References
Key articles
National Comprehensive Cancer Network. NCCN clinical practice guidelines in oncology: systemic light chain amyloidosis [internet publication].Full text
Dispenzieri A, Kyle R, Merlini G, et al. International Myeloma Working Group guidelines for serum-free light chain analysis in multiple myeloma and related disorders. Leukemia. 2009 Feb;23(2):215-24. Abstract
Gillmore JD, Wechalekar A, Bird J, et al. Guidelines on the diagnosis and investigation of AL amyloidosis. Br J Haematol. 2015 Jan;168(2):207-18.Full text Abstract
Dispenzieri A, Buadi F, Kumar SK, et al. Treatment of immunoglobulin light chain amyloidosis: Mayo stratification of myeloma and risk-adapted therapy (mSMART) consensus statement. Mayo Clin Proc. 2015 Aug;90(8):1054-81.Full text Abstract
Adams D, Suhr OB, Hund E, et al. First European consensus for diagnosis, management, and treatment of transthyretin familial amyloid polyneuropathy. Curr Opin Neurol. 2016 Feb;29(suppl 1):S14-26.Full text Abstract
Reference articles
1. Gertz MA, Comenzo R, Falk RH, 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 Aug;79(4):319-28. Abstract
2. International Myeloma Working Group. Criteria for the classification of monoclonal gammopathies, multiple myeloma and related disorders: a report of the International Myeloma Working Group. Br J Haematol. 2003 Jun;121(5):749-57. Abstract
3. Buxbaum JN, Dispenzieri A, Eisenberg DS, et al. Amyloid nomenclature 2022: update, novel proteins, and recommendations by the International Society of Amyloidosis (ISA) Nomenclature Committee. Amyloid. 2022 Dec;29(4):213-9.Full text Abstract
4. Kumar N, Zhang NJ, Cherepanov D, et al. Global epidemiology of amyloid light-chain amyloidosis. Orphanet J Rare Dis. 2022 Jul 19;17(1):278.Full text Abstract
5. Quock TP, Yan T, Chang E, et al. Epidemiology of AL amyloidosis: a real-world study using US claims data. Blood Adv. 2018 May 22;2(10):1046-53.Full text Abstract
6. Bergesio F, Ciciani AM, Santostefano M, et al. Renal involvement in systemic amyloidosis - an Italian retrospective study on epidemiological and clinical data at diagnosis. Nephrol Dial Transplant. 2007 Jun;22(6):1608-18. Abstract
7. Staron A, Connors LH, Zheng L, et al. Race/ethnicity in systemic AL amyloidosis: perspectives on disease and outcome disparities. Blood Cancer J. 2020 Nov 10;10(11):118.Full text Abstract
8. National Cancer Institute: Surveillance, Epidemiology, and End Results Program. Cancer stat facts: myeloma [internet publication].Full text
9. Wadhera RK, Rajkumar SV. Prevalence of monoclonal gammopathy of undetermined significance: a systematic review. Mayo Clin Proc. 2010 Oct;85(10):933-42. Abstract
10. Landgren O, Weiss BM. Patterns of monoclonal gammopathy of undetermined significance and multiple myeloma in various ethnic/racial groups: support for genetic factors in pathogenesis. Leukemia. 2009 Oct;23(10):1691-7.Full text Abstract
11. Hazenberg BP, van Rijswijk MH. Where has secondary amyloid gone? Ann Rheum Dis. 2000 Aug;59(8):577-9.Full text Abstract
12. Lachmann HJ, Goodman HJB, Gilbertson JA, et al. Natural history and outcome in systemic AA amyloidosis. N Engl J Med. 2007 Jun 7;356(23):2361-71.Full text Abstract
13. Lane T, Pinney JH, Gilbertson JA, et al. Changing epidemiology of AA amyloidosis: clinical observations over 25 years at a single national referral centre. Amyloid. 2017 Sep;24(3):162-6. Abstract
14. Pinney JH, Smith CJ, Taube JB, et al. Systemic amyloidosis in England: an epidemiological study. Br J Haematol. 2013 May;161(4):525-32.Full text Abstract
15. Wechalekar AD, Gillmore JD, Hawkins PN. Systemic amyloidosis. Lancet. 2016 Jun 25;387(10038):2641-54. Abstract
16. Rajkumar SV, Gertz MA, Kyle RA. Primary systemic amyloidosis with delayed progression to multiple myeloma. Cancer. 1998 Apr 15;82(8):1501-5.Full text Abstract
17. Kourelis TV, Kumar SK, Gertz MA, et al. Coexistent multiple myeloma or increased bone marrow plasma cells define equally high-risk populations in patients with immunoglobulin light chain amyloidosis. J Clin Oncol. 2013 Dec 1;31(34):4319-24.Full text Abstract
18. Fayand A, Boutboul D, Galicier L, et al. Epidemiology of Castleman disease associated with AA amyloidosis: description of 2 new cases and literature review. Amyloid. 2019 Dec;26(4):197-202. Abstract
19. Bernabei L, Waxman A, Caponetti G, et al. AA amyloidosis associated with Castleman disease: a case report and review of the literature. Medicine (Baltimore). 2020 Feb;99(6):e18978.Full text Abstract
20. Lachmann HJ. Periodic fever syndromes. Best Pract Res Clin Rheumatol. 2017 Aug;31(4):596-609. Abstract
21. Delaleu J, Deshayes S, Rodrigues F, et al. Tumour necrosis factor receptor-1 associated periodic syndrome (TRAPS)-related AA amyloidosis: a national case series and systematic review. Rheumatology (Oxford). 2021 Dec 1;60(12):5775-84.Full text Abstract
22. Rodrigues F, Cuisset L, Cador-Rousseau B, et al. AA amyloidosis complicating cryopyrin-associated periodic syndrome: a study of 86 cases including 23 French patients and systematic review. Rheumatology (Oxford). 2022 Nov 28;61(12):4827-34. Abstract
