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

Gringeri, A; Lundin, Björn LU ; Mackensen, S V; Mantovani, L and Mannucci, P M (2011) In Journal of Thrombosis and Haemostasis 9. p.700-710
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... (More)
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. (Less)
Please use this url to cite or link to this publication:
author
organization
publishing date
type
Contribution to journal
publication status
published
subject
in
Journal of Thrombosis and Haemostasis
volume
9
pages
700 - 710
publisher
Federation of European Neuroscience Societies and Blackwell Publishing Ltd
external identifiers
  • wos:000289156800010
  • pmid:21255253
  • scopus:79953306825
ISSN
1538-7933
DOI
10.1111/j.1538-7836.2011.04214.x
language
English
LU publication?
yes
id
61b279b3-85a7-402b-a572-579521037508 (old id 1777129)
alternative location
http://www.ncbi.nlm.nih.gov/pubmed/21255253?dopt=Abstract
date added to LUP
2011-02-01 09:04:52
date last changed
2017-11-12 04:05:39
@article{61b279b3-85a7-402b-a572-579521037508,
  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 &lt;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&lt;0.02). Plain-film radiology showed signs of arthropathy in 6 patients on prophylaxis (29%) vs. 14 on episodic treatment (74%) (p&lt;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.},
  author       = {Gringeri, A and Lundin, Björn and Mackensen, S V and Mantovani, L and Mannucci, P M},
  issn         = {1538-7933},
  language     = {eng},
  pages        = {700--710},
  publisher    = {Federation of European Neuroscience Societies and Blackwell Publishing Ltd},
  series       = {Journal of Thrombosis and Haemostasis},
  title        = {A Randomized Clinical Trial of Prophylaxis in Children with Hemophilia A (the ESPRIT Study).},
  url          = {http://dx.doi.org/10.1111/j.1538-7836.2011.04214.x},
  volume       = {9},
  year         = {2011},
}