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Thrombosis in Paroxysmal Nocturnal Hemoglobinuria (PNH): From Pathogenesis to Treatment.
Int J Mol Sci
November 2024
Second Propedeutic Department of Internal Medicine, Aristotle University of Thessaloniki, 54642 Thessaloniki, Greece.
Paroxysmal Nocturnal Hemoglobinuria (PNH) constitutes a rare bone marrow failure syndrome characterized by hemolytic anemia, thrombotic events (TEs), and bone marrow aplasia of variable degrees. Thrombosis is one of the major clinical manifestations of the disease, affecting up to 40% of individuals with PNH. Venous thrombosis is more prevalent, affecting mainly unusual sites, such as intrabdominal and hepatic veins.
View Article and Find Full Text PDFBone impairment in atypical hemolytic and uremic syndrome treated by long-term eculizumab.
Pediatr Nephrol
October 2024
Centre de Référence Des Maladies Rénales Rares, Centre de Référence Des Maladies Rares du Calcium Et du Phosphore, Filières Maladies Rares ORKID Et OSCAR, Hospices Civils de Lyon & Université Claude-Bernard Lyon 1, Lyon, France.
Article Synopsis
- - Atypical hemolytic uremic syndrome (aHUS) is a condition caused by issues with the complement system, particularly due to Factor H deficiency, and is typically treated with eculizumab for life.
- - Two young patients with Factor H deficiency on long-term eculizumab therapy exhibited unusual bone issues, including pain and deformities, with diagnostic imaging revealing active bone remodeling and C3c accumulation.
- - The bone alterations observed may either be a side effect of eculizumab treatment or a result of the deficiency of Factor H, indicating a need for further research into the bone health of aHUS patients.
[Not Available].
Cancer Rep (Hoboken)
October 2024
Haematology Unit, Careggi University Hospital, Florence, Italy.
Article Synopsis
- - Thrombotic microangiopathy (TMA) is a serious syndrome linked to microangiopathic hemolytic anemia, low platelet count, and organ damage, particularly in the kidneys, and can be induced by the drug carfilzomib, used for treating multiple myeloma.
- - The report details two cases of multiple myeloma patients who developed TMA while on carfilzomib treatment; both patients saw significant improvements in kidney function and platelet count after receiving eculizumab.
- - In a review of 91 patients receiving carfilzomib, a 2.2% incidence of drug-induced TMA was found, highlighting the need for prompt recognition and management, which includes
A pediatric case with hemolytic uremic syndrome associated with COVID-19, which progressed to end-stage kidney disease.
Turk J Pediatr
May 2024
Department of Pediatric Nephrology, Faculty of Medicine, Mersin University, Mersin, Türkiye.
Background: Hemolytic uremic syndrome (HUS) is a serious cause of acute kidney injury in children. There is a suggestion that coronavirus disease 2019 (COVID-19) may be a trigger for HUS. In this study, we present a pediatric case diagnosed with HUS associated with COVID-19, which progressed to end-stage kidney disease.
View Article and Find Full Text PDFDiagnosis and Management of Pediatric Neuropsychiatric Systemic Lupus Erythematosus: An Update.
Paediatr Drugs
July 2024
Department of Pediatric Rheumatology, Hacettepe University Medical Faculty, Sihhiye Campus, 06100, Ankara, Turkey.
Article Synopsis
- * While MRI is useful for detecting major neurological issues, it often lacks the resolution to identify smaller problems, necessitating the use of advanced imaging techniques like PET or SPECT.
- * Treatment mainly focuses on relieving symptoms and addressing the underlying disease, with strong immunosuppressants like cyclophosphamide and rituximab being recommended, along with ongoing research into new biologic therapies.
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