Systemic amyloidosis
Tóm tắt
Từ khóa
Tài liệu tham khảo
Merlini, 2004, Systemic amyloidosis: are we moving ahead?, Neth J Med, 62, 104
Sipe, 2014, Nomenclature 2014: Amyloid fibril proteins and clinical classification of the amyloidosis, Amyloid, 21, 221, 10.3109/13506129.2014.964858
Esplin, 2013, Current trends in diagnosis and management of cardiac amyloidosis, Curr Probl Cardiol, 38, 53, 10.1016/j.cpcardiol.2012.11.002
Kyle, 1992, Incidence and natural history of primary systemic amyloidosis in Olmsted County, Minnesota, 1950 through 1989, Blood, 79, 1817, 10.1182/blood.V79.7.1817.1817
Pinney, 2013, Systemic amyloidosis in England: an epidemiological study, Br J Haematol, 161, 525, 10.1111/bjh.12286
Hemminki, 2012, Incidence and survival in non-hereditary amyloidosis in Sweden, BMC Public Health, 12, 974, 10.1186/1471-2458-12-974
Valleix, 2012, Hereditary systemic amyloidosis due to Asp76Asn variant β2-microglobulin, N Engl J Med, 366, 2276, 10.1056/NEJMoa1201356
Wechalekar, 2012, 25 years of systemic amyloidosis
Tanskanen, 2008, 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, 40, 232, 10.1080/07853890701842988
Benson, 2008, Leukocyte chemotactic factor 2: A novel renal amyloid protein, Kidney Int, 74, 218, 10.1038/ki.2008.152
Murphy, 2011, Leukocyte chemotactic factor 2 (LECT2)-associated renal amyloidosis, Amyloid, 18, 223, 10.3109/13506129.2011.574354084
Murphy, 2010, Leukocyte chemotactic factor 2 (LECT2)-associated renal amyloidosis: a case series, Am J Kidney Dis, 56, 1100, 10.1053/j.ajkd.2010.08.013
Larsen, 2010, Prevalence and morphology of leukocyte chemotactic factor 2-associated amyloid in renal biopsies, Kidney Int, 77, 816, 10.1038/ki.2010.9
Sousa, 1995, Genetic epidemiology of familial amyloidotic polyneuropathy (FAP)-type I in Póvoa do Varzim and Vila do Conde (north of Portugal), Am J Med Genet, 60, 512, 10.1002/ajmg.1320600606
Holmgren, 1994, Geographical distribution of TTR met30 carriers in northern Sweden: discrepancy between carrier frequency and prevalence rate, J Med Genet, 31, 351, 10.1136/jmg.31.5.351
Hellman, 2008, Heterogeneity of penetrance in familial amyloid polyneuropathy, ATTR Val30Met, in the Swedish population, Amyloid, 15, 181, 10.1080/13506120802193720
Reilly, 1995, Familial amyloid polyneuropathy (TTR ala 60) in North West Ireland: a clinical, genetic, and epidemiological study, J Neurol Neurosurg Psychiatry, 59, 45, 10.1136/jnnp.59.1.45
Jacobson, 1997, Variant-sequence transthyretin (isoleucine 122) in late-onset cardiac amyloidosis in black Americans, N Engl J Med, 336, 466, 10.1056/NEJM199702133360703
Sawaya, 2007, Atomic structures of amyloid cross-beta spines reveal varied steric zippers, Nature, 447, 453, 10.1038/nature05695
Bonar, 1969, Characterization of the amyloid fibril as a cross-beta protein, Proc Soc Exp Biol Med, 131, 1373, 10.3181/00379727-131-34110
Glenner, 1974, Characterization of amyloid, Annu Rev Med, 25, 131, 10.1146/annurev.me.25.020174.001023
Sunde, 1997, Common core structure of amyloid fibrils by synchrotron X-ray diffraction, J Mol Biol, 273, 729, 10.1006/jmbi.1997.1348
Pepys, 1994, Human serum amyloid P component is an invariant constituent of amyloid deposits and has a uniquely homogeneous glycostructure, Proc Natl Acad Sci USA, 91, 5602, 10.1073/pnas.91.12.5602
Dember, 2007, Eprodisate for the treatment of renal disease in AA amyloidosis, N Engl J Med, 356, 2349, 10.1056/NEJMoa065644
