Article
Medicine, General & Internal
Sarah Dunn, Victoria Brocklebank, Andrew Bryant, Sonya Carnell, Thomas J. Chadwick, Sally Johnson, David Kavanagh, Jan Lecouturier, Michal Malina, Eoin Moloney, Yemi Oluboyede, Christopher Weetman, Edwin Kwan Soon Wong, Len Woodward, Neil Sheerin
Summary: This study aims to investigate the safety of stopping eculizumab treatment in patients with atypical haemolytic uraemic syndrome (aHUS) by monitoring disease activity and tracking thrombotic microangiopathy-related serious adverse events. It evaluates the effectiveness and feasibility of this treatment strategy.
Editorial Material
Hematology
Robert A. Brodsky
Summary: Evidence from a prospective study shows that discontinuing eculizumab is safe in most aHUS patients after achieving complete remission, with a relapse risk of less than 25% overall, but up to 50% in patients with rare variants in at least one complement gene.
Article
Allergy
Inge Van de Walle, Karen Silence, Kevin Budding, Liesbeth Van de Ven, Kim Dijkxhoorn, Elisabeth de Zeeuw, Cafer Yildiz, Sofie Gabriels, Jean-Michel Percier, Johanna Wildemann, Jan Meeldijk, Peter J. Simons, Louis Boon, Linda Cox, Rob Holgate, Rolf Urbanus, Henny G. Otten, Jeanette H. W. Leusen, Christophe Blanchetot, Hans de Haard, C. Erik Hack, Peter Boross
Summary: ARGX-117 is a promising new complement inhibitor that uniquely targets both the classical and lectin pathways by binding to the Sushi-2 domain of C2. It prevents complement-mediated cytotoxicity in in vitro models for autoimmune hemolytic anemia and antibody-mediated rejection of organ transplants.
JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY
(2021)
Article
Multidisciplinary Sciences
Hong Si Nga, Lilian Monteiro Pereira Palma, Miguel Ernandes Neto, Ida Maria Maximina Fernandes-Charpiot, Valter Duro Garcia, Roger Kist, Silvana Maria Carvalho Miranda, Pedro Augusto Macedo de Souza, Gerson Marques Pereira, Luis Gustavo Modelli de Andrade
Summary: The study evaluated the incidence of TMA after kidney transplantation in a Brazilian cohort of aHUS patients, stratified by the use of eculizumab. Results showed a higher risk of graft loss due to TMA in patients not treated with eculizumab, highlighting the importance of eculizumab in kidney transplant patients with aHUS.
Review
Immunology
Huiru Chen, Youjia Qiu, Ziqian Yin, Zilan Wang, Yanbing Tang, Hanyu Ni, Jiaye Lu, Zhouqing Chen, Yan Kong, Zhong Wang
Summary: This study conducted a network meta-analysis to compare the relative effects of different drugs for generalized myasthenia gravis (MG). The results showed that rozanolixzumab and batoclimab may be the most effective drugs, but rozanolixzumab was associated with a higher incidence of adverse events. Further head-to-head and extensive observational studies are needed to confirm these findings.
FRONTIERS IN IMMUNOLOGY
(2023)
Article
Biochemistry & Molecular Biology
Nicola Iacomino, Fiammetta Vanoli, Rita Frangiamore, Marta Ballardini, Letizia Scandiffio, Federica Bortone, Francesca Andreetta, Fulvio Baggi, Pia Bernasconi, Carlo Antozzi, Paola Cavalcante, Renato Mantegazza
Summary: The study identifies plasma complement proteins as potential biomarkers for tailoring anti-complement therapy in myasthenia gravis patients, specifically in those with anti-acetylcholine receptor antibody-positive MG. The alterations in complement C2, C3, C5, C3b, and C5a levels may serve as indicators of complement activation status and aid in improving disease treatment through personalized anti-complement therapies.
Article
Medicine, Research & Experimental
Tamas Bakos, Gergely T. Kozma, Janos Szebeni, Gabor Szenasi
Summary: This study investigated the relationship between complement activation and cytokine production using a PBMC model, and found that the use of the C5 inhibitor eculizumab significantly suppressed the production of multiple inflammatory cytokines. These results highlight the abundant formation of the chemokine IL-8 stimulated by C5a, and the inhibitory effect of eculizumab on various inflammatory cytokines, explaining its therapeutic efficacy in severe Covid-19 patients.
BIOMEDICINE & PHARMACOTHERAPY
(2023)
Article
Clinical Neurology
Renato Mantegazza, Gil Wolfe, Srikanth Muppidi, Heinz Wiendl, Kenji P. Fujita, Fanny L. O'Brien, Heather D. E. Booth, James F. Howard
Summary: The study showed that eculizumab helped patients with anti-acetylcholine receptor-positive refractory generalized myasthenia gravis achieve rapid and sustained minimal manifestations (MM) status, supporting its use in this previously difficult-to-treat patient population.
