Use of topical rapamycin in the treatment of superficial lymphatic malformations

• Published in: Journal of the American Academy of Dermatology Vol. 80; no. 2; pp. 508 - 515
• Main Authors: García-Montero, Pablo, del Boz, Javier, Baselga-Torres, Eulalia, Azaña-Defez, José Manuel, Alcaraz-Vera, Manuel, Tercedor-Sánchez, Jesús, Noguera-Morel, Lucero, Vera-Casaño, Ángel
• Format: Journal Article
• Language: English
• Published: United States Elsevier Inc 01-02-2019
• Subjects: Administration, Topical; Adolescent; Child; Child, Preschool; Cohort Studies; Female; Humans; Immunosuppressive Agents - therapeutic use; Lymphatic Abnormalities - diagnosis; Lymphatic Abnormalities - drug therapy; lymphatic malformation; Male; ointment; Prognosis; rapamycin; Retrospective Studies; Severity of Illness Index; sirolimus; Sirolimus - therapeutic use; topical; Treatment Outcome; vascular anomalies; LM; topical; ointment; vascular anomalies; sirolimus; lymphatic malformation; rapamycin
• ISSN: 0190-9622; 1097-6787
• DOI: 10.1016/j.jaad.2018.09.050

The superficial lymphatic component of vascular malformations poses a significant treatment challenge. It is responsible for the majority of symptoms presented, and to date, there is no consensus regarding treatment.
To evaluate the effectiveness of topical rapamycin in treating superficial lymphatic malformations (LM).
A case series study was performed of patients with superficial LM, treated with topical rapamycin. The clinical characteristics of patients and the concentration and application mode of the drug were recorded. The changes in the signs and symptoms observed and associated adverse effects were noted and analyzed.
The study population consisted of 11 patients of an average age of 10.5 years. All were treated with topical rapamycin: 6 patients with a 1% concentration, 1 with a 0.8% concentration, and 4 with a 0.4% concentration. Changes in the clinical appearance of the lesions were observed in all patients. The associated symptoms, present in 9 of 11 patients, improved in every case. The mean follow-up time was 16.1 months.
This study is retrospective, with a small sample size and considerable heterogeneity of lesions and treatment approaches.
Treatment with topical rapamycin modifies the clinical appearance and alleviates symptoms of superficial LM.