9 important updates for World Sickle Cell Day
Click Here to Manage Email Alerts
World Sickle Cell Day is held every June 19.
The observance — established by the United Nations — is designed to increase knowledge and understanding of sickle cell disease, as well as the challenges that patients, their families and their caregivers face.
In conjunction with World Sickle Cell Day, HemOnc Today provides the following nine important updates in research and treatment.
• LentiGlobin (bluebird bio) gene therapy led to reductions in sickle cell disease-related complications and hemolysis, according to results of a phase 1/phase 2 trial. The agent also had an acceptable safety profile. Read more.
• The Sickle Cell Disease Association of America issued an updated provider advisory to ensure patients with sickle cell disease complications receive the best possible care during the COVID-19 pandemic. Read more.
• New recommendations call for increased uniformity of endpoints in clinical trials that evaluate therapies for sickle cell disease. The recommendations also advocate for increased use of patient-reported outcomes and biomarkers as clinical endpoints. Read more.
• The FDA granted rare pediatric disease designation to ARU-1801 (Aruvant/Roivant Sciences), an investigational autologous gene therapy, for the treatment of sickle cell disease. Read more.
• Researchers identified the protein nuclear factor IX as a novel repressor of fetal hemoglobin, a discovery that could lead to the development of new treatments for sickle cell disease. Read more.
• Oral supplementation with arginine resulted in faster resolution of pain associated with vaso-occlusive episodes among younger patients with sickle cell disease, according to the results of a randomized phase 2 trial. Read more.
• Significant induction of fetal hemoglobin after the infusion of CD34-positive cells occurred among a small cohort of patients with sickle cell disease, according to data from a phase 1 gene therapy feasibility study. Read more.
• Young adults with sickle cell disease experienced fragmentation of health care during the transition period after childhood, according to the results of a retrospective study. Researchers also found associations between the frequency of inpatient hospitalizations for disease-related symptoms among young adults and increased mortality risk. Read more.
• A moderate-intensity exercise regimen appeared to improve skeletal muscle microvasculature among patients with sickle cell disease. The regimen also appeared to partially reverse microvascular deficits frequently observed in these patients. Read more.
Collapse
Read more about
Click Here to Manage Email Alerts