{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,30]],"date-time":"2026-10-30T21:02:08Z","timestamp":1793394128330,"version":"4.5.0"},"reference-count":19,"publisher":"Wiley","issue":"2","license":[{"start":{"date-parts":[[2024,11,18]],"date-time":"2024-11-18T00:00:00Z","timestamp":1731888000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/100015661","name":"UCB Pharma","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100015661","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Br J Haematol"],"published-print":{"date-parts":[[2025,2]]},"abstract":"<jats:title>Summary<\/jats:title><jats:p>Primary immune thrombocytopenia (ITP) is an antiplatelet\u2010antibody\u2010mediated disorder with accelerated platelet clearance and decreased platelet production. Rozanolixizumab, a monoclonal IgG4 anti\u2010FcRn antibody, blocks IgG recycling and decreases IgG levels. We report efficacy and safety of rozanolixizumab in adults with persistent\/chronic ITP in 24\u2010week phase 3 studies (TP0003; TP0006), and their 52\u2010week open\u2010label extension (OLE). Primary end\u2010point was durable clinically meaningful platelet response (DCMPR) of \u226550\u2009\u00d7\u200910<jats:sup>9<\/jats:sup>\/L for 8\/12\u2009weeks during Weeks 13\u201325 in the double\u2010blind studies. Operational delays and evolving ITP treatment landscape led the sponsor to terminate these studies early; thus, only 21 and 12 (TP0003) and 20 and 10 (TP0006) patients were randomised to rozanolixizumab or placebo. Forty\u2010three patients enrolled in the OLE: 42 started on every 2\u2010week dosing; 21 later switched to weekly dosing. More rozanolixizumab\u2010treated than placebo\u2010treated patients achieved DCMPR: 4\/21 versus 0 (TP0003) and 1\/20 versus 0 (TP0006). Platelet increases to \u226550\u2009\u00d7\u200910<jats:sup>9<\/jats:sup>\/L were observed on Day 8 in 52.4% (TP0003; 2\/12 placebo) and 45.0% (TP0006; 1\/10 placebo) of rozanolixizumab\u2010treated patients. OLE platelet increases were maintained while on weekly dosing. The most frequent treatment\u2010emergent adverse events overall were headache, pyrexia and nausea, as seen previously. Weekly dosing appears more efficacious than every 2\u2010week dosing.<\/jats:p>","DOI":"10.1111\/bjh.19858","type":"journal-article","created":{"date-parts":[[2024,11,18]],"date-time":"2024-11-18T09:49:45Z","timestamp":1731923385000},"page":"675-688","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":16,"title":["Inhibition of <scp>FcRn<\/scp> with rozanolixizumab in adults with immune thrombocytopenia: Two randomised, double\u2010blind, placebo\u2010controlled phase 3 studies and their open\u2010label extension"],"prefix":"10.1111","volume":"206","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-9845-8292","authenticated-orcid":false,"given":"Nichola","family":"Cooper","sequence":"first","affiliation":[{"name":"Imperial College London  London UK"}],"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 Medicine \u2013 New York Presbyterian Hospital  New York New York USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Maciej","family":"Ka\u017amierczak","sequence":"additional","affiliation":[{"name":"Pozna\u0144 University of Medical Sciences  Pozna\u0144 Poland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yoshitaka","family":"Miyakawa","sequence":"additional","affiliation":[{"name":"Saitama Medical University  Saitama Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sarah","family":"Cluck","sequence":"additional","affiliation":[{"name":"UCB  Slough UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Roc\u00edo","family":"Lled\u00f3 Garc\u00eda","sequence":"additional","affiliation":[{"name":"UCB  Slough UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Birgit","family":"Haier","sequence":"additional","affiliation":[{"name":"UCB  Monheim Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Andreea","family":"Lavrov","sequence":"additional","affiliation":[{"name":"UCB  Monheim Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Puneet","family":"Singh","sequence":"additional","affiliation":[{"name":"UCB  Slough UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rose","family":"Snipes","sequence":"additional","affiliation":[{"name":"UCB  Morrisville North Carolina USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2084-8810","authenticated-orcid":false,"given":"David J.","family":"Kuter","sequence":"additional","affiliation":[{"name":"Hematology Division Massachusetts General Hospital  Boston Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2024,11,18]]},"reference":[{"key":"e_1_2_12_2_1","doi-asserted-by":"publisher","DOI":"10.3324\/haematol.2014.115634"},{"key":"e_1_2_12_3_1","doi-asserted-by":"publisher","DOI":"10.1111\/ejh.12528"},{"key":"e_1_2_12_4_1","doi-asserted-by":"publisher","DOI":"10.3389\/fimmu.2018.00880"},{"key":"e_1_2_12_5_1","doi-asserted-by":"publisher","DOI":"10.3390\/jcm6020016"},{"key":"e_1_2_12_6_1","doi-asserted-by":"publisher","DOI":"10.3390\/jcm10040789"},{"key":"e_1_2_12_7_1","doi-asserted-by":"publisher","DOI":"10.1007\/s12325-015-0251-z"},{"issue":"7","key":"e_1_2_12_8_1","first-page":"1111","article-title":"Generation and characterization of a high affinity anti\u2010human FcRn antibody, rozanolixizumab, and the effects of different molecular formats on the reduction of plasma IgG concentration","volume":"10","author":"Smith B","year":"2018","journal-title":"MAbs"},{"key":"e_1_2_12_9_1","doi-asserted-by":"publisher","DOI":"10.1126\/scitranslmed.aan1208"},{"key":"e_1_2_12_10_1","doi-asserted-by":"publisher","DOI":"10.1182\/bloodadvances.2020002003"},{"key":"e_1_2_12_11_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(23)00077-7"},{"key":"e_1_2_12_12_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2008-07-162503"},{"key":"e_1_2_12_13_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(08)60203-2"},{"key":"e_1_2_12_14_1","doi-asserted-by":"publisher","DOI":"10.1002\/psp4.12739"},{"key":"e_1_2_12_15_1","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000011108"},{"key":"e_1_2_12_16_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(21)00159-9"},{"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","unstructured":"argenx reports topline results from ADVANCE\u2010SC study of VYVGART Hytrulo in primary immune thrombocytopenia: argenx [Internet]. 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