A randomized clinical trial of prophylaxis in children with hemophilia A (the ESPRIT study)

Alessandro Gringeri, B. Lundin, S. Von Mackensen, L. Mantovani, P. M. Mannucci, A. Billio, E. Boeri, G. Castaman, N. Ciavarella, G. Mancuso, M. Morfini, M. G. Mazzucconi, R. Musso, M. Muça Perja, S. Riva, E. Santagostino, A. Rocino, F. A. Scaraggi, M. Schiavoni, G. TagarielloA. Tagliaferri

Research output: Contribution to journalArticlepeer-review

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 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 factorVIII (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 childrenwere 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). Plainfilm 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.

Original languageEnglish
Pages (from-to)700-710
Number of pages11
JournalJournal of Thrombosis and Haemostasis
Volume9
Issue number4
DOIs
Publication statusPublished - 2011

Keywords

  • Episodic treatment
  • Haemo-QoL
  • Hemarthrosis
  • Hemophilia A
  • Prophylaxis
  • Quality of life

ASJC Scopus subject areas

  • Hematology

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