INTRODUCTION Low dose factor VIII prophylactic therapy in hemophilia has not been well established till date. This randomized controlled trial compared the efficacy of twice vs. thrice weekly schedule of low dose prophylactic factor VIII in children with hemophilia A as evaluated by the bleeding rate and clinico-radiological evaluation. METHODS Thirty-three children with severe hemophilia A (≤18 years) were randomized into two groups. Baseline evaluation included detailed history, clinical (HJHS 2.1 score and FISH score) and radiological examination (Pettersson score and ultrasound score). Group 1 received twice weekly factor VIII prophylaxis while group 2 received thrice weekly factor VIII prophylaxis, the dose being 10 U/kg in both groups. All participants were followed up over next 6 months to one year. Data regarding acute bleeding episodes and repeat clinico-radiological assessment at the end of follow up were recorded. RESULTS We analyzed 14 children in twice weekly prophylaxis group and 16 children in thrice weekly prophylaxis group. Statistically insignificant difference was found between the bleeding rates in both the groups after prophylaxis with the median values of monthly bleeding rate being 0.17 and p-value of 0.79. The differences between the initial and final clinical and radiological scores within each group were found to be statistically significant. There was no significant difference in the clinical and radiological scores in between the groups. CONCLUSION Twice weekly FVIII therapy is effective, easily administered prophylactic schedule to prevent long-term complications of hemophilia A. Lay summaryHemophilia A is one of the most common congenital coagulation factor deficiencies. Low dose factor VIII prophylaxis is recommended for hemophilia in resource-poor settings to reduce the bleeding episodes and improve the quality of life, although the optimal schedule for the same has not been well established. A randomized controlled trial on 33 children with hemophilia A (≤18 years) was done to compare the efficacy of twice versus thrice weekly schedule of prophylactic factor VIII. Group 1 received twice weekly factor VIII prophylaxis while group 2 received thrice weekly factor VIII prophylaxis, the dose in both groups being 10 U/kg. They were evaluated by the bleeding rate and clinical (HJHS 2.1 score and FISH score) and radiological scores (Pettersson score and ultrasound score). All participants were followed up over next 6 months to one year. Data regarding acute bleeding episodes and repeat clinico-radiological assessment at end of follow up were recorded. When analyzed, statistically insignificant difference was found between the bleeding rates after the two prophylaxis regimes. There was a significant improvement between initial and final clinical and radiological scores in both the groups and no difference was recorded in between the groups. To conclude, twice weekly FVIII therapy is effective, easily administered prophylactic schedule to prevent long-term complications of hemophilia A.

中文翻译：

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每周两次 A 型血友病儿童每周三次低剂量预防性因子 VIII 治疗：一项开放标签随机试验。

引言 迄今为止，血友病中的低剂量因子 VIII 预防性治疗尚未得到很好的确立。这项随机对照试验比较了 A 型血友病儿童每周两次和三次低剂量预防性因子 VIII 的疗效，通过出血率和临床放射学评估进行评估。方法 33 名患有严重血友病 A（≤18 岁）的儿童被随机分为两组。基线评估包括详细病史、临床（HJHS 2.1 评分和 FISH 评分）和放射检查（Pettersson 评分和超声评分）。第 1 组每周接受两次因子 VIII 预防，而第 2 组每周接受三次因子 VIII 预防，两组的剂量均为 10 U/kg。在接下来的 6 个月至一年内对所有参与者进行了随访。记录有关急性出血事件和随访结束时重复临床放射学评估的数据。结果 我们分析了每周两次预防组中的 14 名儿童和每周三次预防组中的 16 名儿童。预防后两组的出血率差异无统计学意义，月出血率的中位数为 0.17，p 值为 0.79。发现每组内初始和最终临床和放射学评分之间的差异具有统计学意义。组间临床和放射学评分无显着差异。结论 每周两次 FVIII 治疗是有效的、易于实施的预防性时间表，可预防血友病 A 的长期并发症。总结总结血友病 A 是最常见的先天性凝血因子缺乏症之一。对于资源贫乏地区的血友病，建议使用低剂量因子 VIII 预防，以减少出血事件并提高生活质量，尽管尚未确定最佳方案。对 33 名 A 型血友病儿童（≤18 岁）进行了一项随机对照试验，以比较每周两次和三次预防性因子 VIII 的疗效。第 1 组每周接受两次因子 VIII 预防，而第 2 组每周接受三次因子 VIII 预防，两组的剂量均为 10 U/kg。他们通过出血率和临床（HJHS 2.1 评分和 FISH 评分）和放射学评分（Pettersson 评分和超声评分）进行评估。在接下来的 6 个月至一年内对所有参与者进行了随访。记录有关急性出血事件和随访结束时重复临床放射学评估的数据。分析时，发现两种预防方案后出血率之间的差异无统计学意义。两组的初始和最终临床和放射学评分之间都有显着改善，两组之间没有差异。总而言之，每周两次 FVIII 治疗是有效的、易于实施的预防性时间表，可预防血友病 A 的长期并发症。发现两种预防方案后出血率之间的差异无统计学意义。两组的初始和最终临床和放射学评分之间都有显着改善，两组之间没有差异。总而言之，每周两次 FVIII 治疗是有效的、易于实施的预防性时间表，可预防血友病 A 的长期并发症。发现两种预防方案后出血率之间的差异无统计学意义。两组的初始和最终临床和放射学评分之间都有显着改善，两组之间没有差异。总而言之，每周两次 FVIII 治疗是有效的、易于实施的预防性时间表，可预防血友病 A 的长期并发症。