scholarly journals Statin treatment and muscle symptoms: series of randomised, placebo controlled n-of-1 trials

BMJ ◽  
2021 ◽  
pp. n135 ◽  
Author(s):  
Emily Herrett ◽  
Elizabeth Williamson ◽  
Kieran Brack ◽  
Danielle Beaumont ◽  
Alexander Perkins ◽  
...  
Keyword(s):  
Drug Effects ◽  
Statin Treatment ◽  
Term Treatment ◽  
Individual Treatment ◽  
Primary Analysis ◽  
Placebo Period ◽  
Link Type ◽  
Long Term Treatment ◽  
Symptom Scores ◽  
Double Blinded

Abstract Objective To establish the effect of statins on muscle symptoms in people who had previously reported muscle symptoms when taking statins. Design Series of randomised, placebo controlled n-of-1 trials. Setting Primary care across 50 sites in the United Kingdom, December 2016 to April 2018. Participants 200 participants who had recently stopped or were considering stopping treatment with statins because of muscle symptoms. Interventions Participants were randomised to a sequence of six double blinded treatment periods (two months each) of atorvastatin 20 mg daily or placebo. Main outcome measures At the end of each treatment period, participants rated their muscle symptoms on a visual analogue scale (0-10). The primary analysis compared symptom scores in the statin and placebo periods. Results 151 participants provided symptoms scores for at least one statin period and one placebo period and were included in the primary analysis. Overall, no difference in muscle symptom scores was found between the statin and placebo periods (mean difference statin minus placebo −0.11, 95% confidence interval −0.36 to 0.14; P=0.40)). Withdrawals because of intolerable muscle symptoms were 18 participants (9%) during a statin period and 13 (7%) during a placebo period. Two thirds of those completing the trial reported restarting long term treatment with statins. Conclusions No overall effect of atorvastatin 20 mg on muscle symptoms compared with placebo was found in participants who had previously reported severe muscle symptoms when taking statins. Most people completing the trial intended to restart treatment with statins. N-of-1 trials can assess drug effects at the group level and guide individual treatment. Trial registration ISRCTN30952488 , EUDRACT 2016-000141-31, NCT02781064 .

Clinical and diagnostic value of the method of chromato-mass spectrometry of microbial markers in case of damage to the oral mucosa in children by rheumatic diseases

2021 ◽  
Vol 1 (38) ◽  
pp. 49-57
Author(s):  
A. A. Skakodub ◽  
O. I. Admakin ◽  
Ad. A. Mamedov ◽  
N. A. Geppe ◽  
A. V. Simonova
Keyword(s):  
Mass Spectrometry ◽  
Oral Mucosa ◽  
Rheumatic Diseases ◽  
Modern Method ◽  
Diagnostic Value ◽  
Term Treatment ◽  
Individual Treatment ◽  
Oral Microbiota ◽  
Long Term Treatment ◽  
Wide Range

Due to the presence of a large percentage of 42.6% secondary oral infection in children with rheumatic diseases [1, 2], which arose during long-term treatment of shock and maintenance doses of anti-inflammatory therapy, it was important to study the microbiota [16, 17]. This paper for the first time applied a modern method for assessing the microbiota of various biotopes of the affected oral mucosa in children with rheumatic diseases – chromatosis-mass-spectrometry (CMSM), based on the quantitative determination of the level of markers of microorganisms: fatty acids, aldehydes, alcohols [5, 7, 10, 11]. СMSM is a highly sensitive method with a wide diagnostic spectrum. The study of a wide range of microorganisms provides new opportunities in the diagnosis of oral dysbacteriosis and increasing the effectiveness of individual treatment. The aim of the study is to improve the level of diagnosis and treatment of oral mucosal diseases in children with rheumatic diseases, through the use of chromato-mass-spectrometry of the oral microbiota.

Dissociation of symptom scores and bronchial hyperreactivity: Study in asthmatic children on long-term treatment with inhaled beclomethasone dipropionate

1992 ◽  
Vol 13 (2) ◽  
pp. 71-77 ◽  
Author(s):  
Marie R. Benoist ◽  
Jean J. Brouard ◽  
Patrick Rufin ◽  
Serge Waernessyckle ◽  
Jacques de Blic ◽  
...  
Keyword(s):  
Beclomethasone Dipropionate ◽  
Term Treatment ◽  
Bronchial Hyperreactivity ◽  
Asthmatic Children ◽  
Long Term Treatment ◽  
Symptom Scores

scholarly journals High-dose N-acetylcysteine for long-term, regular treatment of early-stage chronic obstructive pulmonary disease (GOLD I–II): study protocol for a multicenter, double-blinded, parallel-group, randomized controlled trial in China

