Systemic Amyloidosis Recognition, Prognosis, and Therapy | Cardiology | JN Learning | AMA Ed Hub [Skip to Content]
[Skip to Content Landing]

Systemic Amyloidosis Recognition, Prognosis, and TherapyA Systematic Review

Educational Objective
To review the diagnosis and management of systemic amyloidosis.
1 Credit CME
Key Points

Question  When should clinicians suspect and how should they diagnose systemic amyloidosis?

Findings  Systemic amyloidosis should be suspected in patients with nondiabetic proteinuria, heart failure with preserved ejection fraction, unexplained peripheral neuropathy, or atypical monoclonal gammopathy of undetermined significance.

Meaning  Late diagnosis of amyloidosis is a barrier to improved outcomes. Early recognition by primary care clinicians is vital for effective therapy to have a meaningful effect on survival.


Importance  Many patients with systemic amyloidosis are underdiagnosed. Overall, 25% of patients with immunoglobulin light chain (AL) amyloidosis die within 6 months of diagnosis and 25% of patients with amyloid transthyretin (ATTR) amyloidosis die within 24 months of diagnosis. Effective therapy exists but is ineffective if end-organ damage is severe.

Objective  To provide evidence-based recommendations that could allow clinicians to diagnose this rare set of diseases earlier and enable accurate staging and counseling about prognosis.

Evidence Review  A comprehensive literature search was conducted by a reference librarian with publication dates from January 1, 2000, to December 31, 2019. Key search terms included amyloid, amyloidosis, nephrotic syndrome, heart failure preserved ejection fraction, and peripheral neuropathy. Exclusion criteria included case reports, non–English-language text, and case series of fewer than 10 patients. The authors independently selected and appraised relevant literature.

Findings  There was a total of 1769 studies in the final data set. Eighty-one articles were included in this review, of which 12 were randomized clinical trials of therapy that included 3074 patients, 9 were case series, and 3 were cohort studies. The incidence of AL amyloidosis is approximately 12 cases per million persons per year and there is an estimated prevalence of 30 000 to 45 000 cases in the US and European Union. The incidence of variant ATTR amyloidosis is estimated to be 0.3 cases per year per million persons with a prevalence estimate of 5.2 cases per million persons. Wild-type ATTR is estimated to have a prevalence of 155 to 191 cases per million persons. Amyloidosis should be considered in the differential diagnosis of adult nondiabetic nephrotic syndrome; heart failure with preserved ejection fraction, particularly if restrictive features are present; unexplained hepatomegaly without imaging abnormalities; peripheral neuropathy with distal sensory symptoms, such as numbness, paresthesia, and dysesthesias (although the autonomic manifestations occasionally may be the presenting feature); and monoclonal gammopathy of undetermined significance with atypical clinical features. Staging can be performed using blood testing only. Therapeutic decision-making for AL amyloidosis involves choosing between high-dose chemotherapy and stem cell transplant or bortezomib-based chemotherapy. There are 3 therapies approved by the US Food and Drug Administration for managing ATTR amyloidosis, depending on clinical phenotype.

Conclusions and Relevance  All forms of amyloidosis are underdiagnosed. All forms now have approved therapies that have been demonstrated to improve either survival or disability and quality of life. The diagnosis should be considered in patients that have a multisystem disorder involving the heart, kidney, liver, or nervous system.

Sign in to take quiz and track your certificates

Buy This Activity

JN Learning™ is the home for CME and MOC from the JAMA Network. Search by specialty or US state and earn AMA PRA Category 1 CME Credit™ from articles, audio, Clinical Challenges and more. Learn more about CME/MOC

Article Information

Accepted for Publication: March 30, 2020.

Corresponding Author: Morie A. Gertz, MD, Division of Hematology, Department of Medicine, Mayo Clinic, 200 First St SW, Mayo West 10, Rochester, MN 55905 (

Author Contributions: Drs Gertz and Dispenzieri had full access to all of the data in the study and take responsibility for the integrity of the data and the accuracy of the data analysis.

Concept and design: All authors.

Acquisition, analysis, or interpretation of data: All authors.

Drafting of the manuscript: All authors.

Critical revision of the manuscript for important intellectual content: All authors.

Statistical analysis: Dispenzieri.

Administrative, technical, or material support: All authors.

