Stem cell transplantation compared with melphalan plus dexamethasone in the treatment of immunoglobulin light-chain amyloidosis

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Abstract

BACKGROUND: Autologous stem cell transplantation (SCT) is a common management strategy for select patients with immunoglobulin light-chain amyloidosis, but no trials have documented improved overall survival. METHODS: Eighty-nine patients with biopsy-proven immunoglobulin light-chain amyloidosis were allowed to select treatment with melphalan plus dexamethasone (n = 34) or SCT (n = 55); all patients were transplant eligible. Treatment preference resulted in imbalanced study arms. Patients who selected SCT were younger, more frequently had an Eastern Cooperative Oncology Group performance status score less than 2, had lower-stage amyloidosis, and had a lower incidence of cardiac amyloidosis. RESULTS: Patients receiving melphalan plus dexamethasone had a 3-year progression-free survival rate of 29.1% and an overall survival rate of 58.8%. Patients undergoing SCT had a 3-year progression-free survival rate of 51.7% and an overall survival rate of 83.6%. An attempt to match patients between the 2 arms in terms of risk produced 24 matched triplet sets (2 SCT patients for each melphalan-dexamethasone patient); there was no difference in hematologic response, but there was better survival after autologous SCT. A propensity score-matched analysis of the cohorts (melphalan plus dexamethasone vs SCT) showed an overall mortality hazard ratio of 2.56 (P <.01). CONCLUSIONS: Although the study had limitations, similar hematologic responses and improved survival were observed after SCT versus melphalan plus dexamethasone.

Original languageEnglish (US)
JournalCancer
DOIs
StateAccepted/In press - 2016

Fingerprint

Immunoglobulin Light Chains
Melphalan
Stem Cell Transplantation
Amyloidosis
Dexamethasone
Survival Rate
Therapeutics
Disease-Free Survival
Survival
Arm
Propensity Score
Cohort Studies
Transplants
Biopsy

Keywords

  • Dexamethasone
  • Immunoglobulin light chains
  • Light-chain amyloidosis
  • Melphalan
  • Stem cell transplantation

ASJC Scopus subject areas

  • Cancer Research
  • Oncology

Cite this

@article{c60448ff563a420e96a7cb729e7084b8,
title = "Stem cell transplantation compared with melphalan plus dexamethasone in the treatment of immunoglobulin light-chain amyloidosis",
abstract = "BACKGROUND: Autologous stem cell transplantation (SCT) is a common management strategy for select patients with immunoglobulin light-chain amyloidosis, but no trials have documented improved overall survival. METHODS: Eighty-nine patients with biopsy-proven immunoglobulin light-chain amyloidosis were allowed to select treatment with melphalan plus dexamethasone (n = 34) or SCT (n = 55); all patients were transplant eligible. Treatment preference resulted in imbalanced study arms. Patients who selected SCT were younger, more frequently had an Eastern Cooperative Oncology Group performance status score less than 2, had lower-stage amyloidosis, and had a lower incidence of cardiac amyloidosis. RESULTS: Patients receiving melphalan plus dexamethasone had a 3-year progression-free survival rate of 29.1{\%} and an overall survival rate of 58.8{\%}. Patients undergoing SCT had a 3-year progression-free survival rate of 51.7{\%} and an overall survival rate of 83.6{\%}. An attempt to match patients between the 2 arms in terms of risk produced 24 matched triplet sets (2 SCT patients for each melphalan-dexamethasone patient); there was no difference in hematologic response, but there was better survival after autologous SCT. A propensity score-matched analysis of the cohorts (melphalan plus dexamethasone vs SCT) showed an overall mortality hazard ratio of 2.56 (P <.01). CONCLUSIONS: Although the study had limitations, similar hematologic responses and improved survival were observed after SCT versus melphalan plus dexamethasone.",
keywords = "Dexamethasone, Immunoglobulin light chains, Light-chain amyloidosis, Melphalan, Stem cell transplantation",
author = "Morie Gertz and Martha Lacy and Angela Dispenzieri and Buadi, {Francis K.} and Dingli, {David M} and Hayman, {Suzanne R.} and Kumar, {Shaji K} and Nelson Leung and John Lust and Rajkumar, {S Vincent} and Russell, {Stephen J} and Suman, {Vera Jean} and Jennifer Le-Rademacher and William Hogan",
year = "2016",
doi = "10.1002/cncr.30051",
language = "English (US)",
journal = "Cancer",
issn = "0008-543X",
publisher = "John Wiley and Sons Inc.",

}

TY - JOUR

T1 - Stem cell transplantation compared with melphalan plus dexamethasone in the treatment of immunoglobulin light-chain amyloidosis

AU - Gertz, Morie

AU - Lacy, Martha

AU - Dispenzieri, Angela

AU - Buadi, Francis K.

