Curative therapy promotes sustained improvement in sickle cell cardiomyopathy

Sickle cell disease, a chronic, genetic blood condition, often leads to cumulative damage to the heart muscle, which results in a condition called cardiomyopathy. Sustained use of standard-of-care treatments for sickle cell disease (hydroxyurea and/or blood transfusions) can reverse some heart damage, but not all. St. Jude scientists, led by corresponding and senior author Akshay Sharma, MBBS, MSc, Department of Bone Marrow Transplantation & Cellular Therapy, and first author Parul Rai, MD, Department of Hematology, investigated whether curative treatments, such as allogeneic hematopoietic stem cell transplantation (HSCT), have a greater impact on improving heart disease, with their findings reported in the British Journal of Haematology.

The investigators had previously reported improvements in myocardial fibrosis, or the buildup of scar tissue in the heart muscle, after HSCT. The current study extended the post-HSCT cardiac follow-up period to 36 months and conducted serial cardiac magnetic resonance (CMR) imaging on children and young adults with sickle cell disease who received HSCT. The impact of HSCT was also compared to the impact of sustained use of hydroxyurea or blood transfusions in an age-matched control group.  

“This study suggests that curing sickle cell disease can fundamentally change the trajectory of cardiac injury,” said Sharma. “Rather than simply preventing further damage, stem cell transplantation was associated with sustained improvement in heart structure and normalization of a marker associated with myocardial fibrosis in nearly all patients assessed three years after transplant. That raises the exciting possibility that some organ damage from sickle cell disease may be reversible.”

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