keyword
https://read.qxmd.com/read/38661088/ravulizumab-facilitates-reduced-burden-of-vascular-access-a-major-benefit-in-paediatric-atypical-haemolytic-uraemic-syndrome
#1
JOURNAL ARTICLE
Freya Bleathman, Joshua Y Kausman, Laine M Hosking, Thomas A Forbes
BACKGROUND: Atypical haemolytic uraemic syndrome (aHUS) is a rare thrombotic microangiopathy resulting from dysregulation of the alternative complement pathway, leading to multi-organ dysfunction and chronic kidney disease. Eculizumab is an anti-C5 monoclonal antibody therapy that has significantly improved aHUS disease control and patient outcomes, however it requires fortnightly intravenous dosing. This often necessitates long term central access and a high hospital attendance burden...
April 25, 2024: Journal of Paediatrics and Child Health
https://read.qxmd.com/read/38658194/carfilzomib-associated-thrombotic-microangiopathy-clinical-features-and-outcomes
#2
JOURNAL ARTICLE
Adrien Joseph, Stéphanie Harel, Laurent Mesnard, Cédric Rafat, Silène Knapp, Anne Rumpler, Carole Philipponnet, Christophe Barba, Jean-Michel Rebibou, David Buob, Alexandre Hertig, Jacques Vargaftig, Jean-Michel Halimi, Bertrand Arnulf, Anne-Sophie Bretaud, Bérangère Joly, Steven Grangé, Paul Coppo
BACKGROUND AND HYPOTHESIS: Carfilzomib, a new proteasome inhibitor indicated for patients with relapsed/refractory myeloma, has been associated with cases of thrombotic microangiopathy (CFZ-TMA). The role of variants in the complement alternative pathway and therapeutic potential of complement blockade with eculizumab remain to be determined. METHODS: We report 37 cases of CFZ-TMA recorded in the French reference center for TMA with their clinical characteristics, genetic analysis and outcome according to treatments...
April 24, 2024: Nephrology, Dialysis, Transplantation
https://read.qxmd.com/read/38646924/novel-factors-potentially-initiating-acute-antibody-mediated-rejection-in-pig-kidney-xenografts-despite-an-efficient-immunosuppressive-regimen
#3
JOURNAL ARTICLE
Kohei Kinoshita, Akihiro Maenaka, Ivy A Rosales, Ahmad Karadagi, Toshihide Tomosugi, David Ayares, Seth Lederman, Robert B Colvin, Tatsuo Kawai, Richard N Pierson, Takaaki Kobayashi, David K C Cooper
Antibody-mediated rejection (AMR) is a common cause of graft failure after pig-to-nonhuman primate organ transplantation, even when the graft is from a pig with multiple genetic modifications. The specific factors that initiate AMR are often uncertain. We report two cases of pig kidney transplantation into immunosuppressed baboons in which we identify novel factors associated with the initiation of AMR. In the first, membranous nephropathy was the initiating factor that was then associated with the apparent loss of the therapeutic anti-CD154 monoclonal antibody in the urine when severe proteinuria was present...