Trials ◽  
2020 ◽  
Vol 21 (1) ◽  
Author(s):  
Heshen Tian ◽  
Yumin Zhou ◽  
Longhui Tang ◽  
Fan Wu ◽  
Zhishan Deng ◽  
...  
Keyword(s):  
Early Stage ◽  
Controlled Trial ◽  
Term Treatment ◽  
Chronic Obstructive ◽  
High Dose ◽  
Obstructive Pulmonary Disease ◽  
Randomized Controlled ◽  
Long Term Treatment ◽  
Double Blinded

Abstract Introduction The presence of increased oxidative stress and airway inflammation has been proven in subjects with chronic obstructive pulmonary disease (COPD). Several studies have demonstrated that drugs with antioxidant and anti-inflammatory properties such as N-acetylcysteine (NAC) can reduce the rate of exacerbations in patients with COPD. However, the beneficial effects of NAC in early-stage COPD are minimally discussed. We are investigating whether high-dose NAC has therapeutic effects in Chinese patients with early-stage COPD. Method and analysis A randomized, double-blinded, placebo-controlled, parallel-group, multicenter clinical trial is evaluating the efficacy and safety of NAC for the long-term treatment of patients with early-stage COPD at 24 centers in China. Subjects aged 40–80 years and recruited by physicians or researchers with special training will be randomized to either NAC 600 mg twice daily group or matching placebo group for 2 years. Measurements will include forced expiratory volume in 1 s (FEV1), the number of COPD exacerbations, health-related quality, and pharmacoeconomic analysis. Discussion Currently, there are no randomized controlled trials with high-dose N-acetylcysteine (600 mg twice daily) for patients with mild-to-moderate COPD (GOLD I–II). We designed this multicenter randomized controlled trial (RCT) to assess the effectiveness, safety, and cost-effectiveness of long-term treatment with high-dose N-acetylcysteine. The results of this trial may guide clinical practice and change the standard of early COPD management. Trial registration Chinese Clinical Trial Registry ChiCTR-IIR-17012604. Registered on 07 September 2017.

scholarly journals Long-term treatment with metformin in obese, insulin-resistant adolescents: results of a randomized double-blinded placebo-controlled trial

2016 ◽  
Vol 6 (8) ◽  
pp. e228-e228 ◽  
Author(s):  
M P van der Aa ◽  
M A J Elst ◽  
E M W van de Garde ◽  
E G A H van Mil ◽  
C A J Knibbe ◽  
...  
Keyword(s):  
Controlled Trial ◽  
Term Treatment ◽  
Insulin Resistant ◽  
Long Term Treatment ◽  
Double Blinded

A 1-year, randomized, placebo-controlled study of donepezil in patients with mild to moderate AD

Neurology ◽  
2001 ◽  
Vol 57 (3) ◽  
pp. 489-495 ◽  
Author(s):  
B. Winblad ◽  
K. Engedal ◽  
H. Soininen ◽  
F. Verhey ◽  
G. Waldemar ◽  
...  
Keyword(s):  
Global Assessment ◽  
Term Treatment ◽  
Controlled Study ◽  
End Point ◽  
Northern European ◽  
Long Term Treatment ◽  
Double Blinded ◽  
To Receive ◽  
State Examination

Objective: To evaluate the long-term clinical efficacy and safety of donepezil versus placebo over 1 year in patients with mild to moderate AD.Methods: Patients (n = 286; mean age, 72.5 years) with possible or probable AD from five Northern European countries were randomized to receive either donepezil (n = 142; 5 mg/day for 28 days, followed by 10 mg/day) or placebo (n = 144) for 1 year.Results: The study was completed by 66.9% of the donepezil- and 67.4% of the placebo-treated patients. The benefit of donepezil over placebo was demonstrated by the Gottfries-Bråne-Steen (a global assessment for rating dementia symptoms) total score at weeks 24, 36, and 52 (p < 0.05) and at the study end point (week 52, last observation carried forward; p = 0.054). Advantages of donepezil over placebo were also observed in cognition and activities of daily living (ADL) assessed by the Mini-Mental State Examination at weeks 24, 36, and 52, and the end point (p < 0.02) and by the Progressive Deterioration Scale at week 52 and the end point (p < 0.05). Adverse events (AE) were recorded for 81.7% of donepezil- and 75.7% of placebo-treated patients, with 7% of donepezil- and 6.3% of placebo-treated patients discontinuing because of AE. Treatment response to donepezil was not predicted by APOE genotype or sex in this population.Conclusion: As the first 1-year, multinational, double-blinded, placebo-controlled study of a cholinesterase inhibitor in AD, these data support donepezil as a well tolerated and effective long-term treatment for patients with AD, with benefits over placebo on global assessment, cognition, and ADL.

