title: 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.
abstract: 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.
authors.affiliation: Hemophilia Work Group, Beijing Children's Hospital affiliated to Capital Medical University, Beijing, China.
authors.forename: L
authors.initials: L
authors.lastname: Tang
authors.forename: R
authors.initials: R
authors.lastname: Wu
authors.forename: J
authors.initials: J
authors.lastname: Sun
authors.forename: X
authors.initials: X
authors.lastname: Zhang
authors.forename: X
authors.initials: X
authors.lastname: Feng
authors.forename: X
authors.initials: X
authors.lastname: Zhang
authors.forename: K-H
authors.initials: KH
authors.lastname: Luke
authors.forename: M-C
authors.initials: MC
authors.lastname: Poon
mesh_terms: D000203:Activities of Daily Living; D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D002681:China; D003029:Coagulants / Q000008:administration & dosage / Q000627:therapeutic use*; D005169:Factor VIII / Q000008:administration & dosage / Q000627:therapeutic use*; D005260:Female; D006395:Hemarthrosis / Q000517:prevention & control; D006467:Hemophilia A / Q000150:complications / Q000188:drug therapy*; D006470:Hemorrhage / Q000517:prevention & control*; D006801:Humans; D008297:Male; D010865:Pilot Projects
chemical_list: D003029:Coagulants; D005169:Factor VIII