One of the most difficult therapeutic dilemmas we face is how to successfully treat complicated vascular anomalies. Determining the approach to therapy is challenging because of the variety of different types of lesions and the lack of data comparing the effectiveness of different therapeutic options. This week Adams et al. (10.1542/peds.2015-3257) share the results of a phase 2 trial of sirolimus, an immunosuppressive agent, in terms of the efficacy and safety of using sirolimus as a treatment for these lesions. Outcomes measured included responsiveness to treatments (captured by functional impairment, quality of life, and radiologic studies) and toxicities and/or infection-related deaths.
The results are optimistic for the majority but still not complication-free for a minority of patients in the trial. While no patient had a complete response after 12 courses of sirolimus (dosing schedule discussed in the article), the number of patients with a partial response is worth paying attention to. If you are following patients with vascular malformations, this is an article you should not miss to learn more about promising new therapies for this problem.