23. Lachmann HJ, Goodman HJ, Andrews PA, et al. AA amyloidosis complicating hyperimmunoglobulinemia D with periodic fever syndrome: a report of two cases. Arthritis Rheum. 2006 Jun;54(6):2010-4.Full text Abstract
24. Larsen CP, Ismail W, Kurtin PJ, et al. Leukocyte chemotactic factor 2 amyloidosis (ALECT2) is a common form of renal amyloidosis among Egyptians. Mod Pathol. 2016 Apr;29(4):416-20.Full text Abstract
25. Comenzo RL, Zhou P, Fleisher M, et al. Seeking confidence in the diagnosis of systemic AL (Ig light-chain) amyloidosis: patients can have both monoclonal gammopathies and hereditary amyloid proteins. Blood. 2006 May 1;107(9):3489-91.Full text Abstract
26. Lachmann HJ, Booth DR, Booth SE, et al. Misdiagnosis of hereditary amyloidosis as AL (primary) amyloidosis. N Engl J Med. 2002 Jun 6;346(23):1786-91.Full text Abstract
27. Merlini G, Dispenzieri A, Sanchorawala V, et al. Systemic immunoglobulin light chain amyloidosis. Nat Rev Dis Primers. 2018 Oct 25;4(1):38. Abstract
28. Pozzi C, Locatelli F. Kidney and liver involvement in monoclonal light chain disorders. Semin Nephrol. 2002 Jul;22(4):319-30. Abstract
29. Randles EG, Thompson JR, Martin DJ, et al. Structural alterations within native amyloidogenic immunoglobulin light chains. J Mol Biol. 2009 May 29;389(1):199-210.Full text Abstract
30. Gu M, Wilton R, Stevens FJ. Diversity and diversification of light chains in myeloma: the specter of amyloidogenesis by proxy. Contrib Nephrol. 2007;153:156-81. Abstract
31. Comenzo RL, Zhang Y, Martinez C, et al. The tropism of organ involvement in primary systemic amyloidosis: contributions of Ig V(L) germ line gene use and clonal plasma cell burden. Blood. 2001 Aug 1;98(3):714-20.Full text Abstract
32. Gertz MA, Leung N, Lacy MQ, et al. Clinical outcome of immunoglobulin light chain amyloidosis affecting the kidney. Nephrol Dial Transplant. 2009 Oct;24(10):3132-7.Full text Abstract
33. Merlini G, Pozzi C. Mechanisms of renal damage in plasma cell dyscrasias: an overview. Contrib Nephrol. 2007;153:66-86. Abstract
34. Gertz MA, Lacy MQ, Dispenzieri A, et al. Amyloidosis: diagnosis and management. Clin Lymphoma Myeloma. 2005 Nov;6(3):208-19. Abstract
35. Shah KB, Inoue Y, Mehra MR. Amyloidosis and the heart: a comprehensive review. Arch Intern Med. 2006 Sep 25;166(17):1805-13.Full text Abstract
36. Muchtar E, Gertz MA, Lacy MQ, et al. Ten-year survivors in AL amyloidosis: characteristics and treatment pattern. Br J Haematol. 2019 Dec;187(5):588-94.Full text Abstract
37. Martinez-Naharro A, Hawkins PN, Fontana M. Cardiac amyloidosis. Clin Med (Lond). 2018 Apr 1;18(suppl 2):s30-s35.Full text Abstract
38. Falk RH, Alexander KM, Liao R, et al. AL (Light-Chain) Cardiac Amyloidosis: A Review of Diagnosis and Therapy. J Am Coll Cardiol. 2016 Sep 20;68(12):1323-41.Full text Abstract
39. Perugini E, Rapezzi C, Reggiani LB, et al. Comparison of ventricular long-axis function in patients with cardiac amyloidosis versus idiopathic restrictive cardiomyopathy. Am J Cardiol. 2005 Jan 1;95(1):146-9. Abstract
40. Palladini G, Merlini G. What is new in diagnosis and management of light chain amyloidosis? Blood. 2016 Jul 14;128(2):159-68.Full text Abstract
41. Gertz MA, Lacy MQ, Dispenzieri A, et al. Amyloidosis. Best Pract Res Clin Haematol. 2005;18(4):709-27. Abstract
42. Freeman R. Autonomic peripheral neuropathy. Lancet. 2005 Apr 2-8;365(9466):1259-70. Abstract
43. Siddiqi OK, Ruberg FL. Cardiac amyloidosis: an update on pathophysiology, diagnosis, and treatment. Trends Cardiovasc Med. 2018 Jan;28(1):10-21. Abstract
44. Kittleson MM, Maurer MS, Ambardekar AV, et al. Cardiac amyloidosis: evolving diagnosis and management: a scientific statement from the American Heart Association. Circulation. 2020 Jul 7;142(1):e7-22.Full text Abstract
45. Grogan M, Scott CG, Kyle RA, et al. Natural History of Wild-Type Transthyretin Cardiac Amyloidosis and Risk Stratification Using a Novel Staging System. J Am Coll Cardiol. 2016 Sep 6;68(10):1014-20.Full text Abstract
46. Kyle RA, Therneau TM, Rajkumar SV, et al. A long-term study of prognosis in monoclonal gammopathy of undetermined significance. N Engl J Med. 2002 Feb 21;346(8):564-9.Full text Abstract
47. Papa R, Lachmann HJ. Secondary, AA, amyloidosis. Rheum Dis Clin North Am. 2018 Nov;44(4):585-603. Abstract
48. Palladini G, Milani P, Merlini G. Management of AL amyloidosis in 2020. Blood. 2020 Dec 3;136(23):2620-7.Full text Abstract
49. Muchtar E, Gertz MA, Kumar SK, et al. Improved outcomes for newly diagnosed AL amyloidosis between 2000 and 2014: cracking the glass ceiling of early death. Blood. 2017 Apr 13;129(15):2111-2119.Full text Abstract
50. Neben-Wittich MA, Wittich CM, Mueller PS, et al. Obstructive intramural coronary amyloidosis and myocardial ischemia are common in primary amyloidosis. Am J Med. 2005 Nov;118(11):1287. Abstract
51. Conceição I, González-Duarte A, Obici L, Schmidt HH, Simoneau D, Ong ML, Amass L. "Red-flag" symptom clusters in transthyretin familial amyloid polyneuropathy. J Peripher Nerv Syst. 2016 Mar;21(1):5-9.Full text Abstract
