scholarly journals Open-label, add-on trial of cetirizine for neuromyelitis optica

2018 ◽  
Vol 5 (2) ◽  
pp. e441 ◽  
Author(s):  
Ilana Katz Sand ◽  
Michelle T. Fabian ◽  
Russell Telford ◽  
Thomas A. Kraus ◽  
Mirna Chehade ◽  
...  
Keyword(s):  
Pilot Study ◽  
Neuromyelitis Optica ◽  
Standard Therapy ◽  
Small Sample Size ◽  
Small Sample ◽  
Open Label ◽  
End Point ◽  
Disease Modifying Therapy ◽  
Significant Difference ◽  
Disease Modifying

ObjectiveThis pilot study preliminarily examined the efficacy and tolerability of cetirizine as an add-on to standard therapy for neuromyelitis optica (NMO).MethodsEligible participants met the Wingerchuk 2006 diagnostic criteria or had a single typical episode along with positive NMO immunoglobulin G. After baseline clinical and laboratory assessments, participants began treatment with cetirizine 10 mg orally daily, in addition to their usual disease-modifying therapy for NMO, and continued for 1 year. The primary end point was the annualized relapse rate (ARR) while on the same disease-modifying therapy before starting cetirizine compared with after taking cetirizine. Additional end points included disability (Expanded Disability Status Scale [EDSS]), relapse severity, tolerability, especially with respect to drowsiness measured by the Epworth Sleepiness Scale (ESS), and laboratory parameters.ResultsThe ARR before cetirizine was 0.4 ± 0.80 and after cetirizine was 0.1 ± 0.24 (p = 0.047). There was no statistically significant difference in the EDSS (mean 3.9 ± 2.18 before the start of the study and 3.2 ± 2.31 at the conclusion of the study, p = 0.500). The ESS remained fairly consistent throughout the study (mean 6.5 ± 5.33 at baseline and 6.9 ± 4.50 at month 12, p = 0.740). Laboratory studies were unrevealing.ConclusionsIn this pilot study, cetirizine was well tolerated, and the prespecified primary efficacy end point was satisfied. However, the open-label design and the small sample size of this pilot study preclude definitive conclusions. Further research is needed.Classification of evidenceThis study provides Class IV evidence that in patients with NMO, the addition of cetirizine to standard therapy is safe, well tolerated, and reduces relapses.

Using intra-articular allogenic lyophilized growth factors in primary knee osteoarthritis: a randomized pilot study

2021 ◽  
Vol 16 (2) ◽  
pp. 113-115
Author(s):  
Rasmia El-Gohary ◽  
Amany Diab ◽  
Hala El-Gendy ◽  
Hossam Fahmy ◽  
Kamel Heshmat Gado
Keyword(s):  
Growth Factors ◽  
Pilot Study ◽  
Knee Osteoarthritis ◽  
Small Sample Size ◽  
Intervention Group ◽  
Small Sample ◽  
Injection Pain ◽  
Post Injection ◽  
Open Label ◽  
Clinical Trial Registration

Objective: Investigating the safety in addition to clinical and structural efficacy of allogenic lyophilized growth factors (L-GFs) in patients with symptomatic primary knee osteoarthritis. Design: A prospective, open-label pilot study. A total of 31-patients randomized into non-intervention and intervention groups. Materials & methods: The intervention group received two intra-articular doses at baseline and after 2-months. Post-injection complications were documented, and the efficacy was assessed by Western Ontario and McMaster Universities Osteoarthritis Index scores and ultrasonography. Results: One dropout from the intervention group. The percentage of improvement of mean Western Ontario and McMaster Universities Osteoarthritis Index-scores and ultrasonography-detected effusion were statistically significant in the intervention group compared with the non-intervention. A brief, mild, post-injection pain was reported by all intervention group. Conclusion: This study provides the safety of intra-articular injection of allogenic L-GFs in knee osteoarthritis. The conclusion of efficacy was limited by small sample size and lack of control injection. Clinical trial registration: NCT04331327 (ClinicalTrials.gov, retrospectively registered).

scholarly journals The Symbiotic Mutualism between Co-Creation and Entrepreneurship

