Sickle Cell Disease: New Treatment Safety
- Riociguat, a medication already approved for pulmonary arterial hypertension, could offer a new approach to managing hypertension and related complications in individuals with sickle cell disease.
- The Centers for Disease Control and Prevention estimates that sickle cell disease affects about 100,000 Americans, notably impacting 1 in every 365 Black or African-American births.
- Prior research revealed that sildenafil,another pulmonary hypertension treatment,triggered unacceptable side effects,including increased pain and hospital admissions,in sickle cell patients.
Riociguat, a drug already used for pulmonary arterial hypertension, shows promise in managing complications related too sickle cell disease. A recent study highlights the safety and tolerability of riociguat, revealing its effectiveness in lowering blood pressure in patients, a crucial finding given the high risk of hypertension in this patient population. The research, published in Lancet Haematology, indicates potential for improved heart function, offering hope for better management of this inherited blood disorder. News Directory 3 provides the latest updates on this and other medical breakthroughs. This early-phase trial involved 130 patients, and while serious adverse events rates were similar between the riociguat and placebo groups, the drug significantly reduced blood pressure compared to the placebo. Future larger studies will be critical. Discover what’s next in the fight against sickle cell disease.
Riociguat Shows Promise treating Sickle Cell Hypertension
Updated June 15, 2025
Riociguat, a medication already approved for pulmonary arterial hypertension, could offer a new approach to managing hypertension and related complications in individuals with sickle cell disease. A recent early-phase clinical trial, featured in Lancet Haematology, indicates the drug is safe, well-tolerated, and effective in lowering blood pressure in this patient population. Researchers also noted preliminary data suggesting improved heart function.
The Centers for Disease Control and Prevention estimates that sickle cell disease affects about 100,000 Americans, notably impacting 1 in every 365 Black or African-American births. These individuals face a heightened risk of vascular problems, including pulmonary hypertension, stroke, and kidney failure, often exacerbated by hypertension. They also experience severe pain episodes when blood flow is obstructed.
Prior research revealed that sildenafil,another pulmonary hypertension treatment,triggered unacceptable side effects,including increased pain and hospital admissions,in sickle cell patients.
The new study aimed to evaluate riociguat’s safety and potential in preventing or reducing clinical complications in sickle cell disease.The double-blind trial involved patients with mild hypertension or protein in their urine, a sign of early kidney disease. Participants were randomly assigned to receive either riociguat or a placebo. Dosages started at 1 milligram, gradually increasing to 2.5 milligrams, taken three times daily for 12 weeks.
Serious adverse events occurred in 22.7% of the riociguat group, compared to 31.3% in the placebo group. These differences were not statistically meaningful. Pain levels and vascular events related to sickle cell disease were similar in both groups. however, riociguat led to a significant blood pressure decrease of 8.20 mmHg,compared to a 1.24 mmHg decrease with the placebo, a statistically significant difference of approximately 6.96 mmHg.
“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 definitely help them better manage their blood pressure and help prevent serious complications down the road,” said Dr. Mark T. Gladwin, university of Maryland School of Medicine.
what’s next
Larger clinical trials are planned to further investigate riociguat’s long-term benefits for sickle cell patients with pulmonary hypertension or kidney disease. Bayer Pharmaceuticals, the drug’s manufacturer, provided funding for the study, which was coordinated by the University of Pittsburgh, with contributions from multiple institutions.