Results 41 to 50 of about 10,942 (165)
European Journal of Haematology, EarlyView.ABSTRACT This retrospective, single‐center study aimed to characterize clonal dynamics of GPI‐deficient cells in patients with paroxysmal nocturnal hemoglobinuria (PNH) or PNH/aplastic anemia (AA) syndrome using multiparameter flow cytometry including FLAER.
Sandra M. Frey +6 more
wiley +1 more source
Nationwide study of eculizumab in paroxysmal nocturnal hemoglobinuria: Evaluation of treatment indications and outcomes [PDF]
, 2023 Eculizumab is an effective treatment for paroxysmal nocturnal hemoglobinuria (PNH). However, considering the risk of life-threatening meningococcal disease, life-long duration and costs, there are strict criteria for initiation of therapy.
the Dutch PNH Working Group
core
Blood Red Cells & Iron: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired hematologic disease characterized by complement-mediated hemolysis with or without overt hemoglobinuria and is associated with bone marrow failure (BMF).
Vinod Pullarkat +13 more
doaj +1 more source
European Journal of Haematology, EarlyView.ABSTRACT Paroxysmal nocturnal hemoglobinuria (PNH) is a rare hematologic disorder caused by a defect of glycosylphosphatidyl‐anchored proteins, leading to an uncontrolled complement‐mediated hemolysis. The advent of complement inhibitors in clinical practice radically changed patients' outcomes and survival.
Bruno Fattizzo +8 more
wiley +1 more source
Predictive Factors of Mortality in Population of Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH): Results from a Korean PNH Registry. [PDF]
, 2017 Paroxysmal nocturnal hemoglobinuria (PNH) is a progressive, systemic, life-threatening disease, characterized by chronic uncontrolled complement activation. A retrospective analysis of 301 Korean PNH patients who had not received eculizumab was performed
민유홍 +7 more
core +1 more source
Internal Medicine Journal, EarlyView.Abstract Complement inhibitor therapy carries a risk of serious infections, including meningococcal disease. Here we provide evidence‐based recommendations and expert consensus for immunisation and prophylactic treatment of patients receiving, or planning to receive, complement inhibitors for neurological conditions in the Australian setting.
Katherine A. Buzzard +13 more
wiley +1 more source
Immunology, EarlyView.We identify four distinct C5 inhibitory epitopes, including two novel antibodies that selectively block membrane attack complex (MAC) assembly while preserving C5 cleavage and C5a generation. These findings establish selective MAC inhibition as a new therapeutic strategy for complement‐mediated diseases.
Rebekah Sian Cooke +5 more
wiley +1 more source
Transfusion, EarlyView.Abstract Background Paroxysmal nocturnal hemoglobinuria (PNH) is a clonal hematopoietic stem cell disorder caused by somatic mutations in the PIGA gene, resulting in loss of glycosylphosphatidylinositol (GPI)‐anchored proteins, including the complement regulatory proteins, CD55 and CD59.
Ganesh Raman +4 more
wiley +1 more source
The importance of terminal complement inhibition in paroxysmal nocturnal hemoglobinuria
Therapeutic Advances in Hematology, 2022 Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, chronic hematologic disorder associated with inappropriate terminal complement activity on blood cells that can result in intravascular hemolysis (IVH), thromboembolic events (TEs), and organ damage ...
Austin G. Kulasekararaj +4 more
doaj +1 more source
American Journal of Hematology, Volume 101, Issue 10, Page 2520-2532, October 2026.ABSTRACT The RACE study (NCT02009747) compared horse antithymocyte globulin (hATG) plus cyclosporine A (CsA) ± eltrombopag as initial immunosuppressive treatment (IST) for severe aplastic anemia. Here we report the final 2‐year analysis of this prospective randomized phase III study.
Antonio M. Risitano +52 more
wiley +1 more source