52. Planté-Bordeneuve V, Said G. Familial amyloid polyneuropathy. Lancet Neurol. 2011 Dec;10(12):1086-97. Abstract
53. Kaku M, Berk JL. Neuropathy associated with systemic amyloidosis. Semin Neurol. 2019 Oct;39(5):578-88. Abstract
54. Gonzalez-Duarte A, Valdés-Ferrer SI, Cantú-Brito C. Characteristics and natural history of autonomic involvement in hereditary ATTR amyloidosis: a systematic review. Clin Auton Res. 2019 Sep;29(suppl 1):1-9.Full text Abstract
55. Vaxman I, Gertz M. When to suspect a diagnosis of amyloidosis. Acta Haematol. 2020;143(4):304-11.Full text Abstract
56. Kapoor M, Rossor AM, Jaunmuktane Z, et al. Diagnosis of amyloid neuropathy. Pract Neurol. 2019 Jun;19(3):250-8. Abstract
57. Kyle RA, Gertz MA. Primary systemic amyloidosis: clinical and laboratory features in 474 cases. Semin Hematol. 1995 Jan;32(1):45-59. Abstract
58. National Comprehensive Cancer Network. NCCN clinical practice guidelines in oncology: systemic light chain amyloidosis [internet publication].Full text
59. Dispenzieri A, Kyle R, Merlini G, et al. International Myeloma Working Group guidelines for serum-free light chain analysis in multiple myeloma and related disorders. Leukemia. 2009 Feb;23(2):215-24. Abstract
60. Katzmann JA, Abraham RS, Dispenzieri A, et al. Diagnostic performance of quantitative kappa and lambda free light chain assays in clinical practice. Clin Chem. 2005 May;51(5):878-81.Full text Abstract
61. Morris KL, Tate JR, Gill D, et al. Diagnostic and prognostic utility of the serum free light chain assay in patients with AL amyloidosis. Intern Med J. 2007 Jul;37(7):456-63. Abstract
62. Gillmore JD, Reilly MM, Coats CJ, et al. Clinical and genetic evaluation of people with or at risk of hereditary ATTR amyloidosis: an expert opinion and consensus on best practice in Ireland and the UK. Adv Ther. 2022 Jun;39(6):2292-301.Full text Abstract
63. Conceição I, Damy T, Romero M, et al. Early diagnosis of ATTR amyloidosis through targeted follow-up of identified carriers of TTR gene mutations. Amyloid. 2019 Mar;26(1):3-9.Full text Abstract
64. Gertz MA. The classification and typing of amyloid deposits. Am J Clin Pathol. 2004 Jun;121(6):787-9. Abstract
65. Gillmore JD, Wechalekar A, Bird J, et al. Guidelines on the diagnosis and investigation of AL amyloidosis. Br J Haematol. 2015 Jan;168(2):207-18.Full text Abstract
66. Hawkins PN, Lavender JP, Pepys MB. Evaluation of systemic amyloidosis by scintigraphy with 123I-labeled serum amyloid P component. N Engl J Med. 1990 Aug 23;323(8):508-13.Full text Abstract
67. Dorbala S, Ando Y, Bokhari S, et al. ASNC/AHA/ASE/EANM/HFSA/ISA/SCMR/SNMMI expert consensus recommendations for multimodality imaging in cardiac amyloidosis: part 1 of 2-evidence base and standardized methods of imaging. Circ Cardiovasc Imaging. 2021 Jul;14(7):e000029.Full text Abstract
68. Tuzovic M, Yang EH, Baas AS, et al. Cardiac amyloidosis: diagnosis and treatment strategies. Curr Oncol Rep. 2017 Jul;19(7):46. Abstract
69. Gertz M, Adams D, Ando Y, et al. Avoiding misdiagnosis: expert consensus recommendations for the suspicion and diagnosis of transthyretin amyloidosis for the general practitioner. BMC Fam Pract. 2020 Sep 23;21(1):198.Full text Abstract
70. Gillmore JD, Maurer MS, Falk RH, et al. Nonbiopsy diagnosis of cardiac transthyretin amyloidosis. Circulation. 2016 Jun 14;133(24):2404-12.Full text Abstract
71. Castano A, Haq M, Narotsky DL, et al. Multicenter study of planar technetium 99m pyrophosphate cardiac imaging: predicting survival for patients with ATTR cardiac amyloidosis. JAMA Cardiol. 2016 Nov 1;1(8):880-9.Full text Abstract
72. Dispenzieri A, Kyle RA, Gertz MA, et al. Survival in patients with primary systemic amyloidosis and raised serum cardiac troponins. Lancet. 2003 May 24;361(9371):1787-9. Abstract
73. Palladini G, Campana C, Klersy C, et al. Serum N-terminal pro-brain natriuretic peptide is a sensitive marker of myocardial dysfunction in AL amyloidosis. Circulation. 2003 May 20;107(19):2440-5.Full text Abstract
74. Zerbini CA, Anderson JJ, Kane KA, et al. Beta 2 microglobulin serum levels and prediction of survival in AL amyloidosis. Amyloid. 2002 Dec;9(4):242-6. Abstract
75. Palladini G, Russo P, Bosoni T, et al. Identification of amyloidogenic light chains requires the combination of serum-free light chain assay with immunofixation of serum and urine. Clin Chem. 2009 Mar;55(3):499-504.Full text Abstract
76. Koyama J, Ray-Sequin PA, Falk RH. Longitudinal myocardial function assessed by tissue velocity, strain, and strain rate tissue Doppler echocardiography in patients with AL (primary) cardiac amyloidosis. Circulation. 2003 May 20;107(19):2446-52.Full text Abstract
77. Weidemann F, Strotmann JM. Use of tissue Doppler imaging to identify and manage systemic diseases. Clin Res Cardiol. 2008 Feb;97(2):65-73. Abstract
78. Dispenzieri A, Gertz MA, Kyle RA, et al. Serum cardiac troponins and N-terminal pro-brain natriuretic peptide: a staging system for primary systemic amyloidosis. J Clin Oncol. 2004 Sep 15;22(18):3751-7. Abstract
