Hematology · Cardiology · 2 h ago
HSCT linked to sustained cardiac remodelling improvements in sickle cell disease
A prospective study followed 20 children and young adults with sickle cell disease after haematopoietic stem cell transplantation (HSCT), using cardiac imaging for up to 36 months. Cardiac measures improved after transplantation, and some echocardiographic findings at 12 months were more favourable than in age-matched participants receiving disease-modifying therapies.
- Twenty HSCT recipients underwent serial cardiac imaging.
- Cardiac volumes and fibrosis marker improved after transplantation.
- Comparison with disease-modifying therapy was not randomized.
In a prospective clinical trial, researchers assessed cardiac changes in 20 children and young adults with sickle cell disease (mean age 14.8 years) undergoing matched-sibling or haploidentical haematopoietic stem cell transplantation. Cardiac magnetic resonance imaging was performed before and after transplantation, with follow-up extending to 36 months. Echocardiographic measures at 12 months were compared with those of age-matched participants receiving disease-modifying therapy.
After HSCT, imaging showed progressive reductions in biventricular and left atrial volumes, while ejection fraction was preserved. Extracellular volume, a surrogate marker of myocardial fibrosis, improved from baseline and had normalized in nearly all participants assessed at 36 months. At 12 months, HSCT recipients had lower tricuspid regurgitant jet velocity, improved diastolic parameters and normalized cardiac output compared with the disease-modifying therapy group.
The findings suggest sustained cardiac structural improvement after HSCT, but the small sample and comparison with age-matched rather than randomized controls limit conclusions about comparative treatment effects. The results do not establish that HSCT should be selected specifically to treat cardiomyopathy.
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British Journal of Haematology: Sustained improvement in sickle cell cardiomyopathy after haematopoietic stem cell transplantation is superior to that with disease‐modifying therapy ↗This is an automated AI-condensed summary that has not yet been reviewed by an editor. Always consult the full item at the original source.