Determining meaningful health‐related quality‐of‐life improvement in persons with haemophilia A using the Haemophilia Quality of Life Questionnaire for Adults (Haem‐A‐QoL)

Abstract

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The effect of emicizumab prophylaxis on long‐term, self‐reported physical health in persons with haemophilia A without factor VIII inhibitors in the HAVEN 3 and HAVEN 4 studies

“…The Haem‐A‐QoL is a frequently utilized haemophilia‐specific instrument for the evaluation of HRQoL in PwHA 19 , 39 and use of this instrument is a strength of this study. Because of the high impact of HA on physical activities, the PH domain is an important facet of the Haem‐A‐QoL when measuring HA interference with HRQoL.…”

“…Haem‐A‐QoL, EQ‐5D‐5L, and missed workdays were also analysed by subgroups based on prior treatment, presence of target joints at baseline, 25 24‐week bleeding rate (a randomization stratification factor used in HAVEN 3), treated annualized bleed rate (ABR; during the first 24 weeks or at discontinuation if < 24 weeks), treated joint ABR (AJBR; during the first 24 weeks or at discontinuation if < 24 weeks) and dosing regimen. The proportions of participants at each timepoint with improvement larger than the responder threshold 18 , 19 were calculated for the Haem‐A‐QoL PH and TS. Only those participants with data both at baseline and the timepoint of interest are included in score change calculations.…”

“…The HAVEN studies found overall improvements in HRQoL through 25 weeks with emicizumab prophylaxis in adolescent and adult PwHA with or without FVIII inhibitors, 12,13,15 as measured by the Haemophilia‐Specific Quality of Life Questionnaire for Adults (Haem‐A‐QoL) and Adolescents (Haemo‐QoL‐SF) 16,17 . The ‘Physical Health’ (PH) domain reflects the detrimental impact of HA on physical health status; a 10‐point reduction in PH is the threshold for clinically meaningful improvement in the physical health of PwHA 18,19 . In HAVEN 3, the mean (standard deviation [SD]) change in PH scores from baseline to Week 25 was –12.2 (26.78) and –15.2 (19.52) in PwHA taking emicizumab QW and Q2W, compared to +1.9 (22.50) without prophylaxis.…”

“… 16 , 17 The ‘Physical Health’ (PH) domain reflects the detrimental impact of HA on physical health status; a 10‐point reduction in PH is the threshold for clinically meaningful improvement in the physical health of PwHA. 18 , 19 In HAVEN 3, the mean (standard deviation [SD]) change in PH scores from baseline to Week 25 was –12.2 (26.78) and –15.2 (19.52) in PwHA taking emicizumab QW and Q2W, compared to +1.9 (22.50) without prophylaxis. In HAVEN 4, PH scores changed –15.1 (21.91) from baseline to Week 25 of Q4W emicizumab.…”

Health‐related quality of life and health status in adolescent and adult people with haemophilia A without factor VIII inhibitors—A non‐interventional study

“…The Haem‐A‐QoL is a frequently utilized instrument for evaluating HRQoL in adult PwH, as it specifically addresses haemophilia‐related factors 25,26 . Because of the high impact of haemophilia on a person's physical activities, the ‘Physical Health’ domain is a particularly important facet of the Haem‐A‐QoL when measuring haemophilia interference with HRQoL; a 10‐point reduction in this domain is considered a threshold for notable improvement in HRQoL in PwH 27 .…”