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Publication Detail
Autologous stem cell transplantation vs bortezomib based chemotheraphy for the firstā€line treatment of systemic light chain amyloidosis in the UK
  • Publication Type:
    Journal article
  • Publication Sub Type:
  • Authors:
    Sharpley FA, Manwani R, Petrie A, Mahmood S, Sachchithanantham S, Lachmann HJ, Martinez De Azcona Naharro A, Gillmore JD, Whelan CJ, Fontana M, Cohen O, Hawkins PN, Wechalekar AD
  • Publisher:
    John Wiley and Sons
  • Publication date:
  • Journal:
    European Journal of Haematology
  • Status:
  • Country:
  • Print ISSN:
  • Language:
  • Keywords:
    multiple myeloma, plasma cell neoplasms, transplantation
OBJECTIVES: The benefit of autologous stem cell transplantation (ASCT) in the treatment of light chain (AL) amyloidosis requires re-evaluation in the modern era. This retrospective case-matched study compares ASCT to bortezomib for the treatment of patients with AL amyloidosis. METHODS: Newly diagnosed patients with AL amyloidosis treated with ASCT or bortezomib between 2001-2018 were identified. Patients were excluded if the time from diagnosis to treatment exceeded 12 months. Patients were matched on a 1:1 basis, using a propensity matched scoring approach. RESULTS: A total of 136 propensity-score matched patients were included (ASCT n= 68, bortezomib n=68). There was no significant difference in overall survival at two years (p=0.908, HR: 0.95, CI:0.41-2.20). For ASCT vs. bortezomib: overall haematological response rate at six months was 90.6% vs. 92.5%; organ response at 12 months: cardiac (70.0% vs. 54%, p>0.999), renal (74% vs.24%, p=0.463)) liver (21% vs. 22%, p=0.048); median progression free survival (50 vs. 42 months p=0.058, HR:0.61, CI:0.37-1.02) and time to next treatment (68 vs. 45 months, p=0.145, HR:0.61, CI:0.31-1.19). More patients required treatment in the bortezomib group compared to ASCT group at 24 months (41 vs. 23, Chi squared p=0.004) and 48 months (57 vs 41, Chi squared p= 0.004). CONCLUSIONS: This small retrospective study suggests that there is no clear survival advantage of ASCT over bortezomib therapy. A prospective randomised controlled trial evaluating ASCT in AL amyloidosis is critically needed.
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