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https://www.readbyqxmd.com/read/28214088/a-case-of-seropositive-autoimmune-autonomic-ganglionopathy-with-diffuse-esophageal-spasm
#1
Nobutoshi Morimoto, Sakuma Takahashi, Tomoki Inaba, Motonori Takamiya, Yasuhiko Kageyama, Mizuki Morimoto, Yoshiaki Takahashi, Hirotake Nishimura, Shunya Nakane, Koji Abe
Autoimmune autonomic ganglionopathy (AAG) is an immune-mediated disorder that leads to various autonomic failures associated with anti-ganglionic acetylcholine receptor antibodies (anti-gAChR-Abs). Diffuse esophageal spasm (DES) is an uncommon esophageal motility disorder. We herein report the case of a 68-year-old woman with DES as a partial symptom of AAG. She presented with chronic esophageal transit failure, constipation, and numbness of the hands and feet, Adie's pupil, thermal hypoalgesia, and decreased deep tendon reflexes...
February 14, 2017: Journal of Clinical Neuroscience: Official Journal of the Neurosurgical Society of Australasia
https://www.readbyqxmd.com/read/28212527/a-multicenter-phase-ii-study-of-salvage-photodynamic-therapy-using-talaporfin-sodium-me2906-and-a-diode-laser-pnl6405epg-for-local-failure-after-chemoradiotherapy-or-radiotherapy-for-esophageal-cancer
#2
Tomonori Yano, Hiroi Kasai, Takahiro Horimatsu, Kenichi Yoshimura, Satoshi Teramukai, Satoshi Morita, Harue Tada, Yoshinobu Yamamoto, Hiromi Kataoka, Naomi Kakushima, Ryu Ishihara, Hajime Isomoto, Manabu Muto
Photodynamic therapy (PDT) showed promising efficacy for local failure after chemoradiotherapy (CRT) for esophageal cancer. However, PDT required long sun shade period. This study aimed to evaluate the safety and efficacy of PDT using second generation photosensitizer, talaporfin sodium for local failure after CRT. This was the multi-institutional non-randomized phase II study. Patients with histologically proven local failure limited within the muscularis propria after 50Gy or more radiotherapy (RT) for esophageal cancer were eligible...
December 20, 2016: Oncotarget
https://www.readbyqxmd.com/read/28212153/new-therapies-for-the-prevention-and-treatment-of-exacerbations-of-bronchiectasis
#3
Hani Abo-Leyah, James D Chalmers
PURPOSE OF REVIEW: Exacerbations of bronchiectasis have a major impact on quality of life, healthcare costs, and long-term risk of complications. Preventing exacerbations is one of the major goals of treatment. Bronchiectasis is increasingly recognized and the impact of bronchiectasis exacerbations on daily clinical practice is also increasing. RECENT FINDINGS: Preventing bronchiectasis exacerbations are dependent on appropriate risk assessment, identifying the patients at highest risk to rationally target preventive therapies...
February 16, 2017: Current Opinion in Pulmonary Medicine
https://www.readbyqxmd.com/read/28211988/intrathecal-enzyme-replacement-therapy-reverses-cognitive-decline-in-mucopolysaccharidosis-type-i
#4
Igor Nestrasil, Elsa Shapiro, Alena Svatkova, Patricia Dickson, Agnes Chen, Amy Wakumoto, Alia Ahmed, Edward Stehel, Sarah McNeil, Curtis Gravance, Elizabeth Maher
Mucopolysaccharidosis type I (MPS I) is an inherited lysosomal storage disease that seriously affects the brain. Severity of neurocognitive symptoms in attenuated MPS subtype (MPS IA) broadly varies partially, due to restricted permeability of blood-brain barrier (BBB) which limits treatment effects of intravenously applied α-L-iduronidase (rhIDU) enzyme. Intrathecal (IT) rhIDU application as a possible solution to circumvent BBB improved brain outcomes in canine models; therefore, our study quantifies effects of IT rhIDU on brain structure and function in an MPS IA patient with previous progressive cognitive decline...
