Enhancing patient engagement in neurofibromatosis clinical trials

Massachusetts General Hospital, Boston MA

Investigators

Abstract

Project Summary Neurofibromatosis 1 (NF1), neurofibromatosis 2 (NF2), and schwannomatosis are a group of related neurogenetic tumor suppressor syndromes that share a predisposition to develop benign and malignant nerve sheath tumors. Because most tumors encountered in patients with NF1, NF2, and schwannomatosis are histologically benign, overall survival is not an appropriate clinical trial outcome for these patients. In 2011, a group of NF researchers agreed to meet periodically to develop novel outcomes for these conditions. The Response Evaluation in Neurofibromatosis and Schwannomatosis (REiNS) International Collaboration consists of specialists from multiple countries and disciplines dedicated to the study of neurofibromatosis, schwannomatosis, and clinical trials. REiNS holds an annual conference during the winter in which recommendations for clinical trial outcomes are discussed. Each year, the Leadership Group of REiNS selects experts on the topics under discussion. These meetings have led to multiple publications that have been incorporated into ongoing clinical trials and that have been published in the peer-reviewed literature. The annual REiNS meeting is a critical forum for generating new ideas and approaches to these rare tumor suppressor syndromes. The 2016 conference topic is Enhancing patient engagement in neurofibromatosis clinical trials and will include outside speakers from the Patient-Centered Outcomes Research Institute (PCORI), the Outcome Measures in Rheumatology initiative (OMERACT), the Food and Drug Administration (FDA), and the National Cancer Institute. The ultimate goal of this conference is to develop patient engagement guidelines for the REiNS International Collaboration.