Development of ‘Diagnosis and Management of von Willebrand Disease’ CE supplement

From: News-Medical.net

An unrestricted grant from Octapharma USA supported the development of a Continuing Education (CE) initiative entitled the “The Diagnosis and Management of von Willebrand Disease,” a program of the National Home Infusion Association (NHIA). The CE supplement was recently published in NHIA’s INFUSION magazine and is available online at www.nhia.org.

Octapharma USA is an affiliate of Octapharma AG, one of the largest human protein products manufacturers in the world. The U.S. Food and Drug Administration (FDA) has granted Octapharma orphan drug exclusivity for wilate® (von Willebrand Factor/Factor VIII Concentrate, Human), the first replacement therapy developed specifically for von Willebrand Disease (VWD).

A Randomized Clinical Trial of Prophylaxis in Children with Hemophilia A (the ESPRIT Study).

From: PubMed.gov

J Thromb Haemost. 2011 Jan 21. doi: 10.1111/j.1538-7836.2011.04214.x. [Epub ahead of print]

Gringeri A, Lundin B, Mackensen SV, Mantovani L, Mannucci PM; and the ESPRIT Study Group.

Department of Medicine and Medical Specialities, Fondazione IRCCS Cà Granda, Ospedale Maggiore Policlinico and University of Milan, Milan, Italy Department of Radiology, University Hospital of Lund, Lund, Sweden Institute of Medical Psychology, University Medical Centre Hamburg-Eppendorf, Hamburg, Germany Centre of Pharmacoeconomics, University Federico II of Naples, Naples, Italy Scientific Direction, Fondazione IRCCS Cà Granda, Ospedale Maggiore Policlinico and University of Milan, Milan, Italy.

Abstract

Background: Prevention of arthropathy is a major goal of hemophilia treatment. While studies in adults have demonstrated an impact of prophylaxis on the incidence of joint bleeds and patients’ well-being in terms of improved quality of life (QoL), it is unclear whether or not prophylaxis influences the outcome and well-being perception of children with hemophilia.

Objective: This randomized controlled study compared the efficacy of prophylaxis with episodic therapy in preventing hemarthroses and image-proven joint damage in children with severe hemophilia A (factor VIII <1%) over a 10-year time period.

Methods: Forty-five children with severe hemophilia A, aged 1-7 years (median 4), with negative clinical-radiological joint score at entry and at least one bleed during the previous 6 months, were consecutively randomized to prophylaxis with recombinant factor VIII (25 IU/Kg 3x week) or episodic therapy with ≥25 IU/Kg every 12-24 hours until complete clinical bleeding resolution. Safety, feasibility, direct costs and QoL were also evaluated.

Results: Twenty-one children were assigned to prophylaxis, 19 to episodic treatment. Children on prophylaxis had fewer hemarthroses than children on episodic therapy: 0.20 vs. 0.52 events/patient/month (p<0.02). Plain-film radiology showed signs of arthropathy in 6 patients on prophylaxis (29%) vs. 14 on episodic treatment (74%) (p<0.05). Prophylaxis was more effective when started early (≤36 months) with patients having less joint bleeds (0.12 joint bleeds/patient/month) and no radiologic signs of arthropathy.

Conclusion: This randomized trial confirms the efficacy of prophylaxis in preventing bleeds and arthropathy in children with hemophilia, particularly when it is initiated early in life.

© 2011 International Society on Thrombosis and Haemostasis