{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,28]],"date-time":"2026-10-28T12:49:35Z","timestamp":1793191775541,"version":"4.5.0"},"reference-count":19,"publisher":"Wiley","issue":"6","license":[{"start":{"date-parts":[[2019,9,12]],"date-time":"2019-09-12T00:00:00Z","timestamp":1568246400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"funder":[{"name":"Chugai Pharmaceutical Co., Ltd"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Haemophilia"],"published-print":{"date-parts":[[2019,11]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Introduction<\/jats:title>\n                    <jats:p>Emicizumab is a recombinant humanized bispecific monoclonal antibody mimicking the cofactor function of activated factor VIII.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Aim<\/jats:title>\n                    <jats:p>In this multicentre, open\u2010label study (HOHOEMI), we evaluated the efficacy, safety and pharmacokinetics of emicizumab in Japanese paediatric patients aged &lt;12\u00a0years with severe haemophilia A without factor VIII (FVIII) inhibitors.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>Emicizumab was administered subcutaneously, with four loading doses of 3\u00a0mg\/kg every week followed by maintenance doses of 3\u00a0mg\/kg every 2\u00a0weeks (Q2W) or 6\u00a0mg\/kg every 4\u00a0weeks (Q4W) in 6 and 7 patients, respectively.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>All patients completed at least 24\u00a0weeks of treatment. Baseline ages ranged from 4\u00a0months to 10\u00a0years, and all patients had been treated with FVIII prophylaxis prior to enrolment except a 4\u2010month\u2010old patient untreated with FVIII previously. In the respective Q2W and Q4W cohorts, 2\/6 and 5\/7 patients experienced no treated bleeding events, and annualized bleeding rates for treated bleeding events were 1.3 (95% confidence interval [CI], 0.6\u20102.9) and 0.7 (95% CI, 0.2\u20102.6). All caregivers preferred emicizumab to the patient's previous treatment. Only one related adverse event (injection site reaction) was observed. There were no thromboembolic events or thrombotic microangiopathy. Individual trough plasma concentrations of emicizumab were within the variability observed in preceding adult\/adolescent studies. All patients tested negative for anti\u2010emicizumab antibodies.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions<\/jats:title>\n                    <jats:p>\n                      Emicizumab administered Q2W or Q4W was efficacious and safe in paediatric patients with severe haemophilia A without inhibitors. This study was registered at\n                      <jats:ext-link xmlns:xlink=\"http:\/\/www.w3.org\/1999\/xlink\" xlink:href=\"http:\/\/www.clinicaltrials.jp\">http:\/\/www.clinicaltrials.jp<\/jats:ext-link>\n                      (JapicCTI\u2010173710).\n                    <\/jats:p>\n                  <\/jats:sec>","DOI":"10.1111\/hae.13848","type":"journal-article","created":{"date-parts":[[2019,9,12]],"date-time":"2019-09-12T20:09:45Z","timestamp":1568318985000},"page":"979-987","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":153,"title":["A multicentre, open\u2010label study of emicizumab given every 2 or 4\u00a0weeks in children with severe haemophilia A without inhibitors"],"prefix":"10.1111","volume":"25","author":[{"given":"Midori","family":"Shima","sequence":"first","affiliation":[{"name":"Department of Pediatrics Nara Medical University Kashihara Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Keiji","family":"Nogami","sequence":"additional","affiliation":[{"name":"Department of Pediatrics Nara Medical University Kashihara Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-2904-3237","authenticated-orcid":false,"given":"Sayaka","family":"Nagami","sequence":"additional","affiliation":[{"name":"Chugai Pharmaceutical Co., Ltd Tokyo Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Seitaro","family":"Yoshida","sequence":"additional","affiliation":[{"name":"Chugai Pharmaceutical Co., Ltd Tokyo Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8166-6522","authenticated-orcid":false,"given":"Koichiro","family":"Yoneyama","sequence":"additional","affiliation":[{"name":"Chugai Pharmaceutical Co., Ltd Tokyo Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3896-5313","authenticated-orcid":false,"given":"Akira","family":"Ishiguro","sequence":"additional","affiliation":[{"name":"Division of Hematology National Center for Child Health and Development Tokyo Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Takashi","family":"Suzuki","sequence":"additional","affiliation":[{"name":"Department of Blood Coagulation Ogikubo Hospital Tokyo Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Masashi","family":"Taki","sequence":"additional","affiliation":[{"name":"Department of Pediatrics St. Marianna University School of Medicine Kawasaki 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