Effectiveness of pharmacological treatments in Duchenne muscular dystrophy: a protocol for a systematic review and meta-analysis

Abstract

IntroductionIn recent years, important advances have been made in the treatment of Duchenne muscular dystrophy (DMD). This protocol proposes a methodology for carrying out a systematic review and meta-analysis that aims to: (1) improve the evidence of the benefits of different pharmacological treatments in boys with DMD, and (2) compare the benefit of treatments specifically aimed at delaying the progression of disease in the functional outcomes.Methods and analysisThis protocol is guided by the Preferred Reporting Items for Systematic Review and Meta-Analysis Protocols (PRISMA-P) and by the Cochrane Collaboration Handbook. A thorough selection of the literature will be done through the MEDLINE, EMBASE and Web of Science databases. The search will be conducted in English and Spanish. The Risk of Bias 2.0 tool from the Cochrane Collaboration will be used to assess the risk of bias. A narrative synthesis of the data will be performed. Meta-analysis will be conducted for effect of treatment on the 6 min walking distance (6MWD), North Star Ambulatory Assessment and Timed Functional Tests. Subgroup analyses will be performed by age or baseline values of the 6MWD, and overall bias.Ethics and disseminationThe approval of an ethical committee is not required. All the included trials will comply with the current ethical standards and the Declaration of Helsinki. The results of this proposed systematic review and meta-analysis will provide a general overview and evidence concerning the effectiveness of pharmacological treatments in Duchenne muscular dystrophy. Findings will be disseminated to academic audiences through peer-reviewed publications, as well as to clinical audiences, patients’ associations and policy makers, and may influence guideline developers in order to improve outcomes for these patients.PROSPERO registration numberCRD42018102207

Validity of bioelectrical impedance to estimate fat-free mass in boys with Duchenne muscular dystrophy

“…Later, it occurs loss of muscular strength and ability to ambulate. Other common DMD complications include scoliosis, heart failure, respiratory insufficiency, fractures of long bones, and vertebrae due to osteoporosis [ 3 ]. Currently, glucocorticoids are the only disease-modifying therapeutic agent shown to improve short-term muscle strength [ 4 ].…”

Efficacy of Chinese herbal injections combined with fluoropyrimidine and oxaliplatin-based chemotherapy for advanced colorectal cancer

“…Third, some studies may lack methodological description like the methods of randomization and lead to a poor methodological quality. [ 24 ]…”

“The Social Network” and Muscular Dystrophies: The Lesson Learnt about the Niche Environment as a Target for Therapeutic Strategies

“…Eventual accumulation of inflammation and fibrosis lead to progressive muscle weakening and loss of muscle mass and function [ 37 ]. The efficiency of regeneration appears to be low [ 38 ]. This progressive muscle wasting condition leads to severe disability and follows with premature death in affected individuals due to respiratory and/or cardiac failure, typically by or before the age of 30.…”