tbuckley/cabot-search huggingface dataset:
AB0545 Interferon Alpha2a versus Cyclosporin A for The Treatment of Severe Ocular Behcet's Disease – A Prospective, Randomised, Single Blind, National Multicenter Trial (INCYTOB)
Authors: I. Kötter, R. Vonthein, B. Schoenfisch, T. Xenitidis, D. Doycheva, J. Henes, N. Stübiger, E. Feist, A. Heiligenhaus, T. Rath, R. Max, F. Mackensen, A. Altenburg, L. Krause, C. Zouboulis, S. Blaschke, M. Horn, T. Neß, S. Goldacker, C. Deuter
Journal: Annals of the Rheumatic Diseases (2016)

<h3>Background</h3> Behcet`s disease (BD) is a variable vessel vasculitis. To date, there are only retrospective case series showing efficacy of interferon alpha for ocular BD. This trial was started in 2007. <h3>Objectives</h3> To prove that interferon alpha2a is at least as effective as cyclosporin A. <h3>Methods</h3> Maintenance dosage of IFN was 3 to 9 Mill iU 3x/week, CSA dosage 3 to 5 mg/kg, adapted to response every two weeks. In case of nonresponse, the patients were switched to the alternative treatment arm after week six. Efficacy was measured by Posterior Uveitis scoring system (PUS), Behcet`s disease current activity form (BDCAF), Health assessment questionnaire (HAQ) and Beck depression Inventory (BDI) were also used. Primary endpoints were time to remission by PUS and number of cross over from one arm to the other. <h3>Results</h3> The study was stopped in 2013 after 37 patients due to slow recruitment. 13 patients per treatment arm (n=26, 65% male, 35% female, 38% German, 50% Turkish, 12% other, mean age 30.5 years) were evaluated. All 13 patients with primarily IFN achieved remission of their ocular disease, 9 of 13 in the CSA arm (p=0.096). The median time to complete PUS remission was 84 vs. 72 days, p=0.899. The probability to achieve remission after one year was 0.92 for IFN (95% CI 0.49–0.99) and 0.62 for CSA (95%CI 0.24–0.81). There were significantly more switches from CSA to IFN (n=7) than vice versa (n=1) (p=0.030). The main reasons were inefficacy (n=4 for CSA) and/or side effects (n=3 for CSA). PUS of the affected eyes improved significantly in both arms during the first year (p&lt;0.001). Visual acuity at baseline was significantly better in the CSA arm than in the IFN arm, hence a significant improvement over time could only be shown in the IFN arm (p&lt;0.003 week 30). BDCAF improved in both arms (p&gt;0.001), as did HAQ (p=0.047). Safety analysis revealed more hair loss (p=0.004) and cytopenia (p&lt;0.001) in the IFN and hypertension (p=0.048) in the CSA arm. <h3>Conclusions</h3> Both treatments are effective for ocular and extraocular BD. There is a tendency towards superiority of interferon alpha considering that there were more switches from CSA to IFN due to inefficacy and side effects. Due to the small sample size, this did not reach significance. A prospective international multicenter trial may help to overcome these limitations. <h3>Acknowledgement</h3> This trial was supported by the German Ministry of Health (BMBF/DLR, No. 01KG0706) and by Roche (supply of interferon alpha2a) <h3>Disclosure of Interest</h3> None declared
AB0719 The Effectiveness of Therapeutic Strategies To Achieve The Goal (T2T) on The Example of Kazan (Russia) Cohort of Patients with Spondyloarthritis
Authors: S. Lapshina, L. Myasoutova, S. Erdes, R. Mukhina, A. Vasilyev
Journal: Annals of the Rheumatic Diseases (2016)

