Treatment With Bortezomib-based Therapy, Followed by Autologous Stem Cell Transplantation, Improves Outcomes in Light Chain Amyloidosis: A Retrospective Study

Tania Jain, Heidi E. Kosiorek, Shu T. Kung, Vishal S. Shah, Amylou Dueck, Veronica Gonzalez-Calle, Susan Luft, Craig B. Reeder, Roberta Adams, Pierre Noel, Jeremy T. Larsen, Joseph R Mikhael, Peter Leif Bergsagel, Alexander Keith Stewart, Rafael Fonseca

Research output: Contribution to journalArticle

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Abstract

We performed a retrospective study to compare the hematologic response, organ response, and overall survival in patients with light chain amyloidosis, receiving bortezomib-based therapy before autologous stem cell transplantation (ASCT) versus non–bortezomib-based therapy or no therapy before ASCT. In the present study, although the patients who received bortezomib before transplantation were at greater risk at baseline, we found significantly better outcomes in these patients. Background: The hematologic response is critical in patients with light chain amyloidosis because a good response is known to improve organ response and overall survival. We present a retrospective analysis to compare the hematologic and organ response in patients who received bortezomib-based therapy before autologous stem cell transplantation (ASCT) versus those who received non–bortezomib-based therapy before ASCT and those who underwent ASCT at diagnosis. Patients and Methods: Of a total of 63 patients who underwent ASCT for light chain amyloidosis, 34 received bortezomib-based therapy before ASCT (Bor-ASCT) and 29 did not receive bortezomib therapy (non-Bor-ASCT). A greater number of patients had involvement of ≥ 3 organs and cardiac involvement in the Bor-ASCT group, suggesting a greater risk at baseline in the Bor-ASCT group. Results: At 3, 6, and 12 months after ASCT, the hematologic response was better in the Bor-ASCT group, with a statistically significance difference at 6 months (partial response or better in 82% vs. 20%; P =.002) and 12 months (partial response or better in 76% vs. 33%; P =.02). Organ responses (66% vs. 21%; P <.001) and median overall survival (not reached vs. 53 months; P =.001) were also greater in the Bor-ASCT group. Conclusion: Our study has shown that bortezomib-based therapy before ASCT improves the hematologic response, organ response and overall survival, potentially by decreasing the light chain load before ASCT.

Original languageEnglish (US)
JournalClinical Lymphoma, Myeloma and Leukemia
DOIs
StateAccepted/In press - Jan 1 2018

Fingerprint

Stem Cell Transplantation
Amyloidosis
Retrospective Studies
Light
Therapeutics
Survival
Bortezomib
Transplantation

Keywords

  • Autologous stem cell transplantation
  • Bortezomib
  • Light chain amyloidosis

ASJC Scopus subject areas

  • Hematology
  • Oncology
  • Cancer Research

Cite this

Treatment With Bortezomib-based Therapy, Followed by Autologous Stem Cell Transplantation, Improves Outcomes in Light Chain Amyloidosis : A Retrospective Study. / Jain, Tania; Kosiorek, Heidi E.; Kung, Shu T.; Shah, Vishal S.; Dueck, Amylou; Gonzalez-Calle, Veronica; Luft, Susan; Reeder, Craig B.; Adams, Roberta; Noel, Pierre; Larsen, Jeremy T.; Mikhael, Joseph R; Bergsagel, Peter Leif; Stewart, Alexander Keith; Fonseca, Rafael.

In: Clinical Lymphoma, Myeloma and Leukemia, 01.01.2018.

Research output: Contribution to journalArticle

Jain, Tania ; Kosiorek, Heidi E. ; Kung, Shu T. ; Shah, Vishal S. ; Dueck, Amylou ; Gonzalez-Calle, Veronica ; Luft, Susan ; Reeder, Craig B. ; Adams, Roberta ; Noel, Pierre ; Larsen, Jeremy T. ; Mikhael, Joseph R ; Bergsagel, Peter Leif ; Stewart, Alexander Keith ; Fonseca, Rafael. / Treatment With Bortezomib-based Therapy, Followed by Autologous Stem Cell Transplantation, Improves Outcomes in Light Chain Amyloidosis : A Retrospective Study. In: Clinical Lymphoma, Myeloma and Leukemia. 2018.
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abstract = "We performed a retrospective study to compare the hematologic response, organ response, and overall survival in patients with light chain amyloidosis, receiving bortezomib-based therapy before autologous stem cell transplantation (ASCT) versus non–bortezomib-based therapy or no therapy before ASCT. In the present study, although the patients who received bortezomib before transplantation were at greater risk at baseline, we found significantly better outcomes in these patients. Background: The hematologic response is critical in patients with light chain amyloidosis because a good response is known to improve organ response and overall survival. We present a retrospective analysis to compare the hematologic and organ response in patients who received bortezomib-based therapy before autologous stem cell transplantation (ASCT) versus those who received non–bortezomib-based therapy before ASCT and those who underwent ASCT at diagnosis. Patients and Methods: Of a total of 63 patients who underwent ASCT for light chain amyloidosis, 34 received bortezomib-based therapy before ASCT (Bor-ASCT) and 29 did not receive bortezomib therapy (non-Bor-ASCT). A greater number of patients had involvement of ≥ 3 organs and cardiac involvement in the Bor-ASCT group, suggesting a greater risk at baseline in the Bor-ASCT group. Results: At 3, 6, and 12 months after ASCT, the hematologic response was better in the Bor-ASCT group, with a statistically significance difference at 6 months (partial response or better in 82{\%} vs. 20{\%}; P =.002) and 12 months (partial response or better in 76{\%} vs. 33{\%}; P =.02). Organ responses (66{\%} vs. 21{\%}; P <.001) and median overall survival (not reached vs. 53 months; P =.001) were also greater in the Bor-ASCT group. Conclusion: Our study has shown that bortezomib-based therapy before ASCT improves the hematologic response, organ response and overall survival, potentially by decreasing the light chain load before ASCT.",
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T2 - A Retrospective Study

