Neurofibromatosis Clinical Trials—REiNS Collaboration 2020 Recommendations

Abstract

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A Systematic Review of Recent and Ongoing Clinical Trials in Patients With the Neurofibromatoses

“…The Response Evaluation in Neurofibromatosis and Schwannomatosis (REiNS) International Collaboration has grown and developed as a response to such need, providing recommendations for clinical trials including those involving selumetinib. 33 Recommendations set forth by the REiNS concerning primary outcome measures play a critical role in the design and execution of future studies, thus also having the potential to impact which areas of study might be prioritized by the scientific community. In this way, the REiNS can have an impact among areas that have been understudied in previous years resulting in greater attention, especially as it seeks to increase patient engagement in clinical trial design.…”

“…In this way, the REiNS can have an impact among areas that have been understudied in previous years resulting in greater attention, especially as it seeks to increase patient engagement in clinical trial design. 33 Notably, the REiNS includes several working groups with emphasis on different areas, including focusing on developing recommendations for reproducible functional outcomes measures. 34 This highlights the need to incorporate measures other than tumor size, such as hearing, visual acuity, or other changes from impairments due to tumor location.…”

A Chinese Collaborative Model for Accelerating Neurofibromatosis Type 1-Associated Research

“…These effective platform trials will bene t all forms of NF1 individuals with better allocation of promising treatments. To improve clinical trial response criteria, an international collaborative group called REINS was formed in 2011 dedicated to developing new standardized consensuses for clinical trials of neuro bromatosis [25][26][27] . This collaboration works on different speci c areas and makes recommendations on disease biomarkers, cutaneous neuro bromas, imaging of tumor response, and functional, neurocognitive and patient-reported outcomes.…”

Advancing neurofibromatosis and schwannomatosis clinical trial design: Consensus recommendations from the Response Evaluation in Neurofibromatosis and Schwannomatosis (REiNS) International Collaboration

“…Three previous supplements published in Neurology ® in 2013, and 2016, and 2021 set forth a variety of recommendations for clinical trial design, gathered stakeholder perspectives on clinical trials, and generated empirical evidence on the reliability of proposed outcomes. 4 – 6 A summary of all currently recommended measures is available at the REiNS website at https://ccrod.cancer.gov/confluence/display/REINS/Recommendations ; these include the use of volumetric magnetic resonance imaging (MRI) analysis to assess response in clinical trials and the use of various functional and patient-reported outcome measures to evaluate clinical benefit. Many of these measures were incorporated into the SPRINT registration trial, which established the efficacy of selumetinib for treatment of inoperable plexiform neurofibromas in pediatric patients with NF1.…”