scholarly journals Induction Therapy with Bortezomib Followed by Bortezomib-High Dose Melphalan and Stem Cell Transplantation for Light Chain Amyloidosis: Results of a Prospective Clinical Trial

2015 ◽  
Vol 21 (8) ◽  
pp. 1445-1451 ◽  
Author(s):  
Vaishali Sanchorawala ◽  
Dina Brauneis ◽  
Anthony C. Shelton ◽  
Stephen Lo ◽  
Fangui Sun ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Induction Therapy ◽  
Light Chain ◽  
Prospective Clinical Trial ◽  
High Dose ◽  
Light Chain Amyloidosis ◽  
High Dose Melphalan

scholarly journals High-dose melphalan and peripheral blood stem cell transplantation for light-chain amyloidosis with cardiac involvement

Blood ◽  
2012 ◽  
Vol 119 (5) ◽  
pp. 1117-1122 ◽  
Author(s):  
Sumit Madan ◽  
Shaji K. Kumar ◽  
Angela Dispenzieri ◽  
Martha Q. Lacy ◽  
Suzanne R. Hayman ◽  
...  
Keyword(s):  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Light Chain ◽  
Cardiac Involvement ◽  
High Dose ◽  
Light Chain Amyloidosis ◽  
Reduced Dose ◽  
Organ Response ◽  
High Dose Melphalan

Abstract High-dose melphalan (HDM) plus stem cell transplantation is an effective treatment for light-chain amyloidosis (AL), but is associated with high treatment-related mortality in patients with cardiac involvement. We studied 187 patients with cardiac involvement with AL who underwent HDM between 1996 and 2008. The median age was 57 years and the median time from diagnosis to HDM was 3.6 months. Half of the patients received reduced-dose melphalan (100-160 mg/m2). The median overall survival (OS) was 66 months, 54 months from diagnosis and HDM, respectively, and 91 patients (49%) were alive at the last follow-up 52 months (median) from HDM. Thirty patients (16%) died within 100 days of transplantation; only low serum albumin predicted early deaths. Overall, hematologic response (HR) and cardiac responses were seen in 66% and 41% of patients, respectively. The median OS for patients with and without HR was not reached and 22 months, respectively (P < .01); and for those with any decrease and no decrease in N-terminal-pro-brain natriuretic peptide was not reached and 26 months, respectively (P < .01). In multivariate analysis of baseline factors, only reduced-dose melphalan predicted shorter OS. HDM is feasible in patients with cardiac amyloidosis, and achievement of HR and organ response is associated with improved survival.

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