Article
Urology & Nephrology
Mendy ter Avest, Hilbert Steenbreker, Romy N. Bouwmeester, Caroline Duineveld, Kioa L. Wijnsma, Lambertus P. W. J. van den Heuvel, Saskia M. C. Langemeijer, Jack F. M. Wetzels, Nicole C. A. J. van de Kar, Rob ter Heine, CUREiHUS Study Grp
Summary: This study investigated the effect of proteinuria on the pharmacokinetics of eculizumab. The findings suggest that patients with severe proteinuria have a higher risk of underexposure to eculizumab.
CLINICAL JOURNAL OF THE AMERICAN SOCIETY OF NEPHROLOGY
(2023)
Article
Urology & Nephrology
Caroline Duineveld, Romy N. Bouwmeester, Kioa L. Wijnsma, F. J. Bemelman, J. W. van der Heijden, S. P. Berger, L. P. W. J. van den Heuvel, Nicole C. A. J. van de Kar, Jack F. M. Wetzels
Summary: Kidney transplantation in aHUS patients in the Netherlands is performed without eculizumab prophylaxis, and eculizumab therapy is given in case of posttransplant aHUS recurrence. The study found a recurrence rate of 23% without eculizumab prophylaxis, and eculizumab treatment effectively improved or stabilized kidney function in most patients.
KIDNEY INTERNATIONAL REPORTS
(2023)
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)
Article
Urology & Nephrology
Gianluigi Ardissino, Donata Cresseri, Francesca Tel, Antenore Giussani, Stefania Salardi, Martina Sgarbanti, Bice Strumbo, Sara Testa, Valentina Capone, Samantha Griffini, Elena Grovetti, Massimo Cugno, Mirco Belingheri, Chiara Tamburello, Evangeline Millicent Rodrigues, Michela Perrone, Massimo Cardillo, Grazia Corti, Dario Consonni, Lucrezia Furian, Silvana Tedeschi, Piergiorgio Messa, Claudio Beretta
Summary: This study presents experience with KTx in aHUS patients, showing that prophylactic use of Eculizumab can significantly reduce relapse rates. Tailored timing of Eculizumab doses based on classical complement pathway activity for each patient has also shown positive results.
JOURNAL OF NEPHROLOGY
(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)
Article
Urology & Nephrology
Massimo Cugno, Valentina Capone, Samantha Griffini, Elena Grovetti, Giulia Pintarelli, Luigi Porcaro, Emilio Clementi, Gianluigi Ardissino
Summary: This study reports on the experience with eculizumab maintenance treatment in patients with aHUS. The interval between eculizumab doses was adjusted based on CCP activity, with the goal of maintaining CCP activity below 30% to prevent relapse. Patients who received eculizumab regularly at tailored intervals showed no relapse during the observation period, and there was an inverse correlation between CCP activity and eculizumab circulating levels.
JOURNAL OF NEPHROLOGY
(2022)
Article
Urology & Nephrology
Esra Baskin, Kibriya Fidan, Bora Gulhan, Kaan Gulleroglu, Nur Canpolat, Alev Yilmaz, Gonul Parmakiz, Birsin Z. Ozcakar, Fatih Ozaltin, Oguz Soylemezoglu
Summary: This study retrospectively analyzed the outcome of pediatric patients with aHUS who discontinued eculizumab treatment. The study found that eculizumab can be safely discontinued with close monitoring in a selected group of patients, and if recurrence occurs, restarting eculizumab immediately can achieve complete remission.
JOURNAL OF NEPHROLOGY
(2022)
Article
Urology & Nephrology
Jean-Michel Halimi, Imad Al-Dakkak, Katerina Anokhina, Gianluigi Ardissino, Christoph Licht, Wai H. Lim, Annick Massart, Franz Schaefer, Johan Vande Walle, Eric Rondeau
Summary: This analysis of the Global aHUS Registry provides further insights into the potential relationship between aHUS and malignant hypertension. The study finds that patients with aHUS and malignant hypertension have a higher need for renal replacement therapy and a higher prevalence of pathogenic variants/anti-complement factor H antibodies. Additionally, the study suggests that eculizumab is effective in treating aHUS with or without malignant hypertension.
