Primary Systemic Amyloidosis (AL)
Conditions
Keywords
AL amyloidosis, Autologous stem cell transplantation, Melphalan, Dexamethasone
Brief summary
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.
Detailed 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.
Interventions
DRUGMelphalan
DRUGDexamethasone
PROCEDUREAutologous stem cell transplantation
Sponsors
Ministry of Health, France
University Hospital, Limoges
Study design
Allocation
RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 70 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| survival | — |
Secondary
| Measure | Time frame |
|---|---|
| hematologic responses | — |
| clinical responses | — |
Countries
France
Outcome results
None listed