Health-related quality of life following valoctocogene roxaparvovec gene therapy for severe hemophilia A in the phase 3 trial GENEr8-1

O’Mahony, Brian; Dunn, Amy L.; Leavitt, Andrew D.; Peyvandi, Flora; Ozelo, Margareth C.; Mahlangu, Johnny; Peerlinck, Kathelijne; Wang, Jiaan-Der; Lowe, Gillian C.; Tan, Chee Wee; Giermasz, Adam; Tran, Huyen; Khoo, Teh-Liane; Cockrell, Erin; Pepperell, Dominic; Chambost, Hervé; López Fernández, Maria Fernanda; Kazmi, Rashid; Majerus, Elaine; Skinner, Mark W.; Klamroth, Robert; Quinn, Jennifer; Yu, Hua; Wong, Wing Yen; Robinson, Tara M.; Pipe, Steven W.

doi:10.1016/j.jtha.2023.08.032

Cited by 9 publications

(2 citation statements)

References 41 publications

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“…In addition to demonstrating improvement from baseline in the mean Total Score, results from this smaller trial ( n = 14 participants) demonstrated improvement in the Treatment, Feelings and Future domains among others, consistent with the HOPE‐B study results 32 . A larger study in haemophilia A patients receiving gene therapy considered a 5.5‐point change from baseline to be clinically meaningful for the Haemo‐QOL‐A Total Score 33 . This is a different questionnaire than the PRO used in the HOPE‐B study.…”

Section: Discussionsupporting

confidence: 75%

Effect of etranacogene dezaparvovec on quality of life for severe and moderately severe haemophilia B participants: Results from the phase III HOPE‐B trial 2 years after gene therapy

Itzler,

Buckner,

Leebeek

et al. 2024

Haemophilia

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IntroductionFor people with haemophilia B (PwHB), bleeding may occur despite prophylaxis, negatively affecting health‐related quality of life (HRQoL). The pivotal phase 3 HOPE‐B trial investigating the adeno‐associated virus gene transfer product, etranacogene dezaparvovec (EDZ), demonstrated sustained factor IX (FIX) activity and bleed protection in PwHB with baseline FIX levels ≤2%.AimAssess how EDZ affects HRQoL in HOPE‐B trial participants.MethodsHRQoL was evaluated using generic and disease‐specific patient reported outcomes (PROs) including the EQ‐5D‐5L and the Hem‐A‐QoL questionnaires. Mean domain and total scores were compared 6 months pre‐ and the first 2 years post‐EDZ administration using repeated measures linear mixed models. The percentage of participants with minimal clinically important improvements in HRQoL was also evaluated.ResultsTwo years post‐EDZ, there were nominally significant increases in the least squares (LS) mean score for the EQ‐5D‐5L Index Value (.04; p = .0129), reflecting better HRQoL. Nominally significant decreases in the LS mean scores, reflecting better HRQoL, were also found for the Hem‐A‐QoL total score (−6.0; p < .0001) and the Treatment (−13.94; p < .0001), Feelings (−9.01; p < .0001), Future (−6.45; p = .0004) and Work/School (−5.21; p = .0098) domains. The percentage of participants with ≥15‐point improvement ranged from 45.83% (95% CI: 31.37%, 60.83%) for Treatment to 13.89% (95% CI: 4.67%, 29.50%) for Family Planning. Results were similar for Year 1.ConclusionIn conclusion, gene therapy with EDZ improved HRQoL in the first and second years in several Hem‐A‐QoL domains, including Treatment, Feelings, Work/School and Future domains, whereas improvement in other aspects of HRQoL were not demonstrated.

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Section: Discussionsupporting

confidence: 75%

Effect of etranacogene dezaparvovec on quality of life for severe and moderately severe haemophilia B participants: Results from the phase III HOPE‐B trial 2 years after gene therapy

Itzler,

Buckner,

Leebeek

et al. 2024

Haemophilia

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“…Evaluating HRQoL not only provides a reliable measurement of patients’ perceptions regarding the overall effectiveness of care but also is crucial in appraising both existing and novel treatment strategies [ 15 , 16 , 17 , 18 , 19 ]. In addition, HRQoL assessments enable cross-country comparisons of healthcare systems, potentially serving as an important tool to exert pressure on health authorities to align national health services with those of countries offering superior care standards [ 20 ].…”

Section: Introductionmentioning

confidence: 99%

A Retrospective Observational Study of Quality of Life in a Northern Greece Population of People with Haemophilia

Moka,

Ntova,

Gavriilaki

et al. 2024

Life

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Haemophilia presents a significant challenge to the quality of life of affected individuals. Evaluating the health-related quality of life (HRQoL) of people with haemophilia (PwH) provides a valuable mean of assessing their perception of overall care outcomes, while also identifying influential factors across various age and condition severity demographics. This observational retrospective study determined the HRQoL of 100 adult PwH in Northern Greece through comprehensive analysis and interpretation of their HRQoL levels, particularly in domains concerning their physical, emotional, and mental well-being, obtained through the Haem-A-QoL index questionnaire. Disease severity and young age were significantly associated with the administration of prophylactic treatment (84.2% of patients with severe haemophilia and 65.2% of patients aged 18–30). The mean Haem-A-QoL score was 40.11 ± 17.38, with the lowest HRQoL observed in the 46–60 age group (46.16), and the highest in the ≥61 age groups (35.16). Notably, the ‘Sports/Leisure’ and ‘Physical Health’ domains exhibited the highest scores, in contrast to ‘Family Planning’ and ‘Relationships/Sexuality’. Individuals with mild haemophilia recorded the lowest mean score (39.38), while those with a severe condition exhibited the highest (41.23). Age, disease severity, and physical activity emerged as primary determinants significantly affecting HRQoL outcomes.

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Three-year outcomes of valoctocogene roxaparvovec gene therapy for hemophilia A

Madan,

Ozelo,

Raheja

et al. 2024

Journal of Thrombosis and Haemostasis

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Health-related quality of life following valoctocogene roxaparvovec gene therapy for severe hemophilia A in the phase 3 trial GENEr8-1

Cited by 9 publications

References 41 publications

Effect of etranacogene dezaparvovec on quality of life for severe and moderately severe haemophilia B participants: Results from the phase III HOPE‐B trial 2 years after gene therapy

Effect of etranacogene dezaparvovec on quality of life for severe and moderately severe haemophilia B participants: Results from the phase III HOPE‐B trial 2 years after gene therapy

A Retrospective Observational Study of Quality of Life in a Northern Greece Population of People with Haemophilia

Three-year outcomes of valoctocogene roxaparvovec gene therapy for hemophilia A

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