Bodin, 2010, Antibodies to human serum amyloid P component eliminate visceral amyloid deposits, Nature, 468, 93, 10.1038/nature09494
Falk, 2005, Diagnosis and management of the cardiac amyloidoses, Circulation, 112, 2047, 10.1161/CIRCULATIONAHA.104.489187
Merlini, 2012, CyBorD: stellar response rates in AL amyloidosis, Blood, 119, 4343, 10.1182/blood-2012-03-413112
Pinney, 2013, Senile systemic amyloidosis: clinical features at presentation and outcome, J Am Heart Assoc, 2, e000098, 10.1161/JAHA.113.000098
Lachmann, 2007, Natural history and outcome in systemic AA amyloidosis, N Engl J Med, 356, 2361, 10.1056/NEJMoa070265
Liao, 2001, Infusion of light chains from patients with cardiac amyloidosis causes diastolic dysfunction in isolated mouse hearts, Circulation, 104, 1594, 10.1161/circ.104.14.1594
Shi, 2010, Amyloidogenic light chains induce cardiomyocyte contractile dysfunction and apoptosis via a non-canonical p38alpha MAPK pathway, Proc Natl Acad Sci USA, 107, 4188, 10.1073/pnas.0912263107
Gillmore, 2009, Diagnosis, pathogenesis, treatment, and prognosis of hereditary fibrinogen A alpha-chain amyloidosis, J Am Soc Nephrol, 20, 444, 10.1681/ASN.2008060614
Sattianayagam, 2012, Hereditary lysozyme amyloidosis—phenotypic heterogeneity and the role of solid organ transplantation, J Intern Med, 272, 36, 10.1111/j.1365-2796.2011.02470.x
Reilly, 1996, Peripheral nerve amyloidosis, Brain Pathol, 6, 163, 10.1111/j.1750-3639.1996.tb00798.x
Sekijima, 2011, High prevalence of wild-type transthyretin deposition in patients with idiopathic carpal tunnel syndrome: a common cause of carpal tunnel syndrome in the elderly, Hum Pathol, 42, 1785, 10.1016/j.humpath.2011.03.004
Gertz, 2005, 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, 79, 319, 10.1002/ajh.20381
Dispenzieri, 2004, Serum cardiac troponins and N-terminal pro-brain natriuretic peptide: a staging system for primary systemic amyloidosis, J Clin Oncol, 22, 3751, 10.1200/JCO.2004.03.029
Sipe, 2012, Amyloid fibril protein nomenclature: 2012 recommendations from the Nomenclature Committee of the International Society of Amyloidosis, Amyloid, 19, 167, 10.3109/13506129.2012.734345
Nilsson, 2010, Structural typing of systemic amyloidoses by luminescent-conjugated polymer spectroscopy, Am J Pathol, 176, 563, 10.2353/ajpath.2010.080797
Foli, 2011, The role of minor salivary gland biopsy in the diagnosis of systemic amyloidosis: results of a prospective study in 62 patients, Amyloid, 18, 80, 10.3109/13506129.2011.574354029
Schönland, 2012, Immunohistochemistry in the classification of systemic forms of amyloidosis: a systematic investigation of 117 patients, Blood, 119, 488, 10.1182/blood-2011-06-358507
Arbustini, 1997, Light and electron microscopy immunohistochemical characterization of amyloid deposits, Amyloid, 4, 157, 10.3109/13506129709014380
Palladini, 2009, Identification of amyloidogenic light chains requires the combination of serum-free light chain assay with immunofixation of serum and urine, Clin Chem, 55, 499, 10.1373/clinchem.2008.117143
Lachmann, 2003, Outcome in systemic AL amyloidosis in relation to changes in concentration of circulating free immunoglobulin light chains following chemotherapy, Br J Haematol, 122, 78, 10.1046/j.1365-2141.2003.04433.x
Deshmukh, 2009, Immunophenotype of neoplastic plasma cells in AL amyloidosis, J Clin Pathol, 62, 724, 10.1136/jcp.2009.065474
Paiva, 2009, Multiparameter flow cytometry quantification of bone marrow plasma cells at diagnosis provides more prognostic information than morphological assessment in myeloma patients, Haematologica, 94, 1599, 10.3324/haematol.2009.009100
