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Liver replacement therapy

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https://www.readbyqxmd.com/read/27906881/stem-cells-and-clinical-practice-new-advances-and-challenges-at-the-time-of-emerging-problems-with-induced-pluripotent-stem-cell-therapies
#1
Mariusz Z Ratajczak, Kamila Bujko, Wojciech Wojakowski
Humans, like other species that reproduce sexually, originate from a fertilized oocyte (zygote), which is a totipotent stem cell giving rise to an adult organism. During the process of embryogenesis, stem cells at different levels of the developmental hierarchy establish all 3 germ layers and give rise to tissue‑committed stem cells, which are responsible for rejuvenation of a given tissue or organ. The robustness of the stem cell compartment is one of the major factors that directly impact life quality as well as lifespan...
November 10, 2016: Polskie Archiwum Medycyny Wewnętrznej
https://www.readbyqxmd.com/read/27898091/restoring-ureagenesis-in-hepatocytes-by-crispr-cas9-mediated-genomic-addition-to-arginase-deficient-induced-pluripotent-stem-cells
#2
Patrick C Lee, Brian Truong, Agustin Vega-Crespo, W Blake Gilmore, Kip Hermann, Stephanie Ak Angarita, Jonathan K Tang, Katherine M Chang, Austin E Wininger, Alex K Lam, Benjamen E Schoenberg, Stephen D Cederbaum, April D Pyle, James A Byrne, Gerald S Lipshutz
Urea cycle disorders are incurable enzymopathies that affect nitrogen metabolism and typically lead to hyperammonemia. Arginase deficiency results from a mutation in Arg1, the enzyme regulating the final step of ureagenesis and typically results in developmental disabilities, seizures, spastic diplegia, and sometimes death. Current medical treatments for urea cycle disorders are only marginally effective, and for proximal disorders, liver transplantation is effective but limited by graft availability. Advances in human induced pluripotent stem cell research has allowed for the genetic modification of stem cells for potential cellular replacement therapies...
November 29, 2016: Molecular Therapy. Nucleic Acids
https://www.readbyqxmd.com/read/27890896/interventional-treatment-of-severe-portal-vein-thrombosis-after-living-donor-liver-transplantation
#3
Youichi Kawano, Satoru Murata, Nobuhiko Taniai, Masato Yoshioka, Atsushi Hirakata, Yoshiaki Mizuguchi, Tetsuya Shimizu, Tomohiro Kanda, Junji Ueda, Hideyuki Takada, Hiroshi Yoshida, Koho Akimaru, Shiro Onozawa, Shinichirou Kumita, Eiji Uchida
Portal vein thrombosis (PVT) is a rare complication of liver transplantation which can lead to graft failure and patient death. Treatment can be difficult, especially in cases of PVT from the intrahepatic portal vein to the proximal jejunal veins. A 55-year-old woman had undergone living-donor liver transplantation with splenectomy for end-stage liver cirrhosis due to hepatitis C with hepatocellular carcinoma. Ten months after transplantation, massive ascites and slight abdominal pain developed, and computed tomography revealed a PVT between the intrahepatic portal vein and the superior mesenteric vein...
2016: Journal of Nippon Medical School, Nippon Ika Daigaku Zasshi
https://www.readbyqxmd.com/read/27887981/predictors-of-mortality-in-the-critically-ill-cirrhotic-patient-is-the-model-for-end-stage-liver-disease-enough
#4
Alagappan Annamalai, Megan Y Harada, Melissa Chen, Tram Tran, Ara Ko, Eric J Ley, Miriam Nuno, Andrew Klein, Nicholas Nissen, Mazen Noureddin
BACKGROUND: Critically ill cirrhotics urgently require liver transplantation but are at high risk for perioperative mortality. The Model for End-Stage Liver Disease (MELD) score, recently updated to incorporate serum sodium, estimates survival probability in patients with cirrhosis but needs further evaluation in the critically ill. The purpose of this study was to evaluate the predictive power of intensive care unit (ICU) admission MELD scores and identify clinical risk factors associated with increased mortality...
