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LAL treatment in adults

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https://www.readbyqxmd.com/read/29628368/sebelipase-alfa-improves-atherogenic-biomarkers-in-adults-and-children-with-lysosomal-acid-lipase-deficiency
#1
Don P Wilson, Mark Friedman, Sachin Marulkar, Tyler Hamby, Eric Bruckert
BACKGROUND: Measures of atherogenic cholesterol, with and without concomitant use of lipid-lowering medications (LLMs), are reported with up to 52 weeks of sebelipase alfa treatment in children and adults with lysosomal acid lipase deficiency (LAL-D) participating in the phase 3 Acid Lipase Replacement Investigating Safety and Efficacy study (NCT01757184). OBJECTIVE: To examine the effects of sebelipase alfa on levels of atherogenic biomarkers in the Acid Lipase Replacement Investigating Safety and Efficacy study...
March 9, 2018: Journal of Clinical Lipidology
https://www.readbyqxmd.com/read/29470730/sacral-nerve-stimulation-versus-percutaneous-tibial-nerve-stimulation-for-faecal-incontinence-a-systematic-review-and-meta-analysis
#2
Constantinos Simillis, Nikhil Lal, Shengyang Qiu, Christos Kontovounisios, Shahnawaz Rasheed, Emile Tan, Paris P Tekkis
AIMS: Percutaneous tibial nerve stimulation (PTNS) and sacral nerve stimulation (SNS) are both second-line treatments for faecal incontinence (FI). To compare the clinical outcomes and effectiveness of SNS versus PTNS for treating FI in adults. METHOD: A literature search of MEDLINE, Embase, Science Citation Index Expanded and Cochrane was performed in order to identify studies comparing SNS and PTNS for treating FI. A risk of bias assessment was performed using The Cochrane Collaboration's risk of bias tool...
May 2018: International Journal of Colorectal Disease
https://www.readbyqxmd.com/read/29424786/no-neurocognitive-advantage-for-immediate-antiretroviral-treatment-in-adults-with-greater-than-500-cd4-t-cell-counts
#3
Edwina J Wright, Birgit Grund, Kevin R Robertson, Lucette Cysique, Bruce J Brew, Gary L Collins, Mollie Poehlman-Roediger, Michael J Vjecha, Augusto César Penalva de Oliveira, Barbara Standridge, Cate Carey, Anchalee Avihingsanon, Eric Florence, Jens D Lundgren, Alejandro Arenas-Pinto, Nicolas J Mueller, Alan Winston, Moses S Nsubuga, Luxshimi Lal, Richard W Price
OBJECTIVE: To compare the effect of immediate versus deferred antiretroviral treatment (ART) on neuropsychological test performance in treatment-naive HIV-positive adults with more than 500 CD4 cells/μl. DESIGN: Randomized trial. METHODS: The START parent study randomized participants to commence immediate versus deferred ART until CD4 less than 350 cells/μl. The START Neurology substudy used eight neuropsychological tests, at baseline, months 4, 8, 12 and annually, to compare groups for changes in test performance...
May 15, 2018: AIDS
https://www.readbyqxmd.com/read/29394260/healthcare-resource-use-and-costs-of-managing-children-and-adults-with-lysosomal-acid-lipase-deficiency-at-a-tertiary-referral-centre-in-the-united-kingdom
#4
Julian F Guest, Andy Ingram, Nadia Ayoub, Christian J Hendriksz, Elaine Murphy, Yusof Rahman, Patrick McKiernan, Helen Mundy, Patrick Deegan
OBJECTIVE: To estimate clinical progression and resource utilisation together with the associated costs of managing children and adults with LAL Deficiency, at a tertiary referral centre in the UK. METHODS: A retrospective chart review was undertaken of patients in the UK with a confirmed diagnosis of LAL Deficiency who were managed at a LAL Deficiency tertiary referral treatment centre. Patients' pathways, treatment patterns, health outcomes and resource use were quantified over differing lengths of time for each patient enabling the NHS cost of patient management in tertiary care to be estimated...
