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Bortezomib-based chemotherapy reduces early mortality and improves outcomes in patients with ultra-high-risk

Kai-Ni Shen1, Cong-Li Zhang1, Zhuang Tian2

  • 1a Department of Hematology , Peking Union Medical College Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College , Beijing , People's Republic of China.

Amyloid : the International Journal of Experimental and Clinical Investigation : the Official Journal of the International Society of Amyloidosis
|May 11, 2019
PubMed

Insights

Bortezomib-based chemotherapy significantly reduces early mortality and improves overall survival in patients with ultra-high-risk amyloid light-chain amyloidosis. This treatment offers better long-term outcomes for this vulnerable patient group.

Area of Science:

  • Hematology
  • Oncology
  • Cardiology

Background:

  • Patients with amyloid light-chain (AL) amyloidosis and advanced cardiac damage face high mortality risks.
  • Bortezomib is a standard treatment, but its efficacy in ultra-high-risk AL amyloidosis (Mayo stage IIIb/IV) remains unproven.

Purpose of the Study:

  • To evaluate the impact of bortezomib-based regimens on early mortality and long-term outcomes.
  • To assess the effectiveness of bortezomib in ultra-high-risk AL amyloidosis patients.

Main Methods:

  • Retrospective analysis of newly diagnosed ultra-high-risk AL amyloidosis patients.
  • Comparison between patients receiving bortezomib-based chemotherapy and those receiving supportive care only.

Main Results:

  • Bortezomib group (n=62) showed median overall survival (OS) of 30 months vs. 2 months in the control group (n=24).
  • Median progression-free survival (PFS) was 15.8 months with bortezomib versus 2 months without (p<.001).
  • Early death rate (within 6 months) was 32.3% with bortezomib, significantly lower than 66.7% in controls (p<.001).

Conclusions:

  • Bortezomib-based regimens are effective in reducing early mortality in ultra-high-risk AL amyloidosis.
  • These regimens improve long-term survival and progression-free survival for this high-risk population.

Keywords:
AL amyloidosisbortezomibearly mortalityoverall survivalultra-high-risk

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