Measurement properties of the Haem‐A‐QoL in haemophilia clinical trials

Mackensen, Sylvia von; Eldar-Lissai, Adi; Auguste, Priscilla; Krishnan, Sangeeta; Maltzahn, Robyn von; Yu, R.; Wyrwich, Kathleen W.

doi:10.1111/hae.13140

Cited by 33 publications

(52 citation statements)

References 22 publications

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“…The strengths of this study include the use of psychometrically validated, haemophilia‐specific and age‐appropriate HRQoL instruments . The inclusion of overall health status measures that are not disease‐specific provides a valuable complement to the disease‐specific outcomes.…”

Section: Discussionmentioning

confidence: 99%

“…The strengths of this study include the use of psychometrically validated, haemophilia-specific and age-appropriate HRQoL instruments. 33,43 The inclusion of overall health status measures that are not disease-specific provides a valuable complement to the disease-specific outcomes. The separate analyses for participants on different previous treatment regimens are also a strength, providing insights on how switching to emicizumab prophylaxis affected outcomes for participants with different treatment histories.…”

Section: Discussionmentioning

confidence: 99%

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The effect of emicizumab prophylaxis on health‐related outcomes in persons with haemophilia A with inhibitors: HAVEN 1 Study

et al. 2018

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Introduction Persons with haemophilia A (PwHA) with inhibitors to factor VIII often experience decreased health‐related outcomes. In HAVEN 1 (NCT02622321), there was a statistically significant reduction in bleeding with emicizumab prophylaxis versus no prophylaxis. Aim Describe health‐related outcomes in PwHA with inhibitors in HAVEN 1. Methods PwHA with inhibitors aged ≥12 years previously on episodic bypassing agents (BPAs) were randomized to emicizumab prophylaxis (Arm A; n = 35) or no prophylaxis (Arm B; n = 18); participants previously on BPA prophylaxis received emicizumab prophylaxis (Arm C; n = 49). Health‐related outcomes assessed at baseline and monthly thereafter: Haemophilia Quality of Life Questionnaire for Adults (Haem‐A‐QoL), Haemophilia‐specific Quality of Life Questionnaire for Children Short Form (Haemo‐QoL SF), EuroQol 5‐Dimensions 5‐Levels (EQ‐5D‐5L) index utility score (IUS) and visual analogue scale (EQ‐VAS) and work/school days. Days hospitalized also recorded. Results At week 25, differences (ANCOVA) in adjusted mean scores (95% confidence interval) favoured Arm A versus B for Haem‐A‐QoL “Total” score (14.0 [5.6, 22.5]; P = 0.002) and “Physical Health” (21.6 [7.9, 35.2]; P = 0.003); EQ‐VAS (−9.7 [−17.6, −1.82]; P = 0.017); and IUS (−0.16 [−0.25, −0.07]; P = 0.001); mean scores are comparable in Arms A and C. Throughout the study, a greater proportion of participants on emicizumab prophylaxis than no prophylaxis exceeded questionnaire‐specific responder thresholds. Mean proportion of missed work days and number of days hospitalized were lower with emicizumab prophylaxis than no prophylaxis. Conclusions In PwHA with inhibitors, emicizumab prophylaxis was associated with substantial and meaningful improvements in health‐related outcomes.

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Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

The effect of emicizumab prophylaxis on health‐related outcomes in persons with haemophilia A with inhibitors: HAVEN 1 Study

et al. 2018

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“…The Haem‐A‐QoL is a validated measure designed to assess aspects of HRQoL in adult PwHA aged ≥18 years, consisting of 46 items in 10 domains (“Physical Health,” “Feelings,” “View of Yourself,” “Sports & Leisure,” “Work & School,” “Dealing With Haemophilia,” “Treatment,” “Future,” “Family Planning” and “Partnership & Sexuality”), which create a “Total” score when combined. Responses, based on participants' experience in the previous 4 weeks, could range from “never” (1) to “all of the time” (5) (5‐point Likert scale).…”

Section: Methodsmentioning

confidence: 99%

Health‐related quality of life and health status in persons with haemophilia A with inhibitors: A prospective, multicentre, non‐interventional study (NIS)

