Outcomes from Autologous Hematopoietic Cell Transplantation versus Chemotherapy Alone for the Management of Light Chain Amyloidosis

Oluchi Oke, Tarsheen Sethi, Stacey Goodman, Sharon Phillips, Ilka Decker, Samuel Rubinstein, Beatrice Concepcion, Sarah Horst, Madan Jagasia, Adetola Kassim, Shelton L. Harrell, Anthony Langone, Daniel Lenihan, Kyle T. Rawling, David Slosky, Robert Frank Cornell

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2 Scopus citations

Abstract

Light chain amyloidosis (AL) results in tissue deposition of misfolded proteins, causing organ dysfunction. In an era of modern therapies, such as bortezomib, reassessment of the benefit of autologous hematopoietic cell transplantation (AHCT) should be considered. In this study, we compared outcomes between patients with AL receiving chemotherapy alone (CT) and those undergoing AHCT. Seventy-four patients with AL were analyzed retrospectively. Two cohorts of patients were studied, those receiving CT (n = 31) and those undergoing AHCT (n = 43). Of the 43 patients in the AHCT cohort, 29 received induction chemotherapy before AHCT, whereas 14 proceeded straight to AHCT without induction therapy. Compared with the CT cohort, patients in the AHCT cohort were younger and had higher ejection fractions, lower brain natriuretic peptide levels, and more severe proteinuria. The majority (87%) of patients in the CT cohort received bortezomib-based treatment. Transplantation-related mortality (TRM) was 7%. Patients receiving AHCT were more likely to achieve complete or very good partial response (P =.048). The median progression-free survival (PFS) and overall survival (OS) were superior in the AHCT cohort (not reached versus 9 months; P <.01 and 74 months versus 8 months; P =.03, respectively). Multivariable analysis demonstrated that improved PFS (hazard ratio, 3.86; 95% confidence interval [CI] 1.3 to 11.5; P =.02) and OS (hazard ratio, 5.6; 95% CI, 1.9 to 16; P <.001) were associated with use of AHCT compared with CT. Patients in the AHCT cohort had deeper and longer durations of response, with superior PFS and OS, compared with those in the CT cohort. Despite the limitations of this study, AHCT should be considered for eligible patients with AL at experienced transplantation centers that can offer this therapy with a low risk of TRM.

Original languageEnglish
Pages (from-to)1473-1477
Number of pages5
JournalBiology of Blood and Marrow Transplantation
Volume23
Issue number9
DOIs
StatePublished - Sep 2017
Externally publishedYes

Keywords

  • Bortezomib
  • Light chain amyloidosis
  • Organ response, chemotherapy
  • Transplantation

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    Oke, O., Sethi, T., Goodman, S., Phillips, S., Decker, I., Rubinstein, S., Concepcion, B., Horst, S., Jagasia, M., Kassim, A., Harrell, S. L., Langone, A., Lenihan, D., Rawling, K. T., Slosky, D., & Cornell, R. F. (2017). Outcomes from Autologous Hematopoietic Cell Transplantation versus Chemotherapy Alone for the Management of Light Chain Amyloidosis. Biology of Blood and Marrow Transplantation, 23(9), 1473-1477. https://doi.org/10.1016/j.bbmt.2017.05.020