Objective: To explore the evaluation of joint injury by HEAD-US-C (Hemophilic Early Arthropathy Detection with UltraSound in China, HEAD-US-C) in patients with moderate or severe hemophilia A treated with prophylaxis vs on-demand. Methods: The patients from June 2015 to July 2017 with moderate or severe hemophilia A were examined by ultrasound imaging of the elbows, knees and ankles; Meanwhile the HEAD-US-C ultrasound assessment scale and hemophilia joint health score scale 2.1 (HJHS2.1) were used to score the joint status. The correlation between the HEAD-US-C and HJHS score was performed in prophylaxis group and on-demand group patients, respectively. Results: A total of 925 cases of joint ultrasonography were conducted in 70 patients with moderate or severe hemophilia A. Among patients with moderate hemophilia, the median (IQR) of HEAD-US-C score and HJHS score in on-demand group were significantly higher than those in the prophylaxis group[1 (0, 6) vs 0.5 (0, 3) , z=0.177, P=0.046],[2 (0, 4) vs 2 (0, 3) z=0.375, P=0.007], even though there was no significant difference of the median (IQR) number of annualized target joints bleeding episodes between on-demand and prophylaxis groups[1 (0, 7) vs 1 (0, 5) , z=1.271, P=0.137]. Unlike in moderate cases, on-demand treatment group had more annualized target joints bleeding episodes than prophylaxis group among patients with severe hemophilia[3 (0, 8) vs 2 (0, 8) , z=0.780 P=0.037]. The prophylaxis group compared favorably with on-demand therapy group in terms of HEAD-US-C score[1 (0, 6) vs 4 (0, 7) , z=2.189, P=0.008], and HJHS score[2 (0, 5) , 4 (1, 6) , z=3646, P<0.001]for the severe hemophilia patients. The positive correlation between HEAD-US-C score and HJHS score was identified (P<0.05) , whether on-demand treatment or prophylaxis groups. The correlation coefficient between HEAD-US-C score and HJHS score in on-demand treatment and prophylaxis groups were 0.739 (95% CI 0.708-0.708) , 0.865 (95% CI 0.848-0.848) respectively, and 95% CI didn't overlap (P<0.05) , indicating that the correlation coefficient in prophylaxis group had stronger correlation than that in on-demand group. Conclusions: Clinical effects of prophylaxis were significantly better than those of on-demand treatment in patients with moderate or se-vere haemophilia A. HEAD-US-C scoring system could effectively evaluate joints damage in hemophilia A patients treated with on-demand or prophylaxis, companied by significantly positive correlation with HJHS clinical evaluation system, and provided objective index for clinical effect assessment.
目的： 探讨血友病关节超声评估量表（Haemophilic Early Arthropathy Detection with UltraSound in China，HEAD-US-C）对中间型/重型血友病A患者按需、预防治疗关节损伤评估的适用性。 方法： 回顾性分析2015年6月至2017年7月70例接受肘、膝、踝关节超声检查的中间型及重型血友病A患者，应用HEAD-US-C超声评估量表及血友病关节健康评分量表2.1版（HJHS2.1）进行关节状况评分。对按需、预防治疗患者HEAD-US-C、HJHS评分进行相关性分析。 结果： 70例中间型及重型血友病A患者共接受919例次关节超声检查。在中间型血友病患者中，按需、预防治疗组患者中位年靶关节出血次数差异无统计学意义[1（0，7）对1（0，5），z=1.271，P=0.137]。按需治疗组中位HEAD-US-C评分[1（0，6）对0.5（0，3），z=0.177，P=0.046]及HJHS评分[2（0，4）对2（0，3），z=0.375，P=0.007]明显高于预防治疗组。重型血友病患者按需、预防治疗组中位HEAD-US-C评分分别为4（0，7）、1（0，6）（z=2.189，P=0.008），中位HJHS评分分别为4（1，6）、2（0，5）（z=3.646，P<0.001），年靶关节出血次数分别为3（0，8）、2（0，8）（z=0.780，P=0.037），按需治疗组均高于预防治疗组。按需、预防治疗组患者HEAD-US-C评分与HJHS评分均存在正相关关系（P值均<0.05）。重型患者按需、预防治疗组HEAD-US-C评分与HJHS评分的相关系数分别为0.739（95%CI 0.708~0.767）、0.865（95%CI 0.848~0.880），95%CI不重合（P<0.05），预防治疗组两评分系统间具有更强的相关性。 结论： 中间型/重型血友病A患者预防治疗疗效明显优于按需治疗。HEAD-US-C超声评估量表可有效评估中间型/重型血友病A患者按需、预防治疗关节损伤状况，与HJHS系统一致性较好，可为临床疗效评估提供客观指标。.
Keywords: Hemophilia A; Joint disease; Replacement therapy; Scoring system; Ultrasonography.