Pirfenidone in Treating Young Patients With Neurofibromatosis Type 1 and Plexiform Neurofibromas

You may be eligible if

• DISEASE CHARACTERISTICS:
• Diagnosis of neurofibromatosis type 1 (NF1) AND
• Plexiform neurofibromas
• * Neurofibromas that have grown along the length of a nerve and may involve multiple fascicles and branches (spinal neurofibromas involve 2 or more levels with connection between the levels or extending laterally along the nerve)
• * Potential to cause significant morbidity such as:
• * Head and neck lesions that could compromise 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 cause limb hypertrophy or loss of function
• * Painful lesions
• Meets at least 1 other diagnostic criteria for NF1
• * 6 or more cafe-au-lait spots (at least 0.5 cm in prepubertal patients or at least 1.5 cm in postpubertal patients)
• * Freckling in the axilla or groin
• * Optic glioma
• * 2 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
• Measurable plexiform neurofibromas
• * At least 3 cm in 1 dimension
• * Tumor resection not feasible
• No history of malignant peripheral nerve sheath tumor or other cancer
• No evidence of an active optic glioma requiring chemotherapy or radiotherapy
• No malignant glioma
• PATIENT CHARACTERISTICS:
• Age
• 3 to 21
• Performance status
• Karnofsky 50-100% (over 10 years of age)
• Lansky 50-100% (10 years and under)
• Life expectancy
• Not specified
• Hematopoietic
• Absolute granulocyte count at least 1,500/mm\^3
• Hemoglobin at least 9.0 g/dL
• Platelet count at least 150,000/mm\^3
• Hepatic
• Bilirubin normal
• SGPT no greater than 2 times upper limit of normal
• No clinically significant hepatic dysfunction that would preclude study participation
• Renal
• Creatinine normal for age OR
• Creatinine clearance at least 70 mL/min
• Cardiovascular
• No clinically significant cardiac dysfunction that would preclude study participation
• Pulmonary
• No clinically significant pulmonary dysfunction that would preclude study participation
• Other
• Not pregnant or nursing
• Negative pregnancy test
• Fertile patients must use effective contraception during and for 2 months after study
• Must be able to take pirfenidone orally
• No serious infections
• No clinically significant unrelated systemic illness or organ dysfunction that would preclude study participation
• PRIOR CONCURRENT THERAPY:
• Biologic therapy
• At least 30 days since prior immunotherapy
• No concurrent immunotherapy
• No concurrent hematopoietic growth factors
• Chemotherapy
• At least 30 days since prior chemotherapy
• No concurrent chemotherapy directed at the tumor
• Endocrine therapy
• At least 30 days since prior hormonal therapy directed at the tumor
• No concurrent hormonal therapy directed at the tumor
• Radiotherapy
• At least 90 days since prior radiotherapy to the site of the plexiform neurofibroma
• No concurrent radiotherapy directed at the tumor
• Surgery
• Not specified
• Other
• Recovered from prior therapy
• More than 30 days since prior investigational agents
• No prior pirfenidone
• No other concurrent investigational agents