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https://www.readbyqxmd.com/read/28087591/clinical-trials-in-antineutrophil-cytoplasmic-antibody-associated-vasculitis-what-we-have-learnt-so-far-and-what-we-still-have-to-learn
#1
Divi Cornec, Emilie Cornec-Le Gall, Ulrich Specks
The prognosis of the antineutrophil cytoplasmic antibody associated vasculitides (AAV), microscopic polyangiitis (MPA), granulomatosis with polyangiitis (GPA) and eosinophilic GPA (EGPA), has been fundamentally improved over the last five decades by the use of glucocorticoids and immunosuppressants, turning them from consistently fatal diseases into chronic conditions. The long-term course is now largely determined by the frequency of disease flares and by accruing damage caused by disease activity and treatment-related comorbidities...
January 12, 2017: Nephrology, Dialysis, Transplantation
https://www.readbyqxmd.com/read/28079584/the-development-and-implementation-of-cognitive-aids-for-critical-events-in-pediatric-anesthesia-the-society-for-pediatric-anesthesia-critical-events-checklists
#2
Anna Clebone, Barbara K Burian, Scott C Watkins, Jorge A Gálvez, Justin L Lockman, Eugenie S Heitmiller
Cognitive aids such as checklists are commonly used in modern operating rooms for routine processes, and the use of such aids may be even more important during critical events. The Quality and Safety Committee of the Society for Pediatric Anesthesia (SPA) has developed a set of critical-event checklists and cognitive aids designed for 3 purposes: (1) as a repository of the latest evidence-based and expert opinion-based information to guide response and management of critical events, (2) as a source of just-in-time information during critical events, and (3) as a method to facilitate a shared understanding of required actions among team members during a critical event...
January 10, 2017: Anesthesia and Analgesia
https://www.readbyqxmd.com/read/28078669/maintenance-phase-in-puva-phototherapy-of-early-stage-mycosis-fungoides-a-critically-appraised-topic
#3
REVIEW
V Grandi, C Delfino, A Pileri, N Pimpinelli
A 65-years-old patient affected by mycosis fungoides stage IB achieved complete remission after a cycle of PUVA phototherapy. As suggested by USCLC guidelines(1) , the patient should be kept in maintenance phase, defined as "period of gradual decrease of frequency of UVL while in clinical remission before discontinuation of phototherapy" by slowly tapering the number of PUVA applications over time up to clinical relapse. USCLC also suggest a standardized schedule for maintenance phase (table 1). Alternatively, the patient could end PUVA therapy and go straight to follow up...
January 12, 2017: British Journal of Dermatology
https://www.readbyqxmd.com/read/28078315/degenerative-ataxias-challenges-in-clinical-research
#4
REVIEW
Sub H Subramony
The degenerative ataxias are a very heterogeneous group of disorders that include numerous genetic diseases as well as apparently "sporadic" entities. There has been an explosion of discoveries related to genetic defects and related pathomechanisms that has brought us to the threshold of meaningful therapies in some but not all of these diseases. There also continues to be lack of knowledge of the causation of disease in a sizeable proportion of these patients. The overall rarity of ataxias as a whole and certainly of the individual genetic entities together with slow and variable progression and variable prognosis in juxtaposition with a rapid development of possible therapies in the horizon such as gene replacement and gene knock-down strategies places the ataxias in a unique position distinct from other similar neurodegenerative diseases...
January 2017: Annals of Clinical and Translational Neurology
https://www.readbyqxmd.com/read/28073682/study-design-for-control-of-heart-rate-in-infant-and-child-tachyarrhythmia-with-heart-failure-using-landiolol-heartful-a-prospective-multicenter-uncontrolled-clinical-trial
#5
Naokata Sumitomo, Hitoshi Horigome, Masaru Miura, Hiroshi Ono, Hideaki Ueda, Kiyohiro Takigiku, Jun Yoshimoto, Naoki Ohashi, Tsugutoshi Suzuki, Koichi Sagawa, Hiroya Ushinohama, Kazuhiro Takahashi, Aya Miyazaki, Heima Sakaguchi, Mari Iwamoto, Motoki Takamuro, Chiho Tokunaga, Tetsuji Nagano
BACKGROUND: Persistent tachycardia in pediatric patients after congenital heart surgery further deteriorates their hemodynamic condition, and may become fatal. Therefore, immediate control of the tachycardia is mandatory in these patients. For this purpose, quick-acting, short-acting, titratable intravenous agents are required. However, there are no agents with such characteristics among the drugs approved for control of pediatric arrhythmias in Japan, and thus novel and effective medications for these patients are awaited...
