ASH published new clinical guidelines on the prevention, diagnosis, and treatment of CNS complications for children and adults with sickle cell disease.
The FDA has granted Priority Review for the Biologics License Application (BLA) for sutimlimab for the treatment of hemolysis in adult patients with cold agglutinin disease, a rare type of autoimmune hemolytic anemia.
Although SCD is often linked to microvasculature occlusion, some sequelae are caused by erythrocyte membrane weakening.
Children with Diamond-Blackfan anemia may benefit from HSCT when a matched sibling donor or matched unrelated donor is not available.
The studies compared the efficacy and safety of vadadustat to darbepoetin alfa in adult patients on dialysis with anemia due to CKD.
What factors influence transplant outcomes in patients with FA with marrow failure that develops into MDS and AL?
Parents of infants and young children with sickle cell disease were satisfied with the patient-centered medical home model experience in the newborn cohort clinic setting.
Although RPI has been a good tool for evaluating of adult bone marrow erythropoietic response to anemia, it is not an adequate indicator in the pediatric population.
Researchers found that individuals with sickle cell disease have lower HIV infection rates, slower progression to AIDS, and lower HIV-associated mortality.
Febrile neutropenia commonly causes morbidity and mortality in children with severe aplastic anemia but little is known about the spectrum of infections.
The Food and Drug Administration (FDA) has granted Orphan Drug designation to FT-4202 (FORMA Therapeutics) for the treatment of sickle cell disease (SCD).
A special commentary article explained that acute kidney injury can be a complication of vaso-occlusive episodes associated with sickle cell disease.
The FDA has approved Reblozyl® (luspatercept–aamt; Bristol-Myers Squibb and Acceleron) for the treatment of anemia in adults with lower-risk myelodysplastic syndromes.
The FDA has approved the New Drug Application for Triferic AVNU®.
Among those with baseline anemia, hemoglobin levels improved with canakinumab versus placebo.
The authors included 6423 patients with SCD in what is believed to be the first study of the incidence of bleeding events in this patient population.
Treatment with androgen deprivation therapy alone is associated with increased risks for anemia compared with radical prostatectomy alone, a study found.
Patients who received hydroxycarbamide therapy scored higher on several measures of neurocognition compared to patients who did not receive hydroxycarbamide.
Using data from a nationwide registry, researchers characterized the clinical profiles of patients with Diamond-Blackfan anemia.
Gender, alcohol consumption, and levels of C-reactive protein and fibrinogen were found to be associated with prevalence of nonanemic absolute iron deficiency.
Patients with sickle cell disease and vitamin D deficiency reported higher rates of emergency room visits and hospital admissions for pain crises.
A panel of researchers developed evidence-based guidelines to support clinical decision making regarding transfusion support for patients with sickle cell disease.
Patients with autoimmune hemolytic anemia who underwent splenectomy experienced increased cumulative incidences of thromboembolism and sepsis.
Younger women had lower thresholds of serum ferritin increase necessary for a red blood cell measurement increase compared with older women and men.
Researchers assessed the safety and efficacy of a pretransplant conditioning regimen in patients with aplastic anemia undergoing allogeneic transplantation.
Of astronauts who went on space missions of short, medium, and long duration, 7%, 10%, and 48%, respectively, were anemic upon return to Earth.
Thrombotic events and sepsis occurred more frequently in patients who underwent splenectomy and were additionally associated with increased mortality risk.
Patients who underwent moderate-intensity endurance training experienced improvements in capillary density and number of capillaries around a fiber.
A multicenter, randomized, parallel group, double-blind, placebo-controlled trial found that canakinumab may be well tolerated by pediatric patients with sickle cell anemia.
Researchers found that the addition of eltrombopag to standard immunosuppression therapy did not provide therapeutic benefit to pediatric patients with SAA.
Patients younger than 10 years of age experienced an overall survival rate of 91% regardless of whether they received transplant from a sibling or unrelated donor.
Clinicians reported informing only 42% of families about hematopoietic cell transplantation, citing concerns about safety and cost.
The FDA has approved Adakveo (crizanlizumab-tmca; Novartis) to reduce the frequency of vaso-occlusive crisis (VOC) in patients aged ≥16 years with sickle cell disease.
Individuals with sickle cell disease were estimated to earn nearly $700,000 less over their lifetime compared with similar individuals without sickle cell disease.
The FDA has approved Reblozyl (luspatercept–aamt; Celgene and Acceleron) for the treatment of anemia in adult patients with beta thalassemia who require regular red blood cell transfusions.
Postoperative anemia was significantly associated with a composite end point of mortality and major ischemic outcomes within 90 days.
Researchers found similar incidences of late events in patients who were treated with and without granulocyte colony-stimulating factor.
Approximately two-thirds of patients who completed a screening questionnaire reported having at least 1 unmet socioeconomic need.
The FDA has approved Ultomiris (ravulizumab-cwvz; Alexion) for the treatment of atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy (TMA) in adult and pediatric patients aged 1 month and older.
More than two-thirds of patients receiving horse antithymocyte globulin plus cyclosporine achieved a very good partial response or better.
Children born to mothers diagnosed with iron-deficiency anemia prior to 30 weeks of gestation were more likely to have a neurodevelopmental disorder.
Nearly two-thirds of ischemic strokes occurred in patients who had not properly completed the STOP protocol.
Low and high hemoglobin levels were associated with increased risk for dementia, white matter hyperintensity volume, and structural connectivity.
In a study of chronic kidney disease patients undergoing vascular access surgery for hemodialysis, severe preoperative anemia was associated with worse outcomes.
Depressive symptoms, spirituality, and missed clinic appointments were all found to correlate with hospital admission in sickle cell disease.
Second-line treatment strategies for severe aplastic anemia include stem cell transplant, immunosuppressive therapy, and thrombopoietin mimetics.
The primary end point was time to readiness for discharge, defined as the difference between the readiness-for-discharge date and time and the start date and time of the first infusion of study drug.
Patients with secondary Evans syndrome demonstrated increased mortality compared with all other cohorts.
Study seeks to recruit up to 45 adults with severe sickle cell disease
Patients with chronic anemia demonstrated a decrease in brain white matter volume proportional to anemia severity.
The FDA has approved Accrufer (ferric maltol; Shield Therapeutics) for the treatment of iron deficiency in adults.