March 2017: American Journal of Medical Genetics. Part A
https://www.readbyqxmd.com/read/28211941/upfront-window-vincristine-irinotecan-treatment-of-high-risk-hepatoblastoma-a-report-from-the-children-s-oncology-group-ahep0731-study-committee
#5
Howard M Katzenstein, Wayne L Furman, Marcio H Malogolowkin, Mark D Krailo, M Beth McCarville, Alexander J Towbin, Greg M Tiao, Milton J Finegold, Sarangarajan Ranganathan, Stephen P Dunn, Max R Langham, Eugene D McGahren, Carlos Rodriguez-Galindo, Rebecka L Meyers
BACKGROUND: The identification of new therapies for high-risk (HR) hepatoblastoma is challenging. Children's Oncology Group study AHEP0731 included a HR stratum to explore the efficacy of novel agents. Herein, the authors report the response rate to the combination of vincristine (V) and irinotecan (I) and the outcome of patients with high-risk hepatoblastoma. METHODS: Patients with newly diagnosed metastatic hepatoblastoma or those with a serum α-fetoprotein (AFP) level <100 ng/mL were eligible...
February 17, 2017: Cancer
https://www.readbyqxmd.com/read/28211589/the-effectiveness-of-intravenous-vitamin-k-in-correcting-cirrhosis-associated-coagulopathy
#6
Ryan M Rivosecchi, Sandra L Kane-Gill, Jeffrey Garavaglia, Adam MacLasco, Heather Johnson
OBJECTIVES: The goal of this study was to evaluate the effectiveness of intravenous (IV) vitamin K in cirrhosis. METHODS: This was a retrospective study of cirrhotic patients, not on anticoagulation, with administration of IV vitamin K and a baseline INR > 1.5. The primary outcome was the effectiveness of therapy defined by a 30% decrease in INR or a reduction in INR to an absolute value of ≤1.5. KEY FINDINGS: A total of 96 patients were included in the cohort...
February 17, 2017: International Journal of Pharmacy Practice
https://www.readbyqxmd.com/read/28210182/successful-cataract-surgery-in-a-patient-with-refractory-wegener-s-granulomatosis-effectively-treated-with-rituximab-a-case-report
#7
Abdullah M Alfawaz
: Wegener's granulomatosis is a granulomatous disorder associated with systemic necrotizing vasculitis. Eye involvement occurs in approximately 50% of Wegener's granulomatosis patients and is an important cause of morbidity. Conventional treatment-related morbidity and failure have led to studies of alternative treatment modalities. In this case of a 35-year-old man with severe Wegener's granulomatosis, conventional therapy failed to induce remission. Despite the standard immunosuppressive therapy, progression of the disease was observed, mainly with ocular manifestations and renal impairment...
July 2016: Saudi Journal of Ophthalmology: Official Journal of the Saudi Ophthalmological Society
https://www.readbyqxmd.com/read/28209746/a-phase-ii-randomized-trial-go27827-of-first-line-folfox-plus-bevacizumab-with-or-without-the-met-inhibitor-onartuzumab-in-patients-with-metastatic-colorectal-cancer
#8
Johanna C Bendell, Howard Hochster, Lowell L Hart, Irfan Firdaus, Joseph R Mace, Joshua J McFarlane, Mark Kozloff, Daniel Catenacci, Jessie J Hsu, Stephen P Hack, David S Shames, See-Chun Phan, Hartmut Koeppen, Allen L Cohn
BACKGROUND: Dysregulated hepatocyte growth factor/mesenchymal-epithelial transition (MET) signaling is associated with poor prognosis and resistance to vascular endothelial growth factor inhibition in metastatic colorectal cancer (mCRC). We report outcomes from a double-blind, multicenter phase II trial of the MET inhibitor onartuzumab in combination with mFOLFOX-6 and bevacizumab for mCRC (GO27827; NCT01418222). MATERIALS AND METHODS: Patients were randomized 1:1 to receive onartuzumab (10 mg/kg intravenously [IV]) or placebo plus mFOLFOX-6 and bevacizumab (5 mg/kg IV)...
