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https://www.readbyqxmd.com/read/29674658/high-dose-carboplatin-etoposide-melphalan-increases-risk-of-thrombotic-microangiopathy-and-organ-injury-after-autologous-stem-cell-transplantation-in-patients-with-neuroblastoma
#1
Sonata Jodele, Christopher E Dandoy, Kasiani Myers, Gregory Wallace, Adam Lane, Ashley Teusink-Cross, Brian Weiss, Stella M Davies
Transplant-associated thrombotic microangiopathy (TA-TMA) is an increasingly recognized complication of hematopoietic cell transplant that can result in multi-organ failure (MOF). Patients undergoing high-dose chemotherapy with autologous stem cell transplant (aHCT) for neuroblastoma require good organ function to receive post-transplant radiation and immunotherapy. We examined TA-TMA incidence and transplant outcomes in patients with neuroblastoma receiving different transplant preparative regimens. Sixty patients underwent aHCT using high-dose chemotherapy: 41 patients received carboplatin/etoposide/melphalan (CEM), 13 patients busulfan/melphalan (Bu/Mel) and six patients received tandem transplant (cyclophosphamide/thiotepa and CEM)...
April 19, 2018: Bone Marrow Transplantation
https://www.readbyqxmd.com/read/29661469/successful-7-year-eculizumab-treatment-of-plasmapheresis-resistant-recurrent-atypical-hemolytic-uremic-syndrome-due-to-complement-factor-h-hybrid-gene-a-case-report
#2
K Vondrák, T Seeman
Atypical hemolytic-uremic syndrome (aHUS) is an extremely rare disease, and up to 70% of the patients have a genetic mutation in the encoding components of complement activation or anti-complement factor H autoantibodies. The risk of recurrence after kidney transplantation is 10% to 80%. Eculizumab, a monoclonal antibody that binds complement protein C5, has shown to be highly effective in patients with aHUS; however, there are only few reports on the efficacy and safety of long-term eculizumab treatment in children with recurrent aHUS...
April 2018: Transplantation Proceedings
https://www.readbyqxmd.com/read/29659362/-recurrent-atypical-hemolytic-uremic-syndrome-after-renal-transplantation-treatment-with-eculizumab
#3
Ana B Latzke, Pehuén Fernández, Carlos Chiurchiu, Daniela Sarmantano, Javier De Arteaga, Walter Douthat, Jorge De la Fuente
Atypical hemolytic uremic syndrome (aHUS) is a rare entity. It is characterized by a thrombotic microangiopathy (nonimmune hemolytic anemia, thrombocytopenia, and acute renal failure), with a typical histopathology of thickening of capillary and arteriolar walls and an obstructive thrombosis of the vascular lumen. The syndrome is produced by a genetic or acquired deregulation of the alternative pathway of the complement system, with high rates of end stage renal disease, post-transplant recurrence, and high mortality...
2018: Medicina
https://www.readbyqxmd.com/read/29657196/atypical-hemolytic-uremic-syndrome-a-monocentric-adult-tunisian-study-and-review-of-literature
#4
R Kherder-Elfekih, M Hajji, L Ben Fatma, H Jebali, I Mami, W Smaoui, M Krid, L Rais, S Beji, K Zouaghi, M Sellami-Kallel
Atypical hemolytic uremic syndrome (aHUS) is characterized by microvascular thrombosis resulting in thrombocytopenia, hemolytic anemia, and multiorgan dysfunction. It is associated with genetic or acquired disorders of regulatory components of the complement system. For our study, we collected data from 16 patients diagnosed with aHUS between January 2010 and January 2014. The mean age was 33.6 years. The female-to-male ratio was 3. The median follow-up duration was 27 ± 3.5 months. The most common clinical presentation was hypertension...
