预防性治疗儿童血友病患者的隐匿性关节。

The hidden joint in children with haemophilia on prophylaxis.

机构信息

Haemophilia Foundation, Buenos Aires, Argentina; Department of Orthopaedics and Traumatology, Juan A. Fernández Hospital, Buenos Aires, Argentina.

出版信息

Thromb Res. 2023 Jun;226:86-92. doi: 10.1016/j.thromres.2023.04.012. Epub 2023 Apr 26.

DOI:10.1016/j.thromres.2023.04.012

PMID:37130495

Abstract

UNLABELLED

Prophylaxis is the gold standard treatment for children with haemophilia (CWH). MRI studies revealed joint damage, even with this treatment; this suggests the presence of subclinical bleeding. In the case of children with haemophilia, it is relevant to detect early signs of joint damage, as this allows the medical team to provide the appropriate treatment and follow-up, in order to avoid arthropathy development and its consequences. The aim of this study is to detect the hidden joint in children with haemophilia on prophylaxis (CWHP) and analyse, by age group, which joint is the most affected. We define the hidden joint in CWH on prophylaxis as the joint that presents joint damage secondary to repetitive bleeding episodes and is detected in the joint evaluation, despite being asymptomatic or with mild symptoms. It is most commonly caused by repetitive subclinical bleeding.

METHODS

This was an observational, analytical, cross-sectional study of 106 CWH on prophylaxis treated in our centre. Patients were divided according to age and type of treatment. Joint damage was defined as a HEAD-US score ≥ 1.

RESULTS

Patients' median age was 12 years. All had severe haemophilia. The median age of onset of prophylaxis was 2.7. Forty-seven (44.3 %) patients received primary prophylaxis (PP) and 59 (55.7 %), secondary prophylaxis. Six hundred and thirty-six joints were analysed. Type of prophylaxis and joint involvement showed statistically significant differences (p < 0.001). However, patients on PP had a greater number of damaged joints at older ages. Twenty-two % (140) of the joints scored ≥1 on HEAD-US. Cartilage was most frequently involved, followed by synovitis, and bone damage. We observed a greater frequency and degree of arthropathy in subjects aged 11 and above. Sixty (12.7 %) joints showed a HEAD-US score ≥ 1, with no history of bleeding. The ankle was the most affected joint, representing the hidden joint according to our definition.

CONCLUSION

Prophylaxis is the best treatment for CWH. However, symptomatic or subclinical joint bleeding may occur. The routine evaluation of joint health is relevant, particularly, of the ankle. In our study, early signs of arthropathy according to age and type of prophylaxis were detected by HEAD-US.

摘要

目的

本研究旨在检测接受预防治疗的血友病儿童（CWHP）的隐匿性关节，并按年龄组分析哪个关节最易受影响。我们将预防治疗的血友病儿童中的隐匿性关节定义为由于反复出血事件引起的关节损伤，尽管无症状或症状轻微，但在关节评估中被检测到的关节。这种关节损伤最常见于反复发生的亚临床出血。

方法

这是一项针对在我们中心接受治疗的 106 名接受预防治疗的血友病儿童的观察性、分析性、横断面研究。根据年龄和治疗类型对患者进行分组。关节损伤定义为 HEAD-US 评分≥1。

结果

患者的中位年龄为 12 岁，均患有重度血友病。预防治疗的中位起始年龄为 2.7 岁。47 名（44.3%）患者接受初级预防（PP），59 名（55.7%）患者接受次级预防。共分析了 636 个关节。预防类型和关节受累在统计学上存在显著差异（p<0.001）。然而，接受 PP 的患者在年龄较大时，受损关节的数量更多。22%（140 个）的关节 HEAD-US 评分为≥1。最常受累的是软骨，其次是滑膜炎和骨损伤。我们观察到年龄在 11 岁及以上的患者中，关节炎的发生率和严重程度更高。60 个（12.7%）关节的 HEAD-US 评分≥1，且无出血史。踝关节是最易受影响的关节，根据我们的定义，它代表隐匿性关节。