Superior survival in primary systemic amyloidosis patients undergoing peripheral blood stem cell transplantation

A case-control study

Angela Dispenzieri, Robert A. Kyle, Martha Lacy, Terry M Therneau, Dirk R. Larson, Matthew F. Plevak, S Vincent Rajkumar, Rafael Fonseca, Philip R. Greipp, Thomas Elmer Witzig, John A. Lust, Steven R. Zeldenrust, Denise S. Snow, Susan R. Hayman, Mark R Litzow, Dennis A. Gastineau, Ayalew Tefferi, David J. Inwards, Ivana Micallef, Stephen Maxted Ansell & 3 others Luis F. Porrata, Michelle A. Elliott, Morie Gertz

Research output: Contribution to journalArticle

195 Citations (Scopus)

Abstract

Primary systemic amyloidosia (AL) is a plasma cell dyscrasia resulting in multisystem failure and death. High-dose chemotherapy with peripheral blood stem cell transplantation (PBSCT) has been associated with higher response rates and seemingly higher overall survival then standard chemotherapy. Selection bias, however, confounds interpretation of these results. We performed a case-match-control study comparing overall survival of 63 AL patients undergoing transplantation with 63 patients not undergoing transplantation. Matching criteria included age, sex, time to presentation, left ventricular ejection fraction, serum creatinine, septal thickness, nerve involvement, 24-hour urine protein, and aerum alkaline phosphatase. According to design, there was no difference between the groups with respect to sex (57% males), age (median, 53 years) left ventricular election fraction (65%), number of patients with peripheral nerve involvement (17%), cardiac interventricular septal wall thickness (12 mm), serum creatinine (1.1 mg/dL [97.24 μmol/L]), and bone marrow plasmacytosis (8%). Sixty-six patients have died (16 cases and 50 controls). For PBSCT and control groups, respectively, the 1-, 2-, and 4-year overall survival rates are 89% and 71%; 81% and 55%; and 71% and 41%. Outside a randomized clinical trial, these results present the strongest data supporting the role of PBSCT in selected patients with AL.

Original languageEnglish (US)
Pages (from-to)3960-3963
Number of pages4
JournalBlood
Volume103
Issue number10
DOIs
StatePublished - May 15 2004

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Peripheral Blood Stem Cell Transplantation
Stem cells
Case-Control Studies
Blood
Chemotherapy
Survival
Creatinine
Transplantation
Alkaline Phosphatase
Bone
Drug Therapy
Paraproteinemias
Selection Bias
Plasmas
Serum
Peripheral Nerves
Stroke Volume
Survival Rate
Randomized Controlled Trials
Bone Marrow

ASJC Scopus subject areas

  • Hematology

Cite this

Superior survival in primary systemic amyloidosis patients undergoing peripheral blood stem cell transplantation : A case-control study. / Dispenzieri, Angela; Kyle, Robert A.; Lacy, Martha; Therneau, Terry M; Larson, Dirk R.; Plevak, Matthew F.; Rajkumar, S Vincent; Fonseca, Rafael; Greipp, Philip R.; Witzig, Thomas Elmer; Lust, John A.; Zeldenrust, Steven R.; Snow, Denise S.; Hayman, Susan R.; Litzow, Mark R; Gastineau, Dennis A.; Tefferi, Ayalew; Inwards, David J.; Micallef, Ivana; Ansell, Stephen Maxted; Porrata, Luis F.; Elliott, Michelle A.; Gertz, Morie.

In: Blood, Vol. 103, No. 10, 15.05.2004, p. 3960-3963.

Research output: Contribution to journalArticle

Dispenzieri, Angela ; Kyle, Robert A. ; Lacy, Martha ; Therneau, Terry M ; Larson, Dirk R. ; Plevak, Matthew F. ; Rajkumar, S Vincent ; Fonseca, Rafael ; Greipp, Philip R. ; Witzig, Thomas Elmer ; Lust, John A. ; Zeldenrust, Steven R. ; Snow, Denise S. ; Hayman, Susan R. ; Litzow, Mark R ; Gastineau, Dennis A. ; Tefferi, Ayalew ; Inwards, David J. ; Micallef, Ivana ; Ansell, Stephen Maxted ; Porrata, Luis F. ; Elliott, Michelle A. ; Gertz, Morie. / Superior survival in primary systemic amyloidosis patients undergoing peripheral blood stem cell transplantation : A case-control study. In: Blood. 2004 ; Vol. 103, No. 10. pp. 3960-3963.
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T1 - Superior survival in primary systemic amyloidosis patients undergoing peripheral blood stem cell transplantation

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AU - Dispenzieri, Angela

AU - Kyle, Robert A.

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AU - Larson, Dirk R.

AU - Plevak, Matthew F.

AU - Rajkumar, S Vincent

AU - Fonseca, Rafael

AU - Greipp, Philip R.

AU - Witzig, Thomas Elmer

AU - Lust, John A.

AU - Zeldenrust, Steven R.

AU - Snow, Denise S.

AU - Hayman, Susan R.

AU - Litzow, Mark R

AU - Gastineau, Dennis A.

AU - Tefferi, Ayalew

AU - Inwards, David J.

AU - Micallef, Ivana

AU - Ansell, Stephen Maxted

AU - Porrata, Luis F.

AU - Elliott, Michelle A.

AU - Gertz, Morie

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N2 - Primary systemic amyloidosia (AL) is a plasma cell dyscrasia resulting in multisystem failure and death. High-dose chemotherapy with peripheral blood stem cell transplantation (PBSCT) has been associated with higher response rates and seemingly higher overall survival then standard chemotherapy. Selection bias, however, confounds interpretation of these results. We performed a case-match-control study comparing overall survival of 63 AL patients undergoing transplantation with 63 patients not undergoing transplantation. Matching criteria included age, sex, time to presentation, left ventricular ejection fraction, serum creatinine, septal thickness, nerve involvement, 24-hour urine protein, and aerum alkaline phosphatase. According to design, there was no difference between the groups with respect to sex (57% males), age (median, 53 years) left ventricular election fraction (65%), number of patients with peripheral nerve involvement (17%), cardiac interventricular septal wall thickness (12 mm), serum creatinine (1.1 mg/dL [97.24 μmol/L]), and bone marrow plasmacytosis (8%). Sixty-six patients have died (16 cases and 50 controls). For PBSCT and control groups, respectively, the 1-, 2-, and 4-year overall survival rates are 89% and 71%; 81% and 55%; and 71% and 41%. Outside a randomized clinical trial, these results present the strongest data supporting the role of PBSCT in selected patients with AL.

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