March 2018: Saudi Journal of Kidney Diseases and Transplantation
https://www.readbyqxmd.com/read/29655452/treatment-of-myasthenia-gravis
#5
REVIEW
Constantine Farmakidis, Mamatha Pasnoor, Mazen M Dimachkie, Richard J Barohn
With specialized care, patients with myasthenia gravis can have very good outcomes. The mainstays of treatment are acetylcholinesterase inhibitors, and immunosuppressive and immunomodulatory therapies. There is good evidence thymectomy is beneficial in thymomatous and nonthymomatous disease. Nearly all of the drugs used for MG are considered "off-label." The 2 exceptions are acetylcholinesterase inhibitors and complement inhibition with eculizumab, which was recently approved by the US Food and Drug Administration for myasthenia gravis...
May 2018: Neurologic Clinics
https://www.readbyqxmd.com/read/29649283/design-and-preclinical-characterization-of-alxn1210-a-novel-anti-c5-antibody-with-extended-duration-of-action
#6
Douglas Sheridan, Zhao-Xue Yu, Yuchun Zhang, Rekha Patel, Fang Sun, Melissa A Lasaro, Keith Bouchard, Bruce Andrien, Andre Marozsan, Yi Wang, Paul Tamburini
Eculizumab, a monoclonal antibody (mAb) directed against complement protein C5, is considered to be the current standard of care for patients with paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome. This study describes the generation and preclinical attributes of ALXN1210, a new long-acting anti-C5 mAb, obtained through select modifications to eculizumab to both largely abolish target-mediated drug disposition (TMDD) and increase recycling efficiency via the neonatal Fc receptor (FcRn)...
2018: PloS One
https://www.readbyqxmd.com/read/29616034/specific-inhibition-of-complement-activation-significantly-ameliorates-autoimmune-blistering-disease-in-mice
#7
Sidonia Mihai, Misa Hirose, Yi Wang, Joshua M Thurman, V Michael Holers, B Paul Morgan, Jörg Köhl, Detlef Zillikens, Ralf J Ludwig, Falk Nimmerjahn
Epidermolysis bullosa acquisita (EBA) is an antibody-mediated blistering skin disease associated with tissue-bound and circulating autoantibodies to type VII collagen (COL7). Transfer of antibodies against COL7 into mice results in a subepidermal blistering phenotype, strictly depending on the complement component C5. Further, activation predominantly by the alternative pathway is required to induce experimental EBA, as blistering was delayed and significantly ameliorated only in factor B-/- mice. However, C5 deficiency not only blocked the activation of terminal complement components and assembly of the membrane attack complex (MAC) but also eliminated the formation of C5a...
2018: Frontiers in Immunology
https://www.readbyqxmd.com/read/29610995/extra-renal-manifestations-of-atypical-hemolytic-uremic-syndrome-in-children
#8
Kibriya Fidan, Nilüfer Göknar, Bora Gülhan, Engin Melek, Zeynep Y Yıldırım, Esra Baskın, Mutlu Hayran, Kaan Gülleroglu, Zeynep B Özçakar, Fatih Ozaltin, Oguz Soylemezoglu
BACKGROUND: Atypical hemolytic uremic syndrome (aHUS) is a chronic disease characterized by thrombotic microangiopathy and a high risk of end-stage kidney disease. Dysregulation and/or excessive activation of the complement system results in thrombotic microangiopathy. Interest in extrarenal manifestations of aHUS is increasing. This study aimed to determine the clinical characteristics of patients with extrarenal manifestations of aHUS in childhood. METHODS: This study included 70 children with extrarenal manifestations of HUS from the national Turkish aHUS Registry...