Review
Clinical Neurology
Francesco Sacca, Chiara Pane, Pablo Ezequiel Espinosa, Maria Pia Sormani, Alessio Signori
Summary: This study conducted a meta-analysis and network meta-analysis of randomized and placebo-controlled trials to evaluate the efficacy of innovative therapies in myasthenia gravis (MG). The results showed that anti-complement and FcRn treatments were effective in MG patients, while rituximab did not provide significant benefit.
EUROPEAN JOURNAL OF NEUROLOGY
(2023)
Article
Biochemistry & Molecular Biology
Weihe Zhang, Minwan Wu, Satish Vadlakonda, Luis Juarez, Xiaogang Cheng, Saritha Muppa, Venkat Chintareddy, Lakshminarayana Vogeti, Debra Kellogg-Yelder, Jason Williams, Kevin Polach, Xilin Chen, Krishnan Raman, Y. S. Babu, Pravin Kotian
Summary: The complement pathways play a crucial role in the immune system, and dysregulation of the alternative pathway has been associated with various diseases. This study identified and developed novel CFD inhibitors that showed potent activity and promising pharmacokinetic properties.
BIOORGANIC & MEDICINAL CHEMISTRY
(2022)
Review
Immunology
Qin Li, Zi Chen
Summary: The current outbreak of COVID-19 has global impacts, affecting various organs and leading to tissue injury and multiorgan failure due to abnormal activation of host immune response. Targeting the complement system could be a potential treatment option for organ damage in COVID-19 patients.
MEDICAL MICROBIOLOGY AND IMMUNOLOGY
(2021)
Review
Immunology
Martin Kolev, Madhumita Das, Monica Gerber, Scott Baver, Pascal Deschatelets, Maciej M. Markiewski
Summary: Recent studies have shown that complement plays a role in accelerating cancer progression by suppressing antitumor immunity in the tumor microenvironment. Activation of intracellular complement is crucial for the survival of immune cells, making it important for tumor cell survival and growth. Additionally, intracellular complement activation is essential for priming antitumor T cell responses.
FRONTIERS IN IMMUNOLOGY
(2022)
Article
Biophysics
Peter Svec, Reem Elfeky, Jacques-Emmanuel Galimard, Christine S. Higham, Arnaud Dalissier, Troy C. Quigg, David Bueno Sanchez, Su Han Lum, Maura Faraci, Theresa Cole, Herbert Pichler, Maria Isabel Benitez-Carabante, Julia Horakova, Marta Gonzalez Vicent, Asaf Yanir, Franca Fagioli, Matthias Wolfl, Nicolas von der Weid, Rachel Protheroe, Gergely Krivan, Carsten Speckmann, Beki James, Simona Lucija Avcin, Yves Bertrand, Marta Verna, Petr Riha, Katharine Patrick, Simone Cesaro, Krzysztof Kalwak, Marc Bierings, Jochen Buchner, Karin Mellgren, Zoltan Prohaszka, Benedicte Neven, Arjan Lankester, Selim Corbacioglu
Summary: A retrospective international study on the treatment of transplantation-associated thrombotic microangiopathy (TA-TMA) with eculizumab in 82 pediatric patients showed a TA-TMA resolution rate of 36.6% and an overall survival rate of 47.1% after 6 months of eculizumab therapy. All patients who did not have resolution of TA-TMA ultimately died, primarily from HSCT-related complications.
BONE MARROW TRANSPLANTATION
(2022)
Review
Medicine, General & Internal
Nicole Ng, Charles A. Powell
Summary: Severe coronavirus disease 2019 leads to multi-organ dysfunction mainly due to maladaptive activation of innate immune pathways and endothelial dysfunction. Clinical trials show that targeting the complement pathways is a promising therapeutic approach.
JOURNAL OF CLINICAL MEDICINE
(2021)
Article
Hematology
Jun Ho Jang, Lily Wong, Bor-Sheng Ko, Sung-Soo Yoon, Katie Li, Irina Baltcheva, Prasanna Kumar Nidamarthy, Raghav Chawla, Guido Junge, Eng Soo Yap
Summary: Iptacopan is a novel oral inhibitor of complement factor B under development for the treatment of PNH. This phase 2 study evaluated the efficacy and safety of iptacopan in PNH patients with active hemolysis. The results showed that iptacopan effectively reduced LDH levels, improved hemolytic markers, and rapidly increased Hb levels in most patients.