Mahlangu

Oldenburg²,

Callaghan

et al. 2019

Haemophilia

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Introduction Real‐world data (RWD) on health‐related outcomes in persons with haemophilia A (PwHA) provide insights into patient needs and can guide clinical study design. A global, prospective, non‐interventional study (NIS; NCT02476942) collected detailed RWD on bleeding outcomes, health‐related quality of life (HRQoL) and health status in PwHA treated per local routine clinical practice. Aim To report HRQoL and health status in the adult/adolescent PwHA with inhibitors cohort in the NIS. Methods This cohort enrolled PwHA aged ≥12 years with high‐titre factor VIII inhibitor history. Participants remained on their usual treatment (no protocol‐specified interventions). Health‐related outcomes: Haemophilia Quality of Life Questionnaire for Adults (Haem‐A‐QoL), Haemophilia‐specific Quality of Life Questionnaire for Children Short Form (Haemo‐QoL SF), EuroQol 5‐Dimensions 5‐Levels (EQ‐5D‐5L) index utility score (IUS) and visual analogue scale (EQ‐VAS). Results One hundred three participants were enrolled on episodic (n = 75) or prophylactic treatment (n = 28); median (range) age, 31 (12‐75) years; median (range) observation time, 26 (4‐70) weeks. Haem‐A‐QoL scores indicated impairments in HRQoL aspects; comparable between episodic/prophylactic regimens and relatively consistent over time. Haemo‐QoL SF scores with both regimens varied over time, and appeared poorer with episodic than prophylactic treatment. IUS and EQ‐VAS were comparable between regimens, stable over time and lower on bleeding days. Mean proportions of missed work and school days were 16% and 23%, respectively; mean (standard deviation) number of days hospitalized was 3.2 (8.8) (comparable between groups). Conclusions These RWD demonstrate that PwHA with inhibitors have impaired HRQoL, despite standard treatment, and that more effective treatment options are needed.

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“…Some studies around the world have evaluated the HRQoL of PWH using a standardized questionnaire called MOS SF‐36 (for Medical Outcome Study Short Form 36), abbreviated SF‐36 . Other studies have used other questionnaires such as the Haem‐A‐QoL to evaluate specific measures in HRQoL of adult PWH …”

Section: Introductionmentioning

confidence: 99%

“…6 Other studies have used other questionnaires such as the Haem-A-QoL to evaluate specific measures in HRQoL of adult PWH. 7,8 The aim of our study was to evaluate for the first time the impact of haemophilia in severe and moderate adult Lebanese PWH on their daily life and social functioning as compared to a sex-and age-matched healthy controls.…”

mentioning

confidence: 99%

The impact of haemophilia on the social status and the health‐related quality of life in adult Lebanese persons with haemophilia

et al. 2019

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Introduction Health‐related quality of life (HRQoL) studies are increasingly needed to prevent and improve the medical care of persons with haemophilia (PWH). Aim We assessed the impact of haemophilia on HRQoL and social status of adult Lebanese PWH compared to a reference population. Methods In this case‐control study, 60 severe and moderate PWH were compared to 112 healthy controls. Detailed socio‐demographic data and disease characteristics were collected, and HRQoL was assessed using the SF‐36 questionnaire. Results Age, body mass index and the percentage of married people were similar in PWH and controls. A greater proportion of controls attained a higher educational level than cases (88.4% vs 59.3%, respectively, P < 0.001). PWH were more likely to have a job requiring physical activity than controls (55.9% vs 31.4%) and more likely to be unemployed (10.2% vs 1.0%), whereas more controls had higher socio‐economic jobs (10.5% vs 1.7%). PWH had significantly (P < 0.001) worse scores in all SF‐36 domains except for energy/fatigue. Affected targeted joints (2.7 ± 1.5) and monthly bleeding frequency (2.9 ± 2.4) were inversely correlated with almost all SF‐36 domains. Only 26.7% of PWH walk normally, and walking abnormalities were inversely correlated with all SF‐36 domains except role‐emotional and emotional well‐being. Conclusion As compared with controls, the majority of Lebanese PWH has difficulties in social integration, has severe physical limitations and psychological impairments.

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Measurement properties of the Haem‐A‐QoL in haemophilia clinical trials

Abstract: These psychometric analyses provide evidence of the reliability, validity and ability to detect change of the Haem-A-QoL to assess the HRQoL of adult males with severe haemophilia A and B in longitudinal clinical trials.

Cited by 33 publications

References 22 publications

The effect of emicizumab prophylaxis on health‐related outcomes in persons with haemophilia A with inhibitors: HAVEN 1 Study

The effect of emicizumab prophylaxis on health‐related outcomes in persons with haemophilia A with inhibitors: HAVEN 1 Study

Health‐related quality of life and health status in persons with haemophilia A with inhibitors: A prospective, multicentre, non‐interventional study (NIS)

The impact of haemophilia on the social status and the health‐related quality of life in adult Lebanese persons with haemophilia

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