Stratification factors included the participants’ age, geographic region and whether they were currently on hydroxyurea, a drug commonly prescribed to patients with SCD. Participants went through a screening period of 28 to 35 days, a treatment period of up to 72 weeks and an end-of-study visit about four weeks after receiving the last dose of the study drug. Researchers randomized participants in each group to receive their dosages once daily. Participants were divided into three groups that received voxelotor in doses of 1500 mg, 900 mg and no medication, respectively. All participants had confirmed sickle cell disease and most had sickle cell anemia.
ANEMIA WITH MISSHAPEN RED BLOOD CELLS TRIAL
The 274 trial participants, ranging from 12 to 65 years old, were from 60 institutions across 12 countries. We believe this drug has the potential to decrease chronic organ failure in patients with this condition.” “These patients are susceptible to strokes, renal failure and other complications that lead to early death. “Chronic organ failure, which is predicted by the severity of anemia, is a leading cause of death for patients with SCD,” said Elliott Vichinsky, MD, the study’s lead researcher, medical director of hematology/oncology at UCSF Benioff Children's Hospital Oakland, and professor at UC San Francisco. The misshapen red blood cells break apart easily and block blood vessels, causing anemia, severe pain and organ damage that can lead to early death. The de-oxygenated hemoglobin causes red blood cells to be sickle-shaped, rather than round. It is a hereditary disorder caused by a defect in a gene that makes hemoglobin, a critical protein in red blood cells that distributes oxygen throughout the body.
Sickle cell disease affects approximately 100,000 individuals in the United States and decreases life expectancy by approximately 30 years, according to the researchers.
The results, published June 14, 2019, in The New England Journal of Medicine, found that 51 percent of the patients receiving the higher dose of voxelotor had a significant increase in hemoglobin at 24 weeks of therapy. The test included three dosage levels, including a placebo group. The 17-month HOPE trial - for Hemoglobin Oxygen Affinity Modulation to Inhibit HbS PolymErization - was designed to evaluate the safety and efficacy of the hemoglobin polymer-inhibitor for adolescents and adults with SCD. A Phase 3 study has found that patients with Sickle Cell Disease (SCD) who took a daily dose of the novel drug voxelotor had less anemia and made healthier red blood cells than patients receiving a placebo.
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