Kyle, 2010, Monoclonal gammopathy of undetermined significance (MGUS) and smoldering (asymptomatic) multiple myeloma: IMWG consensus perspectives risk factors for progression and guidelines for monitoring and management, Leukemia, 24, 1121, 10.1038/leu.2010.60
Hawkins, 1990, Evaluation of systemic amyloidosis by scintigraphy with 123I-labeled serum amyloid P component, N Engl J Med, 323, 508, 10.1056/NEJM199008233230803
Buss, 2012, Longitudinal left ventricular function for prediction of survival in systemic light-chain amyloidosis: incremental value compared with clinical and biochemical markers, J Am Coll Cardiol, 60, 1067, 10.1016/j.jacc.2012.04.043
Selvanayagam, 2007, Evaluation and management of the cardiac amyloidosis, J Am Coll Cardiol, 50, 2101, 10.1016/j.jacc.2007.08.028
Maceira, 2005, Cardiovascular magnetic resonance in cardiac amyloidosis, Circulation, 111, 186, 10.1161/01.CIR.0000152819.97857.9D
Mekinian, 2010, Prognosis assessment of cardiac involvement in systemic AL amyloidosis by magnetic resonance imaging, Am J Med, 123, 864, 10.1016/j.amjmed.2010.03.022
Banypersad, 2013, Quantification of myocardial extracellular volume fraction in systemic AL amyloidosis: an equilibrium contrast cardiovascular magnetic resonance study, Circ Cardiovasc Imaging, 6, 34, 10.1161/CIRCIMAGING.112.978627
Bokhari, 2013, (99m)Tc-pyrophosphate scintigraphy for differentiating light-chain cardiac amyloidosis from the transthyretin-related familial and senile cardiac amyloidoses, Circ Cardiovasc Imaging, 6, 195, 10.1161/CIRCIMAGING.112.000132
Chen, 2012, Molecular imaging of amyloidosis: will the heart be the next target after the brain?, Curr Cardiol Rep, 14, 226, 10.1007/s11886-011-0239-5
Castano, 2012, Technetium pyrophosphate myocardial uptake and peripheral neuropathy in a rare variant of familial transthyretin (TTR) amyloidosis (Ser23Asn): a case report and literature review, Amyloid, 19, 41, 10.3109/13506129.2011.638682
Rapezzi, 2011, Usefulness and limitations of 99mTc-3,3-diphosphono-1,2-propanodicarboxylic acid scintigraphy in the aetiological diagnosis of amyloidotic cardiomyopathy, Eur J Nucl Med Mol Imaging, 38, 470, 10.1007/s00259-010-1642-7
Rapezzi, 2008, Usefulness of 99mTc-DPD scintigraphy in cardiac amyloidosis, J Am Coll Cardiol, 51, 1509, 10.1016/j.jacc.2007.12.038
Perugini, 2005, Noninvasive etiologic diagnosis of cardiac amyloidosis using 99mTc-3,3-diphosphono-1,2-propanodicarboxylic acid scintigraphy, J Am Coll Cardiol, 46, 1076, 10.1016/j.jacc.2005.05.073
Hutt, 2014, Utility and limitations of 3,3-diphosphono-1,2-propanodicarboxylic acid scintigraphy in systemic amyloidosis, Eur Heart J Cardiovasc Imaging, 15, 1289, 10.1093/ehjci/jeu107
Hongo, 2002, Iodine-123 metaiodobenzylguanidine scintigraphic analysis of myocardial sympathetic innervation in patients with AL (primary) amyloidosis, Am Heart J, 144, 122, 10.1067/mhj.2002.123115
Lekakis, 2003, Myocardial adrenergic denervation in patients with primary (AL) amyloidosis, Amyloid, 10, 117, 10.3109/13506120309041733
Antoni, 2013, In vivo visualization of amyloid deposits in the heart with 11C-PIB and PET, J Nucl Med, 54, 213, 10.2967/jnumed.111.102053
Dorbala, 2014, Imaging cardiac amyloidosis: a pilot study using 18F-florbetapir positron emission tomography, Eur J Nucl Med Mol Imaging, 41, 1652, 10.1007/s00259-014-2787-6
