Article
Urology & Nephrology
Gema Ariceta, Bradley P. Dixon, Seong Heon Kim, Gaurav Kapur, Teri Mauch, Stephan Ortiz, Marc Vallee, Andrew E. Denker, Hee Gyung Kang, Larry A. Greenbaum
Summary: Ravulizumab rapidly improved hematologic and kidney parameters in complement inhibitor-naive children with atypical hemolytic uremic syndrome, showing no unexpected safety concerns.
KIDNEY INTERNATIONAL
(2021)
Review
Medicine, General & Internal
Ali Jandal, Weixiong Zhong, Deepak Gopal, Vanessa Horner, Leah Frater-Rubsam, Arjang Djamali, Gauri Bhutani
Summary: We present a case of a female patient with mixed nephritic-nephrotic syndrome and recurrent pancreatitis. Kidney biopsy revealed crescentic membranoproliferative glomerulonephritis with dominant C3 staining on immunofluorescence. Genetic testing revealed a rare C3 variant and a deletion of CFHR3CFHR1. The case highlights the challenges of treating complement-mediated kidney disease and suggests the existence of a C3G/aHUS overlap syndrome.
AMERICAN JOURNAL OF THE MEDICAL SCIENCES
(2023)
Review
Urology & Nephrology
Zhen Chun Tang, Huang Hui, Chunru Shi, Xiangmei Chen
Summary: This comprehensive review and meta-analysis of clinical trials show that eculizumab can reduce the recurrence of atypical hemolytic uremic syndrome in renal transplant patients, decrease the need for dialysis, improve renal function, and reduce the risk of rejection.
Article
Medicine, General & Internal
Ching-Hu Chung, I-Jung Tsai, Min-Hua Tseng, Hsin-Hsu Chou, You-Lin Tain, Jeng-Daw Tsai, Yuan-Yow Chiou, Yee-Hsuan Chiou, Ching-Yuang Lin
Summary: Thrombotic microangiopathy (TMA) syndromes exhibit diverse clinical presentations and etiologies, with common triggers in Taiwan such as pregnancy and systemic lupus erythematosus. The mortality rate in TMA patients treated with plasmapheresis is significantly higher than those without treatment, indicating potential masked underlying etiologies or worsening disease conditions.
Article
Urology & Nephrology
Arnaud Garnier, Karine Brochard, Theresa Kwon, Anne-Laure Sellier-Leclerc, Annie Lahoche, Emma Allain Launay, Francois Nobili, Mathilde Caillez, Sophie Taque, Jerome Harambat, Guylhene Michel-Bourdat, Vincent Guigonis, Marc Fila, Sylvie Cloarec, Djeddi Djamal-Dine, Loic de Parscaux, Lise Allard, Remi Salomon, Tim Ulinski, Veronique Fremeaux-Bacchi, Christophe Morin, Pascale Olivier-Abbal, Helene Colineaux, Francoise Auriol, Catherine Arnaud, Isabelle Kieffer, Clara Brusq
Summary: This study examined the use of eculizumab in children with STEC-HUS and found that it did not improve renal outcomes during the acute phase of the disease, but may reduce long-term kidney sequelae.
JOURNAL OF THE AMERICAN SOCIETY OF NEPHROLOGY
(2023)
Article
Urology & Nephrology
Arnaud Garnier, Karine Brochard, Theresa Kwon, Anne-Laure Sellier-Leclerc, Annie Lahoche, Emma Allain Launay, Francois Nobili, Mathilde Caillez, Sophie Taque, Jerome Harambat, Guylhene Michel-Bourdat, Vincent Guigonis, Marc Fila, Sylvie Cloarec, Djeddi Djamal-Dine, Loic de Parscaux, Lise Allard, Remi Salomon, Tim Ulinski, Veronique Fremeaux-Bacchi, Christophe Morin, Pascale Olivier-Abbal, Helene Colineaux, Francoise Auriol, Catherine Arnaud, Isabelle Kieffer, Clara Brusq
Summary: In pediatric patients with STEC-HUS, treatment with eculizumab does not appear to improve renal outcome during the acute phase of the disease but may reduce long-term kidney sequelae.
JOURNAL OF THE AMERICAN SOCIETY OF NEPHROLOGY
(2023)
Article
Immunology
Yuzhou Zhang, Renee X. Goodfellow, Nicolo Ghiringhelli Borsa, Hannah C. Dunlop, Stephen A. Presti, Nicole C. Meyer, Dingwu Shao, Sarah M. Roberts, Michael B. Jones, Gabriella R. Pitcher, Amanda O. Taylor, Carla M. Nester, Richard J. H. Smith
Summary: C3 glomerulopathy (C3G) and atypical hemolytic uremic syndrome (aHUS) are rare diseases caused by dysregulated activity of the alternative pathway of complement. This study investigated the functional activity of select CFI missense variants in patients with C3G and aHUS. The researchers found that rare CFI variants were associated with altered FI protein levels and activity, highlighting the complexity and multifactorial nature of these complement-mediated renal diseases.
