Inborn errors of immunity reveal molecular requirements for generation and maintenance of human CD4 IL-9-expressing cells.

Background: CD4 T cells play essential roles in adaptive immunity. Distinct CD4 T-cell subsets-T1, T2, T17, T22, T follicular helper, and regulatory T cells-have been identified, and their contributions to host defense and immune regulation are increasingly well defined. IL-9-producing T9 cells were first described in 2008 and appear to play both protective and pathogenic roles in human immunity.

Reducing Hospital Length of Stay and Hepatic Artery Thrombosis Rates for Children Receiving a Liver Transplant: A Single-Center Experience From 2000 to 2021.

Background: Pediatric liver transplantation is a very resource-intensive therapy. This study aimed to identify the changes made between two epochs of management and analyze their influence on length of stay (LOS).

Methods: Data from a single center were obtained from the liver transplant and Pediatric Intensive Care Unit (PICU) databases for 336 transplants (282 children) performed between 2000 and 2021.

Event-free survival of maralixibat-treated patients with Alagille syndrome compared to a real-world cohort from GALA.

Background And Aims: Alagille syndrome (ALGS) is characterized by chronic cholestasis with associated pruritus and extrahepatic anomalies. Maralixibat, an ileal bile acid transporter inhibitor, is an approved pharmacologic therapy for cholestatic pruritus in ALGS. Since long-term placebo-controlled studies are not feasible or ethical in children with rare diseases, a novel approach was taken comparing 6-year outcomes from maralixibat trials with an aligned and harmonized natural history cohort from the G lobal AL agille A lliance (GALA) study.

Long-term outcomes of liver transplantation for homozygous familial hypercholesterolaemia in Australia and New Zealand.

Background And Aims: Homozygous familial hypercholesterolaemia (FH) causes severe cardiovascular disease from childhood. Conventional drug therapy is usually ineffective; lipoprotein apheresis (LA) is often required. Liver transplantation (LT) can correct the metabolic defect but is considered a treatment of last resort.