2024: Xenotransplantation
https://read.qxmd.com/read/38637498/antiviral-cellular-therapy-for-enhancing-t-cell-reconstitution-before-or-after-hematopoietic-stem-cell-transplantation-aces-a-two-arm-open-label-phase-ii-interventional-trial-of-pediatric-patients-with-risk-factor-assessment
#4
MULTICENTER STUDY
Michael D Keller, Patrick J Hanley, Yueh-Yun Chi, Paibel Aguayo-Hiraldo, Christopher C Dvorak, Michael R Verneris, Donald B Kohn, Sung-Yun Pai, Blachy J Dávila Saldaña, Benjamin Hanisch, Troy C Quigg, Roberta H Adams, Ann Dahlberg, Shanmuganathan Chandrakasan, Hasibul Hasan, Jemily Malvar, Mariah A Jensen-Wachspress, Christopher A Lazarski, Gelina Sani, John M Idso, Haili Lang, Pamela Chansky, Chase D McCann, Jay Tanna, Allistair A Abraham, Jennifer L Webb, Abeer Shibli, Amy K Keating, Prakash Satwani, Pawel Muranski, Erin Hall, Michael J Eckrich, Evan Shereck, Holly Miller, Ewelina Mamcarz, Rajni Agarwal, Satiro N De Oliveira, Mark T Vander Lugt, Christen L Ebens, Victor M Aquino, Jeffrey J Bednarski, Julia Chu, Suhag Parikh, Jennifer Whangbo, Michail Lionakis, Elias T Zambidis, Elizabeth Gourdine, Catherine M Bollard, Michael A Pulsipher
Viral infections remain a major risk in immunocompromised pediatric patients, and virus-specific T cell (VST) therapy has been successful for treatment of refractory viral infections in prior studies. We performed a phase II multicenter study (NCT03475212) for the treatment of pediatric patients with inborn errors of immunity and/or post allogeneic hematopoietic stem cell transplant with refractory viral infections using partially-HLA matched VSTs targeting cytomegalovirus, Epstein-Barr virus, or adenovirus...
April 18, 2024: Nature Communications
https://read.qxmd.com/read/38623882/successful-use-of-eculizumab-in-immediate-anca-vasculitis-recurrence-in-a-pediatric-kidney-transplant
#5
Caitlin G Peterson, Rachel L Jackson
BACKGROUND: Kidney transplantation is an acceptable therapy end-stage kidney disease secondary to antineutrophil cytoplasmic antibody-associated vasculitis with risk of disease recurrence ranging from 3% to 17%. Standard posttransplant immunosuppression is the mainstay of therapy after recurrence. Recently, new medications focused on complement regulation and avoidance of steroids have been shown to be effective in treating antineutrophil cytoplasmic antibody (ANCA) vasculitis with no studies in the pediatric population...
May 2024: Pediatric Transplantation
https://read.qxmd.com/read/38536978/bilateral-retinal-venous-occlusion-in-atypical-hemolytic-uremic-syndrome-due-to-complement-factor-h-mutation
#6
JOURNAL ARTICLE
Saban Gonul, Serhat Eker
PURPOSE: Atypical hemolytic uremic syndrome (aHUS) is a rare progressive thrombotic microangiopathy caused by overactivation in the alternative complement pathway. A wide spectrum of environmental triggers, such as viruses, vaccination, drugs, pregnancy, neoplasms, transplant, and autoimmune diseases can cause aHUS in genetically susceptible individuals. In this report, the diagnosis and treatment process of aHUS and bilateral retinal venous occlusion (RVO) will be presented. METHODS: Single-case, retrospective management of ophthalmological and systemic manifestations...
March 27, 2024: Ocular Immunology and Inflammation
https://read.qxmd.com/read/38523374/management-of-pediatric-hemolytic-uremic-syndrome
#7
REVIEW
Bora Gülhan, Fatih Özaltın, Kibriya Fidan, Zeynep Birsin Özçakar, Oğuz Söylemezoğlu
Classical clinical triad of hemolytic uremic syndrome (HUS) is microangiopathic hemolytic anemia, thrombocytopenia, and acute kidney injury associated with endothelial cell injury. Several situations, including infections, medications, malignancies, and transplantation can trigger endothelial damage. On the HUS spectrum, atypical hemolytic uremic syndrome (aHUS) deserves special attention in pediatric patients, as it can cause endstage kidney disease and mortality. A dysfunction in the alternative complement pathway, either acquired or genetic, has been shown to be the main underlying cause...
