Articles tagged with Sickle Cell Anemia
The Georgia Tech-led Nanomedicine Center plans to pursue a clinically viable gene correction technology for single-gene disorders, including sickle cell disease. The team aims to deliver engineered zinc finger nucleases and DNA correction templates into hematopoietic stem cells to produce healthy red blood cells.
A study of 18 patients with sickle cell disease found that inhaling nitric oxide for four hours reduced pain control compared to standard self-administered morphine. Nitric oxide may help restore hemoglobin's natural shape and charge, addressing the root cause of pain.
A $1.5M NIH grant supports Manu Platt's project to develop models for identifying children at risk of stroke due to sickle cell disease. The goal is to create a mathematical model predicting stroke risk and allow for earlier intervention, improving treatment options.
The NCAA's new sickle cell screening program for college athletes aims to prevent rare complications, but experts warn of hasty implementation and unaddressed consequences. The program may identify 400-500 new cases annually, affecting nearly 170,000 athletes.
Researchers are testing Lexiscan, a pharmacologic stress agent, in a multicenter clinical trial to reduce inflammation in sickle cell disease. The trial aims to improve symptoms such as pain and breathing problems, which can be fatal.
A study found that deleting a viral element from human genes can increase production of fetal hemoglobin in red blood cells, which could help alleviate symptoms of sickle cell disease. The researchers believe this natural mechanism may be the key to developing targeted therapies for patients.
A Nationwide Children's Hospital study shows that one-third of sickle cell disease patients receiving narcotics were not prescribed laxatives, highlighting the need for increased attention to constipation prophylaxis. The study found that older children and patients with medical admissions were more likely to receive laxatives.
A large study found 68 abnormal brain findings in 953 children, with only six requiring urgent follow-ups. These findings highlight the need for pediatricians to prepare for discussions about incidental discoveries, which can lead to unnecessary tests and fear.
A clinical trial evaluating a new approach to reduce recurrent strokes in children with sickle cell anemia and iron overload was stopped early. The trial compared hydroxyurea with transfusions, finding no significant benefits for reducing stroke risk.
Adults with sickle cell anemia exhibit lower average nonverbal function scores, as well as difficulties in processing speed, working memory, and executive function. Anemia is associated with neurocognitive impairment in older patients, highlighting the need for early identification and intervention.
A new study published in the Journal of the American Medical Association found that adults with mild sickle cell disease scored lower on brain function tests compared to healthy participants. Cognitive functioning was linked to severity of anemia, and older patients showed greater impairment.
Research suggests that adult patients with few or mild complications of sickle cell disease may develop cognitive problems, such as difficulty organizing thoughts or learning. The study found that more than a third of participants scored below normal levels on cognitive function tests.
African children with sickle-cell disease are at increased risk of bacterial illness, especially invasive pneumococcal disease. Vaccines against pneumococcus can improve quality of life and save lives among these vulnerable children.
Researchers found that lower oxygen saturation is associated with left ventricle hypertrophy and diastolic dysfunction in children with sickle cell disease. The study suggests that the beginning of adult heart disease may start in childhood, highlighting the need for further research.
A study found that patients with sickle cell disease experience a high rate of acute care utilization, with 18- to 30-year-olds at the highest risk for rehospitalization. The analysis included data from eight states and revealed significant disparities in healthcare usage among different age groups.
A new study published in JAMA found that four out of ten people treated for sickle cell disease are readmitted within 30 days, highlighting the need for improved care. The study also revealed higher rates of rehospitalization and emergency department visits among younger patients with Medicaid or public insurance.
The NHLBI and CDC are launching a four-year pilot project to study sickle cell disease and thalassemias in six US states. The Registry and Surveillance System in Hemoglobinopathies (RuSH) project aims to gather comprehensive demographic data on people with these life-threatening diseases.
Scientists have discovered a new type of genetic variation that suggests natural selection can act on gene networks, maintaining alternative states within a single species. This finding may be crucial for understanding how pathogens adapt to new stresses and could provide significant advantages in the battle against diseases.
Statins may protect individuals from serious illness following bacterial infection, including pneumococcal infections that threaten those with sickle cell disease. The cholesterol-lowering drugs employ methods to dampen inflammation and block bacterial toxins, prolonging the lives of mice with sickle cell disease.
Researchers identified statins as a potential new use for treating sickle cell disease in children, finding they reduced bacterial invasion and prevented cell death. The study suggests that prophylactic treatment with statins may reduce the risk of invasive pneumococcal infections in these patients.
