Paroxysmal nocturnal hemoglobinuria (PNH) is characterized by intravascular hemolysis, thrombosis and bone marrow failure. Prior to the availability of specific therapy, PNH led to the death of around half of affected individuals, mainly through thrombotic complications, with a particular grim prognosis for patients presenting with classic PNH. The anti-C5 monoclonal antibody eculizumab has revolutionized treatment, controlling intravascular hemolysis and thrombosis occurrence, with improved long-term survival. However, eculizumab is infused on a lifelong 2 week basis and most of the patients are anemic, with some remaining transfusion-dependent. New anti-C5 agents reproduce the safety and efficacy of eculizumab, with improved patient convenience, while proximal complement inhibitors have been developed to address C3-mediated extravascular hemolysis, aiming to eventually improve hematological response. This review will describe the spectacular medical progress in PNH of the last 20 years, as well as the risks and benefits of a novel approach.
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| http://dx.doi.org/10.1053/j.seminhematol.2022.01.001 | DOI Listing |
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The First Department of Specialty Medicine, Inner Mongolia Corps Hospital of The Chinese People's Armed Police Force, Hohhot, China.
Introduction: Eculizumab is a C5 complement inhibitor approved by the FDA for the targeted treatment of four rare diseases, paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), generalized myasthenia gravis (gMG), and aquaporin-4 immunoglobulin G-positive optic neuromyelitis optica spectrum disorders (AQP4-IgG+NMOSD). The current study was conducted to assess real-world adverse events (AEs) associated with eculizumab through data mining of the FDA Adverse Event Reporting System (FAERS).
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Department of Pharmacy, Sabya General Hospital, Gazan, SAU.
[This retracts the article DOI: 10.7759/cureus.67830.
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Division of Hematology-Oncology, Hanyang University Seoul Hospital, Seoul, Republic of Korea.
Ravulizumab is a second-generation complement component 5 (C5) inhibitor (C5i) approved for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) following positive results from two pivotal trials in patients with PNH originally naive to C5i treatment and eculizumab-experienced patients with PNH. In both trials, after the 26week primary evaluation period, all patients received ravulizumab for up to 6 years. To report ravulizumab treatment outcomes in patients with PNH originally naive to C5i treatment and eculizumab-experienced patients with PNH treated for up to 6 years.
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Pharmacokinetic characterization and exposure-response relationship of crovalimab in the COMMODORE 1, 2 and 3 and COMPOSER trials of patients with paroxysmal nocturnal haemoglobinuria.
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F. Hoffmann-La Roche Ltd, Basel, Switzerland.
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