2021 ◽  
Vol 13 (11) ◽  
pp. 6285
Author(s):  
Sandra Misiak-Kwit ◽  
Małgorzata Wiścicka-Fernando ◽  
Kelaniyage Shihan Dilruk Fernando
Keyword(s):  
Pilot Study ◽  
Small Sample Size ◽  
Strong Association ◽  
Sampling Technique ◽  
Small Sample ◽  
Entrepreneurial Intentions ◽  
Added Value ◽  
Primary Data ◽  
Exploratory Research ◽  
Symbiotic Mutualism

In this manuscript, the authors aim to explore firstly the association between entrepreneurial mindset and co-creation experience, secondly the association between co-creation experience and entrepreneurial intentions, and thirdly the association between entrepreneurial mindset and entrepreneurial intentions within the sustainability context. In this paper, the authors present the results of the pilot study. Primary data were collected from 500 university students from China, Georgia, Poland, Romania, and Sri Lanka by using a convenient sampling technique, and a literature review was the primary method of the concept development. The authors selected the above-mentioned countries to collect primary data by using a convenient sampling technique based on accessibility; they also visited all analysed countries in order to conduct the pilot survey personally. Descriptive statistics and the Spearman’s rank correlation coefficient were applied as primary statistical methods. The findings reveal that there is a very strong association between co-creation experience and entrepreneurial intentions, a very weak negative association between entrepreneurial mindset and co-creation experience, and, surprisingly, a weak association between entrepreneurial mindset and entrepreneurial intentions. The added value of the conducted pilot research involves filling in a gap regarding the relationship between experience and the subjective norm. In the presented pilot research, co-creation experience was compared with not only entrepreneurial mindset but with entrepreneurial intentions as well. An additional value of this exploratory research is compiling an international comparison. The main contribution of this pilot study is examining the symbiotic mutualism between co-creation and entrepreneurship. Among many platforms of associations, the following can be differentiated: creativity, innovativeness, openness, engagement, awareness, motivation, trust (level of social capital), and recognizing the significance of social and sustainable development objectives. Due to the small sample size, the results cannot be generalised. Results refer only to the respondents. However, the findings of the pilot study are the basis for further research studies on symbiotic mutualism between entrepreneurship and co-creation.

scholarly journals Blinding in electric current stimulation in subacute neglect patients with current densities of 0.8 A/m2: a cross-over pilot study

2021 ◽  
Vol 14 (1) ◽  
Author(s):  
Anna Gorsler ◽  
Ulrike Grittner ◽  
Nadine Külzow ◽  
Torsten Rackoll
Keyword(s):  
Pilot Study ◽  
Electric Fields ◽  
Standard Care ◽  
Small Sample Size ◽  
Small Sample ◽  
Related Factors ◽  
Attentional Deficits ◽  
Age Related ◽  
Current Densities ◽  
Improve Standard

Abstract Objective Neglect after stroke is a disabling disorder and its rehabilitation is a major challenge. Transcranial direct current stimulation (tDCS) seems to be a promising adjuvant technique to improve standard care neglect therapy. Since electric fields are influenced by age-related factors, higher current densities are probably needed for effective treatment in aged stroke patients. Validation of treatment efficacy requires sham-controlled experiments, but increased current densities might comprise blinding. Therefore, a pilot study was conducted to test sham adequacy when using current density of 0.8 A/m2. Whether especially neglect patients who mainly suffer from perceptual and attentional deficits are able to differentiate beyond chance active from sham tDCS was investigated in a randomized cross-over design (active/sham stimulation) in 12 early subacute patients with left-sided hemineglect. Stimulation (0.8 A/m2) was performed simultaneous to standard care neglect therapy. Results Odds ratio of correct guessing an atDCS condition compared to wrongly judge an atDCS condition as sham was 10.00 (95%CI 0.65–154.40, p = 0.099). However, given the small sample size and high OR, although likely somewhat overestimated, results require careful interpretation and blinding success in neglect studies with current densities of 0.8 A/m2 should be further confirmed.

