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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
28
oral administration
cervicofacial MRI
Biological dosage of Rapamycin level
Hôpital Jeanne de Flandres, CHU
Lille, France
RECRUITINGHu Robert Debre Aphp - Paris
Paris, France
RECRUITINGResponse rate to rapamycin
Volumetric assessment by MRI. A response is considered as positive if volume decrease is superior to 1/5th of the initial volume.
Time frame: At 3 months
Kinetic of rapamycin response
MRI assessment of the volume
Time frame: At 3, 6 and 12 months
Efficacy of rapamycin on clinical symptoms
Clinical and fiberscopy evaluation by scoring
Time frame: At 3, 6 and 12 months
Pediatric Quality of Life Inventory (PedsQL 4) Scales
Assesses health-related quality of life among children with chronic and acute diseases
Time frame: Baseline, at 3, 6 and 12 months
Biological response to rapamycin
biological effect of mTOR blockage by measuring pAKT, p70S6 kinase, pMEK, and VEGF C, VEGFR3
Time frame: Baseline and at 6 months
Rapamycin side effects
Side effect assessment using the NCI-CTC 3.0 scale
Time frame: Monthly during 1 years
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.