Article
Rheumatology
Katherine E. Lintner, Anjali Patwardhan, Lisa G. Rider, Rabheh Abdul-Aziz, Yee Ling Wu, Emeli Lundstrom, Leonid Padyukov, Bi Zhou, Alaaedin Alhomosh, David Newsom, Peter White, Karla B. Jones, Terrance P. O'Hanlon, Frederick W. Miller, Charles H. Spencer, Chack Yung Yu
ANNALS OF THE RHEUMATIC DISEASES
(2016)
Article
Urology & Nephrology
Andrew L. Schwaderer, Huanyu Wang, SungHwan Kim, Jennifer M. Kline, Dong Liang, Pat D. Brophy, Kirk M. McHugh, George C. Tseng, Vijay Saxena, Evan Barr-Beare, Keith R. Pierce, Nader Shaikh, J. Robert Manak, Daniel M. Cohen, Brian Becknell, John D. Spencer, Peter B. Baker, Chack-Yung Yu, David S. Hains
JOURNAL OF THE AMERICAN SOCIETY OF NEPHROLOGY
(2016)
Article
Multidisciplinary Sciences
Lingling Shi, Yunfei Guo, Chengliang Dong, John Huddleston, Hui Yang, Xiaolu Han, Aisi Fu, Quan Li, Na Li, Siyi Gong, Katherine E. Lintner, Qiong Ding, Zou Wang, Jiang Hu, Depeng Wang, Feng Wang, Lin Wang, Gholson J. Lyon, Yongtao Guan, Yufeng Shen, Oleg V. Evgrafov, James A. Knowles, Francoise Thibaud-Nissen, Valerie Schneider, Chack-Yung Yu, Libing Zhou, Evan E. Eichler, Kwok-Fai So, Kai Wang
NATURE COMMUNICATIONS
(2016)
Article
Rheumatology
Ji Yih Chen, Yee Ling Wu, Mo Yin Mok, Yeong-Jian Jan Wu, Katherine E. Lintner, Chin-Man Wang, Erwin K. Chung, Yan Yang, Bi Zhou, Huanyu Wang, Denise J. H. C. Yu, Alaaedin Alhomosh, Karla Jones, Charles H. Spencer, Haikady N. Nagaraja, Yu Lung Lau, Chak-Sing Lau, C. Yung Yu
ARTHRITIS & RHEUMATOLOGY
(2016)
Review
Immunology
Katherine E. Lintner, Yee Ling Wu, Yan Yang, Charles H. Spencer, Georges Hauptmann, Lee A. Hebert, John P. Atkinson, C. Yung Yu
FRONTIERS IN IMMUNOLOGY
(2016)
Article
Pediatrics
Rabheh Abdul-Aziz, Chack-Yung Yu, Brent Adler, Sharon Bout-Tabaku, Katherine E. Lintner, Melissa Moore-Clingenpeel, Charles H. Spencer
PEDIATRIC RHEUMATOLOGY
(2017)
Article
Genetics & Heredity
Christopher J. Lessard, Indra Adrianto, John A. Ice, Graham B. Wiley, Jennifer A. Kelly, Stuart B. Glenn, Adam J. Adler, He Li, Astrid Rasmussen, Adrienne H. Williams, Julie Ziegler, Mary E. Comeau, Miranda Marion, Benjamin E. Wakeland, Chaoying Liang, Paula S. Ramos, Kiely M. Grundahl, Caroline J. Gallant, Marta E. Alarcon-Riquelme, Graciela S. Alarcon, Juan-Manuel Anaya, Sang-Cheol Bae, Susan A. Boackle, Elizabeth E. Brown, Deh-Ming Chang, Soo-Kyung Cho, Lindsey A. Criswell, Jeffrey C. Edberg, Barry I. Freedman, Gary S. Gilkeson, Chaim O. Jacob, Judith A. James, Diane L. Kamen, Robert P. Kimberly, Jae-Hoon Kim, Javier Martin, Joan T. Merrill, Timothy B. Niewold, So-Yeon Park, Michelle A. Petri, Bernardo A. Pons-Estel, Rosalind Ramsey-Goldman, John D. Reveille, R. Hal Scofield, Yeong Wook Song, Anne M. Stevens, Betty P. Tsao, Luis M. Vila, Timothy J. Vyse, Chack-Yung Yu, Joel M. Guthridge, Kenneth M. Kaufman, John B. Harley, Edward K. Wakeland, Carl D. Langefeld, Patrick M. Gaffney, Courtney G. Montgomery, Kathy L. Moser
AMERICAN JOURNAL OF HUMAN GENETICS
(2012)
Article
Genetics & Heredity
