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<sup>-1</sup> 3 × week) or episodic therapy with ≥ 25 IU kg<sup>-1</sup> 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

Gringeri, A., Lundin, B., Von Mackensen, S., Mantovani, L., Mannucci, P., Esprit, (2011). A randomized clinical trial of prophylaxis in children with hemophilia A (the ESPRIT study). JOURNAL OF THROMBOSIS AND HAEMOSTASIS, 9(4), 700-710 [10.1111/j.1538-7836.2011.04214.x].

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

MANTOVANI, LORENZO GIOVANNI
Penultimo
;
2011

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
Articolo in rivista - Articolo scientifico
Health Services Research; Episodic treatment; Haemo-QoL; Hemarthrosis; Hemophilia A; Prophylaxis; Quality of life; Child; Child, Preschool; Hemophilia A; Humans; Infant; Joint Diseases; Hematology,
English
2011
9
4
700
710
open
Gringeri, A., Lundin, B., Von Mackensen, S., Mantovani, L., Mannucci, P., Esprit, (2011). A randomized clinical trial of prophylaxis in children with hemophilia A (the ESPRIT study). JOURNAL OF THROMBOSIS AND HAEMOSTASIS, 9(4), 700-710 [10.1111/j.1538-7836.2011.04214.x].
File in questo prodotto:
File Dimensione Formato  
2011_JTH_esprit.pdf

accesso aperto

Dimensione 432.24 kB
Formato Adobe PDF
432.24 kB Adobe PDF Visualizza/Apri

I documenti in IRIS sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.

Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/10281/101445
Citazioni
  • Scopus 380
  • ???jsp.display-item.citation.isi??? 366
Social impact