2018: PloS One
https://www.readbyqxmd.com/read/29249276/adjunctive-rifampicin-for-staphylococcus-aureus-bacteraemia-arrest-a-multicentre-randomised-double-blind-placebo-controlled-trial
#5
Guy E Thwaites, Matthew Scarborough, Alexander Szubert, Emmanuel Nsutebu, Robert Tilley, Julia Greig, Sarah A Wyllie, Peter Wilson, Cressida Auckland, Janet Cairns, Denise Ward, Pankaj Lal, Achyut Guleri, Neil Jenkins, Julian Sutton, Martin Wiselka, Gonzalez-Ruiz Armando, Clive Graham, Paul R Chadwick, Gavin Barlow, N Claire Gordon, Bernadette Young, Sarah Meisner, Paul McWhinney, David A Price, David Harvey, Deepa Nayar, Dakshika Jeyaratnam, Tim Planche, Jane Minton, Fleur Hudson, Susan Hopkins, John Williams, M Estee Török, Martin J Llewelyn, Jonathan D Edgeworth, A Sarah Walker
BACKGROUND: Staphylococcus aureus bacteraemia is a common cause of severe community-acquired and hospital-acquired infection worldwide. We tested the hypothesis that adjunctive rifampicin would reduce bacteriologically confirmed treatment failure or disease recurrence, or death, by enhancing early S aureus killing, sterilising infected foci and blood faster, and reducing risks of dissemination and metastatic infection. METHODS: In this multicentre, randomised, double-blind, placebo-controlled trial, adults (≥18 years) with S aureus bacteraemia who had received ≤96 h of active antibiotic therapy were recruited from 29 UK hospitals...
December 14, 2017: Lancet
https://www.readbyqxmd.com/read/29044819/repeated-sleep-quality-assessment-and-use-of-sleep-promoting-interventions-in-icu
#6
Ashika Menear, Rosalind Elliott, Leanne M Aitken, Sara Lal, Sharon McKinley
To describe sleep quality using repeated subjective assessment and the ongoing use of sleep-promoting interventions in intensive care. It is well known that the critically ill experience sleep disruption while receiving treatment in the intensive care unit. Both the measurement and promotion of sleep is challenging in the complex environment of intensive care unit. Repeated subjective assessment of patients' sleep in the intensive care unit and use of sleep-promoting interventions has not been widely reported...
October 16, 2017: Nursing in Critical Care
https://www.readbyqxmd.com/read/28924018/ibrutinib-for-chronic-graft-versus-host-disease-after-failure-of-prior-therapy
#7
MULTICENTER STUDY
David Miklos, Corey S Cutler, Mukta Arora, Edmund K Waller, Madan Jagasia, Iskra Pusic, Mary E Flowers, Aaron C Logan, Ryotaro Nakamura, Bruce R Blazar, Yunfeng Li, Stephen Chang, Indu Lal, Jason Dubovsky, Danelle F James, Lori Styles, Samantha Jaglowski
Chronic graft-versus-host disease (cGVHD) is a serious complication of allogeneic stem cell transplantation with few effective options available after failure of corticosteroids. B and T cells play a role in the pathophysiology of cGVHD. Ibrutinib inhibits Bruton tyrosine kinase in B cells and interleukin-2-inducible T-cell kinase in T cells. In preclinical models, ibrutinib reduced severity of cGVHD. This multicenter, open-label study evaluated the safety and efficacy of ibrutinib in patients with active cGVHD with inadequate response to corticosteroid-containing therapies...
November 23, 2017: Blood
https://www.readbyqxmd.com/read/28912187/pediatric-cardiomyopathies
#8
REVIEW
Teresa M Lee, Daphne T Hsu, Paul Kantor, Jeffrey A Towbin, Stephanie M Ware, Steven D Colan, Wendy K Chung, John L Jefferies, Joseph W Rossano, Chesney D Castleberry, Linda J Addonizio, Ashwin K Lal, Jacqueline M Lamour, Erin M Miller, Philip T Thrush, Jason D Czachor, Hiedy Razoky, Ashley Hill, Steven E Lipshultz
Pediatric cardiomyopathies are rare diseases with an annual incidence of 1.1 to 1.5 per 100 000. Dilated and hypertrophic cardiomyopathies are the most common; restrictive, noncompaction, and mixed cardiomyopathies occur infrequently; and arrhythmogenic right ventricular cardiomyopathy is rare. Pediatric cardiomyopathies can result from coronary artery abnormalities, tachyarrhythmias, exposure to infection or toxins, or secondary to other underlying disorders. Increasingly, the importance of genetic mutations in the pathogenesis of isolated or syndromic pediatric cardiomyopathies is becoming apparent...
September 15, 2017: Circulation Research
https://www.readbyqxmd.com/read/28902626/pediatric-differentiated-thyroid-carcinoma-trends-in-practice-and-outcomes-over-40-years-at-a-single-tertiary-care-institution
#9
Courtney Olmsted, Rajalakshmi Arunachalam, Xiang Gao, Liuska Pesce, Geeta Lal
BACKGROUND: This study aims to analyze changes in characteristics, practice and outcomes of pediatric differentiated thyroid cancer (DTC) at our tertiary care institution. METHODS: Patients <21 years of age diagnosed between 1973 and 2013 were identified. Clinicopathological data, treatment and outcomes were obtained by a retrospective review. RESULTS: Thirteen males and 68 females were divided into Group A (n=35, diagnosed before July 1993) and Group B (n=46, diagnosed after July 1993)...
