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Investigator(s):
Brigitte Widemann, M.D. Principal Investigator Phone: 301-496-7387 widemanb@pbmac.nci.nih.gov
Referral Contact(s):
Pediatric Oncology  Phone: 1-877-624-4878 (Toll free)

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Primary Eligibility:

• Histologically confirmed* neurofibromatosis type 1 (NF1)-associated plexiform neurofibromas (PN) that are inoperable AND have the potential to cause significant morbidity including, but not limited to, any of the following:
• Head and neck lesions that could compromise the airway or great vessels
• Brachial or lumbar plexus lesions that could cause nerve compression and loss of function
• Lesions that could result in major deformity (e.g., orbital lesions) or significant cosmetic problems
• Lesions of the extremity that could cause limb hypertrophy or loss of function
• Painful lesions



[Note: *Histologic confirmation of tumor is not required in the presence of consistent clinical and radiographic findings, but is required if any clinical observation or scan suggests possible malignant transformation.]



• Patients with PN that has not been histologically confirmed must have ≥ 1 other diagnostic criteria for NF1 (according to NIH Consensus Conference criteria), including any of the following:
• Six or more café-au-lait spots (> 0.5 cm in prepubertal patients or > 1.5 cm in postpubertal patients)
• Freckling in the axilla or groin
• Optic glioma
• Two or more Lisch nodules
• Distinctive bony lesion (dysplasia of the sphenoid bone or dysplasia or thinning of long bone cortex)
• First-degree relative with NF1
• Meets one of the following criteria:
• Progressive PN (with or without clinical symptoms) as defined by one of the following (stratum 3):
• Presence of new PN on MRI within the past year
• Measurable increase of the PN (≥ 20% increase in volume, ≥ 13% increase in the product of the two longest perpendicular diameters, or ≥ 6% increase in the longest diameter) on the last two consecutive MRI or CT scans or over a time period of approximately 1 year prior to study entry
• PN with no clinical symptoms and no documented radiographic progression (stratum 1)
• PN with clinical symptoms but no documented radiographic progression (stratum 2)
• Complete tumor resection is not feasible OR patient refuses
• Measurable disease
• No evidence of an optic glioma requiring treatment with chemotherapy or radiotherapy
• No history of malignant peripheral nerve sheath tumor
• Recovered from all prior therapy
• No prior interferon alfa-2b or PEG-interferon alfa-2b
• ≥ 18 months and ≤ 21 years of age
• Karnofsky or Lansky performance status 50–100%
• Absolute granulocyte count > 1,500/μL
• Hemoglobin > 10 g/dL
• Platelet count > 100,000/μL
• Bilirubin < 1.5 mg/dL
• SGPT ≤ 2 x upper limit of normal
• Creatinine clearance ≥ 70 mL/min OR serum creatinine normal based on age as follows:
• 0.8 mg/dL (for patients 5 years of age and under)
• 1 mg/dL (for patients 6–10 years of age)
• 1.2 mg/dL (for patients 11–15 years of age)
• 1.5 mg/dL (for patients over 15 years of age)
• Not pregnant or nursing; fertile patients must use effective contraception
• No medical condition that would preclude study participation
• No other cancer except surgically cured non-melanoma skin cancer or carcinoma in situ of the cervix
• No history of seropositivity for HIV
• No medical condition requiring chronic systemic corticosteroids

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Treatment Plan:

This is a multicenter study. Patients are stratified according to disease status (plexiform neurofibromas [PN] with no clinical symptoms and no documented radiographic progression [stratum 1] vs PN with clinical symptoms but no documented radiographic progression [stratum 2] vs PN with documented radiographic progression with or without clinical symptoms [stratum 3]).

Strata 1 and 2:

• Patients receive PEG-interferon alfa-2b subcutaneously once a week for 4 weeks
• Courses repeat every 4 weeks for up to 1 year in the absence of disease progression or unacceptable toxicity
• Patients who achieve a partial or complete response may continue treatment for up to 2 years

Stratum 3:

• Patients receive PEG-interferon alfa-2b as in strata 1 and 2
• Courses repeat every 4 weeks in the absence of disease progression or unacceptable toxicity

Patients undergo three-dimensional (3-D) MRI at baseline; at 4, 8, and 12 months; and then every 6 months thereafter for volumetric analysis of their plexiform neurofibromas

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Additional Information:

• This trial will be conducted at the NIH Clinical Center in Bethesda, MD. It is open to patients who meet the eligibility requirements, regardless of where they live in the United States.
• There is no charge for medical care received at NIH Clinical Center.
• PDQ (Physicians Data Query) - provides additional details about this study for health care providers.

Reviewed: 11/11/08
Updated: 6/12/08