Slide 1: Health-related quality of life outcomes 4 years after treatment with valoctocogene roxaparvovec
Health-related quality of life outcomes 4 years after treatment with valoctocogene roxaparvovec Bella Madan1, Margareth Ozelo2, Gili Kenet3, 1Guy’s and St Thomas’ NHS Foundation Trust, London, UK; 2Hemocentro UNICAMP, Department of Internal Medicine, School of Medical Sciences, University of Campina
Slide 2: Disclosure: speaker fees from BioMarin
• I have received speaker fees from BioMarin Pharmaceutical Inc. 2
Slide 3: Hemophilia A
Hemophilia A People with hemophilia A lack the Hemophilia A negatively affects health-related blood clotting protein FVIII because quality of life (HRQOL) the gene is faulty Constant Low factor VIII (FVIII) levels cause worry excessive bleeding or bleeding with no apparent cause Current treatments a
Slide 4: Valoctocogene roxaparvovec and HRQOL outcomes
Valoctocogene roxaparvovec and HRQOL outcomes Valoctocogene roxaparvovec is a gene Reduced disease burden of hemophilia A4 therapy for severe hemophilia A that delivers functional genetic instructions for producing FVIII Valoctocogene roxaparvovec provides protection from bleeding with a single infu
Slide 5: GENEr8-1 study design
GENEr8-1 study design Eligibility: 134 participants enrolled and received an infusion of valoctocogene roxaparvovec • Adult men with severe hemophilia A (FVIII ≤1 IU/dL) The modified intention-to-treat (mITT) population • Previously receiving FVIII prophylaxis included the 132 participants who were
Slide 6: Haemo-QOL-A measures HRQOL in people with hemophilia
Haemo-QOL-A measures HRQOL in people with hemophilia Rating Scale Domains Examples (converted to 0–100) Physical 9 items “It is easy for me to lift 0 None of Functioning heavy objects” the time “I feel like a burden to 1 A little of Role Functioning 11 items my family” the time Consequences of 7 ite
Slide 7: Valoctocogene roxaparvovec improved Haemo-QOL-A Total Score across 4 years
Valoctocogene roxaparvovec improved Haemo-QOL-A Total Score across 4 years mITT population The improvements at the end of each year were deemed clinically meaningful1 16 Mean improvement from baseline 14 12 7.3 6.8 6.5 10 6.3 8 6 4 2 Y1 Y2 Y3 Y4 0 = 127 122 113 103 1. Quinn J, et al. Patient Relat O
Slide 8: Consistent improvements for Haemo-QOL-A domain scores
Consistent improvements for Haemo-QOL-A domain scores mITT population 16 10.5 10.7 9.7 9.2 14 7.6 7.2 7.5 12 6.0 6.1 5.3 6.2 10 4.8 8 6 Mean improvement from baseline 4 2 0 Y1 Y2 Y3 Y4 Y1 Y2 Y3 Y4 Y1 Y2 Y3 Y4 Physical Functioning Role Functioning Consequences of Bleeding = 130 125 114 106 129 124 11
Slide 9: HRQOL improvements were partly independent of FVIII activity
HRQOL improvements were partly independent of FVIII activity mITT population Improvement in Haemo-QOL-A Total Score at the end of year 4 was deemed clinically meaningful for participants with FVIII levels below 5%1 16 14 7.6 Mean change from baseline 12 6.3 10 6.2 8 6 4 2 0 ≥40% ≥5% to <40% <5% FVII
Slide 10: HAL measures self-reported functional ability for people with hemophilia
HAL measures self-reported functional ability for people with hemophilia In the previous month, did you have any difficulty due to Domains hemophilia with… Rating Scale Lying/Sitting/ “Standing for long 1 Impossible 8 items Kneeling/Standing periods of time” Function of the “Walking on an Always Leg
Slide 11: HAL Summary Score improved over years
HAL Summary Score improved over 4 years mITT population 8 4.7 4.0 3.0 Mean change from baseline 6 2.4 4 2 0 Year 1 Year 2 Year 3 Year 4 = 124 122 113 104 Results are based on available data at each time point. Error bars represent 95% confidence intervals. Data after participants resumed prophylaxis
Slide 12: The WPAI+CIQ:HS measures impairment at work and school due to hemophilia
The WPAI+CIQ:HS measures impairment at work and school due to hemophilia “In general, how many hours per week do you usually work/attend classes?” 0 Hemophilia had no effect on my work/classwork “During the past seven days, how many hours did you miss from work/class or school because of problems as
Slide 13: WPAI+CIQ:HS activity impairment was reduced over years
WPAI+CIQ:HS activity impairment was reduced over 4 years mITT population 0 Mean percent change from baseline -2 -4 -6 -8 -10 −5.2 −5.1 −5.4 -12 −7.6 -14 Year 1 Year 2 Year 3 Year 4 = 115 117 99 98 Results are based on available data at each time point. Error bars represent 95% confidence intervals.
Slide 14: Conclusions
Haemo-QOL-A HAL • Valoctocogene roxaparvovec • Participants reported improved ability to provides clinically meaningful HRQOL perform daily activities over 4 years improvements over 4 years • The meaningful improvements also apply to participants with FVIII activity below 5% at year 4 WPAI+CIQ:HS •
Slide 15: Acknowledgments
Acknowledgments • Thank you to all trial participants, their families, study site personnel, and investigators • Funding for this study was provided by BioMarin Pharmaceutical Inc. • Medical writing support was provided by Amin Ghane, PhD, of AlphaBioCom, a Red Nucleus company, and funded by BioMari