2024: Turkish Journal of Pediatrics
https://read.qxmd.com/read/38521410/use-of-eculizumab-in-pediatric-patients-with-high-risk-transplant-associated-thrombotic-microangiopathy-outcomes-and-risk-factors-associated-with-response-and-survival-a-retrospective-study-on-behalf-of-the-spanish-group-for-hematopoietic-transplantation-and
#8
JOURNAL ARTICLE
M I Benítez-Carabante, D Bueno, L Alonso García, I López Torija, J Marsal, Fernandez NavarroJM, M L Uria Oficialdegui, M Panesso, B Molina, C Beléndez Bieler, P Palomo, A Pérez Martínez, C Diaz-de-Heredia
INTRODUCTION: Transplant-associated thrombotic microangiopathy (TA-TMA) is associated with high morbidity and mortality. Although with the introduction of eculizumab the survival has significantly improved, there is still a need for improvement, especially in high-risk patients. OBJECTIVES: This study aims to describe the results obtained with eculizumab in a pediatric cohort with the attempt to define which risk factors could determine the response to treatment...
March 21, 2024: Transplantation and cellular therapy
https://read.qxmd.com/read/38503569/eculizumab-for-pregnancy-related-atypical-hemolytic-uremic-syndrome
#9
JOURNAL ARTICLE
Yulia Korotchaeva, Natalia Kozlovskaya, Efim Shifman, Dmitry Kudlay, Sergey Moiseev
No abstract text is available yet for this article.
March 19, 2024: Nephrology, Dialysis, Transplantation
https://read.qxmd.com/read/38485664/the-role-of-anticomplement-therapy-in-the-management-of-the-kidney-allograft
#10
REVIEW
Mehmet Kanbay, Sidar Copur, Zeynep Y Yilmaz, Dilek Ertoy Baydar, Ilmay Bilge, Caner Susal, Burak Kocak, Alberto Ortiz
As the number of patients living with kidney failure grows, the need also grows for kidney transplantation, the gold standard kidney replacement therapy that provides a survival advantage. This may result in an increased rate of transplantation from HLA-mismatched donors that increases the rate of antibody-mediated rejection (AMR), which already is the leading cause of allograft failure. Plasmapheresis, intravenous immunoglobulin therapy, anti-CD20 therapies (i.e., rituximab), bortezomib and splenectomy have been used over the years to treat AMR as well as to prevent AMR in high-risk sensitized kidney transplant recipients...
March 2024: Clinical Transplantation
https://read.qxmd.com/read/38412598/recurrent-c3-glomerulopathy-after-kidney-transplantation
#11
REVIEW
Shota Obata, Pedro A S Vaz de Castro, Leonardo V Riella, Paolo Cravedi
The complement system is part of innate immunity and is pivotal in protecting the body against pathogens and maintaining host homeostasis. Activation of the complement system is triggered through multiple pathways, including antibody deposition, a mannan-binding lectin, or activated complement deposition. C3 glomerulopathy (C3G) is a rare glomerular disease driven by complement dysregulation with high post-transplantation recurrence rates. Its treatment is mainly based on immunosuppressive therapies, specifically mycophenolate mofetil and glucocorticoids...
April 2024: Transplantation Reviews
https://read.qxmd.com/read/38312782/ex%C3%A2-vivo-test-of-complement-dysregulation-in-atypical-hemolytic-uremic-syndrome-kidney-transplant-patients-a-pilot-study
#12
JOURNAL ARTICLE
Caroline Duineveld, Romy N Bouwmeester, Lambertus P W J van den Heuvel, Nicole C A J van de Kar, Jack F M Wetzels
INTRODUCTION: In 2014, a complement assay, which evaluates C5b-9 deposition on endothelial cells, was proposed as a biomarker for atypical hemolytic uremic syndrome (aHUS). Early diagnosis and/or prediction of aHUS (relapse) is pivotal in aHUS kidney transplant recipients who do not receive eculizumab prophylaxis. METHODS: In this pilot study, serum samples of transplanted patients with aHUS in remission without eculizumab and patients with other primary kidney diseases (controls) were blinded and evaluated in the complement assay...
