Results 61 to 70 of about 24,516 (282)

The burden of illness in patients with paroxysmal nocturnal hemoglobinuria receiving treatment with the C5-inhibitors eculizumab or ravulizumab: results from a US patient survey

Annals of Hematology, 2022
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare and life-threatening disease with symptoms of hemolysis and thrombosis. Current therapies for this complement-mediated disease rely predominantly on inhibition of the C5 complement protein.
D. Dingli, J. E. Matos, K. Lehrhaupt, S. Krishnan, M. Yeh, J. Fishman, S. Sarda, S. Baver   +7 more
semanticscholar   +1 more source

Baseline characteristics and disease burden in patients in the International Paroxysmal Nocturnal Hemoglobinuria Registry [PDF]

, 2018
Paroxysmal nocturnal hemoglobinuria is a rare, acquired disease associated with hemolytic anemia, bone marrow failure, thrombosis, and, frequently, poor quality of life.
Bedrosian, Camille L., Bessler, Monica, Brodsky, Robert A., Hillmen, Peter, Kanakura, Yuzuru, Khursigara, Gus, Maciejewski, Jaroslaw P., Muus, Petra, Rosse, Wendell, Schrezenmeier, Hubert, Socié, Gérard, Szer, Jeffrey, Urbano Ispizua, Álvaro   +12 more
core   +1 more source

The safety and efficacy profile of eculizumab in myasthenic crisis: a prospective small case series

Therapeutic Advances in Neurological Disorders
Eculizumab has improved recovery from ventilatory support in myasthenic crisis (MC) cases. However, the safety and efficacy profiles from prospective studies are still lacking. This study aimed to explore eculizumab’s safety and efficacy in a prospective
Jie Song, Xiao Huan, Yuanyi Chen, Yeting Luo, Huahua Zhong, Yuan Wang, Lei Yang, Caihua Xi, Yu Yang, Jianying Xi, Jianming Zheng, Zongtai Wu, Chongbo Zhao, S. Luo   +13 more
semanticscholar   +1 more source

Ravulizumab (ALXN1210) vs eculizumab in adult patients with PNH naive to complement inhibitors: the 301 study.

Blood, 2019
Ravulizumab (ALXN1210), a new complement C5 inhibitor, provides immediate, complete, and sustained C5 inhibition. This phase 3, open-label study assessed the noninferiority of ravulizumab to eculizumab in complement inhibitor-naive adults with paroxysmal
J. W. Lee, F. Sicre de Fontbrune, Lily Wong Lee Lee, V. Pessoa, S. Gualandro, W. Füreder, Vadim Ptushkin, S. Rottinghaus, L. Volles, L. Shafner, R. Aguzzi, R. Pradhan, H. Schrezenmeier, A. Hill   +13 more
semanticscholar   +1 more source

Real‐World Investigation of Satralizumab in Patients With Neuromyelitis Optica Spectrum Disease

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective Satralizumab, a monoclonal antibody targeting the interleukin‐6 receptor, has demonstrated efficacy in clinical trials for neuromyelitis optica spectrum disorder (NMOSD). However, its real‐world effectiveness and safety compared to conventional immunosuppressive therapies remain uncertain.
Li‐Tsung Lin, Hui‐An Lin, Sheng‐Feng Lin   +2 more
wiley   +1 more source

An Anti-C1s Monoclonal, TNT003, Inhibits Complement Activation Induced by Antibodies Against HLA. [PDF]

, 2015
Antibody-mediated rejection (AMR) of solid organ transplants (SOT) is characterized by damage triggered by donor-specific antibodies (DSA) binding donor Class I and II HLA (HLA-I and HLA-II) expressed on endothelial cells.
Fishbein, MC, Gjertson, D, Mulder, A, Panicker, S, Parry, GC, Reed, EF, Thomas, KA, Valenzuela, NM   +7 more
core   +1 more source

Eculizumab discontinuation in children and adults with atypical haemolytic uremic syndrome: a prospective multicentric study.

Blood, 2020
The optimal duration of eculizumab treatment in patients with atypical haemolytic uremic syndrome (aHUS) remains poorly defined. We conducted a prospective national multicentric open-label study in order to assess eculizumab discontinuation in children ...
F. Fakhouri, M. Fila, A. Hummel, D. Ribes, A. Sellier-Leclerc, Simon Ville, C. Pouteil‐Noble, J. Coindre, M. Le Quintrec, E. Rondeau, O. Boyer, F. Provôt, D. Djeddi, W. Hanf, Yahsou Delmas, F. Louillet, A. Lahoche, G. Favre, V. Châtelet, Emma Allain Launay, C. Presne, A. Zaloszyc, S. Caillard, S. Bally, Q. Raimbourg, L. Tricot, C. Mousson, A. Le Thuaut, C. Loirat, V. Frémeaux-Bacchi   +29 more
semanticscholar   +1 more source

Cost-effectiveness of ravulizumab compared with eculizumab for the treatment of paroxysmal nocturnal hemoglobinuria in the Netherlands

European Journal of Health Economics, 2023
Objectives The aim of this study was to evaluate the cost-effectiveness of ravulizumab compared with eculizumab for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH) in the Netherlands.
S. Quist, A. Postma, K. Myrén, L. D. de Jong, M. Postma   +4 more
semanticscholar   +1 more source

Inhibition of Classical and Alternative Complement Pathway by Ravulizumab and Eculizumab

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective To explore the feasibility of classical (CH50) and alternative (AH50) complement pathway activity as potential biomarkers for treatment guidance and monitoring during therapy with ravulizumab in patients with generalized myasthenia gravis (gMG) and compare these to therapeutic drug monitoring under eculizumab.
Lea Gerischer, Frauke Stascheit, Maximilian Mönch, Paolo Doksani, Carla Dusemund, Meret Herdick, Philipp Mergenthaler, Maike Stein, Amani Suboh, Jutta Schröder‐Braunstein, Guido Wabnitz, Jan D. Lünemann, Sophie Lehnerer, Sarah Hoffmann, Andreas Meisel   +14 more
wiley   +1 more source

Eculizumab as Additional Rescue Therapy in Myasthenic Crisis

Muscles
Eculizumab is a monoclonal antibody blocking the terminal complement protein C5. As demonstrated in the phase III randomized, placebo-controlled, REGAIN clinical trial, eculizumab is efficacious in acetylcholine receptor antibody (AChR-Ab)-positive ...
Francesco Crescenzo, Mattia Zanoni, Laura Ferigo, Francesca Rossi, Matteo Grecò, Angelica Lupato, Alessandra Danese, Domenico Ajena, Michelangelo Turazzini   +8 more
doaj   +1 more source