Lipoprotein apheresis improves cardiovascular survival in HoFH
Patients with homozygous familial hypercholesterolaemia who initiate lipoprotein apheresis during childhood and adolescence have a reduced long-term risk for atherosclerotic cardiovascular disease and death, shows follow-up of two international registries.
Pharmacogenomic-guided induction therapy might reduce pediatric AML racial disparities
Using a pharmacogenomic strategy to guide choice of induction therapy for acute myeloid leukaemia might help overcome the disparity in outcomes between Black and White children, suggests research published in JAMA Network Open.
Maralixibat improves itch, bile acid concentrations in paediatric PFIC
Treatment with the ileal bile acid transporter inhibitor maralixibat relieves morning itch for children with different types of progressive familial intrahepatic cholestasis, show phase 3 study findings published in The Lancet Gastroenterology and Hepatology.
Scoring system devised for stratifying children with suspected Sjögren’s syndrome
Researchers have devised the Florida Scoring System to help clinicians stratify children with suspected Sjögren’s syndrome into three distinct classes, thereby improving diagnosis and disease monitoring.
SMA newborn screening improves functional outcomes
Children diagnosed with spinal muscular atrophy through a newborn screening initiative have better functional outcomes than their peers diagnosed after the development of clinical symptoms, the SMARTCARE study findings show.
‘No substantive increase’ in ASD with prenatal topiramate exposure
Children born to women with epilepsy who take topiramate during the second half of their pregnancy do not have a substantially increased risk of autism spectrum disorder, suggests research published in The New England Journal of Medicine.
Sepsis during paediatric leukaemia therapy linked to long-term neurocognitive dysfunction
Severe sepsis during treatment for childhood leukaemia may be associated with an increased risk of moderate-to-severe neurocognitive dysfunction in adulthood, US Study findings indicate.
Umbilical hernia repair ‘should be delayed’ until after age 5 years
The majority of umbilical hernias close spontaneously regardless of size, US researchers have found.
FOXO1 fusion status aids paediatric local rhabdomyosarcoma risk assessment
Considering FOXO1 fusion status improves the risk assessment of children with localised rhabdomyosarcoma, suggest study findings published in Cancer.
Home insulin pump use feasible for hospitalised children with type 1 diabetes
For children with type 1 diabetes who are admitted to hospital, continuing with a home insulin pump is feasible for the prevention of hyperglycemia and hypoglycemia, suggests research published in JAMA Network Open.