November 22, 2016: Journal of the American College of Surgeons
https://www.readbyqxmd.com/read/27887644/copenhagen-comorbidity-in-hiv-infection-cocomo-study-a-study-protocol-for-a-longitudinal-non-interventional-assessment-of-non-aids-comorbidity-in-hiv-infection-in-denmark
#5
Andreas Ronit, Judith Haissman, Ditte Marie Kirkegaard-Klitbo, Thomas Skårup Kristensen, Anne-Mette Lebech, Thomas Benfield, Jan Gerstoft, Henrik Ullum, Lars Køber, Andreas Kjær, Klaus Kofoed, Jørgen Vestbo, Børge Nordestgaard, Jens Lundgren, Susanne Dam Nielsen
BACKGROUND: Modern combination antiretroviral therapy (cART) has improved survival for people living with HIV (PLWHIV). Non-AIDS comorbidities have replaced opportunistic infections as leading causes of mortality and morbidity, and are becoming a key health concern as this population continues to age. The aim of this study is to estimate the prevalence and incidence of non-AIDS comorbidity among PLWHIV in Denmark in the cART era and to determine risk factors contributing to the pathogenesis...
November 26, 2016: BMC Infectious Diseases
https://www.readbyqxmd.com/read/27881559/safety-and-tolerability-of-csl112-a-reconstituted-infusible-plasma-derived-apolipoprotein-a-i-after-acute-myocardial-infarction-the-aegis-i-trial-apoa-i-event-reducing-in-ischemic-syndromes-i
#6
C Michael Gibson, Serge Korjian, Pierluigi Tricoci, Yazan Daaboul, Megan Yee, Purva Jain, John H Alexander, P Gabriel Steg, A Michael Lincoff, John J P Kastelein, Roxana Mehran, Denise M D'Andrea, Lawrence I Deckelbaum, Bela Merkely, Maciej Zarebinski, Ton Oude Ophuis, Robert A Harrington
BACKGROUND: -Human or recombinant apolipoprotein A-I (apoA-I) has been shown to increase high-density lipoprotein-mediated cholesterol efflux capacity and to regress atherosclerotic disease in animal and clinical studies. CSL112 is an infusible, plasma-derived apoA-I that has been studied in normal subjects or those with stable coronary artery disease. This study aimed to characterize the safety, tolerability, pharmacokinetics, and pharmacodynamics of CSL112 in patients with a recent acute myocardial infarction...
November 15, 2016: Circulation
https://www.readbyqxmd.com/read/27878737/sebelipase-alfa-a-review-in-lysosomal-acid-lipase-deficiency
#7
James E Frampton
Sebelipase alfa (Kanuma(®), Kanuma™), the first commercially available recombinant human lysosomal acid lipase (LAL), is approved in various countries worldwide, including those of the EU, the USA and Japan, as a long-term enzyme replacement therapy for patients diagnosed with LAL deficiency (LAL-D), an ultra-rare, autosomal recessive, progressive metabolic liver disease. In an ongoing study in nine infants presenting with early-onset LAL-D (Wolman disease), open-label treatment with sebelipase alfa significantly improved 1-year survival compared with historical controls...
November 23, 2016: American Journal of Cardiovascular Drugs: Drugs, Devices, and Other Interventions
https://www.readbyqxmd.com/read/27876313/lysosomal-acid-lipase-deficiency-expanding-differential-diagnosis
#8
Vassili Valayannopoulos, Eugen Mengel, Anais Brassier, Gregory Grabowski
The differential diagnoses for metabolic liver diseases may be challenging in clinical settings, which represents a critical issue for disorders such as lysosomal acid lipase deficiency (LAL-D). LAL-D is caused by deficient activity of the LAL enzyme, resulting in the accumulation of cholesteryl esters and triglycerides throughout the body, predominately in the liver, spleen, gastrointestinal tract, and blood vessel walls. LAL-D is a progressive, multi-organ disease with early mortality and significant morbidity characterized by a combination of hepatic dysfunction and dyslipidemia...
