Impact of prophylaxis on health‐related quality of life of boys with hemophilia: An analysis of pooled data from 9 countries

Abstract

Background Prophylaxis reduces the frequency of bleeds in boys with severe hemophilia and is the standard care for their management in resource‐abundant countries. The effect of prophylaxis on Health‐Related Quality of Life (HRQoL) has not been established, because the sample sizes of most studies are too small to explore the relationship of multiple factors that influence HRQoL. Methods The aim of this study was to assess the impact of hemophilia severity and treatment regimen on HRQoL and to establish the minimum important difference (MID) using the international level of score distributions. HRQoL data were pooled from 7 studies across 9 countries. HRQoL was measured using the Canadian Hemophilia Outcomes–Kids’ Life Assessment Tool (CHO‐KLAT). A mixed‐effect linear regression analysis was employed to assess the impact of prophylaxis on the CHO‐KLAT score. Results Data from 401 boys with hemophilia were analyzed (57.6% severe hemophilia and 57.6% receiving prophylaxis). The model revealed that receiving prophylaxis was significantly associated with higher HRQoL (regression coefficient 8.5, 95% confidence interval [CI] 3.9‐13.1). Boys with severe hemophilia had a significantly lower HRQoL as compared to boys with moderate and mild hemophilia whose CHO‐KLAT scores were 7.0 and 6.6 points higher, respectively. There was a significant interaction between treatment and disease severity (P = 0.023), indicating prophylaxis has the most significant impact in boys with severe hemophilia. Based on these pooled data, the MID of the CHO‐KLAT was established at 6.5. Conclusions This study confirms the positive effect of prophylaxis on HRQoL in boys with hemophilia in a real‐world setting and provides initial benchmarks for interpreting HRQoL scores based on use of the CHO‐KLAT instrument.

Measuring the impact of changing from standard half‐life (SHL) to extended half‐life (EHL) FVIII prophylaxis on health‐related quality of life (HRQoL) in boys with moderate/severe haemophilia A: Lessons learned with the CHO‐KLAT tool

“…No relationship was found between bleed rates postswitch and changes in CHO‐KLAT scores. In fact, the 10 boys who showed a reduction in their AJBR showed a small but not statistically or clinically significant reduction in mean CHO‐KLAT scores from 78.6 preswitch to 73.9 postswitch ( P = .15), while parent proxy‐reported CHO‐KLAT scores fell slightly from 81.4 preswitch to 79.9 postswitch ( P = .71).…”

Preferences and health utilities related to disease and treatment features for patients with hemophilia A in Canada

“…71.7 (13.1), respectively), whereas increased disease severity was associated with signi cantly lower HRQoL compared to mild or moderate disease. 41 Mean CHO-KLAT score (SD) of Canadian boys was 75.0 (13.0). 41 A CUA comparing primary prophylactic treatment with on-demand treatment in Canadian boys with severe hemophilia A, found that HRQoL and clinical outcomes are substantially improved with prophylaxis compared to on-demand treatment, but it comes at a substantial incremental cost per QALY.…”

“…41 Mean CHO-KLAT score (SD) of Canadian boys was 75.0 (13.0). 41 A CUA comparing primary prophylactic treatment with on-demand treatment in Canadian boys with severe hemophilia A, found that HRQoL and clinical outcomes are substantially improved with prophylaxis compared to on-demand treatment, but it comes at a substantial incremental cost per QALY. 42 In a prior study it was not found that prophylactic treatment improved HRQoL, but there were several limitations noted, including not taking into account the impact of frequent intravenous infusions on HRQoL.…”

Emerging benefits of Fc fusion technology in the context of recombinant factor VIII replacement therapy

“…Prophylactic factor replacement is a well-established standard of care for patients with severe haemophilia A, 1,2 and routine prophylaxis with factor VIII (FVIII) has been shown to reduce the frequency of bleed episodes, prevent joint damage and improve health-related quality of life. 3,4 However, optimal prophylaxis with conventional FVIII products requires frequent intravenous injections, a substantial burden that can negatively impact treatment adherence. 5 Several strategies have been employed to extend the half-life of FVIII in recombinant FVIII (rFVIII) products, thereby reducing the frequency of infusions.…”