<h3>Objectives</h3> To rate the effectiveness of therapeutic strategies to achieve the goal (T2T) on the example of Kazan cohort of patients with spondyloarthritis (SpA) compared with routine practice <h3>Methods</h3> The main group of active observation by rheumatologist and regular correction of therapy according to the T2T criteria for spondyloarthritis included 42 patients: 27 (64.3%) with ankylosing spondylitis (AS) - New York criteria, 15 (35.7%) with axial SpA and positive MRI osteitis. High and very high activity (ASDAS) was at 54.8%, moderate - at 45.2% of patients. The comparison group - 40 patients (65% - AS, 35% - axial SpA) observed in the routine practice with a rheumatologist consultation as needed (just about once in 6 months). Patients were comparable for demographic and clinical characteristics, disease activity, duration of symptoms was not more than 10 years, the average 5,1±2,7. All patients received nonsteroidal anti-inflammatory drugs (NSAIDs), if necessary on a long constant reception. At baseline and after 3, 6 and 12 months BASDAI, ASDAS, BASFI, remission criteria (ASAS) were determined, adherence to treatment was evaluated. <h3>Results</h3> During the observation period in two groups a decrease (p&lt;0.05) of mean values BASDAI, BASFI, ASDAS<sub>CRP</sub> was noticed. For the year of observation in the main group 95.2% of patients showed improvement on ASAS40 criteria, partial remission ASAS criteria was reached at 3 months - 47.6% of patients, at 6 months - 73.8%, at 12 months - 80, 9%, periods of exacerbation were observed in 19.1% of patients. In the comparison group - 55% of patients showed improvement on ASAS40 criteria, after 3 months partial remission ASAS criteria was reached in 17.5% of patients, at 6 months - 35%, 12 months - 47.5% patients, periods of exacerbation were observed in 57.5% of patients. The number of patients, regulary performed physical exercises, in the study group was 73.8%, in the control group - 35%. In the study group 76.1% of patients received NSAIDs continuously, in the control group - 37.5% of patients - only on request <h3>Conclusions</h3> The treatment strategy T2T SpA is an effective tool for the management of patients with this pathology, improves adherence to therapy, helps to control disease activity, improves functional status. <h3>Disclosure of Interest</h3> None declared
A Phase I Study of the Farnesyltransferase Inhibitor Tipifarnib in a Week- on Week-Off Dose Schedule in Acute Myelogenous Leukemia.
Authors: Mark Kirschbaum, Anthony Selwyn Stein, Joseph Tuscano, Jasmine M. Zain, Leslie Popplewell, Margaret R. O’Donnell, Chatchada Karanes, Amrita Krishnan, John J. Wright, Bernadette Pulone, Amalia Rincon, Paul Frankel, Stephen J. Forman, Newman Edward
Journal: Blood (2006)
Circadian Corticosterone Levels Regulate and Integrate Hematopoietic Stem and Progenitor Cell Function and Bone Remodeling Via Notch1 Signaling
Authors: Orit Kollet, Yaron Vagima, Karin Golan, Jonathan Canaani, Gabriele D'Uva, Tomer Itkin, Alexander Kalinkovich, Elias Shezen, Adi Neufeld-Cohen, Diana Rashkovan, Alon Chen, Tsvee Lapidot
Journal: Blood (2010)
Active versus passive humidification for self-ventilating tracheostomy and laryngectomy patients: A systematic review of the literature
Authors: C.Y.Y. Wong, A. Shakir, A. Farboud, H. Whittet
Journal: International Journal of Surgery (2016)

Objective
To determine whether active or passive humidification methods are more effective in preventing pulmonary complications in self-ventilating neck breather patients.

Design
Systematic Review adhering to PRISMA guidance (checklist sourced from www.equator-network.org/).

Setting
Review of current published relevant literature at a tertiary department of Otolaryngology and Head & Neck Surgery.

Participants
We included all separate studies and comparison studies of active and passive humidification techniques in adult and paediatric neck breather patients.

Main Outcome Measures
The primary outcome is the reduction in pulmonary complications. Secondary outcomes include patient compliance; carer and user satisfaction.