JOURNAL OF NEPHROLOGY
(2023)
Article
Urology & Nephrology
Miguel Ernandes Neto, Lucas de Moraes Soler, Halita Vieira Gallindo Vasconcelos, Hong Si Nga, Ariane Moyses Bravin, Julio Cesar Andriotti Borges, Rodrigo Costa Goncalves, Rodrigo Brum Von Kriiger, Raquel Martins Quinino, Viviane Brandao Bandeira de Mello Santana, Maria Izabel de Holanda, Maria Helena Vaisbich, Alice Pignaton Naseri, Gianna Mastroianni Kirsztajn, Lilian Monteiro Pereira Palma, Luis Gustavo Modelli Andrade
Summary: This study analyzed the risk of aHUS relapse in Brazilian patients after eculizumab interruption due to drug shortage. It was found that the cumulative incidence of aHUS relapse at 397 days was 58%, and baseline variables were not associated with relapse rate. The interruption of eculizumab therapy was deemed unsafe due to the high rate of relapse.
JOURNAL OF NEPHROLOGY
(2021)
Article
Urology & Nephrology
Maria Helena Vaisbich, Luis Gustavo Modelli de Andrade, Precil Diego Miranda de Menezes Neves, Lilian Monteiro Pereira Palma, Maria Cristina Ribeiro de Castro, Cassiano Augusto Braga Silva, Maria Izabel Neves de Holanda Barbosa, Maria Goretti Moreira Guimaraes Penido, Oreste Angelo Ferra Neto, Roberta Mendes Lima Sobral, Silvana Maria Carvalho Miranda, Stanley de Almeida Araujo, Igor Gouveia Pietrobom, Henrique Mochida Takase, Claudia Ribeiro, Rafael Marques da Silva, Cesar Augusto Almeida de Carvalho, David Jose Barros Machado, Ana Mateus Simoes Teixeira E Silva, Andreia Ribeiro da Silva, Enzo Ricardo Russo, Flavio Henrique Soares Barros, Jarinne Camilo Landim Nasserala, Luciana Schmitt Cardon de Oliveira, Lucimary de Castro Sylvestre, Rafael Weissheimer, Sueli Oliveira Nascimento, Gilson Bianchini, Fellype de Carvalho Barreto, Valeria Soares Pigozzi Veloso, Patricia Marques Fortes, Vinicius Sardao Colares, Jaelson Guilhem Gomes, Andre Falcao Pedrosa Leite, Pablo Girardelli Mendonca Mesquita, Osvaldo Merege Vieira-Neto
Summary: This study has analyzed the first Brazilian registry for atypical hemolytic uremic syndrome (aHUS), revealing that the disease predominantly affects young adult women with renal involvement. Pediatric patients have lower hemoglobin and platelet levels and higher LDH levels compared to adults. The most common pathogenic variants were found in CFH and the CFHR1-3 deletion.
CLINICAL KIDNEY JOURNAL
(2022)
Article
Urology & Nephrology
Lilian Monteiro Pereira Palma, Renato George Eick, Gustavo Coelho Dantas, Michele Karen dos Santos Tino, Maria Izabel de Holanda
Summary: This study is the largest case series of aHUS in Brazil involving a wide range of patients for which eculizumab was the main treatment. Although eculizumab was started later than advised in the guidelines, most patients were able to stop dialysis at variable intervals. Discontinuation of eculizumab was associated with a 30% relapse of aHUS.
CLINICAL KIDNEY JOURNAL
(2021)
Article
Immunology
Cristina Casas Gonzalez, Veronica Lopez-Jimenez, Teresa Vazquez-Sanchez, Elena Vazquez-Sanchez, Mercedes Cabello, Domingo Hernandez-Marrero
Summary: The atypical hemolytic-uremic syndrome (aHUS) is a disease characterized by non-immune hemolytic anemia, thrombocytopenia, and acute renal failure. The use of eculizumab has improved the prognosis of aHUS and prevented recurrence after kidney transplantation.
