Kidney disease, they develop a new compound for anemia


A new compound developed from the discovery awarded with the Nobel Prize for Medicine in 219 has been shown to be as safe and effective as conventional treatment for anemia in patients with kidney disease, raising hemoglobin levels in both dialysis patients and patients. those who weren’t.

Anemia is a common and sometimes debilitating problem among patients with chronic kidney disease (CKD), which can occur when the kidneys become damaged and limit the production of erythropoietin (EPO), a hormone that signals the body to make red blood cells. Currently, patients with CKD and anemia are treated with erythropoiesis-stimulating agents (ESAs), which must be administered by subcutaneous injection or as part of dialysis.

Researchers at Brigham and Women’s Hospital in the United States examined hypoxia-inducible factor prolyl hydroxylase inhibitors (HIF PH inhibitors), a new class of drugs that can be taken by mouth to treat anemia.

The ASCEND trials, sponsored by GlaxoSmithKline (GSK), tested one of these compounds, daprodustat, in CKD patients on dialysis and in CKD patients without dialysis, and compared the safety and efficacy of the oral drug with conventional treatment.

In two studies published in ‘The New England Journal of Medicine’ and in a simultaneous presentation at Kidney Week of the American Society of Nephrology, they offered data indicating that daprodustat was as safe and effective as ESAs.

“Anemia is a problem for many CKD patients, and having to go to the hospital or have a subcutaneous injection can become a bottleneck for treatment,” explains Ajay Singh, from Brigham’s Division of Renal Medicine and principal investigator. of trials – Oral therapy has the power to be transformative for patient care. “

The development of prolyl hydroxylase inhibitors is based on discoveries about how cells perceive and adapt to oxygen availability by Dr. William G. Kaelin Jr. of Brigham and the Dana-Farber Cancer Institute , and its collaborators. These discoveries were recognized with the Nobel Prize in Medicine in 2019.

While ESAs act as substitutes for EPO to directly stimulate red blood cell production, HIF PH inhibitors act to stabilize proteins known as hypoxia-inducible factors, coercing the body to produce its own EPO and enhancing mobilization. of iron to the bone marrow.

Many concerns have been raised over the years about the safety of ESAs, including the possible increased risk of stroke, myocardial infarction, vascular access thrombosis, tumor progression, and death. The ASCEND phase 3 trials evaluated cardiovascular safety as well as the efficacy of the FHIA inhibitor in increasing hemoglobin. Major cardiovascular adverse events (MACE) were adjudicated by an independent committee throughout the trials.

“We found that oral administration of daprodustat worked as well as conventional therapy – increasing and maintaining hemoglobin levels among non-dialysis patients and maintaining levels among dialysis patients – and was just as safe,” Singh explains. This could lead to a new way of treating people with kidney disease, avoiding injections and stimulating the body to produce red blood cells at the same time. “

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Kidney disease, they develop a new compound for anemia