Martin Chandler, Thomas Moulton, Lena Charafi, Jessica Charlet, Michael Recht
{"title":"达莫克托科格 Alfa Pegol 治疗 ATHNdataset 登记的 A 型血友病患者的疗效。","authors":"Martin Chandler, Thomas Moulton, Lena Charafi, Jessica Charlet, Michael Recht","doi":"10.1111/ejh.14337","DOIUrl":null,"url":null,"abstract":"<p><strong>Objectives: </strong>Health information for 17 109 people living with hemophilia A (PLwHA) is contained within the ATHNdataset. We aimed to evaluate the real-world effectiveness of damoctocog alfa pegol (BAY 94-9027, Jivi®) for hemophilia A.</p><p><strong>Methods: </strong>The ATHNdataset was queried for PLwHA receiving damoctocog alfa pegol between January 1, 2010 and April 30, 2022. Data captured via patient charts were analyzed.</p><p><strong>Results: </strong>At data cutoff, 205 PLwHA were treated with damoctocog alfa pegol: 150 (73.2%) severe (1 female [0.5%]) and 55 (26.8%) mild/moderate (3 [1.5%] female). In total, 32/205 (25.9%) PLwHA received on-demand treatment; 172 (83.9%) received prophylaxis-161 (93.6%) continuous prophylaxis. Documented bleed rates were available for 187 (91.2%) PLwHA, including those on prophylaxis and on-demand regimens, with 150 (80.2%) treated for > 12 months. Overall annualized bleeding rates and proportion of PLwHA with zero bleeds, receiving prophylaxis during the observation period, were mean (SD) 0.26 (1.03) and 138/157 (87.9%), respectively. No new or recurring inhibitors were reported.</p><p><strong>Conclusion: </strong>A low number of bleeds were observed with damoctocog alfa pegol in the real world in both male and female PLwHA. Data should be interpreted with caution owing to limitations of real-world studies and insubstantial data for female PLwHA.</p>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":2.3000,"publicationDate":"2024-11-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Effectiveness of Damoctocog Alfa Pegol to Treat Patients With Hemophilia A Enrolled in the ATHNdataset.\",\"authors\":\"Martin Chandler, Thomas Moulton, Lena Charafi, Jessica Charlet, Michael Recht\",\"doi\":\"10.1111/ejh.14337\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><strong>Objectives: </strong>Health information for 17 109 people living with hemophilia A (PLwHA) is contained within the ATHNdataset. We aimed to evaluate the real-world effectiveness of damoctocog alfa pegol (BAY 94-9027, Jivi®) for hemophilia A.</p><p><strong>Methods: </strong>The ATHNdataset was queried for PLwHA receiving damoctocog alfa pegol between January 1, 2010 and April 30, 2022. Data captured via patient charts were analyzed.</p><p><strong>Results: </strong>At data cutoff, 205 PLwHA were treated with damoctocog alfa pegol: 150 (73.2%) severe (1 female [0.5%]) and 55 (26.8%) mild/moderate (3 [1.5%] female). In total, 32/205 (25.9%) PLwHA received on-demand treatment; 172 (83.9%) received prophylaxis-161 (93.6%) continuous prophylaxis. Documented bleed rates were available for 187 (91.2%) PLwHA, including those on prophylaxis and on-demand regimens, with 150 (80.2%) treated for > 12 months. Overall annualized bleeding rates and proportion of PLwHA with zero bleeds, receiving prophylaxis during the observation period, were mean (SD) 0.26 (1.03) and 138/157 (87.9%), respectively. No new or recurring inhibitors were reported.</p><p><strong>Conclusion: </strong>A low number of bleeds were observed with damoctocog alfa pegol in the real world in both male and female PLwHA. Data should be interpreted with caution owing to limitations of real-world studies and insubstantial data for female PLwHA.</p>\",\"PeriodicalId\":11955,\"journal\":{\"name\":\"European Journal of Haematology\",\"volume\":\" \",\"pages\":\"\"},\"PeriodicalIF\":2.3000,\"publicationDate\":\"2024-11-18\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"European Journal of Haematology\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1111/ejh.14337\",\"RegionNum\":3,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q2\",\"JCRName\":\"HEMATOLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"European Journal of Haematology","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1111/ejh.14337","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"HEMATOLOGY","Score":null,"Total":0}
Effectiveness of Damoctocog Alfa Pegol to Treat Patients With Hemophilia A Enrolled in the ATHNdataset.
Objectives: Health information for 17 109 people living with hemophilia A (PLwHA) is contained within the ATHNdataset. We aimed to evaluate the real-world effectiveness of damoctocog alfa pegol (BAY 94-9027, Jivi®) for hemophilia A.
Methods: The ATHNdataset was queried for PLwHA receiving damoctocog alfa pegol between January 1, 2010 and April 30, 2022. Data captured via patient charts were analyzed.
Results: At data cutoff, 205 PLwHA were treated with damoctocog alfa pegol: 150 (73.2%) severe (1 female [0.5%]) and 55 (26.8%) mild/moderate (3 [1.5%] female). In total, 32/205 (25.9%) PLwHA received on-demand treatment; 172 (83.9%) received prophylaxis-161 (93.6%) continuous prophylaxis. Documented bleed rates were available for 187 (91.2%) PLwHA, including those on prophylaxis and on-demand regimens, with 150 (80.2%) treated for > 12 months. Overall annualized bleeding rates and proportion of PLwHA with zero bleeds, receiving prophylaxis during the observation period, were mean (SD) 0.26 (1.03) and 138/157 (87.9%), respectively. No new or recurring inhibitors were reported.
Conclusion: A low number of bleeds were observed with damoctocog alfa pegol in the real world in both male and female PLwHA. Data should be interpreted with caution owing to limitations of real-world studies and insubstantial data for female PLwHA.
期刊介绍：
European Journal of Haematology is an international journal for communication of basic and clinical research in haematology. The journal welcomes manuscripts on molecular, cellular and clinical research on diseases of the blood, vascular and lymphatic tissue, and on basic molecular and cellular research related to normal development and function of the blood, vascular and lymphatic tissue. The journal also welcomes reviews on clinical haematology and basic research, case reports, and clinical pictures.
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