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Publication Details
AFRICAN RESEARCH NEXUS
SHINING A SPOTLIGHT ON AFRICAN RESEARCH
medicine
Health-related quality of life following valoctocogene roxaparvovec gene therapy for severe hemophilia A in the phase 3 trial GENEr8-1
Journal of Thrombosis and Haemostasis, Volume 21, No. 12, Year 2023
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Description
Background: Severe hemophilia A (HA) negatively impacts health-related quality of life (HRQOL). Objectives: We aimed to analyze HRQOL in adult men with severe HA without inhibitors after valoctocogene roxaparvovec gene transfer in the phase 3 trial GENEr8-1. Methods: Participant-reported outcomes were the hemophilia-specific quality of life questionnaire for adults (Haemo-QOL-A), the EQ-5D-5L instrument, the Hemophilia Activities List (HAL), and the Work Productivity and Activity Impairment Questionnaire: Hemophilia Specific (WPAI+CIQ:HS). Participants completed the questionnaires at baseline and through 104 weeks postinfusion with 6 × 1013 vg/kg of valoctocogene roxaparvovec. Scores were analyzed per participant characteristics and outcomes. Results: For 132 HIV-negative participants, mean change from baseline in Haemo-QOL-A Total Score met the anchor-based clinically important difference (CID: 5.5) by week 12; the mean (SD) increase was 7.0 (12.6) at week 104. At week 104, improvement in Consequences of Bleeding, Treatment Concern, Worry, and Role Functioning domain scores exceeded the CID (6). EQ-5D-5L Utility Index scores improved above the CID at week 52, but not at week 104. EQ-5D-5L visual analog scale and HAL scores increased from baseline to week 104. Participants reported less activity and work impairment at week 104 than baseline. Participants with problem joints had lower mean baseline Haemo-QOL-A Total and domain scores than those without them, but improved over 104 weeks, except for 11 participants with ≥3 problem joints. Participants with 0 bleeds during the baseline prophylaxis period reported Haemo-QOL-A score improvements above the CID, including in the Consequences of Bleeding domain. Conclusion: Valoctocogene roxaparvovec provided clinically meaningful HRQOL improvement for men with severe HA. © 2023 International Society on Thrombosis and Haemostasis
Authors & Co-Authors
O'Mahony, Brian
Ireland, Dublin
Irish Haemophilia Society
Ireland, Dublin
Trinity College Dublin
Dunn, Amy L.
United States, Columbus
Nationwide Children’s Hospital
Leavitt, Andrew D.
United States, San Francisco
University of California, San Francisco
Peyvandi, Flora A.
Italy, Milan
Fondazione Irccs Ca' Granda Ospedale Maggiore Policlinico
Italy, Milan
Università Degli Studi Di Milano
Ozelo, Margareth C.
Brazil, Campinas
Universidade Estadual de Campinas
Mahlangu, Johnny Ndoni
South Africa, Johannesburg
University of the Witwatersrand
Peerlinck, Kathelijne M.J.
Belgium, Leuven
Ku Leuven– University Hospital Leuven
Wang, Jiaan Der
Taiwan, Taichung
Veterans General Hospital-taichung Taiwan
Lowe, Gillian C.
United Kingdom, Birmingham
Queen Elizabeth Hospital Birmingham
Tan, Cheewee
Australia, Adelaide
Royal Adelaide Hospital
Australia, Adelaide
The University of Adelaide
Giermasz, Adam S.
United States, Davis
University of California, Davis
Tran, Huyen Anh M.
Australia, Melbourne
The Alfred
Chambost, Hervé
France, Marseille
Ap-hm Assistance Publique - Hôpitaux de Marseille
López-Fernández, María Fernanda
Spain, Corunna
Complejo Hospitalario Universitario Juan Canalejo
Kazmi, Rashid Saeed
United Kingdom, Southampton
University Hospital Southampton Nhs Foundation Trust
Skinner, Mark W.
Canada, Hamilton
Mcmaster University
Klamroth, Robert
Germany, Berlin
Vivantes Klinikum Im Friedrichshain
Germany, Bonn
Universität Bonn
Quinn, Jennifer
United States, San Rafael
Biomarin Pharmaceutical Inc.
Yu, Hua
United States, San Rafael
Biomarin Pharmaceutical Inc.
Wong, Wing Yen
United States, San Rafael
Biomarin Pharmaceutical Inc.
Robinson, Tara M.
United States, San Rafael
Biomarin Pharmaceutical Inc.
Pipe, Steven W.
United States, Ann Arbor
University of Michigan, Ann Arbor
Statistics
Citations: 2
Authors: 22
Affiliations: 27
Identifiers
Doi:
10.1016/j.jtha.2023.08.032
ISSN:
15387933
Research Areas
Disability
Genetics And Genomics
Infectious Diseases
Participants Gender
Male