Juvenile nasopharyngeal angiofibroma (JNA) is a pathologically benign yet locally aggressive and destructive tumor that develops in the choana and nasopharynx. Historical treatment of JNA has included embolization, surgical resection, and radiation. mTOR signaling way demonstrated to be involved in regulation of growth and angiogenesis of JNA. Sirolimus, as mTOR inhibitor, is a potential target JNA therapy.
The main purpose of the study is to evaluate the efficacy and safety of sirolimus in children and adolescents with primary or recurrent JNA. Efficacy will be estimated based on dynamics of the JNA progression. Historical control group will be used for comparison as standard therapy.
Due to limited experience of sirolimus in JNA in routine practice, study should be conducted in 2 phases: pilot and extended. Decision regarding extended phase will be based on the results of pilot phase.
Confirmed diagnosis of JNA.
Male gender.
Age 7-18 years.
Signed informed consent of the parents or the official representative for patients under 14 years of age, the patient and the patient's parents for persons from 14 to 18 years of age.
None
Single group
Sirolimus
DrugSirolimus 0,8 mg/m2, but \<2 mg per day, once a day (QD), per os, oral solution, 1mg/ml, 60 ml Optimal individual dosage of sirolimus will be determined under control of concentration in blood serum. Therapeutic concentration - 5-15 ng/ml. Blood samples will be collected until therapeutic concertation will be achieved in 2 consecutive samples, but no more than 4 samples (on the 4th, 7th, 10th, 14th days of admission).
The proportion of patients with JNA with positive dynamics
The proportion of patients with JNA with positive dynamics (reduction of tumor volume or stabilization of tumor volume or increase in tumor volume \< 20% of the initial)
The proportion of patients with JNA with positive dynamics
Primary JNA: The proportion of patients with JNA with positive dynamics (reduction of tumor volume or stabilization of tumor volume or increase in tumor volume \< 20% of the initial one
The proportion of patients with JNA with positive dynamics
The proportion of patients with JNA with positive dynamics (reduction of tumor volume or stabilization of tumor volume or increase in tumor volume \< 20% of the initial one
proportion of patients with progression after sirolimus therapy initiation
proportion of patients with progression after sirolimus therapy initiation. Progression define as increase of JNA in volume \>20% from initial one.
The number and proportion (%) of patients receiving sirolimus without tumor progression in post-surgery catamnesis.
Optimization of sirolumus dosage regime: Control of sirolimus concentration in blood serum. Therapeutic concentration - 5-15 ng/ml.
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