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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]
.)
Source Information
From Centre Hospitalier Universitaire, Université et Centre National de la Recherche Scientifique, UMR 6101, Limoges (A.J., F.Q., M.C.); Centre Hospitalier Universitaire, Nantes (P.M.); Hôpital Pitié–Salpêtrière, Assistance Publique–Hôpitaux de Paris and Université Paris VI, Paris (V.L., J.-C.P.); Centre Hospitalier Universitaire, Lille (X.L.); Centre Hospitalier Universitaire, Tours (L.B.); Hôpital Necker, Assistance Publique–Hôpitaux de Paris, Université Paris V, and Centre National de la Recherche Scientifique UM 814, Paris (O.H.); Centre Hospitalier Universitaire Hôpital Purpan, Toulouse (C.R.); Hôpital Saint-Louis, Assistance Publique–Hôpitaux de Paris, Paris (B.A., J.-P.F.); Centre Hospitalier Universitaire, Strasbourg (B.L.); Centre Hospitalier Universitaire, Amiens (B.R.); Centre Henri Becquerel, Rouen (F.J.); Centre Hospitalier Universitaire, Poitiers (F.B.); Centre Hospitalier Universitaire, Rennes (B.G.); Institut de Cancérologie de la Loire, Saint Priest en Jarez (J.J.); and INSERM Unité 702, Université Paris VI, and Hôpital Tenon, Assistance Publique–Hôpitaux de Paris, Paris (P.R.) — all in France.
Address reprint requests to Dr. Jaccard at the Department of Hematology, Centre Hospitalier Universitaire, Limoges, 87000 Limoges, France, or at arnaud.jaccard{at}chu-limoges.fr.
Related Letters:
High-Dose Melphalan versus Melphalan plus Dexamethasone for AL Amyloidosis
Kumar S., Dispenzieri A., Gertz M. A., Mehta J., Lachmann H. J., Wechalekar A. D., Gillmore J. D., Lokhorst H. M., Hazenberg B. P.C., Croockewit A., Comenzo R. L., Steingart R. M., Cohen A. D., Jaccard A., Moreau P., Fermand J.-P.
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N Engl J Med 2008;
358:91-93, Jan 3, 2008.
Correspondence
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