初级预防对智利血友病儿童关节健康的影响

IF 3.7 3区医学 Q1 HEMATOLOGY

Thrombosis research Pub Date : 2025-07-10 DOI:10.1016/j.thromres.2025.109401

Verónica Soto-Arellano , Agustina María Petracco , Carlos Cruz-Montecinos

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引用次数: 0

摘要

初级预防是严重血友病A和b的金标准。对这种治疗进行适当的监测和随访对于取得良好结果至关重要。血友病早期关节病超声（HEAD-US）有助于评估关节状态和检测初始损伤。我们的研究旨在通过HEAD-US评估初级预防对智利儿科人群关节健康的有效性。方法我们选择国内所有15岁以下接受血浆源性FVIII/FIX产品一级预防的严重血友病A或B患者。纳入分析的变量包括关节健康、治疗依从性（Veritas-Pro）、早晨预防治疗、家庭和医院治疗管理以及居住在大都市地区。结果对来自23个公共中心的86名儿童进行了评估（覆盖智利总人口的85%）。接受标准半衰期FVIII/IX预防治疗的儿童中，有一半仍然存在关节损伤，其中踝关节受影响最大。在关节健康的保护因素中，坚持和生活在大都市地区显示出显著的优势比(p <；0.05)。结论50%的儿童存在关节损伤，其中踝关节损伤最为严重。软骨损伤是8岁以上儿童的关键标志，而滑膜炎主要发生在年幼的儿童中。坚持预防和居住在大都市地区是保护性因素。这些结果强调了监测关节健康和使用HEAD-US进行儿童初级预防早期筛查的重要性。

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Joint health in Chilean children with haemophilia on primary prophylaxis

Introduction

Primary prophylaxis is the gold standard in severe haemophilia A and B. Proper monitoring and follow-up of this treatment are essential for achieving good outcomes. Haemophilia Early Arthropathy Ultrasound (HEAD-US) helps assess joint status and detect initial damage.

Our study aimed to evaluate the effectiveness of primary prophylaxis on joint health through HEAD-US in the paediatric population in Chile.

Method

We selected all patients under 15 years old in the country with severe haemophilia A or B who had received primary prophylaxis with plasma-derived FVIII/FIX products. Variables included in the analysis were joint health, treatment adherence (Veritas-Pro), morning prophylaxis treatment, home and hospital administration of treatment, and residence in the metropolitan region.

Results

Eighty-six children from 23 public centres were assessed (covers 85 % of the total population in Chile). Half of the children receiving standard half-life FVIII/IX prophylaxis still present joint damage, with the ankle the most affected joint. Among protective factors for joint health, adherence and living in the metropolitan region showed a significant odds ratio (p < 0.05).

Conclusion

Joint damage was present in 50% of the children, with the ankle being the most affected. Cartilage damage is a key marker in children over eight, while synovitis is predominant in younger ones. Adherence to prophylaxis and living in the metropolitan region are protective factors. These results highlight the importance of monitoring joint health and using HEAD-US for early screening in children on primary prophylaxis.

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来源期刊

Thrombosis research 医学-外周血管病

CiteScore

14.60

自引率

4.00%

发文量

364

审稿时长

31 days

期刊介绍： Thrombosis Research is an international journal dedicated to the swift dissemination of new information on thrombosis, hemostasis, and vascular biology, aimed at advancing both science and clinical care. The journal publishes peer-reviewed original research, reviews, editorials, opinions, and critiques, covering both basic and clinical studies. Priority is given to research that promises novel approaches in the diagnosis, therapy, prognosis, and prevention of thrombotic and hemorrhagic diseases.