79. Kumar S, Dispenzieri A, Lacy MQ, et al. Revised prognostic staging system for light chain amyloidosis incorporating cardiac biomarkers and serum free light chain measurements. J Clin Oncol. 2012 Mar 20;30(9):989-95. Abstract
80. Wechalekar AD, Schonland SO, Kastritis E, et al. A European collaborative study of treatment outcomes in 346 patients with cardiac stage III AL amyloidosis. Blood. 2013 Apr 25;121(17):3420-7.Full text Abstract
81. Palladini G, Dispenzieri A, Gertz MA, et al. 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 Dec 20;30(36):4541-9.Full text Abstract
82. Dispenzieri A, Buadi F, Kumar SK, et al. Treatment of immunoglobulin light chain amyloidosis: Mayo stratification of myeloma and risk-adapted therapy (mSMART) consensus statement. Mayo Clin Proc. 2015 Aug;90(8):1054-81.Full text Abstract
83. mSMART. Mayo consensus on AL amyloidosis: diagnosis, treatment and prognosis. Oct 2020 [internet publication].Full text
84. Muchtar E, Gertz MA, Lacy MQ, et al. Refining amyloid complete hematological response: Quantitative serum free light chains superior to ratio. Am J Hematol. 2020 Nov;95(11):1280-7. Abstract
85. Dorbala S, Ando Y, Bokhari S, et al. ASNC/AHA/ASE/EANM/HFSA/ISA/SCMR/SNMMI expert consensus recommendations for multimodality imaging in cardiac amyloidosis: part 2 of 2 - diagnostic criteria and appropriate utilization. Circ Cardiovasc Imaging. 2021 Jul;14(7):e000030.Full text Abstract
86. Garcia-Pavia P, Rapezzi C, Adler Y, et al. Diagnosis and treatment of cardiac amyloidosis: a position statement of the ESC working group on myocardial and pericardial diseases. Eur Heart J. 2021 Apr 21;42(16):1554-68.Full text Abstract
87. Rajkumar SV, Lacy MQ, Kyle RA. Monoclonal gammopathy of undetermined significance and smoldering multiple myeloma. Blood Rev. 2007 Sep;21(5):255-65. Abstract
88. Vaxman I, Gertz M. Recent advances in the diagnosis, risk stratification, and management of systemic light-chain amyloidosis. Acta Haematol. 2019;141(2):93-106.Full text Abstract
89. Comenzo RL, Gertz MA. Autologous stem cell transplantation for primary systemic amyloidosis. Blood. 2002 Jun 15;99(12):4276-82.Full text Abstract
90. Sidiqi MH, Aljama MA, Muchtar E, et al. Autologous stem cell transplant for immunoglobulin light chain amyloidosis patients aged 70 to 75. Biol Blood Marrow Transplant. 2018 Oct;24(10):2157-9.Full text Abstract
91. Jaccard A, Moreau P, Leblond V, et al. High-dose melphalan versus melphalan plus dexamethasone for AL amyloidosis. N Engl J Med. 2007 Sep 13;357(11):1083-93. Abstract
92. Mhaskar R, Kumar A, Behera M, et al. Role of high-dose chemotherapy and autologous hematopoietic cell transplantation in primary systemic amyloidosis: a systematic review. Biol Blood Marrow Transplant. 2009 Aug;15(8):893-902. Abstract
93. D'Souza A, Dispenzieri A, Wirk B, 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 Nov 10;33(32):3741-9.Full text Abstract
94. Sidiqi MH, Aljama MA, Buadi FK, et al. Stem cell transplantation for light chain amyloidosis: decreased early mortality over time. J Clin Oncol. 2018 May 1;36(13):1323-9.Full text Abstract
95. Miyazaki K, Suzuki K. Autologous hematopoietic cell transplantation versus chemotherapy alone for immunoglobulin light chain amyloidosis: a retrospective study. Clin Lymphoma Myeloma Leuk. 2019 Jul;19(7):413-22. Abstract
96. Oke O, Sethi T, Goodman S, et al. Outcomes from autologous hematopoietic cell transplantation versus chemotherapy alone for the management of light chain amyloidosis. Biol Blood Marrow Transplant. 2017 Sep;23(9):1473-7.Full text Abstract
97. Gertz MA. Immunoglobulin light chain amyloidosis: 2020 update on diagnosis, prognosis, and treatment. Am J Hematol. 2020 Jul;95(7):848-60. Abstract
98. Gustine JN, Staron A, Szalat RE, et al. Predictors of hematologic response and survival with stem cell transplantation in AL amyloidosis: a 25-year longitudinal study. Am J Hematol. 2022 Sep;97(9):1189-99. Abstract
99. Gertz MA, Lacy MQ, Dispenzieri A, et al. Transplantation for amyloidosis. Curr Opin Oncol. 2007 Mar;19(2):136-41. Abstract
100. National Comprehensive Cancer Network. NCCN clinical practice guidelines in oncology: hematopoietic cell transplantation (HCT) [internet publication].Full text
101. Landau H, Smith M, Landry C, et al. Long-term event-free and overall survival after risk-adapted melphalan and SCT for systemic light chain amyloidosis. Leukemia. 2017 Jan;31(1):136-42.Full text Abstract
102. Scott EC, Heitner SB, Dibb W, et al. Induction bortezomib in Al amyloidosis followed by high dose melphalan and autologous stem cell transplantation: a single institution retrospective study. Clin Lymphoma Myeloma Leuk. 2014 Oct;14(5):424-430.e1.Full text Abstract
103. Sanchorawala V, Brauneis D, Shelton AC, et al. Induction Therapy with Bortezomib Followed by Bortezomib-High Dose Melphalan and Stem Cell Transplantation for Light Chain Amyloidosis: Results of a Prospective Clinical Trial. Biol Blood Marrow Transplant. 2015 Aug;21(8):1445-51.Full text Abstract
