ReviewInternational journal of molecular sciences2022
The Arrival of Gene Therapy for Patients with Hemophilia A.
Review in International journal of molecular sciences, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 13 papers, 1 of them a synthesis that pooled 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.
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.
Who cites it
13 citing papers in PubMed, 1 synthesis or guideline pooled it, 23 citations in OpenAlex.
- Liver-related aspects of valoctocogene roxaparvovec gene therapy for hemophilia A: expert guidance for clinical practice.Blood advances · 2024Guideline
- Prospective collection of bleeding rate and factor usage in participants with hemophilia A and B in a noninvestigational product study.Research and practice in thrombosis and haemostasis · 2026Trial
- A phase 1/2 safety and efficacy study of TAK-754 gene therapy: The challenge of achieving durable factor VIII expression in haemophilia A clinical trials.Haemophilia : the official journal of the World Federation of Hemophilia · 2025Trial
- Evolution of Hemophilia Management in Italy: Results from a Delphi Consensus Study.Journal of clinical medicine · 2026Article
- Economic Burden of Haemophilia from a Societal Perspective: A Scoping Review.PharmacoEconomics - open · 2025Review
- Immune Modulation Strategies in Gene Therapy: Overcoming Immune Barriers and Enhancing Efficacy.Current gene therapy · 2025Review
- Comprehensive Coagulation Management in Cardiac Surgery: Anticoagulants, Heparin Resistance, Monitoring, and Bleeding Risks : Coagulation Management in Cardiac Surgery.Galen medical journal · 2025Review
- Role of Pharmacy Professionals in Gene Therapy Based on Adeno-Associated Viruses: Treatment of Hemophilia as a Template of Care.The Canadian journal of hospital pharmacy · 2025Review
- A 360-degree perspective on adeno-associated virus (AAV)-based gene therapy for haemophilia: Insights from the physician, the nurse and the patient.Orphanet journal of rare diseases · 2024Review
- Delivery of DNA-Based Therapeutics for Treatment of Chronic Diseases.Pharmaceutics · 2024Review
- Infrastructural considerations of implementing gene therapy for hemophilia in the Nordic context.Therapeutic advances in hematology · 2023Article
- Bispecific Antibody Format and the Organization of Immunological Synapses in T Cell-Redirecting Strategies for Cancer Immunotherapy.Pharmaceutics · 2022Review
- Treatment With Valoctocogene Roxaparvovec in a Patient With Severe Hemophilia A Led to Sustained Normal FVIII Levels.Haemophilia : the official journal of the World Federation of HemophiliaArticle
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
4 authors at 4 institutions in 1 country.
Funding
Abstract
Historically, the standard of care for hemophilia A has been intravenous administration of exogenous factor VIII (FVIII), either as prophylaxis or episodically. The development of emicizumab, a humanized bispecific monoclonal antibody mimicking activated FVIII, was a subsequent advance in treatment. However, both exogenous FVIII and emicizumab require repeated and lifelong administration, negatively impacting patient quality of life. A recent breakthrough has been the development of gene therapy. This allows a single intravenous treatment that could result in long-term expression of FVIII, maintenance of steady-state plasma concentrations, and minimization (or possibly elimination) of bleeding episodes for the recipient's lifetime. Several gene therapies have been assessed in clinical trials, with positive outcomes. Valoctocogene roxaparvovec (an adeno-associated viral 5-based therapy encoding human B domain-deleted FVIII) is expected to be the first approved gene therapy in European countries, including Italy, in 2022. Some novel challenges exist including refining patient selection criteria, managing patient expectations, further elucidation of the durability and variability of transgene expression and long-term safety, and the development of standardized 'hub and spoke' centers to optimize and monitor this innovative treatment. Gene therapy represents a paradigm shift, and may become a new reference standard for treating patients with hemophilia A.
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
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.