HEAD-US-C quantitative ultrasound assessment scale in evaluation of joint damage in patients with moderate or severe hemophilia A received on-demand versus prophylaxis replacement therapy / 中华血液学杂志

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.

Clinical application and optimization of HEAD-US quantitative ultrasound assessment scale for hemophilic arthropathy / 中华血液学杂志

Objective: To assess the feasibility of HEAD-US scale in the clinical application of hemophilic arthropathy (HA) and propose an optimized ultrasound scoring system. Methods: From July 2015 to August 2017, 1 035 joints ultrasonographic examinations were performed in 91 patients. Melchiorre, HEAD-US (Hemophilic Early Arthropathy Detection with UltraSound) and HEAD-US-C (HEAD-US in China) scale scores were used respectively to analyze the results. The correlations between three ultrasound scales and Hemophilia Joint Health Scores (HJHS) were evaluated. The sensitivity differences of the above Ultrasonic scoring systems in evaluation of HA were compared. Results: All the 91 patients were male, with median age of 16 (4-55) years old, including 86 cases of hemophilia A and 5 cases hemophilia B. The median (P25, P75) of Melchiorre, HEAD-US and HEAD-US-C scores of 1 035 joints were 2(0,6), 1(0,5) and 2(0,6), respectively, and the correlation coefficients compared with HJHS was 0.747, 0.762 and 0.765 respectively, with statistical significance (P<0.001). The positive rates of Melchiorre, HEAD-US-C and HEAD-US scale score were 63.0% (95%CI 59.7%-65.9%), 59.5% (95%CI 56.5%-62.4%) and 56.6% (95%CI 53.6%-59.6%) respectively, and the difference was statistically significant (P<0.001). Even for 336 cases of asymptomatic joints, the positive rates of Melchiorre, HEAD-US-C and HEAD-US scale score were 25.0% (95%CI 20.6%-29.6%), 17.0% (95%CI 12.6%-21.1%) and 11.9% (95%CI 8.4%-15.7%) respectively, and the difference was statistically significant (P<0.001). There were significant changes (P<0.05) in the ultrasonographic score of HA before and after onset of hemorrhage in 107 joints of 40 patients. The difference in variation amplitude of HEAD-US-C scores and HEAD-US scores before and after joint bleeding was statistically significant (P<0.001). Conclusion: Compared with Melchiorre, there were similar good correlations between HEAD-US, HEAD-US-C and HJHS. HEAD-US ultrasound scoring system is quick, convenient and simple to use. The optimized HEAD-US-C scale score is more sensitive than HEAD-US, especially for patients with HA who have subclinical state, which make up for insufficiency of sensitivity in HEAD-US scoring system.

Clinical significance of anticoagulant proteins detection in patients with thrombotic events / 中华血液学杂志

<p><b>OBJECTIVE</b>To investigate the prevalence and the risk of natural anticoagulants such as plasma protein C (PC), protein S (PS) and antithrombin (AT) deficiency in thromboembolic patients with no evident acquired factors.</p><p><b>METHODS</b>Clotting assays on French STAGO autoanalyzer were used to detect the activity of plasma PC, PS and AT in 85 patients with thrombotic disease and 50 sex and age matched healthy controls.</p><p><b>RESULTS</b>Among the 85 enrolled patients (18 arterial and 67 venous thromboembolism), male to female ratio was 1.4 and the median age was 42 years (17-69). The activity of plasma PC, PS and AT in the pre-therapy thrombotic disease group, the thrombo-recurrence group, and the age < or = 45 years group were significantly lower than that is the healthy control group, the first thrombotic episodes group and the age > 45 years group respectively (P < 0.001, P < 0.01, P < 0.01). The overall deficiency rate of these three natural anticoagulants was 30.6%, PS deficiency was the commonest (10.6%), the second was PC deficiency (8.2%), AT deficiency and combined deficiency each accounted for 5.9%.</p><p><b>CONCLUSION</b>The PC, PS and AT protein deficiencies are frequent in Chinese thromboembolic patients, they are the independent risk factors for the thrombotic events and recurrence.</p>