According to the Centers for Disease Control and Prevention, an estimated 100,000 Americans have sickle cell disease, and the disease affects about 1 in 365 black or African American births. People with sickle cell disease are at increased risk for vascular complications that can lead to pulmonary hypertension, stroke, and kidney failure, which can occur when red blood cells block blood flow through small blood vessels in the chest, abdomen, and joints. causes pain. These complications can be exacerbated by high blood pressure.

Unfortunately, previous studies found that sildenafil, an effective treatment for pulmonary hypertension, causes unacceptable side effects in patients with sickle cell disease. It was found that people who took the drug experienced more pain and had more hospitalizations than those who received a placebo treatment.

This new study was designed to test the safety of riociguat and how effective it is at preventing or reducing clinical complications in patients with sickle cell disease.

In this study, patients with sickle cell disease and mild hypertension or protein in the urine (early signs of kidney disease) were randomly assigned to receive either riociguat or a placebo in a double-blind clinical trial. It was done. In both groups, the study drug was started at 1 milligram and gradually increased to 2.5 milligrams, three times a day for 12 weeks. Researchers found that among participants who took riociguat, 22.7% experienced at least one serious adverse event related to treatment. In contrast, in the group receiving a placebo, 31.3% of participants experienced at least one serious adverse event during the study.

The difference was not statistically significant. There were no differences between the two groups in pain severity, pain interference with daily activities, or rates of sickle cell disease-related vascular events. In terms of drug treatment effectiveness, participants who took riociguat saw a reduction in blood pressure of 8.20 mmHg, while those who took a placebo saw a reduction in blood pressure of only about 1.24 mmHg. This result was highly statistically significant, meaning that riociguat was much more effective at lowering blood pressure than placebo, with a difference of approximately 6.96 mmHg. In summary, riociguat was found to be safe and led to significant improvements in blood pressure during the study period.

“Our results are encouraging and open the door to larger clinical trials involving this class of drugs in sickle cell patients with pulmonary hypertension or kidney disease.” Better-tolerated drugs can help better control blood pressure and prevent hypertension, which can lead to serious complications down the road.” John Z. Bowers & Akiko K. said study leader Mark T. Gladwin, M.D., Bowers Distinguished Professor and Dean of UMSOM and vice president for medical affairs at the University of Maryland, Baltimore.

Bayer Pharmaceuticals, the manufacturer of riociguat, provided funding (as well as the drug and placebo) for the study.

The study was led by the University of Pittsburgh’s Clinical Center and Data Coordinating Center. Co-authors of the study included faculty from the University of Illinois at Chicago, Albert Einstein College of Medicine, University of Pittsburgh, Emory University, Duke University, and Johns Hopkins University School of Medicine.