Abstract
BackgroundHealth-related quality of life (HRQoL) has been brought up for decades in haemophilia patients. However, no data to date are available about HRQoL in children with haemophilia using long-term follow up data. This nearly 4-year follow-up study aimed to assess the long-term HRQoL of haemophilia children.MethodsA prospective cohort study among 42 children with haemophilia and their parents was conducted in August 2014 in a children’s hospital; follow-up was completed in January 2018. Primary endpoint was the change in patient HRQoL evaluated by Canadian Haemophilia Outcomes–Kids’ Life Assessment Tool (CHO-KLAT) from baseline to year 4; secondary endpoint was the impact of bleeding rates, physical activity restriction, financial burden and treatment (prophylaxis vs on-demand treatment) on HRQoL, as well as the impact of treatment on event-free survival.ResultsTotally 42 patients (mean age, 5.48[SD, 4.63] years) and 42 parents were included. 38 families completed 4-year follow up. Patients reported a small increase in HRQoL from baseline to year 4. The mean scores of child self-report and parent proxy report of CHO-KLAT at baseline were 60.69 (SD = 20.28) and 61.01 (SD = 12.14), respectively. Scores at follow-up were 64.69 (SD = 13.71) and 65.33 (SD = 15.78), respectively. Haemophilia patients without physical activity restriction, living in urban areas, and receiving prophylactic treatment and home injection, had higher average values for HRQoL scores than the others. Bleeding rates were proportionally negatively correlated with HRQoL. Patients who had received prophylactic treatment had better event-free survival.ConclusionsHaemophilia decreased HRQoL of patients, but this effect weakened after 4 years. HRQoL of children is influenced by severity of haemophilia, bleeding rates, physical activity restriction, financial burden and treatment. Prophylactic treatment is a key factor contributing to event-free survivor prognosis and the optimal form of therapy for childhood haemophilia.
Highlights
Haemophilia, characterized by a bleeding disorder which primarily affects boys, is caused by an inherited deficiency of factor VIII or factor IX [1] and is associated with someZhang et al Health and Quality of Life Outcomes (2019) 17:28 severe joint damage and pain, leading to disability [5]
This study demonstrated that haemophilia decreased Health-related quality of life (HRQoL) of patients, but this effect weakened at year 4 compared with the baseline, the primary outcome
Prophylactic treatment was a key factor contributing to event-free survivor prognosis and an optimal therapy for haemophilia patients
Summary
Haemophilia, characterized by a bleeding disorder which primarily affects boys, is caused by an inherited deficiency of factor VIII (haemophilia A) or factor IX (haemophilia B) [1] and is associated with someZhang et al Health and Quality of Life Outcomes (2019) 17:28 severe joint damage and pain, leading to disability [5]. Injections and limited (sports) participation are common impairments for haemophilia patients, most of whom worry about bleeding, the need for invasive procedures (like blood tests and intravenous therapy) and the risk of permanent disability. This disease and its treatment impact patients’ health-related quality of life (HRQoL). No data to date are available about HRQoL in children with haemophilia using long-term follow up data This nearly 4-year follow-up study aimed to assess the long-term HRQoL of haemophilia children
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