Efficacy of Rapamycin in the Treatment of Cervico-facial Lymphatic Malformations

Inclusion Criteria29

• Patient from 0 to 18 years of age, presenting with poly-cystic suprahyoid or mediastinal lymphatic.
• with chronic pain or functional respiratory or swallowing impairment with a CDS score \< 8
• Curative treatment is not possible or associated with a high risk of morbidity ,mortality and functional and cosmetic impairment
• Karnofsky Score (\> 10 years of age) or Lansky score (≤10 years of age) \> 50%
• Biology
• Neutrophils count≥1.0 x 109/L
• Platelets count ≥ 100 x 109/L
• Hemoglobin ≥ 8 g/dL
• Bilirubin ≤ 1,5 ULN
• Transaminases \< 2,5 ULN
• Serum albumin ≥ 2 g/dL.
• LDL cholesterol \<160 mg/dL
• Triglycerides \< 150 mg/dL
• Negative test of pregnancy if relevant
• Social security affiliation
• At least 2 months after a previous procedure on the malformation
• Other immunosuppressive therapy or long-term general corticosteroid therapy without a 28-day washout period
• renal failure
• Liver failure
• Digestive disease leading to rapamycin malabsorption
• uncontrolled or severe infectious disease
• Patients requiring treatment interfering with CYP3A4 isoenzyme (rifampicin, rifabutin, carbamazepine, phenobarbital, phenytoin) or inhibiting CYP3A4 isoenzyme's activity (ketoconazole, voriconazole, itraconazole, telithromycin, clarithromycin, Diltiazem, Verapamil, nicardipine, clotrimazole, fluconazole , troleandomycin, bromocriptine, cimetidine, danazol, protease inhibitors) -patients requiring treatment by cisapride and metoclopramide
• Concomitant administration of mTOR inhibitor
• Peanuts or soya allergy
• Impossibility to receive informed consent
• Absence of social security affiliation
• refusal to sign consent
• Ongoing pregnancy or breastfeeding
• refusal to participate