Articles tagged with Sickle Cell Anemia
Researchers at MUSC are developing a gene-modifying treatment for sickle cell disease by targeting the LSD 1 enzyme. The potential breakthrough could lead to a disease-modifying treatment and fast-track human clinical trials in as few as three years.
Two studies funded by PCORI will compare strategies to improve care-management transitions for adolescent and young adults with sickle cell disease. The projects aim to reduce emergency department visits, hospital admissions, and improve quality of life among young adults with the condition.
Researchers developed computer models that simulate the assembly of polymer fibers in sickle red blood cells, revealing new details about how different fiber structures produce abnormal cell shapes. The models can also be used to test new approaches to stopping the disease.
Researchers have developed a gene-editing technique using CRISPR that can introduce beneficial mutations into blood cells, potentially treating sickle cell anaemia and other blood disorders. The British-198 mutation already exists in nature and has been shown to boost foetal haemoglobin production.
Researchers found no statistically significant differences in tPA use, administration timeliness, or complications between SCD patients and non-SCD patients. The study suggests tPA is safe for SCD patients and could be used as a complementary therapy.
Research finds that individuals with sickle cell trait have a twofold increased risk of developing kidney failure, similar to the APOL1 gene variants. Hemoglobin C trait did not associate with kidney disease or kidney failure. Early screening and aggressive treatment can help mitigate this risk.
Researchers at CWRU are testing a portable blood-adhesion monitor for sickle cell disease patients. The device aims to predict vaso-occlusive crises and help patients better manage their condition, potentially reducing physical and financial costs.
A study found that standard hemoglobin A1c criteria underestimates long-term glucose levels in African Americans with sickle cell trait, resulting in fewer cases of prediabetes and diabetes. This could lead to missed opportunities for intervention and higher diabetic complications rates
A new study found that hemoglobin A1c readings are lower among individuals with sickle cell trait, suggesting a potential systemic underestimation of blood sugar control. This could lead to false sense of security for patients with diabetes.
Researchers advocate for a multi-drug treatment strategy to tackle vaso-occlusive crises in sickle cell disease, targeting different processes in the pathogenesis. The new approach has shown promising results in reducing crisis episodes, but further studies are needed to confirm its efficacy.
Researchers at Boston University School of Medicine are creating an induced pluripotent stem cell-based library for sickle cell disease, offering a valuable resource for disease modeling and treatment development. The library comprises diverse patient samples, enabling the study of genetic backgrounds and potential therapies.
Researchers found that iron deficiency anemia reduces the risk of malaria by 16% in African children, compared to sickle-cell trait, which only reduces it by 4%. Iron supplementation reverses this protection, increasing the risk of P. falciparum malaria.
Researchers at Stanford University School of Medicine have successfully repaired the gene causing sickle cell disease using CRISPR gene editing technology. The corrected human hematopoietic stem cells were then transplanted into mice and showed promise in producing functioning hemoglobin molecules.
Researchers have successfully corrected the mutated gene responsible for sickle cell disease in stem cells using CRISPR-Cas9 gene editing. The study holds promise for a new treatment by re-infusing edited stem cells into patients, potentially alleviating symptoms and improving lifespan.
African genetic variants associated with increased risk of albuminuria and low glomerular filtration rate among Hispanic/Latino adults, highlighting the importance of ancestry in kidney disease risk, researchers say.
A new report published in Blood shows that some people with mildly symptomatic SCD may live long lives with proper management of the disease. Four women with milder forms of SCD surpassed the US median life expectancy, living up to 86 years old.
Researchers developed a precision-engineered gene therapy virus that selectively silences BCL11A, reducing signs of sickle cell disease and increasing fetal hemoglobin production. The approach has the potential to substantially increase the ratio of non-sickling versus sickling hemoglobin.
Researchers at St. Jude Children's Research Hospital have found a way to use CRISPR gene editing to help fix sickle cell disease and beta-thalassemia in blood cells isolated from patients. The study provides proof-of-principle for a new approach to treat common blood disorders by genome editing.
A study published by Cincinnati Children's Hospital Medical Center reveals the molecular pathways responsible for heart anomalies in sickle cell anemia. The research opens a path to non-invasive diagnosis and development of new targeted therapies, aiming to improve quality of life and reduce mortality among SCA patients.
A team of international researchers has identified biophysical markers that could help assess the effectiveness of treatments for sickle cell disease. The study found improvements in red blood cells' shape and volume when treated with hydroxyurea, suggesting a new mechanism behind the drug's action.
