{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,14]],"date-time":"2026-10-14T03:12:51Z","timestamp":1791947571910,"version":"4.3.4"},"reference-count":31,"publisher":"Wiley","issue":"3","license":[{"start":{"date-parts":[[2023,2,7]],"date-time":"2023-02-07T00:00:00Z","timestamp":1675728000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Haemophilia"],"published-print":{"date-parts":[[2023,5]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Introduction<\/jats:title><jats:p>Since the approval by the EMA of emicizumab for the care of severe haemophilia A without inhibitor, most of the patients of our haemophilia treatment centre started this new treatment. Thanks to the setting of a therapeutic patient education program including three pharmaceutical consultations (PC), we could follow patients\u2019 lifestyle evolution.<\/jats:p><\/jats:sec><jats:sec><jats:title>Aim<\/jats:title><jats:p>The study aimed to assess the perceived clinical evolution, quality of life and treatment satisfaction of patients after 1 year of emicizumab therapy in real\u2010life settings.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>The study was observational, retrospective and monocentric. Every patient over 18 years old receiving emicizumab from June 2020 and who underwent the 3\u00a0PC until March 2022 were included. The clinical evolution was self\u2010estimated by patients with zero\u2010to\u2010six scales before versus 1 year after emicizumab, according to the following parameters: general health state, pain and bleedings (spontaneous or post\u2010traumatic, and patients\u2019 identification ability). Patients\u2019 quality of life was also estimated with the EQ\u20105D\u20103L survey. Their satisfaction, graduated with a zero\u2010to\u2010ten scale, and treatment management were reported during the third PC.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Thirty\u2010eight patients were enrolled. Their general health state improved significantly (<jats:italic>p<\/jats:italic>\u00a0=\u00a0.0023) with an EQ\u20105D\u20103L score at 69.6 (\u00b119.4) out of 100. Although chronic pains remained a persistent issue for 33 (86.8%) patients, their intensity was significantly decreasing after 1 year. Perceived frequency of bleedings was significantly reduced too. On average, the satisfaction of emicizumab therapy was 9.1 (\u00b1 1.02) out of 10.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion<\/jats:title><jats:p>After 1 year of emicizumab therapy, the general health state estimated by patients improved, the pain and the perceived frequency of bleedings diminished. Overall, this treatment received a high patients\u2019 satisfaction rate.<\/jats:p><\/jats:sec>","DOI":"10.1111\/hae.14755","type":"journal-article","created":{"date-parts":[[2023,2,7]],"date-time":"2023-02-07T12:24:48Z","timestamp":1675772688000},"page":"709-715","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":18,"title":["Assessment of the clinical perception, quality of life and satisfaction of patients with severe congenital haemophilia A without inhibitor after 1 year of emicizumab therapy"],"prefix":"10.1111","volume":"29","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-6495-4888","authenticated-orcid":false,"given":"Ga\u00eblle","family":"Oka","sequence":"first","affiliation":[{"name":"Pharmacy Department Cochin Hospital (Assistance Publique \u2013 H\u00f4pitaux de Paris)  Paris France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Val\u00e9rie","family":"Roussel\u2010Robert","sequence":"additional","affiliation":[{"name":"Haemophilia Treatment Centre Cochin Hospital (Assistance Publique \u2013 H\u00f4pitaux de Paris)  Paris France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Clara","family":"Levivien","sequence":"additional","affiliation":[{"name":"Pharmacy