A modified blood adult stem-cell transplant regimen has effectively reversed sickle cell disease in 9 of 10 adults with severe symptoms. The trial's results show that the treatment reduced toxicity and allowed patients to achieve stable mixed donor chimerism, completely replacing their disease-causing cells.
A phase I/II study at Johns Hopkins Medicine has shown that mini stem cell transplantation can safely reverse severe sickle cell disease in nine out of ten patients. The procedure, which uses intravenous transplants of blood-forming stem cells from healthy donors, promotes the coexistence of host and donor cells.
Researchers at Boston Children's Hospital have found a promising new approach to treating sickle cell disease by silencing a gene that suppresses fetal hemoglobin production. Inactivating the BCL11A gene led to a significant increase in gamma-globin production, potentially compensating for defective adult hemoglobin.
Research from Johns Hopkins Children's Center found that children with sickle cell disease who contract H1N1 virus are three times more likely to develop acute chest syndrome and five times more likely to need ICU care. The study highlights the need for these children to be included in flu vaccination lists.
A new study found that only a small percentage of providers prescribe hydroxyurea to eligible children with sickle cell disease due to concerns about future reproductive issues and low patient compliance. Despite its efficacy in preventing pain, many providers prescribe the drug for other complications without sufficient evidence.
Researchers have made significant breakthroughs in understanding the genetic basis of sickle cell disease, exploring the potential of activating fetal hemoglobin to reduce complications. A new study found that activating BCL11A could lead to increased production of healthy hemoglobin, providing a promising avenue for treatment.
The initiative seeks to improve care for patients with sickle cell disease by establishing medical homes, providing comprehensive education, and encouraging specialization in the field. The project aims to reduce pain crises and hospitalizations through better prevention and treatment strategies.
Researchers at the University of Texas Health Science Center at Houston have discovered a potential treatment for priapism, a condition characterized by persistent and painful penile erections. The FDA-approved drug PEG-ADA has been shown to relieve symptoms and prevent a major complication in pre-clinical studies.
A new study aims to prevent brain complications in children with sickle cell disease by comparing two treatments: standard therapy of monthly blood transfusions and chelation vs. a daily dose of hydroxyurea and monthly phlebotomy. The goal is to reduce the risk of first strokes in high-risk patients with sickle cell anemia.
A study published in The Lancet found that vaccination against bacterial infections, such as Streptococcus pneumoniae and Haemophilus influenzae type b, can substantially improve survival rates in African children with sickle-cell anaemia. The researchers also found that these bacteria are the same cause of infection in both developed ...
A recent study published in The Lancet suggests that children in Africa with sickle cell anaemia are dying unnecessarily from bacterial infections. The researchers recommend vaccination programs for all children in Africa to prevent these deadly infections, which can be prevented by vaccination.
Yale scientists have discovered a crucial protein that regulates cell size by controlling the exit of potassium and chloride ions. This finding has significant implications for understanding diseases such as sickle cell anemia and neurological disorders, where cell size imbalances can lead to damage.
The NHLBI has stopped a clinical trial testing sildenafil for pulmonary hypertension in adults with sickle cell disease due to increased risk of severe pain crises and hospitalization. The study found that participants taking sildenafil were more likely to experience serious medical problems compared to those on placebo.
Research found that children with sickle cell disease are less likely to receive transcranial Doppler ultrasound screenings due to limited lab access, despite increased overall screening rates.
A church-based initiative in St. Louis increased first-time African-American blood donations by 60%, with nearly 700 participants donating blood over three years. The Sickle Cell Sabbath Program educated predominantly African-American churches about sickle cell disease and its benefits, leading to more donors.
Four studies showcase treatment advances in blood cancers, including improved progression-free survival for patients with relapsed or refractory chronic lymphocytic leukemia. A new tumor suppressor gene is also discovered in patients with myeloproliferative disorders.
Researchers studied the quality of care for hospitalized sickle cell disease patients, finding that hospitals treating low volumes have higher mortality rates. The study also identified associations between insurance coverage, patient socioeconomic status, and reduced hospital stays.
A study found that ultrasound screening with transcranial Doppler ultrasonography (TCD) significantly reduced stroke incidence among children with sickle cell disease. The technique, combined with regular transfusions for high-risk patients, lowered stroke rates to one-tenth of the pre-TCD era.
Researchers have identified a gene that directly affects the production of fetal hemoglobin, which could lead to the development of new therapies for sickle cell disease and thalassemia. By suppressing a specific gene called BCL11A, HbF production improves dramatically, providing a potential new target for treatments.
The UK Home Office is responsible for the health care of children in detention centres, where many receive inadequate education and healthcare. The Lancet Editorial highlights the appalling failures in care, including missed childhood immunisations and inadequate treatment for illnesses like malaria and sickle-cell disease.