January 7, 2017: Journal of Cardiology
https://www.readbyqxmd.com/read/28072797/effect-of-concomitant-pain-medications-on-response-to-pregabalin-in-patients-with-postherpetic-neuralgia-or-spinal-cord-injury-related-neuropathic-pain
#6
Stephan A Schug, Bruce Parsons, Mary Almas, Ed Whalen
BACKGROUND: Patients with neuropathic pain (NeP) often receive combination therapy with multiple agents in the hopes of improving both pain and any comorbidities that may be present. While pregabalin is often recommended as a first-line treatment of NeP, few studies have examined the effects of concomitant medications on the efficacy of pregabalin. OBJECTIVE: To examine the effects of concomitant medications on the efficacy and safety of pregabalin for the treatment of NeP...
January 2017: Pain Physician
https://www.readbyqxmd.com/read/28068461/a-new-prediction-model-for-evaluating-treatment-resistant-depression
#7
Alexander Kautzky, Pia Baldinger-Melich, Georg S Kranz, Thomas Vanicek, Daniel Souery, Stuart Montgomery, Julien Mendlewicz, Joseph Zohar, Alessandro Serretti, Rupert Lanzenberger, Siegfried Kasper
OBJECTIVE: Despite a broad arsenal of antidepressants, about a third of patients suffering from major depressive disorder (MDD) do not respond sufficiently to adequate treatment. Using the data pool of the Group for the Study of Resistant Depression and machine learning, we intended to draw new insights featuring 48 clinical, sociodemographic, and psychosocial predictors for treatment outcome. METHOD: Patients were enrolled starting from January 2000 and diagnosed according to DSM-IV...
January 3, 2017: Journal of Clinical Psychiatry
https://www.readbyqxmd.com/read/28067166/stem-cell-genetic-therapy-for-fanconi-anemia-a-new-hope
#8
Helmut Hanenberg, Katharina Roellecke, Constanze Wiek
Fanconi anemia (FA) is a rare inherited DNA disorder clinically characterized by congenital malformations, progressive bone marrow failure, and cancer susceptibility. Due to a strong survival advantage of spontaneously corrected 'normal' hematopoietic stem cells (HSCs) in a few patients, FA is considered a model disorder for genetic correction of autologous stem cells, where genetically corrected stem cells and their progeny have a strong in vivo selective advantage, ultimately leading to normal hematopoiesis...
January 9, 2017: Current Gene Therapy
https://www.readbyqxmd.com/read/28067165/gene-therapy-in-fanconi-anemia-a-matter-of-time-safety-and-gene-transfer-tool-efficiency
#9
Verhoeyen Els, Francisco José Román Rodríguez, François-Loïc Cosset, Camille Lévy, Paula Rio
Fanconi anemia (FA) is a rare genetic syndrome characterized by progressive marrow failure. Gene therapy by infusion of FA-corrected autologous hematopoietic stem cells (HSCs) may offer a potential cure since it is a monogenetic disease with mutations in the FANC genes, coding for DNA repair enzymes (See review[1]). However, the collection of hCD34 +-cells in FA patients implies particular challenges because of the reduced numbers of progenitor cells present in their bone marrow (BM)[2] or mobilized peripheral blood[3-5]...
January 9, 2017: Current Gene Therapy
https://www.readbyqxmd.com/read/28067079/mglu5-negative-allosteric-modulators-a-patent-review-2013-2016
#10
Kyle A Emmitte
The pursuit of small molecule mGlu5 NAMs as treatments for a variety of psychiatric and neurodegenerative disorders has developed into a mature field. In addition to extensive preclinical studies, multiple compounds have advanced into clinical trials with the most advanced studies occurring in patients with FXS, PD-LID, and MDD. Areas covered: This review begins with an update of the clinical activity with mGlu5 NAMs, and then moves into a summary of patent applications filed since 2013. The summaries are organized into three separate sections: (1) inventions centered on improvements to existing clinical compounds; (2) new small molecules that maintain the prototypical disubstituted alkyne chemotype found in many mGlu5 NAM compounds; and (3) new small molecules that are not from a disubstituted alkyne chemotype...
