Role of high-dose chemotherapy and autologous hematopoietic cell transplantation in primary systemic amyloidosis: a systematic review

Biol Blood Marrow Transplant. 2009 Aug;15(8):893-902. doi: 10.1016/j.bbmt.2009.01.022. Epub 2009 Apr 2.

Abstract

Significant uncertainty exists regarding the efficacy of high-dose chemotherapy and autologous hematopoietic cell transplantation (AHCT) for the treatment of patients with primary systemic (AL) amyloidosis. We performed a systematic review and meta-analysis to evaluate the efficacy of AHCT versus conventional chemotherapy (CC) in patients with AL amyloidosis using methodology recommended by the Cochrane Collaboration. A comprehensive literature search yielded 820 studies. Twelve studies met the inclusion criteria: 1 randomized controlled trial (RCT), 2 other controlled studies, and 9 single-arm trials. The 1 RCT and 2 controlled studies compared AHCT and CC, and 9 single-arm studies assessed the efficacy of AHCT without a control. The pooled hazard ratio for overall survival (OS) in the 3 controlled studies was 1.79 (95% confidence interval [CI] = 1.11 to 2.91) favoring CC. The pooled proportion for mortality in the single-arm studies (n = 7) was 0.35 (95% CI = 0.25 to 0.46). The pooled odds ratio for complete hematologic response (CHR) from 2 controlled studies was 0.64 (95% CI = 0.25 to 1.64), indicating no difference between AHCT and CC. In the single-arm studies, the pooled proportion for CHR was 0.35 (95% CI = 0.26 to 0.44), and the pooled proportion for treatment-related mortality (TRM) was 0.12 (95% CI = 0.09 to 0.14). In the controlled studies, there was no heterogeneity for any outcome; however, in the single-arm studies, there was a significant heterogeneity for the outcomes of OS, CHR, renal response, and partial hematologic response. Our findings indicate that AHCT does not appear to be superior to CC in improving OS in patients with AL amyloidosis. But the quality of our evidence is low, indicating a need for well-designed and adequately powered RCTs to better address the role of AHCT in AL amyloidosis.

Publication types

  • Comparative Study
  • Meta-Analysis
  • Research Support, Non-U.S. Gov't
  • Review
  • Systematic Review

MeSH terms

  • Amyloidosis / mortality
  • Amyloidosis / therapy*
  • Antineoplastic Agents / administration & dosage*
  • Hematopoietic Stem Cell Transplantation / methods*
  • Hematopoietic Stem Cell Transplantation / mortality
  • Humans
  • Odds Ratio
  • Proportional Hazards Models
  • Survival Rate
  • Transplantation, Autologous
  • Treatment Outcome

Substances

  • Antineoplastic Agents