ABSTRACT
From clinical trials employing intravenous (iv) melphalan and mobilized stem cell support for patients with systemic Primary amyloidosis (AL) one should learned that survival depends on the number of major viscera involved with amyloid, on the degree of cardiac involvement, and on the response of the plasma cell disease to melphalan. Patients with persistent plasma cell disease at 3 months receive adjuvant therapy with either thalidomide and dexamethasone or dexamethasone alone. Primary endpoints are survival, and organ system and hematologic responses. Furthermore, adjuvant therapy is feasible and appears to benefit about 30% of patients (4/13) with improvement in hematologic response, including the achievement of light microscopy. It is important to note how tailored the use of adjuvant therapy is in this trial; the emphasis is on tolerance of these drugs, a problem area particularly with thalidomide in AL patients.
