Outcome of AL amyloidosis after high-dose melphalan and autologous stem cell transplantation: long-term results in a series of 421 patients

Author:

Cibeira Maria Teresa123,Sanchorawala Vaishali12,Seldin David C.12,Quillen Karen12,Berk John L.1,Dember Laura M.1,Segal Adam1,Ruberg Frederick1,Meier-Ewert Hans1,Andrea Nancy T.2,Sloan J. Mark12,Finn Kathleen T.12,Doros Gheorghe4,Blade Joan3,Skinner Martha1

Affiliation:

1. Amyloid Treatment and Research Program, Department of Medicine, Boston University School of Medicine, Boston, MA;

2. Section of Hematology-Oncology, Department of Medicine, Boston University Medical Center, Boston, MA;

3. Hematology Department, Institute of Hematology and Oncology, Institut d'Investigacions Biomèdiques August Pi i Sunyer, Hospital Clínic, University of Barcelona, Barcelona, Spain; and

4. Department of Biostatistics, Boston University School of Public Health, Boston, MA

Abstract

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.

Publisher

American Society of Hematology

Subject

Cell Biology,Hematology,Immunology,Biochemistry

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