October 26, 2017: Journal of Pediatric Endocrinology & Metabolism: JPEM
https://www.readbyqxmd.com/read/28838934/management-of-cardiac-involvement-associated-with-neuromuscular-diseases-a-scientific-statement-from-the-american-heart-association
#10
Brian Feingold, William T Mahle, Scott Auerbach, Paula Clemens, Andrea A Domenighetti, John L Jefferies, Daniel P Judge, Ashwin K Lal, Larry W Markham, W James Parks, Takeshi Tsuda, Paul J Wang, Shi-Joon Yoo
For many neuromuscular diseases (NMDs), cardiac disease represents a major cause of morbidity and mortality. The management of cardiac disease in NMDs is made challenging by the broad clinical heterogeneity that exists among many NMDs and by limited knowledge about disease-specific cardiovascular pathogenesis and course-modifying interventions. The overlay of compromise in peripheral muscle function and other organ systems, such as the lungs, also makes the simple application of endorsed adult or pediatric heart failure guidelines to the NMD population problematic...
September 26, 2017: Circulation
https://www.readbyqxmd.com/read/28804516/the-role-of-sebelipase-alfa-in-the-treatment-of-lysosomal-acid-lipase-deficiency
#11
REVIEW
Angelika L Erwin
Lysosomal acid lipase deficiency (LALD) is a lysosomal storage disorder (LSD) characterized either by infantile onset with fulminant clinical course and very poor prognosis or childhood/adult-onset disease with an attenuated phenotype. The disorder is often misdiagnosed or remains undiagnosed in children and adults due to a rather unspecific clinical presentation with dyslipidemia and steatohepatitis. Until recently, no good treatment options were available for LALD. Despite supportive and symptomatic therapies, death occurred before 1 year of age in patients with infantile-onset disease and patients with childhood/adult-onset LALD suffered from significant complications, such as liver cirrhosis, requiring liver transplantation and early-onset cardiovascular disease...
July 2017: Therapeutic Advances in Gastroenterology
https://www.readbyqxmd.com/read/28578700/a-rare-presentation-of-an-acute-abdomen-an-ileal-diverticular-perforation
#12
Basuru Uvindu Thilakawardana, Sanjay De Mel, Vasitha Abeysuriya, Janaki Hewavisenthi, Chandima De Mel, Lal Chandrasena, Visula Abeysuriya
BACKGROUND: This case report highlights the value of prompt intervention of diagnostic laparoscopy in a patient suspects of having an acute abdomen due to an intestinal perforation, where there is a limitation of performing Contrast Enhanced Computed Tomography of abdomen. A previously healthy young adult presenting with an acute abdomen due to a spontaneous ileal perforation, without any associated risk factors is a rare clinical entity in a developing country. Therefore, entertaining an early diagnosis will possibly prevent a fatal consequence...
June 2, 2017: BMC Research Notes
https://www.readbyqxmd.com/read/28504497/liver-disease-and-dyslipidemia-as-a-manifestation-of-lysosomal-acid-lipase-deficiency-lal-d-clinical-and-diagnostic-aspects-and-a-new-treatment-an-update
#13
Luisa Bay, Cristina Canero Velasco, Mirta Ciocca, Andrea Cotti, Miriam Cuarterolo, Alejandro Fainboim, Eduardo Fassio, Marcela Galoppo, Federico Pinero, Paula Rozenfeld
Lysosomal acid lipase deficiency (LAL-D) is still a little recognized genetic disease with significant morbidity and mortality in children and adults. This document provides guidance on when to suspect LAL-D and how to diagnose it. It is recommended to add lysosomal acid lipase deficiency to the List of differential diagnoses of sepsis, oncological diseases, storage diseases, persistent diarrhea, chronic malnutrition, and hemophagocytic lymphohistiocytosis. It should also be considered in young patients with dyslipidemia and atherosclerosis as well as diseases associated with fatty liver and/or hepatomegaly...
June 1, 2017: Archivos Argentinos de Pediatría
https://www.readbyqxmd.com/read/28303631/surgical-outcomes-of-percutaneous-nephrolithotomy-in-3402-patients-and-results-of-stone-analysis-in-1559-patients
#14
Syed Adibul Hasan Rizvi, Manzoor Hussain, Syed Hassan Askari, Altaf Hashmi, Murli Lal, Mirza Naqi Zafar
OBJECTIVE: To report our experience of a series of percutaneous nephrolithotomy (PCNL) procedures in a single centre over 18 years in terms of patient and stone characteristics, indications, stone clearance and complications, along with the results of chemical analysis of stones in a subgroup. PATIENTS AND METHODS: We retrospectively analysed the outcomes of PCNL in 3402 patients, who underwent the procedure between 1997 and 2014, obtained from a prospectively maintained database...
