Outcomes of Patients with Light Chain Amyloidosis Who Had Autologous Stem Cell Transplantation with 3 or More Organs Involved

Abdullah S. Al Saleh, M. Hasib Sidiqi, Eli Muchtar, Angela Dispenzieri, Francis K. Buadi, David Dingli, Martha Q. Lacy, Rahma M. Warsame, Wilson I. Gonsalves, Taxiarchis V. Kourelis, William J. Hogan, Suzanne R. Hayman, Prashant Kapoor, Shaji K. Kumar, Morie A. Gertz

Research output: Contribution to journalArticlepeer-review

3 Scopus citations

Abstract

Prior reports have suggested that 3 or more organs involved is a contraindication for autologous stem cell transplant (ASCT) in amyloid light chain (AL) amyloidosis. Therefore, most centers limit transplantation to patients who have no more than 2 organs significantly involved. We retrospectively reviewed all patients with AL amyloidosis with ≥3 involved organs and who had ASCT between 1996 and 2015 at Mayo Clinic, Rochester, Minnesota to assess transplant safety and outcomes. Seventy-five patients with ≥3 organs involved underwent ASCT. Median age at diagnosis was 54 years, and 67% were men. The heart was involved in 95%, followed by the kidneys (84%). Thirty-eight patients (51%) had no induction treatment before ASCT. Full-dose melphalan (200 mg/m2) was given in 45%, and the remainder received 140 mg/m2. Overall hematologic response rate was 75%. The median progression-free survival (PFS) and overall survival (OS) were 16 and 68 months, respectively. The 100-day mortality was 16%, and 44 patients (59%) died during follow-up. The most common causes of death were cardiovascular events (32%) and progressive amyloidosis (25%). On multivariable analysis, predictors for PFS were Mayo 2012 stage III/IV (relative risk [RR], 3.3; P =.0012) and hematologic response (at least very good partial response; RR,.4; P =.012). An N-terminal pro–brain natriuretic peptide (NT-proBNP) level of ≥2000 pg/mL was an independent predictor for shorter PFS (RR, 2.6; P =.013). Predictors for OS included any hematologic response (RR,.12; P =.0015), melphalan 200 mg/m2 (RR,.2; P =.014), and Mayo 2012 stage III/IV (RR, 7.7; P =.0002). An NT-proBNP level ≥ 2000 pg/mL was a powerful predictor of OS (RR, 4; P =.013). The number of organs involved (3 versus >3) did not significantly impact PFS or OS. We conclude that the high prevalence and severity of cardiac involvement are the main drivers for the poor outcome in patients who have ≥3 organs involved. Using selection criteria defined for safe transplantation in cardiac amyloidosis should result in low therapy-related mortality independent of the number of organs involved. The severity of cardiac involvement should be the major criterion for transplanting patients with AL amyloidosis that have ≥3 organs involved and not merely the number of organs involved.

Original languageEnglish (US)
Pages (from-to)1520-1525
Number of pages6
JournalBiology of Blood and Marrow Transplantation
Volume25
Issue number8
DOIs
StatePublished - Aug 2019

Keywords

  • Autologous stem cell transplant
  • Light chain amyloidosis
  • Three or more organs

ASJC Scopus subject areas

  • Hematology
  • Transplantation

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