Document Detail

Phase I trial of pegylated interferon-alpha-2b in young patients with plexiform neurofibromas.
MedLine Citation:
PMID:  21242495     Owner:  NLM     Status:  MEDLINE    
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.
R I Jakacki; E Dombi; D M Potter; S Goldman; J C Allen; I F Pollack; B C Widemann
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Publication Detail:
Type:  Clinical Trial, Phase I; Journal Article; Research Support, Non-U.S. Gov't    
Journal Detail:
Title:  Neurology     Volume:  76     ISSN:  1526-632X     ISO Abbreviation:  Neurology     Publication Date:  2011 Jan 
Date Detail:
Created Date:  2011-01-18     Completed Date:  2011-02-14     Revised Date:  2014-07-30    
Medline Journal Info:
Nlm Unique ID:  0401060     Medline TA:  Neurology     Country:  United States    
Other Details:
Languages:  eng     Pagination:  265-72     Citation Subset:  AIM; IM    
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MeSH Terms
Antineoplastic Agents / administration & dosage*,  adverse effects*
Antiviral Agents / administration & dosage,  adverse effects
Child, Preschool
Disease Progression
Drug Administration Schedule
Injections, Subcutaneous
Interferon-alpha / administration & dosage*,  adverse effects*
Magnetic Resonance Imaging
Neurofibroma, Plexiform / drug therapy*,  pathology,  radiography
Polyethylene Glycols / administration & dosage*,  adverse effects*
Recombinant Proteins
Treatment Outcome
Reg. No./Substance:
0/Antineoplastic Agents; 0/Antiviral Agents; 0/Interferon-alpha; 0/Polyethylene Glycols; 0/Recombinant Proteins; 0/peginterferon alfa-2b; 99210-65-8/interferon alfa-2b

From MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine

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