November 10, 2016: Molecular Genetics and Metabolism
https://www.readbyqxmd.com/read/27875571/short-and-long-term-mortality-rates-of-elderly-acute-kidney-injury-patients-who-underwent-continuous-renal-replacement-therapy
#9
Harin Rhee, Keum Sook Jang, Jong Man Park, Jin Suk Kang, Na Kyoung Hwang, Il Young Kim, Sang Heon Song, Eun Young Seong, Dong Won Lee, Soo Bong Lee, Ihm Soo Kwak
BACKGROUND: The world's population is aging faster and the incidence of acute kidney injury (AKI) needing continuous renal replacement therapy (CRRT) is increasing in elderly population. The outcome of AKI needing CRRT in elderly patients is known to be poor. However, the definitions of elderly used in the previous literatures were diverse and, there were few data that compared the long-term mortality rates of these patients with middle aged patients. This study was aimed to evaluate this issue...
2016: PloS One
https://www.readbyqxmd.com/read/27875032/management-of-renal-failure-in-end-stage-liver-disease-a-critical-appraisal
#10
REVIEW
Xingxing S Cheng, Jane C Tan, W Ray Kim
Renal failure is a late consequence of end-stage liver disease (ESLD). Even with liver transplantation, pretransplant renal impairment remains a strong predictor of posttransplant mortality. This review seeks to summarize and critically appraise common therapies used in this setting, including pharmacologic agents, procedures (transjugular intrahepatic portosystemic shunt, renal replacement therapy), and simultaneous liver-kidney transplantation. More experimental extracorporal modalities, eg, albumin dialysis or bioartificial livers, will not be discussed...
December 2016: Liver Transplantation
https://www.readbyqxmd.com/read/27866810/histological-characterisation-of-visceral-changes-in-a-patient-with-type-2-gaucher-disease-treated-with-enzyme-replacement-therapy
#11
Yuko Tezuka, Mitsumasa Fukuda, Shohei Watanabe, Takeshi Nakano, Kentaro Okamoto, Kazuyo Kuzume, Yoshiaki Yano, Mariko Eguchi, Minenori Ishimae, Eiichi Ishii, Tatsuhiko Miyazaki
Gaucher disease is a lysosomal storage disease caused by deficiency of glucocerebrosidase and accumulation of glucocerebroside. Three major sub-types have been described, type 2 is an acute neurological form that exhibits serious general symptoms and poor prognosis, compared with the other types. This case was a girl diagnosed with type 2 Gaucher disease at 12months of age who presented with poor weight gain from infancy, stridor, hypertonia, hepatosplenomegaly, trismus and an eye movement disorder. Enzyme replacement therapy (ERT) was administered, but she had frequent myoclonus and developmental regression...
November 12, 2016: Blood Cells, Molecules & Diseases
https://www.readbyqxmd.com/read/27861428/hepatogenic-differentiation-of-human-induced-pluripotent-stem-cells-on-collagen-coated-polyethersulfone-nanofibers
#12
Maryam Mahmoodinia Maymand, Hamid Reza Soleimanpour-Lichaei, Abdolreza Ardeshirylajimi, Masoud Soleimani, Samaneh Mirzaei, Athena Hajarizadeh, Maryam Kabir Salmani
Many scientists have been fascinated with Induced pluripotent stem cells (iPSCs) for cell replacement therapies. Nanofibrous biocompatible scaffolds have been shown to promote better cell adhesion and improve stem cell differentiation. In the present study, after fabrication using electrospinning technique and surface modifications, the characteristics of Polyethersulfone (PES) nanofibers were determined by scanning electron microscopy (SEM), ATR-FTIR and MTT assay. Then, the hepatogenic potential of iPSCs was evaluated using Real-Time RT-PCR and immunocytochemistry (ICC) after culture on collagen coated polyethersulfone (PES/COL) scaffolds...
