COVID-19 became a global pandemic in 2020 and significantly affected the activity of hematopoietic cell transplants (HCT) worldwide. Despite these challenges, a total of 28,793 transplants, including 18,518 allogeneic and 10,275 autologous transplants, were performed in 719 facilities in 2020 in the Asia-Pacific (AP) region. This represented a 5.
View Article and Find Full Text PDFObjectives: Graft-versus-host disease (GVHD) commonly affects the gastrointestinal (GI) tract among children who undergo hematopoietic stem cell transplantation (HSCT). Studies concerning GI-acute GVHD (aGVHD) in developing countries are limited. Therefore, our aims were (1) to demonstrate clinical characteristics, endoscopic and histopathological findings in children with GI-aGVHD post-HSCT and (2) to compare features, risk factors, and clinical outcomes of children with GI-aGVHD versus non GI-aGVHD.
View Article and Find Full Text PDFJ Clin Res Pediatr Endocrinol
November 2024
Background: Gaucher disease (GD) is one of the most common types of lysosomal storage diseases (LSDs) caused by pathogenic variants of lysosomal β-glucocerebrosidase gene (GBA1), resulting in the impairment of Glucocerebrosidase (GCase) enzyme function and the accumulation of a glycolipid substrate, glucosylceramide (GlcCer) within lysosomes. Current therapeutic approaches such as enzyme replacement therapy and substrate reduction therapy cannot fully rescue GD pathologies, especially neurological symptoms. Meanwhile, delivery of lysosomal enzymes to the endocytic compartment of affected human cells is a promising strategy for treating neuropathic LSDs.
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