Wechalekar, 2011, European collaborative study of treatment outcomes in 347 patients with systemic AL amyloidosis with Mayo stage III disease, Blood, 118, 995, 10.1182/blood.V118.21.995.995
Palladini, 2010, The combination of high-sensitivity cardiac troponin T (hs-cTnT) at presentation and changes in N-terminal natriuretic peptide type B (NT-proBNP) after chemotherapy best predicts survival in AL amyloidosis, Blood, 116, 3426, 10.1182/blood-2010-05-286567
Kristen, 2010, Assessment of disease severity and outcome in patients with systemic light-chain amyloidosis by the high-sensitivity troponin T assay, Blood, 116, 2455, 10.1182/blood-2010-02-267708
Wechalekar, 2013, A European collaborative study of treatment outcomes in 346 patients with cardiac stage III AL amyloidosis, Blood, 121, 3420, 10.1182/blood-2012-12-473066
Dispenzieri, 2006, Absolute values of immunoglobulin free light chains are prognostic in patients with primary systemic amyloidosis undergoing peripheral blood stem cell transplantation, Blood, 107, 3378, 10.1182/blood-2005-07-2922
Wechalekar, 2009, High early mortality and poor outcomes for patients with AL amyloidosis presenting with high serum free light chains - a new risk stratification model, Haematologica, 94, 222
Kourelis, 2013, Coexistent multiple myeloma or increased bone marrow plasma cells define equally high-risk populations in patients with immunoglobulin light chain amyloidosis, J Clin Oncol, 31, 4319, 10.1200/JCO.2013.50.8499
Palladini, 2014, A staging system for renal outcome and early markers of renal response to chemotherapy in AL amyloidosis, Blood, 124, 2325, 10.1182/blood-2014-04-570010
Comenzo, 2012, Consensus guidelines for the conduct and reporting of clinical trials in systemic light-chain amyloidosis, Leukemia, 26, 2317, 10.1038/leu.2012.100
Palladini, 2012, 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, 30, 4541, 10.1200/JCO.2011.37.7614
Gertz, 2012, Immunoglobulin light-chain amyloidosis: growing recognition, new approaches to therapy, active clinical trials, Oncology, 26, 152
Dispenzieri, 2013, Patients with immunoglobulin light chain amyloidosis undergoing autologous stem cell transplantation have superior outcomes compared with patients with multiple myeloma: a retrospective review from a tertiary referral center, Bone Marrow Transplant, 48, 1302, 10.1038/bmt.2013.53
Bochtler, 2011, Hyperdiploidy is less frequent in AL amyloidosis compared with monoclonal gammopathy of undetermined significance and inversely associated with translocation t(11;14), Blood, 117, 3809, 10.1182/blood-2010-02-268987
Gertz, 1991, Classification of amyloidosis by the detection of clonal excess of plasma cells in the bone marrow, J Lab Clin Med, 118, 33
Palladini, 2004, 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, 103, 2936, 10.1182/blood-2003-08-2788
Palladini, 2007, Treatment with oral melphalan plus dexamethasone produces long-term remissions in AL amyloidosis, Blood, 110, 787, 10.1182/blood-2007-02-076034
Wechalekar, 2007, Safety and efficacy of risk-adapted cyclophosphamide, thalidomide, and dexamethasone in systemic AL amyloidosis, Blood, 109, 457, 10.1182/blood-2006-07-035352
Reece, 2011, 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, 118, 865, 10.1182/blood-2011-02-334227
Dispenzieri, 2007, The activity of lenalidomide with or without dexamethasone in patients with primary systemic amyloidosis, Blood, 109, 465, 10.1182/blood-2006-07-032987