Department Cochin Hospital (Assistance Publique \u2013 H\u00f4pitaux de Paris)  Paris France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Isabelle","family":"Lopez","sequence":"additional","affiliation":[{"name":"Pharmacy Department Cochin Hospital (Assistance Publique \u2013 H\u00f4pitaux de Paris)  Paris France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"R\u00e9mi","family":"Pieragostini","sequence":"additional","affiliation":[{"name":"Pharmacy Department Cochin Hospital (Assistance Publique \u2013 H\u00f4pitaux de Paris)  Paris France"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2023,2,7]]},"reference":[{"key":"e_1_2_8_2_1","doi-asserted-by":"publisher","DOI":"10.7326\/M19-1208"},{"key":"e_1_2_8_3_1","doi-asserted-by":"publisher","DOI":"10.7326\/M19-2535"},{"key":"e_1_2_8_4_1","unstructured":"World Federation of Haemophilia.Annual Global Survey 2020. 2020 [cit\u00e9 7 ao\u00fbt 2022]; Disponible sur:https:\/\/www1.wfh.org\/publications\/files\/pdf\u20102045.pdf"},{"issue":"6","key":"e_1_2_8_5_1","first-page":"479","article-title":"Factor VIII replacement is still the standard of care in haemophilia A","volume":"17","author":"Aledort L","year":"2019","journal-title":"Blood Transfus"},{"key":"e_1_2_8_6_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14129"},{"key":"e_1_2_8_7_1","doi-asserted-by":"publisher","DOI":"10.1111\/trf.13016"},{"issue":"5","key":"e_1_2_8_8_1","article-title":"Hemophilia: a disease on the move","volume":"75","author":"P\u00e9ters P","year":"2020","journal-title":"Rev Med Liege"},{"key":"e_1_2_8_9_1","unstructured":"European Medicines Agency.Hemlibra epar public assessment report [Internet]. EMA. 2018 [cit\u00e9 7 ao\u00fbt 2022]. Disponible sur:https:\/\/www.ema.europa.eu\/en\/documents\/assessment\u2010report\/hemlibra\u2010epar\u2010public\u2010assessment\u2010report_en.pdf"},{"key":"e_1_2_8_10_1","unstructured":"European Medicines Agency.Hemlibra epar public assessment report variation [Internet]. EMA. 2019 [cit\u00e9 7 ao\u00fbt 2022]. Disponible sur:https:\/\/www.ema.europa.eu\/en\/documents\/variation\u2010report\/hemlibra\u2010h\u2010c\u20104406\u2010ii\u20100002\u2010epar\u2010assessment\u2010report\u2010variation_en.pdf"},{"key":"e_1_2_8_11_1","doi-asserted-by":"publisher","DOI":"10.3324\/haematol.2019.232132"},{"key":"e_1_2_8_12_1","unstructured":"Roche.Summary of product characteristics \u2010 emicizumab (Hemlibra) [Internet]. 2022 [cit\u00e9 7 ao\u00fbt 2022]. Disponible sur:https:\/\/www.ema.europa.eu\/en\/documents\/product\u2010information\/hemlibra\u2010epar\u2010product\u2010information_en.pdf"},{"key":"e_1_2_8_13_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1703068"},{"key":"e_1_2_8_14_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1803550"},{"issue":"88","key":"e_1_2_8_15_1","article-title":"L\u2019\u00e9ducation th\u00e9rapeutique du patient h\u00e9mophile","volume":"9","author":"Gu\u00e9rois C","year":"2009","journal-title":"Kin\u00e9sith\u00e9rapie Rev"},{"issue":"05","key":"e_1_2_8_16_1","article-title":"Practical guidance of the GTH haemophilia board on the use of emicizumab in patients with haemophilia A","volume":"40","author":"Holstein K","year":"2020","journal-title":"H\u00e4mostaseologie"},{"issue":"253","key":"e_1_2_8_17_1","article-title":"L\u2019\u00e9ducation th\u00e9rapeutique, une n\u00e9cessit\u00e9 pour les personnes h\u00e9mophiles","volume":"68","author":"Gu\u00e9rin N","year":"2019","journal-title":"Rev Infirm"},{"issue":"02","key":"e_1_2_8_18_1","article-title":"Cost\u2010effectiveness and budget impact of emicizumab prophylaxis in haemophilia A patients with inhibitors","volume":"120","author":"Cortesi PA","year":"2020","journal-title":"Thromb