Supervision: Gertz.

Conflict of Interest Disclosures: Dr Gertz reported receiving personal fees from Akccea, Alnylym, Prothena, Janssen, Spectrum, Apellis, Amgen, Physicans Education Resource, Research to Practice, Johnson and Johnson, Proclara, and i3Health outside the submitted work and serving on data and safety monitoring boards for AbbVie and Celgene and receiving royalties from Springer Publishing, grant funding to the institution from the Amyloidosis Foundation and the Macroglobulinemia Foundation (NCI SPORE MM SPORE 5P50 CA186781-04), and personal fees from Sanofi. Dr Dispenzieri reported receiving grants from Celgene, Takeda, Alnylam, Prothena, and Pfizer and personal fees from Intellia and Akcea outside the submitted work.

Additional Contributions: We would like to recognize Patricia Erwin, MLS (Mayo Alix School of Medicine), for expert search services. Ms Erwin was not compensated for her contribution.

Ueda  M , Horibata  Y , Shono  M ,  et al.  Clinicopathological features of senile systemic amyloidosis: an ante- and post-mortem study.   Mod Pathol. 2011;24(12):1533-1544. doi:10.1038/modpathol.2011.117PubMedGoogle ScholarCrossref
Tanskanen  M , Peuralinna  T , Polvikoski  T ,  et al.  Senile systemic amyloidosis affects 25% of the very aged and associates with genetic variation in alpha2-macroglobulin and tau: a population-based autopsy study.   Ann Med. 2008;40(3):232-239. doi:10.1080/07853890701842988PubMedGoogle ScholarCrossref
Kyle  RA , Larson  DR , Kurtin  PJ ,  et al.  Incidence of AL amyloidosis in Olmsted County, Minnesota, 1990 through 2015.   Mayo Clin Proc. 2019;94(3):465-471. doi:10.1016/j.mayocp.2018.08.041PubMedGoogle ScholarCrossref
Lin  HM , Gao  X , Cooke  CE ,  et al.  Disease burden of systemic light-chain amyloidosis: a systematic literature review.   Curr Med Res Opin. 2017;33(6):1017-1031. doi:10.1080/03007995.2017.1297930PubMedGoogle ScholarCrossref
Benson  MD , Buxbaum  JN , Eisenberg  DS ,  et al.  Amyloid nomenclature 2018: recommendations by the International Society of Amyloidosis (ISA) nomenclature committee.   Amyloid. 2018;25(4):215-219. doi:10.1080/13506129.2018.1549825PubMedGoogle ScholarCrossref
Buxbaum  JN , Ruberg  FL .  Transthyretin V122I (pV142I)* cardiac amyloidosis: an age-dependent autosomal dominant cardiomyopathy too common to be overlooked as a cause of significant heart disease in elderly African Americans.   Genet Med. 2017;19(7):733-742. doi:10.1038/gim.2016.200PubMedGoogle ScholarCrossref
Merlini  G , Dispenzieri  A , Sanchorawala  V ,  et al.  Systemic immunoglobulin light chain amyloidosis.   Nat Rev Dis Primers. 2018;4(1):38. doi:10.1038/s41572-018-0034-3PubMedGoogle ScholarCrossref
Siddiqi  OK , Ruberg  FL .  Cardiac amyloidosis: an update on pathophysiology, diagnosis, and treatment.   Trends Cardiovasc Med. 2018;28(1):10-21. doi:10.1016/j.tcm.2017.07.004PubMedGoogle ScholarCrossref
Suhr  OB , Gustavsson  S , Heldestad  V ,  et al.  New insights into the clinical evaluation of hereditary transthyretin amyloidosis patients: a single center’s experience.   Degener Neurol Neuromuscul Dis. 2012;2:93-106. doi:10.2147/DNND.S24652PubMedGoogle Scholar
Sattianayagam  PT , Hahn  AF , Whelan  CJ ,  et al.  Cardiac phenotype and clinical outcome of familial amyloid polyneuropathy associated with transthyretin alanine 60 variant.   Eur Heart J. 2012;33(9):1120-1127. doi:10.1093/eurheartj/ehr383PubMedGoogle ScholarCrossref
