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November 19, 2013; 81 (21 supplement 1) Response evaluation in neurofibromatosis and schwannomatosis (REiNS)

Achieving consensus for clinical trials

The REiNS International Collaboration

Scott R. Plotkin, Jaishri O. Blakeley, Eva Dombi, Michael J. Fisher, C. Oliver Hanemann, Karin S. Walsh, Pamela L. Wolters, Brigitte C. Widemann
First published November 18, 2013, DOI: https://doi.org/10.1212/01.wnl.0000435743.49414.b6
Scott R. Plotkin
From the Neurology Department and Cancer Center (S.R.P.), Massachusetts General Hospital, Boston, MA; Department of Neurology, Neurosurgery, and Oncology (J.O.B.), Johns Hopkins, Baltimore, MD; Pediatric Oncology Branch (E.D., P.L.W., B.C.W.), National Cancer Institute, Bethesda, MD; Division of Oncology, Department of Pediatrics (M.J.F.), The Children's Hospital of Pennsylvania, Philadelphia; Plymouth University Peninsula Schools of Medicine and Dentistry (C.O.H.), Plymouth, United Kingdom; and The Jennifer and Daniel Gilbert Neurofibromatosis Institute (K.S.W.), Children's National Medical Center, Washington, DC.
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Jaishri O. Blakeley
From the Neurology Department and Cancer Center (S.R.P.), Massachusetts General Hospital, Boston, MA; Department of Neurology, Neurosurgery, and Oncology (J.O.B.), Johns Hopkins, Baltimore, MD; Pediatric Oncology Branch (E.D., P.L.W., B.C.W.), National Cancer Institute, Bethesda, MD; Division of Oncology, Department of Pediatrics (M.J.F.), The Children's Hospital of Pennsylvania, Philadelphia; Plymouth University Peninsula Schools of Medicine and Dentistry (C.O.H.), Plymouth, United Kingdom; and The Jennifer and Daniel Gilbert Neurofibromatosis Institute (K.S.W.), Children's National Medical Center, Washington, DC.
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Eva Dombi
From the Neurology Department and Cancer Center (S.R.P.), Massachusetts General Hospital, Boston, MA; Department of Neurology, Neurosurgery, and Oncology (J.O.B.), Johns Hopkins, Baltimore, MD; Pediatric Oncology Branch (E.D., P.L.W., B.C.W.), National Cancer Institute, Bethesda, MD; Division of Oncology, Department of Pediatrics (M.J.F.), The Children's Hospital of Pennsylvania, Philadelphia; Plymouth University Peninsula Schools of Medicine and Dentistry (C.O.H.), Plymouth, United Kingdom; and The Jennifer and Daniel Gilbert Neurofibromatosis Institute (K.S.W.), Children's National Medical Center, Washington, DC.
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Michael J. Fisher
From the Neurology Department and Cancer Center (S.R.P.), Massachusetts General Hospital, Boston, MA; Department of Neurology, Neurosurgery, and Oncology (J.O.B.), Johns Hopkins, Baltimore, MD; Pediatric Oncology Branch (E.D., P.L.W., B.C.W.), National Cancer Institute, Bethesda, MD; Division of Oncology, Department of Pediatrics (M.J.F.), The Children's Hospital of Pennsylvania, Philadelphia; Plymouth University Peninsula Schools of Medicine and Dentistry (C.O.H.), Plymouth, United Kingdom; and The Jennifer and Daniel Gilbert Neurofibromatosis Institute (K.S.W.), Children's National Medical Center, Washington, DC.
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C. Oliver Hanemann
From the Neurology Department and Cancer Center (S.R.P.), Massachusetts General Hospital, Boston, MA; Department of Neurology, Neurosurgery, and Oncology (J.O.B.), Johns Hopkins, Baltimore, MD; Pediatric Oncology Branch (E.D., P.L.W., B.C.W.), National Cancer Institute, Bethesda, MD; Division of Oncology, Department of Pediatrics (M.J.F.), The Children's Hospital of Pennsylvania, Philadelphia; Plymouth University Peninsula Schools of Medicine and Dentistry (C.O.H.), Plymouth, United Kingdom; and The Jennifer and Daniel Gilbert Neurofibromatosis Institute (K.S.W.), Children's National Medical Center, Washington, DC.
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Karin S. Walsh
From the Neurology Department and Cancer Center (S.R.P.), Massachusetts General Hospital, Boston, MA; Department of Neurology, Neurosurgery, and Oncology (J.O.B.), Johns Hopkins, Baltimore, MD; Pediatric Oncology Branch (E.D., P.L.W., B.C.W.), National Cancer Institute, Bethesda, MD; Division of Oncology, Department of Pediatrics (M.J.F.), The Children's Hospital of Pennsylvania, Philadelphia; Plymouth University Peninsula Schools of Medicine and Dentistry (C.O.H.), Plymouth, United Kingdom; and The Jennifer and Daniel Gilbert Neurofibromatosis Institute (K.S.W.), Children's National Medical Center, Washington, DC.
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Pamela L. Wolters
From the Neurology Department and Cancer Center (S.R.P.), Massachusetts General Hospital, Boston, MA; Department of Neurology, Neurosurgery, and Oncology (J.O.B.), Johns Hopkins, Baltimore, MD; Pediatric Oncology Branch (E.D., P.L.W., B.C.W.), National Cancer Institute, Bethesda, MD; Division of Oncology, Department of Pediatrics (M.J.F.), The Children's Hospital of Pennsylvania, Philadelphia; Plymouth University Peninsula Schools of Medicine and Dentistry (C.O.H.), Plymouth, United Kingdom; and The Jennifer and Daniel Gilbert Neurofibromatosis Institute (K.S.W.), Children's National Medical Center, Washington, DC.
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Brigitte C. Widemann
From the Neurology Department and Cancer Center (S.R.P.), Massachusetts General Hospital, Boston, MA; Department of Neurology, Neurosurgery, and Oncology (J.O.B.), Johns Hopkins, Baltimore, MD; Pediatric Oncology Branch (E.D., P.L.W., B.C.W.), National Cancer Institute, Bethesda, MD; Division of Oncology, Department of Pediatrics (M.J.F.), The Children's Hospital of Pennsylvania, Philadelphia; Plymouth University Peninsula Schools of Medicine and Dentistry (C.O.H.), Plymouth, United Kingdom; and The Jennifer and Daniel Gilbert Neurofibromatosis Institute (K.S.W.), Children's National Medical Center, Washington, DC.
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Citation
Achieving consensus for clinical trials
The REiNS International Collaboration
Scott R. Plotkin, Jaishri O. Blakeley, Eva Dombi, Michael J. Fisher, C. Oliver Hanemann, Karin S. Walsh, Pamela L. Wolters, Brigitte C. Widemann
Neurology Nov 2013, 81 (21 supplement 1) S1-S5; DOI: 10.1212/01.wnl.0000435743.49414.b6