scholarly journals NI-19 Use of 11C-methionine PET for decision of discontinuation of adjuvant chemotherapy with temozolomide

2020 ◽  
Vol 2 (Supplement_3) ◽  
pp. ii14-ii14
Author(s):  
Takaaki Beppu ◽  
Yuichi Sato ◽  
Toshiaki Sasaki ◽  
Kazunori Terasaki ◽  
Kuniaki Ogasawara
Keyword(s):  
Adjuvant Chemotherapy ◽  
Small Sample Size ◽  
Standardized Uptake Value ◽  
Progression Free Survival ◽  
Residual Tumor ◽  
Small Sample ◽  
Tumor Type ◽  
Diffuse Astrocytoma ◽  
Significant Difference ◽  
Met Pet

Abstract Background: The aim was to clarify whether positron emission tomography with 11C-methyl-L-methionine (met-PET) is useful to decide on discontinuation of TMZ-adjuvant therapy in patients with residual diffuse astrocytic tumor. Methods: Subjects were 44 patients with residual tumor comprising 17 with IDH1-mutant diffuse astrocytoma (DA), 13 with IDH1-mutant anaplastic astrocytoma (AA), and 14 with IDH1-wild glioblastoma (GB). All patients received TMZ-adjuvant chemotherapy (median, 12 courses), and whether to discontinue or continue TMZ-adjuvant chemotherapy was decided on the basis of the tumor-to-normal ratio in standardized uptake value from met-PET (T/N); patients with T/N < 1.6 immediately discontinued TMZ, and patients with T/N > 1.6 were either to continued or discontinued TMZ. Progression-free survival (PFS) was compared between patients with T/N > 1.6 and T/N < 1.6 in each tumor type. Median observation period was 434 days after met-PET scanning. Results: The number of patient who underwent recurrence was 10 in DA, 7 in AA, and 11 in GB. All patients showing T/N > 1.6 underwent tumor recurrence. PFS was significantly longer in patients with T/N < 1.6 than T/N > 1.6 in DA and AA (p < 0.01 in both types), but was no significant difference between 2 groups in GB (p = 0.06). Sixteen of 17 patients (94%) in DA and AA showed recurrence from residual tumor, whereas 4 of 11 patients (36%) in GB showed recurrent tumor at remote regions which were different from residual tumor. Conclusions: The present study suggested that met-PET is beneficial to decide to discontinue adjuvant chemotherapy with TMZ in patients with residual tumors of DA and AA, but not useful for patients with GB. Reasons for unsuccessful results in GB might have been small sample size, failure of establishing the cut off value in T/N, recurrences at remote regions where not be assessed by met-PET.

Perceptions and Understanding of Diabetes Mellitus Technology in Adults with Type 1 or Type 2 DM: A Pilot Survey from Pakistan

2021 ◽  
pp. 193229682110111
Author(s):  
Sarah Nadeem ◽  
Uswah Siddiqi ◽  
Russell Seth Martins ◽  
Kaleemullah Badini
Keyword(s):  
Diabetes Mellitus ◽  
Pilot Study ◽  
Diabetes Care ◽  
Online Survey ◽  
Small Sample Size ◽  
Developed Countries ◽  
Small Sample ◽  
Cost Of Care ◽  
Cross Sectional ◽  
Specific Patient

Introduction: Diabetes mellitus technology (DMT) is increasingly used for routine management in developed countries, yet its uptake in developing countries is not as consistent. Multiple factors may influence this, including country specific patient perception regarding DMT. We conducted a pilot study in Pakistan to understand this important question which has not been studied yet. Methods: A cross-sectional pilot study was conducted in Pakistan. An anonymous survey exploring perceptions of diabetes technology was circulated on social media platforms, collecting responses over 2 weeks. Target population included adults (≥18 years) living in Pakistan, with DM1 or 2. Results: A total of 40 responses were received. The majority (36/40) reported using conventional glucometers. Nine used continuous glucose monitoring (CGM). Thirty-two of 40 patients believed DMT improved diabetes care, 22 felt it helped decreased risk of Diabetes-related complications. 15/40 stated that DMT results in increased cost of care. Sixteen reported their diabetes care teams had never discussed wearable DMT options whereas 11 disliked them because they did not want a device on their self. Conclusion: In our pilot study we have identified broad themes of opportunity and challenges to DMT use in Pakistan. Patients’ perceptions regarding DMT were generally positive but significant barriers to its acceptance included high cost, lack of discussion between doctor and patient about available technology and personal hesitation. Limitations of our study include sampling bias (online survey) and small sample size, but this data can help inform larger studies, to look at this important topic in greater detail.