April 2, 2018: Pediatric Nephrology: Journal of the International Pediatric Nephrology Association
https://www.readbyqxmd.com/read/29610176/pediatric-malignant-atrophic-papulosis
#9
Yung-Chieh Huang, Jiaan-Der Wang, Fang-Yi Lee, Lin-Shien Fu
Malignant atrophic papulosis (MAP), also known as Degos disease, is an extremely rare disease that is characterized by its unique skin presentation (namely, central, porcelain-white atrophic lesions with a telangiectatic rim). MAP has the following 2 variants: cutaneous MAP is manifested in the skin alone, whereas systemic MAP affects the gastrointestinal tract, central nervous system, lungs, and other internal organs. Some patients who presented with only cutaneous symptoms at first may develop systemic symptoms several years later...
April 2018: Pediatrics
https://www.readbyqxmd.com/read/29606012/adverse-outcomes-in-obstetric-atypical-haemolytic-uraemic-syndrome-a-case-series-analysis
#10
Natalia L Kozlovskaya, Yulia V Korotchaeva, Larisa A Bobrova
OBJECTIVE: The aim of this case series is to raise awareness of obstetric-related atypical haemolytic uraemic syndrome (aHUS) amongst obstetricians and gynaecologists. STUDY DESIGN: Data from 20 consecutive patients, aged 19-38, with obstetric-aHUS manifestation during or immediately after pregnancy are reported. Patients were diagnosed and treatment was initiated between 2012 and 2016. RESULTS: Presentation of aHUS was mainly preceded by preeclampsia and/or haemolysis, elevated liver enzymes and low platelet count syndrome, other obstetric complications, or by diarrhoea...
April 1, 2018: Journal of Maternal-fetal & Neonatal Medicine
https://www.readbyqxmd.com/read/29594148/c3-glomerulopathy-and-atypical-hemolytic-uremic-syndrome-two-important-manifestations-of-complement-system-dysfunction
#11
Ravneet Bajwa, John A DePalma, Taimoor Khan, Anmol Cheema, Sheila A Kalathil, Mohammad A Hossain, Attiya Haroon, Anne Madhurima, Min Zheng, Ali Nayer, Arif Asif
The advances in our understanding of the alternative pathway have emphasized that uncontrolled hyperactivity of this pathway causes 2 distinct disorders that adversely impact the kidney. In the so-called atypical hemolytic uremic syndrome (aHUS), renal dysfunction occurs along with thrombocytopenia, anemia, and target organ injury to multiple organs, most commonly the kidney. On the other hand, in the so-termed C3 glomerulopathy, kidney involvement is not associated with thrombocytopenia, anemia, or other system involvement...
January 2018: Case Reports in Nephrology and Dialysis
https://www.readbyqxmd.com/read/29582962/-atypical-hemolitic-uremic-syndrome-after-administration-of-docetaxel
#12
Alessandro Francesco Colombo, Rosalia Boito, Francesca Bova, Sebastiano Greco, Rosita Lucà, Arcangelo Sellaro, Giuseppe Rizzuto
The purpose of this study is to describe the clinical case of a patient suffering from a gland carcinoma with bilateral inguinal and pelvic lymph node metastases in treatment with weekly administrations of Docetaxel. After two therapy cycles, the patient developed an atypical uremic hemolytic Syndrome (SEUa), treated with infusions of frozen fresh plasma, hemodialysis, and antibiotics. Because of a severe septic secondary complication on an extensive lymph node abscess, the administration of Eculizumab was not possible...
March 2018: Giornale Italiano di Nefrologia: Organo Ufficiale Della Società Italiana di Nefrologia
https://www.readbyqxmd.com/read/29582550/consensus-opinion-on-diagnosis-and-management-of-thrombotic-microangiopathy-in-australia-and-new-zealand
#13
REVIEW
Lucy C Fox, Solomon J Cohney, Joshua Y Kausman, Jake Shortt, Peter D Hughes, Erica M Wood, Nicole M Isbel, Theo de Malmanche, Anne Durkan, Pravin Hissaria, Piers Blombery, Thomas D Barbour
Thrombotic microangiopathy (TMA) arises in a variety of clinical circumstances with the potential to cause significant dysfunction of the kidneys, brain, gastrointestinal tract and heart. TMA should be considered in all patients with thrombocytopenia and anaemia, with an immediate request to the haematology laboratory to look for red cell fragments on a blood film. Whilst TMA of any aetiology generally demands prompt treatment, this is especially so in thrombotic thrombocytopenic purpura (TTP) and atypical haemolytic uraemic syndrome (aHUS), where organ failure may be precipitous, irreversible and fatal...
