Trial Information

Risk Adapted Intravenous Melphalan and Adjuvant Thalidomide and Dexamethasone for Untreated Patients With Primary Systemic Amyloidosis

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.