Safety and efficacy of sirolimus in patients with refractory vascular anomalies

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

“…Tissue growth attributed to pure vascular anomalies in nine cases (Adams et al., 2016; Iacobas et al., 2011; Oza et al., 2016; Parker et al., 2019; Yesil et al., 2016; Youssef et al., 2019).…”

“…In ten studies (Adams et al., 2016; Bessis et al., 2016; Iacobas et al., 2011; Oza et al., 2016; Parker et al., 2014, 2019; Pinto et al., 2018; Sandbank et al., 2019; Yesil et al., 2016; Youssef et al., 2019), data on the duration of treatment ranged from 26 weeks (Parker et al., 2019) to 26 months (Oza et al., 2016). Yesil et al.…”

“…Sirolimus was used as an alternative treatment modality, especially after poor response, failure, worsening and/or recurrence of symptoms after use of the previous treatment options (Adams et al, 2016;Bessis et al, 2016;Erickson et al, 2017;Iacobas et al, 2011;Oza et al, 2016;Parker et al, 2014;Pinto et al, 2018;Sandbank et al, 2019;Yesil et al, 2016). In other studies, sirolimus was used mainly to evaluate the safety and efficacy in patients with PROS (Parker et al, 2019) and vascular anomalies (Adams et al, 2016;Sandbank et al, 2019;Youssef et al, 2019). Sirolimus was given systemically in all but one case, where it was administered topically.…”

“…Combined tissue overgrowth/vascular anomalies in 12 cases (Bessis et al., 2016; Erickson et al., 2017; Parker et al., 2019; Sandbank et al., 2019; Youssef et al., 2019).…”

“…In ten studies (Adams et al, 2016;Bessis et al, 2016;Iacobas et al, 2011;Oza et al, 2016;Parker et al, 2014Parker et al, , 2019Pinto et al, 2018;Sandbank et al, 2019;Yesil et al, 2016;Youssef et al, 2019), data on the duration of treatment ranged from 26 weeks (Parker (Oza et al, 2016). Yesil et al (2016) stopped treatment after 1 year based on positive findings and due to insufficient data in the literature regarding late side effects.…”