{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,3,9]],"date-time":"2026-03-09T09:28:10Z","timestamp":1773048490352,"version":"3.50.1"},"reference-count":25,"publisher":"Wiley","issue":"3","license":[{"start":{"date-parts":[[2016,12,27]],"date-time":"2016-12-27T00:00:00Z","timestamp":1482796800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/100005614","name":"Biogen","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100005614","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Haemophilia"],"published-print":{"date-parts":[[2017,5]]},"abstract":"<jats:sec><jats:title>Introduction<\/jats:title><jats:p>Patients with haemophilia on long\u2010acting prophylactic treatment may experience an improvement in health\u2010related quality of life (<jats:styled-content style=\"fixed-case\">HRQ<\/jats:styled-content>oL) through reductions in breakthrough bleeds and associated complications, including long\u2010term joint damage, compared with episodic treatment.<\/jats:p><\/jats:sec><jats:sec><jats:title>Aim<\/jats:title><jats:p>This analysis examined clinical trial data to understand the psychometric characteristics (reliability, validity and sensitivity to change over time) of the Haem\u2010A\u2010QoL Questionnaire in adult males with haemophilia.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>Two recent, multinational, Phase 3 clinical trials of new, long\u2010acting factor concentrates (A\u2010<jats:styled-content style=\"fixed-case\">LONG<\/jats:styled-content>:<jats:styled-content style=\"fixed-case\">rFVIIIF<\/jats:styled-content>c; B\u2010<jats:styled-content style=\"fixed-case\">LONG<\/jats:styled-content>:<jats:styled-content style=\"fixed-case\">rFIXF<\/jats:styled-content>c) assessed<jats:styled-content style=\"fixed-case\">HRQ<\/jats:styled-content>oL in adolescent and adult males with severe haemophilia A or B respectively. The adults\u2019 baseline assessments, via the 46\u2010item Haem\u2010A\u2010QoL Questionnaire, and change over time at the 6\u2010month assessment were used in the psychometric analyses.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Internal consistency reliability was adequate (Cronbach's alpha &gt; 0.70) for nine of the 10 Haem\u2010A\u2010QoL domains and for \u2018Total Score\u2019 in both trials at baseline (A\u2010<jats:styled-content style=\"fixed-case\">LONG<\/jats:styled-content>,<jats:italic>n<\/jats:italic>= 133; B\u2010<jats:styled-content style=\"fixed-case\">LONG<\/jats:styled-content>,<jats:italic>n<\/jats:italic>= 73). At baseline, several Haem\u2010A\u2010QoL domains and \u2018Total Score\u2019 demonstrated known\u2010groups and convergent validity when compared with other trial measures, including the<jats:styled-content style=\"fixed-case\">EQ<\/jats:styled-content>\u20105D (items and total scores) and joint impairment. Change score correlations (baseline to 28 weeks) between the<jats:styled-content style=\"fixed-case\">EQ<\/jats:styled-content>\u20105D and the Haem\u2010A\u2010QoL \u2018Total Score\u2019, and \u2018Physical Health\u2019 and \u2018Feelings\u2019 domains were moderate in magnitude (\u2502<jats:italic>r<\/jats:italic>\u2502 \u2265 0.33;<jats:italic>P<\/jats:italic>&lt; 0.03), demonstrating sensitivity to change for these outcome measures in A\u2010<jats:styled-content style=\"fixed-case\">LONG<\/jats:styled-content>.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion<\/jats:title><jats:p>These psychometric analyses provide evidence of the reliability, validity and ability to detect change of the Haem\u2010A\u2010QoL to assess the<jats:styled-content style=\"fixed-case\">HRQ<\/jats:styled-content>oL of adult males with severe haemophilia A and B in longitudinal clinical trials.<\/jats:p><\/jats:sec>","DOI":"10.1111\/hae.13140","type":"journal-article","created":{"date-parts":[[2016,12,27]],"date-time":"2016-12-27T13:27:08Z","timestamp":1482845228000},"page":"383-391","source":"Crossref","is-referenced-by-count":38,"title":["Measurement properties of the Haem\u2010A\u2010QoL in haemophilia clinical trials"],"prefix":"10.1111","volume":"23","author":[{"given":"S.","family":"von Mackensen","sequence":"first","affiliation":[{"name":"Institute of Medical Psychology University Medical Centre Hamburg\u2010Eppendorf Hamburg Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"A.","family":"Eldar\u2010Lissai","sequence":"additional","affiliation":[{"name":"Biogen Cambridge MA USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"P.","family":"Auguste","sequence":"additional","affiliation":[{"name":"Evidera Bethesda MD USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"S.","family":"Krishnan","sequence":"additional","affiliation":[{"name":"Biogen Cambridge MA USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"R.","family":"von Maltzahn","sequence":"additional","affiliation":[{"name":"Evidera Bethesda MD USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"R.","family":"Yu","sequence":"additional","affiliation":[{"name":"Evidera Bethesda MD USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"K. W.","family":"Wyrwich","sequence":"additional","affiliation":[{"name":"Evidera Bethesda MD USA"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"311","published-online":{"date-parts":[[2016,12,27]]},"reference":[{"key":"e_1_2_8_2_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2516.2007.01625.x"},{"key":"e_1_2_8_3_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2013-10-529974"},{"key":"e_1_2_8_4_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1305074"},{"key":"e_1_2_8_5_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa067659"},{"key":"e_1_2_8_6_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1538-7836.2011.04214.x"},{"key":"e_1_2_8_7_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2516.2010.02435.x"},{"key":"e_1_2_8_8_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.12242"},{"key":"e_1_2_8_9_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1098-3015(10)67535-3"},{"key":"e_1_2_8_10_1","doi-asserted-by":"publisher","DOI":"10.1007\/978-0-387-78665-0_112"},{"key":"e_1_2_8_11_1","first-page":"65132","article-title":"Guidance for industry on patient\u2010reported outcome measures: use in medical product development to support labeling claims","volume":"74","author":"Food and Drug Administration","year":"2009","journal-title":"Fed Reg"},{"key":"e_1_2_8_12_1","doi-asserted-by":"publisher","DOI":"10.1016\/0168-8510(90)90421-9"},{"key":"e_1_2_8_13_1","unstructured":"Agency for Healthcare Research and Quality.Calculating the U.S. Population\u2010based EQ\u20105D Index Score: Research Initiative in Clinical Economics. February2005.Agency for Healthcare Research and Quality Rockville MD. Available athttp:\/\/www.ahrq.gov\/professionals\/clinicians-providers\/resources\/rice\/EQ5Dscore.html. Accessed February 18 2015."},{"key":"e_1_2_8_14_1","volume-title":"UK Population Norms for EQ\u20105D","author":"Kind P","year":"1999"},{"key":"e_1_2_8_15_1","doi-asserted-by":"publisher","DOI":"10.1002\/acr.20353"},{"key":"e_1_2_8_16_1","volume-title":"Psychometric Theory","author":"Nunnally JC","year":"1994"},{"key":"e_1_2_8_17_1","doi-asserted-by":"publisher","DOI":"10.7249\/CB361"},{"key":"e_1_2_8_18_1","doi-asserted-by":"crossref","first-page":"25","DOI":"10.1093\/oso\/9780198527695.003.0003","volume-title":"Assessing Quality of Life in Clinical Trials","author":"Hays RD","year":"2005"},{"key":"e_1_2_8_19_1","doi-asserted-by":"publisher","DOI":"10.2165\/00019053-200725050-00003"},{"key":"e_1_2_8_20_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2004-06-2283"},{"key":"e_1_2_8_21_1","doi-asserted-by":"publisher","DOI":"10.1586\/erp.11.49"},{"key":"e_1_2_8_22_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2516.2007.01505.x"},{"key":"e_1_2_8_23_1","first-page":"165","article-title":"Prophylaxis in haemophilia should be life\u2010long","volume":"10","author":"Makris M","year":"2012","journal-title":"Blood Transfus"},{"key":"e_1_2_8_24_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2516.2001.00534.x"},{"key":"e_1_2_8_25_1","doi-asserted-by":"publisher","DOI":"10.1160\/TH11-03-0193"},{"key":"e_1_2_8_26_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0049-3848(08)70027-6"}],"container-title":["Haemophilia"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.wiley.com\/onlinelibrary\/tdm\/v1\/articles\/10.1111%2Fhae.13140","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.wiley.com\/onlinelibrary\/tdm\/v1\/articles\/10.1111%2Fhae.13140","content-type":"unspecified","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1111\/hae.13140","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2024,6,21]],"date-time":"2024-06-21T12:50:10Z","timestamp":1718974210000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1111\/hae.13140"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2016,12,27]]},"references-count":25,"journal-issue":{"issue":"3","published-print":{"date-parts":[[2017,5]]}},"alternative-id":["10.1111\/hae.13140"],"URL":"https:\/\/doi.org\/10.1111\/hae.13140","archive":["Portico"],"relation":{},"ISSN":["1351-8216","1365-2516"],"issn-type":[{"value":"1351-8216","type":"print"},{"value":"1365-2516","type":"electronic"}],"subject":[],"published":{"date-parts":[[2016,12,27]]}}}