{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,17]],"date-time":"2026-08-17T14:51:19Z","timestamp":1786978279405,"version":"build-2736575974"},"reference-count":16,"publisher":"Wiley","issue":"11","license":[{"start":{"date-parts":[[2020,9,9]],"date-time":"2020-09-09T00:00:00Z","timestamp":1599609600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/100002429","name":"Amgen","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100002429","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Pediatric Blood &amp; Cancer"],"published-print":{"date-parts":[[2020,11]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Background<\/jats:title>\n                    <jats:p>Treatment for chronic immune thrombocytopenia (cITP) in children is largely limited to immunosuppressive agents. Thrombopoietin receptor agonists (TRAs) have been used to treat cITP in adults for over a decade. The objective of this integrated analysis was to examine the safety and efficacy of the TRA romiplostim in children with ITP.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>We examined efficacy and safety in children with ITP across five romiplostim trials: final data from two double\u2010blind placebo\u2010controlled trials and two open\u2010label extensions, and interim data from an ongoing single\u2010arm trial.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>\n                      Patients (n\u00a0=\u00a024 initially placebo; n\u00a0=\u00a0262 initially romiplostim) had a median age of 10.0\u00a0years (Q1: 6.0, Q3: 13.0), ITP duration of 1.9\u00a0years (Q1: 1.0, Q3: 4.0), and baseline platelet count of 14.3\u00a0\u00d7\u00a010\n                      <jats:sup>9<\/jats:sup>\n                      \/L (Q1: 7.5, Q3: 23.0). Among 282 patients receiving romiplostim, median treatment duration was 65\u00a0weeks (range 8\u2010471\u00a0weeks) and median weekly dose was 6.6\u00a0\u03bcg\/kg (range 0.1\u20109.7\u00a0\u03bcg\/kg). Overall, 89% of romiplostim\u2010treated patients had platelet responses. Nineteen patients (7%) maintained treatment\u2010free responses for \u22656\u00a0months while withholding all ITP therapy. Grade 3 and 4 adverse events of bleeding occurred in 10% and &lt;1% of romiplostim\u2010treated patients, respectively. Twenty\u2010five percent of patients had a serious adverse event, most commonly epistaxis (6%). Seven patients (2%) had neutralizing antibodies against romiplostim postbaseline and none had neutralizing antibodies against endogenous thrombopoietin. Efficacy and safety results appeared similar between children with ITP for \u226412\u00a0months and &gt;12\u00a0months at baseline.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions<\/jats:title>\n                    <jats:p>Across five pediatric clinical trials, romiplostim was well tolerated. Most patients had a platelet response; some maintained responses for at least 6\u00a0months while withholding all ITP therapy.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1002\/pbc.28630","type":"journal-article","created":{"date-parts":[[2020,9,10]],"date-time":"2020-09-10T03:32:11Z","timestamp":1599708731000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":10,"title":["Romiplostim treatment for children with immune thrombocytopenia: Results of an integrated database of five clinical trials"],"prefix":"10.1002","volume":"67","author":[{"given":"Michael D.","family":"Tarantino","sequence":"first","affiliation":[{"name":"The Bleeding and Clotting Disorders Institute and University of Illinois College of Medicine at Peoria  Peoria Illinois"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jenny","family":"Despotovic","sequence":"additional","affiliation":[{"name":"Texas Children's Hematology Center  Houston Texas"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"John","family":"Roy","sequence":"additional","affiliation":[{"name":"Children's Health Queensland &amp; Pathology Queensland The University of Queensland, Saint Lucia  Brisbane Queensland Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"John","family":"Grainger","sequence":"additional","affiliation":[{"name":"Royal Manchester Children's Hospital University of Manchester, Central Manchester University Hospitals NHS Foundation Trust, Manchester Academic Health