不同替代疗法对 a 型严重血友病青少年关节结局的长期影响：一项中国前瞻性队列研究的中期结果

Aims: To explore the long-term effect of different replacement therapies on preventing joint lesions in adolescent patients with severe hemophilia A. And to analyze the correlation among the evaluation indicators.

Methods:We prospectively analyzed long-term regular follow-up data from 49 adolescent patients with severe hemophilia A and their 119 target observation joints, who were divided into on-demand group (n=6), low-dose prophylaxis group (n=17), and intermediate-dose prophylaxis group (n=26). Patients were enrolled from March 2017 to August 2019. The outcome indicators included clinical bleeding phenotype (ABR, AJBR, AOJBR), joint structure (HEAD-US, IPSG-MRI), joint function (HJHS) and quality of life (CHO-KLAT).

Results: The median age of patients in the three groups was 7.78 (6.15-10.66) years, and there were no significant differences in the age of first joint hemorrhage, activity of basic factors, body weight, BMI and other indicators among the three groups. The median follow-up time was 43.13 (27.79-48.03) months. The clinical bleeding phenotype in the on-demand group showed an overall upward or slow downward trend over time, while those in the low and intermediate-dose prophylaxis group showed a significant downward trend. Long-term intermediate-dose prophylaxis can significantly delay even probably reverse the changes of joint structure. Long-term low and intermediate-dose prophylaxis can reverse joint function damage. The quality of life of the three groups was improved compared with the baseline, and the improvement degree of the low-dose prophylaxis group was the highest. There was a low degree of positive correlation between clinical bleeding phenotype and joint structure and function score, there was a significant positive correlation between joint structure and function score.

Conclusions: We advocate that Chinese adolescent patients with severe hemophilia A receive intermediate-dose prophylaxis treatment for a long time, and use comprehensive evaluation methods to monitor joint lesions and treatment effects.

No relevant conflicts of interest to declare.