Problems at work, limited engagement in recreational activities, and less relationship satisfaction contribute to a lower health-related quality of life (HQRoL) and greater pain severity, as reported by adults with hemophilia B, according to a study.
Findings also showed that anxiety and depression in caregivers of children with this disorder are associated with less success at work, and to the children’s own relationship and psychological problems.
The study, “Correlations between patient-reported outcomes and self-reported characteristics in adults with hemophilia B and caregivers of children with hemophilia B: analysis of the B-HERO-S study,” was published in the journal Patient Related Outcome Measures.
Hemophilia B is characterized by a missing or defective factor IX, a clotting protein. Besides spontaneous bleeding episodes, this disorder may be associated with pain, depression and anxiety, all greatly affecting the HRQoL of patients, their loved ones, and caregivers.
Patient-reported outcomes have been used in several studies to evaluate the impact of hemophilia on different aspects of HRQoL. They have included the five-level version of the EuroQol five-dimensional health status measure (EQ-5D-5L), 36-Item Short Form Survey (SF-36), Brief Pain Inventory (BPI), and the Hemophilia Activities List (HAL), which scores the difficulty of performing an activity across seven domains: lying/sitting/kneeling/standing, leg function, arm function, use of transportation, self-care, household tasks, and leisure activities or sports.
“However, the use of PROs in clinical management of patients with hemophilia is limited and inconsistent,” the investigators said.
Researchers here conducted a post-hoc analysis of the B-HERO-S study (NCT02568202), which assessed the impact of hemophilia B on HRQoL and other psychosocial aspects in adult patients and carers of young children with this disorder.
The research team from U.S.-based institutions, the National Hemophilia Foundation, and Novo Nordisk — which funded B-HERO-S — analyzed whether the scores of different PRO instruments correlated with self-reported characteristics in both groups.
This analysis was focused on two different online surveys: one given to 299 adults (213 men) with hemophilia B including four different PROs — EQ-5D-5L, BPI, HAL and the Patient Health Questionnaire (PHQ-9) of depression severity — and the other given to 150 caregivers of affected children (116 women), including the PHQ-9 and a measure of anxiety — the Generalized Anxiety Disorder 7-item.
Other questions related to participants’ demographics, hemophilia treatment, and psychosocial aspects were also included in both surveys.
Results showed that for adult patients — 63% with moderate and 25% with mild hemophilia B — higher pain severity and interference with other aspects of daily living (both assessed by BPI) were linked to work-related problems, reduced engagement in certain activities, problems in relationships with partners or prospective partners, as well as psychological and self-care/treatment issues.
Worse overall health (assessed by EQ-5D-5L) and decreased function (assessed by HAL) also had a negative impact on recreational activities, relationships and work, including not working due to complications from hemophilia and negative experiences with telling an employer or a manager.
In contrast, greater satisfaction with support at work or from a current partner or friends was associated with improved HRQoL, overall health, and less pain.
A greater impact on sexual health and negative experiences telling friends about hemophilia/being bullied because of the disorder correlated with worse overall health and increased pain.
Greater depression severity (assessed by PHQ-9) was linked to experiencing a stressful event and receiving psychological treatment in the past five years. Both greater depression and pain were linked to fewer visits to a hemophilia treatment center.
Data also showed that taking routine factor treatment was associated with better overall health and function and with less pain.
As for caregivers, higher depression and anxiety (assessed by GAD-7) were linked to being overlooked for promotion, not getting hired for a job, and not being able to work flexible hours.
Depression and anxiety in caregivers also correlated with concerns about treatment availability and affordability, stressful events, fewer visits to a hemophilia treatment center, and their child’s functional, relationship, and psychological problems.
Other links included negative experiences with telling somebody their child has hemophilia and the child’s own negative experience in such situations.
Of note, like the adult patients, most children with hemophilia B had moderate (56%) or mild (18%) disease.
Altogether, the results “revealed strong associations between questions related to the psychosocial impact of hemophilia and HRQoL domains, as assessed by PRO instruments in a largely mild-moderate hemophilia population,” the researchers wrote.
“Although these associations do not imply causality, … they do suggest avenues for further investigation and a need for further refining questions on hemophilia psychosocial impact to identify current issues that must be addressed to improve HRQoL,” they added.