TY - JOUR
T1 - Status and Recommendations for Incorporating Biomarkers for Cutaneous Neurofibromas Into Clinical Research
AU - Wallis, Deeann
AU - Stemmer-Rachamimov, Anat
AU - Adsit, Sarah
AU - Korf, Bruce
AU - Pichard, Dominique
AU - Blakeley, Jaishri
AU - Sarin, Kavita Y.
N1 - Funding Information:
K.Y. Sarin is supported by NCI K23 CA211793 and is the D.G. “Mitch” Mitchell Clinical Investigator supported by the Damon Runyon Cancer Research Foundation (CI-104-19).
Funding Information:
The authors acknowledge the support of the Children's Tumor Foundation for the REiNS International Collaboration. The authors would also like to acknowledge the following collaborators for their participation in the REiNS cNF working group: Andrea Baldwin (National Cancer Institute), Andrés Lessing (REiNS patient representative), Ashley Cannon (University of Alabama at Birmingham, coordinating role for working group meetings), Elizabeth Schorry (Cincinnati Children's Hospital), Brigitte Widemann (National Cancer Institute), Christopher Moertel (University of Minnesota), Claas Röhl (REiNS patient representative), Dawn Siegel (Medical College of Wisconsin), Gregg Erickson (REiNS patient representative), Kaleb Yohay (New York University), Khaled Ezzedine (Assistance Publique–Hôpitaux de Paris), Krista Fredrick (REiNS patient representative), Maciej Mrugala (Mayo Clinic), Michael Fisher (Children's Hospital of Philadelphia), Pamela Wolters (National Cancer Institute), Pierre Wolkenstein (Hôpital Henri-Mondor), Robert Kesterson (University of Alabama at Birmingham), and Scott Plotkin (Massachusetts General Hospital, coordinating role for working group meetings).
Publisher Copyright:
© 2021 American Academy of Neurology.
PY - 2021/8/17
Y1 - 2021/8/17
N2 - Objective: To summarize existing biomarker data for cutaneous neurofibroma (cNF) and to inform the incorporation of biomarkers into clinical trial design for cNFs. Methods: The cNF working group, a subgroup of the Response Evaluation in Neurofibromatosis and Schwannomatosis (REiNS) consortium, was formed to review and inform clinical trial design for cNFs. Between June 2018 and February 2020, the cNF working group performed a review of existing data on genetic biomarkers for cNFs in the setting of neurofibromatosis type 1. We also reviewed criteria for successful biomarker application in the clinic. The group then held a series of meetings to develop a consensus report. Results: Our systematic literature review of existing data revealed a lack of validated biomarkers for cNFs. In our report, we summarize the existing signaling, genomic, transcriptomic, histopathologic, and proteomic data relevant to cNF. Finally, we make recommendations for incorporating exploratory aims for predictive biomarkers into clinical trials through biobanking samples. Conclusion: These recommendations are intended to provide both researchers and clinicians with best practices for clinical trial design to aid in the identification of clinically validated biomarkers for cNF.
AB - Objective: To summarize existing biomarker data for cutaneous neurofibroma (cNF) and to inform the incorporation of biomarkers into clinical trial design for cNFs. Methods: The cNF working group, a subgroup of the Response Evaluation in Neurofibromatosis and Schwannomatosis (REiNS) consortium, was formed to review and inform clinical trial design for cNFs. Between June 2018 and February 2020, the cNF working group performed a review of existing data on genetic biomarkers for cNFs in the setting of neurofibromatosis type 1. We also reviewed criteria for successful biomarker application in the clinic. The group then held a series of meetings to develop a consensus report. Results: Our systematic literature review of existing data revealed a lack of validated biomarkers for cNFs. In our report, we summarize the existing signaling, genomic, transcriptomic, histopathologic, and proteomic data relevant to cNF. Finally, we make recommendations for incorporating exploratory aims for predictive biomarkers into clinical trials through biobanking samples. Conclusion: These recommendations are intended to provide both researchers and clinicians with best practices for clinical trial design to aid in the identification of clinically validated biomarkers for cNF.
UR - http://www.scopus.com/inward/record.url?scp=85114521865&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=85114521865&partnerID=8YFLogxK
U2 - 10.1212/WNL.0000000000012426
DO - 10.1212/WNL.0000000000012426
M3 - Article
C2 - 34230199
AN - SCOPUS:85114521865
SN - 0028-3878
VL - 97
SP - S42-S49
JO - Neurology
JF - Neurology
IS - 7
ER -