How Effective is Sirolimus for Slow-Flow Vascular Malformations in Children?

Sirolimus does not impact volume changes detected on MRI scans, but does reduce pain, oozing, bleeding

By Dermsquared Editorial Team | September 15, 2021

Sirolimus treatment has no effect on volume changes detected on magnetic resonance imaging (MRI) scans of children with slow-flow vascular malformations, according to a study published online Sept. 15 in JAMA Dermatology.

Annabel Maruani, M.D., Ph.D., from the University of Nantes in France, and colleagues examined the efficacy and safety of sirolimus for children with slow-flow vascular malformations in a multicenter, observational clinical trial involving 59 children aged 6 to 18 years. Patients underwent an observational period followed by an interventional period when they received oral sirolimus; the switch time was randomly assigned from month 4 to 8. The primary outcome was the change in the volume of vascular malformations detected on MRI scan.

The researchers observed no significant difference between the interventional period and the observational period in the variations in the volume of vascular malformations detected on MRI scans. For children with pure lymphatic malformations, there was a significant decrease noted in volume. Positive effects of sirolimus were seen on pain, especially for combined malformations, and on bleeding, oozing, self-assessed efficacy, and quality of life. There were 231 adverse events reported overall during sirolimus treatment, with the most frequent being mouth ulcer (49.2 percent).

"Questions remain on the optimal age to initiate sirolimus therapy to potentially prevent an increase in the volume of vascular malformations and on how long treatment must be maintained," the authors write.

Several authors disclosed financial ties to the pharmaceutical industry.

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