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

Open access
Cibeira, Maria Teresa, Sanchorawala, Vaishali, Seldin, David C., Quillen, Karen, Berk, John L., Dember, Laura M., Segal, Adam, Ruberg, Frederick, Meier-Ewert, Hans, Andrea, Nancy T., Sloan, J. Mark, Finn, Kathleen T., Doros, Gheorghe, Blade, Joan, Skinner, Martha·2011·Blood

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.

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Metadata

Authors
Cibeira, M. T., Sanchorawala, V., Seldin, D. C., Quillen, K., Berk, J. L., Dember, L. M., Segal, A., Ruberg, F., Meier-Ewert, H., Andrea, N. T., Sloan, J. M., Finn, K. T., Doros, G., Blade, J., & Skinner, M.
Venue
Blood
Published
2011-10-20
Volume
118
Issue
16
Pages
4346-4352
ISSN
  • 0006-4971 (ISSN-L)
  • 1528-0020
OpenAlex
W1999473831
Cited by
289
References
27
FWCI
10.23
Type
journal article
Language
en
Open access
green

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CoChat. (2026). Citation check: Outcome of AL amyloidosis after high-dose melphalan and autologous stem cell transplantation: long-term results in a series of 421 patients. https://app.cochat.ai/papers/10.1182/blood-2011-01-330738
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Cibeira, M. T., Sanchorawala, V., Seldin, D. C., Quillen, K., Berk, J. L., Dember, L. M., Segal, A., Ruberg, F., Meier-Ewert, H., Andrea, N. T., Sloan, J. M., Finn, K. T., Doros, G., Blade, J., & Skinner, M. (2011). Outcome of AL amyloidosis after high-dose melphalan and autologous stem cell transplantation: long-term results in a series of 421 patients. Blood. https://doi.org/10.1182/blood-2011-01-330738

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Contains information from the Semantic Scholar Academic Graph Datasets, made available under the ODC Attribution License (ODC-BY 1.0).

Semantic Scholar’s data is described in: Kinney, R., et al. (2023). The Semantic Scholar Open Data Platform. arXiv:2301.10140.