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https://www.readbyqxmd.com/read/28329498/bullous-pemphigoid-of-infancy-report-and-review-of-infantile-and-pediatric-bullous-pemphigoid
#1
Bárbara R Ferreira, Ana S Vaz, Leonor Ramos, José P Reis, Margarida Gonçalo
A 4-month-old infant was observed with an acute itchy bullous dermatosis, predominantly involving the extremities, which revealed a dermal infiltrate rich in eosinophils, C3 deposits at the dermalepidermal junction, and circulating antibodies to BP180 antigen, confirming the diagnosis of bullous pemphigoid. He was initially treated with deflazacort 1 mg/kg/day, further increased to 2 mg/ kg/day, followed by reduction over seven weeks with complete clinical resolution within this period. We discuss epidemiology, etiology, relationship with vaccination, clinical features, and treatment of thisrelatively rare bullous dermatosis in the pediatric age...
February 16, 2017: Dermatology Online Journal
https://www.readbyqxmd.com/read/28274975/deflazacort-approved-for-duchenne-muscular-dystrophy
#2
Kate Traynor
No abstract text is available yet for this article.
March 15, 2017: American Journal of Health-system Pharmacy: AJHP
https://www.readbyqxmd.com/read/28179743/lixisenatide
#3
Danial E Baker, Terri L Levien
Each month, subscribers to The Formulary Monograph Service receive 5 to 6 well-documented monographs on drugs that are newly released or are in late phase 3 trials. The monographs are targeted to Pharmacy & Therapeutics Committees. Subscribers also receive monthly 1-page summary monographs on agents that are useful for agendas and pharmacy/nursing in-services. A comprehensive target drug utilization evaluation/medication use evaluation (DUE/MUE) is also provided each month. With a subscription, the monographs are are available online to subscribers...
January 2017: Hospital Pharmacy
https://www.readbyqxmd.com/read/28057083/deflazacort-therapeutic-index-relative-potency-and-equivalent-doses-versus-other-corticosteroids
#4
Luca Parente
BACKGROUND: Deflazacort is a synthetic corticosteroid characterized by a favourable pharmacokinetic profile, peculiar pharmacodynamic properties and a good safety profile. However, to the best of our knowledge, no dose-conversion table based on direct comparison of relative potencies between deflazacort and other main corticosteroids is currently available in scientific literature. MAIN BODY: This paper, while reporting a brief review of deflazacort pharmacological properties, its efficacy and tolerability in different clinical areas, has been designed with the specific aim of providing a new dose-conversion table of corticosteroids, including for the first time also deflazacort...
January 5, 2017: BMC Pharmacology & Toxicology
https://www.readbyqxmd.com/read/28043681/long-term-outcome-of-interdisciplinary-management-of-patients-with-duchenne-muscular-dystrophy-receiving-daily-glucocorticoid-treatment
#5
Brenda L Wong, Irina Rybalsky, Karen C Shellenbarger, Cuixia Tian, Mary A McMahon, Meilan M Rutter, Hemant Sawnani, John L Jefferies
OBJECTIVE: To evaluate clinical outcomes and steroid side effects in a cohort of patients with Duchenne muscular dystrophy (DMD) treated with long-term daily glucocorticoid therapy. Although daily glucocorticoid therapy has been shown to extend ambulatory function in DMD, less frequent dosing is often used because of side effect concerns. STUDY DESIGN: Retrospective study of 97 patients with DMD aged 10 to <16 years treated with daily glucocorticoid (89% on deflazacort) for a mean of 8...
March 2017: Journal of Pediatrics
https://www.readbyqxmd.com/read/28018458/successful-treatment-of-tubulointerstitial-nephritis-and-uveitis-with-steroid-and-azathioprine-in-a-12-year-old-boy
#6
Ji Eun Kim, Se Jin Park, Ji Young Oh, Hyeon Joo Jeong, Ji Hong Kim, Jae Il Shin
Tubulointerstitial nephritis and uveitis (TINU) syndrome is a rare disease, often underdiagnosed or misdiagnosed in children. We describe the case of a 12-year-old boy who presented to Severance Hospital with a 1-month history of bilateral conjunctival injection. He was first evaluated by an Ophthalmologist in another hospital and diagnosed with panuveitis. Laboratory tests indicated renal failure, and a renal biopsy confirmed the diagnosis of acute tubulointerstitial nephritis. An extensive exclusion of all possible causes allowed a diagnosis of TINU syndrome...
