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Blood ; 118(16): 4346-52, 2011 Oct 20.
Article in English | MEDLINE | ID: mdl-21828140

ABSTRACT

Previous studies have suggested that, in patients with AL amyloidosis treated with high-dose melphalan and autologous stem-cell transplantation (HDM/SCT), the greatest benefit is seen in those patients achieving a hematologic complete response (CR). We analyzed a series of 421 consecutive patients treated with HDM/SCT at a single referral center and compared outcomes for patients with and without CR. Treatment-related mortality was 11.4% overall (5.6% in the last 5 years). By intention-to-treat analysis, the CR rate was 34% and the median event-free survival (EFS) and overall survival (OS) were 2.6 and 6.3 years, respectively. Eighty-one patients died within the first year after HDM/SCT and were not evaluable for hematologic and organ response. Of 340 evaluable patients, 43% achieved CR and 78% of them experienced an organ response. For CR patients, median EFS and OS were 8.3 and 13.2 years, respectively. Among the 195 patients who did not obtain CR, 52% achieved an organ response, and their median EFS and OS were 2 and 5.9 years, respectively. Thus, treatment of selected AL patients with HDM/SCT resulted in a high organ response rate and long OS, even for those patients who did not achieve CR.


Subject(s)
Amyloidosis/therapy , Antineoplastic Agents, Alkylating/therapeutic use , Hematopoietic Stem Cell Transplantation , Immunoglobulin Light Chains/metabolism , Melphalan/therapeutic use , Myeloablative Agonists/therapeutic use , Adult , Aged , Aged, 80 and over , Amyloidosis/drug therapy , Amyloidosis/metabolism , Antineoplastic Agents, Alkylating/administration & dosage , Disease-Free Survival , Female , Hematologic Tests , Humans , Male , Melphalan/administration & dosage , Middle Aged , Myeloablative Agonists/administration & dosage , Transplantation, Autologous , Treatment Outcome
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