The Agency announced that additional clarification of clinical data was needed prior to completing the review.
Among patients with sickle cell disease, inhibition of BCL11A via gene knockdown is an effect target to induce fetal hemoglobin.
The comparative study aimed to analyze AEs as well as estimate the associated medical costs for 4 IV iron preparations.
Preliminary results suggest that CRISPR-Cas9 editing of BCL11A may benefit patients with sickle cell disease and β-thalassemia by potentially eliminating vaso-occlusive episodes or the need for transfusion.
Investigators aimed to understand patient-reported effects of sickle cell disease on health, wellbeing, and disease management.
For patients with sickle cell disease, HSCT may not be associated with a significant increase in CKD incidence or prevalence following transplantation.
Researchers characterized hospitalization and case fatality rates secondary to COVID-19 among individuals with SCD and compared with the general Black population.
Researchers have identified the binding site for LRF as a therapeutic target for β-hemoglobinopathies.
Researchers evaluated the cumulative incidence of intracranial and extracranial arteriopathy and associated risk factors in children with sickle cell disease.
Investigators compared ovarian reserve and assessed clinical characteristics in young women with sickle cell anemia vs those without the condition.
As physical performance already declines as patients age, how strong is the link between anemia and impacted mobility?
For patients with iron deficiency anemia, oral iron supplementation use alone is comparable to oral supplementation with vitamin C.
Pegcetacoplan is a synthetic cyclic peptide conjugated to a polyethylene glycol polymer that inhibits the complement cascade centrally at C3 and C3b.
Bone marrow transplantation was associated with a relatively low rate of vaso-occlusive crises in patients with sickle cell disease.
Sutimlimab is an investigational monoclonal antibody designed to stop hemolysis by selectively inhibiting C1s in the classical complement pathway.
Endari (L-glutamine; Emmaus Medical) is an amino acid indicated to reduce the acute complications of sickle cell disease in adult and pediatric patients aged 5 years and older.
Patients with sickle cell disease may have an increased risk for venous thromboembolic events, especially pulmonary embolism, following surgical splenectomy.
Study results suggest that brain imaging may be a beneficial part of screening processes for patients with suspected Fanconi anemia.
Study results suggest that for patients with anemia, preoperative iron therapy should not be recommended in major election surgery.
Persistent anemia in the first year after critical illness is common among patients who are hospitalized; however, anemia recovery profiles are needed.
A retrospective analysis suggests that for patients with iron deficiency, intravenous ferumoxytol may be safe and effective when used on a single-dose basis.
Vaso-occlusive crises were linked to an increase in opioid prescriptions in patients with sickle cell disease.
Survey finds variation in practice patterns for obtaining MRI/MRA for stroke prevention and monitoring in patients with sickle cell anemia.
Autologous transplantation with gene-corrected hematopoietic stem cells show engraftment and proliferation in nonconditioned patients with Fanconi anemia.
A current meta-analysis of HIF-PHIs showed that these drugs improve the response rate of hemoglobin and decrease hepcidin levels.
Patients receiving chronic extracorporeal photopheresis therapy are at risk of developing iron deficiency anemia.
In a Japanese study, approximately 1 in 8 CKD patients with anemia failed to respond to darbepoetin alfa therapy.
But no change in opioid use observed for those receiving medical marijuana certification.
Investigators characterized ribosome biogenesis dynamics during erythropoiesis to study the role of ribosome biogenesis in normal erythroid development.
Transferrin saturation and ferritin concentrations improved significantly more in the ferric citrate than ferrous sulfate group over 12 weeks.
Low hemoglobin levels in patients with IgAN increase their risk for end-stage kidney disease, according to new research.
Investigators found sex and racial differences in the risk for end-stage kidney disease among patients with CKD anemia.
The American Society of Hematology released their evidence-based 2020 guidelines on the management of acute and chronic pain in sickle cell disease.
Investigators aimed to determine whether vaporized cannabis would reduce symptoms of chronic pain in adult patients with sickle cell disease.
Couples that are at high risk of having offspring with SCD should be offered pre‐implantation genetic diagnosis treatment to prevent disease transmission.
Investigators compared fixed dose and dose escalation administration of hydroxyurea in pediatric patients with sickle cell anemia.
Although the association between thrombocytosis in iron deficiency anemia is understood, researchers studied the risk for thrombotic complications.
Infection with the virus that causes COVID-19, coupled with use of erythropoiesis-stimulating agents may result in additive prothrombotic effects, according to the authors.
Investigators analyzed graft and survival outcomes among patients with SCD who received liver transplants.
In 2 Japanese trials of nondialysis patients, molidustat proved noninferior to darbepoetin alfa, an erythropoiesis-stimulating agent, in correcting and maintaining hemoglobin levels over 36 weeks.
For patients with SCD, anxiety significantly mediates the effect of sleep disturbances on executive functioning more than depression.
Investigators discuss surgical approaches and outcomes of total and partial splenectomy for the treatment of hereditary hemolytic anemia in children.
A report from the Sickle Cell Disease Implementation Consortium assessed patient-reported outcomes and clinical and psychosocial factors in SCD.
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.