scholarly journals 1611. Evaluating Clinical Outcomes and Efficacy of Daptomycin in Combination with a Beta-Lactam for the Treatment of Vancomycin-Resistant Enterococcus (VRE) Bacteremia

2020 ◽  
Vol 7 (Supplement_1) ◽  
pp. S799-S800
Author(s):  
Nerea Irusta ◽  
Ana Vega ◽  
Yoichiro Natori ◽  
Lilian M Abbo ◽  
Lilian M Abbo ◽  
...  
Keyword(s):  
Clinical Outcomes ◽  
Primary Outcome ◽  
Small Sample Size ◽  
Univariate Analysis ◽  
Small Sample ◽  
Significant Difference ◽  
Vancomycin Resistant ◽  
Secondary Outcomes ◽  
Related Mortality

Abstract Background In-vitro studies have shown synergistic bactericidal activity with daptomycin (DAP) plus β-lactam antimicrobials against vancomycin resistant enterococci (VRE). There is a paucity of data regarding clinical outcomes with this combination in VRE bloodstream infections (BSI). The purpose of this study was to assess the efficacy of DAP plus a β-lactam with in-vitro activity vs. other therapies for treatment of VRE BSI. Methods IRB-approved, single-center, retrospective study of patients with VRE BSI from 01/2018-09/2019. Patients were excluded if < 18 years old, pregnant, or incarcerated. The primary outcome was time-to-microbiological clearance. Secondary outcomes included infection-related mortality, 30-day all-cause mortality, and incidence of recurrent BSI within 30 days of index culture. Targeted DAP doses were ≥ 8mg/kg and based on MIC. Factors associated with significance for outcomes, via univariate analysis, were evaluated with multivariable logistic regression (MLR), removed in a backward-step approach. Results A total of 85 patients were included, 23 of which received DAP plus a β-lactam. The comparator arm included linezolid or DAP monotherapy. Patients with combination therapy had significantly higher Charlson Comorbidity Index (CCI) (p=0.013) and numerically higher Pitt Bacteremia scores (PBS) (p=0.087) (Table 1). There was no difference seen in the primary outcome (Table 2). Secondary outcomes are provided in Table 2. The presence of polymicrobial infection and higher PBS were significantly associated with infection-related mortality (p=0.008 and p=0.005, respectively) by MLR. A Mann Whitney U test indicated that presence of infection-related mortality was greater for patients with higher MICS (U=20.5, p=0.06). The presence of an underlying source may be related to recurrence of BSI (p=0.075). Table 1: Patient Characteristics Table 2. Primary and Secondary Outcomes Conclusion We did not find a significant difference in time-to-microbiological clearance, although patients treated with DAP and a β-lactam had higher CCI and PBS. These results are limited by retrospective design, small sample size, and potential selection bias. Prospective randomized studies are needed to further validate these findings. Disclosures All Authors: No reported disclosures

scholarly journals Perioperative Outcomes Following Kidney-Pancreas Transplantation in Alberta, Canada: Research Letter

2021 ◽  
Vol 8 ◽  
pp. 205435812110293
Author(s):  
Danielle E. Fox ◽  
Robert R. Quinn ◽  
Paul E. Ronksley ◽  
Tyrone G. Harrison ◽  
Hude Quan ◽  
...  
Keyword(s):  
Patient Outcomes ◽  
Postoperative Care ◽  
Pancreas Transplantation ◽  
Type I Diabetes ◽  
Small Sample Size ◽  
Small Sample ◽  
Perioperative Outcomes ◽  
Type I ◽  
Patient Safety Indicators ◽  
Significant Difference