February 16, 2017: Oncologist
https://www.readbyqxmd.com/read/28209305/initial-vasodilatation-in-a-child-with-reversible-cerebral-vasoconstriction-syndrome
#9
Yoshitsugu Oikawa, Yukimune Okubo, Yurika Numata-Uematsu, Yu Aihara, Taro Kitamura, Masaru Takayanagi, Yukitoshi Takahashi, Shigeo Kure, Mitsugu Uematsu
We describe the case of a 10-year-old boy who developed reversible cerebral vasoconstriction syndrome (RCVS) after cerebellitis. He received intravenous immunoglobulin and methylprednisolone to treat the cerebellitis. However, he then presented with a sudden severe headache, vomiting, and generalized tonic-clonic seizure. Brain magnetic resonance angiography (MRA) initially revealed diffuse cerebral vasodilatations, and diffuse multifocal segmental vasoconstrictions developed several days later. His clinical symptoms gradually resolved after several days, in the absence of any specific therapy...
February 10, 2017: Journal of Clinical Neuroscience: Official Journal of the Neurosurgical Society of Australasia
https://www.readbyqxmd.com/read/28209298/utidelone-plus-capecitabine-versus-capecitabine-alone-for-heavily-pretreated-metastatic-breast-cancer-refractory-to-anthracyclines-and-taxanes-a-multicentre-open-label-superiority-phase-3-randomised-controlled-trial
#10
Pin Zhang, Tao Sun, Qingyuan Zhang, Zhongyu Yuan, Zefei Jiang, Xiao Jia Wang, Shude Cui, Yuee Teng, Xi-Chun Hu, Junlan Yang, Hongming Pan, Zhongsheng Tong, Huiping Li, Qiang Yao, Yongsheng Wang, Yongmei Yin, Ping Sun, Hong Zheng, Jing Cheng, Jinsong Lu, Baochun Zhang, Cuizhi Geng, Jian Liu, Roujun Peng, Min Yan, Shaohua Zhang, Jian Huang, Li Tang, Rongguo Qiu, Binghe Xu
BACKGROUND: Utidelone, a genetically engineered epothilone analogue, has shown promise as a potential treatment for breast cancer in phase 1 and 2 trials. The aim of this phase 3 trial was to compare the efficacy and safety of utidelone plus capecitabine versus capecitabine alone in patients with metastatic breast cancer. METHODS: We did a multicentre, open-label, superiority, phase 3, randomised controlled trial in 26 hospitals in China. Eligible participants were female patients with metastatic breast cancer refractory to anthracycline and taxane chemotherapy regimens...
February 10, 2017: Lancet Oncology
https://www.readbyqxmd.com/read/28209296/best-time-to-assess-complete-clinical-response-after-chemoradiotherapy-in-squamous-cell-carcinoma-of-the-anus-act-ii-a-post-hoc-analysis-of-randomised-controlled-phase-3-trial
#11
Robert Glynne-Jones, David Sebag-Montefiore, Helen M Meadows, David Cunningham, Rubina Begum, Fawzi Adab, Kim Benstead, Robert J Harte, Jill Stewart, Sandy Beare, Allan Hackshaw, Latha Kadalayil
BACKGROUND: Guidelines for anal cancer recommend assessment of response at 6-12 weeks after starting treatment. Using data from the ACT II trial, we determined the optimum timepoint to assess clinical tumour response after chemoradiotherapy. METHODS: The previously reported ACT II trial was a phase 3 randomised trial of patients of any age with newly diagnosed, histologically confirmed, squamous cell carcinoma of the anus without metastatic disease from 59 centres in the UK...
