Blood Journal
Leading the way in experimental and clinical research in hematology

A European collaborative study of treatment outcomes in 346 patients with cardiac stage III AL amyloidosis

  1. Ashutosh D. Wechalekar1,*,
  2. Stefan O. Schonland2,
  3. Efstathios Kastritis3,
  4. Julian D. Gillmore1,
  5. Meletios A. Dimopoulos3,
  6. Thirusha Lane1,
  7. Andrea Foli4,
  8. Darren Foard1,
  9. Paolo Milani4,
  10. Lisa Rannigan1,
  11. Ute Hegenbart2,
  12. Philip N. Hawkins1,
  13. Giampaolo Merlini4, and
  14. Giovanni Palladini4
  1. 1 National Amyloidosis Centre, University College London Medical School, London, United Kingdom;
  2. 2 Amyloidosis Centre, Heidelberg University Hospital, Heidelberg, Germany;
  3. 3 Department of Clinical Therapeutics, University of Athens School of Medicine, Athens, Greece;
  4. 4 Amyloidosis Research and Treatment Centre, Fondazione IRCCS Policlinico San Matteo and Department of Molecular Medicine, University of Pavia, Pavia, Italy
  1. * Corresponding author; email: a.wechalekar{at}ucl.ac.uk

Key points

  • Deep clonal responses improve outcomes and can change the natural history of advanced (cardiac stage III) AL amyloidosis.

  • NT-proBNP >8500 ng/L and systolic blood pressure <100 mm of Hg identify a very poor risk subgroup of stage III AL amyloidosis.

Abstract

Treatment outcomes of patients with stage cardiac III AL amyloidosis remain poorly studied. Such cases have been excluded from most clinical studies due to perceived dismal prognosis. We report treatment outcomes of 346 patients with cardiac stage III AL amyloidosis from in UK, Italy, Germany and Greece. Median overall survival (OS) was 7 months with OS at 3, 6, 12 and 24 months of 73%, 55%, 46% and 29%, respectively. 42% died before first response evaluation. On intention to treat (ITT) basis, the overall haematological response rate was 33%, including a complete response rate (CR) of 12%. OS rates at 12 and 24 months, respectively, for 201 response evaluable patients were: for complete responders - 88% and 85%; partial responders - 74% and 53%; and non-responders - 39% and 22%. 45% of responders achieved an organ response. NT-proBNP >8500 ng/L and systolic blood pressure (SBP) <100 mm of Hg were the only factors that independently impacted OS and identified an especially poor prognosis subgroup of patients with median OS of only 3 months. Outcome and organ function of stage III AL amyloidosis without very elevated NT-proBNP and low SBP is improved by a very good haematological response to chemotherapy.

  • Submitted December 18, 2012.
  • Accepted February 26, 2013.