January 2024: KI Reports
https://read.qxmd.com/read/38280096/outcome-of-atypical-hemolytic-uremic-syndrome-role-of-triggers-and-complement-abnormalities-in-the-response-to-c5-inhibition
#13
JOURNAL ARTICLE
Gianluigi Ardissino, Donata Cresseri, Maria Cristina Mancuso, Valentina Capone, Luigi Porcaro, Valeria Amico, Marianna Tangredi, Elena Grovetti, Samantha Griffini, Giuseppe Castellano, Giovanni Montini, Dario Consonni, Massimo Cugno
BACKGROUND: Atypical-hemolytic uremic syndrome (aHUS) is a rare thrombotic microangiopathy often due to uncontrolled complement activation, characterized by high risk of end-stage kidney disease (ESKD). Eculizumab has improved the outcome, however, its efficacy varies among patients and its discontinuation is debated. METHODS: To identify characteristics associated with treatment response, we analyzed 244 aHUS patients referred to our center. Patients were classified according to the presence/absence of complement abnormalities and/or triggers at onset in 4 categories: (1) primary (complement abnormality without trigger), (2) secondary (trigger without complement abnormality), (3) combined (trigger and complement abnormality), (4) idiopathic (no trigger, no complement abnormality)...
January 27, 2024: Journal of Nephrology
https://read.qxmd.com/read/38269581/c5-inhibition-with-eculizumab-prevents-thrombotic-microangiopathy-in-a-case-series-of-pig-to-human-kidney-xenotransplantation
#14
JOURNAL ARTICLE
Maggie E Jones-Carr, Huma Fatima, Vineeta Kumar, Douglas J Anderson, Julie Houp, Jackson C Perry, Gavin A Baker, Leigh McManus, Andrew J Shunk, Paige M Porrett, Jayme E Locke
No abstract text is available yet for this article.
January 25, 2024: Journal of Clinical Investigation
https://read.qxmd.com/read/38222133/is-salvage-of-a-kidney-graft-possible-as-a-result-of-hyperacute-rejection-immediately-after-kidney-transplantation
#15
Irshad Hussain, Salma Mahmoud, Scott Schurman, Reut Hod Dvorai, Rauf Shahbazov
Hyperacute rejection is a rare complication of renal transplantation. It is mainly caused by preformed human leukocyte antigen antibodies and can lead to the loss of the transplanted kidney. Renal transplantation is a highly beneficial treatment for people with end-stage renal disease, greatly improving their quality of life. However, antibody-mediated rejection is a significant challenge for the long-term survival of transplanted kidneys. An 18-year-old male with nephrotic syndrome, who underwent bilateral renal nephrectomy due to severe proteinuria, received a living donor kidney...
December 2023: Curēus
https://read.qxmd.com/read/38164997/d-dimer-and-sinusoidal-obstructive-syndrome-novel-poor-prognostic-features-of-thrombotic-microangiopathy-in-children-after-hematopoietic-cellular-therapy-in-a-single-institution-prospective-cohort-study
#16
JOURNAL ARTICLE
Michelle L Schoettler, Kaley French, Anora Harris, Elyse Bryson, Laura Deeb, Zuri Hudson, Jeremy Obordo, Shanmuganathan Chandrakasan, Suhag Parikh, Benjamin Watkins, Elizabeth Stenger, Muna Qayed, Satheesh Chonat, Adrianna Westbrook, Jeffrey Switchenko, Kirsten M Williams
Transplant-associated thrombotic microangiopathy (TA-TMA) is a common, severe complication of allogeneic hematopoietic cellular therapy (HCT). Even when treated in many studies, morbidity and mortality rates are high. This prospective single-institution cohort study serially enrolled all allogeneic HCT recipients from August 2019-August 2022. Patients were universally screened for TA-TMA and intermediate and high-risk patients were immediately treated with eculizumab. Sub-distribution cox-proportional hazards models were used to identify sub-distribution hazard ratios (sHR)  for multi-organ dysfunction (MOD) and non-relapse-related mortality (NRM)...