Haemost"},{"key":"e_1_2_8_19_1","doi-asserted-by":"publisher","DOI":"10.1007\/s10198-011-0351-x"},{"key":"e_1_2_8_20_1","doi-asserted-by":"publisher","DOI":"10.2147\/PPA.S141392"},{"key":"e_1_2_8_21_1","doi-asserted-by":"crossref","unstructured":"SzendeA JanssenB CabasesJ \u00e9diteurs.Self\u2010Reported Population Health: An International Perspective based on EQ\u20105D. Dordrecht (NL): Springer; 2014.","DOI":"10.1007\/978-94-007-7596-1"},{"key":"e_1_2_8_22_1","doi-asserted-by":"publisher","DOI":"10.1007\/s10198-021-01326-9"},{"key":"e_1_2_8_23_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.12617"},{"key":"e_1_2_8_24_1","doi-asserted-by":"publisher","DOI":"10.1186\/s12955-018-0908-9"},{"key":"e_1_2_8_25_1","doi-asserted-by":"publisher","DOI":"10.1186\/s12891-021-04319-0"},{"key":"e_1_2_8_26_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14184"},{"issue":"03","key":"e_1_2_8_27_1","article-title":"Pharmacokinetics and Pharmacodynamics of Emicizumab in Persons with Hemophilia A with Factor VIII Inhibitors: HAVEN 1 Study","volume":"121","author":"Schmitt C","year":"2021","journal-title":"Thromb Haemost"},{"key":"e_1_2_8_28_1","unstructured":"Centre de R\u00e9f\u00e9rence H\u00e9mophilie et autres d\u00e9ficits constitutionnels en prot\u00e9ines de la coagulation.Protocole national de diagnostic et de soins (PNDS) h\u00e9mophilie [Internet]. 2019 [cit\u00e9 7 ao\u00fbt 2022]. Disponible sur:https:\/\/www.has\u2010sante.fr\/upload\/docs\/application\/pdf\/2019\u201010\/pnds_hemophilie_argumentaire_10.10.19.pdf"},{"key":"e_1_2_8_29_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2017-08-801662"},{"key":"e_1_2_8_30_1","unstructured":"Minist\u00e8re des affaires sociales et de la sant\u00e9.Avis relatif aux prix de sp\u00e9cialit\u00e9s pharmaceutiques publi\u00e9s en application des articles L. 162\u201016\u20105 et L. 162\u201016\u20106 du code de la s\u00e9curit\u00e9 sociale. Journal Officiel n\u00b00076 mars 2017."},{"key":"e_1_2_8_31_1","unstructured":"Minist\u00e8re des solidarit\u00e9s et de la sant\u00e9.Avis relatif aux prix de sp\u00e9cialit\u00e9s pharmaceutiques publi\u00e9s en application des articles L. 162\u201016\u20105 et L. 162\u201016\u20106 du code de la s\u00e9curit\u00e9 sociale. Journal Officiel n\u00b00031 f\u00e9vr 2019."},{"key":"e_1_2_8_32_1","article-title":"\u00c9valuation de l'impact budg\u00e9taire de l'introduction d'un traitement par \u00e9micizumab chez des patients atteints d'h\u00e9mophilie A s\u00e9v\u00e8re cong\u00e9nitale sans inhibiteur","author":"Oka G","year":"2022","journal-title":"Ann Pharm Fr."}],"container-title":["Haemophilia"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1111\/hae.14755","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/full-xml\/10.1111\/hae.14755","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1111\/hae.14755","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2024,5,28]],"date-time":"2024-05-28T14:05:52Z","timestamp":1716905152000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1111\/hae.14755"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2023,2,7]]},"references-count":31,"journal-issue":{"issue":"3","published-print":{"date-parts":[[2023,5]]}},"alternative-id":["10.1111\/hae.14755"],"URL":"https:\/\/doi.org\/10.1111\/hae.14755","archive":["Portico"],"relation":{},"ISSN":["1351-8216","1365-2516"],"issn-type":[{"value":"1351-8216","type":"print"},{"value":"1365-2516","type":"electronic"}],"subject":[],"published":{"date-parts":[[2023,2,7]]},"assertion":[{"value":"2022-07-20","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2023-01-19","order":1,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2023-02-07","order":2,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}