{"database":"biostudies-literature","file_versions":[],"scores":null,"additional":{"omics_type":["Unknown"],"volume":["27(5)"],"submitter":["Skinner MW"],"funding":["F. Hoffmann-La Roche Ltd and Chugai Pharmaceutical Co., Ltd"],"pubmed_abstract":["<h4>Introduction</h4>Severe haemophilia A (HA) has a major impact on health-related quality of life (HRQoL).<h4>Aim</h4>Assess the impact of emicizumab on HRQoL in persons with severe HA (PwHA) without factor VIII (FVIII) inhibitors in the phase 3 HAVEN 3 and 4 studies.<h4>Methods</h4>This pooled analysis examines the HRQoL of PwHA aged ≥ 18 years treated with emicizumab prophylaxis via Haemophilia-Specific Quality of Life Questionnaire for Adults (Haem-A-QoL) and EuroQoL 5-Dimensions 5-levels (EQ-5D-5L). In particular, changes from baseline in Haem-A-QoL 'Physical Health' (PH) domain and 'Total Score' (TS) are evaluated.<h4>Results</h4>Among 176 evaluable participants, 96 (55%) had received prior episodic treatment and 80 (45%) prophylaxis; 70% had ≥ 1 target joint and 51% had experienced"],"journal":["Haemophilia : the official journal of the World Federation of Hemophilia"],"pagination":["854-865"],"full_dataset_link":["https://www.ebi.ac.uk/biostudies/studies/S-EPMC8518882"],"repository":["biostudies-literature"],"pubmed_title":["The effect of emicizumab prophylaxis on long-term, self-reported physical health in persons with haemophilia A without factor VIII inhibitors in the HAVEN 3 and HAVEN 4 studies."],"pmcid":["PMC8518882"],"pubmed_authors":["Shapiro A","Callaghan MU","Shima M","Negrier C","Skinner MW","Lehle M","Jimenez-Yuste V","Levy GG","Oldenburg J","Niggli M","Pipe SW","von Mackensen S","Mahlangu J","Campinha-Bacote A","Paz-Priel I","Chebon S"],"additional_accession":[]},"is_claimable":false,"name":"The effect of emicizumab prophylaxis on long-term, self-reported physical health in persons with haemophilia A without factor VIII inhibitors in the HAVEN 3 and HAVEN 4 studies.","description":"<h4>Introduction</h4>Severe haemophilia A (HA) has a major impact on health-related quality of life (HRQoL).<h4>Aim</h4>Assess the impact of emicizumab on HRQoL in persons with severe HA (PwHA) without factor VIII (FVIII) inhibitors in the phase 3 HAVEN 3 and 4 studies.<h4>Methods</h4>This pooled analysis examines the HRQoL of PwHA aged ≥ 18 years treated with emicizumab prophylaxis via Haemophilia-Specific Quality of Life Questionnaire for Adults (Haem-A-QoL) and EuroQoL 5-Dimensions 5-levels (EQ-5D-5L). In particular, changes from baseline in Haem-A-QoL 'Physical Health' (PH) domain and 'Total Score' (TS) are evaluated.<h4>Results</h4>Among 176 evaluable participants, 96 (55%) had received prior episodic treatment and 80 (45%) prophylaxis; 70% had ≥ 1 target joint and 51% had experienced","dates":{"release":"2021-01-01T00:00:00Z","publication":"2021 Sep","modification":"2026-07-16T16:03:12.39Z","creation":"2026-07-09T11:07:34.98Z"},"accession":"S-EPMC8518882","cross_references":{"pubmed":["34171159"],"doi":["10.1111/hae.14363"]}}