A new study published in Biology of Blood and Marrow Transplantation demonstrates that a reduced-intensity conditioning bone marrow transplant can eliminate symptoms of sickle cell disease in six out of seven patients. This approach eliminates life-threatening side effects associated with traditional bone marrow transplants.
A nationwide clinical trial is underway to investigate the use of unrelated donor marrow and cord blood transplants for severe sickle cell disease. The trial, facilitated by the Blood and Marrow Transplant Clinical Trials Network, aims to extend a promising treatment option to more severely affected patients.
Researchers identified five gene variants that could predict sickle cell disease severity and offer potential therapeutic targets. These variants influence fetal hemoglobin levels, which affect symptom severity in the disease.
A literature review by Johns Hopkins researchers found that hydroxyurea significantly reduces the frequency of intense sickle cell crises and hospital admissions by 68-84% and 18-32%, respectively. However, potential risks include impaired sperm development and unclear effects on leukemia risk.
A study of children with sickle cell disease found a significant decline in lung function over time, with an average loss of 2 percent per year of total lung capacity. The decline is characterized by a restrictive pattern, and certain hemoglobin-beta genotypes are associated with increased risk.
Researchers at the University of North Carolina at Chapel Hill have discovered aggregates of red and white blood cells in patients with sickle cell disease. These clumps may play a role in blocking small blood vessels, causing sudden painful episodes.
A new study found that bosentan prevents experimentally induced vaso-occlusive crises in a mouse model of SCD, reducing blood vessel resistance and preventing organ damage. Bosentan may provide a new way to limit VOCs and thereby reduce mortality and morbidity in individuals with Sickle Cell Disease.
Researchers found that low blood oxygen saturation increases the risk of stroke in children with sickle cell disease. A commonly used predictive model was deemed inadequate, highlighting the need for better screening tools.
Researchers have identified a novel small molecule therapy called senicapoc that shows efficacy in maintaining hydration of red blood cells and increasing hemoglobin levels in patients with sickle cell anemia. Senicapoc, a Gardos channel blocker, works by limiting solute and water loss, thereby preserving RBC hydration.
A new study found that daily pain is far more prevalent and severe than previously thought in adults with sickle cell disease. Over half of the patients reported having pain on most days, with nearly one-third experiencing pain nearly every day.
A Phase II study found that blood transfusion therapy helped shrink brain lesions caused by silent strokes in children with sickle cell disease. The therapy eliminated one lesion completely and showed no new silent strokes after two years. Researchers hope to preserve cognitive function in these children through prolonged transfusions.
Researchers discover that lenalidomide and pomalidomide can increase fetal hemoglobin production in people with sickle-cell disease, potentially providing a new therapy. These findings also suggest the possibility of using these drugs to treat other beta-hemoglobinopathies.
A small study found that eptifibatide, an intravenous blood thinner, appears to be well-tolerated in sickle cell patients and shows potential benefits in reducing inflammation. The results suggest that the drug may have anti-inflammatory effects without causing harm to these patients.
Researchers developed a predictive model using Bayesian network modeling to estimate sickle cell disease severity and predict mortality risk. The model identified laboratory markers and clinical events contributing to the risk of death, providing a personalized disease severity score for therapeutic decisions.
A vaccine introduced in 2000 has significantly reduced the rate of a serious bacterial illness among young children with sickle cell disease (SCD) in the US. The pneumococcal conjugate vaccine has dropped the rate by over 90% among children under 5 years old.
A nationally representative study of over 2,000 people aged 65+ found snacking is crucial to ensure adequate calorie consumption. Researchers also discovered poor dietary intakes among children with sickle cell disease, including low vitamin and mineral levels.
Researchers developed a new imaging technique to capture the movement of white blood cells in real-time, allowing for a better understanding of Sickle Cell disease. The technique, called digital multi-channel videomicroscopy, has the potential to aid investigations and develop therapeutics for this debilitating disease.
Children with sickle cell disease who experienced major complications such as pain and lung disease early in life are not at greater risk for stroke or death during later childhood, according to new research. Children hospitalized for chest problems early on are more likely to see those problems recur up to adulthood.
Researchers at VCU have developed a unique anti-sickling agent called 5-HMF, which has shown promise in treating sickle cell disease by increasing the oxygen affinity of both normal and sickle hemoglobin. This breakthrough could lead to new drug treatments for the debilitating genetic blood disorder.
A hormone called BNP detects pulmonary hypertension in sickle cell disease patients and predicts mortality. High BNP levels correlate with greater pressure in pulmonary arteries and increased risk of death.