Results 71 to 80 of about 20,100 (304)

Eculizumab precision-dosing algorithm for thrombotic microangiopathy in children and young adults undergoing HSCT

Blood Advances, 2022
Key Points This large study examined eculizumab PK/PD in bleeding and nonbleeding patients with TA-TMA. PK/PD model-based eculizumab dosing is needed for bleeding in TA-TMA, whereas fixed-dose regimens are effective in nonbleeding patients.
Kana Mizuno, C. Dandoy, Ashley Teusink‐Cross, S. Davies, A. Vinks, S. Jodele   +5 more
semanticscholar   +1 more source

Eculizumab and recurrent C3 glomerulonephritis [PDF]

Pediatric Nephrology, 2013
Hyperactivity of the alternative complement pathway is the principle defect in C3 glomerulopathies (C3G). Eculizumab, a monoclonal antibody that binds C5 to prevent formation of the membrane attack complex, has been shown to be beneficial in some patients with this disease.In this open-label, proof-of-concept efficacy-and-safety study, a patient with ...
Richard J.H. Smith, Xue Xiao, Billie Fyfe, Yuzhou Zhang, Lynne S. Weiss, Sevgi Gurkan, Sevgi Gurkan   +6 more
openaire   +3 more sources

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

HUS and atypical HUS [PDF]

, 2017
Hemolytic uremic syndrome (HUS) is a thrombotic microangiopathy characterized by intravascular hemolysis, thrombocytopenia, and acute kidney failure. HUS is usually categorized as typical, caused by Shiga toxin-producing Escherichia coli (STEC) infection,
Al-Nouri, Allen, Amaral, Ardissino, Arvidsson, Atefi, Atkinson, Berry, Bhakdi, Bhattacharjee, Boltax, Brady, Bresin, Bruneau, Bu, Caires, Campistol, Canalejo, Carson, Chapin, Chien, Cofiell, Cole, Conway, Conway, Coppo, Cornec-Le Gall, de Córdoba, Del Conde, Delvaeye, Dempsey, Devine, Dhakal, Dragon-Durey, Egbor, El-Husseini, Faguer, Fakhouri, Ferreira, Ferreira, Fremeaux-Bacchi, Frimat, George, Gilbert, Gorsuch, Gottschalk, Grabowski, Greenbaum, Hill, Hornung, Hosler, Huber-Lang, Hughson, Hyvärinen, Iida, Ikeda, Jin, Jokiranta, Kang, Karnchanaphanurach, Karpman, Karpman, Karpman, Kavanagh, Keir, Kielstein, Kim, Kourouklaris, Krisinger, Kronbichler, Laurence, Law, Lechner, Lehtinen, Lemaire, Licht, Liszewski, Loirat, Lupu, Manuelian, Martel, Mathew, Matsukuma, Mele, Meri, Morton, Moskovich, Mukhopadhyay, Nakakuma, Nester, Noris, Noris, Noris, Oikonomopoulou, Orth-Höller, Ozaki, Pangburn, Pangburn, Pape, Pecoraro, Pilzer, Podack, Ricklin, Rinder, Rodríguez-Pintó, Roumenina, Ruggenenti, Schutte, Scully, Sethi, Sims, Sinha, Song, Ståhl, Ståhl, Sung, Szilágyi, T. Sakari Jokiranta, Tawadrous, Tedesco, Thomas, Thurman, Toh, Tsai, Tufano, Vasu, Ward, Warwicker, Wehling, Wetsel, Whaley, Wiedmer, Wig, Woodworth, Zipfel, Zipfel   +135 more
core   +1 more source

Phase 3 Study of Subcutaneous Versus Intravenous Ravulizumab in Eculizumab-Experienced Adult Patients with PNH: Primary Analysis and 1-Year Follow-Up

Advances in Therapy, 2022
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare blood disorder characterized by the destruction of red blood cells (hemolysis) within blood vessels.
M. Yenerel, F. Sicre de Fontbrune, C. Piatek, F. Şahin, W. Füreder, S. Ortiz, M. Ogawa, A. Ozol-Godfrey, J. R. Sierra, J. Szer   +9 more
semanticscholar   +1 more source

Pharmacokinetics and Pharmacodynamics Estimation of Eculizumab in a 2-Year-Old Girl With Atypical Hemolytic Uremic Syndrome: A Case Report With 4-Year Follow-Up

Frontiers in Pediatrics, 2019
Background: Eculizumab has dramatically changed poor outcomes of complement-mediated atypical hemolytic uremic syndrome (aHUS) as first-line treatment. Discontinuation of eculizumab remains challenging, and doctor's visits every 2 weeks for intravenous ...
Ken Saida, Tsuyoshi Fukuda, Kana Mizuno, Masao Ogura, Koichi Kamei, Shuichi Ito   +5 more
doaj   +1 more source

When the environment and mutations affect organ systems [PDF]

, 2017
Atypical hemolytic uremic syndrome (aHUS) is a rare thrombotic microangiopathy (TMA) with a genetic predisposition. Like other TMAs, it presents clinically with thrombocytopenia and microangiopathic hemolytic anemia, which is accompanied by disruption of
Abu Ghanimeh, Mouhanna, Abughanimeh, Omar, Qasem, Abdulraheem, Qasrawi, Ayman   +3 more
core   +1 more source

High‐Efficacy Treatment in Neuromyelitis Optica Specturm Disorder Patients With Seropositive AQP4 Antibodies—A Real‐World Study

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective To compare the effectiveness of high‐efficacy treatments (HET) and low‐efficacy treatments (LET) in NMOSD patients with anti‐aquaporin‐4 antibodies (AQP4‐ab). Methods In this multi‐center study, we analyzed 183 AQP4‐ab seropositive NMOSD patients who received immunosuppressive treatments (IST).
Xiang Li, Binbin Xue, Jia Li, Dewei Xie, Juyuan Pan, Lanbing Zhu, Qiaowen Tong, Jing Lin, Xu Zhang, Junhui Xia, Jie Lin   +10 more
wiley   +1 more source

United States clinical practice experience with eculizumab in myasthenia gravis: symptoms, function, and immunosuppressant therapy use

Journal of Neurology
Background/objectives The phase 3 REGAIN study and its open-label extension demonstrated the efficacy of the complement C5 inhibitor eculizumab in patients with treatment-refractory, acetylcholine receptor antibody–positive generalized myasthenia gravis (
Ali A. Habib, Andrew Klink, Srikanth Muppidi, A. Parthan, S. C. Sader, A. Balanean, A. Gajra, Richard J. Nowak, James F. Howard   +8 more
semanticscholar   +1 more source

Eculizumab for acute relapse of neuromyelitis optica spectrum disorder: Case report

Frontiers in Neurology, 2022
Introduction Eculizumab has been shown to be an effective and typically well-tolerated medication in the treatment of neuromyelitis optica spectrum disorder (NMOSD) in maintaining disease remission in patients who are aquaporin-4 water channel ...
S. Chatterton, J. Parratt, K. Ng
semanticscholar   +1 more source