High-dose melphalan versus melphalan plus dexamethasone for AL amyloidosis.
نویسندگان
چکیده
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].).
منابع مشابه
Oral cyclic melphalan and dexamethasone for patients with AL amyloidosis.
PURPOSE Aggressive treatment of amyloid light chain (AL) amyloidosis with high-dose intravenous melphalan followed by autologous stem cell transplantation (HDM/SCT) is effective in inducing hematologic remission and clinical improvement. However, only selected patients with AL amyloidosis are eligible for HDM/SCT because of amyloid-associated organ dysfunction. PATIENTS AND METHODS We report ...
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disease. In amyloidosis there is a clear relationship between monoclonal gammopathy and AL deposits. In POEMS syndrome, the plasma cell dyscrasia has a pathogenetic role, but no clear relationship has been found between the gammopathy and the clinical manifestations, which rather appear to be due to high VEGF levels. However, both these entities improve when the plasma cell dyscrasia and the mo...
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Primary (AL amyloidosis) is a systemic disease characterized by an amyloid deposition process in many organs, with unsatisfactory survival of patients. The monoclonal light chains form the fibrils that deposit and accumulate in tissues. Renal involvement is very frequent in AL amyloidosis and could lead to development of nephrotic syndrome followed by the renal failure in many cases. Classic th...
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REFERENCES 1 Gillmore JD, Hawkins PN. Amyloidosis and the respiratory tract. Thorax 1999; 54: 444–451. 2 Capizzi SA, Betancourt E, Prakash UB. Tracheobronchial amyloidosis. Mayo Clin Proc 2000; 75: 1148–1152. 3 Evans PA, Pott C, Groenen PJ, et al. Significantly improved PCRbased clonality testing in B-cell malignancies by use of multiple immunoglobulin gene targets. Report of the BIOMED-2 Conce...
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INTRODUCTION Hepatitis B virus (HBV) reactivation (so-called reverse seroconversion) is a rare but known complication of hematopoietic stem cell transplantation, immunosuppressive therapy, or high-dose chemotherapy plus rituximab. This event is linked to a treatment-related fall in titers of antibodies to hepatitis B surface antigen (HBsAb) below the protective threshold level. CASE PRESENTAT...
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ورودعنوان ژورنال:
- The New England journal of medicine
دوره 358 1 شماره
صفحات -
تاریخ انتشار 2007