Jaccard, 2007, High-dose melphalan versus melphalan plus dexamethasone for AL amyloidosis, N Engl J Med, 357, 1083, 10.1056/NEJMoa070484
Cibeira, 2011, Outcome of AL amyloidosis after high-dose melphalan and autologous stem cell transplantation: long-term results in a series of 421 patients, Blood, 118, 4346, 10.1182/blood-2011-01-330738
Landau, 2013, Bortezomib and dexamethasone consolidation following risk-adapted melphalan and stem cell transplantation for patients with newly diagnosed light chain amyloidosis, Leukemia, 27, 823, 10.1038/leu.2012.274
Venner, 2012, Cyclophosphamide, bortezomib, and dexamethasone therapy in AL amyloidosis is associated with high clonal response rates and prolonged progression-free survival, Blood, 119, 4387, 10.1182/blood-2011-10-388462
Kumar, 2012, Lenalidomide, cyclophosphamide, and dexamethasone (CRd) for light-chain amyloidosis: long-term results from a phase 2 trial, Blood, 119, 4860, 10.1182/blood-2012-01-407791
Moreau, 2010, Lenalidomide in combination with melphalan and dexamethasone in patients with newly diagnosed AL amyloidosis: a multicenter phase 1/2 dose-escalation study, Blood, 116, 4777, 10.1182/blood-2010-07-294405
Dispenzieri, 2012, Activity of pomalidomide in patients with immunoglobulin light-chain amyloidosis, Blood, 119, 5397, 10.1182/blood-2012-02-413161
Merlini, 2012, MLN9708, a novel, investigational oral proteasome inhibitor, in patients with relapsed or refractory light-chain amyloidosis (AL): results of a phase 1 study, Blood, 120, 731, 10.1182/blood.V120.21.731.731
Gertz, 2011, Trends in day 100 and 2-year survival after auto-SCT for AL amyloidosis: outcomes before and after 2006, Bone Marrow Transplant, 46, 970, 10.1038/bmt.2010.234
Gertz, 2013, Refinement in patient selection to reduce treatment-related mortality from SCT in amyloidosis, Bone Marrow Transplant, 48, 557, 10.1038/bmt.2012.170
Comenzo, 2002, Autologous stem cell transplantation for primary systemic amyloidosis, Blood, 99, 4276, 10.1182/blood.V99.12.4276
Sanchorawala, 2005, Serum free light-chain responses after high-dose intravenous melphalan and autologous stem cell transplantation for AL (primary) amyloidosis, Bone Marrow Transplant, 36, 597, 10.1038/sj.bmt.1705106
Skinner, 2004, High-dose melphalan and autologous stem-cell transplantation in patients with AL amyloidosis: an 8-year study, Ann Intern Med, 140, 85, 10.7326/0003-4819-140-2-200401200-00008
Landau, 2013, Bortezomib and dexamethasone consolidation following risk-adapted melphalan and stem cell transplantation for patients with newly diagnosed light-chain amyloidosis, Leukemia, 27, 823, 10.1038/leu.2012.274
Sanchorawala, 2015, Induction therapy with bortezomib followed by bortezomib-high dose melphalan and stem cell transplantation for AL amyloidosis: results of a prospective clinical trial, Biol Blood Marrow Transplant, 10.1016/j.bbmt.2015.04.001
Richardson, 2003, A phase 2 study of bortezomib in relapsed, refractory myeloma, N Engl J Med, 348, 2609, 10.1056/NEJMoa030288
Dimopoulos, 2011, Bortezomib for AL amyloidosis: moving forward, Blood, 118, 827, 10.1182/blood-2011-05-355115
Reece, 2009, Weekly and twice-weekly bortezomib in patients with systemic AL amyloidosis: results of a phase 1 dose-escalation study, Blood, 114, 1489, 10.1182/blood-2009-02-203398
Wechalekar, 2008, Efficacy of bortezomib in systemic AL amyloidosis with relapsed/refractory clonal disease, Haematologica, 93, 295, 10.3324/haematol.11627
