Bone marrow transplants may prevent blood vessel disease in patients with sickle cell disease

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Bone marrow transplantation prevented or improved cerebral vascular disease in adults with sickle cell disease, according to a preliminary study to be presented at the American Stroke Association’s International Stroke Conference 2023. Held August 8-10, 2023, the world’s first conference for researchers and clinicians dedicated to the science of stroke and brain health.

Sickle cell disease is a group of genetic disorders caused by mutations in hemoglobin and is a major risk factor for stroke in children and adults. It causes abnormally shaped (sickle-shaped) red blood cells that interact with the lining of blood vessels and can lead to blockage of blood vessels.

John K. Lynch, DO, MPH, lead author of the study and Associate Investigator in the Division of Stroke, National Institute of Neurological Disorders and Stroke (NINDS), a division of the National Institutes of Health, Bethesda, Maryland

“The two major cerebrovascular complications of sickle cell disease are vasculopathy. can result in asymptomatic brain damage throughout the brain,” Lynch said. .

Abnormal narrowing or dilation of blood vessels in the brain can cause stroke and is the leading cause of death and disability in children and adults with sickle cell disease. Bone marrow is at the center of most bones and contains stem cells that turn into red blood cells, white blood cells, or platelets.Prior research on Bone marrow transplantation It has been shown to be useful in children. In this study, researchers investigated the impact of bone marrow transplantation on the development and progression of vascular irregularities in adults with sickle cell disease.

“I was surprised that the bone marrow transplant reversed some of the changes in the brain’s blood vessels. I thought it would stop the changes from getting worse, but I didn’t think it would reverse the changes,” Lynch said. “The focus of our study was to determine whether receiving a stem cell transplant would result in favorable changes in the blood vessels of the brain in patients with sickle cell disease.”

Researchers examined a National Institutes of Health study population of adults with sickle cell disease who underwent a bone marrow transplant between 2004 and 2019. The patient underwent her preoperative and postoperative MRI/MRA imaging, which was reviewed and scored by two independent imaging experts. Presence of stenotic vessels and aneurysms of her eight arteries in the brain. The researchers then compared vascular changes before and after bone marrow transplantation in 87 people (58% men, mean age 32 years) during a follow-up period of more than three years.

Investigation result:

  • Twenty-eight percent of patients had evidence of cerebral vascular problems, 17% had narrowed vessels, and 13% had bulging vessels that were present at the time of bone marrow transplantation.
  • When comparing vascular abnormalities before and after bone marrow transplantation, the researchers found that none of the participants without vascular abnormalities developed narrowing or bulging after transplantation.
  • A review of brain imaging scans showed improvement in stenosis in 62% of patients with vascular abnormalities after bone marrow transplantation.

“Our study is unique in that we were able to examine adults with sickle cell disease over time and compare differences in blood vessels and brain tissue over time,” Lynch said.

“We believe that the reduced number of sickle cells and improved oxygen-carrying capacity of the cells led to the reduced number of strokes after bone marrow transplantation,” Lynch said.

A limitation of this study is that it did not include a comparison group of patients who did not undergo bone marrow transplantation. In addition, some patients were followed for short periods and others for long periods. “We need a more even follow-up time for all study participants to help us understand if vascular damage will take time to stop or improve, or if this will happen quickly.” said Lynch.

“Bone marrow transplantation is being considered more frequently for sickle cell patients, and other less invasive and potentially life-saving treatments are being developed for sickle cell patients. I hope that.”

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