Results 31 to 40 of about 2,146 (159)

Ravulizumab: A Review in Atypical Haemolytic Uraemic Syndrome [PDF]

Drugs, 2021
Ravulizumab (Ultomiris®), a humanized monoclonal antibody that inhibits complement protein C5, is indicated for the treatment of atypical haemolytic uraemic syndrome (aHUS) in several countries, including the USA and those of the EU. Ravulizumab has been re-engineered from eculizumab to extend its terminal elimination half-life, resulting in a more ...
openaire   +2 more sources

Transplacental transfer of ravulizumab in a pregnant woman with neuromyelitis optica: a case report

BMJ Neurology Open
Background Neuromyelitis optica spectrum disorder (NMOSD) is a rare relapsing autoimmune disease of the central nervous system (CNS). Management during pregnancy is challenging due to limited safety data for disease-modifying therapies.Case presentation ...
Anu Jacob, Azza Mahmoud, Luai Eldweik, Asia Mubashir   +3 more
doaj   +1 more source

Exploring individualized crovalimab dosing in PNH through in silico modelling: Potential for improved convenience and cost efficiency

British Journal of Clinical Pharmacology, EarlyView.
Abstract Aim Paroxysmal nocturnal haemoglobinuria (PNH) is a rare, acquired haematopoietic stem cell disorder. Crovalimab, a complement C5‐inhibitor, is approved for PNH and can be self‐administered subcutaneously every 4 weeks, offering a more convenient route than intravenous C5‐inhibitors.
Mendy ter Avest, Simon E. Koele, Nicole C. A. J. van de Kar, Saskia M. C. Langemeijer, Rob ter Heine   +4 more
wiley   +1 more source

Deficiency of high‐molecular‐weight von Willebrand factor mitigates thrombo‐complement injury in an LPS‐induced TMA‐like mouse model

VIEW, EarlyView.
Reduction of high‐molecular‐weight von Willebrand factor disrupts the platelet–complement amplification loop, attenuating microvascular thrombosis, complement deposition, endothelial injury, and organ damage in complement‐mediated thrombotic microangiopathy.
Yang Li, Chihao Zhang, Xiaoqian Xu, Sicong Wang, Xiaofan Hu, Junjie Yao, Xi Wu, Difeng Zhu, Qian Liang, Yeling Lu, Yu Liu, Guanqun Xu, Jiakuan Zhou, Qiulan Ding, Wenman Wu, Ya Li, Jing Dai, Xuefeng Wang   +17 more
wiley   +1 more source

Ravulizumab in treatment-naïve patients with atypical hemolytic uremic syndrome: a real-world case series

Drug Target Insights
Introduction: Atypical hemolytic uremic syndrome (aHUS) is a potentially life-threatening condition associated with poor clinical outcomes if not treated adequately.
Livia Maria Sorrentino, Candida Iacuzzo, Raffaella Sciri, Miriam Zacchia, Mariarosaria Iannuzzi, Paola Marotta, Piercarla Vitale, Chiara Taglioni, Floriana Secondulfo, Daniela Palazzetti, Rocco Baccaro   +10 more
doaj   +1 more source

No evidence for hypogammaglobulinemia in patients with paroxysmal nocturnal hemoglobinuria (PNH) chronically treated with ravulizumab.

PLoS ONE, 2020
IntroductionRavulizumab (ALXN1210) is a long-lasting recycling IgG monoclonal antibody with an increased affinity for the neonatal Fc receptor (FcRn). The FcRn is essential for regulating IgG homeostasis. Saturation of the FcRn pathway is seen under high
Ferras Alashkar, Scott Rottinghaus, Colin Vance, Dörte Herich-Terhürne, Ulrich Dührsen, Roland Assert, Alexander Röth   +6 more
doaj   +1 more source

Inborn errors of immunity in children with neuroinflammation

Developmental Medicine &Child Neurology, EarlyView.
Abstract Inborn errors of immunity (IEIs), an expanding group of monogenic disorders with diverse clinical manifestations, are increasingly recognized to include neuroinflammatory disease. Examples of diseases included under this umbrella are Aicardi–Goutières syndrome, deficiency of adenosine deaminase 2, familial haemophagocytic lymphohistiocytosis ...
Eppie M Yiu, Dilan Dissanayake, Jessie Cunningham, Helen Branson, Ori Scott, E Ann Yeh   +5 more
wiley   +1 more source

Characterizing clinically significant extravascular hemolysis in adults with PNH on ravulizumab or eculizumab treatment

Blood Advances
: In patients with paroxysmal nocturnal hemoglobinuria (PNH), the complement component 5 (C5) inhibitors ravulizumab and eculizumab control terminal complement activity and intravascular hemolysis, which are drivers of morbidity and mortality.
Austin G. Kulasekararaj, Jong Wook Lee, Christopher J. Patriquin, Caroline I. Piatek, Alexander Röth, Wilma Barcellini, Robert A. Brodsky, Jun-ichi Nishimura, Ji Yu, Mohammed Mahdi, Régis Peffault de Latour   +10 more
doaj   +1 more source

Australian consensus recommendations for the management of increased meningococcal infection risk in adults with neurological diseases treated with complement inhibitors

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, Michael Barnett, Heidi Beadnall, Mirasol Forcadela, Lesley‐Ann Hall, Victoria G. Hall, Todd Hardy, Danny Hsu, Paul Kinsella, Mastura Monif, Sudarshini Ramanathan, Stephen W. Reddel, Sean Riminton, Douglas F. Johnson   +13 more
wiley   +1 more source

COVID19 infection in a patient undergoing treatment for Paroxysmal Nocturnal Hemoglobinuria (PNH) with Ravulizumab

Thrombosis Journal, 2021
Background In the recent COVID19 pandemic, patients with hematological disorders were considered at high risk for severe disease. Limited data is available regarding the course of COVID19 infection in this subgroup.
Sufana Shikdar, Azra Borogovac, Elabdallah Mohamad, Mohamad Khawandanah   +3 more
doaj   +1 more source