Science Centre  Manchester UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nichola","family":"Cooper","sequence":"additional","affiliation":[{"name":"Hammersmith Hospital Imperial College London  London UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Donald","family":"Beam","sequence":"additional","affiliation":[{"name":"Cook Children's Medical Center  Fort Worth Texas"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ashok","family":"Raj","sequence":"additional","affiliation":[{"name":"Pediatric Cancer and Blood Disorders Clinic  Louisville Kentucky"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0016-6698","authenticated-orcid":false,"given":"Alexey","family":"Maschan","sequence":"additional","affiliation":[{"name":"Dmitry Rogachev National Research Center for Pediatric Hematology Oncology and Immunology  Moscow Russia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"June","family":"Kim","sequence":"additional","affiliation":[{"name":"Amgen Inc.  Thousand Oaks California"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Melissa","family":"Eisen","sequence":"additional","affiliation":[{"name":"Amgen Inc.  Thousand Oaks California"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2020,9,9]]},"reference":[{"key":"e_1_2_9_2_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2141.2006.06024.x"},{"key":"e_1_2_9_3_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2010-08-302984"},{"key":"e_1_2_9_4_1","doi-asserted-by":"publisher","DOI":"10.1111\/bjh.12889"},{"issue":"1","key":"e_1_2_9_5_1","first-page":"27","article-title":"Childhood immune thrombocytopenia: clinical presentation and management","volume":"12","author":"Faki Osman ME","year":"2012","journal-title":"Sudan J Paediatr"},{"key":"e_1_2_9_6_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2008-07-162503"},{"key":"e_1_2_9_7_1","doi-asserted-by":"publisher","DOI":"10.1002\/pbc.20453"},{"key":"e_1_2_9_8_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.hoc.2009.09.001"},{"key":"e_1_2_9_9_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2010-10-313908"},{"key":"e_1_2_9_10_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(16)00279-8"},{"key":"e_1_2_9_11_1","doi-asserted-by":"publisher","DOI":"10.1002\/pbc.25136"},{"key":"e_1_2_9_12_1","doi-asserted-by":"publisher","DOI":"10.3324\/haematol.2018.202283"},{"issue":"1","key":"e_1_2_9_13_1","first-page":"2334","article-title":"A single\u2010arm, open\u2010label, long\u2010term efficacy and safety study of subcutaneous (SC) romiplostim in children with immune thrombocytopenia (ITP)","volume":"130","author":"Grainger JD","year":"2017","journal-title":"Blood"},{"key":"e_1_2_9_14_1","doi-asserted-by":"publisher","DOI":"10.1111\/bjh.13827"},{"key":"e_1_2_9_15_1","doi-asserted-by":"publisher","DOI":"10.1111\/bjh.15803"},{"key":"e_1_2_9_16_1","doi-asserted-by":"publisher","DOI":"10.1002\/pbc.26003"},{"key":"e_1_2_9_17_1","doi-asserted-by":"publisher","DOI":"10.1007\/s00277-011-1172-9"}],"container-title":["Pediatric Blood &amp; Cancer"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.wiley.com\/onlinelibrary\/tdm\/v1\/articles\/10.1002%2Fpbc.28630","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/pbc.28630","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/full-xml\/10.1002\/pbc.28630","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/pbc.28630","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,9,4]],"date-time":"2023-09-04T16:55:36Z","timestamp":1693846536000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1002\/pbc.28630"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2020,9,9]]},"references-count":16,"journal-issue":{"issue":"11","published-print":{"date-parts":[[2020,11]]}},"alternative-id":["10.1002\/pbc.28630"],"URL":"https:\/\/doi.org\/10.1002\/pbc.28630","archive":["Portico"],"relation":{},"ISSN":["1545-5009","1545-5017"],"issn-type":[{"value":"1545-5009","type":"print"},{"value":"1545-5017","type":"electronic"}],"subject":[],"published":{"date-parts":[[2020,9,9]]},"assertion":[{"value":"2019-11-08","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2020-07-18","order":1,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2020-09-09","order":2,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}],"article-number":"e28630"}}