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High-dose melphalan versus melphalan plus dexamethasone for AL amyloidosis
Arnaud Jaccard1, Philippe Moreau, Veronique Leblond
1Department of Hematology, Centre Hospitalier Universitaire, Université et Centre National de la Recherche Scientifique, UMR 6101, Limoges, France. arnaud.jaccard@chu-limoges.fr
The New England Journal of Medicine
|September 15, 2007
Summary
High-dose chemotherapy with stem-cell rescue did not improve survival for AL amyloidosis patients compared to standard melphalan and dexamethasone. Standard treatment showed better overall survival in this randomized trial.
Area of Science:
- Hematology
- Oncology
- Clinical Trials
Background:
- Immunoglobulin-light-chain (AL) amyloidosis is a serious condition.
- High-dose chemotherapy with autologous stem-cell transplantation (ASCT) has shown promise but lacked direct comparison to standard therapy in randomized trials.
Purpose of the Study:
- To compare the efficacy of high-dose intravenous melphalan plus autologous hematopoietic stem-cell rescue versus standard-dose melphalan plus high-dose dexamethasone in AL amyloidosis patients.
Main Methods:
- A randomized trial was conducted involving 100 newly diagnosed AL amyloidosis patients (ages 18-70).
- Patients were assigned to either high-dose melphalan with stem-cell rescue or standard-dose melphalan with high-dose dexamethasone.
- Overall survival was the primary endpoint, analyzed on an intention-to-treat basis.
Main Results:
- After a median follow-up of 3 years, the median overall survival was significantly shorter in the high-dose melphalan group (22.2 months) compared to the standard-dose melphalan plus dexamethasone group (56.9 months) (P=0.04).
- In patients with high-risk disease, survival was similar between the two treatment arms.
- For low-risk patients, there was a trend towards better survival with standard-dose melphalan plus dexamethasone, though not statistically significant at 3 years (80% vs. 58%).
Conclusions:
- High-dose melphalan with autologous stem-cell rescue is not superior to standard-dose melphalan plus dexamethasone for treating AL amyloidosis.
- Standard-dose melphalan combined with dexamethasone appears to be a more effective treatment strategy for overall survival in AL amyloidosis.
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