AU - Jain, Tania

AU - Kosiorek, Heidi E.

AU - Kung, Shu T.

AU - Shah, Vishal S.

AU - Dueck, Amylou

AU - Gonzalez-Calle, Veronica

AU - Luft, Susan

AU - Reeder, Craig B.

AU - Adams, Roberta

AU - Noel, Pierre

AU - Larsen, Jeremy T.

AU - Mikhael, Joseph R

AU - Bergsagel, Peter Leif

AU - Stewart, Alexander Keith

AU - Fonseca, Rafael

PY - 2018/1/1

Y1 - 2018/1/1

N2 - We performed a retrospective study to compare the hematologic response, organ response, and overall survival in patients with light chain amyloidosis, receiving bortezomib-based therapy before autologous stem cell transplantation (ASCT) versus non–bortezomib-based therapy or no therapy before ASCT. In the present study, although the patients who received bortezomib before transplantation were at greater risk at baseline, we found significantly better outcomes in these patients. Background: The hematologic response is critical in patients with light chain amyloidosis because a good response is known to improve organ response and overall survival. We present a retrospective analysis to compare the hematologic and organ response in patients who received bortezomib-based therapy before autologous stem cell transplantation (ASCT) versus those who received non–bortezomib-based therapy before ASCT and those who underwent ASCT at diagnosis. Patients and Methods: Of a total of 63 patients who underwent ASCT for light chain amyloidosis, 34 received bortezomib-based therapy before ASCT (Bor-ASCT) and 29 did not receive bortezomib therapy (non-Bor-ASCT). A greater number of patients had involvement of ≥ 3 organs and cardiac involvement in the Bor-ASCT group, suggesting a greater risk at baseline in the Bor-ASCT group. Results: At 3, 6, and 12 months after ASCT, the hematologic response was better in the Bor-ASCT group, with a statistically significance difference at 6 months (partial response or better in 82% vs. 20%; P =.002) and 12 months (partial response or better in 76% vs. 33%; P =.02). Organ responses (66% vs. 21%; P <.001) and median overall survival (not reached vs. 53 months; P =.001) were also greater in the Bor-ASCT group. Conclusion: Our study has shown that bortezomib-based therapy before ASCT improves the hematologic response, organ response and overall survival, potentially by decreasing the light chain load before ASCT.

AB - We performed a retrospective study to compare the hematologic response, organ response, and overall survival in patients with light chain amyloidosis, receiving bortezomib-based therapy before autologous stem cell transplantation (ASCT) versus non–bortezomib-based therapy or no therapy before ASCT. In the present study, although the patients who received bortezomib before transplantation were at greater risk at baseline, we found significantly better outcomes in these patients. Background: The hematologic response is critical in patients with light chain amyloidosis because a good response is known to improve organ response and overall survival. We present a retrospective analysis to compare the hematologic and organ response in patients who received bortezomib-based therapy before autologous stem cell transplantation (ASCT) versus those who received non–bortezomib-based therapy before ASCT and those who underwent ASCT at diagnosis. Patients and Methods: Of a total of 63 patients who underwent ASCT for light chain amyloidosis, 34 received bortezomib-based therapy before ASCT (Bor-ASCT) and 29 did not receive bortezomib therapy (non-Bor-ASCT). A greater number of patients had involvement of ≥ 3 organs and cardiac involvement in the Bor-ASCT group, suggesting a greater risk at baseline in the Bor-ASCT group. Results: At 3, 6, and 12 months after ASCT, the hematologic response was better in the Bor-ASCT group, with a statistically significance difference at 6 months (partial response or better in 82% vs. 20%; P =.002) and 12 months (partial response or better in 76% vs. 33%; P =.02). Organ responses (66% vs. 21%; P <.001) and median overall survival (not reached vs. 53 months; P =.001) were also greater in the Bor-ASCT group. Conclusion: Our study has shown that bortezomib-based therapy before ASCT improves the hematologic response, organ response and overall survival, potentially by decreasing the light chain load before ASCT.

KW - Autologous stem cell transplantation

KW - Bortezomib

KW - Light chain amyloidosis

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