ArticleHamostaseologie2023
[Gene therapy of Hemophilia: Recommendations from the German, Austrian, and Swiss Society for Thrombosis and Haemostasis Research (GTH)].
Article in Hamostaseologie, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Cited by 10 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
10 citing papers in PubMed, 1 synthesis or guideline pooled it, 11 citations in OpenAlex.
- The usage of population and disease registries as pre-screening tools for clinical trials, a systematic review.Systematic reviews · 2024Pooled it
- Real-world provider experiences with hemophilia A gene therapy: administration of valoctocogene roxaparvovec.Research and practice in thrombosis and haemostasis · 2026Article
- A roadmap for a patient-centred approach to Pompe disease management.Journal of neurology · 2026Review
- Health care resources and costs associated with delivering gene therapy for hemophilia in clinical practice.Research and practice in thrombosis and haemostasis · 2026Article
- Qualification Criteria of Gene Therapy for Haemophilia-Opinion of the EAHAD Gene Therapy Working Group.Haemophilia : the official journal of the World Federation of Hemophilia · 2025Article
- Cell and gene therapy in Germany: three decades of advanced technologies.Immunotherapy · 2025Article
- Patient perspective on living with mild hemophilia in Germany: results from a nationwide survey.Frontiers in medicine · 2024Article
- Infrastructural considerations of implementing gene therapy for hemophilia in the Nordic context.Therapeutic advances in hematology · 2023Article
- Considerations for the Use of AAV-based Gene Therapy in HIV-Positive Individuals With Haemophilia.Haemophilia : the official journal of the World Federation of HemophiliaReview
- A Comprehensive Approach: Addressing Psychological Needs in Gene Therapy for Haemophilia.Haemophilia : the official journal of the World Federation of HemophiliaReview
Corrections and comments
- Erratum issued
Authors and funding
13 authors at 12 institutions in 3 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Gene therapy has recently become a realistic treatment perspective for patients with hemophilia. Reviewing the literature and our personal experience from clinical trials, we discuss key aspects of hemophilia A and B gene therapy with vectors derived from adeno-associated virus, including predictable results, risks, adverse events, and patient-reported outcomes. Patient selection, informed consent, administration, and monitoring of gene therapy as well as data collection are explained. We also discuss the need for interdisciplinary cooperation with hepatology and other specialties. We emphasize structural and organizational requirements for treatment centers according to the hub-and-spoke model and recommend the use of electronic diaries to ensure safe and timely collection and exchange of data. Electronic diaries will play a key role as a primary source of data for pharmacovigilance, postmarketing clinical studies, national and international registries, as well as health technology and benefit assessment. Reimbursement aspects and the future of gene therapy in adolescents and children are also considered. In a rapidly evolving scientific environment, these recommendations aim to support treatment providers and payers to prepare for the implementation of gene therapy following marketing authorization.
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.