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Natural History of Plexiform Neurofibromas in NF1

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Annual rept. 1 Oct 1999-30 Sep 2000

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The major goals of this study have been to document the usefulness of volumetric MRI as a means of following the growth of plexiform neurofibromas, measuring the rate of growth of plexiform neurofibromas over time, and establishing a consortium of clinical centers to carry out clinical trials of treatment for plexiform neurofibromas in the future. Establishment of this consortium has been considerably more difficult than anticipated, due to the need to have every center approved both by a local IRB and the army IRB. Many minor and a few major differences in wording of informed consents have impeded many centers from completing the IRB approval process. We have tried to streamline this process, now, and expect that 19 centers will be approved by the first quarter of 2001. With this, however, we will have accomplished one of our major goals, to create a consortium of clinical centers, supported by a tissue repository, database, pathology review, MRI review, and statistical support. We have also taken a major step towards demonstration that the volumetric approach can be accomplished in spite of the complexities of imaging of plexiform neurofibromas by showing that three independent observers obtain similar volume measurements. The final goal, that of determining rate of neurofibroma growth, will take longer than expected due to the slow acquisition of IRB approval for all clinical centers, but this is expected to be accomplished as well, albeit after a longer period of study than initially anticipated. The first clinical trial of a farnesyl protein transferase inhibitor is about to begin under the auspices of the National Cancer Institute. We are involved in this study, using the same MRI protocol and tissue repository for that study, It is expected that additional clinical trials will begin over the next sevenal years, and that the approaches and resources of this consortium will facilitate the efforts to test these drugs and determine treatment endpoints in NFI.c

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  • Medicine and Medical Research

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