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Low dose secondary prophylaxis reduces joint bleeding in severe and moderate haemophilic children: a pilot study in
1Hemophilia Work Group, Beijing Children's Hospital Affiliated to Capital Medical University, Beijing, China. runhuiwu@hotmail.com
Insights
Low dose secondary prophylaxis significantly reduced joint bleeds by 83% in children with haemophilia in China. This treatment improved joint function and quality of life, proving cost-effective for developing nations.
Area of Science:
- Pediatric Hematology
- Bleeding Disorders
- Public Health
Background:
- Joint bleeding and disability are common in children with haemophilia, especially with inadequate treatment.
- Developing countries face challenges in managing haemophilia due to economic and treatment limitations.
- Quality of life (QoL) is significantly impacted by joint complications in pediatric haemophilia.
Purpose of the Study:
- To evaluate the efficacy of low-dose secondary prophylaxis in children with haemophilia in China.
- To assess the impact of prophylaxis on joint bleeding frequency and joint function.
- To analyze improvements in QoL, including school attendance and daily activities.
Main Methods:
- A 12-week observation period followed by a 12-week low-dose secondary prophylaxis study.
- Participants: 34 children with moderate to severe haemophilia and established joint disease.
- Prophylaxis regimens: Factor VIII (10 IU/kg twice weekly) for haemophilia A, Factor IX (20 IU/kg weekly) for haemophilia B.
Main Results:
- Joint bleeds reduced by 83% (from 337 to 57) during the prophylaxis period.
- Joint function improved in 66.7% of affected joints, with 23.2% showing moderate to good improvement.
- School attendance, sports participation, and daily activities showed moderate improvements.
Conclusions:
- Low-dose secondary prophylaxis is effective in reducing joint bleeding frequency in pediatric haemophilia.
- The treatment leads to moderate improvements in joint function and overall QoL.
- This approach is a cost-effective strategy for managing haemophilia in developing countries like China.
Abstract:
The most common bleeding in haemophilic patients is in joints, and joint disability is the most common complications in these patients receiving inadequate treatment. Limited by economy and inadequate treatment, developing countries face huge challenge to reduce disability and improve quality of life (QoL) of haemophilic children. The aim of this study was to investigate the effect of low dose secondary prophylaxis in China. Children with moderate and severe haemophilia from the Beijing Children Hospital, Beijing, China, and with established joint disease, were followed for a 12-week observation period followed by a 12-week low dose secondary prophylaxis-study period (for haemophilia A, factor VIII concentrate 10 IU kg(-1) twice weekly; for haemophilia B, factor IX concentrate 20 IU kg(-1) weekly). The reduction of joint bleeding, improvement of joint function and QoL during prophylaxis were analysed. In total 34 children (median age 7.8 years) were analyzable. The number of joint bleeds decreased from a total of 337 (individual range 3-24, mean 9.9) during the observation period to 57 (range 0-6, mean 1.7) during the study period with an overall of reduction 83%. Joint function improved in 66.7% of disease joints, with 23.2% of which were considered good to moderate. School attendance improved in all subjects, sports participation and daily activity improved moderately. Low dose secondary prophylaxis significantly reduces frequency of joint bleeding; with moderate improvement in joint function, school attendance, sport participation and daily activities. Low dose secondary prophylaxis is therefore, cost-effective as applied to developing countries such as China, although there are still unresolved issues.
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