{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,6,2]],"date-time":"2026-06-02T08:10:54Z","timestamp":1780387854043,"version":"3.54.1"},"reference-count":30,"publisher":"Wiley","issue":"7","license":[{"start":{"date-parts":[[2026,4,6]],"date-time":"2026-04-06T00:00:00Z","timestamp":1775433600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"},{"start":{"date-parts":[[2026,4,6]],"date-time":"2026-04-06T00:00:00Z","timestamp":1775433600000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/doi.wiley.com\/10.1002\/tdm_license_1.1"}],"funder":[{"DOI":"10.13039\/100018748","name":"Argenx","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100018748","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["American J Hematol"],"published-print":{"date-parts":[[2026,7]]},"abstract":"<jats:title>ABSTRACT<\/jats:title>\n                  <jats:p>\n                    Primary autoimmune thrombocytopenia (ITP) is characterized by thrombocytopenia, bleeding, and reduced health\u2010related quality of life. In the Phase 3 ADVANCE IV study, intravenous efgartigimod induced significant platelet count responses versus placebo in patients with chronic ITP. ADVANCE SC, a Phase 3, multicenter, randomized, double\u2010blinded, placebo\u2010controlled, parallel\u2010group study, evaluated the efficacy and safety of subcutaneous efgartigimod PH20 in adults with primary ITP. Participants with platelet counts &lt;\u200930\u2009\u00d7\u200910\n                    <jats:sup>9<\/jats:sup>\n                    \/L who received more than one prior ITP therapy. Between December 16, 2020, and October 9, 2023, 207 participants were randomized (2:1) to receive 1000\u2009mg efgartigimod PH20 SC (\n                    <jats:italic>n<\/jats:italic>\n                    \u2009=\u2009137) or placebo (\n                    <jats:italic>n<\/jats:italic>\n                    \u2009=\u200970) once weekly (visits 1\u20134), weekly or biweekly (depending upon response; visits 5\u201316), and at a fixed dosing interval (visits 17\u201324). Most (92.8% [192\/207]) had chronic ITP and 74.9% (155\/207) received at least three previous ITP treatments. The median time since diagnosis was 7.0\u2009years. Superior efficacy of efgartigimod PH20 SC versus placebo was not demonstrated for the primary or secondary endpoints, with a comparable proportion of efgartigimod PH20 SC and placebo\u2010treated participants achieving the primary efficacy endpoint (platelet count \u2265\u200950\u2009\u00d7\u200910\n                    <jats:sup>9<\/jats:sup>\n                    \/L for at least four of the six visits during weeks 19\u201324): \u221213.7% [17\/124] versus 16.2% [11\/68], respectively;\n                    <jats:italic>p<\/jats:italic>\n                    \u2009=\u20090.51; adjusted difference in proportions, \u22123.5% [95% CI, \u221214.7\u20137.0]. Efgartigimod PH20 SC was well tolerated: most adverse events were mild to moderate and comparable between treatment groups. Platelet count increases from baseline were higher than expected with placebo and lower than expected with efgartigimod PH20 SC.\n                  <\/jats:p>\n                  <jats:p>\n                    <jats:bold>Clinical Trial Registration<\/jats:bold>\n                    : The ADVANCE SC trial is registered on\n                    <jats:ext-link xmlns:xlink=\"http:\/\/www.w3.org\/1999\/xlink\" xlink:href=\"http:\/\/clinicaltrials.gov\">ClinicalTrials.gov<\/jats:ext-link>\n                    (NCT04687072)\n                  <\/jats:p>","DOI":"10.1002\/ajh.70303","type":"journal-article","created":{"date-parts":[[2026,4,7]],"date-time":"2026-04-07T03:46:02Z","timestamp":1775533562000},"page":"1494-1506","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Efficacy and Safety of Subcutaneous Efgartigimod\n                    <scp>PH20<\/scp>\n                    in Adults With Primary Immune Thrombocytopenia (\n                    <scp>ADVANCE SC<\/scp>\n                    ): A Multicenter, Randomized, Double\u2010Blinded, Placebo\u2010Controlled, Phase 3 