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Regular prophylaxis with activated prothrombin complex concentrates in pediatric hemophilia
Ayumi Horiguchi1, Yuki Arakawa1, Yuichi Mitani1
1Department of Hematology/Oncology, Saitama Children's Medical Center, Saitama, Japan.
Summary
Regular prophylaxis with activated prothrombin complex concentrates (aPCCs) effectively reduced bleeding in children with hemophilia and inhibitors. While generally safe, one case of nephrotic syndrome highlights the need for careful monitoring.
Area of Science:
- Pediatric Hematology
- Hemophilia Treatment
- Coagulation Disorders
Background:
- Activated prothrombin complex concentrates (aPCCs) are effective for hemophilia prophylaxis in adults.
- Limited data exist on aPCCs prophylaxis in pediatric patients with hemophilia inhibitors.
Purpose of the Study:
- To evaluate the efficacy and safety of regular aPCCs prophylaxis in children with hemophilia and inhibitors.
- To assess the impact of aPCCs on annualized bleeding rates (ABR) in this pediatric population.
Main Methods:
- Single-center retrospective study including nine pediatric patients (<15 years) with severe/moderate hemophilia and inhibitors.
- Patients received prophylactic aPCCs (50-100 U/kg, 2-3 times/week).
- Medical records were reviewed to assess ABR and adverse events.
Main Results:
- Prophylactic aPCCs treatment (median 13 months) reduced the median ABR to 2 (range, 0-17).
- Four patients experienced a 19%-100% reduction in ABR compared to on-demand treatment.
- One patient developed nephrotic syndrome 34 months after initiating prophylaxis.
Conclusions:
- Regular aPCCs prophylaxis is effective in reducing ABR in younger children with hemophilia A and B.
- Nephrotic syndrome is a potential serious adverse event requiring careful monitoring during aPCCs therapy.
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