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Safety and efficacy of tamoxifen in non-ambulant patients with Duchenne muscular dystrophy: a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial (TAMDMD Group B).
Neuromuscul Disord
January 2025
Division of Neuropediatrics and Developmental Medicine, University Children's Hospital Basel (UKBB), University of Basel, Basel, Switzerland. Electronic address:
Most patients with Duchenne muscular dystrophy (DMD) are non-ambulant. Preserving proximal motor function is crucial, rarely studied. Tamoxifen, a selective oestrogen receptor modulator, reduced signs of muscular pathology in a DMD mouse model.
View Article and Find Full Text PDFWhole blood exchange ameliorates acute hemolytic anemia by reducing inflammation and oxidative stress in rats.
FASEB J
January 2025
Department of Blood Transfusion, Xiangya Hospital, Central South University, Changsha, China.
Hemolytic anemia (HA) is characterized by massive destruction of red blood cells (RBCs) and insufficient oxygen supply, which can lead to shock, organ failure, even death. Recent studies have preliminarily demonstrated the therapeutic effectiveness of whole blood exchange (WBE) in the management of acute hemolytic anemia and exhibited potential for reducing the duration of corticosteroid treatment, while the underlying mechanism of WBE therapy was not investigated in preclinical study. Hence, we investigate the therapeutic mechanisms of WBE in HA through established continued WBE therapy in rats creatively.
View Article and Find Full Text PDFMild liver injury following withdrawal of long-term prednisone therapy: A case report.
World J Gastroenterol
January 2025
Department of Biochemistry and Molecular Biology, School of Basic Medical Sciences, Zhengzhou University, Zhengzhou 450001, Henan Province, China.
Background: Liver injury manifesting as hepatic enzyme abnormalities, has been occasionally identified to be a feature of primary or secondary Addison's disease, an uncommon endocrine disease characterized by adrenal insufficiency. There have been no more than 30 reported cases of liver injury explicitly attributed to Addison's disease. Liver injury resulting from adrenal insufficiency due to glucocorticoid withdrawal is exceptionally rarer.
View Article and Find Full Text PDFComparison of two strategies of glucocorticoid withdrawal in patients with rheumatoid arthritis in low disease activity (STAR): a randomised, placebo- controlled, double-blind trial.
Ann Rheum Dis
January 2025
Rheumatology Center, Toulouse University Hospital, Toulouse, France.
Objectives: To compare two strategies-a hydrocortisone replacement strategy and a prednisone tapering strategy-for their success in glucocorticoid discontinuation in patients with rheumatoid arthritis (RA) with low disease activity (LDA).
Methods: The Strategies for glucocorticoid TApering in Rheumatoid arthritis (STAR) study was a double- blind, double-placebo randomised controlled trial including patients with RA receiving a stable dose of glucocorticoid 5 mg/day for ≥3 months and were in LDA for ≥3 months. Patients were randomly assigned in a 1:1 ratio to either replace prednisone with 20 mg/day of hydrocortisone for 3 months, then reduce to 10 mg/day for 3 months before discontinuation or to taper prednisone by 1 mg/day every month until complete discontinuation, contingent on maintaining LDA.
Systematic Review of the Presentation, Treatment, and Outcome of Chronic Disseminated Candidiasis in Children with Cancer or Following Hematopoietic Cell Transplant.
Pediatr Blood Cancer
January 2025
Infectious Diseases Department, Royal Children's Hospital, Parkville, Victoria, Australia.
Chronic disseminated candidiasis (CDC) is a rare complication of immunosuppression. This review describes the presentation, management, and outcomes of CDC in pediatric patients with cancer or following hematopoietic cell transplant (HCT). PubMed, Embase, and Medline were searched identifying 32 studies, describing 95 cases of CDC.
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