Results and Conclusions
Seven studies were included in this review: two RCTs (133 patients), one randomised controlled cross-over trial (29 patients), three randomised prospective studies (171 patients), and one retrospective study (73 patients). Only one study was conducted on paediatric neck breathers. The overall quality of the studies was low. Five studies were at a high risk of bias. Of the remaining two studies, one study had a low risk of bias and the other had an unclear risk. Despite limited subject evidence, results show that passive methods of humidification (mainly HME) is the preferred choice of humidification in the spontaneously breathing neck breather patients group mainly due to the reduction in pulmonary complaints, and better patient compliance.
Extreme Variability of FIP1L1-PDGFRalpha Transcripts In CEL: Analysis of 32 Patients Enrolled In HES0203 Italian Clinical Trial and Correlation with Clinical and Molecular Response After 5 Years Follow-up
Authors: Michela Rondoni, Daniela Cilloni, Francesca Arruga, Cristina Papayannidis, Emanuela Ottaviani, Serena Merante, Francesco Buccisano, Sabrina Colarossi, Ilaria Iacobucci, Stefania Paolini, Fabrizio Pane, Giuseppe Saglio, Francesco Lauria, Michele Baccarani, Giovanni Martinelli
Journal: Blood (2010)
Efficacy of Convalescent Plasma in Relation to Dose of Ebola Virus Antibodies
Authors: Johan van Griensven, Tansy Edwards, Sylvain Baize
Journal: New England Journal of Medicine (2016)

lyze germline-derived data from 402 patients with differentiated thyroid carcinoma.We used these data, which were made available by the Cancer Genome Atlas (TCGA) Research Network, 2 to assess the contribution of known cancer-predisposition genes 3 to differentiated thyroid carcinoma.No mutations were detected in PTEN, APC, or WRN.Instead, to our surprise, we identified 23 patients (5.7%) with a truncating mutation in one of five DNA-repair genes: BRCA1 (in 1 patient), ATM (in 1), CHEK2 (in 4), MSH6 (in 4), and BRCA2 (in 13).These findings suggest a role for DNArepair genes in susceptibility to differentiated thyroid carcinoma, but additional deleterious somatic mutations in these genes were not seen in the associated tumors.Studies have identified an excess of differentiated thyroid carcinoma in CHEK2 mutation carriers, 4 but not in BRCA2 heterozygotes. 5 Further investigations to clarify the clinical significance of these findings are warranted.
Targeting Tumor-Associated Fibroblasts for Therapeutic Delivery in Desmoplastic Tumors
Authors: Lei Miao, Qi Liu, C. Michael Lin, Cong Luo, Yuhua Wang, Lina Liu, Weiyan Yin, Shihao Hu, William Y. Kim, Leaf Huang
Journal: Cancer Research (2016)

The off-target distribution of anticancer nanoparticles to fibroblasts creates a barrier to the effective treatment of desmoplastic tumors. However, we hypothesized that this nanoparticle detriment might be exploited to target the expression of secreted cytotoxic proteins from tumor-associated fibroblasts (TAF) as an anticancer strategy. In addressing this hypothesis, plasmids encoding the secretable TNF-related factor sTRAIL were loaded into lipid-coated protamine DNA complexes and administered by infusion in a murine xenograft model of human desmoplastic bladder carcinoma. Three doses were sufficient to generate approximately 70% of TAFs as sTRAIL-producing cells. sTRAIL triggered apoptosis in tumor cell nests adjacent to TAFs. Furthermore, it reverted residual fibroblasts to a quiescent state due to insufficient activation, further compromising tumor growth and remodeling the microenvironment to favor second-wave nanotherapy. We confirmed the efficacy of this strategy in an orthotopic xenograft model of human pancreatic cancer, where the desmoplastic stroma is well known to be a major barrier to the delivery of therapeutic nanoparticles. Collectively, our results offer a proof of concept for the use of nanoparticles to modify TAFs as an effective strategy to treat desmoplastic cancers. Cancer Res; 77(3); 719-31. ©2016 AACR.
Renal Function in Infants with Sickle Cell Anemia: Baseline Data from the BABY HUG Trial.
Authors: Russell E. Ware, Renee C. Rees, Sharada A. Sarnaik, Rathi V. Iyer, Ofelia A. Alvarez, James F. Casella, Barry L. Shulkin, Eglal Shalaby-Rana, C. Frederic Strife, John H. Miller, Peter A. Lane, Winfred C. Wang, Scott T. Miller
Journal: Blood (2008)
Response Assessment After Induction Therapy in Diffuse Large B Cell Lymphoma (DLBCL): Role of Fluorodeoxyglucose-Positron Emission Tomography (FDG-PET) in Comparison with Computed Tomography (CT).
Authors: Benedetta Puccini, Luigi Rigacci, Patrizia Pregno, Annalisa Chiappella, Renato Alterini, Valentina Carrai, Umberto Vitolo, Alberto Bosi
Journal: Blood (2009)
Outcomes of Autologous and Allogeneic BMT for Mantle Cell Lymphoma.
Authors: Yvette L. Kasamon, Richard J. Jones, Louis F. Diehl, Hassan Nayer, Michael J. Borowitz, Elizabeth Garrett-Mayer, Richard F. Ambinder, Ross A. Abrams, Zhe Zhang, Ian W. Flinn
Journal: Blood (2004)
PTH-033 How Good is Emergency Endoscopy? An Observational Study of Technical and Non-Technical Performance and Procedure Quality
Authors: MK Matharoo, A Haycock, N Sevdalis, S Thomas-Gibson
Journal: Gut (2016)

