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: Molecular and Cellular Biology
PublisherSpringer-Verlag Berlin Heidelberg
Pages625-657
Number of pages33
Volume9783642328640
ISBN (Print)9783642328640, 3642328636, 9783642328633
DOIs
StatePublished - Aug 1 2012
Externally publishedYes

Fingerprint

Neurofibromatosis 1
Toxicity
Tumors
Plexiform Neurofibroma
Clinical Trials
Testing
Therapeutics
Neurofibroma
Neurilemmoma
Glioma
Clinical Studies
Skin

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

Translational/clinical studies in children and adults with neurofibromatosis type 1. / Korf, Bruce; Widemann, Brigitte; Acosta, Maria T.; Packer, Roger J.

Neurofibromatosis Type 1: Molecular and Cellular Biology. Vol. 9783642328640 Springer-Verlag Berlin Heidelberg, 2012. p. 625-657.

Research output: Chapter in Book/Report/Conference proceedingChapter

Korf, B, Widemann, B, Acosta, MT & Packer, RJ 2012, Translational/clinical studies in children and adults with neurofibromatosis type 1. in Neurofibromatosis Type 1: Molecular and Cellular Biology. vol. 9783642328640, Springer-Verlag Berlin Heidelberg, pp. 625-657. https://doi.org/10.1007/978-3-642-32864-0_40
Korf B, Widemann B, Acosta MT, Packer RJ. Translational/clinical studies in children and adults with neurofibromatosis type 1. In Neurofibromatosis Type 1: Molecular and Cellular Biology. Vol. 9783642328640. Springer-Verlag Berlin Heidelberg. 2012. p. 625-657 https://doi.org/10.1007/978-3-642-32864-0_40
Korf, Bruce ; Widemann, Brigitte ; Acosta, Maria T. ; Packer, Roger J. / Translational/clinical studies in children and adults with neurofibromatosis type 1. Neurofibromatosis Type 1: Molecular and Cellular Biology. Vol. 9783642328640 Springer-Verlag Berlin Heidelberg, 2012. pp. 625-657
@inbook{c02586fe860d4264957c18deafd2536e,
title = "Translational/clinical studies in children and adults with neurofibromatosis type 1",
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.",
author = "Bruce Korf and Brigitte Widemann and Acosta, {Maria T.} and Packer, {Roger J.}",
year = "2012",
month = "8",
day = "1",
doi = "10.1007/978-3-642-32864-0_40",
language = "English (US)",
isbn = "9783642328640",
volume = "9783642328640",
pages = "625--657",
booktitle = "Neurofibromatosis Type 1: Molecular and Cellular Biology",
publisher = "Springer-Verlag Berlin Heidelberg",

}

TY - CHAP

T1 - Translational/clinical studies in children and adults with neurofibromatosis type 1

AU - Korf, Bruce

AU - Widemann, Brigitte

AU - Acosta, Maria T.

AU - Packer, Roger J.

PY - 2012/8/1

Y1 - 2012/8/1

N2 - 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.

AB - 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.

UR - http://www.scopus.com/inward/record.url?scp=84942348130&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=84942348130&partnerID=8YFLogxK

U2 - 10.1007/978-3-642-32864-0_40

DO - 10.1007/978-3-642-32864-0_40

M3 - Chapter

SN - 9783642328640

SN - 3642328636

SN - 9783642328633

VL - 9783642328640

SP - 625

EP - 657

BT - Neurofibromatosis Type 1: Molecular and Cellular Biology

PB - Springer-Verlag Berlin Heidelberg

ER -