Management of systemic AL amyloidosis: Recommendations of the Myeloma Foundation of Australia Medical and Scientific Advisory Group

Internal Medicine Journal

Volumn 45, Issue 4, 2015, Pages 371-382

  Weber, N ^a   Mollee, P ^b,c   Augustson, B ^f   Brown, R ^g   Catley, L ^c,d,e   Gibson, J ^g   Harrison, S ^h   Ho, P J ^g   Horvath, N ^m   Jaksic, W ⁿ   Joshua, D ^g   Quach, H ⁱ   Roberts, A W ^j,k   Spencer, A ^l   Szer, J ^j   Talaulikar, D ^o,p   To, B ^m   Zannettino, A ^m   Prince, H M ^h,k  

^a ROYAL BRISBANE AND WOMEN'S HOSPITAL   (Australia)

^b PRINCESS ALEXANDRA HOSPITAL   (Australia)

^c UNIVERSITY OF QUEENSLAND   (Australia)

^d Mater Public Hospitals   (Australia)

^e MATER MEDICAL RESEARCH INSTITUTE   (Australia)

^f SIR CHARLES GAIRDNER HOSPITAL   (Australia)

^g ROYAL PRINCE ALFRED HOSPITAL   (Australia)

^h PETER MACCALLUM CANCER CENTRE   (Australia)

ⁱ ST VINCENT S HOSPITAL   (Australia)

^j ROYAL MELBOURNE HOSPITAL   (Australia)

^k UNIVERSITY OF MELBOURNE   (Australia)

^l ALFRED HOSPITAL   (Australia)

^m South Australia Pathology ^*  (Australia)

ⁿ QUEEN ELIZABETH HOSPITAL   (Australia)

^o CANBERRA HOSPITAL   (Australia)

^p AUSTRALIAN NATIONAL UNIVERSITY   (Australia)

more..

Author keywords

AL; Amyloidosis; Light chain; Plasma cell

Indexed keywords

ALKYLATING AGENT; AMIODARONE; AMYLOID; ANTIARRHYTHMIC AGENT; BENDAMUSTINE; BISPHOSPHONIC ACID DERIVATIVE; BORTEZOMIB; CARFILZOMIB; COLCHICINE; CYCLOPHOSPHAMIDE; DEXAMETHASONE; DIPEPTIDYL CARBOXYPEPTIDASE INHIBITOR; IMMUNOMODULATING AGENT; IXAZOMIB; LENALIDOMIDE; MELPHALAN; PLACEBO; POMALIDOMIDE; PREDNISOLONE; PROTEASOME INHIBITOR; THALIDOMIDE;

AMYLOIDOSIS; AUSTRALIA; AUTOLOGOUS STEM CELL TRANSPLANTATION; DRUG MEGADOSE; HEALTH CARE; HEART TRANSPLANTATION; HUMAN; LIGHT CHAIN; MORTALITY; MULTIPLE MYELOMA; MYELOMA; ORTHOSTATIC HYPOTENSION; OVERALL SURVIVAL; PRACTICE GUIDELINE; PRIORITY JOURNAL; PROGNOSIS; REVIEW; RISK; STAGING;

EID: 84964216447     PISSN: 14440903     EISSN: 14455994     Source Type: Journal    
DOI: 10.1111/imj.12566     Document Type: Review

References (53)