November 15, 2016: ASAIO Journal: a Peer-reviewed Journal of the American Society for Artificial Internal Organs
https://www.readbyqxmd.com/read/27836529/twelve-years-of-experience-with-miglustat-in-the-treatment-of-type-1-gaucher-disease-the-spanish-zagal-project
#13
Pilar Giraldo, Marcio Andrade-Campos, Pilar Alfonso, Pilar Irun, Koldo Atutxa, Antonio Acedo, Abelardo Barez, Margarita Blanes, Vicente Diaz-Morant, Ma Angeles Fernández-Galán, Rafael Franco, Cristina Gil-Cortes, Vicente Giner, Angela Ibañez, Paz Latre, Ines Loyola, Elisa Luño, Roberto Hernández-Martin, Blanca Medrano-Engay, José Puerta, Inmaculada Roig, Javier de la Serna, Olga Salamero, Lucia Villalón, Miguel Pocovi
We report data from a prospective, observational study (ZAGAL) evaluating miglustat 100mg three times daily orally. in treatment-naïve patients and patients with type 1 Gaucher Disease (GD1) switched from previous enzyme replacement therapy (ERT). Clinical evolution, changes in organ size, blood counts, disease biomarkers, bone marrow infiltration (S-MRI), bone mineral density by broadband ultrasound densitometry (BMD), safety and tolerability annual reports were analysed. Between May 2004 and April 2016, 63 patients received miglustat therapy; 20 (32%) untreated and 43 (68%) switched...
October 24, 2016: Blood Cells, Molecules & Diseases
https://www.readbyqxmd.com/read/27833043/efficient-generation-of-pancreatic-%C3%AE-like-cells-from-the-mouse-gallbladder
#14
Yuhan Wang, Feorillo Galivo, Carl Pelz, Annelise Haft, Jonghyeob Lee, Seung K Kim, Markus Grompe
Direct reprogramming is a promising approach for the replacement of β cells in diabetes. Reprogramming of cells originating from the endodermal lineage, such as acinar cells in the pancreas, liver cells and gallbladder cells has been of particular interest because of their developmental proximity to β cells. Our previous work showed that mouse gallbladder epithelium can be partially reprogrammed in vitro to generate islet-like cells (rGBC1). Here, the reprogramming protocol was substantially improved, yielding cells (rGBC2) closer to functional β cells than the 1st generation method with higher conversion efficiency and insulin expression...
October 27, 2016: Stem Cell Research
https://www.readbyqxmd.com/read/27830927/observational-clinical-study-on-organ-dysfunction-associated-with-dosing-of-antibiotics-in-a-paediatric-intensive-care-unit
#15
Sascha Tafelski, Alexander Gratopp, Florian Richter, Sylvia Kramer, Claudia Spies, Klaus D Wernecke, Irit Nachtigall
BACKGROUND: Medication errors are of concern especially in paediatric patients. This study investigates impact of dosing errors of antibiotics on outcome in critically ill paediatric patients. METHODS: Retrospective study including all consecutive patients admitted to one university paediatric intensive care unit (PICU) in 2010 with length of PICU stay >24hrs, age <18 years and antibiotic therapy. Antibiotic dosages were evaluated for compliance with recommended dosing individually adapted for bodyweight, age and organ function...
