• Objective response as assessed by RECIST criteria [ Designated as safety issue: No ]
  
• Toxicity as measured by NCI CTC [ Designated as safety issue: Yes ]
  

OBJECTIVES:

Primary

• Determine the efficacy of sirolimus, in terms of objective response rate, in patients with angiomyolipoma of the kidney secondary to tuberous sclerosis (TSC) or lymphangioleiomyomatosis (LAM).
• Determine the toxicity of this drug in these patients.

Secondary

• Determine changes in other TSC lesions (e.g., tubers, subependymal giant cell astrocytomas, facial angiofibromas, or kidney cysts) in patients with TSC treated with this drug.
• Determine changes in pulmonary disease in patients with LAM treated with this drug.

OUTLINE: This is a multicenter study.

Patients receive oral sirolimus once daily for 12 months in the absence of unacceptable toxicity.

After completion of study treatment, patients are followed at 6 months and 1 year.

PROJECTED ACCRUAL: A total of 13-35 patients will be accrued for this study within 3-30 months.

DISEASE CHARACTERISTICS:

• Diagnosis of angiomyolipoma of the kidney secondary to tuberous sclerosis (TSC) or lymphangioleiomyomatosis (LAM)*

  • Tumor ≥ 2 cm by MRI NOTE: *Diagnosis of LAM by chest CT scan
• No angiomyolipoma-related bleeding or impending bleed

• No evidence of severe LAM, defined as either of the following:

  • Oxygen-dependent disease
  • Poor performance status
• No suspicion of or known renal cell carcinoma

PATIENT CHARACTERISTICS:

Age

• 18 to 65

Performance status

• Not specified

Life expectancy

• Not specified

Hematopoietic

• Hematocrit > 27%
• WBC normal
• Platelet count normal

Hepatic

• SGOT and SGPT < 2 times normal
• Bilirubin < 2 times normal
• Alkaline phosphatase < 2 times normal

Renal

• Creatinine < 4.1 mg/dL
• No evidence of accelerating renal dysfunction
• No acute renal failure

Cardiovascular

• No history of coronary artery disease

Pulmonary

• See Disease Characteristics

Other

• Not pregnant or nursing
• Negative pregnancy test
• Fertile patients must use effective barrier contraception
• No unstable seizures, defined as a recent change in seizure pattern or change in antiepileptic drug regimen
• No active infection

PRIOR CONCURRENT THERAPY:

Biologic therapy

• Not specified

Chemotherapy

• Not specified

Endocrine therapy

• Not specified

Radiotherapy

• Not specified

Surgery

• Not specified

Other

• More than 30 days since prior investigational agents
• More than 6 months since prior vascular embolization therapy for treatment of kidney angiomyolipomas
• No concurrent chronic use of diltiazem, ketoconazole, or rifampin
• No other concurrent investigational agents