Changes over the last two decades in Hemophilia A and B management in a Tertiary Care Pediatric Centre

IF 2.3 Q2 PUBLIC, ENVIRONMENTAL & OCCUPATIONAL HEALTH
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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引用次数: 0

Abstract

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.

过去二十年中,一家三级儿科医疗中心在血友病 A 和血友病 B 管理方面的变化
研究设计我们对儿科血液学和肿瘤学病房近 22 年来收治的 34 名 HA 和 HB 患者进行了一项回顾性观察研究。开始接受预防治疗时的中位年龄从 10.9 岁降至 1.65 岁。2022 年,只有一名患者在使用标准半衰期的 rFVIII,7 名患者在使用延长半衰期 (EHL) 的 rFVIII/rFIX,7 名患者在使用 Emicizumab;1 名患者在使用 Concizumab。平均 ABR 从 2001-2009 年的 5.7 下降到 2019-2022 年的 0.8。我们对19名患者进行了106次超声波检查,以评估HEAD-US评分:其中45.3%的检查结果为0分,在开始预防治疗的年龄低于中位年龄的患者中,HEAD-US评分较低。该评分显示出与既往血肿次数相关的趋势。在我们的队列中,抑制剂在所有患者中的总体流行率为 0.09,在重症 HA/HB 中为 0.14。4名患者需要使用中心静脉置管(CVL),3名患者出现了至少一种严重并发症。在 2017 年之前,我们观察到 4 例未接受预防性治疗的 2 岁以下儿童发生了危及生命的出血。结果我们的实际经验反映了过去 20 年血友病治疗的变化。未来,预计还会有其他药物问世。
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来源期刊
Clinical Epidemiology and Global Health
Clinical Epidemiology and Global Health PUBLIC, ENVIRONMENTAL & OCCUPATIONAL HEALTH-
CiteScore
4.60
自引率
7.70%
发文量
218
审稿时长
66 days
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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