Sirolimus as treatment for 19 patients with refractory cervicofacial lymphatic malformation.

OBJECTIVE

Lymphatic malformations (LMs) are challenging to manage, particularly those involving the cervicofacial region and airway. Traditional therapy is sclerotherapy and/or resection. We aim to establish the emerging therapeutic role of sirolimus.

STUDY

DESIGN

Institutional review board-approved retrospective review.

METHODS

All patients treated for cervicofacial LM with sirolimus at Boston Children's Hospital, Massachusetts, from November 2012 to October 2016 were included. Chart review included response to therapy (defined as reduction in LM bulk by clinical photographs and radiologic imaging), type of LM (microcystic, macrocystic, mixed), extent of disease, duration of therapy, patient/parent-reported quality-of-life, airway status (tracheostomy dependence), and complications (opportunistic infection, hemorrhage, other). Follow-up and clinical outcomes were included up until October 2016.

RESULTS

Nineteen patients were treated with sirolimus for cervicofacial LM from November 2012 to October 2016 at Boston Children's Hospital. Seven patients remain on uninterrupted sirolimus. Of 12 patients who stopped therapy, seven have resumed due to recurrence of symptoms. All patients demonstrated reduction in LM bulk, ranging from modest to significant. All patients with mucosal vesicles (n = 14) resolved or improved on sirolimus. Six patients developed cellulitis, and four had bleeding within the LM during treatment. No patients developed opportunistic or systemic bacterial infection.

CONCLUSION

The use of sirolimus in the management of cervicofacial LM often is efficacious, with limited adverse events. Long-term follow-up, durability of response, and coordination of sirolimus prior to procedural therapies need further study.

LEVEL OF EVIDENCE: 4. Laryngoscope, 128:269-276, 2018.