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Twice Weekly Vs. Thrice Weekly Low-Dose Prophylactic Factor VIII Therapy in Children with Hemophilia A: An Open Label
Sunil Gomber1, Gorika Singhal1, Pooja Dewan1
1Department of Pediatrics, University College of Medical Sciences and Guru Teg Bahadur Hospital, Dilshad Garden, New Delhi, 110095, India.
Insights
Twice weekly low-dose factor VIII prophylaxis is as effective as thrice weekly for children with hemophilia A, significantly improving clinical and radiological scores. This finding supports a simpler, effective treatment schedule to prevent long-term complications.
Area of Science:
- Hematology
- Pediatrics
- Clinical Trials
Background:
- Hemophilia A is a common congenital coagulation disorder.
- Low-dose factor VIII prophylaxis is recommended in resource-poor settings to reduce bleeding and improve quality of life.
- Optimal prophylactic schedules for hemophilia A are not well-established.
Purpose of the Study:
- To compare the efficacy of twice-weekly versus thrice-weekly low-dose prophylactic factor VIII in children with hemophilia A.
- To evaluate the impact of different prophylactic schedules on bleeding rates and clinico-radiological outcomes.
Main Methods:
- A randomized controlled trial involving 33 children (≤18 years) with severe hemophilia A.
- Participants were randomized to receive either twice-weekly or thrice-weekly factor VIII prophylaxis (10 U/kg).
- Evaluations included bleeding rates, clinical scores (HJHS, FISH), and radiological scores (Pettersson, ultrasound) over 6 months to 1 year.
Main Results:
- No statistically significant difference in bleeding rates between the twice-weekly and thrice-weekly prophylaxis groups (median monthly bleeding rate 0.17, p=0.79).
- Both groups showed statistically significant improvements in initial versus final clinical and radiological scores.
- No significant difference in clinical or radiological scores was observed between the two groups.
Conclusions:
- Twice-weekly low-dose factor VIII prophylaxis is an effective and easily administered schedule for children with hemophilia A.
- This regimen can prevent long-term complications associated with hemophilia A.
- The findings support the use of a less frequent prophylactic schedule in clinical practice.
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.
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