SynthesisBMC medical ethics2025
Balancing benefits and burdens: a systematic review on ethical and social dimensions of gene and cell therapies for hereditary blood diseases.
Synthesis in BMC medical ethics, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers.
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
4 citing papers in PubMed.
- Moral Attitudes on Somatic Gene Editing for Inherited Cardiomyopathy: A Qualitative Interview Study with Key Stakeholders in the Netherlands.Journal of bioethical inquiry · 2026Article
- Novel use of a hash-based tokenization for sharing sickle cell disease data without sharing protected health information.Blood advances · 2026Article
- The role of hydroxyurea in modulating miRNA expression in sickle cell disease: molecular mechanisms and therapeutic implications.Annals of hematology · 2026Review
- End-of-Life Care in Sickle Cell Disease and Transfusion-Dependent β-Thalassemia: Clinical, Psychosocial, and Ethical Considerations.Mediterranean journal of hematology and infectious diseases · 2026Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
6 authors.
Funding
Abstract
backgroundSickle cell disease (SCD) and Diamond-Blackfan anemia syndrome (DBAS) are two hereditary blood diseases that present significant challenges to patients, their caregivers, and the healthcare system. Both conditions cause severe health complications and have limited treatment options, leaving many individuals without access to curative therapies like hematopoietic stem cell transplantation. Recent advancements in gene and cell therapies offer the potential for a new curative option, marking a pivotal shift in the management of these debilitating diseases. However, the implementation of these therapies necessitates a nuanced understanding of the ethical and social implications.
methodsIn this mixed methods systematic review, we explore the responsible development and implementation of gene and cell therapies for SCD and DBAS and aim to sketch a path toward ethically and socially sound implementation. Drawing upon principles of Responsible Research & Innovation and the 4A framework of availability, accessibility, acceptability, and affordability, we thematically analyze existing research to illuminate the ethical and social dimensions of these therapies. Following established PRISMA and JBI Manual guidelines, a search across multiple databases yielded 51 peer-reviewed studies with publication dates ranging from 1991 to 2023.
resultsOur thematic analysis shows that the theme of acceptability is heavily shaped by interactions between patients, caregivers, healthcare professionals and researchers, influencing treatment decisions and shaping the development of curative gene and cell therapies. Despite the generally positive perspective on these therapies, factors like the limited treatment options, financial constraints, healthcare professional attitudes, and (historical) mistrust can impede stakeholder decision-making. While acceptability focuses on individual decisions, the themes of availability, accessibility, and affordability are interconnected and primarily driven by healthcare systems, where high research and development costs, commercialization and a lack of transparency challenge equitable access to these therapies. This diminishes the acceptability for patients, revealing a complex interdependence of the themes.
conclusionsThe findings suggest the need for improved communication strategies in clinical practice to facilitate informed decision-making for patients and caregivers. Policy development should focus on addressing pricing disparities and promoting international collaboration to ensure equitable access to therapies. This review has been pre-registered in PROSPERO under registration number CRD42023474305.
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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.