Curative Approach to the Treatment of Beta-Thalassemia and Sickle Cell Disease with Hematopoietic Stem Cell Transplantation.
Level 5 - mechanism / opinion, no new human data
Narrative review without systematic search or meta-analytic synthesis.
PubMed 41753067 · doi:10.3390/jcm15041379
What was done
This narrative review summarizes clinical developments over the past three decades regarding curative therapies—specifically allogeneic hematopoietic stem cell transplantation (allo-HSCT) and gene therapy—for transfusion-dependent beta-thalassemia and sickle cell disease.
What was found
The abstract provides no quantitative data, odds ratios, or survival rates. It notes qualitatively that allo-HSCT from matched-sibling or matched-unrelated donors represents the primary curative standard, with haploidentical HSCT expanding access, and highlights that early transplantation reduces irreversible organ damage and vasculopathy.
Why it matters
It highlights the expanding landscape of curative donor options and gene therapies for severe hemoglobinopathies while framing the global access barriers driven by high cost and infrastructure requirements.
Limits
As a narrative review abstract, it presents no primary data, sample sizes, systematic selection methodology, or statistical comparisons of outcomes across transplantation platforms.
Cited by
- supports Genetic disorders such as sickle cell anemia and thalassemia can only be cured through stem cell transplantation.