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A 3D illustration of sickle-shaped red blood cells in the bloodstream seen in sickle cell disease.
People with sickle cell disease are at high risk for vascular complications that can lead to pulmonary hypertension, stroke, kidney failure, and pain in the chest, abdomen, and joints.
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New Drug Controls Sickle Cell Disease-Linked Hypertension

Treatment for lung condition could help patients with SCD control complications from hypertension and kidney damage

University of Maryland School of Medicine
Published:Apr 12, 2024
|2 min read
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A drug approved to treat pulmonary arterial hypertension may be effective at managing hypertension and end-organ damage in patients with sickle cell disease (SCD), according to a new study published in Lancet Haematology. An early-phase randomized clinical trial involving 130 patients with SCD found that the drug, called riociguat, was safe to use, well tolerated in these patients, and significantly improved their blood pressure. Preliminary efficacy data suggested the medication may improve heart function.

An estimated 100,000 Americans have SCD, and the disease occurs in about one out of every 365 Black or African-American births, according to the CDC. People with SCD are at high risk for vascular complications that can lead to pulmonary hypertension, stroke, and kidney failure as well as severe pain when red blood cells impede blood flow through tiny blood vessels in the chest, abdomen, and joints. Hypertension can worsen these complications.

Unfortunately, previous research found that sildenafil, an effective treatment for pulmonary hypertension, caused unacceptable side effects in patients with SCD. It found that those who took this drug experienced high levels of pain that caused increased hospitalization compared to those who took a placebo treatment. The new study tested the safety and efficacy of riociguat in mitigating clinical complications for patients with SCD.

Trial testing the efficacy of riociguat

In the double-blind clinical trial, patients with SCD and mild hypertension or proteinuria were randomly assigned to receive either riociguat or a placebo. Both groups received the study drug at a starting dose of 1 mg, which was gradually increased up to 2.5 mg, thrice a day for 12 weeks. The researchers found that among the participants who took riociguat, 22.7 percent experienced at least one serious adverse event related to the treatment. In comparison, 31.3 percent of participants in the group that received the placebo had at least one serious adverse event during the study.

The differences were not statistically significant. There were no differences between the two groups in the rates of pain severity, pain interference in their daily lives, and vascular events related to their SCD. 

Blood pressure in participants who took riociguat dropped by 8.20 mmHg, while those on the placebo saw a decrease of about 1.24 mmHg. The result was highly statistically significant, meaning riociguat was more effective at lowering blood pressure, with a difference of approximately 6.96 mmHg. 

Riociguat was found to be safe and significantly improved blood pressure throughout the study. “Our results are encouraging and open the door to larger clinical trials involving this class of drugs in patients with sickle cell disease who have pulmonary hypertension or kidney disease. Having a drug that’s easy to tolerate can help them better manage their blood pressure and help prevent serious complications down the road,” said study leader Mark T. Gladwin, MD, who is the John Z. and Akiko K. Bowers Distinguished Professor and dean of UMSOM, and vice president for medical affairs at University of Maryland, Baltimore, MD.

- This press release was originally published on the University of Maryland School of Medicine website