<h3>Introduction</h3> Patients undergoing emergency endoscopy are acutely unwell, often comorbid with higher mortality compared to routine cases. Therapeutic intervention is likely and procedures may be conducted out of hours in less familiar environments. There is a longer learning curve, posing challenges for skill acquisition and retention. NCEPOD identified good decision-making and teamwork as areas for improvement for patient safety in urgent cases. The Upper GI Bleed Toolkit sets quality standards for 24/7 service provision. However, variation exists on when urgent procedures are performed, equipment availability, endoscopy teams, procedure setting and access to senior supervision. This study investigated whether the endoscopist’s performance in technical and non-technical skill is also implicated. <h3>Methods</h3> Aims: To compare performance in urgent/emergency (E) and routine (R) GI endoscopy in a tertiary unit. A prospective observational comparative study was conducted with live ratings of technical skill (DOPS 1–4*) non-technical skill (ENTS 1–4*), degree of completion of a 13 item endoscopy safety checklist (SC) and Patient Safety Incidents (PSI). Over 2 months, consecutive E cases were live rated by an independent trained observer using validated tools. R cases matched for endoscopist experience and procedure were rated within the following 24 h. Wilcoxon and t tests were used to compare paired E and R data and Spearman’s rank was used to examine correlation between the different variables. (*1 = poor 4 = excellent) <h3>Results</h3> 41 E &amp; 41 R cases performed by independent endoscopists on the on-call GIB rota were assessed. All variables were poorer for E compared to R cases (Table). ENTS, DOPS and SC completion were positively correlated. PSI were inversely correlated with ENTS, DOPS and SC completion (Image) <h3>Conclusion</h3> Performance in urgent/emergency endoscopy was significantly poorer than for routine procedures and resulted in more PSI. Different technical and non-technical skills may be required for emergency endoscopy. In these complex higher-risk cases, poorer non-technical skills and suboptimal checklist completion may be negatively impacting technical performance in endoscopists who perform well in the routine setting. These offer targeted training opportunities and performance assessments to enhance safety and quality. <h3>Disclosure of Interest</h3> None Declared
Multilineage Dysplasia Confers Poor Prognosis to Patients with De Novo Acute Myeloid Leukemia with Intermediate-Risk Cytognetics and Wild-Type NPM1.
Authors: Jose Tomas Navarro, Maria Rozman, Leonor Arenillas, Ana Aventin, Teresa Gimenez, Esther Alonso, Granada Perea, Mireia Camós, Mayda Navarrete, Esperanza Tuset, Lourdes Florensa, Fuensanta Millá, Josep F Nomdedeu, Esmeralda de la Banda, Marina Díaz-Beyá, Salut Brunet, Jorge Sierra, Jordi Esteve
Journal: Blood (2012)
Nrf2 Deficiency Leads to Reduced Hematopoietic Stem Cell Self-Renewal and Increased Sensitivity to Genotoxic Stressors Through Impaired P53 Function
Authors: Liang Li, Yin Wei Ho, Ling Li, Qin Huang, Min Li, Stephen J. Forman, Smita Bhatia, Ravi Bhatia
Journal: Blood (2012)
Porphyrin‐Based Carbon Dots for Photodynamic Therapy of Hepatoma
Authors: Yang Li, Xiaohua Zheng, Xiaoyu Zhang, Shi Liu, Qing Pei, Min Zheng, Zhigang Xie
Journal: Advanced Healthcare Materials (2016)

