A multicenter phase 2 trial of stem cell transplantation for immunoglobulin light-chain amyloidosis (E4A97): An Eastern Cooperative Oncology Group Study

Morie A. Gertz, E. Blood, D. H. Vesole, Rafat Abonour, H. M. Lazarus, P. R. Greipp

Research output: Contribution to journalArticle

41 Citations (Scopus)

Abstract

Stem cell transplantation was introduced as a new therapeutic modality for amyloidosis. The purpose of the current study was to determine the feasibility and toxicity of stem cell transplantation for amyloidosis in a cooperative group setting in which most participating institutions would have limited experience in managing the disorder. A total of 30 patients with biopsy-proven amyloidosis shown to be immunoglobulin light-chain type were enrolled on this trial. The protocol required mobilization of a minimum of 6 × 108 mononuclear cells/kg or 5 × 106 CD34+ cells/kg ideal body weight. These targets had to be achieved within seven collections. Patients with advanced hepatic, renal, or cardiac failure were excluded. End points included objective response rate and overall survival. The secondary end point of the protocol was nonhematologic toxicity. Accrual to the study was faster than expected. The overall response rate (hematologic and organ) was 64%, with three treatment-related deaths. Another patient died before day 30 of sudden cardiac death not treatment related. The median follow-up of surviving patients is 30.3 months. Median survival has not been reached. Stem cell transplantation for selected patients with amyloidosis is feasible in a cooperative group setting. A multicenter phase 3 trial of high-dose therapy is indicated.

Original languageEnglish
Pages (from-to)149-154
Number of pages6
JournalBone Marrow Transplantation
Volume34
Issue number2
DOIs
StatePublished - Jul 2004

Fingerprint

Immunoglobulin Light Chains
Stem Cell Transplantation
Amyloidosis
Ideal Body Weight
Sudden Cardiac Death
Liver Failure
Therapeutics
Renal Insufficiency
Survival Rate
Heart Failure
Biopsy
Survival

Keywords

  • Amyloidosis
  • Myeloma
  • Transplantation

ASJC Scopus subject areas

  • Hematology
  • Transplantation

Cite this

A multicenter phase 2 trial of stem cell transplantation for immunoglobulin light-chain amyloidosis (E4A97) : An Eastern Cooperative Oncology Group Study. / Gertz, Morie A.; Blood, E.; Vesole, D. H.; Abonour, Rafat; Lazarus, H. M.; Greipp, P. R.

In: Bone Marrow Transplantation, Vol. 34, No. 2, 07.2004, p. 149-154.

Research output: Contribution to journalArticle

@article{e546f73dd70e4a6abd745ce5606d1eaa,
title = "A multicenter phase 2 trial of stem cell transplantation for immunoglobulin light-chain amyloidosis (E4A97): An Eastern Cooperative Oncology Group Study",
abstract = "Stem cell transplantation was introduced as a new therapeutic modality for amyloidosis. The purpose of the current study was to determine the feasibility and toxicity of stem cell transplantation for amyloidosis in a cooperative group setting in which most participating institutions would have limited experience in managing the disorder. A total of 30 patients with biopsy-proven amyloidosis shown to be immunoglobulin light-chain type were enrolled on this trial. The protocol required mobilization of a minimum of 6 × 108 mononuclear cells/kg or 5 × 106 CD34+ cells/kg ideal body weight. These targets had to be achieved within seven collections. Patients with advanced hepatic, renal, or cardiac failure were excluded. End points included objective response rate and overall survival. The secondary end point of the protocol was nonhematologic toxicity. Accrual to the study was faster than expected. The overall response rate (hematologic and organ) was 64{\%}, with three treatment-related deaths. Another patient died before day 30 of sudden cardiac death not treatment related. The median follow-up of surviving patients is 30.3 months. Median survival has not been reached. Stem cell transplantation for selected patients with amyloidosis is feasible in a cooperative group setting. A multicenter phase 3 trial of high-dose therapy is indicated.",
keywords = "Amyloidosis, Myeloma, Transplantation",
author = "Gertz, {Morie A.} and E. Blood and Vesole, {D. H.} and Rafat Abonour and Lazarus, {H. M.} and Greipp, {P. R.}",
year = "2004",
month = "7",
doi = "10.1038/sj.bmt.1704539",
language = "English",
volume = "34",
pages = "149--154",
journal = "Bone Marrow Transplantation",
issn = "0268-3369",
publisher = "Nature Publishing Group",
number = "2",

}

TY - JOUR

T1 - A multicenter phase 2 trial of stem cell transplantation for immunoglobulin light-chain amyloidosis (E4A97)

T2 - An Eastern Cooperative Oncology Group Study

AU - Gertz, Morie A.

AU - Blood, E.

AU - Vesole, D. H.

AU - Abonour, Rafat

AU - Lazarus, H. M.

AU - Greipp, P. R.

PY - 2004/7

Y1 - 2004/7

N2 - Stem cell transplantation was introduced as a new therapeutic modality for amyloidosis. The purpose of the current study was to determine the feasibility and toxicity of stem cell transplantation for amyloidosis in a cooperative group setting in which most participating institutions would have limited experience in managing the disorder. A total of 30 patients with biopsy-proven amyloidosis shown to be immunoglobulin light-chain type were enrolled on this trial. The protocol required mobilization of a minimum of 6 × 108 mononuclear cells/kg or 5 × 106 CD34+ cells/kg ideal body weight. These targets had to be achieved within seven collections. Patients with advanced hepatic, renal, or cardiac failure were excluded. End points included objective response rate and overall survival. The secondary end point of the protocol was nonhematologic toxicity. Accrual to the study was faster than expected. The overall response rate (hematologic and organ) was 64%, with three treatment-related deaths. Another patient died before day 30 of sudden cardiac death not treatment related. The median follow-up of surviving patients is 30.3 months. Median survival has not been reached. Stem cell transplantation for selected patients with amyloidosis is feasible in a cooperative group setting. A multicenter phase 3 trial of high-dose therapy is indicated.

AB - Stem cell transplantation was introduced as a new therapeutic modality for amyloidosis. The purpose of the current study was to determine the feasibility and toxicity of stem cell transplantation for amyloidosis in a cooperative group setting in which most participating institutions would have limited experience in managing the disorder. A total of 30 patients with biopsy-proven amyloidosis shown to be immunoglobulin light-chain type were enrolled on this trial. The protocol required mobilization of a minimum of 6 × 108 mononuclear cells/kg or 5 × 106 CD34+ cells/kg ideal body weight. These targets had to be achieved within seven collections. Patients with advanced hepatic, renal, or cardiac failure were excluded. End points included objective response rate and overall survival. The secondary end point of the protocol was nonhematologic toxicity. Accrual to the study was faster than expected. The overall response rate (hematologic and organ) was 64%, with three treatment-related deaths. Another patient died before day 30 of sudden cardiac death not treatment related. The median follow-up of surviving patients is 30.3 months. Median survival has not been reached. Stem cell transplantation for selected patients with amyloidosis is feasible in a cooperative group setting. A multicenter phase 3 trial of high-dose therapy is indicated.

KW - Amyloidosis

KW - Myeloma

KW - Transplantation

UR - http://www.scopus.com/inward/record.url?scp=4043124700&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=4043124700&partnerID=8YFLogxK

U2 - 10.1038/sj.bmt.1704539

DO - 10.1038/sj.bmt.1704539

M3 - Article

C2 - 15156165

AN - SCOPUS:4043124700

VL - 34

SP - 149

EP - 154

JO - Bone Marrow Transplantation

JF - Bone Marrow Transplantation

SN - 0268-3369

IS - 2

ER -