Badanie skuteczności sirolimusu w leczeniu językowych mikrocytowych malformacji limfatycznych u dzieci i dorosłych

Lingual microcystic lymphatic malformations (LMLMs) are rare conditions that appear as clusters of small cysts filled with lymph fluid or blood. These cysts can cause problems such as oozing, bleeding, infections, and difficulties with speaking, chewing, or breathing. They can also lead to pain and affect appearance. The study aims to evaluate the effectiveness and safety of a treatment using a solution called sirolimus, which is applied directly to the affected area on the tongue. This treatment is compared to the usual care, which involves no treatment.

The study involves applying a 1 mg/mL solution of sirolimus once a day to the front part of the tongue where the malformation is located. The treatment is given to both children and adults over a period of 24 weeks. The progress is checked at different times: after 4, 8, 12, 16, 20, and 24 weeks. The main goal is to see if the treatment helps reduce the severity of the malformations compared to not receiving any treatment.

Throughout the study, the severity of the condition is assessed by experts using photographs, and patients also provide feedback on symptoms like oozing, bleeding, and discomfort. The study also monitors the safety of the treatment by checking for any side effects and measuring the levels of sirolimus in the blood. This helps ensure that the treatment is safe for use over the study period.