November 2016: Korean Journal of Pediatrics
https://www.readbyqxmd.com/read/28018451/eosinophilic-gastroenteritis-in-an-18-year-old-male-with-prolonged-nephrotic-syndrome
#7
Da Min Choi, Jung Eun Pyun, Hyung Eun Yim, Kee Hwan Yoo, Jung Ok Shim, Eun Jung Lee, Nam Hee Won
Eosinophilic gastroenteritis is a rare disease characterized by prominent eosinophilic tissue infiltration of the gastrointestinal tract. Here, we report a case of eosinophilic gastroenteritis in an 18-year-old patient with prolonged nephrotic syndrome who presented with abdominal pain and peripheral hypereosinophilia. During the previous 2 years, he had visited local Emergency Department several times because of epigastric pain and nausea. He had been treated with steroid-dependent nephrotic syndrome since 3 years of age...
November 2016: Korean Journal of Pediatrics
https://www.readbyqxmd.com/read/27843100/isolation-and-structural-characterization-of-novel-photolytic-degradation-impurities-of-deflazacort-using-q-tof-2d-nmr-and-ftir
#8
Ch Krishnam Raju, Avadhesh Kumar Pandey, Kaushik Ghosh, Arunima Pola, Sanath Kumar Goud, Mrunal A Jaywant, Sameer G Navalgund, Koduru V Surendranath
Forced Degradation of Deflazacort drug substance in ultraviolet light condition resulted into a number of significant degradation products. Two of these degradation products were found to be unknown during the study and marked as DD-I and DD-II. Thus, the objective of this work is to investigate and identify these two novel degradation products of DFZ. The isolation method for these new degradation products were developed using a new reverse-phase high performance liquid chromatography (HPLC). DD-I and DD-II, eluting at 0...
January 30, 2017: Journal of Pharmaceutical and Biomedical Analysis
https://www.readbyqxmd.com/read/27746500/retrospective-cohort-study-comparing-the-efficacy-of-prednisolone-and-deflazacort-in-children-with-muscular-dystrophy-a-6-years-experience-in-a-south-indian-teaching-hospital
#9
Harish Petnikota, Vrisha Madhuri, Sangeet Gangadharan, Indira Agarwal, Belavendra Antonisamy
BACKGROUND: Muscular dystrophies are inherited myogenic disorders characterized by progressive muscle wasting and weakness of variable distribution and severity. They are a heterogeneous group characterized by variable degree of skeletal and cardiac muscle involvement. The most common and the most severe form of muscular dystrophy is DMD. Currently, there is no curative treatment for muscular dystrophies. Several drugs have been studied to retard the progression of the muscle weakness...
September 2016: Indian Journal of Orthopaedics
https://www.readbyqxmd.com/read/27566742/efficacy-and-safety-of-deflazacort-vs-prednisone-and-placebo-for-duchenne-muscular-dystrophy
#10
Robert C Griggs, J Phillip Miller, Cheryl R Greenberg, Darcy L Fehlings, Alan Pestronk, Jerry R Mendell, Richard T Moxley, Wendy King, John T Kissel, Valerie Cwik, Michel Vanasse, Julaine M Florence, Shree Pandya, Jordan S Dubow, James M Meyer
OBJECTIVE: To assess safety and efficacy of deflazacort (DFZ) and prednisone (PRED) vs placebo in Duchenne muscular dystrophy (DMD). METHODS: This phase III, double-blind, randomized, placebo-controlled, multicenter study evaluated muscle strength among 196 boys aged 5-15 years with DMD during a 52-week period. In phase 1, participants were randomly assigned to receive treatment with DFZ 0.9 mg/kg/d, DFZ 1.2 mg/kg/d, PRED 0.75 mg/kg/d, or placebo for 12 weeks. In phase 2, placebo participants were randomly assigned to 1 of the 3 active treatment groups...