1. Kyle RA, Linos A, Beard CM, Linke RP, Gertz MA, O'Fallon WM etal. Incidence and natural history of primary systemic amyloidosis in Olmsted County, Minnesota, 1950 through 1989. Blood 1992; 79: 1817-1822.
2. Mollee P, Renaut P, Gottlieb D, Goodman H. How to diagnose amyloidosis. Intern Med J 2014; 44: 7-17.
3. The Oxford Levels of Evidence 2. Oxford centre for evidence-based medicine. [cited 2014 Jan 6]. Available from URL: http://www.cebm.net/index.aspx?o=5653
4. Kyle R, Gertz M. Primary systemic amyloidosis: clinical and laboratory features in 474 cases. Semin Hematol 1995; 32: 45-59.
5. Falk RH, Comenzo RL, Skinner M. The systemic amyloidoses. N Engl J Med 1997; 337: 898-909.
6. Quach H, Prince HM. Clinical practice guideline: multiple myeloma.[cited 2014 Jan 6]. Available from URL: http://www.myeloma.org.au
7. Dispenzieri A, Gertz MA, Kyle RA, Lacy MQ, Burritt MF, Therneau TM etal. Serum cardiac troponins and N-terminal pro-brain natriuretic peptide: a staging system for primary systemic amyloidosis. J Clin Oncol 2004; 22: 3751-3757.
8. Palladini G, Barassi A, Klersy C, Pacciolla R, Milani P, Sarais G etal. 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.
9. Palladini G, Foli A, Milani P, Russo P, Albertini R, Lavatelli F etal. Best use of cardiac biomarkers in patients with AL amyloidosis and renal failure. Am J Hematol 2012; 87: 465-471.
10. Bodin K, Ellmerich S, Kahan MC, Tennent GA, Loesch A, Gilbertson JA etal. Antibodies to human serum amyloid P component eliminate visceral amyloid deposits. Nature 2010; 468: 93-97.
11. Kyle RA, Greipp PR, Garton JP, Gertz MA. Primary systemic amyloidosis. Comparison of melphalan/prednisone versus colchicine. Am J Med 1985; 79: 708-716.
12. Kyle RA, Gertz MA, Greipp PR, Witzig TE, Lust JA, Lacy MQ etal. A trial of three regimens for primary amyloidosis: colchicine alone, melphalan and prednisone, and melphalan, prednisone, and colchicine. N Engl J Med 1997; 336: 1202-1207.
13. Palladini G, Perfetti V, Obici L, Caccialanza R, Semino A, Adami F etal. 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 2004; 103: 2936-2938.
14. Lebovic D, Hoffman J, Levine BM, Hassoun H, Landau H, Goldsmith Y etal. Predictors of survival in patients with systemic light-chain amyloidosis and cardiac involvement initially ineligible for stem cell transplantation and treated with oral melphalan and dexamethasone. Br J Haematol 2008; 143: 369-373.
15. Jaccard A, Moreau P, Leblond V, Leleu X, Benboubker L, Hermine O etal. High-dose melphalan versus melphalan plus dexamethasone for AL amyloidosis. N Engl J Med 2007; 357: 1083-1093.
16. Dietrich S, Schonland SO, Benner A, Bochtler T, Kristen AV, Beimler J etal. 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 2010; 116: 522-528.
17. Mollee P, Tiley C, Cunningham I, Moore J, Prince HM, Cannell P etal. A phase II study of risk-adapted intravenous melphalan in patients with AL amyloidosis. Br J Haematol 2012; 157: 766-769.
18. Mikhael JR, Schuster SR, Jimenez-Zepeda VH, Bello N, Spong J, Reeder CB etal. Cyclophosphamide-bortezomib-dexamethasone (CyBorD) produces rapid and complete hematologic response in patients with AL amyloidosis. Blood 2012; 119: 4391-4394.
19. Venner CP, Lane T, Foard D, Rannigan L, Gibbs SDJ, Pinney JH etal. Cyclophosphamide, bortezomib, and dexamethasone therapy in AL amyloidosis is associated with high clonal response rates and prolonged progression-free survival. Blood 2012; 119: 4387-4390.
20. Venner CP, Lane T, Foard D, Rannigan L, Mahmood S, Gibbs SDJ. A matched comparison of cyclophosphamide, bortezomib and dexamethasone (CVD) versus cyclophosphamide, thalidomide and dexamethasone (CTD) in the treatment of Mayo cardiac stage III patients with AL amyloidosis. Blood 2012; 120: 2966a.