068 Evaluation of the long-term treatment effect of teriflunomide on cognitive outcomes and association with brain volume change: data from temso and its extension study

2018 ◽  
Vol 89 (6) ◽  
pp. A28.1-A28 ◽  
Author(s):  
Till Sprenger ◽  
Jeannette Lechner-Scott ◽  
Maria P Sormani ◽  
Jerry S Wolinsky ◽  
Jens Wuerfel ◽  
...  
Keyword(s):  
Cognitive Function ◽  
Volume Change ◽  
Brain Volume ◽  
Term Treatment ◽  
Cognitive Outcomes ◽  
Link Type ◽  
Brain Volume Loss ◽  
Long Term Treatment ◽  
Z Scores

IntroductionIn a blinded SIENA (Structural Image Evaluation using Normalisation of Atrophy) analysis of TEMSO (NCT00134563), teriflunomide significantly reduced brain volume loss (BVL) over 2 years vs placebo. Further analysis indicated a strong correlation between 2 year BVL and disability worsening, showing better disability outcomes for patients with lower rates of BVL. Here, we explore the relationship between BVL and long-term changes in cognitive function in TEMSO and its extension (NCT00803049).MethodsThe effect of teriflunomide on cognitive function was assessed by change from baseline in Paced Auditory Serial Addition Test (PASAT)−3 scores in the TEMSO core (n=1086) and extension (n=740) studies. To evaluate change in PASAT-3 scores over 5 years, the TEMSO population was categorised into groups defined by percentage brain volume change from baseline to Year 2 (assessed by SIENA).ResultsAdjusted mean changes from baseline to Week 96 in PASAT-3 raw/Z-scores were –0.265/–0.022 and 0.870/0.073 for placebo and teriflunomide 14 mg, respectively (difference vs placebo, p=0.0435 in both instances). Long-term improvements in PASAT-3 Z-scores were observed with teriflunomide 14 mg treatment: mean (SD) changes from baseline at Weeks 156 and 276 were 0.194 (0.634) and 0.200 (0.677), respectively. Mean (SD) units of change from baseline in raw PASAT-3 scores for teriflunomide 14 mg–treated patients at Weeks 156 and 276 were 2.36 (7.73) and 2.43 (8.24), respectively. In an association analysis, the group with least BVL from baseline to Year 2 demonstrated a significant improvement in PASAT-3 score with teriflunomide treatment over 5 years vs the group with most BVL.ConclusionTeriflunomide significantly improved PASAT-3 performance vs placebo over 5 years in the TEMSO core and extension studies. Slower rates of BVL over 2 years correlated with better long-term PASAT-3 improvement. This study suggests that BVL earlier in the disease course predicts longer-term cognitive function.Study supportSanofi.

scholarly journals Short-term and long-term safety and efficacy of tenofovir alafenamide, tenofovir disoproxil fumarate and entecavir treatment of acute-on-chronic liver failure associated with hepatitis B

2021 ◽  
Vol 21 (1) ◽  
Author(s):  
Juan Li ◽  
Chunhua Hu ◽  
Yi Chen ◽  
Rou Zhang ◽  
Shan Fu ◽  
...  
Keyword(s):  
Hepatitis B ◽  
Term Treatment ◽  
Efficacy And Safety ◽  
Short Term ◽  
Serum Cystatin C ◽  
Short Term Treatment ◽  
Link Type ◽  
Long Term Treatment ◽  
Significant Difference