February 10, 2017: Lancet Oncology
https://www.readbyqxmd.com/read/28209267/assessment-of-volume-status-and-appropriate-fluid-replenishment-in-the-setting-of-nephrotic-syndrome
#12
Pierluigi Marzuillo, Stefano Guarino, Andrea Apicella, Rosaria Marotta, Vincenzo Tipo, Laura Perrone, Angela La Manna, Giovanni Montini
BACKGROUND: When the permeability of the glomerular filtration barrier increases, leading to proteinuria, nephrotic syndrome (NS) occurs. First episodes or relapses of NS can be concurrent with acute gastroenteritis (AGE) infections. This condition can cause further deterioration of the hypovolemic state, as intravascular water is lost through both AGE-related vomiting/diarrhea and NS-related fluid shifting into the interstitium. In this case report, we wish to raise the issues about the difficult management of children presenting with both NS and AGE...
February 10, 2017: Journal of Emergency Medicine
https://www.readbyqxmd.com/read/28208675/pneumonia-acute-respiratory-distress-syndrome-and-early-immune-modulator-therapy
#13
REVIEW
Kyung-Yil Lee
Acute respiratory distress syndrome (ARDS) is caused by infectious insults, such as pneumonia from various pathogens or related to other noninfectious events. Clinical and histopathologic characteristics are similar across severely affected patients, suggesting that a common mode of immune reaction may be involved in the immunopathogenesis of ARDS. There may be etiologic substances that have an affinity for respiratory cells and induce lung cell injury in cases of ARDS. These substances originate not only from pathogens, but also from injured host cells...
February 11, 2017: International Journal of Molecular Sciences
https://www.readbyqxmd.com/read/28208199/systemic-thrombolytic-therapy-for-acute-pulmonary-embolism-who-is-a-candidate
#14
Stavros V Konstantinides, Stefano Barco
Pulmonary embolism (PE) is a major cause of both acute and long-term morbidity for a large number of patients worldwide, and massive PE is frequently fatal. Right ventricular (RV) dysfunction is a key determinant of prognosis in the acute phase of PE. Patients with clinically overt RV failure, that is, with cardiogenic shock or persistent hypotension at presentation (acute high-risk PE), are clearly in need of immediate reperfusion treatment with systemic thrombolysis or, alternatively, surgical or catheter-directed techniques...
February 2017: Seminars in Respiratory and Critical Care Medicine
https://www.readbyqxmd.com/read/28207759/investigations-on-therapeutic-glucocerebrosidases-through-paired-detection-with-fluorescent-activity-based-probes
#15
Wouter W Kallemeijn, Saskia Scheij, Sascha Hoogendoorn, Martin D Witte, Daniela Herrera Moro Chao, Cindy P A A van Roomen, Roelof Ottenhoff, Herman S Overkleeft, Rolf G Boot, Johannes M F G Aerts
Deficiency of glucocerebrosidase (GBA) causes Gaucher disease (GD). In the common non-neuronopathic GD type I variant, glucosylceramide accumulates primarily in the lysosomes of visceral macrophages. Supplementing storage cells with lacking enzyme is accomplished via chronic intravenous administration of recombinant GBA containing mannose-terminated N-linked glycans, mediating the selective uptake by macrophages expressing mannose-binding lectin(s). Two recombinant GBA preparations with distinct N-linked glycans are registered in Europe for treatment of type I GD: imiglucerase (Genzyme), contains predominantly Man(3) glycans, and velaglucerase (Shire PLC) Man(9) glycans...
2017: PloS One
https://www.readbyqxmd.com/read/28207600/advancements-in-the-critical-care-management-of-status-epilepticus
#16
Andrew Bauerschmidt, Andrew Martin, Jan Claassen
PURPOSE OF REVIEW: Status epilepticus has a high morbidity and mortality. There are little definitive data to guide management; however, new recent data continue to improve understanding of management options of status epilepticus. This review examines recent advancements regarding the critical care management of status epilepticus. RECENT FINDINGS: Recent studies support the initial treatment of status epilepticus with early and aggressive benzodiazepine dosing...
