Overview
To evaluate the efficacy of Rapamycin in extended cervicofacial lymphatic malformations in pediatric patients. Rapamycin is administered oral for a 6 month period.
The success rate is determined by volume reduction superior to 1/5e of the initial volume measured by MRI, impact on QOL and reduction of bleeding in case of mucosal involvement.
Eligibility
Inclusion Criteria:
- 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
Exclusion Criteria:
- Non-respect of inclusion criteria
- 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