Results 21 to 30 of about 1,547 (116)

Population Pharmacokinetic and Pharmacokinetic/Pharmacodynamic Analyses of Pegcetacoplan in Patients with Paroxysmal Nocturnal Hemoglobinuria

Drugs in R&D
Background and Objective Paroxysmal nocturnal hemoglobinuria is a rare blood disorder characterized by life-threatening hemolysis and thrombosis. Complement C5 inhibitor therapy improves symptoms and life prognosis; however, it can result in insufficient
Ryan L. Crass, Brandon Smith, Sven Adriaens, Sunny Chapel, Grant Langdon   +4 more
doaj   +2 more sources

Cost-Utility Analysis Comparing Pegcetacoplan to Anti-C5 Monoclonal Antibodies in the Treatment of Paroxysmal Nocturnal Hemoglobinuria [PDF]

ClinicoEconomics and Outcomes Research
Sergio Di Matteo,1 Roberto Freilone,2 Giacomo Matteo Bruno,1,3 Rosario Notaro,4 Sabrin Moumene,1 Nicoletta Martone,5 Cristina Teruzzi,5 Antonio Ciccarone,1 Giorgio Lorenzo Colombo1,3 1Center of Research, SAVE Studi - Health Economics and Outcomes ...
Di Matteo S, Freilone R, Bruno GM, Notaro R, Moumene S, Martone N, Teruzzi C, Ciccarone A, Colombo GL   +8 more
doaj   +1 more source

Real-World Study of US Adults with Paroxysmal Nocturnal Hemoglobinuria Treated with Pegcetacoplan

Hematology Reports
Background: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired, life-threatening disease characterized by complement-mediated hemolysis.
Brian Mulherin, Apeksha Shenoy, Lily Arnett, Weiqi Jiao, Joseph Guarinoni, Sujata Sarda, Jinny Min, David Dingli   +7 more
doaj   +2 more sources

Disease-modifying treatments in paroxysmal nocturnal hemoglobinuria: a systematic review of economic evaluations [PDF]

Cost Effectiveness and Resource Allocation
Background Paroxysmal Nocturnal Hemoglobinuria (PNH) is a rare, life-threatening hematologic disorder characterized by chronic hemolysis, bone marrow and organ failure, and thrombotic events.
Shaghayegh Moradi, Yasaman Khajeamiri, Sara Zandi, Mohammadreza Keramati, Ghader Mohammadnezhad   +4 more
doaj   +2 more sources

Pegcetacoplan versus Eculizumab in Paroxysmal Nocturnal Hemoglobinuria [PDF]

, 2021
Background Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired disease characterized by chronic complement-mediated hemolysis. C5 inhibition controls intravascular hemolysis in untreated PNH but cannot address extravascular hemolysis ...
Panse, Jens Peter, Peffault de la Tour, Régis, Höchsmann, Britta, Szer, Jeff, Ilene Weitz, Panse, Jens, Röth, Alexander, Jeff Szer, Weitz, Ilene, Régis Peffault de la Tour, de la Tour, Régis Peffault, Tan, Lisa, Carlos de Castro, Deschatelets, Pascal, Kensuke Usuki, Griffin, Morag, Hillmen, Peter, Hisakazu Nishimori, Jens Panse, Britta Höchsmann, Temitayo Ajayi, Mohamed Hamdani, de Castro, Carlos, Antonio Risitano, Kiladjian, Jean-Jacques, Hamdani, Mohamed, Usuki, Kensuke, Risitano, Antonio, Jean-Jacques Kiladjian, Lisa Tan, Alexander Röth, Nishimori, Hisakazu, Cedric Francois, Ajayi, Temitayo, Grossi, Federico, Peter Hillmen, Morag Griffin, Francois, Cedric, Pascal Deschatelets, Federico Grossi   +39 more
core   +2 more sources

Central retinal vein occlusion following intravitreal injections: a case series highlighting multifactorial risk [PDF]

International Journal of Retina and Vitreous
Introduction Intravitreal injections are standard treatment for various manifestations of age-related macular degeneration (AMD) but are associated with rare complications, including central retinal vein occlusion (CRVO).
Michael Antonietti, Carolina Mercado, William E. Smiddy, Stephen G. Schwartz   +3 more
doaj   +2 more sources

Analysis of Costs per Responder in US Adults with Paroxysmal Nocturnal Hemoglobinuria with a Suboptimal Response to Prior Eculizumab Treatment

Hematology Reports, 2023
European Society for Blood and Marrow Transplantation (EBMT) hematologic response categories comprehensively assess complement inhibitor responses in patients with paroxysmal nocturnal hemoglobinuria (PNH).
Jesse Fishman, Seri Anderson, Sandra E. Talbird, David Dingli   +3 more
doaj   +1 more source

Association of complement C3 inhibitor pegcetacoplan with reduced photoreceptor degeneration beyond areas of geographic atrophy

Scientific Reports, 2022
Preservation of photoreceptors beyond areas of retinal pigment epithelium atrophy is a critical treatment goal in eyes with geographic atrophy (GA) to prevent vision loss.
Maximilian Pfau, Steffen Schmitz-Valckenberg, Ramiro Ribeiro, Reza Safaei, Alex McKeown, Monika Fleckenstein, Frank G. Holz   +6 more
doaj   +1 more source

Trial of Pegcetacoplan in C3 Glomerulopathy and Immune-Complex MPGN.

VALIANT Trial Investigators Group: Huynh-Do, Clinic of Nephrology and HypertensionBackground C3 glomerulopathy and primary immune-complex membranoproliferative glomerulonephritis (MPGN) generally result in glomerular C3 deposition and irreversible kidney
Delmas, Yahsou, Han, Seung Hyeok, Mastrangelo, Antonio, Kinnman, Nils, Szamosi, Johan, Wallace, Dean, Van De Kar, Nicole, Pickering, Matthew C, Nester, Carla M, Deschatelets, Pascal, Dixon, Bradley P, Bomback, Andrew S, Fakhouri, Fadi, Zecher, Daniel, Ariceta, Gema, Vivarelli, Marina, Mizuno, Masashi, Abad-Franch, Lydia, Remuzzi, Giuseppe, Li, Li, López-Lázaro, Luis, Le Quintrec, Moglie, Isbel, Nicole, Gale, Daniel P, Licht, Christoph, Walker, Patrick D, Wang, Zhongshen, Greenbaum, Larry A, Francois, Cedric, Neves de Holanda, Maria Izabel   +29 more
core   +6 more sources

Pegcetacoplan in paroxysmal nocturnal hemoglobinuria: A systematic review on efficacy and safety

Research and Practice in Thrombosis and Haemostasis, 2022
Introduction Pegcetacoplan, a pegylated penta‐decapeptide, targets complement C3 to control both intravascular and extravascular hemolysis. This systematic review aims to study the efficacy and safety of pegcetacoplan in paroxysmal nocturnal ...
Sangam Shah, Rajan Chamlagain, Ziyaul Haq Musalman, Yagya Raj Adhikari, Santosh Chhetri, Sujan Paudel, Krishna Gundabolu, Prajwal Dhakal   +7 more
doaj   +1 more source