AU - Dingli, David M

AU - Hayman, Suzanne R.

AU - Kumar, Shaji K

AU - Leung, Nelson

AU - Lust, John

AU - Rajkumar, S Vincent

AU - Russell, Stephen J

AU - Suman, Vera Jean

AU - Le-Rademacher, Jennifer

AU - Hogan, William

PY - 2016

Y1 - 2016

N2 - BACKGROUND: Autologous stem cell transplantation (SCT) is a common management strategy for select patients with immunoglobulin light-chain amyloidosis, but no trials have documented improved overall survival. METHODS: Eighty-nine patients with biopsy-proven immunoglobulin light-chain amyloidosis were allowed to select treatment with melphalan plus dexamethasone (n = 34) or SCT (n = 55); all patients were transplant eligible. Treatment preference resulted in imbalanced study arms. Patients who selected SCT were younger, more frequently had an Eastern Cooperative Oncology Group performance status score less than 2, had lower-stage amyloidosis, and had a lower incidence of cardiac amyloidosis. RESULTS: Patients receiving melphalan plus dexamethasone had a 3-year progression-free survival rate of 29.1% and an overall survival rate of 58.8%. Patients undergoing SCT had a 3-year progression-free survival rate of 51.7% and an overall survival rate of 83.6%. An attempt to match patients between the 2 arms in terms of risk produced 24 matched triplet sets (2 SCT patients for each melphalan-dexamethasone patient); there was no difference in hematologic response, but there was better survival after autologous SCT. A propensity score-matched analysis of the cohorts (melphalan plus dexamethasone vs SCT) showed an overall mortality hazard ratio of 2.56 (P <.01). CONCLUSIONS: Although the study had limitations, similar hematologic responses and improved survival were observed after SCT versus melphalan plus dexamethasone.

AB - BACKGROUND: Autologous stem cell transplantation (SCT) is a common management strategy for select patients with immunoglobulin light-chain amyloidosis, but no trials have documented improved overall survival. METHODS: Eighty-nine patients with biopsy-proven immunoglobulin light-chain amyloidosis were allowed to select treatment with melphalan plus dexamethasone (n = 34) or SCT (n = 55); all patients were transplant eligible. Treatment preference resulted in imbalanced study arms. Patients who selected SCT were younger, more frequently had an Eastern Cooperative Oncology Group performance status score less than 2, had lower-stage amyloidosis, and had a lower incidence of cardiac amyloidosis. RESULTS: Patients receiving melphalan plus dexamethasone had a 3-year progression-free survival rate of 29.1% and an overall survival rate of 58.8%. Patients undergoing SCT had a 3-year progression-free survival rate of 51.7% and an overall survival rate of 83.6%. An attempt to match patients between the 2 arms in terms of risk produced 24 matched triplet sets (2 SCT patients for each melphalan-dexamethasone patient); there was no difference in hematologic response, but there was better survival after autologous SCT. A propensity score-matched analysis of the cohorts (melphalan plus dexamethasone vs SCT) showed an overall mortality hazard ratio of 2.56 (P <.01). CONCLUSIONS: Although the study had limitations, similar hematologic responses and improved survival were observed after SCT versus melphalan plus dexamethasone.

KW - Dexamethasone

KW - Immunoglobulin light chains

KW - Light-chain amyloidosis

KW - Melphalan

KW - Stem cell transplantation

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U2 - 10.1002/cncr.30051

DO - 10.1002/cncr.30051

M3 - Article

C2 - 27142462

AN - SCOPUS:84977471563

JO - Cancer

JF - Cancer

SN - 0008-543X

ER -