scholarly journals Clinical Efficacy and Safety of Ivacaftor/Lumacaftor Combination in Patients with Cystic Fibrosis: International Studies Review

Author(s):  
Nataliya Yu. Kashirskaya ◽  
Nika V. Petrova ◽  
Rena A. Zinchenko

Cystic fibrosis is an autosomal recessive disease caused by structure abnormalities in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. It is characterized by severe course and poor prognosis without or with insufficient treatment. Approval of pathogenetic therapy medications, CFTR modulators (potentiators and correctors), for clinical use in 2012 in the United States has reduced mortality from this disease. This article provides the overview of studies on clinical efficacy and safety of ivacaftor/lumacaftor combination (Iva/Lum) — the first licensed CFTR modulator medication for homozygous patients with F508del variant. It was shown that Iva/Lum increases lung function, reduce the number of acute conditions of bronchopulmonary process (including those that require antibiotics and hospitalization), partially restores pancreas exocrine function, increases body weight and mass growth index, and improves quality of life. It allows considering it as favorable effect on the course and prognosis of cystic fibrosis. It was also noted that the early onset of the drug administration (from the age of two) positively affects the prognosis of the disease, increasing life expectancy and improving quality of life.

2019 ◽  
pp. 52-57
Author(s):  
O. N. Minushkin ◽  
L. V. Maslovsky ◽  
M. I. Bulanova ◽  
O. F. Shaposhnikovа

A study of the clinical efficacy and safety of the drug Samelix (ademetionine, manufacturer – JSC «Canonfarma production», Russia) in 30 patients with chronic alcoholic liver disease (steatohepatitis mild to moderate currents, cirrhosis of the liver grade a ChildPugh) with the syndrome of cholestasis. Purpose of the study: evaluate the clinical efficacy and safety of Samelix (ademetionine) in 30 patients with chronic alcoholic liver disease. Objectives of the study: evaluate the effect of the drug on biochemical parameters, evaluate the effect of the drug on the clinical manifestation of the disease based on the individual diary data during the course therapy, assess the quality of life through the SF-36 questionnaire before and after treatment; report adverse events. The results of the study showed that therapy with this drug leads to a significant positive dynamics of biochemical parameters, regression of clinical manifestations of the disease, a significant increase in the quality of life. Good and excellent results of therapy were observed in 76.7% of cases. The drug is safe and well tolerated.


CHEST Journal ◽  
2010 ◽  
Vol 137 (3) ◽  
pp. 642-650 ◽  
Author(s):  
Alexandra L. Quittner ◽  
Michael S. Schechter ◽  
Lawrence Rasouliyan ◽  
Tmirah Haselkorn ◽  
David J. Pasta ◽  
...  

2020 ◽  
Vol 21 (6) ◽  
pp. 427-435 ◽  
Author(s):  
Cheng Cui ◽  
Siqi Tu ◽  
Valerie Sia Jie En ◽  
Xiaobei Li ◽  
Xueting Yao ◽  
...  

Background: As the number of severe acute respiratory syndrome coronavirus 2 (SARS-COV-2) infected people is greatly increasing worldwide, the international medical situation becomes very serious. Potential therapeutic drugs, vaccine and stem cell replacement methods are emerging, so it is urgent to find specific therapeutic drugs and the best treatment regimens. After the publications on hydroxychloroquine (HCQ) with anti- SARS-COV-2 activity in vitro, a small, non-randomized, open-label clinical trial showed that HCQ treatment was significantly associated with reduced viral load in patients with coronavirus disease-19 (COVID-19). Meanwhile, a large prophylaxis study of HCQ sulfate for COVID-19 has been initiated in the United States. HCQ offered a promising efficacy in the treatment of COVID-19, but the optimal administration is still being explored. Methods: We used the keyword "hydroxychloroquine" to conduct a literature search in PubMed to collect relevant literature on the mechanism of action of HCQ, its clinical efficacy and safety, pharmacokinetic characteristics, precautions for clinical use and drug interactions to extract and organize information. Results: This paper reviews the mechanism, clinical efficacy and safety, pharmacokinetic characteristics, exposureresponse relationship and precautions and drug interactions of HCQ, and summarizes dosage recommendations for HCQ sulfate. Conclusion: It has been proved that HCQ, which has an established safety profile, is effective against SARS-CoV-2 with sufficient pre-clinical rationale and evidence. Data from high-quality clinical trials are urgently needed worldwide.


