SCD News Briefs

thrombosis

Direct Oral Anticoagulants as an Alternative Treatment for SCD

Direct oral anticoagulants compare favorably with vitamin K antagonists in lowering bleeding rates in patients with sickle cell disease (SCD), according to a study published in Blood Advances. A major complication of SCD is the risk of venous thromboembolism, which is associated with a higher risk of mortality. Scientists have discovered that all 3 components…

Stroke

Prophylactic Hydroxyurea Linked With Reduced Stroke Risk in SCD

Prophylactic use of hydroxyurea in children with sickle cell disease (SCD) significantly reduces middle cerebral artery blood velocity as assessed by transcranial doppler ultrasound (TCD), thus potentially reducing stroke risk in these patients, as published in the Journal of Clinical Medicine. The researchers provided further evidence supporting the use of hydroxyurea in patients with SCD,…

sickle cell disease

Proposed Genome Editing Technology Aims to Transform SCD Treatment

Precision BioSciences will use their novel genome editing technology called ARCUS® to develop a potentially curative treatment for sickle cell disease (SCD) and other hemoglobinopathies in collaboration with Novartis Pharma. The technology will use a custom nuclease to insert, in vivo, a therapeutic transgene at a “safe harbor” location in the genome. “We believe that…

sleep

Sleep Disturbances Affect Neurocognitive Function in SCD

Sleep disturbances such as decreased oxygen saturation may lead to neurocognitive dysfunction in patients with sickle cell disease (SCD), according to a study recently published in Sleep Medicine. Specifically, nocturnal oxygen saturation (SpO2) seems to be independently related to verbal comprehension and working memory, which are characteristically diminished in these patients, as Tucker and the…

pregnant woman

Children Born to Women With SCD Face Increased Risk of ADHD and Obesity

A new study comparing the developmental outcomes of children born to women with sickle cell disease (SCD) with those of matched controls found the exposed children to be at higher risk of developing attention-deficit/hyperactivity disorder (ADHD) and obesity. The study, published in eJHaem, used data from the Boston Birth Cohort (BBC), a prospective birth cohort…

FDA orphan drug designation

FDA Grants Two Designations for Novel SCD Therapies Under Investigation

Global Blood Therapeutics (GBT) announced that 2 novel treatments for sickle cell disease (SCD), inclacumab and GBT021601 (GBT601), were granted Orphan Drug and Rare Pediatric Disease designations by the US Food and Drug Administration (FDA). Inclacumab, a novel P-selectin inhibitor, is being studied in 2 trials to assess its ability to reduce vaso-occlusive crises (VOCs) and…

malaria

Malaria Linked With the Exacerbation of Sickle Cell Disease

Ghanaian researchers have discovered that malaria can induce vaso-occlusive crises in patients with sickle cell disease, requiring them to receive prompt treatment to prevent further clinical deterioration, according to a study published in PLoS One. Sickle cell disease is a particularly pressing problem in Ghana, affecting about 5000 newborns per year. From 2013 to 2014,…

mitochondria

Retention of Functional Mitochondria in RBCs May Lead to SCD Complications

Functional mitochondria are retained in red blood cells (RBCs) of patients with sickle cell disease (SCD), possibly causing immune responses and leading to complications, according to an article published in the British Journal of Haematology. Patients with SCD had a significant increase in mature RBCs (CD71–) staining positive for mitochondria (MitoTracker+) than healthy blood donors…

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