Di Girolamo  M , Monno  D , Pirro  MR , Nowakowski  M .  Approach to diagnosis in systemic amyloidosis: initial findings and time from symptoms onset to diagnosis (light-chain amyloidosis vs. transthyretin): problems and observations.   Amyloid. 2011;18(suppl 1):83-85. doi:10.3109/13506129.2011.574354030PubMedGoogle ScholarCrossref
Lousada  I , Comenzo  RL , Landau  H , Guthrie  S , Merlini  G .  Light chain amyloidosis: patient experience survey from the amyloidosis research consortium.   Adv Ther. 2015;32(10):920-928. doi:10.1007/s12325-015-0250-0PubMedGoogle ScholarCrossref
Carr  AS , Shah  S , Choi  D ,  et al.  Spinal stenosis in familial transthyretin amyloidosis.   J Neuromuscul Dis. 2019;6(2):267-270. doi:10.3233/JND-180348PubMedGoogle ScholarCrossref
Barge-Caballero  G , López-Bargiela  P , Pombo-Otero  J , Pardo-Martínez  P .  Quadriceps tendon rupture in wild-type transthyretin amyloidosis (ATTRwt).   Eur Heart J. 2019;40(16):1307. doi:10.1093/eurheartj/ehz128PubMedGoogle ScholarCrossref
Cortese  A , Vegezzi  E , Lozza  A ,  et al.  Diagnostic challenges in hereditary transthyretin amyloidosis with polyneuropathy: avoiding misdiagnosis of a treatable hereditary neuropathy.   J Neurol Neurosurg Psychiatry. 2017;88(5):457-458. doi:10.1136/jnnp-2016-315262PubMedGoogle ScholarCrossref
Engineer  DP , Kute  VB , Patel  HV , Shah  PR .  Clinical and laboratory profile of renal amyloidosis: a single-center experience.   Saudi J Kidney Dis Transpl. 2018;29(5):1065-1072. doi:10.4103/1319-2442.243966PubMedGoogle ScholarCrossref
Ritts  AJ , Cornell  RF , Swiger  K , Singh  J , Goodman  S , Lenihan  DJ .  Current concepts of cardiac amyloidosis: diagnosis, clinical management, and the need for collaboration.   Heart Fail Clin. 2017;13(2):409-416. doi:10.1016/j.hfc.2016.12.003PubMedGoogle ScholarCrossref
Mohammed  SF , Mirzoyev  SA , Edwards  WD ,  et al.  Left ventricular amyloid deposition in patients with heart failure and preserved ejection fraction.   JACC Heart Fail. 2014;2(2):113-122. doi:10.1016/j.jchf.2013.11.004PubMedGoogle ScholarCrossref
Di Nunzio  D , Recupero  A , de Gregorio  C , Zito  C , Carerj  S , Di Bella  G .  Echocardiographic findings in cardiac amyloidosis: inside two-dimensional, doppler, and strain imaging.   Curr Cardiol Rep. 2019;21(2):7. doi:10.1007/s11886-019-1094-zPubMedGoogle ScholarCrossref
Cappelli  F , Baldasseroni  S , Bergesio  F ,  et al.  Echocardiographic and biohumoral characteristics in patients with AL and TTR amyloidosis at diagnosis.   Clin Cardiol. 2015;38(2):69-75. doi:10.1002/clc.22353PubMedGoogle ScholarCrossref
Oda  S , Utsunomiya  D , Nakaura  T ,  et al.  Identification and assessment of cardiac amyloidosis by myocardial strain analysis of cardiac magnetic resonance imaging.   Circ J. 2017;81(7):1014-1021. doi:10.1253/circj.CJ-16-1259PubMedGoogle ScholarCrossref
Finsterer  J , Iglseder  S , Wanschitz  J , Topakian  R , Löscher  WN , Grisold  W .  Hereditary transthyretin-related amyloidosis.   Acta Neurol Scand. 2019;139(2):92-105. doi:10.1111/ane.13035PubMedGoogle ScholarCrossref
Sperry  BW , Reyes  BA , Ikram  A ,  et al.  Tenosynovial and cardiac amyloidosis in patients undergoing carpal tunnel release.   J Am Coll Cardiol. 2018;72(17):2040-2050. doi:10.1016/j.jacc.2018.07.092PubMedGoogle ScholarCrossref
Thompson  CA , Kyle  R , Gertz  M , Heit  J , Pruthi  R , Pardanani  A .  Systemic AL amyloidosis with acquired factor X deficiency: a study of perioperative bleeding risk and treatment outcomes in 60 patients.   Am J Hematol. 2010;85(3):171-173. doi:10.1002/ajh.21603PubMedGoogle Scholar
