Cutaneous Neurofibromas and Quality of Life in Adults With Neurofibromatosis Type 1.

ABSTRACT:

Importance

There is a burgeoning interest in therapeutic development for cutaneous neurofibromas (cNFs), a major cause of morbidity in persons with neurofibromatosis type 1 (NF1). To determine meaningful clinical trial outcomes, deeper understanding is needed regarding how cNFs are associated with quality of life (QoL). However, this understanding has been hampered by challenges in recruiting participants with this rare genetic disease.

Objective

To develop a large, crowdsourced validated registry of persons with NF1 and determine the association of specific cNF features with QoL, pain, and itch.

Design, setting, and participants

From May 2021 to December 2023, a decentralized platform was developed and recruited persons 40 years or older with NF1 and at least 1 cNF f

SUBMITTER: Lin MJ 

PROVIDER: S-EPMC11359107 | biostudies-literature | 2024 Oct

REPOSITORIES: biostudies-literature