New insights and modern treatment of AL amyloidosis

Current Hematologic Malignancy Reports

Volumn 8, Issue 4, 2013, Pages 291-298

  Chaulagain, Chakra P ^a   Comenzo, Raymond L ^a  

^a TUFTS MEDICAL CENTER   (United States)

Author keywords

AL amyloidosis; Clonal plasma cell disease; Immunomodulatory agents; Proteasome inhibitors; Response criteria

Indexed keywords

AMYLOID A PROTEIN; BENDAMUSTINE; BIOLOGICAL MARKER; BORTEZOMIB; CARFILZOMIB; CYCLOPHOSPHAMIDE; DEXAMETHASONE; IXAZOMIB CITRATE; LENALIDOMIDE; MELPHALAN; PARAPROTEIN; POMALIDOMIDE; PREDNISONE; THALIDOMIDE; TROPONIN I; TROPONIN T;

ALBUMINURIA; AMYLOIDOSIS; ARTICLE; AUTOLOGOUS STEM CELL TRANSPLANTATION; AUTONOMIC NEUROPATHY; BONE MARROW BIOPSY; CARPAL TUNNEL SYNDROME; COMBINATION CHEMOTHERAPY; CYTOTOXICITY; DIASTOLIC HEART FAILURE; DISEASE COURSE; DRUG MEGADOSE; EARLY DIAGNOSIS; ELECTROCARDIOGRAM; FINE NEEDLE ASPIRATION BIOPSY; FLUID RETENTION; HEART LEFT VENTRICLE HYPERTROPHY; HEMATOLOGIC DISEASE; HUMAN; HYPERTENSION; LASER MICRODISSECTION; LIGHT CHAIN AMYLOIDOSIS; MASS SPECTROMETRY; MONOCLONAL IMMUNOGLOBULINEMIA; MORTALITY; MULTIPLE MYELOMA; MYELODYSPLASTIC SYNDROME; NEPHROTIC SYNDROME; OVERALL SURVIVAL; PARESTHESIA; PLASMA CELL; PROGNOSIS; PURPURA; TREATMENT RESPONSE;

AMYLOIDOSIS; ANTINEOPLASTIC AGENTS; COMBINED MODALITY THERAPY; DRUG THERAPY, COMBINATION; HUMANS; IMMUNOGLOBULIN LIGHT CHAINS; IMMUNOSUPPRESSIVE AGENTS; MYELOABLATIVE AGONISTS; RISK FACTORS; STEM CELL TRANSPLANTATION; TRANSPLANTATION, AUTOLOGOUS;

EID: 84890560462     PISSN: 15588211     EISSN: 1558822X     Source Type: Journal    
DOI: 10.1007/s11899-013-0175-0     Document Type: Article

References (59)

