Rapamycin dosage: Biological dosage of Rapamycin level
Study summary
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
Sex
ALL
Min age
—
Max age
18 Years
Healthy volunteers
No
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
Primary outcome measure(s)
Response rate to rapamycin — At 3 months Volumetric assessment by MRI. A response is considered as positive if volume decrease is superior to 1/5th of the initial volume.
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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