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Atypical hemolytic uremic syndrome (aHUS) is an important differential diagnosis in thrombotic microangiopathy (TMA). The absence of definitive biomarkers usually allows for aHUS to be diagnosed only through a process of exclusion. Due to the unfavorable prognosis if adequate therapy is delayed or not provided, differential diagnostic considerations and initiation of treatment must occur promptly.
View Article and Find Full Text PDFComplement-Mediated Hemolytic Uremic Syndrome Due to MCP/CD46 Mutation: A Case Report.
J Investig Med High Impact Case Rep
January 2025
Marshall University, Huntington, WV, USA.
Thrombotic microangiopathy (TMA) is a severe condition characterized by microangiopathic hemolytic anemia, thrombocytopenia, and end-organ damage, often involving the kidneys. Complement-mediated hemolytic uremic syndrome (cHUS), a rare form of TMA, arises from dysregulated alternative complement pathway activation, frequently due to genetic mutations. We report the case of a 23-year-old male presenting with TMA secondary to a heterozygous mutation in the membrane cofactor protein (MCP/CD46) gene.
View Article and Find Full Text PDFMonocytes serve as Shiga toxin carriers during the development of hemolytic uremic syndrome.
Cell Mol Biol Lett
January 2025
State Key Laboratory of Pharmaceutical Biotechnology, Department of Gastroenterology, Drum Tower Hospital, Nanjing University Medical School, Nanjing University, Nanjing, 210093, Jiangsu, China.
Shiga toxin (Stx)-induced hemolytic uremic syndrome (HUS) poses a life-threatening complication for which a definitive treatment remains elusive. To exert its cytotoxic effect on renal cells, Stx must be delivered from the infected intestines to the kidney. However, the mechanism underlying Stx delivery remains unclear.
View Article and Find Full Text PDFThrombocytopenia in Critically Ill Children: A Review for Practicing Clinicians.
Children (Basel)
January 2025
Division of Critical Care Medicine, Nicklaus Children's Hospital, 3100 SW 62nd Avenue, Miami, FL 33155, USA.
Thrombocytopenia frequently occurs in patients before, during, and after admission to Pediatric Intensive Care Units (PICUs). In critically ill children, it is often due to multifactorial causes and can be a sign of significant organ dysfunction. This review summarizes the potential causes/mechanisms of thrombocytopenia in acutely ill children, their identification, and treatments, with special attention paid to septic patients.
View Article and Find Full Text PDFAdverse drug events (ADEs) risk signal mining related to eculizumab based on the FARES database.
Front Pharmacol
January 2025
The First Department of Specialty Medicine, Inner Mongolia Corps Hospital of The Chinese People's Armed Police Force, Hohhot, China.
Introduction: Eculizumab is a C5 complement inhibitor approved by the FDA for the targeted treatment of four rare diseases, paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), generalized myasthenia gravis (gMG), and aquaporin-4 immunoglobulin G-positive optic neuromyelitis optica spectrum disorders (AQP4-IgG+NMOSD). The current study was conducted to assess real-world adverse events (AEs) associated with eculizumab through data mining of the FDA Adverse Event Reporting System (FAERS).
Methods: Disproportionality analyses, including Reporting Ratio Ratio (ROR), Proportional Reporting Ratio (PRR), Bayesian Confidence Propagation Neural Network (BCPNN), and Multi-Item Gamma Poisson Shrinker (MGPS) algorithms were used to quantify the signals of eculizumab-associated AEs.
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