A Stanford-led study of nearly 50,000 active-duty U.S. Army soldiers between 2011 and 2014 found no significant increase in mortality among those carrying sickle cell trait, contradicting earlier assumptions.
A small study found that long-term opioid treatment increased pain intensities, fatigue, and impaired daily activities in patients with sickle cell disease. Central sensitization, a phenomenon amplifying painful sensations, was also more pronounced in those on opioids.
Children with sickle cell disease show significant improvement in lung function after receiving hydroxyurea treatment. The study found a reduction of over one-third in the annual pulmonary function decline, indicating potential benefits for patients.
Researchers at Case Western Reserve University have received funding for innovative diagnostic devices targeting three major blood disorders: malaria, cystic fibrosis, and sickle cell anemia. The technologies aim to improve diagnosis speed, accuracy, and accessibility in resource-constrained settings.
A new study suggests that hydroxyurea can be effective in reducing the risk of stroke for some children with sickle cell disease. The Transcranial Doppler with Transfusions Changing to Hydroxyurea (TWiTCH) study, stopped early due to positive preliminary results, showed that transitioning from regular blood transfusions to daily doses ...
A clinical trial is planned to test the efficacy of pomalidomide, an FDA-approved multiple myeloma drug, on patients with sickle cell disease. The study shows that pomalidomide increases production of fetal hemoglobin, which can reverse the course of the disease.
A pediatric study of 341 children with sickle cell disease found that prasugrel did not significantly reduce the rate of pain crises or severe lung complications. The trial, conducted across 13 developed and developing nations, was one of the largest and geographically broadest trials on sickle cell disease to date.
A new mobile biochip device, the HemeChip, can rapidly screen for sickle cell disease with just a few drops of blood, providing an easy-to-use and cost-effective tool for equitable diagnosis. This technology has the potential to make a huge difference in developing nations worldwide by enabling early treatment.
A study found that after 6 months of hydroxyurea treatment, sickle cell disease patients' kidney function improved significantly. The urinary albumin/creatine ratio decreased, indicating a potential renal benefit of HU in sickle cell disease.
A team of engineers has developed a simple technique for diagnosing and monitoring sickle cell disease using magnetic levitation and a smartphone. The test is quick, inexpensive, and can be performed in regions with limited medical resources.
A researcher from Florida Atlantic University has developed a novel way to monitor sickle cell disease using a smartphone. The technology uses a portable smart sensor and phone application to analyze blood test results, enabling patients to track abnormal activities in their blood cells and take early intervention steps.
Researchers at UI Health have successfully transplanted stem cells from healthy siblings into adult patients with sickle cell disease, achieving a 92% cure rate and eliminating the need for chemotherapy. The procedure offers a new prospect for adults with the disease, who previously had limited treatment options.
Research found that depleting the microbiome with antibiotics can reduce neutrophil activity in sickle cell disease, preventing acute crises and reducing organ damage. The study also suggests antibiotics may help treat septic shock by eliminating pro-inflammatory neutrophils.
Danitza Nébor, a Jackson Laboratory postdoctoral associate, is driven by her personal connection to sickle-cell disease. She searches for genetic modifiers to reduce the severity of the disease in patients with two copies of the sickle-cell gene.
Researchers introduce single-letter DNA change into human red blood cells, increasing oxygen-carrying haemoglobin production and alleviating symptoms of sickle cell anaemia. The approach is effective, safe, and non-inherited, offering a promising alternative to conventional gene therapy.
A new study found that 44% of adults with sickle cell disease experience sleep disordered breathing, which lowers oxygen levels at night. The study suggests screening for sleep disturbances using a questionnaire or oxygen desaturation index to identify potential risks in this population.
Scientists have made a breakthrough in treating patients with sickle cell disease by engineering custom blood cells that can evade the immune system. Lab-grown stem cells were reprogrammed and edited using CRISPR to replace the defective gene, resulting in healthy red blood cells that function just as well as those from unaffected donors.
The Georgia Health Policy Center has received a $2.7 million grant from the CDC to study transfusion-related complications in patients with hemoglobin disorders, aiming to improve their outcomes. Researchers will explore approaches for reducing complications, developing data-driven solutions, and empowering patients and providers.
Children taking hydroxyurea for sickle cell disease show inconsistent results, with some experiencing improved symptoms despite negative laboratory tests. A new study aims to clarify the issue by monitoring medication adherence and exploring genetic differences between responders and non-responders.
A novel biochip has been developed to evaluate red blood cell properties in sickle cell patients, providing a potential tool for monitoring and managing the disease. The research aims to identify and predict flare-ups, allowing for earlier treatment and prevention of complications.