November 15, 2016: Neurology
https://www.readbyqxmd.com/read/27542440/identifying-evidence-of-cardio-renal-syndrome-in-patients-with-duchenne-muscular-dystrophy-using-cystatin-c
#11
Chet R Villa, Ahmad Kaddourah, Jacob Mathew, Thomas D Ryan, Brenda L Wong, Stuart L Goldstein, John L Jefferies
Patients with Duchenne muscular dystrophy (DMD) develop dilated cardiomyopathy and are at risk for kidney injury. Creatinine based estimated glomerular filtration rate (eGFR) is limited by low muscle mass with low serum creatinine levels in DMD. We assessed the relationship between cardiac function, modified Schwartz eGFR and cystatin C eGFR in patients with DMD. Ninety-three patients with DMD were screened for renal dysfunction in an outpatient neuromuscular clinic. Patients with new nephrotoxic medications, recent hospitalization or decompensated heart failure were excluded from the analysis...
October 2016: Neuromuscular Disorders: NMD
https://www.readbyqxmd.com/read/27525172/prednisone-and-deflazacort-in-duchenne-muscular-dystrophy-do-they-play-a-different-role-in-child-behavior-and-perceived-quality-of-life
#12
Susan Sienko, Cathleen Buckon, Eileen Fowler, Anita Bagley, Loretta Staudt, Mitell Sison-Williamson, Kathy Zebracki, Craig M McDonald, Michael Sussman
The aim of this study was to determine whether prednisone and deflazacort play a different role in child behavior and perceived health related psychosocial quality of life in ambulant boys with Duchenne Muscular Dystrophy. As part of a prospective natural-history study, parents of sixty-seven ambulant boys with DMD (27 taking prednisone, 15 taking deflazacort, 25 were steroid naïve) completed the Child Behavior Checklist (CBCL) for assessment of behavioral, emotional and social problems and both parents and boys with DMD completed the PedsQL™4...
June 17, 2016: PLoS Currents
https://www.readbyqxmd.com/read/27500011/can-quantitative-muscle-strength-and-functional-motor-ability-differentiate-the-influence-of-age-and-corticosteroids-in-ambulatory-boys-with-duchenne-muscular-dystrophy
#13
Cathleen Buckon, Susan Sienko, Anita Bagley, Mitell Sison-Williamson, Eileen Fowler, Loretta Staudt, Kent Heberer, Craig M McDonald, Michael Sussman
BACKGROUND: In the absence of a curative treatment for Duchenne Muscular Dystrophy (DMD), corticosteroid therapy (prednisone, deflazacort) has been adopted as the standard of care, as it slows the progression of muscle weakness and enables longer retention of functional mobility. The ongoing development of novel pharmacological agents that target the genetic defect underlying DMD offer hope for a significant alteration in disease progression; however, substantiation of therapeutic efficacy has proved challenging...
2016: PLoS Currents
https://www.readbyqxmd.com/read/27328118/vertebral-fractures-in-duchenne-muscular-dystrophy-patients-managed-with-deflazacort
#14
Amardeep Singh, Emily K Schaeffer, Christopher W Reilly
BACKGROUND: Corticosteroids are widely used in the management of patients with Duchenne muscular dystrophy (DMD). They improve quality of life in these patients by prolonging ambulation and preserving cardiorespiratory status. However, corticosteroid treatment is associated with a decrease in bone mineral density (BMD) and an increased risk of vertebral fractures (VF). The purpose of this study was to investigate the prevalence of VF in patients with DMD undergoing long-term treatment with the corticosteroid deflazacort...
June 18, 2016: Journal of Pediatric Orthopedics
https://www.readbyqxmd.com/read/27264291/deflazacort-hypersensitivity-a-difficult-to-manage-case-of-systemic-allergic-dermatitis-and-literature-review
#15
Leonardo Bianchi, Katharina Hansel, Elettra Antonelli, Veronica Bellini, Luigi Rigano, Luca Stingeni
No abstract text is available yet for this article.