Porphyrin-containing carbon dots (CDs) possess ultrasmall size, excellent water solubility, and photostability. These CDs can effectively generate cytotoxic singlet oxygen upon irradiation, and induce the cell apoptosis. Photodynamic ability of CDs inhibits the growth of hepatoma. This work not only sheds light on developing functional carbon dots, but also highlights the importance of special-structure precursor molecules in synthesizing functional CDs. As a service to our authors and readers, this journal provides supporting information supplied by the authors. Such materials are peer reviewed and may be re-organized for online delivery, but are not copy-edited or typeset. Technical support issues arising from supporting information (other than missing files) should be addressed to the authors. Please note: The publisher is not responsible for the content or functionality of any supporting information supplied by the authors. Any queries (other than missing content) should be directed to the corresponding author for the article.
Salvage Chemotherapy Regimens for Acute Myeloid Leukemia: Is One Better?.
Authors: Samantha L Price, Timothy J. George, Gene A Wetzstein, Jeffrey E Lancet, Alan F. List, Viet Q. Ho, Hugo F. Fernandez, Javier Pinilla-Ibarz, Mohamed A Kharfan-Dabaja, Rami S. Komrokji
Journal: Blood (2009)
Predictive value of therapeutic monitoring with plasma Epstein-Barr virus DNA in Extranodal NK/T cell lymphoma, nasal type
Authors: Chengcheng Guo, He Huang, Ying Tian, Xueying Li, Mengping Zhang, Huangming Hong, Xiaohong Fu, Xiaoting Lin, Shanshan Li, Raj Shrestha Prem, Zhongjun Xia, Tongyu Lin
Journal: Blood (2014)
PWE-086 What Motivates Patients to Enter Clinical Trials Involving Endoscopic or Surgical Treatment? Qualitative Interviews of Patients Approached for The Bride Study (NCT01733719)
Authors: S Budihal, C Jackson, JS deCaestecker
Journal: Gut (2016)

<h3>Introduction</h3> Poor recruitment is an issue for randomised trials. We aimed to explore patient attitudes towards recruitment in an RCT comparing ablative therapies in Barrett’s early neoplasia (BRIDE) &amp; to a proposed trial comparing surgery to endotherapy. <h3>Methods</h3> Patients sampled from among 100 entering/declining BRIDE in each of 6 UK centres, had telephone interviews by an experienced qualitative researcher using a topic guide developed with patients, audio-recorded, anonymised &amp; transcribed verbatim. Transcript analysis used the constant comparative approach, managed by NVivo software. Scrutiny of initial transcripts during 3–4 intensive readings generated open codes (short descriptors summarising points), grouped into themes. Data from other transcripts contradicting the codes/themes were explored &amp; the coding frame revised, resulting in a set of issues important to patients that can help/hinder recruitment and retention. <h3>Results</h3> 18 (16 men, age 47–85) were interviewed. Main findings: 1) Gaining informed consent is time-consuming, but necessary as there is potential for misunderstanding. 2) Some patients received information about BRIDE before results of investigations. Recruitment process should be designed to prevent this. 3) Recruiters need a pleasant attitude, honesty, respect for potential participants, &amp; good interpersonal skills. If not their own doctor, the approach should be from someone suitably qualified &amp; introduced by the doctor/their team. 4) Presence of others during recruitment (eg Macmillan nurse or multiple professionals) may frighten patients into interpreting their diagnosis as serious/terminal. 5) Some believed that treatment had been chosen based on individual need, &amp; others that trial participation was the only route to a particular treatment &amp; better follow up, misunderstanding randomisation. 6) The expectation that taking part could benefit people like themselves or their descendants facilitated successful recruitment. 7) Inconvenience/additional costs to patients and their families should be minimised (e.g. many more visits to hospital). 8) Recruitment was facilitated by awareness that outcomes &amp; side effects of the 2 treatments were roughly equivalent. Surgery was not seen as equivalent, but for most was riskier, involving additional suffering &amp; longer recovery. It was seen as less preferable &amp; would need to offer some probability of advantage over endoscopy. <h3>Conclusion</h3> Communication, openness &amp; trust are key; patients need to understand randomisation does not ensure a particular treatment. Reducing expense/inconvenience assists recruitment. Randomisation to surgery requires clear potential benefits to be acceptable. (NIHR RfPB Grant No PB-PG-0711-25066) <h3>Disclosure of Interest</h3> None Declared
Intraductal cholangioscopy-guided electrohydraulic lithotripsy as a rescue therapy for impacted common bile duct stones within a Dormia basket
Authors: Khalil Aloreidi, Bhavesh Patel, Muslim Atiq
Journal: Endoscopy (2016)

