Title : Givinostat efficacy in patients with muscular dystrophy: A systematic review
Abstract:
Muscular Dystrophy (MD) is a group of hereditary disorders characterized by progressive generalized muscle weakness and degeneration. Recent studies evaluated the role of Givinostat in treating MD. So, we aimed in our systematic review to assess the efficacy of Givinostat in MD. PubMed, ScienceDirect, and Cochrane databases were searched using a pre-defined search strategy from inception until April-2024 following the standard method of Cochrane Handbook and the PRISMA-statement guidelines. All clinical trials studying Givinostat in patients with MD were included. Three clinical trials with a total of 250 participants included, two of these studies tested Givinostat efficacy in DMD and the other in BMD. One of these primarily studied givinostat histological effect in DMD and revealed that it increased the fraction of muscle tissue by 29.1%, and cross-sectional area by 77%. And reduced the amount of necrosis by 43.5% and fatty replacement by 37.5%. The second study tested its effect in BMD and reported that mean total fibrosis didn’t change in treatment or placebo group, and the two groups didn’t differ at Month-12 (least squares mean [LSM] difference 1.04%; p = 0.8282). MRI fat fraction didn’t change as well. The third study tested the effect of Givinostat in DMD and showed the geometric LSM ratio at 12-months of treatment was 1·27 (95% CI 1·17–1·37) in givinostat group and 1·48 (1·32–1·66) in placebo group (p=0·035). Our systematic review revealed that givinostat has favourable outcome in functional tests and histological findings in DMD patients, but not in patients with BMD.
Audience Take Away Notes:
- Recently many medications have been tested for its efficacy in Muscular dystrophy, our abstract is summarizing the currently evidence which may aid child neurologists in updating them regards the latest evidence of its use.
- It will help neurologists in tailoring their choice of medications for patients with Muscular dystrophy.
- Yes, this research that other faculty could use to expand their research or teaching.
- Yes, our abstract summarizes the current evidence on Givinostat in Muscular dystrophy which would save practitioners time looking for all clinical trials evidence on Givinostat.
- Yes, our abstract clearly shows the need of bigger and longer randomized clinical trial to support the current evidence.
