Health-related quality of life in children with haemophilia in China: a 4-year follow-up prospective cohort study

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.

Analysis of the Clinical Factors Affecting the Quality of Life in Children with Hemophilia A

“…Repeated joint bleeding causes severe joint damage and pain that causes disability (4). Regular visits to the hospital, repeated injections, and activity restrictions are common disorders in hemophiliacs in addition to feelings of anxiety about recurrent bleeding and the risk of permanent disability that will affect the quality of life (5). The fact that until now there is no definitive cure for hemophilia and lifelong therapy is required, assessment of the patient's quality of life becomes an important parameter (6,7).…”

Health‐related quality of life and caregiver burden of emicizumab in children with haemophilia A and factor VIII inhibitors—Results from the HAVEN 2 study

“…[1][2][3] A clinical hallmark of severe HA is recurrent spontaneous bleeds, particularly into joints, and a substantial corresponding impact on physical health and HRQoL. [4][5][6][7][8] Approximately 30% of previously untreated persons with haemophilia A (PwHA) develop one of the most challenging complications of haemophilia treatment: neutralizing alloantibodies (inhibitors) against FVIII, [9][10][11] preventing effective FVIII prophylaxis. 12,13 Standard treatment for PwHA with FVIII inhibitors is bypassing agents (BPAs); however, their haemostatic effects are suboptimal and unpredictable, and their use is further burdened by the need for frequent intravenous injections over prolonged periods of time.…”

“…Haemophilia A (HA), a congenital bleeding disorder characterized by deficiency of coagulation protein factor (F)VIII, has a negative impact on the health‐related quality of life (HRQoL) of affected people 1‐3 . A clinical hallmark of severe HA is recurrent spontaneous bleeds, particularly into joints, and a substantial corresponding impact on physical health and HRQoL 4‐8 …”

Health-Related Quality of Life of Patients With Haemophilia - A Cross-Sectional Survey in the Northeast of China

“…In recent years, the HRQoL of haemophilia patients has attracted increasing attention from the research community [8,33]. The EQ-5D is perhaps the most widely used instruments for assessing HRQoL in patients with various disease conditions [12].…”