organ damage in patients with sickle cell disease, according to a recent study. The study involved 130 patients with sickle cell disease and found that the drug riociguat was safe and well tolerated, and it significantly improved their blood pressure. The early phase randomized clinical trial also suggested that the medication might improve heart function.
Organ damage is a concern for patients with sickle cell disease, as demonstrated in a recent study published in Lancet Haematology. A clinical trial involving 130 sickle cell disease patients revealed that the drug riociguat was both safe and well-tolerated, and it also led to improved blood pressure. The preliminary data also suggests that the medication may have the potential to improve heart function.
Sickle cell disease affects an estimated 100,000 Americans, with about 1 in every 365 Black or African-American births being affected, according to the Centers for Disease.Control and Prevention. Individuals with sickle cell disease have a high susceptibility to vascular complications that can result in pulmonary hypertension, stroke, and kidney failure, as well as intense pain when red blood cells obstruct blood flow in small blood vessels in the chest, abdomen, and joints. These complications can be exacerbated by hypertension.
Regrettably, earlier studies revealed that sildenafil, a successful treatment for pulmonary hypertension, led to undesirable side effects in sickle cell disease patients. It was discovered that those who used this medication experienced elevated levels of pain, which resulted in increased hospital admissions.In comparison to those who were given a fake treatment, this new study aimed to examine the safety and effectiveness of riociguat in preventing or reducing clinical complications for patients with sickle cell disease. The participants had mild hypertension or protein in their urine, which is an early indicator of kidney disease. They were randomly divided into two groups, one receiving riociguat and the other a placebo, in a double-blind clinical trial. Both groups started with a 1 milligram dose of the study drug, gradually increasing to 2.5 milligrams three times a day over 12 weeks. The researchers discovered that riociguat had a significant impact on the patients’ conditions.22.7 percent of the participants who were given riociguat experienced a serious adverse event related to the treatment, whereas 31.3 percent of those who received the placebo had at least one serious adverse event during the study.
The differences between the two groups were not statistically significant. There were no variations in the rates of pain severity, pain interference in their daily lives, and in vascular events related to their sickle cell disease. In terms of the drug treatment’s effectiveness, the participants who took riociguat experienced an 8.20 mmHg drop in their blood pressure.The findings showed that those who were given a placebo experienced a decrease of around 1.24 mmHg in blood pressure. This difference was considered to be highly statistically significant, indicating that riociguat was much more effective at lowering blood pressure compared to the placebo, with a variance of approximately 6.96 mmHg. In conclusion, the study found that riociguat was safe and resulted in a significant improvement in blood pressure over the course of the trial.
“These results are promising and pave the way for larger clinical trials involving this category of medications in patients with sickle cell disease who have pulmonary hypertension or kidney disease. Having a medication that is easy to tolerate can help them better manage their condition.””Maintaining healthy blood pressure is crucial for preventing serious health issues in the future,” explained Mark T. Gladwin, MD, the study leader and John Z. and Akiko K. Bowers Distinguished Professor and Dean of UMSOM, as well as Vice President for Medical Affairs at University of Maryland, Baltimore.
The study received funding from Bayer Pharmaceuticals, the manufacturer of riociguat, who also provided the drug and placebo for the research.
The University of Pittsburgh oversaw the clinical and data coordinating centers for the study, with contributing faculty from the University of Illinois at Chicago, Albert Einstein College of Medicine, University of Pittsburgh, and Emory University.The study was conducted by researchers from various prestigious institutions including University of Pittsburgh, Duke University, and Johns Hopkins School of Medicine. The journal reference for this study is as follows: Mark T Gladwin, Victor R Gordeuk, Payal C Desai, Caterina Minniti, Enrico M Novelli, Claudia R Morris, Kenneth I Ataga, Laura De Castro, Susanna A Curtis, Fuad El Rassi, Hubert James Ford, Thomas Harrington, Elizabeth S Klings, Sophie Lanzkron, Darla Liles, Jane Little, Alecia Nero, Wally Smith, James G Taylor, Ayanna Baptiste, Ward Hagar, Julie Kanter, Amy Kinzie, Temeia Martin, Amina Rafique, Marilyn J Telen, Christina M Lalama, Gregory J Kato, Kaleab Z Abebe. The research is on Riociguat.A trial called STERIO-SCD tested the use of a new drug in patients with sickle cell disease and high blood pressure or protein in the urine. The trial was randomised, double-blind, and placebo-controlled, and was conducted in phase 1-2.