Author(s):  
Elliot Friedman ◽  
Beth LeBreton ◽  
Lindsay Fuzzell ◽  
Elizabeth Wehrpsann

By many estimates the majority of adults over age 65 have two or more chronic medical conditions (multimorbidity) and are consequently at increased risk of adverse functional outcomes. Nonetheless, many older adults with multimorbidity are able to maintain high levels of function and retain good quality of life. Research presented here is designed to understand the influences that help ensure better functional outcomes in these older adults. This chapter presents findings that draw on data from the Midlife in the United States study. The independent and interactive contributions of diverse factors to multimorbidity and changes in multimorbidity over time are reviewed. The degree that multimorbidity increases risk of cognitive impairment and disability is examined. The role of inflammation as a mediator is considered. Multimorbidity is increasingly the norm for older adults, so better understanding of factors contributing to variability in multimorbidity-related outcomes can lead to improved quality of life.


2021 ◽  
Vol 10 (10) ◽  
pp. 2187
Author(s):  
Omrit Feldman ◽  
Eran Goldstien ◽  
Benjamin Rolnik ◽  
Ariel B. Ganz ◽  
Shahar Lev-Ari

Stuttering is a speech disorder that can cause disturbances in the timing and flow of speech. In addition to being a communication disorder, stuttering is often accompanied by a reduction in the quality of life and has impacts on social status, mental well-being, self-acceptance, and the chances of integration into the labor market. The Inquiry Based Stress Reduction (IBSR) program, developed in the United States by Byron Katie in 1986, is the clinical application of “The Work” method (Thework.com) and represents an emerging mindfulness and cognitive-reframing method. IBSR has been demonstrated to improve mental health and well-being in adults and may alleviate psychological and psychosocial symptoms of stuttering. The purpose of this trial was to examine the effect of a 12-week IBSR intervention on the overall stuttering experience and indicators of anxiety, psychological flexibility, and well-being among adults who stutter (AWS). This study was a randomized controlled clinical trial. Participants were randomized to IBSR (n = 28) and control (n = 28) groups. Validated questionnaires of overall stuttering experience (OASES-A), anxiety (STAI), psychological flexibility (PFQ), and satisfaction with life (SWLS) were completed before, after, and one month after the intervention. An intention-to-treat approach was implemented for analysis. Our results show that participants in the IBSR intervention group exhibited a greater improvement in their overall stuttering experience as compared to the control group, as well as in general information on stuttering awareness and perception, reactions to stuttering, communication in daily situations, and quality of life. In addition, we found a greater reduction in anxiety levels and an increase in satisfaction-with-life scores in the IBSR group. These results indicate that IBSR can improve the overall stuttering experience.


2021 ◽  
Vol 28 (3) ◽  
pp. 265-276
Author(s):  
Naira Khachatryan ◽  
Maxwell Pistilli ◽  
Maureen G. Maguire ◽  
Angela Y. Chang ◽  
Marissa R. Samuels ◽  
...  

2021 ◽  
pp. 002073142199484
Author(s):  
Vicente Navarro

This article analyses the political changes that have been occurring in the United States (including the elections for the presidency of the country) and their consequences for the health and quality of life of the population. A major thesis of this article is that there is a need to analyse, besides race and gender, other categories of power - such as social class - in order to understand what happens in the country. While the class structure of the United States is similar to that of major Western European countries, the political context is very different. The U.S. political context has resulted in the very limited power of its working class, which explains the scarcity of labor, political and social rights in the country, such as universal access to health care.


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