A recent clinical trial has shown that riociguat, a medication designed to stimulate the nitric oxide receptor soluble guanylate cyclase, does not increase rates of vaso-occlusive events in patients with sickle cell disease. The study aimed to examine whether riociguat would cause similar increases in pain crises, as seen in a previous trial of phosphodiesterase 5 inhibitors.
This phase 1–2, randomised, double-blind, placebo-controlled trial enrolled 130 patients aged 18 or older with confirmed sickle cell disease. Participants were given either riociguat or a placebo for 12 weeks, with doses escalating based on blood pressure responses. The primary endpoint was the incidence of serious treatment-emergent adverse events.
The results were promising. The proportion of patients experiencing serious adverse events was lower in the riociguat group (22.7%) compared to the placebo group (31.3%), though the difference was not statistically significant. Additionally, the rates of vaso-occlusive events, pain severity, and pain interference were similar between the two groups, suggesting that riociguat did not exacerbate the symptoms of sickle cell disease.
One of the key findings was the significant reduction in blood pressure among those taking riociguat, with a mean decrease of 8.2 mm Hg compared to just 1.2 mm Hg in the placebo group. These study findings suggest that riociguat is a safe treatment option with potential haemodynamic benefits, providing valuable insights for future trials in sickle cell disease.
Helena Bradbury, EMJ
Reference
Gladwin MT et al. Riociguat in patients with sickle cell disease and hypertension or proteinuria (STERIO-SCD): a randomised, double-blind, placebo controlled, phase 1–2 trial. The Lancet Haematology. 2024;11(5):e345-57.
