Management of neurofibromatosis type 1-associated plexiform neurofibromas

Author:

Fisher Michael J1,Blakeley Jaishri O2ORCID,Weiss Brian D3,Dombi Eva4ORCID,Ahlawat Shivani5,Akshintala Srivandana4,Belzberg Allan J6,Bornhorst Miriam7ORCID,Bredella Miriam A8,Cai Wenli8,Ferner Rosalie E9,Gross Andrea M4,Harris Gordon J8,Listernick Robert10,Ly Ina11,Martin Staci4,Mautner Victor F12,Salamon Johannes M13,Salerno Kilian E14,Spinner Robert J15,Staedtke Verena2,Ullrich Nicole J16,Upadhyaya Meena17,Wolters Pamela L4,Yohay Kaleb18,Widemann Brigitte C4

Affiliation:

1. Division of Oncology, The Children’s Hospital of Philadelphia and the University of Pennsylvania Perelman School of Medicine , Philadelphia, Pennsylvania , USA

2. Division of Neuro-Oncology, Department of Neurology, Johns Hopkins University School of Medicine , Baltimore, Maryland , USA

3. Division of Oncology, Cincinnati Children’s Hospital Medical Center, University of Cincinnati College of Medicine , Cincinnati, Ohio , USA

4. Pediatric Oncology Branch, National Cancer Institute , Bethesda, Maryland , USA

5. Russell H. Morgan Department of Radiology & Radiological Science, Johns Hopkins University School of Medicine , Baltimore, Maryland , USA

6. Department of Neurosurgery, Johns Hopkins University School of Medicine , Baltimore, Maryland , USA

7. Family Neurofibromatosis Institute, Center for Neuroscience and Behavioral Medicine,Children’s National Hospital, Washington, District of Columbia , USA

8. Department of Radiology, Massachusetts General Hospital and Harvard Medical School , Boston, Massachusetts , USA

9. Neurofibromatosis Service, Department of Neurology, Guy’s Hospital, Guy’s & St. Thomas’ NHS Foundation Trust , London , UK

10. Department of Pediatrics, Ann & Robert H. Lurie Children’s Hospital of Chicago, Feinberg School of Medicine, Northwestern University , Chicago, Illinois , USA

11. Stephen E. and Catherine Pappas Center for Neuro-Oncology, Massachusetts General Hospital , Boston, Massachusetts , USA

12. Department of Neurology, University Medical Center Hamburg-Eppendorf , Hamburg , Germany

13. Department for Diagnostic and Interventional Radiology and Nuclear Medicine, University Medical Center Hamburg-Eppendorf , Hamburg , Germany

14. Radiation Oncology Branch, National Cancer Institute , Bethesda, Maryland , USA

15. Department of Neurologic Surgery, Mayo Clinic , Rochester, Minnesota , USA

16. Department of Neurology, Boston Children’s Hospital , Boston, Massachusetts , USA

17. Division of Cancer and Genetics, Cardiff University , Wales , UK

18. Grossman School of Medicine, Department of Neurology , New York, New York , USA

Abstract

Abstract Plexiform Neurofibromas (PN) are a common manifestation of the genetic disorder neurofibromatosis type 1 (NF1). These benign nerve sheath tumors often cause significant morbidity, with treatment options limited historically to surgery. There have been tremendous advances over the past two decades in our understanding of PN, and the recent regulatory approvals of the MEK inhibitor selumetinib are reshaping the landscape for PN management. At present, there is no agreed upon PN definition, diagnostic evaluation, surveillance strategy, or clear indications for when to initiate treatment and selection of treatment modality. In this review, we address these questions via consensus recommendations from a panel of multidisciplinary NF1 experts.

Funder

AstraZeneca

Array BioPharma

Exelixis

Publisher

Oxford University Press (OUP)

Subject

Cancer Research,Neurology (clinical),Oncology

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