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http://dx.doi.org/10.1007/s00403-024-03088-9 | DOI Listing |
J Comp Eff Res
January 2025
ICON plc, Insights, Evidence & Value - Health Economics & Epidemiology, Langen, Germany.
To compare the efficacy and safety of lanadelumab versus other approved long-term prophylaxis (LTP) treatments in patients with pediatric hereditary angioedema (HAE) aged <12 years. A systematic literature review was conducted to identify studies of LTP in patients with HAE aged <12 years. Two studies met the inclusion criteria in an indirect treatment comparison of efficacy and safety data in pediatric HAE patients.
View Article and Find Full Text PDFClin Infect Dis
January 2025
Department of Clinical Microbiology, Rigshospitalet, Copenhagen, Denmark.
The recent US Food and Drug Administration approval of pivmecillinam-an oral prodrug of the amidinopenicillin antibiotic mecillinam-presents a valuable opportunity to address the need for new treatments for uncomplicated urinary tract infection (uUTI). We report findings of a systematic literature review of the safety profile of pivmecillinam/mecillinam based on more than 40 years' experience, mainly in Europe and Canada, to describe its tolerability profile and identify any important safety signals. In total, 110 eligible publications were identified describing use of pivmecillinam/mecillinam as monotherapy or in combination, for treatment of uUTI or other infectious conditions.
View Article and Find Full Text PDFAm J Gastroenterol
January 2025
Division of Gastroenterology and Hepatology, University of Michigan.
The number of alcohol use disorder patients with mild (n=101) and severe alcoholic liver disease (n=112) increased between 2013 and 2023 (p=0.06). Naltrexone was prescribed in 65%, acamprosate 26% and disulfiram 9%.
View Article and Find Full Text PDFMol Genet Metab Rep
March 2025
Translational Medical Center, National Center of Neurology and Psychiatry, Tokyo, Japan.
Background: The efficacy and safety of avalglucosidase alfa for Pompe disease (PD) have been demonstrated in a global Phase 3 trial (COMET) in patients with late-onset PD (LOPD) and a global Phase 2 trial (Mini-COMET) in patients with infantile-onset PD (IOPD). This case series examines the individual results of three Japanese patients enrolled in these trials.
Methods: Case reports were assembled from data collected in the COMET and Mini-COMET trials.
Front Neurol
January 2025
Unidade Local de Saúde de São João, Porto, Portugal.
Background: Anti-CD20 monoclonal antibodies are a class of immunosuppressive drugs widely used in the treatment of central nervous system (CNS) inflammatory diseases, with well-established efficacy and safety. Although rare, these therapies can be associated with serious adverse events including hematological and infectious complications. This study aims to evaluate their safety and tolerability profile in real-world clinical practice.
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