Melphalan, Thalidomide, and Dexamethasone in Treating Patients With Newly Diagnosed, Previously Untreated Primary Systemic Amyloidosis

Memorial Sloan Kettering Cancer Center (MSK)

Status and phase

Completed

Phase 2

Conditions

Multiple Myeloma and Plasma Cell Neoplasm

Treatments

Biological: filgrastim

Drug: thalidomide

Drug: melphalan

Drug: dexamethasone

Study type

Interventional

Funder types

Other

NIH

Identifiers

NCT00089167

MSKCC-02031

02-031

Details and patient eligibility

About

RATIONALE: Drugs such as melphalan, thalidomide, and dexamethasone may be effective in treating patients with primary systemic amyloidosis.

PURPOSE: This phase II trial is studying how well giving melphalan together with thalidomide and dexamethasone works in treating patients with primary systemic amyloidosis.

Full description

OBJECTIVES:

Primary

Determine the 2-year and overall progression-free survival of patients with newly diagnosed, previously untreated primary systemic (AL) amyloidosis treated with risk-adapted melphalan followed by thalidomide and dexamethasone.

Secondary

Determine plasma cell disease response in these patients at 3, 12, and 24 months after treatment with this regimen.
Determine amyloid-related disease response in these patients at 12 and 24 months after treatment with this regimen.
Determine the prognostic significance of immunoglobulin light-chain variable-region germline gene expression by AL plasma cell clones in patients treated with this regimen.
Determine whether there is molecular minimal residual disease at 12 and 24 months in patients achieving a complete hematologic response after treatment with this regimen.

OUTLINE: Patients are stratified according to the extent of amyloid-related disease (low-risk vs high-risk).

High-risk disease: Patients receive 2 courses of low-dose melphalan IV, dexamethasone, and filgrastim (G-CSF). After 3 months, patients receive thalidomide and dexamethasone if plasma cell disease persists.
Low-risk disease: Patients receive 1 course of high-dose melphalan IV and G-CSF. Patients then receive thalidomide and dexamethasone as in high-risk disease regimen.

Patients are followed at 3, 12, and 24 months.

PROJECTED ACCRUAL: A total of 82 patients will be accrued for this study.

Sex

All

Ages

18+ years old

Volunteers

No Healthy Volunteers

Inclusion and exclusion criteria

DISEASE CHARACTERISTICS:

Diagnosis of primary systemic (AL) amyloidosis within the past 12 months
- High- or low-risk disease, determined by the extent of systemic organ involvement with disease and patient age

PATIENT CHARACTERISTICS:

Age

18 and over

Performance status

SWOG 0-3

Life expectancy

Not specified

Hematopoietic

Not specified

Hepatic

Not specified

Renal

Not specified

Cardiovascular

No New York Heart Association class III or IV congestive heart failure
No restrictive cardiomyopathy requiring oxygen
No myocardial infarction within the past 6 months
No symptomatic cardiac arrhythmia within the past 60 days

Other

No other active malignancy within the past 5 years except adequately treated basal cell or squamous cell skin cancer, carcinoma in situ of the cervix, or adequately treated stage I cancer in complete remission

PRIOR CONCURRENT THERAPY:

Biologic therapy