104. Afrough A, Saliba RM, Hamdi A, et al. Impact of Induction Therapy on the Outcome of Immunoglobulin Light Chain Amyloidosis after Autologous Hematopoietic Stem Cell Transplantation. Biol Blood Marrow Transplant. 2018 Nov;24(11):2197-2203.Full text Abstract
105. Huang X, Wang Q, Chen W, et al. Induction therapy with bortezomib and dexamethasone followed by autologous stem cell transplantation versus autologous stem cell transplantation alone in the treatment of renal AL amyloidosis: a randomized controlled trial. BMC Med. 2014 Jan 6;12:2.Full text Abstract
106. Cornell RF, Zhong X, Arce-Lara C, et al. Bortezomib-based induction for transplant ineligible AL amyloidosis and feasibility of later transplantation. Bone Marrow Transplant. 2015 Jul;50(7):914-7.Full text Abstract
107. Cohen AD, Zhou P, Chou J, 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 Oct;139(2):224-33.Full text Abstract
108. Landau H, Hassoun H, Rosenzweig MA, et al. Bortezomib and dexamethasone consolidation following risk-adapted melphalan and stem cell transplantation for patients with newly diagnosed light-chain amyloidosis. Leukemia. 2013 Apr;27(4):823-8.Full text Abstract
109. Milani P, Basset M, Nuvolone M, et al. Indicators of profound hematologic response in AL amyloidosis: complete response remains the goal of therapy. Blood Cancer J. 2020 Sep 1;10(8):90.Full text Abstract
110. Tandon N, Sidana S, Dispenzieri A, et al. Impact of involved free light chain (FLC) levels in patients achieving normal FLC ratio after initial therapy in light chain amyloidosis (AL). Am J Hematol. 2018 Jan;93(1):17-22.Full text Abstract
111. Muchtar E, Dispenzieri A, Leung N, et al. Optimizing deep response assessment for AL amyloidosis using involved free light chain level at end of therapy: failure of the serum free light chain ratio. Leukemia. 2019 Feb;33(2):527-531.Full text Abstract
112. Al Saleh AS, Sidiqi MH, Sidana S, et al. Impact of consolidation therapy post autologous stem cell transplant in patients with light chain amyloidosis. Am J Hematol. 2019 Oct;94(10):1066-1071.Full text Abstract
113. Sanchorawala V, Wright DG, Rosenzweig M, et al. Lenalidomide and dexamethasone in the treatment of AL amyloidosis: results of a phase 2 trial. Blood. 2007 Jan 15;109(2):492-6.Full text Abstract
114. Specter R, Sanchorawala V, Seldin DC, et al. Kidney dysfunction during lenalidomide treatment for AL amyloidosis. Nephrol Dial Transplant. 2011 Mar;26(3):881-6.Full text Abstract
115. Dispenzieri A, Lacy MQ, Zeldenrust SR, et al. The activity of lenalidomide with or without dexamethasone in patients with primary systemic amyloidosis. Blood. 2007 Jan 15;109(2):465-70. Abstract
116. Sitia R, Palladini G, Merlini G. Bortezomib in the treatment of AL amyloidosis: targeted therapy? Haematologica. 2007 Oct;92(10):1302-7.Full text Abstract
117. Kastritis E, Anagnostopoulos A, Roussou M, et al. Treatment of light chain (AL) amyloidosis with the combination of bortezomib and dexamethasone. Haematologica. 2007 Oct;92(10):1351-8. Abstract
118. Comenzo RL, Reece D, Palladini G, et al. Consensus guidelines for the conduct and reporting of clinical trials in systemic light-chain amyloidosis. Leukemia. 2012 Nov;26(11):2317-25. Abstract
119. Gavriatopoulou M, Musto P, Caers J, et al. European myeloma network recommendations on diagnosis and management of patients with rare plasma cell dyscrasias. Leukemia. 2018 Sep;32(9):1883-1898.Full text Abstract
120. Kastritis E, Leleu X, Arnulf B, et al. Bortezomib, melphalan, and dexamethasone for light-chain amyloidosis. J Clin Oncol. 2020 Oct 1;38(28):3252-60.Full text Abstract
121. Kastritis E, Palladini G, Minnema MC, et al. Daratumumab-based treatment for immunoglobulin light-chain amyloidosis. N Engl J Med. 2021 Jul 1;385(1):46-58.Full text Abstract
122. Palladini G, Russo P, Nuvolone M, et al. Treatment with oral melphalan plus dexamethasone produces long-term remissions in AL amyloidosis. Blood. 2007 Jul 15;110(2):787-8. Abstract
123. Palladini G, Milani P, Foli A, et al. Oral melphalan and dexamethasone grants extended survival with minimal toxicity in AL amyloidosis: long-term results of a risk-adapted approach. Haematologica. 2014 Apr;99(4):743-50.Full text Abstract
124. Mikhael JR, Schuster SR, Jimenez-Zepeda VH, et al. Cyclophosphamide-bortezomib-dexamethasone (CyBorD) produces rapid and complete hematologic response in patients with AL amyloidosis. Blood. 2012 May 10;119(19):4391-4.Full text Abstract
125. 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 May 10;119(19):4387-90.Full text Abstract
126. Jaccard A, Comenzo RL, Hari P, et al. Efficacy of bortezomib, cyclophosphamide and dexamethasone in treatment-naïve patients with high-risk cardiac AL amyloidosis (Mayo Clinic stage III). Haematologica. 2014 Sep;99(9):1479-85.Full text Abstract
127. Palladini G, Sachchithanantham S, Milani P, et al. A European collaborative study of cyclophosphamide, bortezomib, and dexamethasone in upfront treatment of systemic AL amyloidosis. Blood. 2015 Jul 30;126(5):612-5.Full text Abstract
128. Manwani R, Cohen O, Sharpley F, et al. A prospective observational study of 915 patients with systemic AL amyloidosis treated with upfront bortezomib. Blood. 2019 Dec 19;134(25):2271-2280.Full text Abstract