January 8, 2017: Expert Opinion on Therapeutic Patents
https://www.readbyqxmd.com/read/28066606/lobectomy-and-limited-resection-in-small-sized-peripheral-non-small-cell-lung-cancer
#11
Terumoto Koike, Teruaki Koike, Seijiro Sato, Takehisa Hashimoto, Tadashi Aoki, Katsuo Yoshiya, Yasushi Yamato, Takehiro Watanabe, Kohei Akazawa, Shin-Ichi Toyabe, Masanori Tsuchida
BACKGROUND: Although lobectomy is the standard surgical procedure for non-small cell lung cancer (NSCLC), recent studies show favorable outcomes after limited resection in patients with small-sized peripheral tumors. We conducted a randomized controlled trial of such patients to estimate postoperative outcomes and pulmonary function following these surgical techniques. METHODS: Between 2005 and 2008, eligible patients with tumors of 2 cm or less were randomly assigned 1:1 to undergo lobectomy or limited resection; 32 and 33 NSCLC patients in each group, respectively, were analyzed...
November 2016: Journal of Thoracic Disease
https://www.readbyqxmd.com/read/28065636/neuroprotection-and-neurotoxicity-in-the-developing-brain-an-update-on-the-effects-of-dexmedetomidine-and-xenon
#12
Azeem Alam, Ka Chun Suen, Zac Hana, Robert D Sanders, Mervyn Maze, Daqing Ma
Growing and consistent preclinical evidence, combined with early clinical epidemiological observations, suggest potentially neurotoxic effects of commonly used anesthetic agents in the developing brain. This has prompted the FDA to issue a safety warning for all sedatives and anesthetics approved for use in children under three years of age. Recent studies have identified dexmedetomidine, the potent α2-adrenoceptor agonist, and xenon, the noble gas, as effective anesthetic adjuvants that are both less neurotoxic to the developing brain, and also possess neuroprotective properties in neonatal and other settings of acute ongoing neurologic injury...
January 5, 2017: Neurotoxicology and Teratology
https://www.readbyqxmd.com/read/28065262/analyzing-amyloid-%C3%AE-peptide-modulation-profiles-and-binding-sites-of-%C3%AE-secretase-modulators
#13
J Trambauer, A Fukumori, B Kretner, H Steiner
γ-Secretase is a key player in the pathogenesis of Alzheimer's disease (AD). The intramembrane-cleaving enzyme initially cleaves a C-terminal fragment of the amyloid precursor protein (APP) at the ɛ-site within its transmembrane domain to release the APP intracellular domain. Subsequent stepwise carboxy-terminal trimming cleavages eventually release amyloid-β (Aβ) peptides of 37-43 amino acids into the extracellular space. Aβ42 as well as the much less abundant Aβ43 species are highly aggregation prone and can deposit as plaques in the brains of affected patients, which are widely believed to be causative of AD...
2017: Methods in Enzymology
https://www.readbyqxmd.com/read/28064388/stem-cell-recipes-of-bone-marrow-and-fish-just-what-the-stroke-doctors-ordered
#14
REVIEW
Eleonora Napoli, Cesar V Borlongan
Stem cell therapy for stroke has advanced from the laboratory to the clinic, but remains as an experimental treatment. Two lines of transplant regimens have emerged, namely the "early bird" peripheral injections in subacute stroke patients and the "late night" direct intracerebral treatments in chronic stroke patients. Autologous bone marrow-derived stem cells, which only required minimal manipulations during graft cell preparation, gained fast-track entry into the clinic, while gene modified stem cells necessitated overcoming more stringent regulatory criteria before they were approved for clinical use...
January 7, 2017: Stem Cell Reviews
https://www.readbyqxmd.com/read/28062555/antibody-therapeutics-targeting-a%C3%AE-and-tau
#15
Gilbert Gallardo, David M Holtzman
The astonishing findings that active and passive immunization against amyloid-β (Aβ) in mouse models of Alzheimer's disease (AD) dramatically decreased amyloid burden led to a rapid initiation of human clinical trials with much enthusiasm. However, methodological issues and adverse effects relating to these clinical trials arose, challenging the effectiveness and safety of these reagents. Efforts are now underway to develop safer immunotherapeutic approaches toward Aβ and the treatment of individuals at risk for AD before or in the earliest stages of cognitive decline with new hopes...
