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
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)
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)
Review
Pediatrics
Lilian Monteiro P. Palma, Maria Helena Vaisbich-Guimaraes, Meera Sridharan, Cheryl L. Tran, Sanjeev Sethi
Summary: Thrombotic microangiopathy (TMA) is a clinical-pathological entity characterized by specific clinical features, with different underlying causes in children and adults. In children, rare genetic factors and transplantation-related risks need to be considered in addition to common causes. This article describes the etiology of TMA and provides guidance for clinical evaluation and treatment.
PEDIATRIC NEPHROLOGY
(2022)
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
Genetics & Heredity
Dervla M. Connaughton, Pratibha Bhai, Paul Isenring, Mohammed Mahdi, Bekim Sadikovic, Laila C. Schenkel
Summary: Atypical hemolytic uremic syndrome (aHUS) is a complex disease characterized by various genetic variants. A next-generation sequencing (NGS) panel was used to analyze a large cohort of Canadian patients, and it was found that only a small percentage of patients had pathogenic/likely pathogenic variants. The study highlights the challenges in variant classification and the importance of considering variable expressivity and incomplete penetrance in interpreting genetic data in aHUS patients.
JOURNAL OF MOLECULAR MEDICINE-JMM
(2023)
Article
Immunology
S. Elhadad, J. Chapin, D. Copertino, K. Van Besien, J. Ahamed, J. Laurence
Summary: The alternative complement pathway is involved in microvascular endothelial cell injury in thrombotic microangiopathies. Inhibition of MASP2 can reduce plasma-induced MVEC injury, suggesting potential therapeutic benefits in these disorders.
CLINICAL AND EXPERIMENTAL IMMUNOLOGY
(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
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
Romy N. Bouwmeester, Caroline Duineveld, Kioa L. Wijnsma, Frederike J. Bemelman, Joost W. van der Heijden, Joanna A. E. van Wijk, Antonia H. M. Bouts, Jacqueline van de Wetering, Eiske Dorresteijn, Stefan P. Berger, Valentina Gracchi, Arjan D. van Zuilen, Mandy G. Keijzer-Veen, Aiko P. J. de Vries, Roos W. G. van Rooij, Flore A. P. T. Engels, Wim Altena, Renee de Wildt, Evy van Kempen, Eddy M. Adang, Mendy ter Avest, Rob ter Heine, Elena B. Volokhina, Lambertus P. W. J. van den Heuvel, Jack F. M. Wetzels, Nicole C. A. J. van de Kar
Summary: The study found that discontinuing eculizumab after 3 months of therapy is safe and cost-effective for patients with aHUS in native kidneys. Most patients showed full recovery of hematological thrombotic microangiopathy parameters after treatment, with the majority also experiencing renal response.
KIDNEY INTERNATIONAL REPORTS
(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
Urology & Nephrology
Sjoerd A. M. E. G. Timmermans, Jan G. M. C. Damoiseaux, Alexis Werion, Chris P. Reutelingsperger, Johann Morelle, Pieter van Paassen
Summary: Thrombotic microangiopathy (TMA) syndromes represent severe endothelial damage caused by various mechanisms. The study found that assessment of serum-induced ex vivo C5b9 formation and screening for rare variants in complement genes can better categorize TMA, with complement playing a major role in driving the disease, indicating potential therapeutic and prognostic implications.
KIDNEY INTERNATIONAL REPORTS
(2021)
Article
Hematology
Vanessa Vilani Addad, Lilian Monteiro Pereira Palma, Maria Helena Vaisbich, Abner Macola Pacheco Barbosa, Naila Camila da Rocha, Marilia Mastrocolla de Almeida Cardoso, Juliana Tereza Coneglian de Almeida, Monica A. P. de Paula de Sordi, Juliana Machado-Rugolo, Lucas Frederico Arantes, Luis Gustavo Modelli de Andrade
Summary: In this retrospective cohort study, an algorithm was developed to classify Thrombotic Microangiopathy (TMA) using clinical characteristics and biochemical exams. The study found that secondary TMA was the most common type, while primary TMA mainly consisted of TTP and aHUS. The model showed high accuracy in predicting TMA in most categories.
THROMBOSIS JOURNAL
(2023)
Review
Pharmacology & Pharmacy
Tommaso Mazzierli, Federica Allegretta, Enrico Maffini, Marco Allinovi
Summary: Drug-induced thrombotic microangiopathy (DITMA) accounts for 10%-13% of all thrombotic microangiopathy (TMA) cases and about 20%-30% of secondary TMAs, just behind pregnancy-related and infection-related forms. The scientific literature on DITMA is limited, leading to poor understanding of its mechanisms and management. In this review, we provide an updated list of TMA-associated drugs and describe the clinical features of DITMA, as well as analyze the association between signs/symptoms and specific causative drugs. We also discuss the role of complement system and genetic deregulation in DITMA and propose a treatment approach based on recent literature data.
FRONTIERS IN PHARMACOLOGY
(2023)
Review
Urology & Nephrology
Ramy M. Hanna, Kammi Henriksen, Kamyar Kalantar-Zadeh, Antoney Ferrey, Richard Burwick, Kenar D. Jhaveri
Summary: Thrombotic microangiopathies (TMAs) are a group of disorders characterized by microangiopathic hemolytic anemia and end-organ dysfunction. There are different types of TMAs, including thrombotic thrombocytopenic purpura, Shiga toxin-mediated hemolytic uremic syndrome, and atypical hemolytic uremic syndrome. Other forms of TMA, such as drug-induced TMA, rheumatological disease-induced TMA, and renal-limited TMA, exist in an intermediate space and may overlap with atypical hemolytic uremic syndrome clinically. Hematopoietic stem cell transplant-TMA is a more resistant form of TMA with different therapeutic needs and clinical course. The understanding of TMA syndromes is an emerging field in nephrology, rheumatology, and hematology, and further research is needed to unravel the relationships and distinctions between different subclasses of TMA syndromes.
ADVANCES IN CHRONIC KIDNEY DISEASE
(2022)