{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,16]],"date-time":"2026-10-16T13:50:56Z","timestamp":1792158656408,"version":"4.4.0"},"reference-count":19,"publisher":"Wiley","issue":"4","license":[{"start":{"date-parts":[[2026,7,21]],"date-time":"2026-07-21T00:00:00Z","timestamp":1784592000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"},{"start":{"date-parts":[[2026,7,21]],"date-time":"2026-07-21T00:00:00Z","timestamp":1784592000000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/doi.wiley.com\/10.1002\/tdm_license_1.1"}],"funder":[{"DOI":"10.13039\/501100012112","name":"Swedish Orphan Biovitrum","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100012112","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":[[2026,10]]},"abstract":"<jats:title>Summary<\/jats:title>\n                  <jats:p>\n                    Avatrombopag treatment resulted in significantly higher platelet response rates in patients when compared with placebo in a phase 3b study of patients with paediatric immune thrombocytopenia (ITP) (NCT04516967). These post hoc analyses of the 12\u2010week core phase further assessed the efficacy of avatrombopag, considering the influence of baseline characteristics and protocol\u2010defined dose adjustments on the achievement and sustainability of response. The cumulative percentage of patients who achieved a platelet response (2 consecutive platelet counts \u226550\u2009\u00d7\u200910\n                    <jats:sup>9<\/jats:sup>\n                    \/L) by days 15 and 22 was 70.4% and 75.9%, respectively, with avatrombopag (placebo, 0% at both days). Only minor differences were observed by baseline characteristics. Among the 92.6% of patients (50\/54) treated with avatrombopag who achieved a clinically meaningful platelet response (platelet count \u226530\u2009\u00d7\u200910\n                    <jats:sup>9<\/jats:sup>\n                    \/L), the mean percentage of time with response was 62.5%. Protocol\u2010specified dose holds due to a platelet count &gt;250\u2009\u00d7\u200910\n                    <jats:sup>9<\/jats:sup>\n                    \/L occurred for 61.1% of patients, which may have impacted the efficacy outcomes. 78.3% of non\u2010durable responders had \u22651 dose holds prompted by a platelet count of 250\u2013400\u2009\u00d7\u200910\n                    <jats:sup>9<\/jats:sup>\n                    \/L, a platelet count that would not prompt a dose hold in real\u2010world clinical practice. Avatrombopag demonstrated a clinically meaningful platelet response across the clinical and demographic subgroups in this heavily pre\u2010treated and refractory paediatric ITP population.\n                  <\/jats:p>","DOI":"10.1111\/bjh.70709","type":"journal-article","created":{"date-parts":[[2026,7,22]],"date-time":"2026-07-22T00:54:50Z","timestamp":1784681690000},"page":"1818-1827","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Avatrombopag for persistent and chronic immune thrombocytopenia in children: A post hoc analysis of clinical characteristics of response from a phase 3b clinical trial"],"prefix":"10.1111","volume":"209","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-7302-0449","authenticated-orcid":false,"given":"Rachael F.","family":"Grace","sequence":"first","affiliation":[{"name":"Department of Pediatrics, Dana\u2010Farber\/Boston Children's Cancer and Blood Disorders Center Harvard Medical School  Boston Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3435-757X","authenticated-orcid":false,"given":"G\u00f6ksel","family":"Leblebisatan","sequence":"additional","affiliation":[{"name":"Department of Pediatric Hematology \u00c7ukurova University Medical Faculty  Adana Turkey"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6194-6826","authenticated-orcid":false,"given":"Yesim","family":"Aydinok","sequence":"additional","affiliation":[{"name":"Department of Pediatric Hematology and Oncology Ege University School of Medicine  Izmir Turkey"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3842-8788","authenticated-orcid":false,"given":"Sule","family":"Unal","sequence":"additional","affiliation":[{"name":"Department of Pediatric Hematology, Faculty of Medicine Hacettepe University  Ankara Turkey"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"John","family":"Grainger","sequence":"additional","affiliation":[{"name":"Department of Haematology Royal Manchester Children's Hospital  Manchester UK"},{"name":"University of Manchester  Manchester UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2842-9266","authenticated-orcid":false,"given":"Amanda B.","family":"Grimes","sequence":"additional","affiliation":[{"name":"Texas Children's Cancer and Hematology Center Texas Children's Hospital  Houston Texas USA"},{"name":"Division of Hematology and Oncology, Department of Pediatrics Baylor College of Medicine  Houston Texas USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0439-402X","authenticated-orcid":false,"given":"Michele P.","family":"Lambert","sequence":"additional","affiliation":[{"name":"Children's Hospital of Philadelphia  Philadelphia Pennsylvania USA"},{"name":"Perelman School of Medicine University of Pennsylvania  Philadelphia Pennsylvania USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jessica","family":"Zhang","sequence":"additional","affiliation":[{"name":"Sobi, Inc.  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Morrisville North Carolina USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"name":"the AVA\u2010PED\u2010301 Study Group","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2026,7,21]]},"reference":[{"key":"e_1_2_11_2_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2008-07-162503"},{"key":"e_1_2_11_3_1","doi-asserted-by":"publisher","DOI":"10.1159\/000492187"},{"key":"e_1_2_11_4_1","doi-asserted-by":"publisher","DOI":"10.1182\/bloodadvances.2019000812"},{"key":"e_1_2_11_5_1","doi-asserted-by":"publisher","DOI":"10.1182\/asheducation-2018.1.568"},{"key":"e_1_2_11_6_1","unstructured":"Dova Pharmaceuticals Inc.Doptelet: Prescribing information.2025. Available from:https:\/\/doptelet.com\/themes\/pdf\/prescribing\u2010information.pdf"},{"key":"e_1_2_11_7_1","unstructured":"Novartis Pharmaceuticals Corporation.Promacta: Prescribing information.2025. 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Lancet.2015;386(10004):1649\u20131658. 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