Verma S P, Dutta T K, Mahadevan S, Nalini P, Basu D, Biswal N, Ramesh A, Charles D, Vinod K V, Harichandra Kumar K T
Clinical Haematology, Jawaharlal Institute of Post Graduate Medical Education and Research (JIPMER), Puducherry, India.
Medicine, Division of Clinical Haematology, Jawaharlal Institute of Post Graduate Medical Education and Research (JIPMER), Puducherry, India.
Haemophilia. 2016 May;22(3):342-8. doi: 10.1111/hae.12838. Epub 2016 Mar 14.
Current factor prophylaxis strategy practised in developed countries is not feasible in resource constraint developing countries like India.
The aim of this study was to investigate the efficacy and safety of very low-dose factor prophylaxis in India.
Children of 1-10 years of age with severe haemophilia were randomized to Prophylaxis group and Episodic (On demand) group. Children in prophylaxis group received very low-dose factor VIII (FVIII) concentrate, i.e. 10 units kg(-1) body weights on 2 days a week. Episodic group received factor concentrate in standard recommended doses. The study period was 11.5 months.
In total 21 children were enrolled in this study, 11 assigned to prophylaxis and 10 to episodic group. Children on prophylaxis had 11 joint bleeds in comparison to 57 joint bleeds in episodic group. Mean number of haemarthrosis per patient per month were 0.08 (0.08 ± 0.13) in prophylaxis group compared to 0.48 (0.48 ± 0.34) in episodic group (P < 0.05). Total FVIII consumption was 87.51 and 56.32 units kg(-1) month(-1) in prophylaxis and episodic group respectively (P = ns). Overall median hospital emergency visits were 1 day in prophylaxis group and 9 days in episodic group (P ≤ 0.05). Median days of absenteeism from school were 25 days in episodic group and 3 days in prophylaxis group (P < 0.05). No significant complications were noted in prophylaxis group and compliance was 98%.
To conclude, low-dose FVIII prophylaxis is cost effective, efficacious and a safe method of preventing joint bleeds and consequent joint damages.
发达国家目前实行的因子预防策略在像印度这样资源有限的发展中国家是不可行的。
本研究的目的是调查在印度采用极低剂量因子预防的疗效和安全性。
将1至10岁的重度血友病儿童随机分为预防组和按需治疗组。预防组儿童每周2天接受极低剂量的凝血因子VIII(FVIII)浓缩剂,即每公斤体重10单位。按需治疗组接受标准推荐剂量的因子浓缩剂。研究期为11.5个月。
本研究共纳入21名儿童,11名被分配到预防组,10名被分配到按需治疗组。预防组儿童发生11次关节出血,而按需治疗组为57次关节出血。预防组每名患者每月的平均关节积血次数为0.08(0.08±0.13),按需治疗组为0.48(0.48±0.34)(P<0.05)。预防组和按需治疗组的FVIII总消耗量分别为每月每公斤体重87.51和56.32单位(P=无显著差异)。预防组总的医院急诊就诊中位数为1天,按需治疗组为9天(P≤0.05)。按需治疗组的缺课天数中位数为25天,预防组为3天(P<0.05)。预防组未观察到明显并发症,依从性为98%。
总之,低剂量FVIII预防是一种经济有效、安全的预防关节出血及后续关节损伤的方法。