The treatment of bile duct stones has evolved from surgery to endoscopic management, which has a success rate of 90 % [1]. Impaction of the lithotripsy basket during endoscopic removal of a common bile duct (CBD) stones is seen in up to 5.9 % of the cases [2] [3] and represents one of the most feared complications; as it usually results in open surgical intervention.
Single Cell-Level Signaling Profiling of Acute Myeloid Leukemia Following Treatment with Axl Kinase Inhibitor BGB324
Authors: Monica Hellesøy, Katarzyna Wnuk-Lipinska, Anna Boniecka, Eline Milde Nævdal, Hakon Reikvam, Sonja Loges, Jorge E. Cortes, Jørn Skavland, Øystein Bruserud, Jim Lorens, David Micklem, Gro Gausdal, Bjorn T. Gjertsen
Journal: Blood (2015)
Frequent Loss of HLA Alleles From Hematopoietic Stem Cells in Patients with Hepatitis-Associated Aplastic Anemia
Authors: Takamasa Katagiri, Aiko Sato-Otsubo, Koichi Kashiwase, Satoko Morishima, Yusuke Sato, Yuka Mori, Motohiro Kato, Masashi Sanada, Yasuo Morishima, Shigeki Ohtake, Seishi Ogawa, Shinji Nakao
Journal: Blood (2011)
High Frequency of Aberrant Phenotype in Multiple Myeloma Patients Detected by Multiparametric Flow Cytometry.
Authors: Luiz Arthur Calheiros, Eliza Y.S. Kimura, Manuella S.S. Almeida, Maria de Lourdes L.F. Chauffaille, Jandey G. Bigonha, Alex F. Sandes, Wolney G. Barreto, Carolina L. Escaramuzi, Gisele W.B. Colleoni, Mihoko Yamamoto
Journal: Blood (2004)
A Single Institution Analysis of Incidence of Recurrent Chromosomal Abnormalities in Adult ALL Patients According to Race, Gender, and Age
Authors: Victoria Chiou, Jacob Tiegs, Scott Isom, Mark J. Pettenati, Susan Lyerly, Leslie R. Ellis, Timothy Pardee, Berenzon Dmitriy, Bayard L. Powell, Denise A Levitan
Journal: Blood (2011)
Bortezomib Resistance Is Mediated by Increased Signaling through the Insulin-Like Growth Factor-1/Akt Axis.
Authors: Deborah J. Kuhn, Chad C. Bjorklund, Valeria Magarotto, Jairo A. Matthews, Michael Wang, Veerabhadran Baladandayuthapani, Robert Z. Orlowski
Journal: Blood (2009)
Teledermatology as a tool to improve access to care for medically underserved populations: A retrospective descriptive study
Authors: Erica R. Leavitt, Stephen Kessler, Stacey Pun, Tania Gill, Loraine A. Escobedo, Myles Cockburn, Adam Sutton, Ashley B. Crew
Journal: Journal of the American Academy of Dermatology (2016)
SP0110 What Makes An Effective Mentor and An Effective Mentee?
Authors: J. Hafler
Journal: Annals of the Rheumatic Diseases (2016)

Mentoring is a key teaching skill that is considered to be a core component of the duties of all medical school faculty. Effective mentoring requires understanding the needs of the mentee. How do we learn how to be an effective mentor? How we we learn how to be an effective mentee? As Daloz (1986) said "I resolved to look more closely at what good teachers and mentors actually do as they guide their charges along on their transformational journeys." In this session I will explore what good mentors and good mentees can do toward achieving successful outcomes and address specific characteristics of effective mentors. This session will be of interest to mentors and mentees in research and clinical areas. <h3>Disclosure of Interest</h3> None declared