Muchtar  E , Gertz  MA , Kyle  RA ,  et al.  A modern primer on light chain amyloidosis in 592 patients with mass spectrometry-verified typing.   Mayo Clin Proc. 2019;94(3):472-483. doi:10.1016/j.mayocp.2018.08.006PubMedGoogle Scholar
Katzmann  JA , Kyle  RA , Benson  J ,  et al.  Screening panels for detection of monoclonal gammopathies.   Clin Chem. 2009;55(8):1517-1522. doi:10.1373/clinchem.2009.126664PubMedGoogle ScholarCrossref
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;32(9):1883-1898. doi:10.1038/s41375-018-0209-7PubMedGoogle ScholarCrossref
Quarta  CC , Gonzalez-Lopez  E , Gilbertson  JA ,  et al.  Diagnostic sensitivity of abdominal fat aspiration in cardiac amyloidosis.   Eur Heart J. 2017;38(24):1905-1908. doi:10.1093/eurheartj/ehx047PubMedGoogle ScholarCrossref
Kimmich  C , Schönland  S , Kräker  S ,  et al.  Amyloid in bone marrow smears in systemic light-chain amyloidosis.   Amyloid. 2017;24(1):52-59. doi:10.1080/13506129.2017.1314959PubMedGoogle ScholarCrossref
Mollee  P , Renaut  P , Gottlieb  D , Goodman  H .  How to diagnose amyloidosis.   Intern Med J. 2014;44(1):7-17. doi:10.1111/imj.12288PubMedGoogle ScholarCrossref
Gillmore  JD , Maurer  MS , Falk  RH ,  et al.  Nonbiopsy diagnosis of cardiac transthyretin amyloidosis.   Circulation. 2016;133(24):2404-2412. doi:10.1161/CIRCULATIONAHA.116.021612PubMedGoogle ScholarCrossref
Vrana  JA , Gamez  JD , Madden  BJ , Theis  JD , Bergen  HR  III , Dogan  A .  Classification of amyloidosis by laser microdissection and mass spectrometry-based proteomic analysis in clinical biopsy specimens.   Blood. 2009;114(24):4957-4959. doi:10.1182/blood-2009-07-230722PubMedGoogle ScholarCrossref
Gilbertson  JA , Theis  JD , Vrana  JA ,  et al.  A comparison of immunohistochemistry and mass spectrometry for determining the amyloid fibril protein from formalin-fixed biopsy tissue.   J Clin Pathol. 2015;68(4):314-317. doi:10.1136/jclinpath-2014-202722PubMedGoogle ScholarCrossref
Kourelis  TV , Kyle  RA , Dingli  D ,  et al.  Presentation and outcomes of localized immunoglobulin light chain amyloidosis: the Mayo Clinic experience.   Mayo Clin Proc. 2017;92(6):908-917. doi:10.1016/j.mayocp.2017.02.016PubMedGoogle ScholarCrossref
Geller  HI , Singh  A , Mirto  TM ,  et al.  Prevalence of monoclonal gammopathy in wild-type transthyretin amyloidosis.   Mayo Clin Proc. 2017;92(12):1800-1805. doi:10.1016/j.mayocp.2017.09.016PubMedGoogle ScholarCrossref
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;104(7):1451-1459. doi:10.3324/haematol.2018.205336PubMedGoogle ScholarCrossref
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;22(18):3751-3757. doi:10.1200/JCO.2004.03.029PubMedGoogle ScholarCrossref
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;30(9):989-995. doi:10.1200/JCO.2011.38.5724PubMedGoogle ScholarCrossref
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;126(5):612-615. doi:10.1182/blood-2015-01-620302PubMedGoogle ScholarCrossref
Kyriakou  P , Mouselimis  D , Tsarouchas  A ,  et al.  Diagnosis of cardiac amyloidosis: a systematic review on the role of imaging and biomarkers.   BMC Cardiovasc Disord. 2018;18(1):221. doi:10.1186/s12872-018-0952-8PubMedGoogle ScholarCrossref
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;68(10):1014-1020. doi:10.1016/j.jacc.2016.06.033PubMedGoogle ScholarCrossref
Gillmore  JD , Damy  T , Fontana  M ,  et al.  A new staging system for cardiac transthyretin amyloidosis.   Eur Heart J. 2018;39(30):2799-2806. doi:10.1093/eurheartj/ehx589PubMedGoogle ScholarCrossref
