Myeloablative chemotherapy with stem cell rescue for the treatment of primary systemic amyloidosis: A status report

Research output: Contribution to journalArticle

68 Citations (Scopus)

Abstract

Stem cell transplantation has been incorporated in the treatment of primary systemic amyloidosis for 5 years. Results reported to date suggest that the response rates are substantially better than those for patients treated with low-dose traditional melphalan and prednisone chemotherapy. Unexpectedly high mortality rates have, however, been reported with stem cell transplantation, reaching 40% in some series. This unexpectedly high mortality appears to be related to multiorgan failure of tissues infiltrated with amyloid deposits. Deaths have been reported from gastrointestinal tract hemorrhage, gastrointestinal tract perforation, sudden cardiac death, and renal failure. The best patient for transplantation appears to have single organ involvement, an age < 55 years, the absence of renal insufficiency, and no symptomatic cardiac dysfunction. Patients eligible to receive stem cell transplant represent a highly selected population, and before conclusions about the efficacy of transplantation are drawn, comparison with a matched control group is necessary. Amyloidosis should be considered an indication for stem cell transplantation in the context of a clinical trial so that results can be compiled and reported for an accurate assessment of response rate, survival, relapse rates and treatment-related toxicities.

Original languageEnglish (US)
Pages (from-to)465-470
Number of pages6
JournalBone Marrow Transplantation
Volume25
Issue number5
StatePublished - 2000

Fingerprint

Stem Cell Transplantation
Stem Cells
Drug Therapy
Renal Insufficiency
Gastrointestinal Tract
Transplantation
Melphalan
Gastrointestinal Hemorrhage
Mortality
Sudden Cardiac Death
Amyloid Plaques
Amyloidosis
Prednisone
Research Design
Therapeutics
Survival Rate
Heart Failure
Clinical Trials
Transplants
Recurrence

Keywords

  • Amyloid
  • Amyloidosis
  • Chemotherapy
  • Nephrotic syndrome
  • Transplantation

ASJC Scopus subject areas

  • Hematology
  • Transplantation

Cite this

@article{be486232d66c4a7a83f814657d6ae0ee,
title = "Myeloablative chemotherapy with stem cell rescue for the treatment of primary systemic amyloidosis: A status report",
abstract = "Stem cell transplantation has been incorporated in the treatment of primary systemic amyloidosis for 5 years. Results reported to date suggest that the response rates are substantially better than those for patients treated with low-dose traditional melphalan and prednisone chemotherapy. Unexpectedly high mortality rates have, however, been reported with stem cell transplantation, reaching 40{\%} in some series. This unexpectedly high mortality appears to be related to multiorgan failure of tissues infiltrated with amyloid deposits. Deaths have been reported from gastrointestinal tract hemorrhage, gastrointestinal tract perforation, sudden cardiac death, and renal failure. The best patient for transplantation appears to have single organ involvement, an age < 55 years, the absence of renal insufficiency, and no symptomatic cardiac dysfunction. Patients eligible to receive stem cell transplant represent a highly selected population, and before conclusions about the efficacy of transplantation are drawn, comparison with a matched control group is necessary. Amyloidosis should be considered an indication for stem cell transplantation in the context of a clinical trial so that results can be compiled and reported for an accurate assessment of response rate, survival, relapse rates and treatment-related toxicities.",
keywords = "Amyloid, Amyloidosis, Chemotherapy, Nephrotic syndrome, Transplantation",
author = "Morie Gertz and Martha Lacy and Angela Dispenzieri",
year = "2000",
language = "English (US)",
volume = "25",
pages = "465--470",
journal = "Bone Marrow Transplantation",
issn = "0268-3369",
publisher = "Nature Publishing Group",
number = "5",

}

TY - JOUR

T1 - Myeloablative chemotherapy with stem cell rescue for the treatment of primary systemic amyloidosis

T2 - A status report

AU - Gertz, Morie

AU - Lacy, Martha

AU - Dispenzieri, Angela

PY - 2000

Y1 - 2000

N2 - Stem cell transplantation has been incorporated in the treatment of primary systemic amyloidosis for 5 years. Results reported to date suggest that the response rates are substantially better than those for patients treated with low-dose traditional melphalan and prednisone chemotherapy. Unexpectedly high mortality rates have, however, been reported with stem cell transplantation, reaching 40% in some series. This unexpectedly high mortality appears to be related to multiorgan failure of tissues infiltrated with amyloid deposits. Deaths have been reported from gastrointestinal tract hemorrhage, gastrointestinal tract perforation, sudden cardiac death, and renal failure. The best patient for transplantation appears to have single organ involvement, an age < 55 years, the absence of renal insufficiency, and no symptomatic cardiac dysfunction. Patients eligible to receive stem cell transplant represent a highly selected population, and before conclusions about the efficacy of transplantation are drawn, comparison with a matched control group is necessary. Amyloidosis should be considered an indication for stem cell transplantation in the context of a clinical trial so that results can be compiled and reported for an accurate assessment of response rate, survival, relapse rates and treatment-related toxicities.

AB - Stem cell transplantation has been incorporated in the treatment of primary systemic amyloidosis for 5 years. Results reported to date suggest that the response rates are substantially better than those for patients treated with low-dose traditional melphalan and prednisone chemotherapy. Unexpectedly high mortality rates have, however, been reported with stem cell transplantation, reaching 40% in some series. This unexpectedly high mortality appears to be related to multiorgan failure of tissues infiltrated with amyloid deposits. Deaths have been reported from gastrointestinal tract hemorrhage, gastrointestinal tract perforation, sudden cardiac death, and renal failure. The best patient for transplantation appears to have single organ involvement, an age < 55 years, the absence of renal insufficiency, and no symptomatic cardiac dysfunction. Patients eligible to receive stem cell transplant represent a highly selected population, and before conclusions about the efficacy of transplantation are drawn, comparison with a matched control group is necessary. Amyloidosis should be considered an indication for stem cell transplantation in the context of a clinical trial so that results can be compiled and reported for an accurate assessment of response rate, survival, relapse rates and treatment-related toxicities.

KW - Amyloid

KW - Amyloidosis

KW - Chemotherapy

KW - Nephrotic syndrome

KW - Transplantation

UR - http://www.scopus.com/inward/record.url?scp=0034012359&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=0034012359&partnerID=8YFLogxK

M3 - Article

C2 - 10713619

AN - SCOPUS:0034012359

VL - 25

SP - 465

EP - 470

JO - Bone Marrow Transplantation

JF - Bone Marrow Transplantation

SN - 0268-3369

IS - 5

ER -