129. Moreau P, Jaccard A, Benboubker L, et al. Lenalidomide in combination with melphalan and dexamethasone in patients with newly diagnosed AL amyloidosis: a multicenter phase 1/2 dose-escalation study. Blood. 2010 Dec 2;116(23):4777-82.Full text Abstract
130. Sanchorawala V, Patel JM, Sloan JM, et al. Melphalan, lenalidomide and dexamethasone for the treatment of immunoglobulin light chain amyloidosis: results of a phase II trial. Haematologica. 2013 May;98(5):789-92.Full text Abstract
131. Hegenbart U, Bochtler T, Benner A, et al. Lenalidomide/melphalan/dexamethasone in newly diagnosed patients with immunoglobulin light chain amyloidosis: results of a prospective phase 2 study with long-term follow up. Haematologica. 2017 Aug;102(8):1424-1431.Full text Abstract
132. Kastritis E, Dialoupi I, Gavriatopoulou M, et al. Primary treatment of light-chain amyloidosis with bortezomib, lenalidomide, and dexamethasone. Blood Adv. 2019 Oct 22;3(20):3002-9.Full text Abstract
133. Wechalekar AD, Goodman HJ, Lachmann HJ, et al. Safety and efficacy of risk-adapted cyclophosphamide, thalidomide, and dexamethasone in systemic AL amyloidosis. Blood. 2007 Jan 15;109(2):457-64. Abstract
134. Venner CP, Gillmore JD, Sachchithanantham S, et al. A matched comparison of cyclophosphamide, bortezomib and dexamethasone (CVD) versus risk-adapted cyclophosphamide, thalidomide and dexamethasone (CTD) in AL amyloidosis. Leukemia. 2014 Dec;28(12):2304-10.Full text Abstract
135. Liu B, Wang Y, Ning X, et al. A comparative study of cyclophosphamide, thalidomide and dexamethasone (CTD) versus bortezomib and dexamethasone (BDex) in light-chain amyloidosis. Curr Probl Cancer. 2021 Apr;45(2):100669.Full text Abstract
136. Seldin DC, Choufani EB, Dember LM, et al. Tolerability and efficacy of thalidomide for the treatment of patients with light chain-associated (AL) amyloidosis. Clin Lymphoma. 2003 Mar;3(4):241-6. Abstract
137. Gertz MA, Lacy MQ, Lust JA, et al. Phase II trial of high-dose dexamethasone for untreated patients with primary systemic amyloidosis. Med Oncol. 1999 Jul;16(2):104-9. Abstract
138. Mahmood S, Venner CP, Sachchithanantham S, et al. Lenalidomide and dexamethasone for systemic AL amyloidosis following prior treatment with thalidomide or bortezomib regimens. Br J Haematol. 2014 Sep;166(6):842-8.Full text Abstract
139. Palladini G, Milani P, Foli A, et al. A phase 2 trial of pomalidomide and dexamethasone rescue treatment in patients with AL amyloidosis. Blood. 2017 Apr 13;129(15):2120-23.Full text Abstract
140. Sanchorawala V, Palladini G, Kukreti V, et al. A phase 1/2 study of the oral proteasome inhibitor ixazomib in relapsed or refractory AL amyloidosis. Blood. 2017 Aug 3;130(5):597-605.Full text Abstract
141. Cohen OC, Sharpley F, Gillmore JD, et al. Use of ixazomib, lenalidomide and dexamethasone in patients with relapsed amyloid light-chain amyloidosis. Br J Haematol. 2020 May;189(4):643-9.Full text Abstract
142. ter Haar NM, Oswald M, Jeyaratnam J, et al. Recommendations for the management of autoinflammatory diseases. Ann Rheum Dis. 2015 Sep;74(9):1636-44.Full text Abstract
143. Okuda Y, Ohnishi M, Matoba K, et al. Comparison of the clinical utility of tocilizumab and anti-TNF therapy in AA amyloidosis complicating rheumatic diseases. Mod Rheumatol. 2014 Jan;24(1):137-43.Full text Abstract
144. Lane T, Wechalekar AD, Gillmore JD, et al. Safety and efficacy of empirical interleukin-1 inhibition using anakinra in AA amyloidosis of uncertain aetiology. Amyloid. 2017 Sep;24(3):189-193.Full text Abstract
145. Lachmann HJ, Gilbertson JA, Gillmore JD, et al. Unicentric Castleman's disease complicated by systemic AA amyloidosis: a curable disease. QJM. 2002 Apr;95(4):211-8.Full text Abstract
146. Lidar M, Livneh A. Familial Mediterranean fever: clinical, molecular and management advancements. Neth J Med. 2007 Oct;65(9):318-24.Full text Abstract
147. Wu B, Xu T, Li Y, et al. Interventions for reducing inflammation in familial Mediterranean fever. Cochrane Database Syst Rev. 2018 Oct 19;10:CD010893.Full text Abstract
148. Hoffman HM, Throne ML, Amar NJ, et al. Efficacy and safety of rilonacept (interleukin-1 Trap) in patients with cryopyrin-associated periodic syndromes: results from two sequential placebo-controlled studies. Arthritis Rheum. 2008 Aug;58(8):2443-52.Full text Abstract
149. Hoffman HM, Throne ML, Amar NJ, et al. Long-term efficacy and safety profile of rilonacept in the treatment of cryopryin-associated periodic syndromes: results of a 72-week open-label extension study. Clin Ther. 2012 Oct;34(10):2091-103.Full text Abstract
150. van der Hilst JCh, Moutschen M, Messiaen PE, et al. Efficacy of anti-IL-1 treatment in familial Mediterranean fever: a systematic review of the literature. Biologics. 2016 Apr 4;10:75-80. Abstract
151. Yilmaz S, Cinar M, Simsek I, et al. Tocilizumab in the treatment of patients with AA amyloidosis secondary to familial Mediterranean fever. Rheumatology (Oxford). 2015 Mar;54(3):564-5.Full text Abstract
