CC-5013 With or Without Dexamethasone in Treating Patients With Primary Systemic Amyloidosis

OBJECTIVES:

Primary

• Determine the tolerability of CC-5013 in patients with primary systemic (AL) amyloidosis.
• Determine the objective hematologic response rate in patients treated with this drug.
• Determine amyloid organ disease response in patients treated with this drug.

Secondary

• Determine hematologic and amyloid organ disease response in patients who do not achieve a response to CC-5013 alone and are subsequently treated with CC-5013 and dexamethasone.
• Determine the toxicity of CC-5013 in combination with dexamethasone in these patients.

OUTLINE: Patients receive oral CC-5013 once daily on days 1-21. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. Patients not achieving a hematologic response continue to receive CC-5013 as before and also receive oral dexamethasone twice daily on days 1-4, 9-12, and 17-20 of every other 28-day course for up to 6 courses of combination therapy. Patients who maintain a hematologic response after 6 courses of combination therapy may receive CC-5013 alone in the absence of disease progression or unacceptable toxicity. Patients not achieving a hematologic response after the initiation of dexamethasone are removed from the study.

Patients are followed annually.

PROJECTED ACCRUAL: A total of 15-25 patients will be accrued for this study within 5-12.5 months.

Inclusion criteria

DISEASE CHARACTERISTICS:

• Histologically confirmed primary systemic (AL) amyloidosis

• Tissue amyloid deposits or positive fat aspirate
• Meets 1 of the following criteria for AL type disease:

• Serum or urine monoclonal protein by immunofixation electrophoresis
• Plasmacytosis of bone marrow by monoclonal staining for kappa- or lambda-light chain isotype

PATIENT CHARACTERISTICS:

Age

• 18 and over

Performance status

• SWOG 0-2

Life expectancy

• Not specified

Hematopoietic

• White blood count> 3,000/mm^3
• Hemoglobin > 8 g/dL
• Platelet count > 100,000/mm^3
• Absolute neutrophil count > 1,000/mm^3

Hepatic

• Bilirubin ≤ 2 times upper limit of normal (ULN)
• aspartate aminotransferase (AST) and Alanine Aminotransferase (ALT) ≤ 2 times ULN

PRIOR CONCURRENT THERAPY:

Biologic therapy

• Prior thalidomide for AL amyloidosis allowed

Chemotherapy

• More than 4 weeks since prior cytotoxic chemotherapy

Endocrine therapy

• Prior steroids for AL amyloidosis allowed

Radiotherapy

• More than 4 weeks since prior radiotherapy

Surgery

• Prior surgery allowed

Other

• Recovered from all prior therapy

Exclusion criteria

• No secondary or familial amyloidosis
• No multiple myeloma, defined as ≥ 30% plasma cells in bone marrow biopsy specimen OR lytic bone lesions
• No prior CC-5013

Renal

• No dialysis

Cardiovascular

• No symptomatic cardiac arrhythmia
• No oxygen-dependent restrictive cardiomyopathy

Other

• No untreated or uncontrolled infection
• No other malignancy except basal cell skin cancer or carcinoma in situ of the cervix or breast
• No other serious medical illness that would preclude study participation
• No history of hypersensitivity reaction to thalidomide
• HIV negative
• Not pregnant or nursing
• Negative pregnancy test
• Fertile patients must use effective contraception