{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,25]],"date-time":"2026-09-25T14:09:28Z","timestamp":1790345368142,"version":"4.1.0"},"reference-count":37,"publisher":"Wiley","issue":"6","license":[{"start":{"date-parts":[[2020,10,21]],"date-time":"2020-10-21T00:00:00Z","timestamp":1603238400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"},{"start":{"date-parts":[[2020,10,21]],"date-time":"2020-10-21T00:00:00Z","timestamp":1603238400000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/doi.wiley.com\/10.1002\/tdm_license_1.1"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Haemophilia"],"published-print":{"date-parts":[[2020,11]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Introduction<\/jats:title>\n                    <jats:p>Persons with haemophilia A (PwHA) with factor (F)VIII inhibitors, including children, have impaired health\u2010related quality of life (HRQoL). The HAVEN 2 study (NCT027955767) of paediatric PwHA with FVIII inhibitors demonstrated that subcutaneous emicizumab prophylaxis resulted in low annualizedbleed rates.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Aim<\/jats:title>\n                    <jats:p>We assessed the impact of emicizumab prophylaxis on the HRQoL of children and their caregivers participating in HAVEN 2.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>Children aged 8\u201011\u00a0years self\u2010reported HRQoL using the Haemophilia\u2010Specific Quality of Life Assessment Instrument for Children and Adolescents Short Form (Haemo\u2010QoL SF II). Caregivers of children aged 0\u201011\u00a0years completed the Adapted Inhibitor\u2010Specific Quality of Life Assessment with Aspects of Caregiver Burden. All scores were transformed to a 0\u2010100 scale, where lower scores reflect a better HRQoL. The number of missed days from school\/day care and hospitalizations was also recorded.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>In HAVEN 2 (n\u00a0=\u00a088), the median age was 6.5\u00a0years (range: 1\u201015\u00a0years); 85 participants were aged\u00a0&lt;\u00a012\u00a0years and included in this analysis, and 34 participants were aged 8\u201011\u00a0years, thereby eligible to complete the Haemo\u2010QoL SF II questionnaire. The mean (standard deviation, n) baseline Haemo\u2010QoL SF II \u2018Total\u2019 score was 30.2 (14.9, 30), indicating moderate impairment; with emicizumab, mean score decreased by \u22129.62 (7.73, 17) points to 23.0 (13.93, 20) by Week 49. The most improved domains were \u2018Sports &amp; School\u2019 and \u2018Physical Health\u2019. Caregivers reported similar improvements.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusion<\/jats:title>\n                    <jats:p>Prophylactic emicizumab is accompanied by substantial and sustained improvements in HRQoL of paediatric PwHA with FVIII inhibitors and their caregivers.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1111\/hae.14183","type":"journal-article","created":{"date-parts":[[2020,10,21]],"date-time":"2020-10-21T03:08:04Z","timestamp":1603249684000},"page":"1009-1018","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":37,"title":["Health\u2010related quality of life and caregiver burden of emicizumab in children with haemophilia A and factor VIII inhibitors\u2014Results from the HAVEN 2 study"],"prefix":"10.1111","volume":"26","author":[{"given":"Maria Elisa","family":"Mancuso","sequence":"first","affiliation":[{"name":"Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico Angelo Bianchi Bonomi Hemophilia and Thrombosis Center  Milan Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5781-7669","authenticated-orcid":false,"given":"Johnny","family":"Mahlangu","sequence":"additional","affiliation":[{"name":"Faculty of Health Sciences University of the Witwatersrand and NHLS  Johannesburg South Africa"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"suffix":"Jr","given":"Robert","family":"Sidonio","sequence":"additional","affiliation":[{"name":"Emory University and Children's Healthcare of Atlanta  Atlanta GA USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Peter","family":"Trask","sequence":"additional","affiliation":[{"name":"Genentech, Inc.  South San Francisco CA USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marianne","family":"Uguen","sequence":"additional","affiliation":[{"name":"F. Hoffmann\u2010La Roche Ltd  Basel Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tiffany","family":"Chang","sequence":"additional","affiliation":[{"name":"Genentech, Inc.  South San Francisco CA USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-5922-7061","authenticated-orcid":false,"given":"Midori","family":"Shima","sequence":"additional","affiliation":[{"name":"Nara Medical University  Kashihara Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6013-1254","authenticated-orcid":false,"given":"Guy","family":"Young","sequence":"additional","affiliation":[{"name":"Children's Hospital Los Angeles University of Southern California Keck School of Medicine  Los Angeles CA USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1585-4100","authenticated-orcid":false,"given":"Johannes","family":"Oldenburg","sequence":"additional","affiliation":[{"name":"University of Bonn  Bonn Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sylvia","family":"von Mackensen","sequence":"additional","affiliation":[{"name":"Department of Medical Psychology University Medical Centre Hamburg\u2010Eppendorf  Hamburg Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2020,10,21]]},"reference":[{"key":"e_1_2_10_2_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.amepre.2009.12.016"},{"key":"e_1_2_10_3_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.13754"},{"key":"e_1_2_10_4_1","doi-asserted-by":"publisher","DOI":"10.2147\/PPA.S196103"},{"key":"e_1_2_10_5_1","doi-asserted-by":"publisher","DOI":"10.1097\/MPH.0000000000000117"},{"key":"e_1_2_10_6_1","doi-asserted-by":"publisher","DOI":"10.1186\/s12955-019-1083-3"},{"key":"e_1_2_10_7_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1355-0691.2004.00876.x"},{"key":"e_1_2_10_8_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2516.2006.01204.x"},{"key":"e_1_2_10_9_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.13275"},{"key":"e_1_2_10_10_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.critrevonc.2011.01.002"},{"key":"e_1_2_10_11_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1208024"},{"key":"e_1_2_10_12_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2014-07-586347"},{"key":"e_1_2_10_13_1","doi-asserted-by":"publisher","DOI":"10.1111\/jth.13888"},{"key":"e_1_2_10_14_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1516437"},{"key":"e_1_2_10_15_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1538-7836.2007.02663.x"},{"key":"e_1_2_10_16_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2011-08-369132"},{"key":"e_1_2_10_17_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.12246"},{"key":"e_1_2_10_18_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2516.2007.01518.x"},{"key":"e_1_2_10_19_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2516.2009.01999.x"},{"key":"e_1_2_10_20_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.12019"},{"key":"e_1_2_10_21_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.12501"},{"key":"e_1_2_10_22_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2516.2007.01494.x"},{"key":"e_1_2_10_23_1","doi-asserted-by":"publisher","DOI":"10.1002\/pbc.24856"},{"key":"e_1_2_10_24_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.13736"},{"key":"e_1_2_10_25_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.13684"},{"key":"e_1_2_10_26_1","doi-asserted-by":"publisher","DOI":"10.1038\/nm.2942"},{"key":"e_1_2_10_27_1","unstructured":"European Medicines Agency.HEMLIBRA\u00ae solution for injection: emicizumab piIEa. 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