Background: Simultaneous kidney-pancreas transplantation (SPK) has benefits for patients with kidney failure and type I diabetes mellitus, but is associated with greater perioperative risk compared with kidney-alone transplantation. Postoperative care settings for SPK recipients vary across Canada and may have implications for patient outcomes and hospital resource use. Objective: To compare outcomes following SPK transplantation between patients receiving postoperative care in the intensive care unit (ICU) compared with the ward. Design: Retrospective cohort study using administrative health data. Setting: In Alberta, the 2 transplant centers (Calgary and Edmonton) have different protocols for routine postoperative care of SPK recipients. In Edmonton, SPK recipients are routinely transferred to the ICU, whereas in Calgary, SPK recipients are transferred to the ward. Patients: 129 adult SPK recipients (2002-2019). Measurements: Data from the Canadian Institute for Health Information Discharge Abstract Database (CIHI-DAD) were used to identify SPK recipients (procedure codes) and the outcomes of inpatient mortality, length of initial hospital stay (LOS), and the occurrence of 16 different patient safety indicators (PSIs). Methods: We followed SPK recipients from the admission date of their transplant hospitalization until the first of hospital discharge or death. Unadjusted quantile regression was used to determine differences in LOS, and age- and sex-adjusted marginal probabilities were used to determine differences in PSIs between centers. Results: There were no perioperative deaths and no major differences in the demographic characteristics between the centers. The majority of the SPK transplants were performed in Edmonton (n = 82, 64%). All SPK recipients in Edmonton were admitted to the ICU postoperatively, compared with only 11% in Calgary. There was no statistically significant difference in the LOS or probability of a PSI between the 2 centers (LOS for Edmonton vs Calgary:16 vs 13 days, P = .12; PSIs for Edmonton vs Calgary: 60%, 95% confidence interval [CI] = 0.50-0.71 vs 44%, 95% CI = 0.29-0.59, P = .08). Limitations: This study was conducted using administrative data and is limited by variable availability. The small sample size limited precision of estimated differences between type of postoperative care. Conclusions: Following SPK transplantation, we found no difference in inpatient outcomes for recipients who received routine postoperative ICU care compared with ward care. Further research using larger data sets and interventional study designs is needed to better understand the implications of postoperative care settings on patient outcomes and health care resource utilization.

scholarly journals A Pilot Study to Investigate the Efficacy of Nicotine Oral Soluble Film, Lozenge and Gum in Relief of Acute Smoking Cue-provoked Craving for Cigarette in Low Dependence Smokers

2014 ◽  
Vol 10 (2) ◽  
pp. 87-95 ◽  
Author(s):  
Daniel Du ◽  
James Borders ◽  
Alex Selmani ◽  
William Waverczak
Keyword(s):  
Pilot Study ◽  
Early Time ◽  
Controlled Study ◽  
Nicotine Gum ◽  
Open Label ◽  
Time Points ◽  
Smoking Cues ◽  
Significant Difference ◽  
Nicotine Lozenge

Introduction: A new nicotine film that releases nicotine quickly may lead to faster craving relief.Aims: This study compares the efficacy of 2.5 mg nicotine film with 2 mg nicotine lozenge and 2 mg nicotine gum on relieving provoked craving in low dependence smokers.Methods: A randomised, open-label, active comparators controlled study was conducted in 120 subjects. Subjects were abstinent from smoking for 4 hours prior to being provoked with smoking cues. After post-provocation craving assessment, subjects were administered one dose of the 3 treatments: nicotine film, lozenge, or gum. Craving intensity was then assessed at 50 seconds, 3, 5, 7, 15, 20, 25 and 30 minutes after administration.Results/Findings: Three treatments reduced craving with similar maximum effects. The effect was maintained up to 30 minutes. Nicotine film significantly reduced more craving than lozenge at 50 seconds, 3 and 5 minutes. It also significantly reduced more craving than gum at 50 seconds and 3 minutes. There was no significant difference between lozenge and gum.Conclusions: Nicotine film, lozenge and gum have similar maximum craving relief. Nicotine film significantly reduced more craving than lozenge and gum at early time points. Nicotine film may be particularly useful to provide acute craving relief.

