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Renal disease
Published in Catherine Nelson-Piercy, Handbook of Obstetric Medicine, 2020
Plasmapheresis is not needed for HELLP syndrome, which usually improves with conservative therapy. It is the treatment of choice for TTP (thrombotic thrombocytopenic purpura). Eculizumab is a recombinant, humanized, monoclonal antibody directed against C5. It has replaced plasma exchange as the gold standard in the management of complement-mediated primary aHUS (atypical hemolytic uremic syndrome).Dialysis may become necessary in AKI to prevent or treat uraemia, acidosis, hyperkalaemia or fluid overload, but a requirement for long-term renal replacement therapy is very unusual.
Cobalamin C, D, F, G diseases; methylmalonic aciduria and variable homocystinuria
Published in William L. Nyhan, Georg F. Hoffmann, Aida I. Al-Aqeel, Bruce A. Barshop, Atlas of Inherited Metabolic Diseases, 2020
William L. Nyhan, Georg F. Hoffmann, Aida I. Al-Aqeel, Bruce A. Barshop
The excretion of methylmalonic acid in the urine was statistically significantly higher in patients with Cbl X (p<0.01) than with Cbl C [13]. Plasma concentrations of homocysteine were not statistically different, but the range, both higher and lower, was greater for the homocysteine in plasma; normal levels were recorded in four. The most commonly defective abnormality of intercellular cobalamin metabolism is Cbl C [29]. Elevated levels of methylmalonic acid and homocysteine and decreased production of methionine are the biochemical hallmarks of the disease. Macular and retinal degeneration are unique features of Cbl C disease [30]. Deterioration of visual activity was documented [31]. Maculopathy, retinopathy, nystagmus, strabismus and optic atrophy were commonly encountered. Among the clinical manifestations, an atypical hemolytic uremic syndrome is uncommon [32]. It occurs generally within the first 30 days of life. It has also been reported [33] in a 20-year old man with malignant hypertension and renal failure. Biopsy revealed glomerular arteriolar thrombotic microangiopathy.
The complement system in health and disease
Published in Gabriel Virella, Medical Immunology, 2019
Atypical hemolytic uremic syndrome (aHUS) is a rare disease that causes blood clots that, over time, reduce significantly the blood flow in the kidneys, impairing its ability to remove waste products, which ultimately leads to kidney failure. aHUS is associated with mutations in proteins of the alternative pathway, primarily factor H, that lead to uncontrolled complement activation. Recent clinical trials have shown that eculizumab therapy improves outcomes, which has led to the approval of its use in aHUS.
Pharmacotherapeutic options for the prevention of kidney transplant rejection: the evidence to date
Published in Expert Opinion on Pharmacotherapy, 2022
Goce Spasovski, Lada Trajceska, Irena Rambabova-Bushljetik
Recently, immunosuppressant minimization strategies are considered, based on the associated complications (toxicity and/or adverse reactions) [10]. Thus, individual CNI concentration levels are monitored in order to prevent more frequent AR episodes while limiting their nephrotoxicity [11]. Steroid discontinuation resulted in somewhat increased AR possibilities, although with preserved graft function and survival [12]. MMF is frequently reduced alleviating gastrointestinal and hematological side effects and infections. Nowadays, a couple of new drugs (belatacept and alemtuzumab) may be a treatment of choice for induction of donor-specific immunotolerance or mixed chimerism [13–15]. Of note, the combined immunosuppression should minimize the individual impact on morbidity and mortality, while the overall drug effectiveness would be improved. However, the problem of unrecognized production of de novo or preexisting anti-HLA antibodies and development of chronic antibody-mediated rejection (cAMR) and transplant glomerulopathy (TG) as sequel that prevents long-term graft survival need further analysis and update in the treatment. Finally, therapeutical possibilities for the small proportion of patients with cancers and atypical hemolytic uremic syndrome (aHUS) should be further evaluated. The actual immunosuppressants with their mechanism of action and possible side effects are presented in Table 1.
A US cost-minimization model comparing ravulizumab versus eculizumab for the treatment of atypical hemolytic uremic syndrome
Published in Journal of Medical Economics, 2020
Yan Wang, Karissa Johnston, Evan Popoff, Karl-Johan Myren, Antoinette Cheung, Claudio Faria, Ioannis Tomazos
Atypical hemolytic uremic syndrome (aHUS) is a rare, systemic, and unpredictable disease that can cause severe, progressive organ damage and death in patients of all ages if left untreated1–4. Estimates of disease prevalence are variable, and range from 2.2–9.4 cases per million individuals among the general population aged ≥20 years5. aHUS is characterized by systemic thrombotic microangiopathy (TMA), resulting from uncontrolled activation of the complement system3,4,6. Patients typically experience recurring episodes of hemolytic anemia, thrombocytopenia, and acute kidney injury that ultimately progresses to end-stage renal disease (ESRD)6, although some patients also develop extra-renal symptoms and organ damage7.
Belgian consensus statement on the diagnosis and management of patients with atypical hemolytic uremic syndrome
Published in Acta Clinica Belgica, 2018
Kathleen J Claes, Annick Massart, Laure Collard, Laurent Weekers, Eric Goffin, Jean-Michel Pochet, Karin Dahan, Johann Morelle, Brigitte Adams, Nilufer Broeders, Patrick Stordeur, Daniel Abramowicz, Jean-Louis Bosmans, Koen Van Hoeck, Peter Janssens, Lissa Pipeleers, Patrick Peeters, Steven Van Laecke, Elena Levtchenko, Ben Sprangers, Lambertus van den Heuvel, Nathalie Godefroid, Johan Van de Walle
In January 2017, a protocol with recommendations for the diagnosis and treatment of patients with a (possible) diagnosis of atypical Hemolytic Uremic Syndrome was proposed. This protocol was developed by a Belgian working group consisting of clinicians, both adult and pediatric nephrologists, biologists and a geneticist working in university hospitals, with a particular interest and expertise in diagnosis and treatment of aHUS. The recent breakthroughs in diagnosis and treatment of aHUS urged us to address some highly relevant questions in the diagnostic and therapeutic field of aHUS. Therefore, the following text is a general consensus document on the diagnosis and the therapeutic approach of this disease. Since aHUS is an ultra-rare condition and randomized controlled trials are absent, evidence-based guidelines cannot be provided. Consensus was reached on diagnostic approach, treatment duration in native aHUS and the use and duration of eculizumab as a preventive treatment in renal transplantation. The working group will have a meeting at least every year in order to update the diagnostic and treatment recommendations based on the last available scientific evidence and clinical experience.