High-dose melphalan versus melphalan plus dexamethasone for AL amyloidosis.
BACKGROUND
High-dose chemotherapy followed by autologous hematopoietic stem-cell transplantation has been reported to provide higher response rates and better overall survival than standard chemotherapy in immunoglobulin-light-chain (AL) amyloidosis, but these two strategies have not been compared in a randomized study.


METHODS
We conducted a randomized trial comparing high-dose intravenous melphalan followed by autologous hematopoietic stem-cell rescue with standard-dose melphalan plus high-dose dexamethasone in patients with AL amyloidosis. Patients (age range, 18 to 70 years) with newly diagnosed AL amyloidosis were randomly assigned to receive intravenous high-dose melphalan plus autologous stem cells or oral melphalan plus oral high-dose dexamethasone.


RESULTS
Fifty patients were enrolled in each group. The results were analyzed on an intention-to-treat basis, with overall survival as the primary end point. After a median follow-up of 3 years, the estimated median overall survival was 22.2 months in the group assigned to receive high-dose melphalan and 56.9 months in the group assigned to receive melphalan plus high-dose dexamethasone (P=0.04). Among patients with high-risk disease, overall survival was similar in the two groups. Among patients with low-risk disease, there was a nonsignificant difference between the two groups in overall survival at 3 years (58% in the group assigned to receive high-dose melphalan vs. 80% in the group assigned to receive melphalan plus high-dose dexamethasone; P=0.13).


CONCLUSIONS
The outcome of treatment of AL amyloidosis with high-dose melphalan plus autologous stem-cell rescue was not superior to the outcome with standard-dose melphalan plus dexamethasone. (ClinicalTrials.gov number, NCT00344526 [ClinicalTrials.gov].).
Jaccard
Arnaud
A
Department of Hematology, Centre Hospitalier Universitaire, Université et Centre National de la Recherche Scientifique, UMR 6101, Limoges, France. arnaud.jaccard@chu-limoges.fr
Moreau
Philippe
P
Leblond
Veronique
V
Leleu
Xavier
X
Benboubker
Lotfi
L
Hermine
Olivier
O
Recher
Christian
C
Asli
Bouchra
B
Lioure
Bruno
B
Royer
Bruno
B
Jardin
Fabrice
F
Bridoux
Frank
F
Grosbois
Bernard
B
Jaubert
Jérome
J
Piette
Jean-Charles
JC
Ronco
Pierre
P
Quet
Fabrice
F
Cogne
Michel
M
Fermand
Jean-Paul
JP
D000368:Aged; D000686:Amyloidosis / Q000188:drug therapy / Q000401:mortality / Q000628:therapy*; D003131:Combined Modality Therapy; D003907:Dexamethasone / Q000008:administration & dosage*; D018450:Disease Progression; D004359:Drug Therapy, Combination; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007147:Immunoglobulin Light Chains; D053208:Kaplan-Meier Estimate; D008297:Male; D008558:Melphalan / Q000008:administration & dosage*; D008875:Middle Aged; D019653:Myeloablative Agonists / Q000008:administration & dosage*
D007147:Immunoglobulin Light Chains; D019653:Myeloablative Agonists; D003907:Dexamethasone; D008558:Melphalan