Prophylactic Therapy Achieving Zero Annual Bleed Rates Gives Hemophilia Patients Normal Quality Of Life

Date: Feb-16-2013Children with hemophilia A achieving annual bleed rates of zero on prophylactic therapy have similar quality of life and physical health to the healthy population, according to a study reported at the 6th Annual Congress of the European Association for Hemophilia and Allied Disorders (6-8 February 2013; Warsaw, Poland).

The multinational online survey collected information from parents or other caregivers for 470 children with severe hemophilia A (Factor VIII levels Children with an annual bleed rate of zero had similarly high quality of life scores to healthy population norms (mean total PedsQL scores of 81.08 vs 83.84). They reported good joint function, with 96% having full range of joint motion. Two-thirds (65%) of the children had no joint pain.

"Efforts to maintain a zero annual bleed rate in children with severe hemophilia A may help ensure optimal outcomes," said the research group, led by Josh Epstein, from Baxter Healthcare Corporation, Westlake Village, California, USA. They recommended individualised prophylactic therapy to increase the number of patients free from bleeds.

"An annual bleed rate of zero is our aim for prophylactic treatment of hemophilia A," commented Dr Elena Santagostino, Head of the Angelo Bianchi Bonomi Hemophilia and Thrombosis Centre, Milan, Italy. She noted that preventing bleeding into joints is essential to stop the development of the disabling arthropathy that occurs with repeated bleeds that occur without prophylactic therapy in patients with hemophilia.

"Annual bleed rate is a key clinical metric for the optimal management of people with hemophilia, enabling the efficacy of their treatment to be evaluated and tailored to individual needs," said Dr Santagostino. She considered that a personalised approach to hemophilia management, including monitoring annual bleed rate, may help to prevent, reduce or even eliminate bleeding episodes.

Dr Pedro Pina, Medical Director Hemophilia, Baxter Healthcare, Zurich, Switzerland, noted that a recent study showed that a personalised regimen with Advate (recombinant FVIII therapy processed without any blood-based additives), in which dosage was tailored to each patient's pharmacokinetic profile for factor VIII, achieved the same efficacy as standard prophylaxis with fixed dosing every two days.

Individualised dosing had the advantage of reducing the number of weekly infusions. Nearly half (42%) of patients treated prophylactically with Advate experienced zero bleeds.

He said that a phase II/III study with BAX 855, a recombinant factor VIII with a longer half life than Advate, has recently started recruiting to investigate its use as prophylaxis in hemophilia A.

Susan Mayor PhD, freelance medical journalist, London, UK

Susan Mayor's attendance at EAHAD was funded by Baxter

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