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Peginterferon alfa-2b in Treating Young Patients with Unresectable, Sympotomatic or Life-Threatening Plexiform Neurofibromas Associated with Neurofibromatosis Type 1
This Phase II, multicenter study will determine how plexiform neuofibroma (PNF) tumors respond to weekly dosages of Peginterferon alfa-2b (also known as PEG-Intron), a long-acting form of interferon. Researchers will also study side effects of the drug and evaluate “volumetric analysis,” a new method of measuring changes in tumor size using magnetic resonance imaging (MRI) scans. Although benign, PNF tumors can become disfiguring, disabling and even life-threatening. They are a relatively common problem in people with a type of nerocutaneous syndrome called neurofibromatosis type 1 (NF1), a condition characterized by changes in skin coloring and the growth of tumors along nerves in the skin, brain and other parts of the body.
The trial is enrolling participants of both genders, ages 18 months to 21 years, who satisfy study criteria, including having NF1 and progressive, symptomatic or life-threatening PNFs that are not surgically removable. A subgroup of patients with NF1 and no symptoms associated with their PNF will also be included.
Males: 18 months to 21 years
Females: 18 months to 21 years
Following a series of diagnostic tests, PEG-Intron will be given in weekly doses via subcutaneous injections at home for up to 2 years. Participants will be monitored closely with physical examinations, blood tests and MRI scans.
Frequency of visits: Varies from 6 to 24 weeks
Duration: Up to 1 to 2 years, depending on response
National Cancer Institute
Study Description at National Institutes of Health
National Cancer Institute
Cancer and Blood Disorder Research
For more information about the study or enrollment, please contact:
Angela Connelly, RN, BSN
Children's Hospital's main campus is located in the Lawrenceville neighborhood. Our main hospital address is:
UPMC Children’s Hospital of Pittsburgh
One Children’s Hospital Way
4401 Penn Ave.
Pittsburgh, PA 15224
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