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) Selleck IBET762 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. Haemophilia is an X-linked recessive hereditary disease. The main clinical manifestation of haemophilia is recurrent bleeding into joints, resulting in severe disability, poor attendance to school, work or other social activities. Prophylaxis has demonstrated click here effectiveness in preventing haemarthrosis and is recommended as the management of choice for severe haemophilia children by the World Health Organization (WHO) and World Federation of Hemophilia (WFH; ). learn more Primary prophylaxis aims to maintain perfect joint status. Secondary prophylaxis initiated after occurrence of joint disability, aims to maintain the basic joint activity and function . Currently, the standard prophylaxis formula is to use moderate-full dose, such as 20–40 IU kg−1 once to three times per week for HA . There are critical barriers in carrying
out treatments, particularly prophylaxis. These include factor concentrates shortage and cost, especially in the developing countries like China. Due to these barriers, haemophilia children can hardly get treatment and have a high rate of disability . Given the problem of inadequate concentrate support and the presence of unsatisfactory joint status, treatment goal in developing country has to be compromised from ‘having perfect joint’ to ‘keeping basic ability of daily living’. With this treatment goal, low-dose secondary prophylaxis is for the time being, the preventative management strategy of choice in developing countries. We have previously demonstrated feasibility and benefit of low-dose (10 IU kg−1 twice weekly) secondary prophylaxis for HA children with arthropathy in a single Beijing centre study . The objective of this clinical trial is to confirm that a similar short-term low-dose secondary prophylaxis for a similar haemophilia population remains feasible and beneficial when carried out at multiple centres in different areas of China.