Sickle cell disease carriers in sub-Saharan Africa require aggressive public health education to raise awareness of the risks of having children with the disease. The disorder causes significant physical and emotional challenges, as well as potential life-threatening complications.
A nationwide team of experts led by UT Southwestern hematologist Dr. George Buchanan developed the first comprehensive, evidence-based guidelines for managing sickle cell disease from birth to end of life. The guidelines consist of over 500 specific directions for physicians caring for patients with sickle cell disease.
A recent study found that sickle cell patients who experience discrimination are 53% more likely to not adhere to their doctors' orders. The researchers analyzed the experiences of 291 patients with sickle cell disease and found a significant association between perceived discrimination and non-adherence.
A new evidence-based guideline for managing sickle cell disease recommends the use of hydroxyurea and transfusion therapy for many individuals with SCD. The guideline aims to facilitate improved and more accessible care for affected individuals, despite limited high-quality evidence.
A new test for sickle cell disease can identify the condition in just 12 minutes and costs as little as 50 cents, offering hope for rural clinics around the globe. The test is based on separating cells by density using polymers and water, making it simple and low-cost to run.
A new trial suggests that regular blood transfusion therapy can significantly reduce the recurrence of silent strokes and overt strokes in children with sickle cell anemia. The study found that monthly blood transfusions reduced repeat cerebral infarcts by 58% in children with pre-existing silent strokes.
A NIH-funded study found that regular monthly blood transfusions prevented recurrent brain blood vessel blockage in children with sickle cell anemia, reducing the risk of long-term cognitive problems and poor academic performance. The study suggests early screening for silent strokes can help manage the disease and prevent complications.
Researchers found that monthly transfusions reduced the risk of silent strokes and subsequent brain damage in children with sickle cell anemia. The treatment, which raises circulating blood levels and lowers sickled cells, also decreased the occurrence of painful crises.
Monthly blood transfusions significantly lower the risk of recurrent strokes in children with sickle cell disease who have already suffered a silent stroke. Children receiving regular transfusions had a 58% lower risk of suffering repeat silent or overt strokes compared to those without treatment.
Researchers use protein-engineering techniques to force chromatin fiber into looped structures that activate genes regulating fetal hemoglobin, which is not affected by the inherited mutation causing sickle cell disease. The approach may give rise to a new therapy for the debilitating blood disorder.
A new study found that two beneficial variants of a gene controlling red blood cell development have spread from Africa to nearly all human populations globally. These variants promote fetal haemoglobin production in adulthood, leading to milder symptoms of inherited blood disorders like sickle cell anaemia and thalassaemia.
Drs. DeBaun and Hebbel will present their lecture on sickle cell disease, highlighting improved understanding but continued challenges. Their research focuses on cerebrovascular injury and asthma, with implications for treatment and care.
A drug candidate developed to treat sickle cell disease has been acquired by Baxter International, advancing its clinical development activities. Aes-103 significantly reduces patients' pain in Phase II clinical trials, offering a potential breakthrough treatment.
A new bone marrow transplantation method has shown promising results in treating adults with severe sickle cell disease, achieving long-term stable donor engraftment and improved hemoglobin levels. The study's findings suggest that this procedure can be applied to older adults with fewer toxic effects.
A recent study shows that half of patients with severe sickle cell disease have safely stopped immunosuppressant medication after a modified blood stem-cell transplant. The trial reversed the condition in nearly all patients and allowed them to achieve stable mixed donor chimerism.
Researchers identified differences in pneumococcal genome that explain why vaccines don't protect children with sickle cell disease from infections. The study's findings will aid efforts to improve vaccine effectiveness and inform research into new protection methods.
A new assessment tool for young adults with sickle cell disease has been developed to gauge their readiness for adult care. The tool, which was found to be effective in pinpointing areas of need, includes a comprehensive guidebook addressing personal concerns and knowledge gaps.
Researchers found that low oxygen and nitric oxide levels increase red blood cell adhesion to blood vessel walls, leading to debilitating pain crises in sickle cell patients. Restoring normal nitric oxide levels can substantially reduce this adhesion, making it a potentially new therapeutic target.
The new guidelines recommend regular screening for pulmonary hypertension in SCD adults, and intensifying SCD therapy for those with PH. Patients with symptomatic PH should be treated, while management is guided by hemodynamic numbers obtained through catheterization.
The American Thoracic Society has developed guidelines to identify and manage patients with sickle cell disease who are at increased risk of mortality from pulmonary hypertension. Guidelines recommend treatment with hydroxyurea, chronic transfusion therapy, or indefinite anticoagulant therapy in patients with confirmed pulmonary hypert...