Amyloidosis - Symptoms, Causes, Images, and Treatment Options

Diseases

Highlights & Basics

Key Highlights

Amyloidosis is a rare disease. Immunoglobulin light chain (AL) amyloidosis (also called primary systemic amyloidosis) is the most common type of amyloidosis.
Amyloidosis occurs when amyloid proteins are deposited in tissue and organs. It may have a primary cause, may be inherited, or may be secondary to other diseases.
Amyloidosis usually presents with unexplained weight loss, fatigue, and edema resistant to diuretic therapy.
Serum and urine immunofixation electrophoresis confirms the presence of monoclonal light chains in AL amyloidosis. Biopsy verification of amyloid deposits is essential for diagnosis. Accurate classification of amyloid deposit in tissues is necessary prior to initiating appropriate therapy.
Resulting clinical syndromes of amyloidosis include nephrotic syndrome, neuropathy, cardiomyopathy, and conduction abnormalities.

Classic periorbital purpura

Morie A. Gertz, MD; courtesy of Mayo Clinic

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History & Exam
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Definition

Epidemiology

Etiology

Pathophysiology

Images

Bilateral periorbital ecchymosis (amyloid purpura) in a patient with AL amyloidosis
Classic periorbital purpura
Macroglossia in a patient with AL amyloidosis
Congo red stain blood vessel in a bone marrow biopsy demonstrating green birefringence pathognomonic of amyloidosis
Electron micrograph demonstrating classical amyloid fibrils
Renal biopsy demonstrating amyloid deposits as amorphous replacement of the glomerular architecture

Citations

Key Articles

National Comprehensive Cancer Network. NCCN clinical practice guidelines in oncology: systemic light chain amyloidosis [internet publication].[Full Text]
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.[Abstract][Full Text]
Wechalekar AD, Cibeira MT, Gibbs SD, et al. Guidelines for non-transplant chemotherapy for treatment of systemic AL amyloidosis: EHA-ISA working group. Amyloid. 2023 Mar;30(1):3-17.[Abstract][Full Text]
Sanchorawala V, Boccadoro M, Gertz M, et al. Guidelines for high dose chemotherapy and stem cell transplantation for systemic AL amyloidosis: EHA-ISA working group guidelines. Amyloid. 2022 Mar;29(1):1-7.[Abstract]
Muchtar E, Dispenzieri A, Gertz MA, et al. Treatment of AL amyloidosis: Mayo stratification of myeloma and risk-adapted Ttherapy (mSMART) consensus statement 2020 update. Mayo Clin Proc. 2021 Jun;96(6):1546-77.[Abstract][Full Text]

