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Plasma Cell Disorders

Outcome of AL amyloidosis after high-dose melphalan and autologous stem cell transplantation in Sweden, long-term results from all patients treated in 1994–2009

Abstract

High-dose melphalan and autologous stem cell transplantation (HDM/ASCT) is widely used in immunoglobulin light chain (AL) amyloidosis, but the benefit is debated mainly because of the high treatment-related mortality (24% in a randomised study comparing HDM/ASCT with oral melphalan/dexamethasone). We report here on the long-term outcome of all patients treated with HDM/ASCT for AL amyloidosis in Sweden between 1994 and 2009. Seventy-two patients were treated at eight Swedish centres. Median follow-up was 67.5 months. At least partial response (organ or haematological) was seen in 64% of the patients. Median overall survival was 98 months or 8.2 years, with 5-year survival 63.9% and 10-year survival 43.4%. In patients with cardiac involvement or multiple organ involvement, survival was significantly shorter, median overall survival 49 and 56 months, respectively. All mortality within 100 days from ASCT was 12.5% for all patients and 17.2% in the patients with cardiac involvement. For patients treated in the earlier time period (1994–2001), 100-day mortality was 23.8% compared with 7.8% in the later period (2002–2009). In conclusion, long survival times can be achieved in patients with AL amyloidosis treated with HDM/ASCT, also in smaller centres. Early mortality is high, but with a decreasing trend over time.

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References

  1. Falk RH, Comenzo RL, Skinner M . The systemic amyloidoses. N Engl J Med 1997; 337: 898–909.

    Article  CAS  Google Scholar 

  2. Kyle RA, Gertz MA . Primary systemic amyloidosis: clinical and laboratory features in 474 cases. Semin Hematol 1995; 32: 45–59.

    CAS  Google Scholar 

  3. Skinner M, Sanchorawala V, Seldin DC, Dember LM, Falk RH, Berk JL et al. High-dose melphalan and autologous stem-cell transplantation in patients with AL-amyloidosis: an 8-year study. Ann Intern Med 2004; 140: 85–93.

    Article  CAS  Google Scholar 

  4. Goodman HJB, Gillmore JD, Lachmann HJ, Wechalekar D, Bradwell AR, Hawkins PN . Outcome of autologous stem cell transplantation for AL amyloidosis in the UK. Br J Haematol 2006; 134: 417–425.

    Article  Google Scholar 

  5. Cibeira MT, Sanchorawala V, Seldin DC, Quillen K, Berk JL, Dember LM et al. Outcome of AL amyloidosis after high-dose melphalan and autologous stem cell transplantation: long-term results in a series of 421 patients. Blood 2011; 118: 4346–4352.

    Article  CAS  Google Scholar 

  6. Jimenez-Zepeda VH, Franke N, Reece DE, Trudel S, Chen C, Delgado DH et al. Autologous stem cell transplant is an effective therapy for carefully selected patients with AL amyloidosis: experience of a single institution. Br J Haematol 2014; 164: 722–728.

    Article  CAS  Google Scholar 

  7. Jaccard A, Moreau P, Leblond V, Leleu X, Benboubker L, Hermine O . High-dose melphalan versus melphalan plus dexamethasone for AL amyloidosis. N Engl J Med 2007; 357: 1083–1093.

    Article  CAS  Google Scholar 

  8. Dispenzieri A, Kyle RA, Lacy MQ, Therneau TM, Larson DR, Plevak MF et al. Superior survival in primary systemic amyloidosis patients undergoing peripheral blood stem cell transplantation: a case control study. Blood 2004; 103: 3960–3963.

    Article  CAS  Google Scholar 

  9. Gertz MA, Lacy MQ, Dispenzieri A, Kumar SK, Dingli D, Leung N et al. Refinement in patient selection to reduce treatment-related mortality from autologous stem cell transplantation in amyloidosis. Bone Marrow Transplant 2013; 48: 557–561.

    Article  CAS  Google Scholar 

  10. Gertz MA, Comenzo R, Falk RH, Fermand JP, Hazenberg BP, Hawkins PN et al. Definition of organ involvement and treatment response in immunoglobulin light chain amyloidosis (AL): a consensus opinion from the 10th International Symposium on Amyloid and Amyloidosis, Tours, France, 18-22 April 2004. Am J Hematol 2005; 79: 319–328.

    Article  Google Scholar 

  11. Sanchorawala V, Skinner M, Quillen K, Finn KT, Doros G, Seldin DC . Long-term outcome of patients with al amyloidosis treated with high-dose melphalan and stem-cell transplantation. Blood 2007; 110: 3561–3563.

    Article  CAS  Google Scholar 

  12. Dispenzieri A, Gertz MA, Kyle RA, Lacy MQ, Burritt MF, Therneau TM et al. Prognostication of survival using cardiac troponins and N-terminal pro-brain natriuretic peptide in patients with primary systemic amyloidosis undergoing peripheral blood stem cell transplantation. Blood 2004; 104: 1881–1887.

    Article  CAS  Google Scholar 

  13. Palladini G, Barassi A, Klersy C, Pacciolla R, Milani P, Sarais G et al. The combination of high-sensitivity cardiac troponin T (hs-cTnT) at presentation and changes in N-terminal natriuretic peptide type B (NT-proBNP) after chemotherapy best predicts survival in AL amyloidosis. Blood 2010; 116: 3426–3430.

    Article  CAS  Google Scholar 

  14. Schonland SO, Bochtler T, Perz J, Hansberg M, Mangatter A, Ho AD et al. Results of two consecutive phase II trials of patients with systemic AL amyloidosis treated with high-dose melphalan after induction and mobilisation chemotherapy. Amyloid 2010; 17: 80–81.

    Google Scholar 

  15. Gertz MA, Lacy MQ, Dispenzieri A, Ansell SM, Elliott MA, Gastineau DA et al. Risk-adjusted manipulation of melphalan dose before stem cell transplantation in patients with amyloidosis is associated with a lower response rate. Bone Marrow Transplant 2004; 34: 1025–1031.

    Article  CAS  Google Scholar 

  16. Tsai SB, Seldin DC, Quillen K, Berk JL, Ruberg FL, Meier-Ewert H et al. High-dose melphalan and stem cell transplantation for patients with AL amyloidosis: trends in treatment-related mortality over the past 17 years at a single referral center. Blood 2012; 120: 4445–4446.

    Article  CAS  Google Scholar 

  17. D’Souza A, Dispenzieri A, Wirk B, Zhang M-J, Huang J, Gertz MA et al. Improved outcomes after autologous hematopoietic cell transplantation for light chain amyloidosis: a Center for International Blood and Marrow Transplant Research Study. J Clin Oncol 2015; 33: 3741–3749.

    Article  Google Scholar 

  18. Herrmann S, Gertz MA, Stegall MD, Dispenzieri A, Cosio FC, Kumar S et al. Long-term outcomes of patients with light chain amyloidosis (AL) after renal transplantation with or without stem cell transplantation. Nephrol Dial Transplant 2011; 26: 2032–2036.

    Article  Google Scholar 

Download references

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Correspondence to S Rosengren.

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Rosengren, S., Mellqvist, UH., Nahi, H. et al. Outcome of AL amyloidosis after high-dose melphalan and autologous stem cell transplantation in Sweden, long-term results from all patients treated in 1994–2009. Bone Marrow Transplant 51, 1569–1572 (2016). https://doi.org/10.1038/bmt.2016.249

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