{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,30]],"date-time":"2026-10-30T21:02:41Z","timestamp":1793394161554,"version":"4.5.0"},"reference-count":38,"publisher":"American Society of Hematology","issue":"17","content-domain":{"domain":["ashpublications.org"],"crossmark-restriction":true},"short-container-title":[],"published-print":{"date-parts":[[2020,9,8]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:p>Primary immune thrombocytopenia (ITP) is a predominantly immunoglobulin G (IgG)-autoantibody-mediated disease characterized by isolated thrombocytopenia. Rozanolixizumab, a subcutaneously infused humanized monoclonal anti-neonatal Fc receptor (FcRn) antibody, reduced serum IgG in healthy volunteers. In this phase 2, multicenter, open-label study, patients with persistent\/chronic primary ITP received 1 to 5 once-weekly subcutaneous infusions of rozanolixizumab (cumulative doses, 15-21 mg\/kg). Primary objectives were safety and tolerability, and secondary objectives were clinical efficacy (change in platelet count) and pharmacodynamic effect (change in IgG). In all, 51 (77.3%) of 66 patients reported 1 or more adverse events (AEs), all mild-to-moderate, most commonly headaches (26 [39.4%] of 66), of which 15 were treatment related. Four patients had serious AEs, but none were treatment related. No AEs resulted in discontinuation of the study drug. No serious infections occurred. Platelet counts of \u226550 \u00d7 109\/L were achieved at least once at any time after multiple infusions (5 \u00d7 4, 3 \u00d7 7, or 2 \u00d7 10 mg\/kg: 35.7%, 35.7%, and 45.5% of patients, respectively) or single infusions (15 or 20 mg\/kg: 66.7% and 54.5% patients, respectively). Minimum mean IgG levels and maximum mean platelet counts both occurred by day 8 in the higher (15 and 20 mg\/kg) single-dose cohorts and maximum platelet count occurred by day 11 onward in the multiple-dose cohorts. No clinically meaningful changes occurred in IgA, IgM, IgE, or albumin levels. In patients with persistent\/chronic primary ITP, rozanolixizumab demonstrated a favorable safety profile and rapid, substantial platelet increases concordant with substantial IgG reductions, especially with single doses. By day 8, in the 15 and 20 mg\/kg single-dose cohorts, &amp;gt;50% patients achieved clinically relevant platelet responses (\u226550 \u00d7 109\/L), coinciding with the lowest mean IgG levels. These data support phase 3 development of rozanolixizumab in persistent\/chronic primary ITP. This trial was registered at www.clinicaltrials.gov as #NCT02718716.<\/jats:p>","DOI":"10.1182\/bloodadvances.2020002003","type":"journal-article","created":{"date-parts":[[2020,9,4]],"date-time":"2020-09-04T15:39:58Z","timestamp":1599233998000},"page":"4136-4146","update-policy":"https:\/\/doi.org\/10.1182\/blood.2019cm0000","source":"Crossref","is-referenced-by-count":112,"title":["Phase 2 multiple-dose study of an FcRn inhibitor, rozanolixizumab, in patients with primary immune thrombocytopenia"],"prefix":"10.1182","volume":"4","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-3411-6357","authenticated-orcid":false,"given":"Tadeusz","family":"Robak","sequence":"first","affiliation":[{"name":"Department of Hematology, Medical University of Lodz, Copernicus Memorial Hospital, Lodz, Poland;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Maciej","family":"Ka\u017amierczak","sequence":"additional","affiliation":[{"name":"Department of Hematology and Bone Marrow Transplantation, Pozna\u0144 University of Medical Sciences, Pozna\u0144, Poland;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5673-4490","authenticated-orcid":false,"given":"Isidro","family":"Jarque","sequence":"additional","affiliation":[{"name":"Department of Hematology, Hospital Universitario y Polit\u00e9cnico La Fe, Valencia, Spain;"},{"name":"Centro de Investigaci\u00f3n Biom\u00e9dica en Red de C\u00e1ncer, Instituto de Salud Carlos III, Madrid, Spain;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Vasile","family":"Musteata","sequence":"additional","affiliation":[{"name":"Division of Hematology, Arensia Exploratory Medicine, Institute of Oncology, Chisinau, Moldova;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jacek","family":"Treli\u0144ski","sequence":"additional","affiliation":[{"name":"Department of Hematology, Medical University of Lodz, Copernicus Memorial Hospital, Lodz, Poland;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nichola","family":"Cooper","sequence":"additional","affiliation":[{"name":"Department of Immunology and Inflammation, Imperial College Healthcare National Health Service Trust, London, United Kingdom;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Peter","family":"Kiessling","sequence":"additional","affiliation":[{"name":"Union Chimique Belge (UCB) Pharma, Monheim-am-Rhein, Germany;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ute","family":"Massow","sequence":"additional","affiliation":[{"name":"Union Chimique Belge (UCB) Pharma, Monheim-am-Rhein, Germany;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Franz","family":"Woltering","sequence":"additional","affiliation":[{"name":"Union Chimique Belge (UCB) Pharma, Monheim-am-Rhein, Germany;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rose","family":"Snipes","sequence":"additional","affiliation":[{"name":"UCB Pharma, Raleigh, NC;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Juan","family":"Ke","sequence":"additional","affiliation":[{"name":"UCB Pharma, Slough, United Kingdom;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Grant","family":"Langdon","sequence":"additional","affiliation":[{"name":"PTx Solutions Ltd, London, United Kingdom;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"James B.","family":"Bussel","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Weill Cornell Medicine, New York, NY; 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