Van Creveldkliniek, Department of Haematology, University Medical Center Utrecht, Utrecht; Julius Center for Health Sciences and Primary Care, University Medical Center Utrecht, Utrecht.
Haemophilia. 2013 Nov;19(6):944-50. doi: 10.1111/hae.12197. Epub 2013 Jun 4.
Outcome assessment in haemophilia is important to assess results of prophylactic treatment. Recently, the Haemophilia Joint Health Score (HJHS) was developed to assess early joint damage in children with haemophilia. Thus, the aim of this study was to assess reliability and explore validity of the HJHS in teenagers and young adults with haemophilia. Twenty-two patients with haemophilia (mean age 20.4, range 14-30, including 15 severe) were assessed by the HJHS1.0, Haemophilia Activities List (HAL), SF36 and self-evaluation was performed using a Visual Analogue Scale (VAS) scale. A subset of 12 patients were assessed by three physiotherapists to establish interobserver reliability (intraclass correlation coefficient: ICC). Total HJHS1.0 scores were calculated without overall global gait. Validity was explored by the assessment of Pearson's correlation with all outcome parameters and recent Pettersson scores. Overall outcome was good, with median HJHS score of 5.5 of a maximum 144 (range 0-34), median patients' VAS of 96.5 and maximum scores for HAL and SF36 physical functioning for the majority of patients. Pettersson scores were low (median 3.5 of 78, N = 18). Interobserver reliability was good (ICC 0.84), with limits of agreement of ±7.2 points. ICC was unaffected by different score calculation methods. Exploration of validity in 22 patients showed weak correlations of HJHS scores with patients' VAS (0.33) and HAL (-0.40) and strong correlations with SF36-PF (-0.66) and Pettersson scores (0.86). These results suggest that interobserver reliability of the HJHS1.0 in teenagers and young adults with limited joint damage is excellent. Preliminary data on validity were similar or better than those in children.
血友病的结局评估对于评估预防性治疗的结果很重要。最近，开发了血友病关节健康评分（HJHS）来评估儿童血友病的早期关节损伤。因此，本研究旨在评估 HJHS 在青少年和年轻血友病患者中的可靠性，并探索其有效性。22 名血友病患者（平均年龄 20.4 岁，范围 14-30 岁，包括 15 名重度患者）接受了 HJHS1.0、血友病活动清单（HAL）、SF36 评估，并使用视觉模拟量表（VAS）进行自我评估。12 名患者的子集由三名物理治疗师进行评估，以建立观察者间可靠性（组内相关系数：ICC）。没有总体步态的情况下计算了总 HJHS1.0 评分。通过与所有结局参数和最近的 Pettersson 评分的 Pearson 相关性评估来探索有效性。总体结局良好，HJHS 评分中位数为 144 分中的 5.5 分（范围 0-34），患者 VAS 中位数为 96.5，大多数患者的 HAL 和 SF36 身体功能评分最高。Pettersson 评分较低（中位数 18 分中的 3.5 分）。观察者间可靠性良好（ICC 0.84），一致性界限为±7.2 分。ICC 不受不同评分计算方法的影响。在 22 名患者中，HJHS 评分与患者 VAS（0.33）和 HAL（-0.40）的相关性较弱，与 SF36-PF（-0.66）和 Pettersson 评分（0.86）的相关性较强。这些结果表明，HJHS1.0 在有限关节损伤的青少年和年轻血友病患者中的观察者间可靠性极佳。有效性的初步数据与儿童相似或更好。