January 6, 2017: Cold Spring Harbor Perspectives in Medicine
https://www.readbyqxmd.com/read/28062025/amniotic-fluid-technical-update-on-physiology-and-measurement
#16
Kenneth I Lim, Kimberly Butt, Kentia Naud, Mila Smithies
OBJECTIVE: OUTCOMES: EVIDENCE: A MEDLINE and KFINDER search was used to identify relevant articles, with review of bibliography identified article including Cochrane reviews and recent review articles. VALUES: The evidence collected was reviewed by the Diagnostic Imaging Committee of the Society of Obstetricians and Gynecologists of Canada. The recommendations were made according to the guidelines developed by The Canadian Task Force on Preventative Health Care (Table 1)...
January 2017: Journal of Obstetrics and Gynaecology Canada: JOGC, Journal D'obstétrique et Gynécologie du Canada: JOGC
https://www.readbyqxmd.com/read/28061482/genomic-mutation-driven-metastatic-breast-cancer-therapy-a-single-center-experience
#17
Yuan Yuan, Susan E Yost, Yate-Ching Yuan, Nicola M Solomon, Isa Mambetsariev, Sumanta Pal, Paul Frankel, Ravi Salgia, Susan L Neuhausen, Joanne Mortimer
BACKGROUND: Next-Generation Sequencing (NGS) has made genomic mutation-driven therapy feasible for metastatic breast cancer (MBC) patients. We frequently submit tumor tissue from MBC patients for targeted NGS of tumor using the Illumina HiSeq 2000 platform (FoundationOne®, Foundation Medicine, MA). Herein, we report the results and clinical impact of this test in MBC patients. PATIENTS AND METHODS: We identified patients with MBC treated at City of Hope from January 2014 to May 2016 who underwent NGS...
January 3, 2017: Oncotarget
https://www.readbyqxmd.com/read/28059608/near-future-of-tumor-immunology-anticipating-resistance-mechanisms-to-immunotherapies-a-big-challenge-for-clinical-trials
#18
João Paulo Portela Catani, Sandy Adjemian, Rachel P Riechelmann, Bryan E Strauss
The success of immunotherapies brings hope for the future of cancer treatment. Even so, we are faced with a new challenge, that of understanding which patients will respond initially and, possibly, develop resistance. The examination of the immune profile, especially approaches related to the immunoscore, may foretell which tumors will have a positive initial response. Ideally, the mutation load would also be analysed, helping to reveal tumor associated antigens that are predictive of an effective cytolytic attack...
January 6, 2017: Human Vaccines & Immunotherapeutics
https://www.readbyqxmd.com/read/28055269/targeting-pd-1-pathway-a-new-hope-for-gastrointestinal-cancers
#19
Burak Bilgin, Mehmet An Sendur, Muhammed Bülent Akıncı, Didem Şener Dede, Bülent Yalçın
BACKGROUND: VEGF, HER2 and EGFR targeted agents are currently used in gastric, esophageal and colorectal cancers. However, treatment outcomes are still poor in most of the Gastrointestinal (GI) cancers. Immune checkpoints are one of the most promising immunotherapy approaches. In this review article, we aimed to discuss the efficacy and safety of anti-PD-1/PD-L1 therapies in GI cancers, including gastric, esophageal and colorectal cancer in the era of published or reported recent studies...
January 5, 2017: Current Medical Research and Opinion
https://www.readbyqxmd.com/read/28051882/are-sigma-modulators-an-effective-opportunity-for-cancer-treatment-a-patent-overview-1996-2016
#20
Simona Collina, Emanuele Bignardi, Marta Rui, Daniela Rossi, Raffaella Gaggeri, Alice Zamagni, Michela Cortesi, Anna Tesei
Although several molecular targets against cancer have been identified, there is a continuous need for new therapeutic strategies. Sigma Receptors (SRs) overexpression has been recently associated to different cancer conditions. Therefore, novel anticancer agents targeting SRs may increase the specificity of therapies, overcoming some of the common drawbacks of conventional chemotherapy. Areas covered: The present review focuses on patent documents disclosing SR modulators with possible application in cancer therapy and diagnosis...
January 4, 2017: Expert Opinion on Therapeutic Patents
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