Pediatricians

Study Sheds Light on Pathology of End-Stage Lung Disease in IPF and CF

Adaptive and innate lymphocyte populations contribute to interleukin (IL)-17A-dependent pathologies in the end-stage of chronic lung diseases such as idiopathic pulmonary fibrosis (IPF) and cystic fibrosis (CF), according to a new study published in Clinical and Translational Immunology. The study also showed that pathogens drive the secretion of IL-17A. IL-17A and IL-22 play an important…

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…

Scintigraphy Scan

Bone Scintigraphy Facilitates Early Diagnosis of SM

Employing Tc-99m hydroxyl diphosphonate (HDP) bone scintigraphy can be useful in diagnosing systemic mastocytosis (SM), according to a case report published in the International Journal of Paediatric Orthopaedics. The researchers observed diffuse skeletal uptake on the bone scan, and a bone biopsy revealed mast cell infiltration. “Diagnosing [SM] remains challenging due to the contradictory results…

vertebrae

Study Finds Decreased Vertebral Height in Children With SMA

Newly characterized anatomic variations in the vertebral bodies of children with spinal muscular atrophy (SMA) may not only explain the spinal deformity frequently seen in these patients, but also influence their treatment, according to a study recently published in World Neurosurgery. “Surgeons should be aware of anatomical differences for choosing implant devices accordingly,” the authors…

anxiety

High Prevalence of Anxiety and Depression Found Among People With CF

Researchers confirmed that the prevalence of depression and anxiety among individuals with cystic fibrosis (CF) and their caregivers remains worryingly high, as published in Social Psychiatry and Psychiatric Epidemiology. One of the major shifts that have taken place in CF care is the slow transition of the disease from one that is severely time-limiting to…

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…

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