摘要
目的研究中国重型血友病A成人患者短期足量预防治疗下的疗效特点。方法对既往按需或低剂量预防治疗的13例重型血友病A成人患者进行关节评估(靶关节超声、HJHS关节评分),回顾性收集按需或低剂量预防治疗时患者的年出血情况,然后前瞻性观察短期足量预防治疗下患者出血情况及关节评估状况的变化,同时调查患者平时活动强度(IPAQ短问卷),测量足量预防治疗下患者72 h FVIII:C谷浓度。结果 13位重型血友病A成人患者中位年龄26.0(20.5～29.0)岁,接受短期足量预防治疗的中位剂量31.0(29.1～33.0)U/kg,3次/周,72 h FVⅢ:C谷浓度1.7%(1.3%～3.4%)。随访3月期间,所有患者的年化出血次数、年化关节出血次数较既往明显减少(P=0.001,P=0.001),但仅有4人(30.8%)实现了"零出血",7人(53.8%)实现了关节"零出血",仍有9人(69.2%)存在突破性出血。关节超声与HJHS评分评估6人(46.2%)靶关节损害程度较前加重,7人(53.8%)则无明显进展。相比于关节未进展组,关节进展组患者可能存在关节基线状态较严重、随访前及随访期间的出血次数较高、体力活动强度较高、而FVIII:C的基线活性较低等状况偏差。结论目前短期足量预防治疗虽可以明显减少出血及部分阻止关节损害的进展,但尚无法实现所有中国重型血友病A成人患者的"零出血"目标,亦无法完全阻止其关节的进一步损害。对于不同临床出血表型、关节状态及体力活动强度的成人患者,可能需要更多评估方法的个体化治疗方案及必要的理疗和手术干预。
Objective To investigate the efficacy of short-term full-dose prophylaxis in adult Chinese patients with severe hemophilia A. Methods Thirteen adult Chinese patients with severe hemophilia A receiving on-demand or low-dose prophylaxis underwent ultrasound examination of the target joints and evaluation of Hemophilia Joint Health Score（HJHS）.The data of annual bleeding episodes in the period of on-demand or low-dose prophylaxis were collected retrospectively from the patients, and the changes in bleeding and joint condition（ultrasound findings of the target joints and HJHS） were observed during short-term full-dose prophylaxis. The activity intensity of the patients was assessed using the IPAQ questionnaire, and the 72 h FVIII trough activity was measured during full-dose prophylaxis. Results The median age of the 13 patients was 26.0（20.5-29.0） years. For full-dose prophylaxis, the patients received a median therapeutic dose of 31.0（29.1-33.0） IU/kg,administered for 3 times per week; the median 72 h FVIII trough activity of patients was 1.7%（1.3-3.4%）. During the follow-up period for 3 months, the annual bleeding rates（ABR） and annual joint bleeding rates（AJBR） decreased significantly in all the patients（P=0.001 and 0.001, respectively）, but zero bleeding was achieved in only 4 patients（30.8%） and zero joint bleeding in7 patients（53.8%）; 9 patients（69.2%） still experienced breakthrough bleeding. The damage severity of target joints assessed by ultrasound and HJHS in 6 patients（46.2%）was worse than before and no obvious progression of target joints damage was found in 7 patients（53.8%）. Compared with the patients without progression, the patients with worsened joint damage had poorer baseline joint condition, higher bleeding frequencies before and during the follow-up, a higher intensity of physical activity, and a lower baseline FVIII activity. Conclusion At present, although short-term full-dose prophylaxis can significantly reduce the bleeding and partially pr
作者
孙雪岩
庄金木
周璇
李惠萍
柳竹琴
孙竞
SUN Xueyan;ZHUANG Jinmu;ZHOU Xuan;LI Huiping;LIU Zhuqin;SUN Jing(Department of Hematology,Nanfang Hospital,Southern Medical University,Guangzhou,510515,China)
出处
《南方医科大学学报》
CAS
CSCD
北大核心
2018年第10期1222-1227,共6页
Journal of Southern Medical University
基金
南方医科大学临床重点研究项目(LC2016ZD016)
关键词
足量预防治疗
成人
重型血友病A
full-dose prophylaxis
severe hemophilia A
adult patients