152. Hashkes PJ, Spalding SJ, Giannini EH, et al. Rilonacept for colchicine-resistant or -intolerant familial Mediterranean fever: a randomized trial. Ann Intern Med. 2012 Oct 16;157(8):533-41.Full text Abstract
153. Ben-Zvi I, Kukuy O, Giat E, et al. Anakinra for Colchicine-Resistant Familial Mediterranean Fever: A Randomized, Double-Blind, Placebo-Controlled Trial. Arthritis Rheumatol. 2017 Apr;69(4):854-862.Full text Abstract
154. De Benedetti F, Gattorno M, Anton J, et al. Canakinumab for the Treatment of Autoinflammatory Recurrent Fever Syndromes. N Engl J Med. 2018 May 17;378(20):1908-1919.Full text Abstract
155. Ozen S, Ben-Cherit E, Foeldvari I, et al. Long-term efficacy and safety of canakinumab in patients with colchicine-resistant familial Mediterranean fever: results from the randomised phase III CLUSTER trial. Ann Rheum Dis. 2020 Oct;79(10):1362-1369.Full text Abstract
156. Kacar M, Savic S, van der Hilst JCH. The Efficacy, Safety and Tolerability of Canakinumab in the Treatment of Familial Mediterranean Fever: A Systematic Review of the Literature. J Inflamm Res. 2020;13:141-149.Full text Abstract
157. Adams D, Suhr OB, Hund E, et al. First European consensus for diagnosis, management, and treatment of transthyretin familial amyloid polyneuropathy. Curr Opin Neurol. 2016 Feb;29(suppl 1):S14-26.Full text Abstract
158. Ando Y, Coelho T, Berk JL, et al. Guideline of transthyretin-related hereditary amyloidosis for clinicians. Orphanet J Rare Dis. 2013 Feb 20;8:31.Full text Abstract
159. Tsuchiya A, Yazaki M, Kametani F, et al. Marked regression of abdominal fat amyloid in patients with familial amyloid polyneuropathy during long-term follow-up after liver transplantation. Liver Transpl. 2008 Apr;14(4):563-70.Full text Abstract
160. Ericzon BG, Wilczek HE, Larsson M, et al. Liver Transplantation for Hereditary Transthyretin Amyloidosis: After 20 Years Still the Best Therapeutic Alternative? Transplantation. 2015 Sep;99(9):1847-54.Full text Abstract
161. Okumura K, Yamashita T, Masuda T, et al. Long-term outcome of patients with hereditary transthyretin V30M amyloidosis with polyneuropathy after liver transplantation. Amyloid. 2016;23(1):39-45.Full text Abstract
162. Cristóbal Gutiérrez H, Pelayo-Negro AL, Gómez Gómez D, et al. Overview of treatments used in transthyretin-related hereditary amyloidosis: a systematic review. Eur J Hosp Pharm. 2020 Jul;27(4):194-201.Full text Abstract
163. Milani P, Mussinelli R, Perlini S, et al. An evaluation of patisiran: a viable treatment option for transthyretin-related hereditary amyloidosis. Expert Opin Pharmacother. 2019 Dec;20(18):2223-8. Abstract
164. Adams D, Gonzalez-Duarte A, O'Riordan WD, et al. Patisiran, an RNAi therapeutic, for hereditary transthyretin amyloidosis. N Engl J Med. 2018 Jul 5;379(1):11-21. Abstract
165. Adams D, Polydefkis M, González-Duarte A, et al. Long-term safety and efficacy of patisiran for hereditary transthyretin-mediated amyloidosis with polyneuropathy: 12-month results of an open-label extension study. Lancet Neurol. 2021 Jan;20(1):49-59.Full text Abstract
166. Benson MD, Waddington-Cruz M, Berk JL, et al. Inotersen treatment for patients with hereditary transthyretin amyloidosis. N Engl J Med. 2018 Jul 5;379(1):22-31. Abstract
167. Brannagan TH, Wang AK, Coelho T, et al. Early data on long-term efficacy and safety of inotersen in patients with hereditary transthyretin amyloidosis: a 2-year update from the open-label extension of the NEURO-TTR trial. Eur J Neurol. 2020 Aug;27(8):1374-1381.Full text Abstract
168. Coelho T, Maia LF, Martins da Silva A, et al. Tafamidis for transthyretin familial amyloid polyneuropathy: a randomized, controlled trial. Neurology. 2012 Aug 21;79(8):785-92.Full text Abstract
169. Barroso FA, Judge DP, Ebede B, et al. Long-term safety and efficacy of tafamidis for the treatment of hereditary transthyretin amyloid polyneuropathy: results up to 6 years. Amyloid. 2017 Sep;24(3):194-204.Full text Abstract
170. Greene MJ, Klimtchuk ES, Seldin DC, et al. Cooperative stabilization of transthyretin by clusterin and diflunisal. Biochemistry. 2015 Jan 20;54(2):268-78.Full text Abstract
171. Berk JL, Suhr OB, Obici L, et al. Repurposing diflunisal for familial amyloid polyneuropathy: a randomized clinical trial. JAMA. 2013 Dec 25;310(24):2658-67.Full text Abstract
172. Sekijima Y, Tojo K, Morita H, et al. Safety and efficacy of long-term diflunisal administration in hereditary transthyretin (ATTR) amyloidosis. Amyloid. 2015;22(2):79-83. Abstract
173. Dubrey SW, Davidoff R, Skinner M, et al. Progression of ventricular wall thickening after liver transplantation for familial amyloidosis. Transplantation. 1997 Jul 15;64(1):74-80.Full text Abstract
174. Herlenius G, Wilczek HE, Larsson M, et al. Ten years of international experience with liver transplantation for familial amyloidotic polyneuropathy: results from the Familial Amyloidotic Polyneuropathy World Transplant Registry. Transplantation. 2004 Jan 15;77(1):64-71.Full text Abstract
175. Maurer MS, Schwartz JH, Gundapaneni B, et al. Tafamidis treatment for patients with transthyretin amyloid cardiomyopathy. N Engl J Med. 2018 Sep 13;379(11):1007-16. Abstract