November 2017: BJU International
https://www.readbyqxmd.com/read/28197978/managing-cardiovascular-risk-in-lysosomal-acid-lipase-deficiency
#15
REVIEW
James J Maciejko
Lysosomal acid lipase deficiency (LAL-D) is a rare, life-threatening, autosomal recessive, lysosomal storage disease caused by mutations in the LIPA gene, which encodes for lysosomal acid lipase (LAL). This enzyme is necessary for the hydrolysis of cholesteryl ester and triglyceride in lysosomes. Deficient LAL activity causes accumulation of these lipids in lysosomes and a marked decrease in the cytoplasmic free cholesterol concentration, leading to dysfunctional cholesterol homeostasis. The accumulation of neutral lipid occurs predominantly in liver, spleen, and macrophages throughout the body, and the aberrant cholesterol homeostasis causes a marked dyslipidemia...
June 2017: American Journal of Cardiovascular Drugs: Drugs, Devices, and Other Interventions
https://www.readbyqxmd.com/read/28018078/subungual-osteochondroma-nail-sparing-excision
#16
Anurag Tiwari, Nidhi Agrawal, Tarun Verma, Hitesh Lal
Subungual osteochondroma is a relatively uncommon benign bone tumor affecting mostly children and young adults, and is a major source of pain and nail deformity. Treatment consists of marginal excision and meticulous wound closure. In this report, we present three cases of subungual osteochondroma arising from the dorsal aspect of distal phalanx of the great toe, which were managed by marginal excision with preservation of nail apparatus under digital block anesthesia with the use of a ring tourniquet. We emphasize on the use of ring tourniquet made by glove's finger and the technique of preserving the nail apparatus to prevent nail deformity...
October 2016: Journal of Clinical Orthopaedics and Trauma
https://www.readbyqxmd.com/read/27878737/sebelipase-alfa-a-review-in-lysosomal-acid-lipase-deficiency
#17
REVIEW
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...
December 2016: American Journal of Cardiovascular Drugs: Drugs, Devices, and Other Interventions
https://www.readbyqxmd.com/read/27799810/novel-treatment-options-for-lysosomal-acid-lipase-deficiency-critical-appraisal-of-sebelipase-alfa
#18
REVIEW
Kim Su, Emma Donaldson, Reena Sharma
Lysosomal acid lipase deficiency (LAL-D) is a rare disorder of cholesterol metabolism with an autosomal recessive mode of inheritance. The absence or deficiency of the LAL enzyme gives rise to pathological accumulation of cholesterol esters in various tissues. A severe LAL-D phenotype manifesting in infancy is associated with adrenal calcification and liver and gastrointestinal involvement with characteristic early mortality. LAL-D presenting in childhood and adulthood is associated with hepatomegaly, liver fibrosis, cirrhosis, and premature atherosclerosis...
2016: Application of Clinical Genetics
https://www.readbyqxmd.com/read/27733791/protective-and-ameliorative-effect-of-sea-buckthorn-leaf-extract-supplementation-on-lead-induced-hemato-biochemical-alterations-in-wistar-rats
#19
Rizwana Zargar, Pratiksha Raghuwanshi, Ankur Rastogi, Aditi Lal Koul, Pallavi Khajuria, Aafreen Wahid Ganai, Sumeet Kour
AIM: To evaluate the protective and ameliorative effect of aqueous sea buckthorn leaf extract (SLE) on hemato-biochemical profile in lead intoxicated Wistar rats. MATERIALS AND METHODS: An experiment was conducted for 60 days. 36 adult male Wistar rats with a mean body weight of 177.8±12.6 g were divided into five groups and were subjected to various daily oral treatment regimens. Group I served as a negative control receiving only feed and water, Group II (positive control for lead) received lead acetate at 250 ppm in drinking water, and Group III (positive control for SLE) received SLE at 100 mg/kg b...
September 2016: Veterinary World
https://www.readbyqxmd.com/read/27437275/self-mutilating-behaviour-in-severe-meningococcal-infection-an-interesting-association
#20
Anju Dinkar, Jitendra Singh, Virendra Atam, Krishna Kumar Sahani, Munna Lal Patel
Neisseria meningitidis most commonly manifests as asymptomatic colonization in the nasopharynx of healthy adolescents and adults. It may rarely present as invasive disease which may be either bacterial meningitis or meningococcal septicaemia. Hereby we report a case presented with fever and rashes, irritability followed by self mutilating behaviour who was diagnosed as a case of invasive meningococcal infection. He responded well to treatment with intravenous ceftriaxone and self mutilating behaviour was subsided completely after treatment...
May 2016: Journal of Clinical and Diagnostic Research: JCDR
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