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

Blood - Tập 121 - Trang 3420-3427 - 2013
Ashutosh D. Wechalekar1, Stefan O. Schonland2, Efstathios Kastritis3, Julian D. Gillmore1, Meletios A. Dimopoulos3, Thirusha Lane1, Andrea Foli4, Darren Foard1, Paolo Milani4, Lisa Rannigan1, Ute Hegenbart2, Philip N. Hawkins1, Giampaolo Merlini4, Giovanni Palladini4
1National Amyloidosis Centre, University College London Medical School, London, United Kingdom
2Amyloidosis Centre, Heidelberg University Hospital, Heidelberg, Germany
3Department of Clinical Therapeutics, University of Athens School of Medicine, Athens, Greece
4Amyloidosis Research and Treatment Centre, Fondazione IRCCS Policlinico San Matteo and Department of Molecular Medicine, University of Pavia, Pavia, Italy

Tóm tắt

Key PointsDeep clonal responses improve outcomes and can change the natural history of advanced (cardiac stage III) AL amyloidosis. NT-proBNP >8500 ng/L and SBP <100 mm Hg identify a very poor risk subgroup of stage III AL amyloidosis.

Tài liệu tham khảo

Merlini, 2011, Amyloidosis: pathogenesis and new therapeutic options., J Clin Oncol, 29, 1924, 10.1200/JCO.2010.32.2271 Falk, 2005, Diagnosis and management of the cardiac amyloidoses., Circulation, 112, 2047, 10.1161/CIRCULATIONAHA.104.489187 Dispenzieri, 2003, Survival in patients with primary systemic amyloidosis and raised serum cardiac troponins., Lancet, 361, 1787, 10.1016/S0140-6736(03)13396-X Palladini, 2003, Serum N-terminal pro-brain natriuretic peptide is a sensitive marker of myocardial dysfunction in AL amyloidosis., Circulation, 107, 2440, 10.1161/01.CIR.0000068314.02595.B2 Kristen, 2010, Assessment of disease severity and outcome in patients with systemic light-chain amyloidosis by the high-sensitivity troponin T assay., Blood, 116, 2455, 10.1182/blood-2010-02-267708 Wechalekar, 2011, Abnormal N-terminal fragment of brain natriuretic peptide in patients with light chain amyloidosis without cardiac involvement at presentation is a risk factor for development of cardiac amyloidosis., Haematologica, 96, 1079, 10.3324/haematol.2011.040493 Dispenzieri, 2004, Serum cardiac troponins and N-terminal pro-brain natriuretic peptide: a staging system for primary systemic amyloidosis., J Clin Oncol, 22, 3751, 10.1200/JCO.2004.03.029 Dispenzieri, 2004, 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, 104, 1881, 10.1182/blood-2004-01-0390 Kumar, 2012, Revised prognostic staging system for light chain amyloidosis incorporating cardiac biomarkers and serum free light chain measurements., J Clin Oncol, 30, 989, 10.1200/JCO.2011.38.5724 Palladini, 2004, Association of melphalan and high-dose dexamethasone is effective and well tolerated in patients with AL (primary) amyloidosis who are ineligible for stem cell transplantation., Blood, 103, 2936, 10.1182/blood-2003-08-2788 Kastritis, 2010, Bortezomib with or without dexamethasone in primary systemic (light chain) amyloidosis., J Clin Oncol, 28, 1031, 10.1200/JCO.2009.23.8220 Venner, 2012, Cyclophosphamide, bortezomib, and dexamethasone therapy in AL amyloidosis is associated with high clonal response rates and prolonged progression-free survival., Blood, 119, 4387, 10.1182/blood-2011-10-388462 Mikhael, 2012, Cyclophosphamide-bortezomib-dexamethasone (CyBorD) produces rapid and complete hematologic response in patients with AL amyloidosis., Blood, 119, 4391, 10.1182/blood-2011-11-390930 Schönland, 2012, Current status of hematopoietic cell transplantation in the treatment of systemic amyloid light-chain amyloidosis., Bone Marrow Transplant, 47, 895, 10.1038/bmt.2011.152 Merlini, 2012, CyBorD: stellar response rates in AL amyloidosis., Blood, 119, 4343, 10.1182/blood-2012-03-413112 Palladini, 2010, 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, 116, 3426, 10.1182/blood-2010-05-286567 Lachmann, 2002, Misdiagnosis of hereditary amyloidosis as AL (primary) amyloidosis., N Engl J Med, 346, 1786, 10.1056/NEJMoa013354 Wechalekar, 2007, Safety and efficacy of risk-adapted cyclophosphamide, thalidomide, and dexamethasone in systemic AL amyloidosis., Blood, 109, 457, 10.1182/blood-2006-07-035352 Sanchorawala, 2007, Lenalidomide and dexamethasone in the treatment of AL amyloidosis: results of a phase 2 trial., Blood, 109, 492, 10.1182/blood-2006-07-030544 Dispenzieri, 2007, The activity of lenalidomide with or without dexamethasone in patients with primary systemic amyloidosis., Blood, 109, 465, 10.1182/blood-2006-07-032987 Gertz, 2005, 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, 79, 319, 10.1002/ajh.20381 Oken, 1982, Toxicity and response criteria of the Eastern Cooperative Oncology Group., Am J Clin Oncol, 5, 649, 10.1097/00000421-198212000-00014 Merlini, 2006, Dangerous small B-cell clones., Blood, 108, 2520, 10.1182/blood-2006-03-001164 Kyle, 1999, Long-term survival (10 years or more) in 30 patients with primary amyloidosis., Blood, 93, 1062, 10.1182/blood.V93.3.1062 Wechalekar, 2005, Efficacy of risk adapted cyclophosphamide, thalidomide and dexamethasone in systemic AL amyloidosis [abstract]., Blood, 106, 976A, 10.1182/blood.V106.11.3496.3496 Jaccard, 2007, High-dose melphalan versus melphalan plus dexamethasone for AL amyloidosis., N Engl J Med, 357, 1083, 10.1056/NEJMoa070484 Reece, 2011, Efficacy and safety of once-weekly and twice-weekly bortezomib in patients with relapsed systemic AL amyloidosis: results of a phase 1/2 study., Blood, 118, 865, 10.1182/blood-2011-02-334227 Palladini, 2009, Treatment of patients with advanced cardiac AL amyloidosis with oral melphalan, dexamethasone, and thalidomide., Ann Hematol, 88, 347, 10.1007/s00277-008-0600-y Dietrich, 2010, Treatment with intravenous melphalan and dexamethasone is not able to overcome the poor prognosis of patients with newly diagnosed systemic light chain amyloidosis and severe cardiac involvement., Blood, 116, 522, 10.1182/blood-2009-11-253237 Madan, 2012, High-dose melphalan and peripheral blood stem cell transplantation for light-chain amyloidosis with cardiac involvement., Blood, 119, 1117, 10.1182/blood-2011-07-370031 Bodin, 2010, Antibodies to human serum amyloid P component eliminate visceral amyloid deposits., Nature, 468, 93, 10.1038/nature09494 Ward, 2011, Doxycycline reduces fibril formation in a transgenic mouse model of AL amyloidosis., Blood, 118, 6610, 10.1182/blood-2011-04-351643