M.P. Esposto , E. Bonetti , R. Balter , V. Pezzella , A. Zaccaron , V. Vitale , G. Caddeo , M. Chinello , G. Tridello , G. Poli , S. Cesaro
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In 2022, only one patient was on Standard-half-life-rFVIII, 7 patients were on Extended Half-Life (EHL)-rFVIII/rFIX and 7 patients were on Emicizumab; 1 patient was on Concizumab. The mean ABR passed from 5,7 in the period 2001–2009 to 0,8 in the period 2019–2022. We performed 106 ultrasonographic exams in 19 patients to evaluate the HEAD-US score: a percentage of 45,3 % of these exams revealed a score of 0 and the HEAD-US score resulted inferior in patients who started prophylaxis at an age below the median age at the initiation of prophylaxis. The score showed a tendency to correlate with the number of previous hemarthrosis. In our cohort, the overall prevalence of inhibitors is 0,09 among all patients, and 0,14 in severe HA/HB. 4 patients needed a Central Venous Line (CVL), and 3 patients developed at least one severe complication. Before 2017, we observed 4 cases of life-threatening bleeding in children less than 2 years old not on prophylaxis.</p></div><div><h3>Results</h3><p>our real-life experience reflects the changes in hemophilia management in the last two decades. In the future, other drugs are expected.</p></div>","PeriodicalId":46404,"journal":{"name":"Clinical Epidemiology and Global Health","volume":null,"pages":null},"PeriodicalIF":2.3000,"publicationDate":"2024-08-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2213398424002598/pdfft?md5=65f4d58bc023f7bf4b1e5c99be0a56bd&pid=1-s2.0-S2213398424002598-main.pdf","citationCount":"0","resultStr":"{\"title\":\"Changes over the last two decades in Hemophilia A and B management in a Tertiary Care Pediatric Centre\",\"authors\":\"M.P. Esposto , E. Bonetti , R. Balter , V. Pezzella , A. Zaccaron , V. Vitale , G. Caddeo , M. Chinello , G. Tridello , G. Poli , S. Cesaro\",\"doi\":\"10.1016/j.cegh.2024.101762\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<div><h3>Objective</h3><p>The treatment of Hemophilia A and B has significantly evolved.</p></div><div><h3>Study design</h3><p>we conducted a retrospective, observational study among patients with HA and HB managed in a Pediatric Hematology and Oncology Unit for almost 22 years for a total of 34 patients.</p></div><div><h3>Results</h3><p>Until 2009, more than 80 % of patients were treated only on demand, while in 2022,100 % of patients with moderate and severe disease were receiving continuous prophylaxis. The median age at the start of prophylaxis passed from 10,9 to 1,65 years. In 2022, only one patient was on Standard-half-life-rFVIII, 7 patients were on Extended Half-Life (EHL)-rFVIII/rFIX and 7 patients were on Emicizumab; 1 patient was on Concizumab. The mean ABR passed from 5,7 in the period 2001–2009 to 0,8 in the period 2019–2022. 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Changes over the last two decades in Hemophilia A and B management in a Tertiary Care Pediatric Centre
Objective
The treatment of Hemophilia A and B has significantly evolved.
Study design
we conducted a retrospective, observational study among patients with HA and HB managed in a Pediatric Hematology and Oncology Unit for almost 22 years for a total of 34 patients.
Results
Until 2009, more than 80 % of patients were treated only on demand, while in 2022,100 % of patients with moderate and severe disease were receiving continuous prophylaxis. The median age at the start of prophylaxis passed from 10,9 to 1,65 years. In 2022, only one patient was on Standard-half-life-rFVIII, 7 patients were on Extended Half-Life (EHL)-rFVIII/rFIX and 7 patients were on Emicizumab; 1 patient was on Concizumab. The mean ABR passed from 5,7 in the period 2001–2009 to 0,8 in the period 2019–2022. We performed 106 ultrasonographic exams in 19 patients to evaluate the HEAD-US score: a percentage of 45,3 % of these exams revealed a score of 0 and the HEAD-US score resulted inferior in patients who started prophylaxis at an age below the median age at the initiation of prophylaxis. The score showed a tendency to correlate with the number of previous hemarthrosis. In our cohort, the overall prevalence of inhibitors is 0,09 among all patients, and 0,14 in severe HA/HB. 4 patients needed a Central Venous Line (CVL), and 3 patients developed at least one severe complication. Before 2017, we observed 4 cases of life-threatening bleeding in children less than 2 years old not on prophylaxis.
Results
our real-life experience reflects the changes in hemophilia management in the last two decades. In the future, other drugs are expected.
期刊介绍：
Clinical Epidemiology and Global Health (CEGH) is a multidisciplinary journal and it is published four times (March, June, September, December) a year. The mandate of CEGH is to promote articles on clinical epidemiology with focus on developing countries in the context of global health. We also accept articles from other countries. It publishes original research work across all disciplines of medicine and allied sciences, related to clinical epidemiology and global health. The journal publishes Original articles, Review articles, Evidence Summaries, Letters to the Editor. All articles published in CEGH are peer-reviewed and published online for immediate access and citation.
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