Elham Gheysvandi, Iman Dianat, Rashid Heidarimoghadam, Leili Tapak, Akram Karimi-Shahanjarini, Forouzan Rezapur-Shahkolai
Neck and shoulder pain among elementary school students: prevalence and its risk factors
BMC public health
rett syndrome rare genetic neurodevelopmental disorder predominantly impacts females presents loss acquired skills impaired communication stereotypic hand movements given limited treatment options rett syndrome dire need effective interventions evaluate safety efficacy trofinetide randomized controlled trials rcts report rett syndrome patients identified 109 articles four databases scopus pubmed web science cochrane central removing duplicates narrowed 59 articles assessment included rcts evaluated efficacy safety trofinetide patients rett syndrome three studies eligible inclusion two independent reviewers evaluated identified studies titles abstracts full texts extracting pertinent data assessed quality studies using cochrane risk bias rob 20 tool conducted metaanalysis using fixed effects model case insignificant heterogeneity otherwise used random effects model based nature outcome analyzed mean difference odds ratio analysis conducted using revman version 53 among analyzed outcomes 181 patients trofinetide group 134 patients placebo group significant improvement rett syndrome behavior questionnaire rsbq scores observed 200 mg dosage overall mean difference 353 p 0001 clinical global impressionimprovement cgii scores improved considerably 200 mg dosage overall mean difference 034 p 00001 substantial changes observed motor behavioral assessment mba top 3 caregiver concerns evaluated treatment emergent adverse events teaes across various dosages noted significant associations diarrhea 200 mg vomiting 200 mg irritability 200 mg however find significant association dosages incidence decreased appetite trofinetide demonstrated potential improving rsbq cgii scores 200 mg dosage although substantial changes found mba top 3 caregiver concerns adverse events linked specific dosages