| Phase I trial of pegylated interferon-alpha-2b in young patients with plexiform neurofibromas. | |
| | |
MedLine Citation:
|
PMID: 21242495 Owner: NLM Status: MEDLINE |
Abstract/OtherAbstract:
|
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. |
| | |
Authors:
|
R I Jakacki; E Dombi; D M Potter; S Goldman; J C Allen; I F Pollack; B C Widemann |
Related Documents
:
|
146395 - Muscle changes in patients with varicose veins. 14512905 - Significance of dual-basin drainage in patients with truncal melanoma undergoing sentin... 532845 - Proteinaceous lymphadenopathy with hypergammaglobulinemia. 1759765 - Erica predicts response to tamoxifen in elderly women with breast cancer. 16686295 - Outcome of patients on chronic peritoneal dialysis undergoing peritoneal catheter remov... 18815815 - Kidney growth in children with congenital hypothyroidism. |
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: 2012-01-18 |
Medline Journal Info:
|
Nlm Unique ID: 0401060 Medline TA: Neurology Country: United States |
Other Details:
|
Languages: eng Pagination: 265-72 Citation Subset: AIM; IM |
Affiliation:
|
Children's Hospital of Pittsburgh, 4401 Penn Avenue, Pittsburgh, PA 15224, USA. regina.jakacki@chp.edu |
Export Citation:
|
APA/MLA Format Download EndNote Download BibTex |
| MeSH Terms | |
Descriptor/Qualifier:
|
Adolescent Adult Antineoplastic Agents / administration & dosage*, adverse effects* Antiviral Agents / administration & dosage, adverse effects Child Child, Preschool Disease Progression Drug Administration Schedule Female Humans Infant Injections, Subcutaneous Interferon-alpha / administration & dosage*, adverse effects* Magnetic Resonance Imaging Male Neurofibroma, Plexiform / drug therapy*, pathology, radiography Polyethylene Glycols / administration & dosage*, adverse effects* Recombinant Proteins Treatment Outcome |
| Chemical | |
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
Previous Document: A novel DCC mutation and genetic heterogeneity in congenital mirror movements.
Next Document: Adverse antiepileptic drug effects in new-onset seizures: a case-control study.