January 2, 2024: American Journal of Hematology
https://read.qxmd.com/read/38154068/pediatric-transplant-associated-thrombotic-microangiopathy-healthcare-utilization-and-implications-of-eculizumab-therapy
#17
JOURNAL ARTICLE
Michelle L Schoettler, Leslie Elaine Lehmann, Pei-Chi Kao, Nan Chen, Sonata Jodele, Satheesh Chonat, Kirsten M Williams, Wendy B London, Christine Duncan, Christopher E Dandoy
The healthcare utilization (HCU) burden of transplant associated thrombotic microangiopathy (TA-TMA) and its treatments are unknown. The objective of this study was to investigate inpatient costs associated with meeting criteria for transplant associated thrombotic microangiopathy (TA-TMA) in the first-year post-HCT. This IRB-approved retrospective multicenter study included serial children who underwent HCT from 1/1/2015 to 7/1/2019. A standardized unit cost (adjusted for geographic location, differences in cost of living, and inflation) for inpatient hospitalization was extracted from the Pediatric Health Information System data and linked to clinical data...
December 28, 2023: Blood Advances
https://read.qxmd.com/read/38146570/beyond-immunosuppression-the-intricate-relationship-between-tacrolimus-and-microangiopathy
#18
REVIEW
Ripudaman S Munjal, Jagdish Sharma, Srinija Polishetti, Pushkar Sai Valleru, Himanshi Banker, Ramansh Bandhu Gupta, Fnu Anamika, Rohit Jain
Tacrolimus, widely known as Prograf, has become the preferred immunosuppressant for preventing graft rejection in solid organ transplant recipients, particularly in steroid-sparing regimens. Its efficacy and reduced risk of acute and chronic rejection compared to cyclosporine have made it the preferred treatment option for transplant patients. However, tacrolimus has drawbacks as it is associated with adverse effects, such as renal tubular necrosis, kidney failure, hypertension, metabolic acidosis, and new-onset diabetes mellitus...
November 2023: Curēus
https://read.qxmd.com/read/38145741/compassionate-use-narsoplimab-for-severe-refractory-transplant-associated-thrombotic-microangiopathy-in-children
#19
JOURNAL ARTICLE
Michelle L Schoettler, Seema Patel, Elyse Bryson, Laura Deeb, Benjamin Watkins, Muna Qayed, Shanmuganathan Chandrakasan, Taylor Fitch, Katherine Silvis, Jayre Jones, Satheesh Chonat, Kirsten M Williams
BACKGROUND: Transplant-associated thrombotic microangiopathy (TA-TMA) is a common and potentially severe complication of hematopoietic cell transplantation. TA-TMA directed therapy with eculizumab, a C5 inhibitor, has resulted in a survival benefit in some studies. However, children with TA-TMA refractory to complement C5 inhibition with eculizumab (rTA-TMA) have mortality rates exceeding 80%, and there are no other known therapies. Narsoplimab, an inhibitor of the effector enzyme of the lectin pathway, MASP-2, has been studied in adults with TA-TMA as first line therapy with a response rate of 61%...
December 23, 2023: Transplantation and cellular therapy
https://read.qxmd.com/read/38102212/eculizumab-treatment-in-paediatric-patients-diagnosed-with-ahus-after-haematopoietic-stem-cell-transplantation-a-hsct-tma-case-series-from-japanese-ahus-post-marketing-surveillance
#20
JOURNAL ARTICLE
Shuichi Ito, Atsuro Saito, Ayako Sakurai, Kenichiro Watanabe, Shuhei Karakawa, Takako Miyamura, Tomoko Yokosuka, Hideaki Ueki, Hiroaki Goto, Hiroshi Yagasaki, Mariko Kinoshita, Michio Ozeki, Norifumi Yokoyama, Hirofumi Teranishi
Haematopoietic stem-cell transplantation (HSCT)-associated thrombotic microangiopathy (HSCT-TMA) is a serious complication with high mortality. Accumulating evidence suggests that complement dysregulation is potentially involved in the development of HSCT-TMA. We retrospectively analysed the clinical characteristics and outcomes of thirteen paediatric patients who were diagnosed with atypical haemolytic uremic syndrome and treated with eculizumab to manage HSCT-TMA during post-marketing surveillance in Japan...
March 2024: Bone Marrow Transplantation
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