Publications by authors named "Michael Geffner"
Article Synopsis
- Paroxysmal nocturnal hemoglobinuria (PNH) causes uncontrolled complement activation leading to hemolysis, but treatment with C5 inhibitors may not fully prevent blood transfusion needs.
- A phase 2 trial involving 12 eculizumab-treated patients tested danicopan, an oral drug targeting an alternative complement pathway, showing a significant increase in hemoglobin levels and a reduction in transfusions over 24 weeks.
- Danicopan was well-tolerated with manageable side effects and demonstrated improved fatigue scores, indicating its potential as an effective treatment option for PNH patients.
Article Synopsis
- Paroxysmal nocturnal hemoglobinuria (PNH) is a blood disorder caused by a lack of certain complement regulators, leading to serious hemolysis (destruction of red blood cells).
- Danicopan, an innovative oral medication, targets a specific factor in the complement system to help reduce hemolysis in PNH patients.
- In a phase 2 trial with 10 patients, danicopan was shown to significantly lower lactate dehydrogenase levels (an indicator of hemolysis) and was well-tolerated, although two patients discontinued the study for various reasons.
Article Synopsis
- - Complement is crucial for the innate immune system's defense against infections, but improper activation can lead to chronic diseases.
- - Complement Factor D (CFD) is key for activating the Alternative Pathway of complement, which amplifies responses but isn't necessary for other pathways, making it a target for selective inhibition.
- - Recent advancements in CFD inhibitors, especially reversible small molecules like danicopan, show promise for modulating complement activity without increasing infection risk.
Objectives: Pancreatitis is the most common major complication of endoscopic retrograde cholangiopancreatography (ERCP). Inflammatory cytokines are released during acute pancreatitis. Interleukin-10 (IL-10) is a potent inhibitor of cytokines and has been shown to attenuate pancreatitis in animal models and pilot human studies.
View Article and Find Full Text PDFChronic hepatitis C virus (HCV) infection is usually asymptomatic in children, but significant liver disease may occur. We evaluated the efficacy, safety, and pharmacokinetics of interferon alfa-2b and ribavirin in children with chronic HCV. We determined the optimal ribavirin dose in an initial cohort of a phase 1 study and then subsequently used it, in combination with interferon alfa-2b, in a second cohort of this study and a phase 3 trial.
View Article and Find Full Text PDFBackground And Aims: Hepatitis C virus (HCV) reinfection after liver transplantation is frequent and leads to chronic hepatitis and cirrhosis. The use of antiviral therapy in this situation remains controversial. This study aimed to assess the safety and efficacy of interferon alfa-2b plus ribavirin for recurrent hepatitis C following liver transplantation.
View Article and Find Full Text PDFAnterior ischemic optic neuropathy, infarction of the optic nerve head owing to inadequate perfusion through the posterior ciliary arteries, is a common cause of visual loss in adults but is rarely reported in children, in part because the diagnosis is overlooked. We report two cases of young children undergoing chronic peritoneal dialysis, who suffered bilateral visual loss from anterior ischemic optic neuropathy. Predisposing local anatomic and multiple systemic factors included a small optic nerve head with little cupping, possible intraocular hypertension, and systemic hypotension, hypovolemia, and anemia.
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