Publications by authors named "Monika Oliver"
Article Synopsis
- Paroxysmal nocturnal haemoglobinuria (PNH) is a rare blood disorder causing serious issues due to chronic hemolysis and can significantly affect patients' quality of life.
- A post hoc analysis evaluated the effectiveness of pegcetacoplan, a targeted complement C3 inhibitor, in PNH patients with poor bone marrow function from the PEGASUS and PRINCE studies.
- Results showed that while normalisation of certain health parameters was challenging, a significant percentage of patients experienced clinically meaningful improvements in hemoglobin, LDH levels, and fatigue after treatment with pegcetacoplan.
Systemic lupus erythematosus (SLE) can present with a diverse array of hematologic manifestations, among which atypical hemolytic uremic syndrome (aHUS) is a rare entity. SLE-triggered aHUS has significant morbidity and mortality without timely intervention, yet its frequency remains uncertain and optimal strategies for complement-directed therapies are largely expert-driven. We performed a comprehensive literature review and present a case of a 23-year-old female newly diagnosed with SLE/class IV lupus nephritis who developed aHUS that rapidly responded to the C5 antagonist, eculizumab.
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- Paroxysmal nocturnal hemoglobinuria (PNH) is a rare blood disorder that leads to anemia, increased risk of blood clots, and organ damage, with a high mortality rate when only supportive care is provided.
- The introduction of targeted therapies like eculizumab and newer options such as ravulizumab and pegcetacoplan have significantly improved patient outcomes by increasing hemoglobin levels and reducing complications.
- Despite advances in treatment, challenges remain, including managing breakthrough hemolysis and infection risks, as well as ensuring patient adherence to therapies that are increasingly self-administered and complex.
Article Synopsis
- Paroxysmal nocturnal haemoglobinuria (PNH) is a rare blood disorder that leads to severe fatigue and decreased quality of life, making effective treatment essential.
- A post hoc analysis of the PEGASUS phase 3 trial compared the effectiveness of two treatments, pegcetacoplan and eculizumab, specifically focusing on improvements in patient-reported fatigue using the FACIT-fatigue scale.
- Results showed that patients receiving pegcetacoplan had significantly greater improvements in fatigue and related health measures compared to those receiving eculizumab, indicating pegcetacoplan is a more effective treatment option for managing fatigue in PNH.