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The overall effectiveness of prophylaxis in severe haemophilia
J Panicker1, I Warrier, R Thomas
1Department of Paediatrics and Paediatric Haematology-Oncology, Children's Hospital of Michigan, Beaubien Boulevard, Detroit, MI, USA.
Insights
Prophylaxis significantly reduces bleeding episodes, emergency room visits, and hospitalizations in children with haemophilia compared to on-demand treatment. This approach improves long-term health outcomes and quality of life.
Area of Science:
- Hematology
- Pediatric Medicine
- Pharmacology
Background:
- Hemophilia is a genetic bleeding disorder requiring lifelong management.
- On-demand treatment for hemophilia can lead to chronic joint damage and reduced quality of life.
- Prophylaxis aims to prevent bleeding episodes and long-term complications.
Purpose of the Study:
- To compare the effectiveness of prophylactic factor replacement therapy versus on-demand treatment in children with hemophilia.
- To evaluate changes in bleeding episodes, joint health, and healthcare utilization.
Main Methods:
- Retrospective review of 25 children with severe hemophilia A or B.
- Comparison of outcomes (factor usage, bleeds, ER visits, hospitalizations) during prophylaxis versus on-demand therapy.
- Analysis of primary and secondary prophylaxis regimens.
Main Results:
- Prophylaxis significantly reduced mean annual major bleeds from 15.5 to 1.9.
- Significant decreases were observed in target joints, emergency room visits, and hospitalizations.
- Despite increased factor usage, prophylaxis led to fewer bleeds and reduced healthcare utilization.
Conclusions:
- Prophylaxis is effective in reducing bleeding frequency and severity in children with hemophilia.
- Preventing new target joint bleeds through prophylaxis improves long-term morbidity and quality of life.
- Prophylactic therapy offers a better long-term outlook for pediatric hemophilia patients.
Abstract:
The aim of this retrospective review was to assess the overall effectiveness of prophylaxis when compared with on-demand treatment of haemophilic patients. Twenty-five children (22 with severe haemophilia A and three with severe haemophilia B) were evaluated. Five haemophilia A patients received primary prophylaxis (instituted before the onset of any joint bleed) while the other 17 haemophilia A and all three haemophilia B patients were on secondary prophylaxis. We compared factor usage, number of bleeding episodes, emergency room (ER) visits and hospitalizations while on prophylaxis to those while on demand therapy. All subjects were male, the median age at time of review was 11.4 years and at start of prophylaxis was 4.5 years. Thirteen of the 25 patients (52%) required indwelling venous catheters for access, seven of these had one or more (one-six) episodes of line sepsis. Haemophilia A patients received an average of 23.8 U kg(-1) (20-30 U kg(-1)) of recombinant factor VIII three times a week while haemophilia B patients received 50 U kg(-1) recombinant FIX twice weekly. There was a significant reduction in the mean number of major bleeds on prophylaxis from 15.5 to 1.9 per year and a significant decrease in target joints, ER visits and hospitalizations. Although factor usage per year was higher on prophylaxis, there was an overall reduction in number of bleeds and resultant decrease in hospitalizations and ER visits. By preventing new target joints, prophylaxis can lead to reduction in long-term morbidity and a better quality of life despite increased central lines and higher factor usage.