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

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.