21. Palladini G, Foli A, Milani P, Obici L, Lavatelli F, Nuvolone M. Melphalan and dexamethasone (MDex) vs. bortezomib, melphalan and dexamethasone (BMDex) in AL amyloidosis: a matched case control study. XIIIth International Symposium on Amyloidosis Abstract Book: University Medical Center Groningen. 2012: 204a.
22. Seldin DC, Choufani EB, Dember LM, Wiesman JF, Berk JL, Falk RH etal. Tolerability and efficacy of thalidomide for the treatment of patients with light chain-associated (AL) amyloidosis. Clin Lymphoma 2003; 3: 241-246.
23. Palladini G, Perfetti V, Perlini S, Obici L, Lavatelli F, Caccialanza R etal. The combination of thalidomide and intermediate-dose dexamethasone is an effective but toxic treatment for patients with primary amyloidosis (AL). Blood 2005; 105: 2949-2951.
24. Wechalekar AD, Goodman HJB, Lachmann HJ, Offer M, Hawkins PN, Gillmore JD. Safety and efficacy of risk-adapted cyclophosphamide, thalidomide, and dexamethasone in systemic AL amyloidosis. Blood 2007; 109: 457-464.
25. Sanchorawala V, Wright DG, Rosenzweig M, Finn KT, Fennessey S, Zeldis JB etal. Lenalidomide and dexamethasone in the treatment of AL amyloidosis: results of a phase 2 trial. Blood 2007; 109: 492-496.
26. Dispenzieri A, Lacy MQ, Zeldenrust SR, Hayman SR, Kumar SK, Geyer SM etal. The activity of lenalidomide with or without dexamethasone in patients with primary systemic amyloidosis. Blood 2007; 109: 465-470.
27. Moreau P, Jaccard A, Benboubker L, Royer B, Leleu X, Bridoux F etal. Lenalidomide in combination with melphalan and dexamethasone in patients with newly diagnosed AL amyloidosis: a multicenter phase 1/2 dose-escalation study. Blood 2010; 116: 4777-4782.
28. Kastritis E, Terpos E, Roussou M, Gavriatopoulou M, Pamboukas C, Boletis I etal. A phase I/II study of lenalidomide with low dose oral cyclophosphamide and low dose dexamethasone (RdC) in AL amyloidosis. Blood 2012; 119: 5384-5390.
29. Kumar SK, Hayman SR, Buadi FK, Roy V, Lacy MQ, Gertz MA etal. Lenalidomide, cyclophosphamide, and dexamethasone (CRd) for light-chain amyloidosis: long-term results from a phase 2 trial. Blood 2012; 119: 4860-4867.
30. Comenzo RL, Gertz MA. Autologous stem cell transplantation for primary systemic amyloidosis. Blood 2002; 99: 4276-4282.
31. Saba N, Sutton D, Ross H, Siu S, Crump R, Keating A etal. High treatment-related mortality in cardiac amyloid patients undergoing autologous stem cell transplant. Bone Marrow Transplant 1999; 24: 853-855.
32. Gertz M, Lacy M, Dispenzieri A, Hayman S, Kumar S, Buadi F etal. Troponin T level as an exclusion criterion for stem cell transplantation in light-chain amyloidosis. Leuk Lymphoma 2008; 49: 36-41.
33. Jimenez-Zepeda VH, Franke N, Reece DE, Trudel S, Chen S, Delgado DH etal. 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.
34. Palladini G, Perfetti V, Merlini G. Therapy and management of systemic AL (primary) amyloidosis. Swiss Med Wkly 2006; 136: 715-720.
35. Gertz MA, Lacy MQ, Dispenzieri A, Hayman SR, Kumar SK, Dingli D etal. Autologous stem cell transplant for immunoglobulin light chain amyloidosis: a status report. Leuk Lymphoma 2010; 51: 2181-2187.
36. Cibeira MT, Sanchorawala V, Seldin DC, Quillen K, Berk JL, Dember LM etal. 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.
37. Seldin DC, Andrea N, Berenbaum I, Berk JL, Connors L, Dember LM etal. High-dose melphalan and autologous stem cell transplantation for AL amyloidosis: recent trends in treatment-related mortality and 1-year survival at a single institution. Amyloid 2011; 18(Suppl 1): 122-124.
