Treatment-related knowledge and skills of patients with haemophilia and their informal caregivers

Background Replacement therapy in haemophilia plays an important role in the effective management of this rare bleeding disorder and requires a high level of knowledge and practical skills. Objective To evaluate and to compare the knowledge and skills about the medicines and their management among people with haemophilia treated with clotting factor concentrates, and their informal caregivers. Setting Eight Hospital Pharmacies working closely with Haemophilia Care Centres in France. Method In this cross-sectional and multi-centre study, 26-item questionnaire was specifically developed to assess the knowledge and skills. Face-to-face interviews using a questionnaire were conducted with patients and caregivers. Main outcome measure Level of knowledge and skills about disease, treatment and medication management. Results This study included 80 patients and 55 caregivers. Although most interviewees had basic knowledge of the treatment, only 43.7 % knew the effect of clotting factor concentrates on the haemostasis process. Similarly, only 12.7 % of the patients and 30.9 % of the caregivers referred to inhibitors as adverse effects. Despite intensive training for home treatment, 55.7 % reported difficulties with reconstitution or injection. The knowledge required for the responsible management of their medications had not been totally acquired: only 17 participants were indeed familiar with the medication storage conditions; 29 patients and 9 caregivers had already experienced an emergency which they had to treat with no medicine available at home; and 47.4 % of participants had already thrown away an unused drug vial. Conclusion This study shows the need of a regular update and to renew awareness of haemophilia treatment specificities among these populations. The identified needs suggest that we should more invest in educational techniques or therapeutic education programs more focused on medication management.