Best Practices in Gene Therapy for Sickle Cell Disease and Transfusion-dependent β-Thalassemia.
Level 5 - mechanism / opinion, no new human data
Expert opinion and narrative review describing institutional best practices without new empirical data
PubMed 40058646 · doi:10.1016/j.jtct.2025.02.025
What was done
This narrative review outlines standardized institutional approaches and best practices for implementing and administering commercially available autologous gene-therapy-based hematopoietic stem cell transplantation (HSCT) for patients with sickle cell disease (SCD) and transfusion-dependent β-thalassemia (TDT).
What was found
The abstract reports no numerical data, clinical trial metrics, or quantitative patient outcomes. It summarizes institutional operational frameworks, policies, and procedural considerations necessary to manage the complex patient care pathway associated with these cell-based gene therapies.
Why it matters
With the recent commercial approval of gene therapies for hemoglobinopathies, sharing institutional implementation strategies assists other centers in establishing safe, standardized delivery pipelines for patients lacking HLA-matched donors.
Limits
The publication is an expert opinion and narrative guide rather than a systematic review or empirical comparative trial. The abstract provides no quantitative data on clinical efficacy, adverse events, long-term outcomes, or cost-effectiveness, and recommendations reflect the experience of specific treating institutions.
Cited by
- supports Genetic disorders such as sickle cell anemia and thalassemia can only be cured through stem cell transplantation.