Autologous Hematopoietic Cell Transplantation Versus Chemotherapy Alone for Immunoglobulin Light Chain Amyloidosis: A Retrospective Study

Clin Lymphoma Myeloma Leuk. 2019 Jul;19(7):413-422.e5. doi: 10.1016/j.clml.2019.02.009. Epub 2019 Mar 1.


Introduction: Immunoglobulin light chain (AL) amyloidosis is caused by the deposition of monoclonal immunoglobulin light chains, for which autologous hematopoietic cell transplantation (AHCT) is one of the most effective therapies. In small studies comparing AHCT with chemotherapy alone, AHCT was associated with better survival.

Patients and methods: In this study, we compared the outcomes of AHCT with those of chemotherapy alone in 232 patients. We retrospectively reviewed the outcomes in 74 patients who underwent AHCT with those of 158 patients treated only with chemotherapy.

Results: The median event-free survival (EFS) (73 vs. 9 months; P < .001) and overall survival (OS) (not achieved vs. 39 months; P < .001) were superior in the AHCT group versus those in the chemotherapy group. On multivariate analysis, AHCT was significantly associated with better EFS (hazard ratio, 0.410; 95% confidence interval, 0.241-0.697; P = .0010) and OS (hazard ratio, 0.313; 95% confidence interval, 0.155-0.636; P = .0013) than chemotherapy alone. Even when patients with severe findings (mean left ventricular thickness > 12 mm, brain natriuretic peptide level > 400 pg/mL, and creatinine level > 2.0 mg/dL) and elderly patients (age > 65 years) were excluded, both EFS and OS were significantly better in the AHCT group than in the chemotherapy group upon univariate and multivariate analyses.

Conclusion: AHCT yielded better EFS and OS than chemotherapy alone in patients with AL amyloidosis. AHCT should be considered for eligible patients.

Keywords: Bortezomib; Dexamethasone; High dose melphalan; Immunomodulatory drug; Melphalan.

MeSH terms

  • Adult
  • Aged
  • Aged, 80 and over
  • Antineoplastic Agents / administration & dosage
  • Antineoplastic Agents / adverse effects
  • Antineoplastic Agents / therapeutic use*
  • Female
  • Hematopoietic Stem Cell Transplantation* / adverse effects
  • Hematopoietic Stem Cell Transplantation* / methods
  • Humans
  • Immunoglobulin Light-chain Amyloidosis / diagnosis
  • Immunoglobulin Light-chain Amyloidosis / etiology
  • Immunoglobulin Light-chain Amyloidosis / mortality
  • Immunoglobulin Light-chain Amyloidosis / therapy*
  • Male
  • Middle Aged
  • Retrospective Studies
  • Severity of Illness Index
  • Treatment Outcome


  • Antineoplastic Agents