Evidence map›Paper›PMID 42064204›Full record

Trial reportResearch and practice in thrombosis and haemostasis2026

Durability of efficacy, safety, and quality of life 5 years after valoctocogene roxaparvovec gene transfer for severe hemophilia A: final phase 3 GENEr8-1 trial results.

Andrew D Leavitt, Johnny Mahlangu, Priyanka Raheja, Emily Symington, Doris V Quon, Adam Giermasz, Gili Kenet, Gillian Lowe, Nigel S Key, Carolyn M Millar and 12 more

Abstract readClinical Trial, Phase IIIMulticenter Study
PubMed Full text at PMC Publisher
In one paragraph

Trial report in Research and practice in thrombosis and haemostasis, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

2 citing papers in PubMed.

  1. Article
  2. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

22 authors.

Andrew D LeavittAdult Hemophilia Treatment Center, Department of Medicine, University of California San Francisco, San Francisco, California, USA.
Johnny MahlanguDepartment of Molecular Medicine and Haematology, Faculty of Health Sciences, University of the Witwatersrand, Johannesburg, South Africa.
Priyanka RahejaThe Royal London Hospital Haemophilia Centre, Barts Health NHS Trust, London, UK.
Emily SymingtonCambridge University Hospitals NHS Foundation Trust, Cambridge, UK.
Doris V QuonOrthopaedic Hemophilia Treatment Center, Los Angeles, California, USA.
Adam GiermaszHemophilia Treatment Center, University of California Davis, Sacramento, California, USA.
Gili KenetThe National Hemophilia Center and Amalia Biron Research Institute of Thrombosis and Hemostasis, Sheba Medical Center, Tel Hashomer, Tel Aviv University, Tel Aviv, Israel.
Gillian LoweWest Midlands Adult Haemophilia Comprehensive Care Centre, University Hospitals Birmingham NHS Foundation Trust, Birmingham, UK.
Nigel S KeyUNC Blood Research Center, University of North Carolina, Chapel Hill, North Carolina, USA.
Carolyn M MillarCentre for Haematology, Imperial College London, London, UK.
Steven W PipeDepartments of Pediatrics and Pathology, University of Michigan, Ann Arbor, Michigan, USA.
Sheng-Chieh ChouDivision of Hematology, Department of Internal Medicine, National Taiwan University Hospital, Taipei, Taiwan.
Robert KlamrothVascular Medicine and Haemostaseology, Vivantes Klinikum im Friedrichshain, Berlin, Germany.
Jane MasonQueensland Haemophilia Centre, Cancer Care Services, Royal Brisbane and Women's Hospital, Brisbane, Queensland, Australia.
Hervé ChambostAP-HM, Department of Pediatric Hematology Oncology, Children Hospital La Timone & Aix Marseille University, INSERM, INRA, C2VN, Marseille, France.
Flora PeyvandiFondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milan, Italy.
Elaine MajerusDepartment of Medicine, Washington University in St Louis, St Louis, Missouri, USA.
Dominic PepperellDepartment of Haematology, Fiona Stanley Hospital, Murdoch, Western Australia, Australia.
Chee Wee TanRoyal Adelaide Hospital, Adelaide, South Australia, Australia.
Hua YuBioMarin Pharmaceutical Inc, Novato, California, USA.
Konstantia-Maria ChaveleBioMarin UK Ltd, London, UK.
Margareth C OzeloHemocentro UNICAMP, Department of Internal Medicine, School of Medical Sciences, University of Campinas, Campinas, Sao Paulo, Brazil.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Background: Valoctocogene roxaparvovec, a gene therapy for severe hemophilia A, enables endogenous factor VIII (FVIII) expression and confers bleed control. Objectives: To present the final 5-year efficacy and safety results from the phase 3 GENEr8-1 trial. Methods: Adult men ( Results: Median follow-up was 261.1 weeks; 128 of 134 participants completed the study. For the 112 participants who enrolled from a previous noninterventional study (rollover population), mean ABR for treated bleeds declined by 83.3% ( Conclusion: Valoctocogene roxaparvovec provides durable hemostatic efficacy, FVIII activity, and improved health-related quality of life for ≥5 years, with no new safety signals.

Indexed as

Gene Therapy AgentsGenetic TherapyHemophilia AAdultFactor VIIIHemorrhageHumansMaleQuality of LifeRecombinant Fusion ProteinsFactor VIIIRecombinant Fusion ProteinsValoctocogene Roxaparvovecadeno-associated virusclinical trialgene therapyhealth-related quality of lifehemophilia A

Identifiers

PMID42064204
PMCPMC13125186

What OpenQuestion holds

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Registered trials

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.