Kastritis, 2010, Bortezomib with or without dexamethasone in primary systemic (light chain) amyloidosis, J Clin Oncol, 28, 1031, 10.1200/JCO.2009.23.8220
Mikhael, 2012, Cyclophosphamide-bortezomib-dexamethasone (CyBorD) produces rapid and complete hematologic response in patients with AL amyloidosis, Blood, 119, 4391, 10.1182/blood-2011-11-390930
Sanchorawala, 2010, Durable hematologic complete responses can be achieved with lenalidomide in AL amyloidosis, Blood, 116, 1990, 10.1182/blood-2010-07-295485
Kastritis, 2012, A phase 1/2 study of lenalidomide with low-dose oral cyclophosphamide and low-dose dexamethasone (RdC) in AL amyloidosis, Blood, 119, 5384, 10.1182/blood-2011-12-396903
Palladini, 2013, A phase II trial of cyclophosphamide, lenalidomide and dexamethasone in previously treated patients with AL amyloidosis, Haematologica, 98, 433, 10.3324/haematol.2012.073593
Venner, 2014, A matched comparison of cyclophosphamide, bortezomib and dexamethasone (CVD) versus risk-adapted cyclophosphamide, thalidomide and dexamethasone (CTD) in AL amyloidosis, Leukemia, 8, 2304, 10.1038/leu.2014.218
Lane, 2013, AA amyloidosis complicating the hereditary periodic fever syndromes, Arthritis Rheum, 65, 1116, 10.1002/art.37827
Obici, 2012, AA amyloidosis: basic knowledge, unmet needs and future treatments, Swiss Med Wkly, 142, w13580
Nakamura, 2010, Etanercept can induce resolution of renal deterioration in patients with amyloid A amyloidosis secondary to rheumatoid arthritis, Clin Rheumatol, 29, 1395, 10.1007/s10067-010-1469-4
Obici, 2011, Favourable and sustained response to anakinra in tumour necrosis factor receptor-associated periodic syndrome (TRAPS) with or without AA amyloidosis, Ann Rheum Dis, 70, 1511, 10.1136/ard.2010.143438
Kuemmerle-Deschner, 2011, Two-year results from an open-label, multicentre, phase III study evaluating the safety and efficacy of canakinumab in patients with cryopyrin-associated periodic syndrome across different severity phenotypes, Ann Rheum Dis, 70, 2095, 10.1136/ard.2011.152728
Lachmann, 2009, Use of canakinumab in the cryopyrin-associated periodic syndrome, N Engl J Med, 360, 2416, 10.1056/NEJMoa0810787
Kisilevsky, 2002, Novel glycosaminoglycan precursors as anti-amyloid agents part II, J Mol Neuroscience, 19, 45, 10.1007/s12031-002-0009-3
Kisilevsky, 2007, Heparan sulfate as a therapeutic target in amyloidogenesis: prospects and possible complications, Amyloid, 14, 21, 10.1080/13506120601116419
Sattianayagam, 2011, The role of liver transplantation in hereditary non-neuropathic systemic amyloidosis, Gastroenterology, 140, S-963, 10.1016/S0016-5085(11)63987-5
de Carvalho, 2002, Long-term quantitative evaluation of liver transplantation in familial amyloid polyneuropathy (Portuguese V30M), Amyloid, 9, 126, 10.3109/13506120208995245
Stangou, 1998, Progressive cardiac amyloidosis following liver transplantation for familial amyloid polyneuropathy: implications for amyloid fibrillogenesis, Transplantation, 66, 229, 10.1097/00007890-199807270-00016
Ericzon, 2008, Domino liver transplantation: risks and benefits, Transplant Proc, 40, 1130, 10.1016/j.transproceed.2008.03.020
Pradotto, 2012, Amyloid polyneuropathy following domino liver transplantation, Muscle Nerve, 45, 918, 10.1002/mus.23265
Antonini, 2013, Reversibility of acquired amyloid polyneuropathy after liver retransplantation, Am J Transplant, 13, 2734, 10.1111/ajt.12378
Abdelfatah, 2014, Domino liver transplantation as a cause of acquired familial amyloid polyneuropathy, Amyloid, 21, 136, 10.3109/13506129.2014.885894