1. Falk RH, Comenzo RL, Skinner M. The Systemic Amyloidoses. N Engl J Med. 1997;337:898-909.
2. Kyle RA, Linos A, Beard CM, et al. Incidence and natural history of primary systemic amyloidosis in Olmsted County, Minnesota, 1950 through 1989. Blood. 1992;79(7):1817-22.
3. Pepys MB. Amyloidosis. Ann Rev med. 2006;57:223-42.
4. Comenzo RL. Current and emerging views and treatments of systemic immunoglobulin light-chain (AL) amyloidosis. Contrib Nephrol. 2007;153:195-210.
5. Brambilla F, Lavatelli F, Merlini G, Mauri P. Clinical proteomics for diagnosis and typing of systemic amyloidoses. Proteomics Clin Appl. 2013;7(1-2):136-43.
6. Comenzo RL, Zhou P, Fleisher M, Clark B, Teruya-Feldstein J. Seeking confidence in the diagnosis of systemic AL (Ig light-chain) amyloidosis: patients can have both monoclonal gammopathies and hereditary amyloid proteins. Blood. 2006;107(9):3489-91.
7. Merlini G, Westermark P. The systemic amyloidoses: clearer understanding of the molecular mechanisms offers hope for more effective therapies. J Int Med. 2004;255:159-78.
8. Comenzo RL. How I treat amyloidosis. Blood. 2009;114(15):3147-57. This is an excellent review for readers who want to grasp the fundamental basics of AL amyloidosis. Written in a case based format supported by in-depth and critical analysis of the literature in the field makes it a must read article by all who are interested in this disease.
9. Gertz MA, Kyle RA. Primary systemic smyloidosis-a diagnostic primer. Mayo Clin Proc. 1989;64(12):1505-19.
10. Merlini G, Wechalekar AD, Palladini G. Systemic light chain amyloidosis: an update for treating physicians. Blood. 2013 May 13. This is another must-read up-to-date review article likely to have wide spread reading and citations in the field of AL amyloidosis. The authors have done a commendable job in presenting the review concise yet complete for clinicians who diagnose and treat patients with AL amyloidosis.
11. Comenzo RL. Managing systemic light-chain amyloidosis. J Natl Compr Canc Netw. 2007;5(2):179-87.
12. Comenzo RL, Reece D, Palladini G, Seldin D, Sanchorawala V, Landau H, et al. Consensus guidelines for the conduct and reporting of clinical trials in systemic light-chain amyloidosis. Leukemia. 2012;26(11):2317-25. This article will have far reaching influences because it provides the basis for design and conduct of future clinical trial and pharma-academia collaboration for innovative strategies to our fight against AL amyloidosis.
13. Vrana JA, Gamez JD, Madden BJ, Theis JD, 3rd Bergen HR, Dogan A. Classification of amyloidosis by laser microdissection and mass spectrometry-based proteomic analysis in clinical biopsy specimens. Blood. 2009;114(24):4957-9. Laser microdissection and mass spectrometry has revolutionized the accuracy of amyloid typing and helping clinicians and researchers to distinguish AL from non-AL amyloidosis.
14. Hoffman JE, Hassoun H, Landau H, Comenzo RL. Coincidental Gammopathies in Patients with Systemic Amyloidosis and Transthyretin Gene Mutations. ASH 2010 Poster 2948 (II-828)
15. Dispenzieri A, Gertz MA, Kyle RA, Lacy MQ, Burritt MF, Therneau TM, et al. Serum cardiac troponins and N-terminal pro-brain natriuretic peptide: a staging system for primary systemic amyloidosis. J Clin Oncol. 2004;22(18):3751-7.
16. Katmann JA, Kyle RA, Benson J, et al. Screning panels for detection of monoclonal gammopathies. Clin Chem. 2009;55(8):1517-22.
17. Kyle RA, Gertz MA. Primary Systemic amyloidosis: clinical and laboratory features in 474 cases. Semin Hematol. 1995;32(1):45-59.
18. Lachmann HJ, Gallimore R, Gillmore JD, Carr-Smith HD, Bradwell AR, Pepys MB, et al. Outcome in systemic AL amyloidosis in relation to changes in concentration of circulating free immunoglobulin light chains following chemotherapy. Br J Haematol. 2003;122(1):78-84.
19. DA Palladini G, Gertz MA, Kumar S, Wechalekar A, Hawkins PN, Schönland S, et al. Validation of the Criteria of Response to Treatment In AL Amyloidosis. Blood. 2010;116:1364a.
