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Keywords:

  • amyloidosis therapy;
  • amyloidosis complications;
  • immunoglobulin light chain;
  • stem cell transplantation;
  • therapeutic use of alkylating agents;
  • humans;
  • prognosis

Abstract

BACKGROUND:

The current study was conducted to determine characteristics distinguishing the 10-year survivor group in patients with systemic immunoglobulin light chain (AL) amyloidosis who underwent autologous stem cell transplantation (SCT).

METHODS:

The study group included all 74 patients with AL amyloidosis who underwent high-dose melphalan treatment supported by autologous SCT since the beginning of the Mayo Clinic's SCT program until prior to August 2001.

RESULTS:

A total of 32 patients (43%) patients survived for > 10 years. Statistically significant baseline differences in the 10-year survivor group included: 1) the number of organs involved; 2) septal thickness; 3) total cholesterol; and 4) urine total protein. The number of organs involved was the only predictor found on multivariable analysis. Depth of the response to therapy, as measured by the lowest posttransplantation serum free light chain level, was found to be the most significant indicator of durability of response.

CONCLUSIONS:

Autologous SCT can offer durable benefit for patients with AL amyloidosis. The number of organs involved offers the greatest pretreatment prognostic value, whereas the lowest posttransplantation serum free light chain level offers the best posttreatment prognostic value. Cancer 2012. © 2012 American Cancer Society.