重型血友病A成人短期足量预防治疗的疗效

Efficacy of Short-Term Adequate Preventive Therapy in Adults with Severe Hemophilia A

目的:探讨重型血友病A成人患者短期足量预防治疗的疗效。方法:回顾性分析30例血友病A成人患者的临床及随访资料,记录患者预防治疗前3个月的年化出血次数(ABR)、年化关节出血次数(AJBR)、血友病关节健康评分(HJHS)及关节超声评分情况,然后前瞻性观察患者在短期足量预防治疗3个月后的ABR、AJBR、HJHS评分及超声评分情况。结果:所有受试者进行短期足量预防治疗时的ABR、AJBR明显少于按需或低剂量预防治疗时患者的出血次数(P<0.05)。但本研究30例患者中,仅有10例(33.33%)可达到"零出血",12例(40.00%)实现关节"零出血",16例(53.33%)仍存在突破性出血;14例患者(46.67%)靶关节损害程度较前加重,16例患者(53.33%)无明显进展,无1例患者的靶关节改善。结论:重型血友病A成人短期足量预防治疗虽然可以在不同程度上降低出血情况,防止部分关节进行性损坏,但仍然无法实现零出血和完全改善关节破坏。建议急性出血按需治疗后可继续进行短期足量预防治疗,减少出血次数,有条件者可进行长期足量预防治疗。

Objective: To explore the efficacy of short-term adequate preventive therapy in adult patients with severe hemophilia A. Methods: 30 adult hemophilia A patients were selected as research objects, and the annual number of bleeding(ABR), annual number of joint bleeding(AJBR), hemophilia joint health score(HJHS) and joint ultrasound score were recorded 3 months before prevention and treatment. Then, the ABR, AJBR, HJHS scores and ultrasound scores of the patients were prospectively observed after 3 months of short-term adequate preventive treatment. Results: ABR and AJBR in all subjects were significantly less than the bleeding frequency of patients with on-demand or low-dose prophylaxis(P<0.05). In the 30 patients, only 10 cases(33.33%) achieved "zero bleeding", 12 cases(40.00%) achieved joint "zero bleeding", and 16 cases(53.33%) still had breakthrough bleeding;14 patients(46.67%) had aggravated target joint damage, while 16 patients(53.33%) had no significant progress, and target joint improvement was not observed in 1 patient. Conclusion: Short-term adequate preventive treatment for severe hemophilia A in adults can reduce bleeding and prevent partial joint damage to varying degrees, but it is still unable to achieve zero bleeding and completely improve joint damage.