat":[265],"[P/M])":[267],"then":[269],"met":[270,465,496],"criteria.":[273],"ACTIVATE-T/LTE":[275],"among":[286],"status.":[293,458],"Results:":[294],"40%":[297],"(N=40)":[303,404],"response;":[307,408],"showed":[317],"similar":[318],"levels":[322],"after":[323],"switching":[324],"These":[327],"treatment":[333,537],"(Figure":[334,490],"1a).":[335],"All":[336,471],"16":[337],"responses":[347,431,439],"LTE;":[351],"15":[352],"M/M":[353,364],"evaluable":[356],"for":[357,422,467],"assessment":[359],"LTE.":[362,514],"13/15":[363],"(86.7%)":[366],"maintained":[367,384,437,480],"\u22651.5":[368],"BL":[373,390],"up":[374,486],"19.5":[376],"mos":[377,489],"time":[380,393,414],"points;":[381],"\u22651":[385],"points.":[394],"None":[395],"had":[405,412],"17":[409],"P/M":[410],"sufficient":[413],"(24":[415],"wks":[416],"treatment)":[418],"assessment.":[425],"6/17":[426],"(35%)":[428],"follow-up.":[441],"(N=27),":[444],"37%":[445],"22%":[453],"9":[462],"(33.3%)":[464],"criteria":[466],"response.":[470],"6":[472],"21.9":[488],"1b).":[491],"One":[492],"additional":[493],"pt,":[494],"primary":[498],"endpoint,":[499],"but":[500],"free":[504],"did":[507],"receive":[509],"any":[510],"Image:Summary/Conclusion:":[515],"improved":[517],"reduced":[520],"over":[528],"time,":[529],"demonstrating":[530],"consistency":[532],"durability":[535]},"counts_by_year":[{"year":2023,"cited_by_count":1}],"updated_date":"2026-11-04T10:54:05.112534","created_date":"2022-06-25T00:00:00"}