Background With a dramatic expansion in stem cell research, there is a conflict in the literature whether stem cell therapy is effective and safe in muscular dystrophies.

Methods We performed electronic searches on Web of Science, PubMed, Embase, and Cochrane library from inception to August 2023 to identify studies on the efficacy and safety of stem cell therapy compared to usual care in muscular dystrophies. The Risk of Bias version 2 tool was used for quality assessment.

Results Of 4,328 studies screened, 23 were assessed. Twenty studies had limitations of non-randomized controlled trial (RCT) design. Three RCTs (53 patients with Duchenne muscular dystrophy) were included, and of these, two had low risk of bias. Two studies were from the USA with majorly non-ambulatory participants and one from Europe with ambulatory. The nature of stem cell therapies included autologous transplantation of muscle-derived CD133+ stem cells in abductor digiti minimi muscle (n=1) and allogeneic cardiosphere-derived cells (n=2, one intracoronary and one intravenous route). Studies failed to evaluate critical motor efficacy outcomes (e.g., improvement in muscle strength beyond six months, independent ambulation, need for respiratory support). However, improvement in performance of upper limb function at 12-mo (n=2), scar size reduction and improvement in inferior wall systolic thickening at 12-mo by MRI (n=1) was identified. No serious adverse events causally related to stem cell therapies were identified.

Interpretation Considering a few RCTs and clinical heterogeneity, the systematic review does not identify high-quality evidence demonstrating improved critical outcomes with stem cell therapy for muscular dystrophies.

Parth Lal
Postgraduate Institute of Medical Education and Research
India

Akansha Singh
National Institute of Pharmaceutical Education and Research
India

Nagita Devi
National Institute of Pharmaceutical Education and Research
India

Sandeep Negi
Postgraduate Institute of Medical Education and Research
India

Kiran Parkash
Government Medical College and Hospital, Sector 32
India

Renu Suthar
Postgraduate Institute of Medical Education and Research
India

Dipika Bansal
National Institute of Pharmaceutical Education and Research
India

Jitendra Kumar Sahu
Postgraduate Institute of Medical Education and Research
India