For a complete list of the members of the ESPRIT study group see Appendix 1.
A randomized clinical trial of prophylaxis in children with hemophilia A (the ESPRIT Study)
Article first published online: 4 APR 2011
© 2011 International Society on Thrombosis and Haemostasis
Journal of Thrombosis and Haemostasis
Volume 9, Issue 4, pages 700–710, April 2011
How to Cite
GRINGERI, A., LUNDIN, B., VON MACKENSEN, S., MANTOVANI, L., MANNUCCI, P. M. and THE ESPRIT STUDY GROUP (2011), A randomized clinical trial of prophylaxis in children with hemophilia A (the ESPRIT Study). Journal of Thrombosis and Haemostasis, 9: 700–710. doi: 10.1111/j.1538-7836.2011.04214.x
- Issue published online: 4 APR 2011
- Article first published online: 4 APR 2011
- Accepted manuscript online: 21 JAN 2011 03:32PM EST
- Received 20 August 2010, accepted 29 December 2010
- episodic treatment;
- hemophilia A;
- quality of life
Summary. 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 perception of well- 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-radiologic 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−1 3 × week) or episodic therapy with ≥25 IU kg−1 every 12–24 h 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 per patient per month (P < 0.02). Plain-film radiology showed signs of arthropathy in six 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 fewer joint bleeds (0.12 joint bleeds per patient per 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.