Efficacy of mTOR inhibitors (sirolimus) in isolated limb overgrowth: a systematic review.

We systematically assessed the literature on the use of sirolimus for the treatment of isolated limb overgrowth conditions and its various modalities of administration in PubMed, Scopus, Ovid MEDLINE, Web of Science, Google Scholar, Cochrane Database of Systematic Reviews, references of journals and grey literature using pre-trialled Medical Subject Headings terms and articles. Eleven articles were included, and 39 patients were identified for review. Sirolimus was given orally in 38 patients and topically in one patient. Sirolimus was found to be highly effective in treatment of isolated limb overgrowth conditions with improvement of symptoms (physical, emotional, social) reported in all but one patient. Dosage and adverse effects seemed to be closely correlated. The result of our study suggests that sirolimus should be considered as an adjuvant or first-line management in isolated limb overgrowth in prospective trials.