Hamour, 2008, Heart transplantation for homozygous familial transthyretin (TTR) V122I cardiac amyloidosis, Am J Transplant, 8, 1056, 10.1111/j.1600-6143.2008.02162.x
Fuchs, 2005, Heart transplantation in a 68-year-old patient with senile systemic amyloidosis, Am J Transplant, 5, 1159, 10.1111/j.1600-6143.2005.00805.x
Stangou, 2010, Hereditary fibrinogen A alpha-chain amyloidosis: phenotypic characterization of a systemic disease and the role of liver transplantation, Blood, 115, 2998, 10.1182/blood-2009-06-223792
Gillmore, 2010, Hereditary fibrinogen A alpha-chain amyloidosis: clinical phenotype and role of liver transplantation, Blood, 115, 4313, 10.1182/blood-2010-01-261750
Dubrey, 2012, Amyloid diseases of the heart: current and future therapies, QJM, 105, 617, 10.1093/qjmed/hcr259
Rowczenio, 2011, Amyloidogenicity and clinical phenotype associated with five novel mutations in apolipoprotein A-I, Am J Pathol, 179, 1978, 10.1016/j.ajpath.2011.06.024
Gillmore, 2006, Organ transplantation in hereditary apolipoprotein AI amyloidosis, Am J Transplant, 6, 2342, 10.1111/j.1600-6143.2006.01507.x
Johnson, 2012, The transthyretin amyloidoses: from delineating the molecular mechanism of aggregation linked to pathology to a regulatory-agency-approved drug, J Mol Biol, 421, 185, 10.1016/j.jmb.2011.12.060
Coelho, 2012, Tafamidis for transthyretin familial amyloid polyneuropathy: a randomized, controlled trial, Neurology, 79, 785, 10.1212/WNL.0b013e3182661eb1
Kolstoe, 2010, Trapping of palindromic ligands within native transthyretin prevents amyloid formation, Proc Natl Acad Sci USA, 107, 20483, 10.1073/pnas.1008255107
Sekijima, 2006, Orally administered diflunisal stabilizes transthyretin against dissociation required for amyloidogenesis, Amyloid, 13, 236, 10.1080/13506120600960882
Miller, 2004, Native state stabilization by NSAIDs inhibits transthyretin amyloidogenesis from the most common familial disease variants, Lab Invest, 84, 545, 10.1038/labinvest.3700059
Berk, 2013, Repurposing diflunisal for familial amyloid polyneuropathy: a randomized clinical trial, JAMA, 310, 2658, 10.1001/jama.2013.283815
Adams, 2013, Recent advances in the treatment of familial amyloid polyneuropathy, Ther Adv Neurol Disord, 6, 129, 10.1177/1756285612470192
Coelho, 2013, Safety and efficacy of RNAi therapy for transthyretin amyloidosis, N Engl J Med, 369, 819, 10.1056/NEJMoa1208760
Solomon, 2003, Therapeutic potential of chimeric amyloid-reactive monoclonal antibody 11-1F4, Clin Cancer Res, 9, 3831S
Hrncic, 2000, Antibody-mediated resolution of light chain-associated amyloid deposits, Am J Pathol, 157, 1239, 10.1016/S0002-9440(10)64639-1
Wall, 2010, Radioimmunodetection of amyloid deposits in patients with AL amyloidosis, Blood, 116, 2241, 10.1182/blood-2010-03-273797
Wall, 2012, AL amyloid imaging and therapy with a monoclonal antibody to a cryptic epitope on amyloid fibrils, PLoS One, 7, e52686, 10.1371/journal.pone.0052686
Tennent, 1995, Serum amyloid P component prevents proteolysis of the amyloid fibrils of Alzheimer disease and systemic amyloidosis, Proc Natl Acad Sci USA, 92, 4299, 10.1073/pnas.92.10.4299
Pepys, 2002, Targeted pharmacological depletion of serum amyloid P component for treatment of human amyloidosis, Nature, 417, 254, 10.1038/417254a
Gillmore, 2010, Sustained pharmacological depletion of serum amyloid P component in patients with systemic amyloidosis, Br J Haematol, 148, 760, 10.1111/j.1365-2141.2009.08036.x