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Abstract

The neurofibromatoses (NF)—including neurofibromatosis 1 (NF1), neurofibromatosis 2 (NF2), and schwannomatosis—are related tumor-suppressor syndromes characterized by a predisposition to multiple tumor types and other disease manifestations, which often result in functional disability, reduced quality of life, pain, and, in some cases, malignancy. With increasing knowledge of the biology and pathogenesis of NF, clinical trials with targeted agents directed at NF tumors have become available. Most clinical trials for patients with NF have used designs and endpoints similar to oncology trials. However, differences in the disease manifestations and natural history of NF (compared to cancers) require the development of new designs and endpoints to perform meaningful NF clinical trials. The Response Evaluation in Neurofibromatosis and Schwannomatosis (REiNS) International Collaboration was established in 2011 at the Children's Tumor Foundation meeting to achieve consensus within the NF community about the design of future clinical trials, with a specific emphasis on endpoints. The REiNS Collaboration includes 7 working groups that focus on imaging of tumor response; functional, visual, patient-reported, and neurocognitive outcomes; whole-body MRI; and disease biomarkers. This supplement includes the first series of recommendations by the REiNS Collaboration. The hope is that these recommendations will be used by members of the group and by researchers outside of the REiNS International Collaboration to standardize the measurement of outcomes and thus improve clinical trials for patients with NF. Ultimately, we plan to engage industry partners and national regulatory agencies in this process to facilitate the approval of drugs for patients with NF.

Glossary

FDA=
US Food and Drug Administration;
NF=
neurofibromatosis;
REiNS=
Response Evaluation in Neurofibromatosis and Schwannomatosis

Footnotes

  • REiNS International Collaboration members are listed on the Neurology® Web site at www.neurology.org.

  • Go to Neurology.org for full disclosures. Funding information and disclosures deemed relevant by the authors, if any, are provided at the end of the article.

  • Supplemental data at www.neurology.org

  • Received May 9, 2013.
  • Accepted in final form August 13, 2013.
  • © 2013 American Academy of Neurology
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  • Article
    • Abstract
    • Glossary
    • ORGANIZATION OF THE REiNS INTERNATIONAL COLLABORATION
    • SUMMARY OF PUBLISHED CLINICAL TRIALS FOR NEUROFIBROMATOSIS
    • WHY CURRENT ENDPOINTS ARE NOT APPROPRIATE FOR NF TRIALS
    • FUTURE GOALS/PLANS
    • AUTHOR CONTRIBUTIONS
    • STUDY FUNDING
    • DISCLOSURE
    • Acknowledgment
    • Footnotes
    • REFERENCES
  • Figures & Data
  • Info & Disclosures
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Topics Discussed

  • All Pediatric
  • Neurofibromatosis
  • Clinical trials Methodology/study design
  • Nerve tumor

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