Publications by authors named "J P Panse"
Complement C5 inhibitor treatment with ravulizumab or eculizumab for paroxysmal nocturnal hemoglobinuria (PNH) improves outcomes and survival. Some patients remain anemic due to clinically significant extravascular hemolysis (cs-EVH: hemoglobin [Hgb] ≤9.5 g/dL and absolute reticulocyte count [ARC] ≥120×109/L).
View Article and Find Full Text PDFIntroduction: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired, non-malignant hematologic disease characterized by complement-mediated hemolysis (with or without hemoglobinuria), fatigue, increased susceptibility to thrombosis, and bone marrow dysfunction. The development of complement inhibitors has transformed outcomes for patients with PNH, but patients may still experience pharmacodynamic breakthrough hemolysis (BTH), which can be caused by exposure to a complement amplifying condition (CAC), such as vaccination, infection, or surgery.
Materials And Methods: A 13-member expert panel used a validated methodology (a RAND/UCLA modified Delphi panel) to develop consensus on how to classify pharmacodynamic BTH in patients with complement-inhibitor treated PNH.
Introduction: Men are generally more susceptible to bacterial infections than women. Central venous catheters (CVCs), often used to administer systemic treatment in patients with cancer, are an important source of infection. However, little is known about sex-specific differences of CVC-related bloodstream infections (CRBSIs) in patients with cancer.
View Article and Find Full Text PDFParoxysmal Nocturnal Hemoglobinuria, Pathophysiology, Diagnostics, and Treatment.
Transfus Med Hemother
October 2024
Article Synopsis
- Paroxysmal nocturnal hemoglobinuria (PNH) causes intravascular hemolysis due to a lack of complement regulation, leading to symptoms like anemia, pain, and fatigue, but can be treated with complement inhibitors to reduce disease-related complications and mortality.
- Eculizumab and ravulizumab, terminal complement inhibitors, improve survival rates, but many patients still experience issues from extravascular hemolysis, necessitating new treatment options.
- Proximal complement inhibitors (like pegcetacoplan, danicopan, and iptacopan) can help normalize hemoglobin levels and enhance quality of life for patients with significant extravascular hemolysis, though a clear treatment algorithm for choosing among
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.