FRONTIERS IN IMMUNOLOGY
(2022)
Article
Pediatrics
Kazuki Tanaka, Brigitte Adams, Alvaro Madrid Aris, Naoya Fujita, Masayo Ogawa, Stephan Ortiz, Marc Vallee, Larry A. Greenbaum
Summary: The study demonstrated that pediatric patients with aHUS who switched from chronic eculizumab to ravulizumab treatment experienced stable kidney and hematologic parameters, with low occurrence of adverse events.
PEDIATRIC NEPHROLOGY
(2021)
Review
Immunology
Maryam Saleem, Sana Shaikh, Zheng Hu, Nicola Pozzi, Anuja Java
Summary: Thrombotic microangiopathy (TMA) is a condition characterized by microangiopathic hemolytic anemia, thrombocytopenia, and organ injury caused by endothelial cell damage and microthrombi formation. Membranous nephropathy can be either primary, associated with circulating autoantibodies, or secondary, associated with infections, drugs, cancer, or other autoimmune diseases. Differentiating between primary and secondary TMA as well as primary and secondary membranous nephropathy is crucial for timely treatment, but can be challenging for clinicians, particularly after kidney transplant.
FRONTIERS IN IMMUNOLOGY
(2022)
Article
Immunology
Margarita Lopez-Trascasa, Angel Alonso-Melgar, Marta Melgosa-Hijosa, Laura Espinosa-Roman, Maria Dolores Lledin-Barbancho, Eugenia Garcia-Fernandez, Santiago Rodriguez de Cordoba, Pilar Sanchez-Corral
Summary: Pathogenic gain-of-function mutations in complement Factor B were found to be the cause of atypical HUS in 2007. A four-month-old boy diagnosed with hypocomplementemic aHUS in 2000 suffered severe complications and end-stage renal disease by the age of 3, eventually undergoing a combined liver and kidney transplantation in 2009. After experiencing post-transplant complications, including B-cell non-Hodgkin lymphoma and liver rejection, the patient currently maintains normal kidney and liver functions 12 years post-surgery, suggesting that CLKT was an acceptable therapeutic option.
FRONTIERS IN IMMUNOLOGY
(2021)
Review
Urology & Nephrology
Dominique Suzanne Genest, Christopher J. Patriquin, Christoph Licht, Rohan John, Heather N. Reich
Summary: Thrombotic microangiopathy (TMA) is a common pathological lesion caused by endothelial injury and dysfunction. Renal-limited forms of TMA are not uncommon and often pose diagnostic challenges. Prompt investigation and treatment are important to reduce morbidity and mortality. Advances in understanding the pathophysiology of TMA have led to the development of diagnostic tools and targeted therapies. This article reviews the clinical presentation, pathologic hallmarks, and treatment strategies for TMA involving the kidney.
AMERICAN JOURNAL OF KIDNEY DISEASES
(2023)
Review
Medicine, General & Internal
Ana Avila, Eva Gavela, Asuncion Sancho
Summary: Thrombotic microangiopathy is a rare but serious complication affecting kidney transplant recipients, characterized by systemic or localized symptoms. Treatment options include plasma exchange and complement blockade.
FRONTIERS IN MEDICINE
(2021)
Article
Pediatrics
Corentin Tanne, Etienne Javouhey, Olivia Boyer, Morgan Recher, Emma Allain-Launay, Catherine Monet-Didailler, Caroline Rouset-Rouviere, Amelie Ryckewaert, Francois Nobili, Francine Arfbez Gindre, Jerome Rambaud, Anita Duncan, Julien Berthiller, Justine Bacchetta, Anne-Laure Sellier-Leclerc
Summary: This study describes the occurrence of myocarditis in pediatric patients with hemolytic uremic syndrome (HUS) and identifies leukocyte count and platelet transfusion as possible risk factors for myocarditis.
PEDIATRIC NEPHROLOGY
(2022)
Review
Medicine, General & Internal
Sjoerd A. M. E. G. Timmermans, Pieter van Paassen
Summary: Thrombotic microangiopathy (TMA) is a rare and life-threatening condition, often affecting the brain and kidneys. Complement dysregulation plays a crucial role in TMA, particularly in secondary atypical HUS patients presenting with coexisting conditions such as hypertensive emergency, pregnancy, and kidney transplantation, shifting the paradigm of the disease.
JOURNAL OF CLINICAL MEDICINE
(2021)
Article
Urology & Nephrology
Thomas Barbour, Marie Scully, Gema Ariceta, Spero Cataland, Katherine Garlo, Nils Heyne, Yosu Luque, Jan Menne, Yoshitaka Miyakawa, Sung-Soo Yoon, David Kavanagh
Summary: Ravulizumab demonstrates long-term efficacy and safety in adults with aHUS, providing additional clinical benefits beyond 6 months of treatment, with most adverse events occurring early during the initial evaluation period and decreasing over time.
KIDNEY INTERNATIONAL REPORTS
(2021)