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January 18, 2011; 76 (3) Articles

Phase I trial of pegylated interferon-α-2b in young patients with plexiform neurofibromas

R.I. Jakacki, E. Dombi, D.M. Potter, S. Goldman, J.C. Allen, I.F. Pollack, B.C. Widemann
First published January 17, 2011, DOI: https://doi.org/10.1212/WNL.0b013e318207b031
R.I. Jakacki
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E. Dombi
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D.M. Potter
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Citation
Phase I trial of pegylated interferon-α-2b in young patients with plexiform neurofibromas
R.I. Jakacki, E. Dombi, D.M. Potter, S. Goldman, J.C. Allen, I.F. Pollack, B.C. Widemann
Neurology Jan 2011, 76 (3) 265-272; DOI: 10.1212/WNL.0b013e318207b031

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Abstract

Objective: Interferon has antiproliferative and antiangiogenic properties. We sought to evaluate preliminary efficacy and determine the recommended phase II dose (RP2D) for pegylated interferon-α-2b (PI) in patients with unresectable progressive or symptomatic plexiform neurofibromas (PN).

Methods: PI was administered weekly in cohorts of 3–6 patients during the dose-finding phase and continued for up to 2 years. Twelve patients were treated at the RP2D to further evaluate toxicity and activity.

Results: Thirty patients (median age 9.3 years, range 1.9–34.7 years) were enrolled. No dose-limiting toxicity (DLT) was seen in patients treated at the 3 μg/kg dose level (DL) during the first 4 weeks. All 5 patients treated at the 4.5 μg/kg DL came off study or required dose reductions for behavioral toxicity or fatigue. Similar DLT on the 3 μg/kg DL became apparent over time. There was 1 DLT (myoclonus) in 12 patients enrolled at the 1.0 μg/kg DL. Eleven of 16 patients with pain showed improvement and 13 of 14 patients with a palpable mass had a decrease in size. Five of 17 patients (29%) who underwent volumetric analysis had a 15%–22% decrease in volume. Three of 4 patients with documented radiographic progression prior to enrollment showed stabilization or shrinkage.

Conclusions: The RP2D of PI for pediatric patients with PN is 1 μg/kg/wk. Clinical and radiographic improvement and cessation of growth can occur.

Classification of evidence: This study provides Class III evidence that pegylated interferon-α-2b in patients with unresectable, progressive, symptomatic, or life-threatening PNs results in radiographic reduction or stabilization of PN size.

Footnotes

  • Study funding: Supported by Schering-Plough Corp., who supplied the pegylated interferon-α-2b.

  • AUC
    area under the curve
    DL
    dose level
    DLT
    dose-limiting toxicity
    FDA
    Food and Drug Administration
    IFN
    interferon
    MTD
    maximum tolerated dose
    NF1
    neurofibromatosis type 1
    NHT
    nonhematologic toxicity
    PEG
    polyethylene glycol
    PI
    pegylated interferon-α-2b
    PN
    plexiform neurofibroma
    RP2D
    recommended phase II dose
    STIR
    short T1-inversion recovery
    TSH
    thyroid-stimulating hormone
    VA
    volumetric analyses.

  • Received March 12, 2010.
  • Accepted September 17, 2010.
  • Copyright © 2011 by AAN Enterprises, Inc.
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