Considerations for development of therapies for cutaneous neurofibroma.

Objective: The only therapies currently available for cutaneous neurofibromas (cNF) are procedural. The goals of the Therapies Development Working Group were to (1) summarize currently available treatment options for cNF, (2) define key considerations for drug discovery and development generally, and specifically for cNF, and (3) outline recommendations for the successful development of medical therapies for cNF.

Methods: The subgroup reviewed published and unpublished data on procedural, drug/device, and medical treatment approaches utilized for cNFs via literature search.

A high-throughput molecular data resource for cutaneous neurofibromas.

Neurofibromatosis type 1 (NF1) is a genetic disorder with a range of clinical manifestations such as widespread growth of benign tumours called neurofibromas, pain, learning disorders, bone deformities, vascular abnormalities and even malignant tumours. With the establishment of the Children's Tumour Foundation biobank, neurofibroma samples can now be collected directly from patients to be analysed by the larger scientific community. This work describes a pilot study to characterize one class of neurofibroma, cutaneous neurofibromas, by molecularly profiling of ~40 cutaneous neurofibromas collected from 11 individual patients.

Electrosurgical excision technique for the treatment of multiple cutaneous lesions in neurofibromatosis type I.

Neurofibromatosis I (NF1) is an autosomal dominant disease that presents with multiple cutaneous lesions often numbering into the 500-1000 range. In addition to the psychosocial implications, there are limited surgical options for this condition. A series of 97 consecutive patients with NF1 presented with numerous cutaneous lesions.