Health-related Quality-of-Life Outcomes 4 Years After Treatment with Valoctocogene Roxaparvovec

Highlights From the ISTH 2024 Congress

Health-related Quality-of-Life Outcomes 4 Years After Treatment with Valoctocogene Roxaparvovec

Presenting Author: Bella Madan, MD, FRCP, FRCPath

Madan B¹ Ozelo M,² Kenet G,³ Chou SC,⁴ Pipe S,⁵ Leavitt A⁶ Careta F,⁷ Lynch P,⁸ Fletcher S,⁹ Goshen D,¹⁰ Ruiz A,¹¹ Dashiell-Aje E,¹² Rivat C,¹² Mahlangu, J,¹³

Guy's & St. Thomas' NHS Foundation Trust, London, England, United Kingdom
Hemocentro UNICAMP, Department of Internal Medicine, School of Medical Sciences, University of Campinas, Campinas, Brazil
National Hemophilia Center Sheba Medical center, Tel Hashomer & Amalia Biron Research Institute of Thrombosis & Hemostasis, Tel Aviv University, Israel
Department of Internal Medicine, National Taiwan University Hospital, Taipei, Taiwan (Republic of China)
University of Michigan, Ann Arbor, Michigan, United States
Adult Hemophilia Treatment Center, University of California San Francisco, California, United States
Universidade Federal do Espírito Sant, Vitoria, Brazil
Believe Ltd, Hawthorne, California, United States
Oxford University Hospitals NHS Trust, Oxford, England, United Kingdom
Israeli Hemophilia Association, Ramat Gan, Tel Aviv, Israel
National Bleeding Disorders Foundation, Oakland Park, Florida, United States
BioMarin Pharmaceutical Inc., Novato, California, United States
Hemocentro UNICAMP, Department of Internal Medicine, School of Medical Sciences, University of Campinas

Figure 1: Haemo-Quality of Life (QoL)

This figure shows the changes in Haemo-Quality of Life (QoL)-Assessment total score in patients treated with valoctogene roxaparvovec over 4 years. Patients reported QoL improvements each year after receiving gene therapy compared to baseline of participants using FVIII prophylaxis. Moreover, the improvement each year reached the 5.5-point threshold which was considered to be clinically meaningful.

Figure 2: (HAL) Summary Score

This figure shows the hemophilia activities list (HAL) summary score in patients treated with valoctogene roxaparvovec over 4 years. The HAL comprises 42 items distributed across seven domains: **lying down/sitting/kneeling/standing, functions of the legs, functions of the arms, use of transportation, self‐care, household tasks and leisure activities and sports**. At each timepoint, participants reported an improvement on average in the HAL summary score compared with using regular FVIII prophylaxis at baseline.