Reversal of Cerebral Arteriopathy Post-Hematopoietic Stem Cell Transplant for Sickle Cell Disease

Pediatrics. 2024 Jan 1;153(2):e2023062643. doi: 10.1542/peds.2023-062643.


Sickle cell disease (SCD) is a chronic hematologic disorder which causes progressive cerebral arteriopathy beginning in childhood. As a result, arterial ischemic stroke is a major cause of morbidity and mortality in SCD, and SCD is a leading cause of childhood stroke worldwide. Allogenic hematopoietic stem cell transplant (HSCT) may be curative for individuals with SCD. Long-term outcomes and effects are currently being studied. In this report, we describe a child with SCD who presented with arterial ischemic stroke at 6 years of age and was found to have a severe form of cerebral large vessel arteriopathy by catheter-directed angiography. The patient initially underwent revascularization surgery by indirect superficial temporal artery to middle cerebral artery bypass, and 1 year later, he underwent curative HSCT. Approximately 3 years after HSCT, repeat catheter-directed angiography revealed a striking reversal of cerebral large vessel arteriopathy. This article reveals a previously unrecognized and potentially beneficial effect of HSCT that may ameliorate cerebral large vessel arteriopathy and improve cerebrovascular health for children with SCD.

MeSH terms

  • Anemia, Sickle Cell* / complications
  • Anemia, Sickle Cell* / therapy
  • Child
  • Hematopoietic Stem Cell Transplantation* / adverse effects
  • Humans
  • Ischemic Stroke*
  • Stroke* / diagnostic imaging
  • Stroke* / etiology
  • Stroke* / therapy