March 27, 2018: Internal Medicine Journal
https://www.readbyqxmd.com/read/29578925/successful-management-of-a-rare-cause-of-hemolytic-uremic-syndrome-with-eculizumab-in-a-child
#14
Caner Alparslan, Önder Yavaşcan, Belde Kasap Demir, Bahriye Atmiş, Aysun Karabay Bayazit, Göksel Leblebisatan, Elif P Öncel, Demet Alaygut, Fatma Mutlubaş, Nejat Aksu
BACKGROUND: Hemolytic uremic syndrome (HUS) is characterized by microangiopathic hemolytic anemia, acute renal failure, and thrombocytopenia. It very rarely coexists with acute lymphoblastic leukemia (ALL) emerging before, simultaneously, or after the diagnosis has been made, and management of the patient may be difficult. CASE: We present the case of a 7-year-old boy who was diagnosed with HUS and initially managed by hemodialysis (HD). Thereafter, HUS progressed, and neurological findings developed...
March 23, 2018: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/29572749/eculizumab-treatment-in-severe-pediatric-stec-hus-a-multicenter-retrospective-study
#15
Lucas Percheron, Raluca Gramada, Stéphanie Tellier, Remi Salomon, Jérôme Harambat, Brigitte Llanas, Marc Fila, Emma Allain-Launay, Anne-Laure Lapeyraque, Valerie Leroy, Anne-Laure Adra, Etienne Bérard, Guylhène Bourdat-Michel, Hassid Chehade, Philippe Eckart, Elodie Merieau, Christine Piètrement, Anne-Laure Sellier-Leclerc, Véronique Frémeaux-Bacchi, Chloe Dimeglio, Arnaud Garnier
BACKGROUND: Hemolytic uremic syndrome related to Shiga-toxin-secreting Escherichia coli infection (STEC-HUS) remains a common cause of acute kidney injury in young children. No specific treatment has been validated for this severe disease. Recently, experimental studies highlight the potential role of complement in STEC-HUS pathophysiology. Eculizumab (EC), a monoclonal antibody against terminal complement complex, has been used in severe STEC-HUS patients, mostly during the 2011 German outbreak, with conflicting results...
March 23, 2018: Pediatric Nephrology: Journal of the International Pediatric Nephrology Association
https://www.readbyqxmd.com/read/29567368/efficacy-of-eculizumab-in-severe-adamts13-deficient-thrombotic-thrombocytopenic-purpura-ttp-refractory-to-standard-therapies
#16
Ernesto Vigna, Annamaria Petrungaro, Anna Perri, Dario Terzi, Anna Grazia Recchia, Francesco Mendicino, Antonella La Russa, Sabrina Bossio, Laura De Stefano, Francesco Zinno, Renzo Bonofiglio, Fortunato Morabito, Massimo Gentile
Thrombotic thrombocytopenic purpura (TTP) is a rare microangiopathic hemolytic anemia (MAHA) defined by mechanical hemolytic anemia, severe thrombocytopenia, and systemic visceral ischemia due to systemic platelet-rich microthrombi. Forty percent of patients with autoimmune TTP experience one or multiple relapses. Patients with refractory TTP are currently managed by corticosteroids, twice-daily PEX, and the anti-CD20 monoclonal antibody rituximab. Herein, we report two cases of severe TTP, refractory to those standard agents...