Abstract Background & Aims There is limited evidence on the efficacy and safety of nucleos(t) ide analogues (NAs) in the treatment of HBV-ACLF. Our objective was to evaluate the outcomes among TAF, TDF and ETV, three first-line antivirals against chronic hepatitis B, in patients with HBV-ACLF. Methods Patients with HBV-related ACLF were recruited and received daily TAF (25 mg/d), TDF (300 mg/d) and ETV (0.5 mg/d). They were prospectively followed-up. The primary endpoint was overall survival at week 12 and week 48, the secondary endpoints were virological response and biochemical response. Results Forty gender and age matched eligible subjects were recruited and divided into three groups: TAF group, TDF group and ETV group. By week 48, 8 (80%) patients in TAF group, 6 (60%) patients in TDF group and 17 (85%) patients in ETV group survived without liver transplantation (P = 0.251). After 4 weeks of NAs treatment, all three groups showed paralleling reduction of HBV DNA levels. All three groups presented similar biochemical responses at week 4, patients treated with TAF showed a priority in total bilirubin reduction, albumin and cholesterol maintenance. Additionally, although there was no significant difference in changes of serum urea, serum creatinine, serum cystatin C and estimated GFR among the three groups by treatment week 4, TDF showed unfavorable renal safety even in short -term treatment. The treatment using NAs was well-tolerated and there was no serious drug-related adverse event reported. Conclusions TAF, TDF and ETV are of similar efficacy and safety in short-term and long-term treatment of HBV-ACLF. Trial registration This study is ongoing and is registered with ClinicalTrials.gov, NCT03640728 (05/02/2019).

scholarly journals Risk of Recurrent Coronary Events in Patients With Familial Hypercholesterolemia; A 10-Years Prospective Study

2021 ◽  
Vol 11 ◽  
Author(s):  
Kjell-Erik Arnesen ◽  
Ann Vinh Phung ◽  
Karoline Randsborg ◽  
Irene Mork ◽  
Marlene Thorvall ◽  
...  
Keyword(s):  
Familial Hypercholesterolemia ◽  
Low Density Lipoprotein ◽  
Density Lipoprotein ◽  
Statin Treatment ◽  
Term Treatment ◽  
Lipid Lowering ◽  
Treat To Target ◽  
Atherosclerotic Cardiovascular Disease ◽  
Pcsk9 Inhibitors ◽  
Long Term Treatment

Background and Aim: Real world evidence on long term treatment of patients with familial hypercholesterolemia (FH) is important. We studied the effects of intensive lipid lowering medication (LLM) and optimized lifestyle in the study TTTFH–Treat To Target FH.Materials and Methods: Adults with a first known total cholesterol of mean (95% CI) 9.8 mmol/L (9.5, 10.1) were included consecutively in their routine consultation during 2006. Of the patients 86.4% had a pathogenic FH-mutation and the remaining were clinically diagnosed. We included 357 patients and 279 met for follow-up after median 10.0 (min 8.1, max 12.8) years.Results: Mean (95% CI) low density lipoprotein (LDL-C) was reduced from 3.9 (3.8, 4.1) to 3.0 (2.9, 3.2). More men than women used high intensity statin treatment, 85.2 and 60.8%, respectively. Women (n = 129) had higher LDL-C; 3.3 mmol/L (3.0, 3.5), than men; (n = 144) 2.8 mmol/L (2.6, 3.0), p = 0.004. Add-on PCSK9 inhibitors (n = 25) reduced mean LDL-C to 2.0 (1.4, 2.6) mmol/L. At enrollment 57 patients (20.4%) had established atherosclerotic cardiovascular disease (ASCVD), and 46 (80.4%) of them experienced a new event during the study period. Similarly, 222 (79.6%) patients had no detectable ASCVD at enrollment, and 29 of them (13.1%) experienced a first-time event during the study period.Conclusion: A mean LDL-C of 3.0 mmol/L was achievable in FH, treated intensively at a specialized clinic with few users of PCSK9 inhibitors. LDL-C was higher (0.5 mmol/L) in women than in men. In patients with ASCVD at enrollment, most (80.7%) experienced a new ASCVD event in the study period. The FH patients in primary prevention had more moderate CV risk, 13% in ten years.

Hypnotherapy is effective in the long-term treatment of functional dyspepsia

2001 ◽  
Vol 120 (5) ◽  
pp. A115-A115 ◽  
Author(s):  
E CALVERT ◽  
L HOUGHTON ◽  
P COOPER ◽  
P WHORWELL
Keyword(s):  
Functional Dyspepsia ◽  
Term Treatment ◽  
Long Term Treatment

1608: Long-Term Treatment with High Doses of Sildenafil Does Not Up-Regulate the Levels of Phosphodiesterase 5 (Pde5) in the Rat Penis

2004 ◽  
Vol 171 (4S) ◽  
pp. 424-424 ◽  
Author(s):  
Monica G. Ferrini ◽  
Eliane G. Valente ◽  
Jacob Rajfer ◽  
Nestor F. Gonzalez-Cadavid
Keyword(s):  
Term Treatment ◽  
Long Term Treatment ◽  
High Doses ◽  
Phosphodiesterase 5
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