The relative burden of haemophilia A and the impact of target joint development on health-related quality of life: results from the ADVATE Post-Authorization Safety Surveillance (PASS) study

Abstract

Studies with haemophilia A (HA) patients have shown burden in health-related quality of life (HRQOL) when compared with general population norms. In the current study, HA patients' SF-36v2 health survey scores were compared with general population norms and to patients with other chronic conditions. The impact of target joints (TJs) on HRQOL was also examined. The sample was a subset of HA patients enrolled in the Post-Authorization Safety Surveillance (PASS) programme: a prospective open-label study in which ADVATE [Antihaemophilic Factor (Recombinant), Plasma/Albumin-Free Method] was prescribed. A total of 205 patients who were ≥ 18 years old and had SF-36v2 baseline scores were selected for this study. To measure the burden of HA on HRQOL, manova analyses compared these SF-36v2 scores to age- and gender-matched general population US and EU norms and to patients from other chronic condition groups. manova and correlational analyses examined the relations among TJ, age and SF-36v2 scores. Comparisons with general population norms confirm that HA negatively impacts physical, but not mental, HRQOL. Comparison with other chronic conditions shows the physical burden of HA is greater than for chronic back pain but similar to diabetes and rheumatoid arthritis, while the mental burden of HA is less than for all three patient groups. The presence of TJs was negatively associated with physical HRQOL, although this association was much larger for older patients (45+ years) than for younger ones. Physical, but not mental, HRQOL is diminished in HA patients. Target joints are associated with lower physical HRQOL, although this effect is moderated by age.

Improvement in health‐related quality of life with recombinant factor IX prophylaxis in severe or moderately severe haemophilia B patients: results from the BAX326 Pivotal Study

“…The most plausible explanation is that these patients experience frequent bleeding episodes and have joint damage, as evidenced by a median annual bleed rate (ABR) of 14 bleeds per year and one target joints at baseline prior to receiving BAX326. Similar to the previous studies in haemophilia A and B [9,12,13,[18][19][20], our findings also showed that the burden is more pronounced with respect to the physical rather than the mental aspects of HRQoL.…”

Adherence to treatment regimen and bleeding rates in a prospective cohort of youth and young adults on low-dose daily prophylaxis for severe hemophilia A

“…Age was an important factor across all populations, as the PCS generally decreased over the years. In our study, mean MCS was unchanged at the end of the study (51.5 ± 9.45 compared to 52.4 ± 7.83) but still higher than the US general norms (47.7 ± 0.24) reported by Klamroth et al [29], whereas mean PCS was comparable to baseline (52.5 vs 49.8) and US general norms (50.7 ± 0.23).…”

“…This low variability could be explained by the fact that our subjects were already on a LDDP regimen at study entry, but also by the low number of subjects enrolled in our study. In the Advate PASS study, Klamroth et al assessed HRQoL in HA patients compared to healthy subjects, and to populations with other chronic diseases [29]. In their study, the PCS was reduced in HA (42.3 ± 0.77) compared to age-matched healthy subjects (54.5 ± 0.24) or those with chronic back pain (46.9 ± 0.4), but comparable to subjects with rheumatoid arthritis (43.6 ± 1.89).…”

Dental health and oral health‐related quality of life in children with congenital bleeding disorders

“…OHR-QoL also allows for a shift from traditional medical/dental criteria to assessment and care that focus on a person's social and emotional experience and physical functioning in defining appropriate treatment goals and outcome [2].By this measure, the discrepancy between the patient's wants, needs or demands, and what the patient has, will be determined. [3] Despite numerous reports on general health and quality of life in CBD patients [4][5][6][7][8], to our knowledge, few published studies have assessed the dental health as well as OHR-QoL in CBD, especially in children. [3,9].…”