176. Damy T, Garcia-Pavia P, Hanna M, et al. Efficacy and safety of tafamidis doses in the tafamidis in transthyretin cardiomyopathy clinical trial (ATTR-ACT) and long-term extension study. Eur J Heart Fail. 2021 Feb;23(2):277-85.Full text Abstract
177. Rapezzi C, Elliott P, Damy T, et al. Efficacy of Tafamidis in Patients With Hereditary and Wild-Type Transthyretin Amyloid Cardiomyopathy: Further Analyses From ATTR-ACT. JACC Heart Fail. 2021 Feb;9(2):115-123.Full text Abstract
178. Adams D, Tournev IL, Taylor MS, et al. Efficacy and safety of vutrisiran for patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy: a randomized clinical trial. Amyloid. 2023 Mar;30(1):1-9.Full text Abstract
179. Kaufman GP, Schrier SL, Lafayette RA, et al. Daratumumab yields rapid and deep hematologic responses in patients with heavily pretreated AL amyloidosis. Blood. 2017 Aug 17;130(7):900-2.Full text Abstract
180. Schwotzer R, Manz MG, Pederiva S, et al. Daratumumab for relapsed or refractory AL amyloidosis with high plasma cell burden. Hematol Oncol. 2019 Dec;37(5):595-600.Full text Abstract
181. Sanchorawala V, Sarosiek S, Schulman A, et al. Safety, tolerability, and response rates of daratumumab in relapsed AL amyloidosis: results of a phase 2 study. Blood. 2020 Apr 30;135(18):1541-1547.Full text Abstract
182. Kimmich CR, Terzer T, Benner A, et al. Daratumumab for systemic AL amyloidosis: prognostic factors and adverse outcome with nephrotic-range albuminuria. Blood. 2020 Apr 30;135(18):1517-1530.Full text Abstract
183. Lecumberri R, Krsnik I, Askari E, et al. Treatment with daratumumab in patients with relapsed/refractory AL amyloidosis: a multicentric retrospective study and review of the literature. Amyloid. 2020 Sep;27(3):163-167.Full text Abstract
184. Kastritis E, Roussakis P, Kostopoulos IV, et al. Short daratumumab consolidation in patients with AL amyloidosis or LCCD improves complete response rates and modifies bone marrow microenvironment. Blood. 2020;136(supp 1):25.Full text
185. Solomon A, Weiss DT, Wall JS. Therapeutic potential of chimeric amyloid-reactive monoclonal antibody 11-1F4. Clin Cancer Res. 2003 Sep 1;9(10 Pt 2):s3831-8.Full text Abstract
186. Solomon A, Weiss DT, Wall JS. Immunotherapy in systemic primary (AL) amyloidosis using amyloid-reactive monoclonal antibodies. Cancer Biother Radiopharm. 2003 Dec;18(6):853-60. Abstract
187. Edwards CV, Gould J, Langer AL, et al. Analysis of the phase 1a/b study of chimeric fibril-reactive monoclonal antibody 11-1F4 in patients with relapsed or refractory AL amyloidosis. Amyloid. 2017 Mar;24(sup1):58-9.Full text Abstract
188. Valent J, Silowsky J, Kurman MR, et al. CAEL-101 is well-tolerated in AL amyloidosis patients receiving concomitant cyclophosphamide-bortezomib-dexamethasone (CyborD): a phase 2 dose-finding study (NCT04304144). Blood. 2020;136 (suppl 1):26-7.Full text
189. Parker TL, Rosenthal A, Sanchorawala V, et al. A phase II study of isatuximab (SAR650984) (NSC-795145) for patients with previously treated AL amyloidosis (SWOG S1702; NCT#03499808). Blood. 2020;136(supp 1):20–21.Full text
190. ClinicalTrials.org. CARDIO-TTRansform: a study to evaluate the efficacy and safety of eplontersen (formerly known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in participants with transthyretin-mediated amyloid cardiomyopathy (ATTR CM). Mar 2023 [internet publication].Full text
191. ClinicalTrials.org. NEURO-TTRansform: a study to evaluate the efficacy and safety of eplontersen (formerly known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in participants with hereditary transthyretin-mediated amyloid polyneuropathy. Mar 2023 [internet publication].Full text
192. Gillmore JD, Gane E, Taubel J, et al. CRISPR-Cas9 in vivo gene editing for transthyretin amyloidosis. N Engl J Med. 2021 Aug 5;385(6):493-502.Full text Abstract
193. ClinicalTrials.org. Study to evaluate safety, tolerability, pharmacokinetics, and pharmacodynamics of NTLA-2001 in patients with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN) and patients with transthyretin amyloidosis-related cardiomyopathy (ATTR-CM). Dec 2022 [internet publication].Full text
194. Gertz MA, Lacy MQ, Dispenzieri A, et al. Effect of hematologic response on outcome of patients undergoing transplantation for primary amyloidosis: importance of achieving a complete response. Haematologica. 2007 Oct;92(10):1415-8. Abstract
195. Staron A, Zheng L, Doros G, et al. Marked progress in AL amyloidosis survival: a 40-year longitudinal natural history study. Blood Cancer J. 2021 Aug 4;11(8):139.Full text Abstract
196. Dittrich T, Benner A, Kimmich C, et al. Performance analysis of AL amyloidosis cardiac biomarker staging systems with special focus on renal failure and atrial arrhythmia. Haematologica. 2019 Jul;104(7):1451-9.Full text Abstract
197. Muchtar E, Therneau TM, Larson DR, et al. Comparative analysis of staging systems in AL amyloidosis. Leukemia. 2019 Mar;33(3):811-814.Full text Abstract
Use of this content is subject to our disclaimer