Bleeding rates with severe hem A fall with prophylaxis, study finds
Drop in annual rate of 91% seen at 4 years after switch from on-demand use
Prophylactic, or preventive, treatment with factor VIII (FVIII) replacement therapy for four years reduced annual bleeding rates by 91% for people with severe hemophilia A, a study from Taiwan reported.
After switching from on-demand treatment to prophylaxis, patients also experienced a 90.8% reduction in annual joint bleed rates and reported a significant improvement in quality of life.
Although costs increased with this switch, prophylactic treatment became more cost effective over time due to bleed prevention.
“This study provides valuable data for healthcare policymakers, highlighting the long-term benefits of prophylaxis as a preventative approach for managing bleeding and improving overall well-being in patients with severe [hemophilia A],” the researchers wrote.
Prophylactic replacement therapy aims to prevent bleeds, reduce their rates
The study, “Evaluating the Effectiveness of Prophylactic Strategies for Hemophilia A Management: A Real-World, Longitudinal Observational Study,” was published in Drugs – Real World Outcomes.
Hemophilia is caused by the lack or dysfunction of specific clotting factors, which are proteins that help form blood clots to prevent excessive bleeding. In hemophilia A, the missing clotting factor is FVIII.
The gold standard disease treatment is replacement therapy, in which a version of the missing clotting factor is administered to patients. Replacement therapy can be given prophylactically to prevent or reduce the frequency of bleeding episodes, or reactively to treat active bleeds on an on-demand basis.
The use of prophylactic or on-demand treatment and their specific regimens vary between countries based on differences in healthcare policies and economic considerations.
In Taiwan, intermediate-dose prophylaxis with standard half-life FVIII products for severe hemophilia A has been covered under the country’s national health insurance system since 2014. As of 2019, 73.4% of hemophilia patients received prophylactic therapy, but some still preferred on-demand treatment.
“There remains a lack of consensus regarding the cost effectiveness and quality of life of different treatment modalities in Taiwan. Therefore, pharmacoeconomic evaluations of severe hemophilia treatments are needed,” the researchers wrote.
The team examined data covering 35 males (six under age 18) with severe hemophilia A and without FVIII inhibitors — neutralizing antibodies that can render FVIII replacement therapies less effective — being treated at any of five hemophilia centers in Taiwan. All had switched from on-demand treatment to intermediate-dose prophylaxis, and were followed for a mean of 3.6 years.
One year after this switch, patients’ mean annual bleeding rate had decreased by 76.9%. Their mean annual joint bleeding rate similarly fell by 72.5% over the same period.
At four years after moving to prophylactic treatment, patients’ mean annual bleeding rate dropped by 91%, and their mean joint annual bleeding rate declined by 90.8%.
Prophylaxis seen as particularly beneficial for children
Researchers also assessed whether switching to prophylactic treatment altered clinical outcomes. Pediatric patients reported significant improvements in their daily life with the change to prophylactic treatment, while no significant gains were observed in adult patients.
“Prophylactic treatment initiated at a young age can have a larger beneficial effect and help maintain joint health as children grow into adulthood,” the researchers wrote.
Following the switch, patients also reported significant improvements in their overall quality of life.
In evaluating financial considerations, the researchers found that the average annual cost of FVIII replacement therapy rose by 41% in the first year of the move to prophylactic treatment, and it held steady in subsequent years.
But in its fourth year, intermediate-dose prophylaxis had averted an average of 28.6 annual bleeding episodes in pediatric patients and 24 in adults, compared with their bleed rates with on-demand therapy.
“When compared with the reactive treatment, prophylactic treatment was more expensive but more effective in preventing bleeding episodes,” the researchers wrote.
“This 4-year study provided compelling evidence for the long-term benefits of switching to intermediate-dose prophylaxis in severe [hemophilia A],” the team concluded.
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