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Abstract
Objective: To longitudinally analyze changes in plexiform neurofibroma (PN) volume in relation to age and body growth in children and young adults with neurofibromatosis type 1 and inoperable, symptomatic, or progressive PNs, using a sensitive, automated method of volumetric MRI analysis.
Methods: We included patients 25 years of age and younger with PNs entered in a natural history study or in treatment trials who had volumetric MRI over ≥16 months.
Results: We studied 49 patients (median age 8.3 years) with 61 PNs and a median evaluation period of 34 months (range 18 to 70). The PN growth rates varied among patients, but were constant within patients. Thirty-four patients (69%) experienced ≥20% increase in PN volume during the observation period. PN volume increased more rapidly than body weight over time (p = 0.026). Younger patients had the most rapid PN growth rate.
Conclusions: Volume increase of plexiform neurofibromas is a realistic and meaningful trial endpoint. In most patients plexiform neurofibroma growth rate exceeded body growth rate. The youngest patients had the fastest plexiform neurofibroma growth rate, and clinical drug development should be directed toward this population. Age stratification for clinical trials for plexiform neurofibromas should be considered.
Footnotes
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Additional material related to this article can be found on the Neurology Web site. Go to www.neurology.org and scroll down the Table of Contents for the February 27 issue to find the title link for this article.
Commentary, see page 627
This article was previously published in electronic format as an Expedited E-Pub on January 10, 2007, at www.neurology.org.
This research was supported by the Intramural Research Program of the NIH, National Cancer Institute, Center for Cancer Research. This research was also supported by the U.S. Army through three clinical trial awards: 1) natural history of plexiform neurofibromas in NF1, grant no. NF70002; 2) phase II randomized, cross-over, double-blinded, placebo-controlled trial of the farnesyltransferase inhibitor R115777 in pediatric patients with neurofibromatosis type 1 and progressive plexiform neurofibromas, grant no. NF000027; 3) phase I/phase II study of pirfenidone in NF1 and plexiform neurofibromas, grant no. NF010042.
Disclosure: The authors report no conflicts of interest.
Received March 21, 2006. Accepted in final form July 21, 2006.
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