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Malignant Peripheral Nerve Sheath Tumors in Neurofibromatosis Type 1: A Multicenter Project With 3 Clinical Trials

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Annual rept. 5 Apr 2004-4 Apr 2005

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A major goal of this CTDA proposal is to optimize NF1 subject recruitment into 3 clinical trials related to MPNSTs. By developing a well- publicized network of NF1 Clinic Centers and Sarcoma Centers, we plan to offer enrollment to all individuals in North America and Europe who have MPNST and NF1. One study is a case-control study to identify risk factors for MPNST. Some individuals will be eligible for a clinical trial of neoadjuvant chemotherapy, and this will be offered to them as a treatment option through sarcoma clinics. Finally, subjects with symptomatic plexiform neurofibromas will be offered enrollment in a PET scanning surveillance study limited to specific PET centers. This CTDA project has been successful in being directly responsible for the implementation of 1 of the 3 clinical trials. Our initial efforts led to the submission of a clinical trial for neoadjuvant chemotherapy in MPNST DAMD-NF043129 PI - David Viskochil. This proposal was not funded, however it was revised by Brigitte Widemann, M.D. as principal investigator with a dedicated focus on treatment of MPNSTs within an oncology Consortium SAPC. This revision entitled PHASE II TRIAL OF NEOADJUVANT CHEMOTHERAPY IN SPOPADIC AND NEUROFIBROMATOSIS TYPE I-ASSOCIATED HIGH GRADE UNRESPECTABLE MPNSTs was submitted in February, 2005, and it has been favorably reviewed for funding through the 2005 DOD NF Program. In addition, website development is continuing at the University of Utah for the recruitment and the enrollment of NF1 patients with MPNST into a natural history study.

Subject Categories:

  • Biochemistry
  • Anatomy and Physiology

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