National Sickle Cell Awareness Month: New insights into gene therapy, pain management
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September is National Sickle Cell Awareness Month.
The congressional designation is intended to focus attention on the need for improved treatments and additional research for the condition, which affects an estimated 100,000 people in the United States.
In conjunction with this observation, Healio provides the following updates in sickle cell disease research and treatment.
1. The first three patients who received OTQ923 (Intellia Therapeutics, Novartis) — an investigational gene therapy — experienced dramatic reductions in complications related to sickle cell disease. Read more.
2. Researchers at Children’s Hospital of Philadelphia and Perelman School of Medicine at University of Pennsylvania developed an in vivo proof-of-concept model for the delivery of gene editing tools to treat blood disorders. Read more.
3. Evidence suggests two investigational gene therapies provide net health benefits compared with standard approaches for sickle cell disease, according to a revised evidence report from Institute for Clinical and Economic Review. Read more.
4. Sickle cell disease is known to be associated with severe pain, but the current care model primarily focuses on acute vaso-occlusive pain. A perspective published in The New England Journal of Medicine proposed a shift to a biopsychosocial model that would address other types of chronic daily pain patients with sickle cell disease face. Read more.
5. Treatment with exagamglogene autotemcel (CRISPR Therapeutics, Vertex Pharmaceuticals) conferred clinically meaningful increases in fetal hemoglobin and total hemoglobin levels among patients with beta-thalassemia and sickle cell disease, results of two studies showed. Read more.
6. Unemployment, depression, female sex and older age appeared associated with increased pain frequency among patients with sickle cell disease. Read more.
7. Despite having been established as the primary disease-modifying therapy for individuals with sickle cell anemia, hydroxyurea remains underused among children with the disease. Read more.
8. An analysis of individuals in the United States with public insurance showed that those with sickle cell disease died more than 20 years earlier than the average person. Read more.
9. Fewer than 4% of eligible individuals with sickle cell disease received prescriptions for newer FDA-approved pain medications. Read more.
Reference:
- NIH National Heart, Lung, and Blood Institute. What is sickle cell disease? Available at: https://www.nhlbi.nih.gov/health/sickle-cell-disease. Accessed Sept. 13, 2023.
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