TRANSPLANTATION PROCEEDINGS
(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)
Article
Pediatrics
Melissa Muff-Luett, Keia R. Sanderson, Rachel M. Engen, Rima S. Zahr, Scott E. Wenderfer, Cheryl L. Tran, Sheena Sharma, Yi Cai, Susan Ingraham, Erica Winnicki, Donald J. Weaver, Tracy E. Hunley, Stefan G. Kiessling, Meredith Seamon, Robert Woroniecki, Yosuke Miyashita, Nianzhou Xiao, Abiodun A. Omoloja, Sarah J. Kizilbash, Asif Mansuri, Mahmoud Kallash, Yichun Yu, Ashley K. Sherman, Tarak Srivastava, Carla M. Nester
Summary: This study investigated the broader use and outcomes of pediatric patients exposed to eculizumab, revealing a significant number of patients receiving off-label indications for C5 blockade. Although adverse events related to the medication were low, the cohort mortality rate was relatively high. Prospective studies in homogeneous disease cohorts are needed to confirm the role of C5 blockade in kidney outcomes.
PEDIATRIC NEPHROLOGY
(2021)
Article
Health Care Sciences & Services
Agnieszka Furmanczyk-Zawiska, Anna Kubiak-Dydo, Ewelina Uzarowska-Gaska, Marta Kotlarek-Lysakowska, Katarzyna Salata, Monika Kolanowska, Michal Swierniak, Pawel Gaj, Beata Leszczynska, Maria Daniel, Krystian Jazdzewski, Magdalena Durlik, Anna Wojcicka
Summary: Atypical hemolytic uremic syndrome (aHUS) is a rare disease caused by genetic defects, with variable clinical outcomes, including renal failure. This study presents cases of five family members affected by aHUS, demonstrating the highly variable penetrance of the disease.
JOURNAL OF PERSONALIZED MEDICINE
(2021)
Article
Pediatrics
Dineke Westra, Elena B. Volokhina, Renate G. van der Molen, Thea J. A. M. van der Velden, Annelies Jeronimus-Klaasen, Joop Goertz, Valentina Gracchi, Eiske M. Dorresteijn, Antonia H. M. Bouts, Mandy G. Keijzer-Veen, Joanna A. E. van Wijk, Jaap A. Bakker, Anja Roos, Lambert P. van den Heuvel, Nicole C. A. J. van de Kar
PEDIATRIC NEPHROLOGY
(2017)
Article
Genetics & Heredity
Lambertus van den Heuvel, Kristian Riesbeck, Omaima El Tahir, Valentina Gracchi, Mariann Kremlitzka, Servaas A. Morre, A. Marceline van Furth, Birendra Singh, Marcin Okroj, Nicole van de Kar, Anna M. Blom, Elena Volokhina
JOURNAL OF HUMAN GENETICS
(2018)
Review
Pediatrics
Kioa L. Wijnsma, Caroline Duineveld, Jack F. M. Wetzels, Nicole C. A. J. van de Kar
PEDIATRIC NEPHROLOGY
(2019)
Article
Food Science & Technology
Elena B. Volokhina, Wouter J. C. Feitz, Lonneke M. Elders, Thea J. A. M. van der Velden, Nicole C. A. J. van de Kar, Lambertus P. W. J. van den Heuvel
Article
Food Science & Technology
Wouter J. C. Feitz, Nicole C. A. J. van de Kar, Ian Cheong, Thea J. A. M. van der Velden, Carolina G. Ortiz-Sandoval, Dorothea Orth-Hoeller, Lambert P. J. W. van den Heuvel, Christoph Licht
Review
Pediatrics
Marina Vivarelli, Nicole van de Kar, Raffaella Labbadia, Francesca Diomedi-Camassei, Joshua M. Thurman
Summary: C3 glomerulopathy is a challenging clinical entity with new therapeutic options emerging. Understanding complement dysregulation and its role in various glomerular diseases is crucial for optimizing patient care. Ongoing clinical trials with complement inhibitors are shedding light on the potential treatment options for these conditions.
