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. 2011 Oct 20;118(16):4346-52.
doi: 10.1182/blood-2011-01-330738. Epub 2011 Aug 9.

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

Maria Teresa Cibeira  1 , Vaishali Sanchorawala, David C Seldin, Karen Quillen, John L Berk, Laura M Dember, Adam Segal, Frederick Ruberg, Hans Meier-Ewert, Nancy T Andrea, J Mark Sloan, Kathleen T Finn, Gheorghe Doros, Joan Blade, Martha Skinner
Affiliations

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

Maria Teresa Cibeira et al. 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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Figures

Figure 1
Figure 1
Flow chart showing the outcome of 421 consecutive patients with AL amyloidosis treated with HDM-SCT.
Figure 2
Figure 2
EFS and OS by intention-to-treat analysis in 421 patients with AL amyloidosis treated with HDM/SCT. Median EFS, 2.6 years (95% CI, 2.0-3.4 years); median OS, 6.3 years (95% CI, 5.4-7.4 years).
Figure 3
Figure 3
EFS and OS of patients who achieved a hematologic CR (n = 145) after HDM/SCT compared with those who did not (non-CR, n = 195). EFS was significantly longer for CR patients (8.3 vs 2 years, P < .0001), as was OS (13.2 vs 5.9 years, P < .0001).
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References

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