March 15, 2018: Transfusion and Apheresis Science
https://www.readbyqxmd.com/read/29563942/atypical-hemolytic-uremic-syndrome-and-eculizumab-therapy-in-children
#17
REVIEW
Seong Heon Kim, Hye Young Kim, Su Young Kim
Hemolytic uremic syndrome (HUS) is often encountered in children with acute kidney injury. Besides the well-known shiga toxin-producing Escherichia coli -associated HUS, atypical HUS (aHUS) caused by genetic complement dysregulation has been studied recently. aHUS is a rare, chronic, and devastating disorder that progressively damages systemic organs, resulting in stroke, end-stage renal disease, and death. The traditional treatment for aHUS is mainly plasmapheresis or plasma infusion; however, many children with aHUS will progress to chronic kidney disease despite plasma therapy...
February 2018: Korean Journal of Pediatrics
https://www.readbyqxmd.com/read/29558000/hemolytic-uremic-syndrome-with-dual-caution-in-an-infant-cobalamin-c-defect-and-complement-dysregulation-successfully-treated-with-eculizumab
#18
Ulkem Kocoglu Barlas, Hasan Serdar Kıhtır, Nilufer Goknar, Melike Ersoy, Nihal Akcay, Esra Sevketoglu
BACKGROUND: Hemolytic uremic syndrome (HUS) is a clinical syndrome characterized by hemolytic anemia, thrombocytopenia, and acute kidney injury. Atypical hemolytic uremic syndrome (aHUS) is a devastating disease with significant mortality and high risk of progression to end-stage kidney disease. It is mostly caused by dysregulation of the alternative complement pathway. Cobalamin C (Cbl C) defect is a genetic disorder of cobalamin metabolism and is a rare cause of HUS. CASE-DIAGNOSIS/TREATMENT: We present a 6-month-old male infant who was admitted to the pediatric intensive care unit (PICU) due to restlessness, severe hypertension, anemia, respiratory distress, and acute kidney injury...
March 20, 2018: Pediatric Nephrology: Journal of the International Pediatric Nephrology Association
https://www.readbyqxmd.com/read/29552364/clinical-relapses-of-atypical-hus-on-eculizumab-clinical-gap-for-monitoring-and-individualised-therapy
#19
Chia Wei Teoh, Kathleen Mary Gorman, Bryan Lynch, Timothy H J Goodship, Niamh Marie Dolan, Mary Waldron, Michael Riordan, Atif Awan
Atypical hemolytic uremic syndrome (aHUS) is caused by dysregulation of the complement system. A humanised anti-C5 monoclonal antibody (eculizumab) is available for the treatment of aHUS. We present the first description of atypical HUS in a child with a coexistent diagnosis of a POL-III leukodystrophy. On standard eculizumab dosing regime, there was evidence of ongoing C5 cleavage and clinical relapses when immunologically challenged. Eculizumab is an effective therapy for aHUS, but the recommended doses may not be adequate for all patients, highlighting the need for ongoing efforts to develop a strategy for monitoring of treatment efficacy and potential individualisation of therapy...
2018: Case Reports in Nephrology
https://www.readbyqxmd.com/read/29551496/cr1-gene-polymorphisms-in-chinese-patients-with-paroxysmal-nocturnal-hemoglobinuria
#20
Zhangbiao Long, Yali Du, Hongmin Li, Bing Han
Patients with paroxysmal nocturnal hemoglobinuria (PNH) who have minor allele of the complement receptor 1 (CR1) gene, displayed more sub-optimal responder to eculizumab compared with major allele. To investigate polymorphism of the CR1 gene in Chinese patients with PNH and its correlation with clinical features and the potential impact on eculizumab efficiency, we genotyped CR1 rs2274567, rs3811381 and the intron 27 Hind III restriction fragment length polymorphism in 95patients with PNH and 96 controls. The results indicated that the genotypes of CR1 rs2274567, rs3811381 and the intron 27 Hind III in PNH patients and controls both consist with Hardy-Weinberg equilibrium...
March 15, 2018: Gene
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