Impact of severe haemophilia A on patients' health status: results from the guardian^™ 1 clinical trial of turoctocog alfa (NovoEight^®)

Abstract

Summary Haemophilia and its treatment interfere with patients' life and may affect adherence to treatment. This study explored the impact of severe haemophilia A on patients' health status, especially in young adults (YA), using data from guardian™ 1, a multinational, open‐label, non‐controlled phase 3 trial investigating safety and efficacy of turoctocog alfa (NovoEight®) in previously treated patients aged 12 years and older with severe haemophilia A (FVIII ≤ 1%). Health status was assessed using the EuroQoL‐5 dimensions (EQ‐5D‐3L), covering 5 dimensions of health (mobility, self‐care, usual activities, pain/discomfort and anxiety/depression), and a visual analogue scale (VAS) measuring self‐rated overall health status. EQ‐5D was administered pretreatment (screening/baseline) and posttreatment (end‐of‐trial). Baseline responses to the EQ‐5D dimensions and VAS were described overall and by age and compared to reference values from UK general population. Guardian™ 1 included 150 patients (16 adolescents, 83 YA aged 16–29 and 51 adults aged 30+). All five dimensions of patients' health status were impacted at baseline. The percentage of haemophilia patients reporting problems was consistently significantly greater than age‐matched general population reference values. Likewise, for all age groups mean baseline EQ‐5D VAS score was significantly lower for haemophilia patients (YA: 78.0) than for the general population (YA aged 18–29: 87.3). The health status of patients with severe haemophilia A entering guardian™ 1 was markedly poorer than that of the general population, particularly regarding mobility and pain. YA patients reported better health status than older patients, but considerably lower than that of the general YA population.

Mental health disorders in haemophilia: Systematic literature review and meta‐analysis

“…While most studies investigated depression alone, six studies reported anxiety and depression without separating those affected by depression, anxiety or both . All six studies used the EuroQoL five dimension scale (EQ5D), and therefore, this resulted in low statistical heterogeneity among studies ( I 2 = 0%, P = .60).…”

“…Our search strategy identified 2315 studies, of which 28 studies met our eligibility criteria (Figure 1). Twenty‐seven studies had a cross‐sectional design, and one study was a retrospective chart review (Table 1). None of the studies were prospective in nature.…”

Turoctocog alfa in the treatment of individuals with hemophilia A: review of quality of life data collected in Phase III trials

“…For the full analysis set of the trial as well as for the adults aged 30-60 years, the change from baseline in EQ-5D VAS did not differ according to the treatment regimen received within the 12 months preceding the trial [33]. In the young adults, as indicated in Figure 1, the baseline EQ-5D VAS was comparable between the group who had received prophylaxis and the group who had received on-demand prior to the trial; mean change in EQ-5D VAS from baseline to the end-of-treatment visit showed an improvement (no information on statistical significance available) for both groups, however the improvement was greater in the individuals previously treated with on-demand regimen before the trial (5.2 point improvement, SD = 10.3) than individuals previously treated with prophylaxis (1.2 point improvement, SD = 18.5).…”

“…guardian 1 enabled the health status of young adults aged 16-29 years to be compared with both the full analysis set of the trial, and to the gender and agespecific reference values from the UK general population using data from the EQ-5D. Young adults (16-29 years, n = 83) and older adults (30-60 years, n = 51) with hemophilia A reported more problems in all EQ-5D domains and in particular for the Mobility and Pain/Discomfort domains than young adults and adults in the respective reference populations [33,34]. For all EQ-5D domains, adults 30-60 years of age reported more problems than young adults, supporting the degenerative nature of hemophilia.…”

Joint Mobility and Physical Function of Danish Hemophilia Patients: A Three-Wave Panel Study Spanning 24 Years

“…The most common manifestations of hemophilia are joint bleeds, leading to painful arthritis, limited joint mobility, and impaired physical functioning. As a result, PWH experience a reduced quality of life (QOL) [3-7]. …”