Trial"],"prefix":"10.1002","volume":"101","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-9845-8292","authenticated-orcid":false,"given":"Nichola","family":"Cooper","sequence":"first","affiliation":[{"name":"Department of Immunology and Inflammation Imperial College  London UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1507-2851","authenticated-orcid":false,"given":"Catherine M.","family":"Broome","sequence":"additional","affiliation":[{"name":"Division of Hematology Oncology MedStar Georgetown University  Washington DC USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yoshitaka","family":"Miyakawa","sequence":"additional","affiliation":[{"name":"Department of Hematology Saitama Medical University Hospital  Saitama Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Vickie","family":"McDonald","sequence":"additional","affiliation":[{"name":"Barts Health NHS Trust  London UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hanny","family":"Al\u2010Samkari","sequence":"additional","affiliation":[{"name":"Massachusetts General Hospital  Boston Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Abderrahim","family":"Khelif","sequence":"additional","affiliation":[{"name":"Department of Clinical Hematology, Farhat Hached Hospital Sousse University  Sousse Tunisia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Spero R.","family":"Cataland","sequence":"additional","affiliation":[{"name":"Division of Hematology The Ohio State Univesrity Wexner Medical Center  Columbus Ohio USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Wilma","family":"Barcellini","sequence":"additional","affiliation":[{"name":"SC Ematologia, Fondazione IRCCS ca\u2019 Granda Ospedale Maggiore Policlinico  Milan Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Renchi","family":"Yang","sequence":"additional","affiliation":[{"name":"State Key Laboratory of Experimental Hematology, Institute of Hematology and Blood Diseases Hospital Chinese Academy of Medical Sciences and Peking Union Medical College  Tianjin China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7941-2443","authenticated-orcid":false,"given":"Heng","family":"Mei","sequence":"additional","affiliation":[{"name":"Institute of Hematology, Union Hospital, Tongji Medical College Huazhong University of Science and Technology  Wuhan China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Filip","family":"Matthijssens","sequence":"additional","affiliation":[{"name":"Argenx  Ghent Belgium"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anna","family":"Hultberg","sequence":"additional","affiliation":[{"name":"Argenx  Ghent Belgium"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Giorgia","family":"Ciurlia","sequence":"additional","affiliation":[{"name":"Argenx  Ghent Belgium"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Domenica","family":"Gandini","sequence":"additional","affiliation":[{"name":"Argenx  Ghent Belgium"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jaume","family":"Ayguasanosa","sequence":"additional","affiliation":[{"name":"Argenx  Ghent Belgium"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kristof","family":"De Beuf","sequence":"additional","affiliation":[{"name":"Argenx  Ghent Belgium"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"S\u00e9gol\u00e8ne","family":"Pastouret","sequence":"additional","affiliation":[{"name":"Argenx  Ghent Belgium"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Waleed","family":"Ghanima","sequence":"additional","affiliation":[{"name":"Department of Hemato\u2010Oncology, \u00d8stfold Hospital Trust, Norway and Institute of Clinical Medicine University of Oslo  Oslo Norway"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2253-2502","authenticated-orcid":false,"given":"Francesco","family":"Rodeghiero","sequence":"additional","affiliation":[{"name":"The Hematology Project Foundation Department of Cell Therapy and Hematology, San Bortolo Hospital  Vicenza Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-2884-9247","authenticated-orcid":false,"given":"James B.","family":"Bussel","sequence":"additional","affiliation":[{"name":"Weill