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NEUROLOGY 2009;73:1273-1279
© 2009 American Academy of Neurology

Characteristics of children enrolled in treatment trials for NF1-related plexiform neurofibromas

A. Kim, MD, A. Gillespie, RN, MS, E. Dombi, MD, A. Goodwin, RN, W. Goodspeed, RN, E. Fox, MD, F. M. Balis, MD and B. C. Widemann, MD

From the Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, Bethesda, MD.

Address correspondence and reprint requests to Dr. AeRang Kim, National Cancer Institute, Pediatric Oncology Branch, 10 Center Dr., Bldg. 10-CRC, Room 1-3872, Bethesda, MD 20892

Objective: To describe the characteristics of children enrolled in treatment trials for neurofibromatosis type 1 (NF1)–related plexiform neurofibroma (PN), PN tumor burden, PN-related complications, and treatment outcomes and to highlight the differences between characteristics of children with NF1 vs children with cancers entered on early phase drug trials.

Methods: Pre-enrollment characteristics and complications of PN were retrospectively analyzed in a cohort of 59 children with NF1-related PN treated on 1 of 7 clinical trials at the NIH between 1996 and 2007. Outcome was analyzed in a subset of 19 patients enrolled in phase I trials. Comparisons to children with cancer were made from a similar analysis performed recently.

Results: The median age at enrollment was 8 years. The median PN volume was 555 mL. Most patients had no prior chemotherapy or radiation, but nearly half had previous surgery for PN. PN-associated complications and NF1 manifestations were common, including pain (53%), other tumors (18%), and hypertension (8%). Investigational drug therapy was well tolerated. A median of 10 treatment cycles was administered. Patients with NF1-related PN were younger, had better performance score, had less prior therapy, and remained on study longer than cancer patients.

Conclusions: Children with NF1-related plexiform neurofibroma (PN) enrolled in clinical trials had large tumors with substantial morbidity. Clinical trials in these children provide information about drug tolerance, cumulative toxicity, and pharmacokinetics in a younger population than early phase pediatric cancer trials. This report may aid in the evaluation of the applicability of traditional pediatric cancer trial designs and endpoints for NF1-related PN.

Abbreviations: DLT = dose-limiting toxicities; ECOG = Eastern Cooperative Oncology Group; MTD = maximum tolerated dose; NF1 = neurofibromatosis type 1; PK = pharmacokinetic; PN = plexiform neurofibroma.

kimaer{at}mail.nih.gov

Supported by the Intramural Research Program of the NIH, National Cancer Institute, Center for Cancer Research.

The views expressed do not necessarily represent the views of the NIH or the US government.

Disclosure: Author disclosures are provided at the end of the article.

Received February 17, 2009. Accepted in final form July 23, 2009.