{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,3,14]],"date-time":"2026-03-14T05:24:01Z","timestamp":1773465841763,"version":"3.50.1"},"reference-count":16,"publisher":"BMJ","issue":"9","funder":[{"DOI":"10.13039\/501100000801","name":"Bayer\n HealthCare","doi-asserted-by":"publisher","award":["N\/A"],"award-info":[{"award-number":["N\/A"]}],"id":[{"id":"10.13039\/501100000801","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["bmj.com"],"crossmark-restriction":true},"short-container-title":["BMJ Open"],"published-print":{"date-parts":[[2021,9]]},"abstract":"\n Introduction<\/jats:title>\n Haemophilia A is a rare bleeding disorder caused by defects in\n coagulation factor VIII (FVIII). Damoctocog alfa pegol (BAY 94\u20139027,\n Jivi, Bayer, Germany) is a site-specifically PEGylated,\n extended-half-life, recombinant FVIII, approved for use in previously\n treated patients (PTPs) aged \u226512 years with haemophilia A. However, a\n real-world evidence regarding routine clinical use of damoctocog alfa\n pegol is limited.<\/jats:p>\n <\/jats:sec>\n \n Methods and analysis<\/jats:title>\n HEM-POWR is a multinational, multicentre, non-interventional,\n prospective, postmarketing cohort study evaluating the effectiveness and\n safety of real-world treatment with damoctocog alfa pegol. Estimated\n enrolment is \u2265200 PTPs with haemophilia A, receiving damoctocog alfa\n pegol (on-demand, prophylaxis or intermittent prophylaxis (as per local\n label)), observed for 36 months. Primary outcomes are total bleeding\n events and annualised bleeding rate; secondary outcomes include\n long-term safety, joint health, pharmacokinetics, patient-reported\n outcomes (PROs) from validated questionnaires and perioperative\n haemostasis. Where applicable, reasons for switching to damoctocog alfa\n pegol, choice of treatment regimen and dose will also be captured.\n Exploratory and descriptive statistical analyses will be performed, and\n will be stratified by parameters including, but not limited to,\n prophylaxis regimen and haemophilia severity. Patients can record bleeds\n and consumption in electronic (e) Diaries, ePROs, and can access\n non-promotional study information (videos explaining study procedures)\n via an online patient portal. Optionally, patients can enrol in the\n LIFE-ACTIVE substudy designed to investigate the relationship between\n activity (measured by the ActiGraph CP Insight watch) and effectiveness\n parameters collected from HEM-POWR.<\/jats:p>\n <\/jats:sec>\n \n Ethics and dissemination<\/jats:title>\n Study approval was obtained by local independent ethics committees\n and authorities in participating study centres across Europe, the\n Americas and Asia. Informed consent from patients or their legal\n representative is a requirement for participation. The study results\n will be submitted for publication in a peer-reviewed scientific journal\n and presented at scientific conferences.<\/jats:p>\n <\/jats:sec>\n \n Trial registration numbers<\/jats:title>\n NCT03932201<\/jats:ext-link>, EUPAS26416.<\/jats:p>\n <\/jats:sec>\n \n Protocol version and date<\/jats:title>\n V.1.2, 27 September 2019.<\/jats:p>\n <\/jats:sec>","DOI":"10.1136\/bmjopen-2020-044997","type":"journal-article","created":{"date-parts":[[2022,2,14]],"date-time":"2022-02-14T12:19:13Z","timestamp":1644841153000},"page":"e044997","update-policy":"https:\/\/doi.org\/10.1136\/crossmarkpolicy","source":"Crossref","is-referenced-by-count":5,"title":["Design of the HEM-POWR study: a prospective, observational study of\n real-world treatment with damoctocog alfa pegol in patients with haemophilia\n A"],"prefix":"10.1136","volume":"11","clinical-trial-number":[{"clinical-trial-number":"nct03932201","registry":"10.18810\/clinical-trials-gov","type":"postResults"}],"author":[{"given":"Martin","family":"Sanabria","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Mar\u00eda Teresa","family":"\u00c1lvarez 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