How to score the impact of treatment on cutaneous neurofibromas in clinical trials

Abstract

Most patients with neurofibromatosis 1 (NF1) develop a variable number of cutaneous neurofibromas (cNFs) which have a major impact on patients’ quality of life0. A major problem is how to score the response to therapy in clinical practice and trials. A measurement instrument recognized by all experts in the field is required to be able to judge the efficacy of treatments and the benefits perceived by the patients. Fertitta and colleagues propose a system for assessing cNFs severity and impact with a list of items validated by a consensus of experts and patients.This qualitative approach has the advantage of integrating multiple parameters of interest, based in part on a “patient-centric” approach.