Bone Mineral Density in Haemophilila: A Treatment Outcome

Sammanfattning: Background: Osteoporosis is increasingly recognised as one of the major public health problems throughout the world and has massive socioeconomic implications. It has previously been shown that patients with severe haemophilia and not receiving any prophylactic treatment render a high risk of reduced bone density. Aims: To evaluate Bone Mineral Density (BMD) in patients with haemophilia of different severity types and treatment. To describe the type, intensity and duration of physical activity among adult patients with severe haemophilia. To assess levels of quality of life in severe haemophilia on long-term prophylaxis and to determine whether BMD is correlated to the patients’ perceived quality of life. The overall goal of this project is to evaluate the utility of BMD as an indicator for treatment quality. Methods: Thirty-eight adult patients with severe haemophilia: (Group A: patients who started prophylaxis at the age of ≤ 3 years; Group B: at the age of >3 years), 16 patients with mild haemophilia and 190 controls. BMD (BMD g/cm2) was measured by dual energy x-ray absorptiometry (DXA). Physical activity was assessed using the self-report Modifiable Activity Questionnaire (MAQ). Joints were evaluated according to the HJHS score. Quality of life was assessed using the self-report SF-36 Questionnaire. In Sweden, prophylaxis is usually started as primary prophylaxis at a very early age and continued indefinitively. Results: There was no significant difference in BMD at all measured sites between those with mild and severe haemophilia. Based on mean T-score both groups had normal BMD (T-score >-1). Patients with severe haemophilia divided into 2 groups (A and B): Group A have mean BMD T-score >-1.0 (i.e. normal score) at all measured sites, and Group B have mean BMD T-score <-1,0 at hip region, and >-1.0 at lumbar spine and total body. Moreover, patients in group A have significantly higher BMD at hip region compared to patients in group B. Time involved and intensity of all aspects of physical activity for Group A were almost similar to their healthy peers. Group B had significantly lower vigorous leisure and total physical activities than Group A and their healthy peers. Group A has almost similar scores in the SF-36 domains compared to the reference population. Group B scores in the SF-36 domains were lower compared to the reference population. Moreover, patients in group A have significantly higher scores of physical function (PF), social function (SF), and physical component summary (PCS) domains compared to patients in group B. In group B, significant correlations were found between BMD at femoral neck and total body with physical domains. Conclusions: The results indicate that adequate long-term prophylaxis since early childhood may preserve normal BMD and prevent or retards chronic joint disease, allowing patients to participate in physical activity and lead a life comparable to life of the general population.