scholarly journals Beyond the limitation of randomized controlled trials (RCTs)-current drug repositioning by using human induced pluripotent stem (iPS) cells technology-

2017 ◽  
Author(s):  
M.D. Midori Okabe

AbstractTo ensure the clinical value of medical interventions, randomized controlled trials (RCTs) are necessary. However, the results of conventional RCTs cannot show individual therapeutic efficacy and safety for medical intervention to a targeted patient. It is the most important weak point of conventional RCTs. Here I show that the new clinical research method by using human induced pluripotent stem (iPS) cells technology will be able to complement the most important weak point of conventional RCTs.As the representative examples, I show the new clinical values of statins (inhibitors of 3-hydroxy-3-methylglutaryl-coenzyme A reductase) found by using human iPS cells technology in achondroplasia or hanatophoric dysplasia (type 1) case and hepatitis C virus (HCV) infection case. Furthermore, they are also important examples for drug repositioning.Therefore, my article would be valuable as a scientific communication for physicians and/or scientists.

2013 ◽  
Vol 2013 ◽  
pp. 1-8 ◽  
Author(s):  
Changtai Zhu ◽  
Hao Cao ◽  
Xifa Zhou ◽  
Chunlei Dong ◽  
Judong Luo ◽  
...  

Objective. To evaluate the clinical value of Danshen injection and Huangqi injection for the treatment of liver cirrhosis.Methods. The Chinese Biomedical Literature Database (CBM), Chinese Scientific Journals Full-Text Database (VIP), Wanfang Database, China National Knowledge Infrastructure (CNKI), PubMed, and EMBASE database were searched to collect the literatures about the randomized controlled trials involving the treatment of liver cirrhosis with Danshen injection combined with Huangqi injection, and the data analyses were performed using RevMan 4.2 software.Results. A total of 11 studies involving 1086 patients (trials group: 554 cases, control group: 532 cases) were included in this study. Compared with those in control group, the meta-analysis showed-that the total effectiveness rate and the level of serum albumin increased, while serum total bilirubin, alanine transmninase, type III procollagen, hyaluronic acid, laminin, and type-IV collagen decreased in trials group. The Jadad score ranged from 1 to 2 and the funnel plot analysis suggests that publication bias may occur.Conclusions. Danshen injection combined with Huangqi injection may promote the curative efficacy of liver cirrhosis, which is a promising novel treatment approach. The exact outcome needs to perform rigorously designed, multicenter, and large randomized controlled trials.


2020 ◽  
Vol 16 ◽  
Author(s):  
Alexander K. C. Leung ◽  
Amy A.M. Leung ◽  
Alex H.C. Wong ◽  
Kam Lun Hon

Background: Hiccups are a universal phenomenon. They are usually benign and self-limited. Persistent or intractable hiccups, although rare, can be debilitating and may indicate the presence of an underlying pathological process. Objective: To familiarize physicians with the pathophysiology, etiology, evaluation, and management of children with hiccups. Methods: A search was conducted on December 10, 2019 in Pubmed Clinical Queries using the key terms "hiccup" OR “hiccough” OR “singultus”. The selected publication types included all clinical trials (including open trials, non-randomized controlled trials, and randomized controlled trials), observational studies, and reviews (including meta-analysis and narrative reviews) published within the past 10 years. Only papers published in the English literature were included in this review. The information retrieved from the above search was used in the compilation of the present article. Results: Overdistension of the stomach is the most commonly identifiable cause of acute hiccups, followed by gastroesophageal reflux and gastritis. Other causes of hiccups, notably persistent and intractable hiccups, include an underlying gastrointestinal, neurological, cardiovascular, pulmonary, infectious, and psychogenic disorder. Persistent or intractable hiccups can be a harbinger of serious medical pathology. A detailed history and thorough physical examination may provide clues for the etiology of the hiccups. The treatment of hiccups should be directed at the underlying cause whenever possible. Bouts of acute hiccups less than 48 hours rarely require medical intervention as they usually resolve within minutes. Treatment may be considered when hiccups are bothersome, persistent, or intractable. Treatment modalities include lifestyle changes, physical maneuvers, pharmacotherapy, and very rarely, surgical intervention. Conclusion: Acute hiccups are usually benign and self-limiting. Persistent or intractable hiccups can be a harbinger of serious medical pathology. The underlying cause should be treated if possible. There are no formal guidelines to the treatment of hiccups. Currently, most of the methods proposed are based on case reports and anecdotal evidence. Terminating an episode of hiccups can be very challenging for a clinician but may tremendously improve the patient’s quality of life. It is hoped that future well-designed and better-powered studies will provide us with more information on the efficacy of various treatment modalities for hiccups.


2015 ◽  
Vol 2015 ◽  
pp. 1-10 ◽  
Author(s):  
Min He ◽  
Yu Wu ◽  
Mengmeng Wang ◽  
Wenwen Chen ◽  
Weian Yuan ◽  
...  

Oxymatrine (OMTR) is widely used for the treatment of chronic hepatitis B (CHB) in China. Several reports revealed that combination of OMTR and lamivudine reduced the incidence of tyrosine- (Y-) methionine- (M-) aspartic acid- (D-) aspartic acid (D) (YMDD) mutations in CHB patients. The aim of this study was to evaluate the clinical value of oxymatrine in preventing lamivudine induced YMDD mutation using meta-analysis of data from published randomized controlled trials (RCTs) and to provide some useful information for clinical treatment and future research of YMDD mutation. The Cochrane Central Register of Controlled Trials, Medline, Science Citation Index, EMBASE, China National Knowledge Infrastructure, Wanfang Database, and China Biomedical Database were searched to identify RCTs that evaluated the incidence of YMDD-motif mutation to lamivudine therapy and lamivudine plus OMTR therapies in CHB patients. Data analysis was carried out with the use of RevMan 5.3.2. The literature search yielded 324 studies, and 16 RCTs matched the selection criteria. Overall, the incidence of YMDD mutation was significantly lower in patients treated with lamivudine plus OMTR than in patients treated with lamivudine alone (11.14% versus 28.18%; RR: 0.41; 95% CI: 0.33–0.52;p<0.05). The exact outcome needs to perform rigorously designed, multicenter, and large randomized controlled trials.


Methodology ◽  
2017 ◽  
Vol 13 (2) ◽  
pp. 41-60
Author(s):  
Shahab Jolani ◽  
Maryam Safarkhani

Abstract. In randomized controlled trials (RCTs), a common strategy to increase power to detect a treatment effect is adjustment for baseline covariates. However, adjustment with partly missing covariates, where complete cases are only used, is inefficient. We consider different alternatives in trials with discrete-time survival data, where subjects are measured in discrete-time intervals while they may experience an event at any point in time. The results of a Monte Carlo simulation study, as well as a case study of randomized trials in smokers with attention deficit hyperactivity disorder (ADHD), indicated that single and multiple imputation methods outperform the other methods and increase precision in estimating the treatment effect. Missing indicator method, which uses a dummy variable in the statistical model to indicate whether the value for that variable is missing and sets the same value to all missing values, is comparable to imputation methods. Nevertheless, the power level to detect the treatment effect based on missing indicator method is marginally lower than the imputation methods, particularly when the missingness depends on the outcome. In conclusion, it appears that imputation of partly missing (baseline) covariates should be preferred in the analysis of discrete-time survival data.


2020 ◽  
Vol 146 (12) ◽  
pp. 1117-1145
Author(s):  
Kathryn R. Fox ◽  
Xieyining Huang ◽  
Eleonora M. Guzmán ◽  
Kensie M. Funsch ◽  
Christine B. Cha ◽  
...  

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