38. Mhaskar R, Kumar A, Behera M, Kharfan-Dabaja MA, Djulbegovic B. Role of high-dose chemotherapy and autologous hematopoietic cell transplantation in primary systemic amyloidosis: a systematic review. Biol Blood Marrow Transplant 2009; 15: 893-902.
39. Comenzo RL, Reece D, Palladini G, Seldin D, Sanchorawala V, Landau H etal. Consensus guidelines for the conduct and reporting of clinical trials in systemic light-chain (AL) amyloidosis. Leukemia 2012; 26: 2317-2325.
40. Palladini G, Dispenzieri A, Gertz MA, Kumar S, Wechalekar A, Hawkins PN etal. New criteria for response to treatment in immunoglobulin light chain amyloidosis based on free light chain measurement and cardiac biomarkers: impact on survival outcomes. J Clin Oncol 2012; 30: 4541-4549.
41. Dispenzieri A, Lacy MQ, Katzmann JA, Rajkumar SV, Abraham RS, Hayman SR etal. Absolute values of immunoglobulin free light chains are prognostic in patients with primary systemic amyloidosis undergoing peripheral blood stem cell transplantation. Blood 2006; 107: 3378-3383.
42. Lachmann HJ, Gallimore R, Gillmore JD, Carr-Smith HD, Bradwell AR, Pepys MB etal. Outcome in systemic AL amyloidosis in relation to changes in concentration of circulating free immunoglobulin light chains following chemotherapy. Br J Haematol 2003; 122: 78-84.
43. Palladini G, Lavatelli F, Russo P, Perlini S, Perfetti V, Bosoni T etal. Circulating amyloidogenic free light chains and serum N-terminal natriuretic peptide type B decrease simultaneously in association with improvement of survival in AL. Blood 2006; 107: 3854-3858.
44. Perlini S, Musca F, Salinaro F, Fracchioni I, Palladini G, Obici L etal. Functional correlates of N-terminal natriuretic peptide type B (NT-proBNP) response to therapy in cardiac light chain (AL) amyloidosis. Amyloid 2011; 18(Suppl 1): 91-92.
45. Mollee P, Tate J, Pretorius CJ. Evaluation of the N Latex free light chain assay in the diagnosis and monitoring of AL amyloidosis. Clin Chem Lab Med 2013; 51: 2303-2310.
46. Dispenzieri A, Dingli D, Kumar SK, Rajkumar SV, Lacy MQ, Hayman S etal. Discordance between serum cardiac biomarker and immunoglobulin-free light-chain response in patients with immunoglobulin light-chain amyloidosis treated with immune modulatory drugs. Am J Hematol 2010; 85: 757-759.
47. Wechalekar AD, Gillmore JD, Foard D, Rannigan L, Lane T, Pinney JH etal. Evaluation of an early switch to second line chemotherapy in AL amyloidosis among patients who fail to achieve a very good partial response to frontline treatment. XIIIth International Symposium on Amyloidosis. Groningen, The Netherlands: International Society of Amyloidosis 2012.
48. Palladini G, Dispenzieri A, Gertz M, Kumar S, Wechalekar A, Hawkins PN. Reply to S Girnius etal. J Clin Oncol 2013; 31: 2750-2751.
49. Gertz MA, Lacy MQ, Dispenzieri A. Therapy for immunoglobulin light chain amyloidosis: the new and the old. Blood Rev 2004; 18: 17-37.
50. Kapoor P, Thenappan T, Singh E, Kumar S, Greipp PR. Cardiac amyloidosis: a practical approach to diagnosis and management. Am J Med 2011; 124: 1006-1015.
51. Kristen AV, Dengler TJ, Hegenbart U, Schonland SO, Goldschmidt H, Sack F-U etal. Prophylactic implantation of cardioverter-defibrillator in patients with severe cardiac amyloidosis and high risk for sudden cardiac death. Heart Rhythm 2008; 55: 235-240.
52. Lacy MQ, Dispenzieri A, Hayman SR, Kumar S, Kyle R, Rajkumar SV etal. Autologous stem cell transplant after heart transplant for light chain (AL) amyloid cardiomyopathy. J Heart Lung Transplant 2008; 27: 823-829.
53. Gertz MA, Kyle RA, O'Fallon WM. Dialysis support of patients with primary systemic amyloidosis. A study of 211 patients. Arch Intern Med 1992; 152: 2245-2250.