Michael Mueller, Paula Barros, Abigail S. Witherden, Amy L. Roberts, Zhou Zhang, Helmut Schaschl, Chack-Yung Yu, Matthew E. Hurles, Catherine Schaffner, R. Andres Floto, Laurence Game, Karyn Meltz Steinberg, Richard K. Wilson, Tina A. Graves, Evan E. Eichler, H. Terence Cook, Timothy J. Vyse, Timothy J. Aitman
AMERICAN JOURNAL OF HUMAN GENETICS
(2013)
Article
Rheumatology
Kenneth M. Kaufman, Jian Zhao, Jennifer A. Kelly, Travis Hughes, Adam Adler, Elena Sanchez, Joshua O. Ojwang, Carl D. Langefeld, Julie T. Ziegler, Adrienne H. Williams, Mary E. Comeau, Miranda C. Marion, Stuart B. Glenn, Rita M. Cantor, Jennifer M. Grossman, Bevra H. Hahn, Yeong Wook Song, Chack-Yung Yu, Judith A. James, Joel M. Guthridge, Elizabeth E. Brown, Graciela S. Alarcon, Robert P. Kimberly, Jeffrey C. Edberg, Rosalind Ramsey-Goldman, Michelle A. Petri, John D. Reveille, Luis M. Vila, Juan-Manuel Anaya, Susan A. Boackle, Anne M. Stevens, Barry I. Freedman, Lindsey A. Criswell, Bernardo A. Pons-Estel, Joo-Hyun Lee, Ji-Seon Lee, Deh-Ming Chang, R. Hal A. Scofield, Gary S. Gilkeson, Joan T. Merrill, Timothy B. Niewold, Timothy James Vyse, Sang-Cheol Bae, Marta E. Alarcon-Riquelme, Chaim O. Jacob, Kathy Moser Sivils, Patrick M. Gaffney, John B. Harley, Amr H. Sawalha, Betty P. Tsao
ANNALS OF THE RHEUMATIC DISEASES
(2013)
Article
Rheumatology
Rachel Kaiser, Kimberly E. Taylor, Yun Deng, Jian Zhao, Yonghong Li, Joanne Nititham, Monica Chang, Joseph Catanese, Ann B. Begovich, Elizabeth E. Brown, Jeffrey C. Edberg, Gerald McGwin, Graciela S. Alarcon, Rosalind Ramsey-Goldman, John D. Reveille, Luis M. Vila, Michelle Petri, Robert P. Kimberly, Xuebing Feng, Lingyun Sun, Nan Shen, Wei Li, Jian-Xin Lu, Edward K. Wakeland, Quan-Zhen Li, Wanling Yang, Yu-Lung Lau, Fei-Lan Liu, Deh-Ming Chang, Chack-Yung Yu, Yeong W. Song, Hwee Siew Howe, Betty P. Tsao, Lindsey A. Criswell
ARTHRITIS AND RHEUMATISM
(2013)
Article
Rheumatology
D. J. Birmingham, L. A. Hebert, H. Song, W. T. Noonan, B. H. Rovin, H. N. Nagaraja, C. Y. Yu
Article
Endocrinology & Metabolism
Suzanne E. Kingery, Yee Ling Wu, Bi Zhou, Robert P. Hoffman, C. Yung Yu
PEDIATRIC DIABETES
(2012)
Article
Genetics & Heredity
Daisuke Sakurai, Jian Zhao, Yun Deng, Jennifer A. Kelly, Elizabeth E. Brown, John B. Harley, Sang-Cheol Bae, Marta E. Alarcon-Riqueime, Jeffrey C. Edberg, Robert P. Kimberly, Rosalind Ramsey-Goldman, Michelle A. Petri, John D. Reveille, Luis M. Vila, Graciela S. Alarcon, Kenneth M. Kaufman, Timothy J. Vyse, Chaim O. Jacob, Patrick M. Gaffney, Kathy Moser Sivils, Judith A. James, Diane L. Kamen, Gary S. Gilkeson, Timothy B. Niewold, Joan T. Merrill, R. Hal Scofield, Lindsey A. Criswell, Anne M. Stevens, Susan A. Boackle, Jae-Hoon Kim, Jiyoung Choi, Bernardo A. Pons-Estel, Barry I. Freedman, Juan-Manuel Anaya, Javier Martin, C. Yung Yu, Deh-Ming Chang, Yeong Wook Song, Carl D. Langefeld, Weiling Chen, Jennifer M. Grossman, Rita M. Cantor, Bevra H. Hahn, Betty P. Tsao
Review
Endocrinology & Metabolism
Melanie Copenhaver, Chack-Yung Yu, Robert P. Hoffman
CURRENT DIABETES REVIEWS
(2019)