PEDIATRIC NEPHROLOGY
(2022)
Article
Biochemistry & Molecular Biology
Wouter J. C. Feitz, Petra A. van Setten, Thea J. A. M. van der Velden, Christoph Licht, Lambert P. J. W. van den Heuvel, Nicole C. A. J. van de Kar
Summary: Hemolytic uremic syndrome (HUS) is a disease characterized by a triad of symptoms caused by infection with Shiga toxin-producing Escherichia coli bacteria. While predominantly affecting children, the syndrome leads to kidney damage. Research has shown that cells isolated from pediatric and adult kidneys do not significantly differ in their biological responses to Shiga toxin.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2021)
Article
Immunology
Marloes A. H. M. Michels, Rianne J. F. Maas, Thea J. A. M. van der Velden, Nicole C. A. J. van de Kar, Lambertus P. W. J. van den Heuvel, Elena B. Volokhina
Summary: Properdin plays a crucial role in complement system by promoting C5 activation and lytic activity independently of its function in new convertase formation.
JOURNAL OF IMMUNOLOGY
(2021)
Article
Microbiology
Wouter J. C. Feitz, Romy Bouwmeester, Thea J. A. M. van der Velden, Susan Goorden, Christoph Licht, Lambert P. J. W. van den Heuvel, Nicole C. A. J. van de Kar
Summary: This study suggests that inhibiting the synthesis of Gb3 could be a potential therapeutic target for protecting against (further) endothelial damage caused by Stx in the future.
Article
Biochemistry & Molecular Biology
Wouter J. C. Feitz, Samuel Suntharalingham, Meraj Khan, Carolina G. Ortiz-Sandoval, Nades Palaniyar, Lambert P. van den Heuvel, Nicole C. A. J. van de Kar, Christoph Licht
Summary: Stx2a induces NET formation in neutrophils through the NOX-dependent pathway, with neutrophils from acute STEC-HUS patients showing lower ROS and NET formation compared to the recovery phase and healthy controls.
Article
Health Care Sciences & Services
Merel L. Kimman, Adrienne H. Rotteveel, Marlies Wijsenbeek, Remy Mostard, Nelleke C. Tak, Xana van Jaarsveld, Marjolein Storm, Kioa L. Wijnsma, Marielle Gelens, Nicole C. A. J. van de Kar, Jack Wetzels, Carmen D. Dirksen
PATIENT-PATIENT CENTERED OUTCOMES RESEARCH
(2017)
Meeting Abstract
Pediatrics
Anne Schijvens, Natasja Dors, Nicole Van De Kar, Michiel Schreuder
PEDIATRIC NEPHROLOGY
(2017)
Meeting Abstract
Pediatrics
Lambertus Van Den Heuvel Van Den Heuvel, Kristian Riesbeck, Omaima El Tahir, Valentina Gracchi, Mariann Kremlitzka, Servaas Morre, Marceline Van Furth, Birendra Singh, Marcin Okroj, Nicole Van De Kar, Anna Blom, Elena Volokhina
PEDIATRIC NEPHROLOGY
(2017)
Meeting Abstract
Pediatrics
Elena Volokhina, Kioa Wijnsma, Renate Van der Molen, Nel Roeleveld, Thea Van der Velden, Joop Goertz, Fred Sweep, Roger Bruggemann, Jack Wetzels, Nicole Van de Kar, Lambertus Van den Heuvel
PEDIATRIC NEPHROLOGY
(2017)
Meeting Abstract
Pediatrics
Kioa Wijnsma, Susan Veissi, Diego Comerci, Nicole Van De Kar, Juan Ugalde, Bert Van Den Heuvel
PEDIATRIC NEPHROLOGY
(2017)