Early Low-Dose Treatment Effective in Preventing Bleeding in Hemophilia A, Study Reports

Low-dose recombinant factor VIII lowered bleeding rates in Chinese children with hemophilia, and the earlier the preventive treatment began, the more effective it was in minimizing bleeding episodes, according to a new study.

The research, “The Efficacy of Recombinant FVIII Low-Dose Prophylaxis in Chinese Pediatric Patients With Severe Hemophilia A: A Retrospective Analysis From the ReCARE Study,” was published in the journal Clinical and Applied Thrombosis/Hemostasis.

Hemophilia A is unusually common in China, but costs and limited treatment availability often prevent patients from obtaining treatment. Earlier studies have suggested that treatment with low-dose recombinant factor VIII may be a solution in areas with limited healthcare resources.

A retrospective study, ReCARE (NCT02263066), was designed to assess treatment choices in Chinese children with hemophilia A. Researchers at Capital Medical University used data from the trial to investigate low-dose preventive treatment.

The study covered 57 children with a median age of 8.2 years. Three children received primary preventive treatment — therapy started before the second large joint bleed in those younger than 3.

Twenty-one patients received secondary treatment. It was administered to children after two or more joint bleeds but before a child developed joint disease, and to children older than 3. The final 32 patients received tertiary treatment, started after two bleeds or the onset of joint disease at any age.

Low-dose treatment lowered the annual bleeding rate in all three groups. The later the treatment started, the higher the rate, however.

In the three children with primary early treatment, the annual number of bleeds dropped to zero from four. Children with secondary treatment had an average number of eight bleeds before treatment, but the rate dropped to 1.9 after the therapy began. Those with tertiary treatment, who had an average rate of 20 before therapy, saw their bleeding rate reduced to 9.

Since only three patients were in the primary group, the research team compared results in the two remaining groups. They found that the later the treatment started, the higher the bleeding rate.

Researchers noted a similar pattern in annual rates of joint bleeds. Although the treatment lowered rates, the 5.1 annual rate in the tertiary group was higher than the 0.8 rate in the secondary group. The difference in rates was not statistically significant, however.

The length of treatment period could not explain the differences in annual bleeding rates, the researchers said. “[F]urther randomized prospective research is warranted to support this conclusion, including assessment of the joint structures and functions by assessment tools in a longer prophylactic (preventive-treatment) duration study cohort,” they concluded.