Prophylactic treatment is better than episodic or on-demand treatment for the prevention of bleeding in hemophilia, a new study published in PLOS One found. Moreover, the bleeding rate seemed to have a dose-response effect in patients with hemophilia A.
There are different prophylactic and episodic treatment options for hemophilia, and a better understanding of the effect of these treatments can help ensure patients are offered the right option.
To this aim, a team of researchers conducted a systemic review of the literature for randomized controlled trials comparing different factor replacement therapies in patients with hemophilia published up to December 2020.
They identified 9 randomized controlled trials. Of these, 6 compared prophylactic treatment with episodic treatment in patients with hemophilia A. The authors found that the annualized bleeding rate was lower in the low, intermediate, and high-dose prophylactic groups compared to episodic treatment groups.
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Moreover, the annualized joint bleeding rate was lower in all 3 prophylactic groups compared to the episodic treatment group.
However, none of the studies compared the different doses of prophylactic treatment and all results had a very low certainty of the evidence, the authors said. “Thus, future studies are needed to confirm these results and inform decision making.”
Hemophilia is a genetic disease characterized by clotting factor deficiency leading to spontaneous bleeding episodes. Hemophilia A is caused by a mutation in the gene encoding so-called factor VIII while hemophilia B is caused by a mutation in the gene encoding factor IX.
Treatment consists of clotting factor replacement therapy, which can be prophylactic or on-demand.
Delgado-Flores CJ, García-Gomero D, Salvador-Salvador S, Montes-Alvis J, Herrera-Cunti C, Taype-Rondan A. Effects of replacement therapies with clotting factors in patients with hemophilia: a systematic review and meta-analysis. PLOS One. 2022;17(1):e0262273. doi:10.1371/journal.pone.0262273