22 April 2024

New results from an early clinical trial have offered hope that a drug for pulmonary arterial hypertension may be effective in the care of sickle cell patients.

The drug may be able to help hypertension and end organ damage in these patients, researchers have reported in the Lancet Haematology.

The Phase 1-2 study, STERIO-SCD, found that the drug riociguat appears to be safe and shows signs of effectiveness in patients with sickle cell disease.

Researchers at the University of Maryland, USA, tested the drug after other drugs were found to have unacceptable side effects.

The randomised control trial involved 130 patients with mild hypertension or having protein in the urine – a marker for developing kidney disease.

There were no statistically significant differences in adverse events, the researchers report. 22.7% of those receiving riociguat experienced at least one adverse event,  compared with 31.3% of those receiving a placebo.

The drug achieved a drop in blood pressure of 8.2 mmHg amongst users. Those on placebo achieved 1.24 mmHg reductions – a difference reported as “highly statistically significant”.

Study leader Professor Mark Gladwin said: “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.”


Gladwin MT, Gordeuk VR, Desai PC, Minniti C, Novelli EM, Morris CR, Ataga KI, De Castro L, Curtis SA, El Rassi F, Ford HJ, Harrington T, Klings ES, Lanzkron S, Liles D, Little J, Nero A, Smith W, Taylor JG 6th, Baptiste A, Hagar W, Kanter J, Kinzie A, Martin T, Rafique A, Telen MJ, Lalama CM, Kato GJ, Abebe KZ. (2024) “Riociguat in patients with sickle cell disease and hypertension or proteinuria (STERIO-SCD): a randomised, double-blind, placebo controlled, phase 1–2 trial.” Lancet Haematology, 27 March 2024, doi: 10.1016/S2352-3026(24)00045-0

Link: https://www.thelancet.com/journals/lanhae/article/PIIS2352-3026(24)00045-0/abstract

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