20. Bochtler T, Hegenbart U, Benner A, Kunz C, Hose D, Seckinger A et al. Prognostic significance of cytogenetic aberrations in light chain amyloidosis patients treated with melphalan / dexamethasone as first-line therapy. XIIIth International Symposium on Amyloidosis. Groningen, The Netherlands, May 6-10, 2012. Abstract 15.15 - OP 40, p 52.
21. Zhou P, Hoffman J, Landau H, Hassoun H, Iyer L, Comenzo RL. Clonal plasma cell pathophysiology and clinical features of disease are linked to clonal plasma cell expression of cyclin D1 in systemic light-chain amyloidosis. Clin Lymphoma Myeloma Leuk. 2012;12(1):49-58. Both references 21 and 22 are about the information in the genetics of clonal plasma cells in the outcome of patients with AL amyloidosis. The emphasis in the genetic information should be the part of work up in clinics and in the translational researches in AL.
22. Bryce AH, Ketterling RP, Gertz MA, Lacy M, Knudson RA, Zeldenrust S, et al. Translocation t(11;14) and survival of patients with light chain (AL) amyloidosis. Haematologica. 2009;94(3):380-6. Both references 21 and 22 are about the information in the genetics of clonal plasma cells in the outcome of patients with AL amyloidosis. The emphasis in the genetic information should be the part of work up in clinics and in the translational researches in AL.
23. Trinkaus-Randall V, Walsh MT, Steeves S, Monis G, Connors LH, Skinner M. Cellular response of cardiac fibroblasts to amyloidogenic light chains. Am J Pahol. 2005;166(1):197-208.
24. Palladini G, Comenzo RL. The challenge of systemic immunoglobulin light-chain amyloidosis (Al). Subcell Biochem. 2012;65:609-42.
25. Merlini G. CyBorD: stellar response rates in AL amyloidosis. Blood. 2012;119(19):4343-5.
26. Dubrey SW, Comenzo RL. Amyloid diseases of the heart: current and future therapies. QJM. 2012;105(7):617-31.
27. Wechalekar AD, Schonland SO, Kastritis E, Gillmore JD, Dimopoulos MA, Lane T, et al. A European collaborative study of treatment outcomes in 346 patients with cardiac stage III AL amyloidosis. Blood. 2013;121(17):3420-7.
28. Dietrich S, Schönland SO, Benner A, Bochtler T, Kristen AV, Beimler J, et al. 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(4):522-8.
29. Reece DE, Hegenbart U, Sanchorawala V, Merlini G, Palladini G, Bladé J, et al. 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. 2011;118(4):865-73.
30. Sitia R, Palladini G, Merlini G. Bortezomib in the treatment of AL amyloidosis: targeted therapy? Haematologica. 2007;92(10):1302-7.
31. Mikhael JR, Schuster SR, Jimenez-Zepeda VH, Bello N, Spong J, Reeder CB. et al. Cyclophosphamide-bortezomib-dexamethasone (CyBorD) produces rapid and complete hematologic response in patients with AL amyloidosis. Blood. 2012;119(19):4391-4.
32. Venner CP, Lane T, Foard D, et al. Cyclophosphamide, bortezomib, and dexamethasone therapy in AL amyloidosis is associated with high clonal response rates and prolonged progression-free survival. Blood. 2012;119(19):4387-90.
33. Shah GL, Kaul E, Fallo S, Cossor FI, Smith H, Klein AK, et al. Subcutaneous Bortezomib in Combination Regimens in Newly Diagnosed Patients with Myeloma or Systemic AL Amyloidosis: High Response Rates and Minimal Toxicity. Blood (ASH Annual Meeting Abstracts #2968). 2012;120(21):2968.
34. Cohen AD, Zhou P, Chou J, Teruya-Feldstein J, Reich L, Hassoun H, et al. Risk-adapted autologous stem cell transplantation with adjuvant dexamethasone +/- thalidomide for systemic light-chain amyloidosis: results of a phase II trial. Br J Haematol. 2007;139(2):224-33
35. Landau H, Hassoun H, Rosenzweig MA, Maurer M, Liu J, Flombaum C, et al. Bortezomib and dexamethasone consolidation following risk-adapted melphalan and stem cell transplantation for patients with newly diagnosed light-chain amyloidosis. Leukemia. 2013;27(4):823-8.
36. Landau H, Hassoun H, Bello C, Hoover E, Riedel ER, Nimer SD, et al. Consolidation with bortezomib and dexamethasone following risk-adapted melphalan and stem cell transplant in systemic AL amyloidosis. Amyloid. 2011;18 Suppl 1:135-6.
