Natural History of Plexiform Neurofibromas in NF1
Abstract
The three major goals of this study were to: (1) validate volumetric MRI in the measurement of plexiform neurofibromas; (2) obtain a body of normative data on the growth rate of plexiform neurofibromas; and (3) develop an infrastructure to support future clinical trials. Although the first aim will not be fully achieved until all of the data are available and analyzed, we have made significant progress by demonstrating tumor volumes can be reliably measured by multiple observers. The reproducibility study has been completed and submitted for publication. Regarding the second aim, this year saw a continued increase in patient enrollment mainly due to the addition of three new clinical sites. Most plexiform neurofibromas have been imaged only once or twice, but we have included data representing longitudinal follow-up of one tumor as an example of the type of growth data we are hoping to collect. Finally, with respect to building infrastructure, we have a stable group of participating centers and have streamlined the process of signing on new centers. We have also worked collaboratively with the group at the National Cancer Institute who is running a clinical trial of a farnesyl protein transferase to coordinate the natural history study with this clinical trial. The study got off to a slow start due to administrative problems related to the process of obtaining IRB approval for centers but data is now steadily accumulating, making it clear that the objectives of the study will be achieved.
Document Details
- Document Type
- Technical Report
- Publication Date
- Oct 01, 2001
- Accession Number
- ADA400083
Entities
People
- Bruce R. Korf
Organizations
- Brigham and Women's Hospital