Results 1 to 10 of about 898 (115)

Efficacy and Safety of Iptacopan in Patients With C3 Glomerulopathy [PDF]

Kidney International Reports, 2023
Introduction: Complement 3 glomerulopathy (C3G) is a rare inflammatory kidney disease mediated by dysregulation of the alternative complement pathway. No targeted therapy exists for this aggressive glomerulonephritis.
Guido Junge, Teresa Cavero
exaly   +11 more sources

Iptacopan monotherapy resulted in increased hemoglobin level in patients with PNH and hemoglobin ≥10 g/dL after anti‐C5 therapy [PDF]

HemaSphere
Patients with paroxysmal nocturnal hemoglobinuria (PNH) on anti‐C5 often experience extravascular hemolysis with anemia. Iptacopan, the first oral proximal complement inhibitor targeting factor B, has shown efficacy and safety in PNH patients.
Austin Kulasekararaj, Flore S. deFontbrune, Anna Gaya, Ilene Weitz, David J. Kuter, Bhumika J. Patel, David J. Araten, Abhay Singh, Jun H. Jang, Richard J. Kelly, Eros Di Bona, Michael Loschi, Vinod Pullarkat, Jörg Schubert, Rosario Notaro, Mustafa N. Yenerel, Joan Beckman, Nicolas Blin, Irina Murakhovskaya, Jens Panse, Eloy Roman, Alexander Röth, Hubert Schrezenmeier, Srinivas Tantravahi, Régis P. deLatour, Navin Mahajan, Luca Monaco, Tao Ding, Tomasz Lawniczek, Philippe Ferber, Marion Dahlke, Antonio M. Risitano   +31 more
doaj   +3 more sources

Targeting the Alternative Complement Pathway With Iptacopan to Treat IgA Nephropathy: Design and Rationale of the APPLAUSE-IgAN Study

Kidney International Reports, 2023
Introduction: Targeting the alternative complement pathway (AP) is an attractive therapeutic strategy because of its role in immunoglobulin A nephropathy (IgAN) pathophysiology.
Hernán Trimarchi, Dmitrij Kollins
exaly   +5 more sources

Direct Switch From Iptacopan to Pegcetacoplan in a Patient With Paroxysmal Nocturnal Hemoglobinuria [PDF]

eJHaem
Introduction Paroxysmal nocturnal hemoglobinuria (PNH) is a rare clonal hematologic disorder treated with complement inhibitors. Currently, patients with suboptimal response to C5 inhibitors may be switched to proximal complement inhibitors, such as ...
Vitalii Latyshev, Zalina Fidarova, Elena Lukina, Elena Parovichnikova   +3 more
doaj   +3 more sources

Case Report: Iptacopan in a paroxysmal nocturnal hemoglobinuria patient with severe renal insufficiency [PDF]

Frontiers in Medicine
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare disorder characterized by complement-mediated hemolysis, thrombosis, and bone marrow failure. Iptacopan, an oral factor B inhibitor, has demonstrated efficacy in managing PNH but has not been studied in
Xiaoqin Wang
exaly   +4 more sources

Recurrence of IgA nephropathy in a kidney transplant patient successfully treated with iptacopan—a case report [PDF]

Frontiers in Immunology
IgA nephropathy recurrence after kidney transplantation is a common complication, which is most often detected histologically. Recurrence after transplant affects long-term graft survival, with prognosis being worse with increasing levels of proteinuria,
Jay Pandav, Basheer Kummangal, Justin Smith, Angelo De Mattos, Megan Stack, Zoha Ahmad, Nicole Andeen, Vanderlene Kung, Ali Olyaei, Shehzad Rehman   +9 more
doaj   +2 more sources

Efficacy analysis of iptacopan in a patient with thrombotic microangiopathy after allogeneic hematopoietic stem cell transplantation: a case report [PDF]

Frontiers in Immunology
To investigate the efficacy of Iptacopan in transplantation-associated thrombotic microangiopathy (TA-TMA) after allogeneic hematopoietic stem cell transplantation (allo-HSCT), we report the case of a 43-year-old male with Acute Myeloid Leukemia ...
Zixuan Zhao, Lin Guo, Jiao Ge, Wei Wu, Yanhua Yue, Yan Qiu, Feng Li, Wenxi Hua, Weiying Gu, Yan Lin   +9 more
doaj   +2 more sources

Switching between complement inhibitors in paroxysmal nocturnal hemoglobinuria: Analysis of strategy, efficacy, and safety. [PDF]

Hemasphere
Abstract Paroxysmal nocturnal hemoglobinuria (PNH) is an ultra‐orphan disease. In 2026, approved complement inhibitors (CIs) for PNH include three C5 inhibitors (C5i) and three proximal inhibitors (PIs). Clinical trials for the approved PI pegcetacoplan, iptacopan, and C5i + danicopan had clear protocols for changing from terminal to PI, extrapolated ...
Griffin M, Fattizzo B, Lee JW, Kelly RJ, Trikha R, Ueda Y, Nishimura JI, Patriquin CJ, Röth A, Muus P, Panse J, Álvarez MG, Pike A, Munir T, Gandhi S, Kulasekararaj A.   +15 more
europepmc   +2 more sources

Complement-targeted therapies for C3 glomerulopathy and atypical hemolytic uremic syndrome: a time-limited rapid systematic review with narrative synthesis [PDF]

Frontiers in Medicine
C3 glomerulopathy (C3G) and atypical hemolytic uremic syndrome (aHUS) are rare complement-mediated kidney diseases with differing injury sites and therapeutic targets. We conducted a time-limited rapid systematic review to synthesize clinical evidence on
Qishun Wu, Zhiliang Yu, Zhangli Wu, Ling Yang, Xin Lin   +4 more
doaj   +2 more sources

Methodological pitfalls in indirect treatment comparisons: insights from a recent systematic review and analysis for C3 glomerulopathy [PDF]

Journal of Comparative Effectiveness Research
Aim: Indirect treatment comparisons (ITCs), as outlined in NICE and ISPOR guidance, require careful evaluation of cross-trial heterogeneity to ensure valid comparisons, particularly in rare diseases with limited evidence.
Brian Hutton, James Fotheringham, Andrew Ervin, Raisa Sidhu, Corey Lourenco, Imtiaz A Samjoo, Chris Cameron, Bridget Dunne   +7 more
doaj   +2 more sources