37. Comenzo RL, Fein DE, Hassoun H, et al. Long-term outcomes of patients with systemic light chain amyloidosis (AL) treated at diagnosis with risk-adapted stem cell transplant and consolidation with novel agents. Blood (ASH Annual Meeting Abstracts). 2012;120(21):3150.
38. Palladini G, Perfetti V, Obici L, et al. 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(8):2936-8.
39. Palladini G, Russo P, Nuvolone M, et al. Treatment with oral melphalan plus dexamethasone produces long-term remissions in AL amyloidosis. Blood. 2007;110(2):787-8.
40. Kyle RA, Gertz MA, Greipp PR, Witzig TE, Lust JA, Lacy MQ, et al. A trial of three regimens for primary amyloidosis: colchicine alone, melphalan and prednisone, and melphalan, prednisone, and colchicines. N Engl J Med. 1997;336(17):1202-7.
41. Skinner M, Anderson J, Simms R, Falk R, Wang M, Libbey C, et al. Treatment of 100 patients with primary amyloidosis: a randomized trial of melphalan, prednisone, and colchicine versus colchicine only. Am J Med. 1996;100(3):290-8.
42. Gertz MA, Kyle RA. Acute leukemia and cytogenetic abnormalities complicating melphalan treatment of primary systemic amyloidosis. Arch Intern Med. 1990;150(3):629-33.
43. A Trial for Systemic Light-chain (AL) Amyloidosis (EMN-03). ClinicalTrials.gov Identifier:NCT01078454
44. Wechalekar AD, Goodman H, Lachmann H, Offer M, Hawkins P, Gillmore J. Safety and efficacy of risk-adapted cyclophosphamide, thalidomide, and dexamethasone in systemic AL amyloidosis. Blood. 2007;109(2):457-64.
45. Palladini G, Russo P, Foli A, Milani P, Lavatelli F, Obici L, et al. Salvage therapy with lenalidomide and dexamethasone in patients with advanced AL amyloidosis refractory to melphalan, bortezomib, and thalidomide. Ann Hematol. 2012;91(1):89-92.
46. Dispenzieri A, Buadi F, Laumann K, LaPlant B, Hayman SR, Kumar SK, et al. Activity of pomalidomide in patients with immunoglobulin light-chain amyloidosis. Blood. 2012;119(23):5397-404.
47. Kastritis E, Wechalekar AD, Dimopoulos MA, Merlini G, Hawkins PN, Perfetti V, et al. Bortezomib with or without dexamethasone in primary systemic (light chain) amyloidosis. J Clin Oncol. 2010;28(6):1031-7.
48. Lu J, Wang H, Huang XJ. Curative effect observation of patients with primary systemic amyloidosis treated by the combination of bortezomib with dexmethasone and cyclophosphamide. Zhonghua Xue Ye Xue Za Zhi. 2013;34(4):345-8. article in Chinese.
49. A Safety Study of Carfilzomib in Patients with Previously-Treated Systemic Light Chain Amyloidosis. ClinicalTrials.gov Identifier:NCT01789242
50. Study of Oral MLN9708 in Adult Patients with Relapsed or Refractory Light Chain Amyloidosis. ClinicalTrials.gov Identifier:NCT01318902
51. Study of Dexamethasone Plus MLN9708 or Physician's Choice of Treatment in Relapsed or Refractory Systemic Light Chain (AL) Amyloidosis. ClinicalTrials.gov Identifier:NCT01659658
52. Phase II Study of the Combination of Bendamustine and Dexamethasone in Patients with Relapsed AL Amyloidosis. ClinicalTrials.gov Identifier:NCT01222260
53. A Phase I/II Trial of Pomalidomide and Dexamethasone in Subjects with Previously-Treated AL Amyloidosis. ClinicalTrials.gov Identifier:NCT01570387
54. Pomalidomide and Dexamethasone (PDex) in AL Amyloidosis. ClinicalTrials.gov Identifier:NCT01510613
55. Comenzo RL. Who knows how to treat systemic light chain amyloidosis? Oncology (Williston Park). 2011;25(7):626, 628-9, 632-3
56. Suhr OB, Ericzon BG. Selection of hereditary transthyretin amyloid patients for liver transplantation: the Swedish experience. Amyloid. 2012;19 Suppl 1:78-80.
57. Coelho T, Maia LF. Martins da Silva A, Waddington Cruz M, Planté-Bordeneuve V, Lozeron P et al. Tafamidis for transthyretin familial amyloid polyneuropathy: a randomized, controlled trial. Neurology. 2012;79(8):785-92.
58. Dispenzieri A, Gertz MA, Buadi F. What do I need to know about immunoglobulin light chain (AL) amyloidosis? Blood Rev. 2012;26(4):137-54.
59. Comenzo RL, Gertz MA. Autologous stem cell transplantation for primary systemic amyloidosis. Blood. 2002;99(12):4276-82.