Cornell Medical College  New York New York USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"name":"the ADVANCE SC Investigator Trial Group","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2026,4,6]]},"reference":[{"key":"e_1_2_12_2_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2141.2006.06024.x"},{"key":"e_1_2_12_3_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.hoc.2013.03.001"},{"key":"e_1_2_12_4_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2008-07-162503"},{"key":"e_1_2_12_5_1","doi-asserted-by":"publisher","DOI":"10.1186\/1477-7525-6-13"},{"key":"e_1_2_12_6_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.tmrv.2020.09.003"},{"key":"e_1_2_12_7_1","doi-asserted-by":"publisher","DOI":"10.1111\/jth.14419"},{"key":"e_1_2_12_8_1","first-page":"2417","article-title":"The Role of CD8+ T\u2010Cell Clones in Immune Thrombocytopenia","volume":"141","author":"Malik A.","year":"2023","journal-title":"Blood"},{"key":"e_1_2_12_9_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41418-021-00797-w"},{"key":"e_1_2_12_10_1","doi-asserted-by":"publisher","DOI":"10.2147\/JBM.S259101"},{"key":"e_1_2_12_11_1","doi-asserted-by":"publisher","DOI":"10.1182\/bloodadvances.2019000966"},{"key":"e_1_2_12_12_1","doi-asserted-by":"publisher","DOI":"10.1111\/bjh.19078"},{"key":"e_1_2_12_13_1","doi-asserted-by":"publisher","DOI":"10.1007\/s00018-010-0318-6"},{"key":"e_1_2_12_14_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(21)00159-9"},{"key":"e_1_2_12_15_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.neurot.2024.e00378"},{"key":"e_1_2_12_16_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(24)00309-0"},{"key":"e_1_2_12_17_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(23)01460-5"},{"key":"e_1_2_12_18_1","doi-asserted-by":"publisher","DOI":"10.1111\/bjh.19858"},{"key":"e_1_2_12_19_1","doi-asserted-by":"publisher","DOI":"10.1002\/ajh.25680"},{"key":"e_1_2_12_20_1","doi-asserted-by":"publisher","DOI":"10.1182\/bloodadvances.2020002003"},{"key":"e_1_2_12_21_1","doi-asserted-by":"publisher","DOI":"10.1038\/nbt1143"},{"key":"e_1_2_12_22_1","doi-asserted-by":"publisher","DOI":"10.1172\/JCI97911"},{"key":"e_1_2_12_23_1","doi-asserted-by":"publisher","DOI":"10.1002\/ajh.25125"},{"key":"e_1_2_12_24_1","doi-asserted-by":"publisher","DOI":"10.1111\/bjh.15573"},{"key":"e_1_2_12_25_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(08)60203-2"},{"key":"e_1_2_12_26_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood.2019003599"},{"key":"e_1_2_12_27_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2141.2009.07717.x"},{"key":"e_1_2_12_28_1","doi-asserted-by":"publisher","DOI":"10.1007\/s40259-018-0295-0"},{"key":"e_1_2_12_29_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2006-05-024539"},{"key":"e_1_2_12_30_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2024-206889"},{"key":"e_1_2_12_31_1","doi-asserted-by":"publisher","DOI":"10.1080\/1744666X.2025.2497840"}],"container-title":["American Journal of Hematology"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/ajh.70303","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/full-xml\/10.1002\/ajh.70303","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/ajh.70303","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,6,2]],"date-time":"2026-06-02T07:48:41Z","timestamp":1780386521000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1002\/ajh.70303"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,4,6]]},"references-count":30,"journal-issue":{"issue":"7","published-print":{"date-parts":[[2026,7]]}},"alternative-id":["10.1002\/ajh.70303"],"URL":"https:\/\/doi.org\/10.1002\/ajh.70303","archive":["Portico"],"relation":{},"ISSN":["0361-8609","1096-8652"],"issn-type":[{"value":"0361-8609","type":"print"},{"value":"1096-8652","type":"electronic"}],"subject":[],"published":{"date-parts":[[2026,4,6]]},"assertion":[{"value":"2026-03-09","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2026-03-18","order":2,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2026-04-06","order":3,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}