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Phase II trial of pirfenidone in adults with neurofibromatosis type 1

D Babovic-Vuksanovic1, K Ballman, V Michels

  • 1Department of Medical Genetics, Mayo Clinic, 200 First Street SW, Rochester, MN 55905, USA. dbabovic@mayo.edu

Neurology
|October 13, 2006
PubMed

Insights

Pirfenidone showed potential in a phase II trial for neurofibromatosis type 1 (NF1) patients. While some saw tumor volume decrease, further investigation is needed for this condition lacking effective therapies.

Area of Science:

  • Oncology
  • Genetics
  • Pharmacology

Background:

  • Neurofibromatosis type 1 (NF1) is a genetic disorder.
  • NF1 is associated with tumor development.
  • Effective therapies for NF1 are limited.

Purpose of the Study:

  • To evaluate the efficacy of oral pirfenidone in patients with NF1.
  • To assess tumor volume changes in NF1 patients treated with pirfenidone.

Main Methods:

  • An open-label phase II clinical trial was conducted.
  • Twenty-four patients with NF1 were enrolled.
  • Tumor volume was monitored using three-dimensional MRI.

Main Results:

  • Four out of 24 patients experienced a tumor volume decrease of 15% or more.
  • Three patients showed tumor progression.
  • Seventeen patients had stable tumor volume.

Conclusions:

  • Pirfenidone demonstrates potential as a therapeutic option for NF1.
  • Further research is warranted to explore pirfenidone's role in NF1 management.
  • Pirfenidone may address the unmet need for effective NF1 therapies.

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