February 14, 2017: Current Opinion in Critical Care
https://www.readbyqxmd.com/read/28207512/incidence-and-risk-factors-of-acute-kidney-injury-associated-with-continuous-intravenous-high-dose-vancomycin-in-critically-ill-patients-a-retrospective-cohort-study
#17
Guillaume Lacave, Vincent Caille, Fabrice Bruneel, Catherine Palette, Stéphane Legriel, David Grimaldi, Mathilde Eurin, Jean-Pierre Bedos
For vancomycin therapy of severe infections, the Infectious Diseases Society of America recommends high vancomycin trough levels, whose potential for inducing nephrotoxicity is controversial. We evaluated the incidence and risk factors of acute kidney injury (AKI) in critically ill patients given continuous intravenous vancomycin with target serum vancomycin levels of 20 to 30 mg/L.We retrospectively studied 107 continuous intravenous vancomycin treatments of ≥48 hours' duration with at least 2 serum vancomycin levels ≥20 mg/L in critically ill patients...
February 2017: Medicine (Baltimore)
https://www.readbyqxmd.com/read/28202000/safety-and-efficacy-of-lobaplatin-combined-with-5-fluorouracil-as-first-line-induction-chemotherapy-followed-by-lobaplatin-radiotherapy-in-locally-advanced-nasopharyngeal-carcinoma-preliminary-results-of-a-prospective-phase-ii-trial
#18
Liang-Ru Ke, Wei-Xiong Xia, Wen-Ze Qiu, Xin-Jun Huang, Jing Yang, Ya-Hui Yu, Hu Liang, Guo-Ying Liu, Yan-Fang Ye, Yan-Qun Xiang, Xiang Guo, Xing Lv
BACKGROUND: Due to improvements in imaging and radiological techniques as well as the use of chemotherapy, distant metastasis has become the predominant mode of treatment failure in patients with locally advanced nasopharyngeal carcinoma (LA-NPC). Platinum-based systemic chemotherapy has shown survival benefits and is now the standard strategy for systemic therapy in patients with LA-NPC. Notably, the third-generation platinum reagent lobaplatin has shown anti-tumor effects in several solid tumors with lower incidences of gastrointestinal, hepatic and renal toxicity relative to other platinum drugs...
February 15, 2017: BMC Cancer
https://www.readbyqxmd.com/read/28199478/evaluation-of-o6-benzylguanine-potentiated-topical-carmustine-for-mycosis-fungoides-a-phase-1-2-clinical-trial
#19
Joselin D Tacastacas, Derek V Chan, Sean Carlson, Stanton L Gerson, Afshin Dowlati, Pingfu Fu, Kurt Lu, Sarah Groft, Julie Rosenjack, Kord Honda, Thomas McCormick, Kevin D Cooper
Importance: In a phase 1 trial, single-dose O6-benzylguanine with topical carmustine for patients with early stage (stage IA through stage IIA) cutaneous T-cell lymphoma, mycosis fungoides (MF) type, resulted in clinical responses proportional to inhibition of O6-alkylguanine DNA alkyltransferase activity, but a maximum tolerated dose (MTD) was not reached. Objective: To determine whether dose escalation of carmustine in combination with dual-dose O6-benzylguanine to prolong alkyltransferase inhibition could reach an MTD...
February 15, 2017: JAMA Dermatology
https://www.readbyqxmd.com/read/28199114/curcumin-loaded-blood-stable-polymeric-micelles-for-enhancing-therapeutic-effect-on-erythroleukemia
#20
Feirong Gong, Dan Chen, Xin Teng, Junhua Ge, Xianfeng Ning, Ya-Ling Shen, Jian Li, Shanfeng Wang
Curcumin has high potential in suppressing many types of cancer and overcoming multi-drug resistance in a multi-faceted manner by targeting diverse molecular targets. However, the rather low systemic bioavailability resulted from its extremely low aqueous solubility and rapid metabolism and excretion in vivo has hampered its application in cancer therapy. With the primary aim to increase the aqueous solubility of curcumin while retaining its stability in the blood circulation before it accesses the tumor site, here we report preparation of curcumin-loaded N-(tert-butoxycarbonyl)-L-phenylalanine end-capped methoxy-poly(ethylene glycol)-block-poly(ε-caprolactone) (mPEG-PCL-Phe(Boc)) micelles with high stability both in vitro and in vivo and anti-tumor efficacy...
February 15, 2017: Molecular Pharmaceutics
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