Jaccard  A , Moreau  P , Leblond  V ,  et al; Myélome Autogreffe (MAG) and Intergroupe Francophone du Myélome (IFM) Intergroup.  High-dose melphalan versus melphalan plus dexamethasone for AL amyloidosis.   N Engl J Med. 2007;357(11):1083-1093. doi:10.1056/NEJMoa070484PubMedGoogle ScholarCrossref
Sidiqi  MH , Aljama  MA , Buadi  FK ,  et al.  Stem cell transplantation for light chain amyloidosis: decreased early mortality over time.   J Clin Oncol. 2018;36(13):1323-1329. doi:10.1200/JCO.2017.76.9554PubMedGoogle ScholarCrossref
Varga  C , Comenzo  RL .  High-dose melphalan and stem cell transplantation in systemic AL amyloidosis in the era of novel anti-plasma cell therapy: a comprehensive review.   Bone Marrow Transplant. 2019;54(4):508-518. doi:10.1038/s41409-018-0284-4PubMedGoogle ScholarCrossref
Cibeira  MT , Bladé  J .  Upfront CyBorD in AL amyloidosis.   Blood. 2015;126(5):564-566. doi:10.1182/blood-2015-06-648113PubMedGoogle ScholarCrossref
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;99(4):743-750. doi:10.3324/haematol.2013.095463PubMedGoogle ScholarCrossref
Kastritis  E , Leleu  X , Arnulf  B ,  et al.  A randomized phase III trial of melphalan and dexamethasone (MDex) versus bortezomib, melphalan and dexamethasone (BMDex) for untreated patients with AL amyloidosis.   Blood. 2016;128(22):646-646. doi:10.1182/blood.V128.22.646.646Google ScholarCrossref
Palladini  G , Jaccard  A , Milani  P ,  et al.  Circulating free light chain measurement in the diagnosis, prognostic assessment and evaluation of response of AL amyloidosis: comparison of Freelite and N latex FLC assays.   Clin Chem Lab Med. 2017;55(11):1734-1743. doi:10.1515/cclm-2016-1024PubMedGoogle ScholarCrossref
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;126(5):612-615. doi:10.1182/blood-2015-01-620302PubMedGoogle ScholarCrossref
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;49(7):545-551. doi:10.1080/07853890.2017.1304649PubMedGoogle ScholarCrossref
Dispenzieri  A , Kastritis  E , Wechalekar  AD ,  et al  Primary results from the phase 3 tourmaline-al1 trial of ixazomib-dexamethasone versus physician's choice of therapy in patients (Pts) with relapsed/refractory primary systemic al amyloidosis (RRAL).   Blood. 2019;134(supplement 1):139-139. doi:10.1182/blood-2019-124409Google ScholarCrossref
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;129(15):2120-2123. doi:10.1182/blood-2016-12-756528PubMedGoogle ScholarCrossref
Bochtler  T , Hegenbart  U , Kunz  C ,  et al.  Translocation t(11;14) is associated with adverse outcome in patients with newly diagnosed AL amyloidosis when treated with bortezomib-based regimens.   J Clin Oncol. 2015;33(12):1371-1378. doi:10.1200/JCO.2014.57.4947PubMedGoogle ScholarCrossref
Bochtler  T , Hegenbart  U , Kunz  C ,  et al.  Prognostic impact of cytogenetic aberrations in AL amyloidosis patients after high-dose melphalan: a long-term follow-up study.   Blood. 2016;128(4):594-602. doi:10.1182/blood-2015-10-676361PubMedGoogle ScholarCrossref
Trial of venetoclax (ABT-199) and dexamethasone for relapsed or refractory systemic AL amyloidosis. identifier: NCT03000660. Updated May 3, 2019. Accessed June 9, 2020.
Popat  R , Dowling  E , Achhala  S ,  et al.  Real world data of the impact of first cycle daratumumab on multiple myeloma and AL amyloidosis services.   Br J Haematol. 2018;182(6):936-939. doi:10.1111/bjh.14897PubMedGoogle ScholarCrossref
