학술논문

Trends in phenotype in the English paediatric neurofibromatosis type 2 cohort stratified by genetic severity.
Document Type
Article
Source
Clinical Genetics. Aug2019, Vol. 96 Issue 2, p151-162. 12p. 6 Charts.
Subject
*NEUROFIBROMATOSIS 2
*VESTIBULAR nerve
*ACOUSTIC neuroma
*SPINAL surgery
*PHENOTYPES
Language
ISSN
0009-9163
Abstract
Childhood onset neurofibromatosis type 2 can be severe and genotype dependent. We present a retrospective phenotypic analysis of all ascertained children in England 1.0). Focal cortical dysplasia occurred in 26% group 3 and 4% 2A. A total of 48% of group 3 underwent ≥1 major intervention (intracranial/spinal surgery/Bevacizumab/radiotherapy) compared to 35% of 2A; with 23% group 3 undergoing spinal surgery (schwannoma/ependymoma/meningioma resection) compared to 4% of 2A. Mean age starting Bevacizumab was 12.7 in group 3 and 14.9 years in 2A. In conclusion, group 3 phenotype manifests earlier with greater tumour load, poorer visual outcomes and more intervention. [ABSTRACT FROM AUTHOR]