Intensive Versus Conventional Treatment in Patients With Primary Amyloidosis

University of Limoges (UL)

Status and phase: Completed; Phase 3

Conditions: Primary Systemic Amyloidosis (AL)

Treatments: Drug: Dexamethasone; Drug: Melphalan; Procedure: Autologous stem cell transplantation

Study type: Interventional

Funder types: Other

Identifiers

NCT00344526

I00001

Details and patient eligibility

About

AL amyloidosis is caused by a clonal plasma cell dyscrasia and characterized by progressive deposition of amyloid fibrils derived from monoclonal Ig light chains, leading to multisystem organ failure and death. The prognosis for AL amyloidosis with conventional treatment remains poor, Autologous stem cell transplantation (ASCT) for AL amyloidosis produces high hematologic and organ responses. However, treatment-related mortality remains high and reported series are subject to selection bias.

Full description

A prospective randomized trial was conducted to compare in AL amyloidosis ASCT (melphalan 140 or 200 mg/m2 depending on age and clinical status supported with ASCT collected with G-CSF alone) and the oral regimen M-Dex (melphalan 10 mg/m2 and dexamethasone 40 mg for 4 days each months up to 18 months). The objectives were to compare survival and hematologic and clinical responses.

Sex: All

Ages: 18 to 70 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• below 70 years of age
• biopsy proven systemic AL amyloidosis
• no more than 2 prior courses of chemotherapy
• ECOG performance status < 3
• Informed written consent

Exclusion criteria

• localized amyloidosis
• HIV seropositivity
• previous myelodysplasia
• concomitant serious disease

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)