Kaufman  GP , Schrier  SL , Lafayette  RA , Arai  S , Witteles  RM , Liedtke  M .  Daratumumab yields rapid and deep hematologic responses in patients with heavily pretreated AL amyloidosis.   Blood. 2017;130(7):900-902. doi:10.1182/blood-2017-01-763599PubMedGoogle ScholarCrossref
A study to evaluate the efficacy and safety of daratumumab in combination with cyclophosphamide, bortezomib and dexamethasone (CyBorD) compared to cybord alone in newly diagnosed systemic amyloid light-chain (AL) amyloidosis. identifier: NCT03201965. Updated May 1, 2020. Accessed June 9, 2020.
Comenzo  R , Kastritis  E , Maurer  MS ,  et al. Subcutaneous daratumumab+cyclophosphamide, bortezomib and dexamethasone (CYBORD) in patients with newly diagnosed amyloid light chain (AL) amyloidosis. Abstract presented at: European Hematology Association Meeting; June 15, 2019; Amsterdam, the Netherlands.
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;30(36):4541-4549. doi:10.1200/JCO.2011.37.7614PubMedGoogle ScholarCrossref
Milani  P , Basset  M , Russo  F , Foli  A , Merlini  G , Palladini  G .  Patients with light-chain amyloidosis and low free light-chain burden have distinct clinical features and outcome.   Blood. 2017;130(5):625-631. doi:10.1182/blood-2017-02-767467PubMedGoogle ScholarCrossref
Sachchithanantham  S , Roussel  M , Palladini  G ,  et al.  European collaborative study defining clinical profile outcomes and novel prognostic criteria in monoclonal immunoglobulin m-related light chain amyloidosis.   J Clin Oncol. 2016;34(17):2037-2045. doi:10.1200/JCO.2015.63.3123PubMedGoogle ScholarCrossref
Muchtar  E , Dispenzieri  A , Leung  N ,  et al.  Depth of organ response in AL amyloidosis is associated with improved survival: grading the organ response criteria.   Leukemia. 2018;32(10):2240-2249. doi:10.1038/s41375-018-0060-xPubMedGoogle ScholarCrossref
Dyck  PJB , González-Duarte  A , Obici  L ,  et al.  Development of measures of polyneuropathy impairment in hATTR amyloidosis: from NIS to mNIS + 7.   J Neurol Sci. 2019;405:116424. doi:10.1016/j.jns.2019.116424PubMedGoogle Scholar
Varga  C , Lentzsch  S , Comenzo  RL .  Beyond NEOD001 for systemic light-chain amyloidosis.   Blood. 2018;132(18):1992-1993. doi:10.1182/blood-2018-07-865857PubMedGoogle ScholarCrossref
Monteiro  C , Mesgazardeh  JS , Anselmo  J ,  et al.  Predictive model of response to tafamidis in hereditary ATTR polyneuropathy.   JCI Insight. 2019;4(12):126526. doi:10.1172/jci.insight.126526PubMedGoogle Scholar
Maurer  MS , Schwartz  JH , Gundapaneni  B ,  et al; ATTR-ACT Study Investigators.  Tafamidis treatment for patients with transthyretin amyloid cardiomyopathy.   N Engl J Med. 2018;379(11):1007-1016. doi:10.1056/NEJMoa1805689PubMedGoogle ScholarCrossref
Judge  DP , Heitner  SB , Falk  RH ,  et al.  Transthyretin stabilization by AG10 in symptomatic transthyretin amyloid cardiomyopathy.   J Am Coll Cardiol. 2019;74(3):285-295. doi:10.1016/j.jacc.2019.03.012PubMedGoogle ScholarCrossref
Carvalho  A , Rocha  A , Lobato  L .  Liver transplantation in transthyretin amyloidosis: issues and challenges.   Liver Transpl. 2015;21(3):282-292. doi:10.1002/lt.24058PubMedGoogle ScholarCrossref
Berk  JL , Suhr  OB , Obici  L ,  et al; Diflunisal Trial Consortium.  Repurposing diflunisal for familial amyloid polyneuropathy: a randomized clinical trial.   JAMA. 2013;310(24):2658-2667. doi:10.1001/jama.2013.283815PubMedGoogle ScholarCrossref
