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- Title
High-dose melphalan and autologous stem-cell transplantation in patients with AL amyloidosis: an 8-year study.
- Authors
Skinner, Martha; Sanchorawala, Vaishali; M. Dember, Laura; H. Falk, Rodney; L. Berk, John; J. Anderson, Jennifer; O'Hara, Carl; T. Finn, Kathleen; A. Libbey, Caryn; Wiesman, Janice; Quillen, Karen; Swan, Niall; G. Wright, Daniel; Seldin, David C; Dember, Laura M; Falk, Rodney H; Berk, John L; Anderson, Jennifer J; Finn, Kathleen T; Libbey, Caryn A
- Abstract
<bold>Background: </bold>AL amyloidosis is a fatal disease resulting from tissue deposition of amyloid fibrils derived from monoclonal immunoglobulin light chains. Treatment with oral chemotherapy is minimally effective.<bold>Objective: </bold>To test survival and organ response in a large sample of patients treated with high-dose intravenous melphalan (100 to 200 mg/m2) and autologous blood stem-cell transplantation.<bold>Design: </bold>8-year longitudinal analysis of clinical effectiveness.<bold>Setting: </bold>University-affiliated specialty referral clinic.<bold>Patients: </bold>701 consecutive new patients with AL amyloidosis.<bold>Intervention: </bold>High-dose chemotherapy and autologous stem-cell transplantation for patients who met eligibility requirements based on organ involvement and clinical status.<bold>Measurements: </bold>Survival analysis of all patients evaluated and a detailed analysis of treatment outcome in the subgroup that received high-dose melphalan and stem-cell transplantation.<bold>Results: </bold>Among 701 patients with AL amyloidosis, 394 (56%) were eligible for high-dose melphalan and stem-cell transplantation; 82 did not proceed with treatment because of patient choice or disease progression. Median survival of the 312 patients who initiated treatment was 4.6 years. A complete hematologic response, defined as no evidence of an underlying plasma cell dyscrasia 1 year after treatment, was achieved in 40% of patients and was associated with prolonged survival. Statistically significant improvements occurred in end-organ disease and were greater in patients with a complete hematologic response. Mortality rate within 100 days of treatment with high-dose melphalan and stem-cell transplantation was 13%; patients with cardiomyopathy had the highest mortality rates.<bold>Conclusions: </bold>Treatment of selected patients with AL amyloidosis by using high-dose melphalan and stem-cell transplantation resulted in hematologic remission, improved 5-year survival, and reversal of amyloid-related disease in a substantial proportion.
- Subjects
AMYLOIDOSIS; AMYLOID beta-protein; MONOCLONAL antibodies; DRUG therapy; PLASMA cell diseases; CARDIOMYOPATHIES; IMMUNOGLOBULINS; STEM cells; TRANSPLANTATION immunology
- Publication
Annals of Internal Medicine, 2004, Vol 140, Issue 2, p85
- ISSN
0003-4819
- Publication type
journal article
- DOI
10.7326/0003-4819-140-2-200401200-00008