Guideline-Based Care Might Reduce Cerebrovascular Illness in Sickle Cell Disease

According to a story from Healio, a recent study has found that use of hydroxyurea in alignment with treatment guidelines has the potential to bring down symptoms of cerebrovascular disease in people living with sickle cell disease. This finding was based on results from a single-center study. As treatments have improved for people living with sickle cell, patients are seeing major improvements in long-term survival.

However, cerebrovascular disease is still a common cause of morbidity in this patient population. A third of patients are believed to experience silent cerebral infarcts by their teen years. This results in increased risk of neurocognitive impairment and stroke. Hydroxyurea appears to protect patients from cerebrovascular progression and strokes, even in people who already have abnormal cerebral blood flow. Guidelines first issued in 2014 recommend use of hydroxyurea early in a patient’s life, starting at just nine months old.

This was a retrospective study that drew on data from an institutional registry, drawing on data from 2009-2023. Data from a total of 446 patients was used, with a median follow-up duration of five years. The goal of the study was to determine whether the guidelines resulted in increased use of hydroxyurea, and whether this use had any effect on the development of cerebrovascular disease symptoms.

Hydroxyurea usage increased significantly following the release of the guidelines, increasing from 29% to 72%. The researchers reported a 14.8% incidence rate cerebrovascular disease events. The data also suggested improvements in cerebrovascular disease-free survival following the guideline release, but this figure was not statistically significant. An overrepresentation of patients in the post-guideline period and the relatively small sample size may have played a role in this result.

The scientists aim to expand their research with a larger cohort involving 1000 patients in order to get more substantive data. While two gene therapies are FDA-approved in sickle cell disease, hydroxyurea is still expected to play a significant role in treatment.

About Sickle Cell Disease

Sickle cell disease is a genetic, inherited disorder of the blood. This disorder is characterized by an abnormality affecting hemoglobin, which is found in red blood cells and is responsible for carrying oxygen. The abnormality causes blood cells to lose their typical circular shape and instead take on an elongated, sickle-like appearance. This is caused by a genetic mutation that may have arisen as a defense against malaria, although this benefit only occurs in people with sickle cell trait, not the disease. Symptoms begin to appear at around six months old and include swelling of the hands and feet, stroke, bacterial infections, and acute episodes of severe pain termed sickle cell crisis. Severity of disease varies, but these attacks can result in serious declines in health and organ damage. Treatment is mostly symptomatic, but bone marrow transplant has been curative in children. The disease most frequently affects people of African ancestry. Life expectancy is between 40 and 60. To learn more about sickle cell disease, click here.

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