Referenced Articles

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35. Pozzi C, Locatelli F. Kidney and liver involvement in monoclonal light chain disorders. Semin Nephrol. 2002 Jul;22(4):319-30.[Abstract]
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37. 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]
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56. Benson MD, James S, Scott K, et al. Leukocyte chemotactic factor 2: a novel renal amyloid protein. Kidney Int. 2008 Jul;74(2):218-22.[Abstract][Full Text]
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76. 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]
77. 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.[Abstract][Full Text]
78. Muchtar E, Dispenzieri A, Lacy MQ, et al. Overuse of organ biopsies in immunoglobulin light chain amyloidosis (AL): the consequence of failure of early recognition. Ann Med. 2017 Nov;49(7):545-551.[Abstract][Full Text]
79. Gertz MA. The classification and typing of amyloid deposits. Am J Clin Pathol. 2004 Jun;121(6):787-9.[Abstract]
80. Gertz MA, Dispenzieri A, Muchtar E. Importance of FISH genetics in light chain amyloidosis. Oncotarget. 2017 Oct 10;8(47):81735-6.[Full Text]
81. Muchtar E, Dispenzieri A, Kumar SK, et al. Interphase fluorescence in situ hybridization in untreated AL amyloidosis has an independent prognostic impact by abnormality type and treatment category. Leukemia. 2017 Jul;31(7):1562-9.[Abstract]
82. 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.[Abstract][Full Text]
83. 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.[Abstract][Full Text]
84. Tuzovic M, Yang EH, Baas AS, et al. Cardiac amyloidosis: diagnosis and treatment strategies. Curr Oncol Rep. 2017 Jul;19(7):46.[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 1 of 2-evidence base and standardized methods of imaging. Circ Cardiovasc Imaging. 2021 Jul;14(7):e000029. [Abstract][Full Text]
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88. 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]
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96. Al Saleh AS, Sidiqi MH, Muchtar E, et al. Prognostic role of beta-2 microglobulin in patients with light chain amyloidosis treated with autologous stem cell transplantation. Biol Blood Marrow Transplant. 2020 Aug;26(8):1402-1405.[Abstract][Full Text]
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98. Choufani EB, Sanchorawala V, Ernst T, et al. Acquired factor X deficiency in patients with amyloid light-chain amyloidosis: incidence, bleeding manifestations, and response to high-dose chemotherapy. Blood. 2001 Mar 15;97(6):1885-7.[Abstract][Full Text]
99. 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.[Abstract][Full Text]
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102. Hoscheit M, Kamal A, Cline M. Gastroparesis in a patient with sastric AL amyloidosis. Case Rep Gastroenterol. 2018 May-Aug;12(2):317-21.[Abstract][Full Text]
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104. Muchtar E, Kumar SK, Gertz MA, et al. Staging systems use for risk stratification of systemic amyloidosis in the era of high-sensitivity troponin T assay. Blood. 2019 Feb 14;133(7):763-6.[Abstract][Full Text]
105. 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.[Abstract][Full Text]
106. 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]
107. 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.[Abstract][Full Text]
108. Adams D, Ando Y, Beirão JM, et al. Expert consensus recommendations to improve diagnosis of ATTR amyloidosis with polyneuropathy. J Neurol. 2021 Jun;268(6):2109-22.[Abstract][Full Text]
109. Maurer MS, Bokhari S, Damy T, et al. Expert consensus recommendations for the suspicion and diagnosis of transthyretin cardiac amyloidosis. Circ Heart Fail. 2019 Sep;12(9):e006075.[Abstract][Full Text]
110. 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.[Abstract][Full Text]
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112. Wechalekar AD, Cibeira MT, Gibbs SD, et al. Guidelines for non-transplant chemotherapy for treatment of systemic AL amyloidosis: EHA-ISA working group. Amyloid. 2023 Mar;30(1):3-17.[Abstract][Full Text]
113. Sanchorawala V, Boccadoro M, Gertz M, et al. Guidelines for high dose chemotherapy and stem cell transplantation for systemic AL amyloidosis: EHA-ISA working group guidelines. Amyloid. 2022 Mar;29(1):1-7.[Abstract]
114. Muchtar E, Dispenzieri A, Gertz MA, et al. Treatment of AL amyloidosis: Mayo stratification of myeloma and risk-adapted Ttherapy (mSMART) consensus statement 2020 update. Mayo Clin Proc. 2021 Jun;96(6):1546-77.[Abstract][Full Text]
115. mSMART. Mayo consensus on AL amyloidosis: diagnosis, treatment and prognosis. Apr 2023 [internet publication].[Full Text]
116. 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.[Abstract][Full Text]
117. 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.[Abstract][Full Text]
118. 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.[Abstract][Full Text]
119. 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]
120. 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.[Abstract][Full Text]
121. 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]
122. 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]
123. Chakraborty R, Milani P, Palladini G, et al. Role of autologous haematopoietic cell transplantation in the treatment of systemic light chain amyloidosis in the era of anti-CD38 monoclonal antibodies. Lancet Haematol. 2023 Nov;10(11):e936-40.[Abstract][Full Text]
124. Gertz MA. Immunoglobulin light chain amyloidosis: 2024 update on diagnosis, prognosis, and treatment. Am J Hematol. 2024 Feb;99(2):309-24.[Abstract][Full Text]
125. Sharpley FA, Petrie A, Mahmood S, et al. A 24-year experience of autologous stem cell transplantation for light chain amyloidosis patients in the United Kingdom. Br J Haematol. 2019 Dec;187(5):642-52.[Abstract]
126. 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]
127. National Comprehensive Cancer Network. NCCN clinical practice guidelines in oncology: hematopoietic cell transplantation (HCT) [internet publication].[Full Text]
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