HEAD-US-C超声评估量表对中间型/重型血友病A患者按需和预防替代治疗关节损伤评价

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. CONCLUSION: 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.