scholarly journals Nutritional Support and Clinical Outcome of Severe and Critical Patients With COVID-19 Pneumonia

2020 ◽  
Vol 7 ◽  
Author(s):  
Xiangfeng Yue ◽  
Min Li ◽  
Yu Wang ◽  
Jing Zhang ◽  
Xinyi Wang ◽  
...  
Keyword(s):  
Protein Intake ◽  
Small Sample Size ◽  
Controlled Trial ◽  
Antiviral Treatment ◽  
Small Sample ◽  
Nutrition Support ◽  
Calorie Intake ◽  
Viral Shedding ◽  
Significant Difference ◽  
Critical Patients

Background: In 2020, a novel coronavirus has spread throughout the world. More than four hundred thousand people have died of SARS-CoV-2 pneumonia, most of which were severe and critical patients. No effective antiviral treatment has been verified thus far. Nutrition support has become one of the important treatments for severe and critical patients.Methods: In this retrospective study, 26 severe patients and 22 critical patients with laboratory confirmed COVID-19 were enrolled. We recorded the diet and nutritional treatments in severe and critical patients. Baseline characteristics and clinical outcomes of severe and critical patients were also collected.Results: Average calorie intake of severe patients (19.3 kcal/kg/d) was higher than critical patients (15.3 kcal/kg/d) (P = 0.04). Protein intake was similar in the two groups (0.65 and 0.62 g/kg per day, respectively; P = 0.29). There was no significant difference in the median duration of viral shedding between the severe and critical patients (P = 0.354).Conclusions: A permissive underfeeding strategy that restricts non-protein calories but preserves protein intake is feasible for critical patients with SARS-CoV-2 pneumonia. Viral shedding duration of critical patients was the same as severe patients who received standard feeding. Nevertheless, evidence of the conclusion is not sufficient because of small sample size. To show the real clinical benefit of permissive low-calorie and adequate protein intake in critical SARS-CoV-2 pneumonia patients, a large and pragmatic randomized controlled trial is needed.

scholarly journals Survival of patients with melanoma metastatic brain metastases in the era of novel systemic therapies in Cardiff, Wales

2019 ◽  
Vol 21 (Supplement_4) ◽  
pp. iv13-iv13
Author(s):  
Joseph Pasquale ◽  
Joanita Ocen ◽  
James Powell ◽  
Satish Kumar
Keyword(s):  
Targeted Therapy ◽  
Brain Metastases ◽  
Small Sample Size ◽  
Brain Lesion ◽  
Small Sample ◽  
Simultaneous Treatment ◽  
Local Progression ◽  
Significant Difference ◽  
Retrospective Nature ◽  
The Uk

Abstract The incidence of malignant melanoma (MM) is increasing in the UK; it’s projected to rise by 7% by 2035. MM has high predisposition to developing brain metastases (BM) with 50–60% of patients being affected. Stereotactic radiosurgery (SRS) and surgery, key interventions in managing patients with BM, have been shown to improve survival outcomes of patients. Patients’ prognosis and survival has also significantly improved with the advent of novel therapies in the last few years. It was noted that the Cardiff Neuro-Oncology multidisciplinary team were receiving increasing amount of referrals for consideration of surgery or SRS in patients with MM. 106 MDT referrals were retrospectively reviewed. 31 patients had surgery, 20 patients had SRS and the remaining 54 patients had WBRT. There was no significant difference in the patient distribution. The majority of patients had 1 brain lesion in both groups (in similar proportions). The 12 month survival for the surgical cohort was 65% for immunotherapy group, 55%- targeted therapy and 30%- no therapy. For the SRS group the 12 month survival for immunotherapy was 45%, targeted therapy- 40% and 20%- no therapy. The median OS for surgery versus SRS was 8 and 7 months respectively. The results suggest that simultaneous treatment with surgery or SRS in conjunction with SACT does improve survival. Interpretation of results will have to be taken with caution due to the retrospective nature and the small sample size. Going forward, we will delve deeper and review local progression rates and SACT timing/sequencing in our practice.

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