Benson  MD , Waddington-Cruz  M , Berk  JL ,  et al.  Inotersen treatment for patients with hereditary transthyretin amyloidosis.   N Engl J Med. 2018;379(1):22-31. doi:10.1056/NEJMoa1716793PubMedGoogle ScholarCrossref
Adams  D , Gonzalez-Duarte  A , O’Riordan  WD ,  et al.  Patisiran, an RNAi therapeutic, for hereditary transthyretin amyloidosis.   N Engl J Med. 2018;379(1):11-21. doi:10.1056/NEJMoa1716153PubMedGoogle ScholarCrossref
Merlini  G .  AL amyloidosis: from molecular mechanisms to targeted therapies.   Hematology Am Soc Hematol Educ Program. 2017;2017(1):1-12. doi:10.1182/asheducation-2017.1.1PubMedGoogle ScholarCrossref
Suzuki  T , Kusumoto  S , Yamashita  T ,  et al.  Labial salivary gland biopsy for diagnosing immunoglobulin light chain amyloidosis: a retrospective analysis.   Ann Hematol. 2016;95(2):279-285. doi:10.1007/s00277-015-2549-yPubMedGoogle ScholarCrossref
Moore  PT , Burrage  MK , Mackenzie  E , Law  WP , Korczyk  D , Mollee  P .  The utility of 99mTc-DPD scintigraphy in the diagnosis of cardiac amyloidosis: an Australian experience.   Heart Lung Circ. 2017;26(11):1183-1190. doi:10.1016/j.hlc.2016.12.017PubMedGoogle ScholarCrossref
Adams  D , Cauquil  C , Labeyrie  C .  Familial amyloid polyneuropathy.   Curr Opin Neurol. 2017;30(5):481-489. doi:10.1097/WCO.0000000000000476PubMedGoogle ScholarCrossref
Kristen  AV , Dengler  TJ , Katus  HA .  Suspected cardiac amyloidosis: endomyocardial biopsy remains the diagnostic gold-standard.   Am J Hematol. 2007;82(4):328. doi:10.1002/ajh.20745PubMedGoogle ScholarCrossref
Obici  L , Kuks  JB , Buades  J ,  et al; European Network for TTR-FAP (ATTReuNET).  Recommendations for presymptomatic genetic testing and management of individuals at risk for hereditary transthyretin amyloidosis.   Curr Opin Neurol. 2016;29(suppl 1):S27-S35. doi:10.1097/WCO.0000000000000290PubMedGoogle ScholarCrossref
Wechalekar  AD , Gillmore  JD , Hawkins  PN .  Systemic amyloidosis.   Lancet. 2016;387(10038):2641-2654. doi:10.1016/S0140-6736(15)01274-XPubMedGoogle ScholarCrossref
Hwa  YL , Kumar  SK , Gertz  MA ,  et al.  Induction therapy pre-autologous stem cell transplantation in immunoglobulin light chain amyloidosis: a retrospective evaluation.   Am J Hematol. 2016;91(10):984-988. doi:10.1002/ajh.24453PubMedGoogle ScholarCrossref
Shen  KN , Zhang  CL , Tian  Z ,  et al.  Bortezomib-based chemotherapy reduces early mortality and improves outcomes in patients with ultra-high-risk light-chain amyloidosis: a retrospective case control study.   Amyloid. 2019;26(2):66-73. doi:10.1080/13506129.2019.1594759PubMedGoogle ScholarCrossref
If you are not a JN Learning subscriber, you can either:
Subscribe to JN Learning for one year
Buy this activity
If you are not a JN Learning subscriber, you can either:
Subscribe to JN Learning for one year
Buy this activity
With a personal account, you can:
  • Access free activities and track your credits
  • Personalize content alerts
  • Customize your interests
  • Fully personalize your learning experience
Education Center Collection Sign In Modal Right

Name Your Search

Save Search
With a personal account, you can:
  • Track your credits
  • Personalize content alerts
  • Customize your interests
  • Fully personalize your learning experience

Lookup An Activity



My Saved Searches

You currently have no searches saved.

With a personal account, you can:
  • Access free activities and track your credits
  • Personalize content alerts
  • Customize your interests
  • Fully personalize your learning experience
Education Center Collection Sign In Modal Right