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.
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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
100
Service des Maladies du Sang
Lille, France
Service d'Hématologie et de Thérapie cellulaire
Limoges, France
Service d'hématologie clinique
Nantes, France
Service d'hématologie Clinique, Groupe Hospitalier Pitié-Salpétrière
Paris, France
survival
hematologic responses
clinical responses
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Service d'hématologie clinique, Hôpital Necker
Paris, France
Service d'immuno-hématologie, Hôpital Saint-Louis
Paris, France
Service d'hématologie
Toulouse, France
Hématologie Clinique
Tours, France