Adolescents and young adults with sickle cell disease (SCD) who don’t regularly take the medication hydroxyurea (or who don’t adhere to prescribed instructions) are generally those with medication-taking barriers such as forgetfulness, lack of access, poor medication knowledge, fear of side effects, or doubts about the drug’s treatment, according to new research. Patients…
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In an analysis of adults with sickle cell disease, researchers found evidence that transfusions with stored units of older blood were associated with higher rates of hospital admission due to infections. The study, “Older red cell units are associated with an increased incidence of infection in chronically transfused adults…
Individuals with sickle cell trait (SCT), a common hemoglobin gene variant in African-Americans, have an increased risk of developing kidney disease, new research shows. These findings may have important implications in genetic counseling for individuals with SCT. The study “Sickle Cell Trait and the Risk of ESRD in Blacks” appeared…
Clinical trial results show that Zepatier (elbasvir/grazoprevir) is highly effective against hepatitis C virus (HCV) infection in sickle cell disease patients and others with inherited blood disorders. The study with these findings, “Elbasvir/Grazoprevir for Patients With Hepatitis C Virus Infection and Inherited Blood Disorders: A Phase III Study,” was published in the journal…
A 13-year-old boy with sickle cell disease has become the world’s first patient to be successfully treated with gene therapy. The unidentified teenager remains free from sickle crises and other signs of the disease 15 months after his treatment, which was conducted as part of a Phase 1/2 clinical trial…
Intensifying treatment with hydroxyurea in young children with sickle cell anemia is safe and improves several disease parameters in the long run, researchers demonstrated in a clinical trial (NCT00305175) comparing toddlers with older children. Based on the positive findings, researchers argue that the practice of giving small children higher…
The Bristol-Myers Squibb Foundation, Texas Children’s Cancer and Hematology Centers and Baylor College of Medicine plan to jointly create a treatment network for children with cancer and blood diseases in three sub-Saharan African countries. The $100 million initiative, Global HOPE (Hematology-Oncology Pediatric Excellence), aims to build the medical capacity needed to diagnose and properly…
Modus Therapeutics, a company developing treatments specifically for sickle cell disease, has successfully completed a financing campaign that raised a total of 32 million Swedish krona (about €3.4 million euros) to develop sevuparin. Sevuparin is a proprietary polysaccharide drug with the potential to restore blood flow and prevent…
Researchers in Alicante, Spain, established levels of normality for hemoglobin A in newborn screening by taking into account factors such as the babies being born prematurely and their mothers having immigrated from an area of the world where the prevalence of genetics mutations causing defects in the hemoglobin gene is high.
Using Stem Cells from Closely Matched Siblings Increases Sickle Cell Transplant Success, Study Finds
Using stem cells from a closely matched sibling increases the chance that a sickle-cell disease transplant will succeed, and the patient survive, a study reports. The study, “Sickle cell disease: an international survey of results of HLA-identical sibling hematopoietic stem cell transplantation,” was published in the journal Blood. It…
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