Translational/clinical studies in children and adults with neurofibromatosis type 1

Bruce Korf, Brigitte Widemann, Maria T. Acosta, Roger J. Packer

Research output: Chapter in Book/Report/Conference proceedingChapter

Abstract

Despite considerable challenges, the management of children and adults with NF1 has rapidly entered the therapeutic era. A multitude of new agents are available which could potentially benefit patients, and a clinical trial infrastructure is now in place to more effectively and expeditiously study new therapies. Almost all of the clinical manifestations of neurofibromatosis type 1 are therapeutic targets. Preclinical testing offers the possibility of identifying agents which are most likely to positively alter clinical outcome. Such preclinical work has already been translated into therapeutic trials for children and adults with NF1 and plexiform neurofibromas, gliomas, malignant peripheral nerve sheath tumors, and even neurocognitive disorders. Clinical trials are also being proposed for skeletal dysplasia and dermal neurofibromas. The potential benefits of such trials need to be carefully balanced against the potential long-term toxicity caused by such interventions and the realization that many manifestations of NF1 are lifelong conditions that may require chronic treatment.

Original languageEnglish (US)
Title of host publicationNeurofibromatosis Type 1
Subtitle of host publicationMolecular and Cellular Biology
PublisherSpringer-Verlag Berlin Heidelberg
Pages625-657
Number of pages33
Volume9783642328640
ISBN (Electronic)9783642328640
ISBN (Print)3642328636, 9783642328633
DOIs
StatePublished - Aug 1 2012

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ASJC Scopus subject areas

  • Medicine(all)
  • Biochemistry, Genetics and Molecular Biology(all)

Cite this

Korf, B., Widemann, B., Acosta, M. T., & Packer, R. J. (2012). Translational/clinical studies in children and adults with neurofibromatosis type 1. In Neurofibromatosis Type 1: Molecular and Cellular Biology (Vol. 9783642328640, pp. 625-657). Springer-Verlag Berlin Heidelberg. https://doi.org/10.1007/978-3-642-32864-0_40