Department of Pediatrics, Peking Union Medical College Hospital, Chinese Academe of Medical Sciences and Peking Union Medical College, Beijing, China.
Department of Pediatrics, Beijing Shijitan Hospital, Capital Medical University, Beijing, China.
Haemophilia. 2021 Mar;27(2):237-244. doi: 10.1111/hae.14256. Epub 2021 Feb 6.
To explore the long-term joint outcomes of low-dose prophylaxis in Chinese children with severe haemophilia A and to analyse their related factors.
We retrospectively analysed follow-up data from 21 severe haemophilia A children on regular low-dose prophylaxis for 6-10 years. We used International Prophylaxis Study Group magnetic resonance imaging score (IPSG MRI score), Hemophilia Joint Health Score (HJHS), number of target joints, and Hemophilia-Specific Quality of Life Index (Haemo-QoL) to evaluate joint outcomes. Factors associated with these outcomes were evaluated by statistical analysis.
(1) The children were 1.75 to 17 years age at prophylaxis initiation. Median prophylactic factor VIII dose was 22.9 IU/kg per week. (2) At the end of follow-up: (a) The total IPSG MRI scores were 2-24 with 90.5% children exhibiting moderate to severe joint involvement (score 7-24); (b) The HJHS ranged 2-27, with 0-10 for 46.7% children and >10 for 53.3% children. There was a positive correlation between the MRI score and HJHS (p < .05); (c) Compared to their on-demand treatment period before prophylaxis, target joints numbers decreased, and no child needed auxiliary devices to walk; (d) Joint outcomes were positively correlated with the age at initiation of low-dose prophylaxis (p < .05) and negatively correlated with the treatment dose.
Long-term low-dose prophylaxis had positive effect on joint outcomes compared with on-demand treatment. However, a certain degree of joint damage remained in all children indicating the need for improving the current strategy of low-dose prophylaxis.
探索中国重度血友病 A 患儿低剂量预防治疗的长期关节结局，并分析其相关因素。
我们回顾性分析了 21 例接受定期低剂量预防治疗 6-10 年的重度血友病 A 患儿的随访数据。我们使用国际预防治疗研究组磁共振成像评分（IPSG MRI 评分）、血友病关节健康评分（HJHS）、靶关节数和血友病特定生活质量指数（Haemo-QoL）评估关节结局。通过统计分析评估与这些结局相关的因素。
（1）患儿预防治疗开始时年龄为 1.75-17 岁，中位数预防用因子 VIII 剂量为 22.9 IU/kg/周。（2）随访结束时：（a）总 IPSG MRI 评分为 2-24 分，90.5%的患儿存在中重度关节受累（评分 7-24 分）；（b）HJHS 评分为 2-27 分，46.7%的患儿为 0-10 分，53.3%的患儿为>10 分，MRI 评分与 HJHS 呈正相关（p<0.05）；（c）与预防治疗前按需治疗期相比，靶关节数减少，无患儿需要辅助装置行走；（d）关节结局与低剂量预防治疗开始时的年龄呈正相关（p<0.05），与治疗剂量呈负相关。
与按需治疗相比，长期低剂量预防治疗对关节结局有积极影响。然而，所有患儿仍存在一定程度的关节损伤，这表明需要改进当前的低剂量预防治疗策略。