November 10, 2016: Minerva Pediatrica
https://www.readbyqxmd.com/read/27830550/gunn-rats-as-a-surrogate-model-for-evaluation-of-hepatocyte-transplantation-based-therapies-of-crigler-najjar-syndrome-type-1
#16
Zsuzsanna Polgar, Yanfeng Li, Xia Li Wang, Chandan Guha, Namita Roy-Chowdhury, Jayanta Roy-Chowdhury
Liver transplantation has been established as a curative therapy for acute and chronic liver failure, as well as liver-based inherited metabolic diseases. Because of the complexity of organ transplantation and the worldwide shortage of donor organs, hepatocyte transplantation is being developed as a bridging therapy until donor organs become available, or for amelioration of inherited liver-based diseases. The Gunn rat is a molecular and metabolic model of Crigler-Najjar syndrome type 1, which is characterized by lifelong unconjugated hyperbilirubinemia due to the lack of uridinediphosphoglucuronate glucuronosyltransferase-1 (UGT1A1)-mediated bilirubin glucuronidation...
2017: Methods in Molecular Biology
https://www.readbyqxmd.com/read/27830548/fetal-liver-stem-progenitor-cell-transplantation-a-model-to-study-tissue-mass-replacement-and-cell-based-therapies
#17
Mladen I Yovchev, Michael Oertel
Liver transplantation is the only therapeutic treatment for patients with end-stage liver diseases. However, donor organ scarcity is the major limitation, and therefore, alternative strategies are urgently needed. The ultimate goal for successful cell-based therapies is the ability of transplanted cells to efficiently engraft and reconstitute injured liver mass. To evaluate the repopulation capacity of transplanted cells, it is essential to identify their specific characteristics, as well as to study the mechanism(s) Through which transplanted donor cells replace tissue mass in hepatic microenvironments, using well-established cell transplantation models...
2017: Methods in Molecular Biology
https://www.readbyqxmd.com/read/27827549/advances-in-identifying-urine-serum-biomarkers-in-alpha-1-antitrypsin-deficiency-for-more-personalized-future-treatment-strategies
#18
Ilaria Ferrarotti, Angelo Guido Corsico, Jan Stolk, Stefania Ottaviani, Marco Fumagalli, Sabina Janciauskiene, Paolo Iadarola
Alpha1-antitrypsin deficiency (AATD) is a genetic disorder characterized by reduced serum levels of alpha1-antitrypsin (AAT) and increased risk for developing both early-onset lung emphysema and chronic liver disease. Laboratory diagnosis of AATD is not just a matter of degree, although the AAT serum level is the most important determinant for risk of lung damage. While being a single-gene disease, the clinical phenotype of AATD is heterogeneous. The current standard of care for patients affected by AATD-associated pulmonary emphysema is replacement therapy with weekly i...
November 9, 2016: COPD
https://www.readbyqxmd.com/read/27826603/liberation-from-renal-replacement-therapy-after-cadaveric-liver-transplantation
#19
Ibtesam A Hilmi, Ali Al-Khafaji, Daniela Damian, Bedda L Rosario-Rivera, Ali Abdullah, John A Kellum
BACKGROUND: Renal failure requiring renal replacement therapy (RRT) is common in patients with end-stage liver disease (ESLD) and is associated with worse outcomes following liver transplantation (LT). We investigated the factors associated with liberation from posttransplant RRT and studied the impact of RRT on patient and graft outcomes. METHODS: A 5-year retrospective study of ESLD patients who received pretransplant RRT was conducted. Variables associated with liberation from RRT at 30 days and at 1-year posttransplant were analyzed...
November 2016: Transplantation Direct
https://www.readbyqxmd.com/read/27818959/amlodipine-induced-gingival-overgrowth-in-a-child-after-liver-transplant
#20
André Guollo, Ana Paula Molina Vivas, Rodrigo Nascimento Lopes, Gilda Porta, Fábio Abreu Alves
Drug-induced gingival overgrowth (GO) has been associated with phenytoin, cyclosporine, and calcium channel blocker therapies. This study reports the case of an 11-year-old girl who was referred for evaluation of GO, which had occurred over the last 6 months. Her medical history included a liver transplant due to biliary atresia 3 years ago, immunosuppressive therapy, and hypertension, which is why she was started on a daily intake of amlodipine. The intraoral examination showed generalized GO, and the treatment consisted of a gingivectomy...
July 2016: Autopsy & case reports
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