Document Detail

Short-term low-dose secondary prophylaxis for severe/moderate haemophilia A children is beneficial to reduce bleed and improve daily activity, but there are obstacle in its execution: a multi-centre pilot study in China.
MedLine Citation:
PMID:  23231016     Owner:  NLM     Status:  In-Data-Review    
We recently showed in a single centre trial that low-dose secondary prophylaxis in severe/moderate haemophilia patients with arthropathy is feasible and beneficial. However, this regimen has not been validated in a multicentre setting and what obstacles are there to prophylaxis remain unclear. (i) Benefit study: to confirm the benefits of similar prophylaxis protocol in severe/moderate haemophilia A (HA) in a multicentre setting in China. (ii) Follow-up obstacle study: to investigate obstacles in compliance to prophylaxis treatment. (i) Benefit study: severe/moderate HA children with arthropathy from 15 centres were enrolled to undergo an 8-week on-demand treatment, followed by 6 to 12-week low-dose secondary prophylaxis. Outcomes compared in the two periods include joint and severe bleeding, daily activities and factor consumption. (ii) Obstacle study: questionnaires to investigators to collect data on patient and centre factors contributing to inability to comply with prophylaxis. We enrolled 191 patients from 15 centres. Sixty-six (34.6%) from three centres completed the prophylaxis protocol, and they had significantly decreased bleeding (78.8% haemarthrosis and 68.9% severe bleedings) and improved daily activities with no increase in factor consumption over that in the on-demand therapy period. The remaining 125 patients from 12 centres were not compliant to the prophylaxis protocol; questionnaire data indicated that the major obstacles were inability of patients/parents to accept (41.7%) or to adhere (33.3%) to the prophylaxis protocol, mostly because of failure to understand the benefits and to accept the frequent injections. Non-availability of a centre comprehensive care team was another important determinant. Short-term low-dose secondary prophylactic therapy is beneficial without increasing factors consumption for severe/moderate HA with arthropathy in a multi-centre setting in China. Obstacles to overcome must include improvement in comprehensive care and in education to patient/parents and healthcare personnel.
L Tang; R Wu; J Sun; X Zhang; X Feng; X Zhang; K-H Luke; M-C Poon
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Publication Detail:
Type:  Journal Article    
Journal Detail:
Title:  Haemophilia : the official journal of the World Federation of Hemophilia     Volume:  19     ISSN:  1365-2516     ISO Abbreviation:  Haemophilia     Publication Date:  2013 Jan 
Date Detail:
Created Date:  2012-12-12     Completed Date:  -     Revised Date:  -    
Medline Journal Info:
Nlm Unique ID:  9442916     Medline TA:  Haemophilia     Country:  England    
Other Details:
Languages:  eng     Pagination:  27-34     Citation Subset:  IM    
Copyright Information:
© 2012 Blackwell Publishing Ltd.
Hemophilia Work Group, Beijing Children's Hospital affiliated to Capital Medical University, Beijing, China.
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