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

Sumit Madan, Shaji K Kumar, Angela Dispenzieri, Martha Lacy, Suzanne R. Hayman, Francis K. Buadi, David M Dingli, S Vincent Rajkumar, William Hogan, Nelson Leung, Martha Grogan, Morie Gertz

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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 reduceddose melphalan (100-160 mg/m 2). 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 reduceddose melphalan predicted shorter OS. HDM is feasible in patients with cardiac amyloidosis, and achievement of HR and organ response is associated with improved survival.

Original languageEnglish (US)
Pages (from-to)1117-1122
Number of pages6
JournalBlood
Volume119
Issue number5
DOIs
StatePublished - Feb 2 2012

Fingerprint

Peripheral Blood Stem Cell Transplantation
Melphalan
Amyloidosis
Stem cells
Blood
Light
Survival
Brain Natriuretic Peptide
Stem Cell Transplantation
Serum Albumin
Multivariate Analysis
Transplantation
Mortality
Therapeutics

ASJC Scopus subject areas

  • Hematology
  • Biochemistry
  • Cell Biology
  • Immunology

Cite this

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title = "High-dose melphalan and peripheral blood stem cell transplantation for light-chain amyloidosis with cardiac involvement",
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 reduceddose melphalan (100-160 mg/m 2). 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 reduceddose melphalan predicted shorter OS. HDM is feasible in patients with cardiac amyloidosis, and achievement of HR and organ response is associated with improved survival.",
author = "Sumit Madan and Kumar, {Shaji K} and Angela Dispenzieri and Martha Lacy and Hayman, {Suzanne R.} and Buadi, {Francis K.} and Dingli, {David M} and Rajkumar, {S Vincent} and William Hogan and Nelson Leung and Martha Grogan and Morie Gertz",
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T1 - High-dose melphalan and peripheral blood stem cell transplantation for light-chain amyloidosis with cardiac involvement

AU - Madan, Sumit

AU - Kumar, Shaji K

AU - Dispenzieri, Angela

AU - Lacy, Martha

AU - Hayman, Suzanne R.

AU - Buadi, Francis K.

AU - Dingli, David M

AU - Rajkumar, S Vincent

AU - Hogan, William

AU - Leung, Nelson

AU - Grogan, Martha

AU - Gertz, Morie

PY - 2012/2/2

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N2 - 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 reduceddose melphalan (100-160 mg/m 2). 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 reduceddose melphalan predicted shorter OS. HDM is feasible in patients with cardiac amyloidosis, and achievement of HR and organ response is associated with improved survival.

AB - 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 reduceddose melphalan (100-160 mg/m 2). 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 reduceddose 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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