July 2016: Contact Dermatitis
https://www.readbyqxmd.com/read/27149418/corticosteroids-for-the-treatment-of-duchenne-muscular-dystrophy
#16
REVIEW
Emma Matthews, Ruth Brassington, Thierry Kuntzer, Fatima Jichi, Adnan Y Manzur
BACKGROUND: Duchenne muscular dystrophy (DMD) is the most common muscular dystrophy of childhood. Untreated, this incurable disease, which has an X-linked recessive inheritance, is characterised by muscle wasting and loss of walking ability, leading to complete wheelchair dependence by 13 years of age. Prolongation of walking is a major aim of treatment. Evidence from randomised controlled trials (RCTs) indicates that corticosteroids significantly improve muscle strength and function in boys with DMD in the short term (six months), and strength at two years (two-year data on function are very limited)...
May 5, 2016: Cochrane Database of Systematic Reviews
https://www.readbyqxmd.com/read/27039228/corticosteroid-treatment-and-growth-patterns-in-ambulatory-males-with-duchenne-muscular-dystrophy
#17
Molly M Lamb, Nancy A West, Lijing Ouyang, Michele Yang, David Weitzenkamp, Katherine James, Emma Ciafaloni, Shree Pandya, Carolyn DiGuiseppi
OBJECTIVES: To evaluate growth patterns of ambulatory males with Duchenne muscular dystrophy (DMD) treated with corticosteroids compared with ambulatory, steroid-naïve males with DMD and age-matched unaffected general-population males and to test associations between growth and steroid treatment patterns among treated males. STUDY DESIGN: Using data from the Muscular Dystrophy Surveillance, Tracking, and Research Network, we identified a total of 1768 height, 2246 weight, and 1755 body mass index (BMI) measurements between age 2 and 12 years for 324 ambulatory males who were treated with corticosteroids for at least 6 months...
June 2016: Journal of Pediatrics
https://www.readbyqxmd.com/read/26885481/toxic-epidermal-necrolysis-associated-with-deflazacort-therapy-with-nephrotic-syndrome
#18
Eun Chae Lee, Geun A Kim, Ja Wook Koo
Toxic epidermal necrolysis (TEN) is a drug-related fatal disease. Extensive necrosis of the epidermis can lead to serious complications. This report describes two cases of TEN, associated with deflazacort (DFZ), in two boys, aged 4 years and 14 years, with nephrotic syndrome (NS). The 14-year-old male teenager received DFZ following NS relapse. After 17 days, pruritic papules appeared on the lower extremities. Another case involved a 4-year-old boy receiving DFZ and enalapril. After a 41-day DFZ treatment period, erythematous papules appeared on the palms and soles...
December 2014: Kidney Research and Clinical Practice
https://www.readbyqxmd.com/read/26833937/practice-guideline-update-summary-corticosteroid-treatment-of-duchenne-muscular-dystrophy-report-of-the-guideline-development-subcommittee-of-the-american-academy-of-neurology
#19
REVIEW
David Gloss, Richard T Moxley, Stephen Ashwal, Maryam Oskoui
OBJECTIVE: To update the 2005 American Academy of Neurology (AAN) guideline on corticosteroid treatment of Duchenne muscular dystrophy (DMD). METHODS: We systematically reviewed the literature from January 2004 to July 2014 using the AAN classification scheme for therapeutic articles and predicated recommendations on the strength of the evidence. RESULTS: Thirty-four studies met inclusion criteria. RECOMMENDATIONS: In children with DMD, prednisone should be offered for improving strength (Level B) and pulmonary function (Level B)...
February 2, 2016: Neurology
https://www.readbyqxmd.com/read/26813773/ifn-beta-1a-as-glucocorticoids-sparing-therapy-in-a-patient-with-clippers
#20
María Rico, Javier Villafani, Alberto Tuñón, Valentín Mateos, Pedro Oliva-Nacarino
BACKGROUND: Chronic lymphocytic inflammation with pontine perivascular enhancement responsive to steroids (CLIPPERS) is a recently described inflammatory disease of the central nervous system, distinguished by brainstem- and spinal cord-centered lesions with a characteristic contrast enhancement on MRI, a lymphocytic perivascular infiltrate on pathological exam, and a dramatic response to and dependence on steroids therapy. Since its initial description in 2010, different glucocorticoid-sparing agents, mostly immunosuppressant drugs, have been used to minimize the dosage, but these therapies also carry the risk of important secondary effects...
January 27, 2016: American Journal of Case Reports
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