Findings from the first study of etavopivat in people with sickle cell disease: a plain language summary of publication

Plain Language SummaryWhat is this summary about?This plain language summary describes findings from the first clinical study of a medicine called etavopivat. Etavopivat is being studied to see if it might help people with sickle cell disease, which is also known as SCD. SCD is an inherited condition in which red blood cells can become crescent moon (sickle) shaped. Normally, red blood cells transport oxygen around the body. Sickle red blood cells cannot transport oxygen around the body very well. They are also rigid and sticky, can block small blood vessels, and can break down more quickly than normal red blood cells. This study aimed to discover the effects of taking etavopivat tablets once every day by mouth in a small group of people with SCD to understand (1) what dose of etavopivat they were able to tolerate, (2) what side effects they experienced, and (3) how taking etavopivat affected red blood cell shape and function.What were the results?The study included 3 groups of people with SCD. Groups 1 and 2 took etavopivat for a short time (days to weeks) to learn how tolerable each dose of treatment was to them. This summary focuses on the findings from Group 3, in which 15 people with SCD took 400 mg of etavopivat tablets by mouth once a day for 3 months. Researchers found improvement in the transport of oxygen by their red blood cells. They also found a slower rate of red blood cell breakdown. In most people with SCD who took etavopivat, red blood cells had a lower tendency to change to the sickle shape. Eight of the 15 people taking etavopivat had side effects that were or might have been related to etavopivat. Overall, these side effects were considered manageable. Five people had serious side effects. One of which was possibly related to etavopivat.What do the results mean?Researchers found that etavopivat was well tolerated in a small group of people with SCD. After taking etavopivat for 3 months, people with SCD appeared to have improvements in the health of their red blood cells and the transport of oxygen by the blood around the body. Though only a small number of people took part, study findings were encouraging enough to support planning for a larger study. This will help researchers discover whether etavopivat is a potential treatment option for people with SCD.How to say (download PDF and double click sound icon to play sound)…Diphosphoglycerate: Die-FOS-fuh-gli-suh-rateEtavopivat: Eh-TAV-o-piv-atGamma-glutamyl transferase: gam-UH-glu-TAH-myl-trans-fuh-rayzHemoglobin: hee-muh-GLOW-binPlacebo: pluh-SEE-bohPyruvate kinase: pai-ROO-vayt-KAI-nayzClinical study: A type of study that looks at how well a new treatment works in people.Side effect: Any effect (unwanted or unexpected) that a person experiences during the study. Side effects may or may not be caused by a medicine or treatment they used.Serious side effect: A side effect that is life threatening, requires hospital care, or causes lasting problems.This is an abstract of the Plain Language Summary of Publication article.View the full Plain Language Summary PDF of this article to read the full-textLink to original article hereTrial registration: ClinicalTrials.gov identifier: NCT03815695.