Diflunisal for ATTR Cardiac Amyloidosis

Congestive Heart Failure

Volumn 18, Issue 6, 2012, Pages 315-319

  Castaño, Adam ^a   Helmke, Stephen ^a   Alvarez, Julissa ^a   Delisle, Susan ^a   Maurer, Mathew S ^a  

^a Columbia College of Physicians and Surgeons ^*  (United States)

Author keywords

[No Author keywords available]

Indexed keywords

DIFLUNISAL; PREALBUMIN; TROPONIN I;

AGED; ARTICLE; COHORT ANALYSIS; CONTROLLED STUDY; DRUG EFFICACY; DRUG SAFETY; DRUG TOLERABILITY; DRUG WITHDRAWAL; FEMALE; FOLLOW UP; GLOMERULUS FILTRATION RATE; HEART AMYLOIDOSIS; HEART EJECTION FRACTION; HEART LEFT VENTRICLE MASS; HEART STRESS; HUMAN; MAJOR CLINICAL STUDY; MALE; MULTICENTER STUDY; MUTANT; OPEN STUDY; PRIORITY JOURNAL; RANDOMIZED CONTROLLED TRIAL; WILD TYPE;

AGED; AMYLOID NEUROPATHIES, FAMILIAL; ANTI-INFLAMMATORY AGENTS, NON-STEROIDAL; COHORT STUDIES; DIFLUNISAL; FEMALE; HEART DISEASES; HUMANS; MALE; TREATMENT OUTCOME;

EID: 84869881017     PISSN: 15275299     EISSN: 17517133     Source Type: Journal    
DOI: 10.1111/j.1751-7133.2012.00303.x     Document Type: Article

References (34)

1. Benson MD. Amyloidosis. In: Scriver CR, Beaudet AL, Sly WS, Valle D, eds. The Metabolic and Molecular Bases of Inherited Disease, 7th ed. New York, NY: McGraw-Hill; 1995:7.
2. Lachmann HJ, Booth DR, Booth SE, et al. Misdiagnosis of hereditary amyloidosis as AL (primary) amyloidosis. N Engl J Med. 2002;346:1786-1791.
3. Bhuiyan T, Helmke S, Patel AR, et al. Pressure-volume relationships in patients with transthyretin (ATTR) cardiac amyloidosis secondary to V122I mutations and wild-type transthyretin: Transthyretin Cardiac Amyloid Study (TRACS). Circ Heart Fail. 2011;4:121-128.
4. Robbins J. Thyroxine-binding proteins. Prog Clin Biol Res. 1976;5:331-355.
5. Connors LH, Lim A, Prokaeva T, et al. Tabulation of human transthyretin (TTR) variants, 2003. Amyloid. 2003;10:160-184.
6. Rapezzi C, Quarta CC, Riva L, et al. Transthyretin-related amyloidoses and the heart: a clinical overview. Nat Rev Cardiol. 2010;7:398-408.
7. 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;40:232-239.
8. Cornwell GG III, Murdoch WL, Kyle RA, et al. Frequency and distribution of senile cardiovascular amyloid. A clinicopathologic correlation. Am J Med. 1983;75:618-623.
9. Mirzoyev SA, Edwards WD, Mohammed SF, et al., eds. Cardiac Amyloid Deposition is Common in Elderly Patients with Heart Failure and Preserved Ejection Fraction. Chicago, IL: AHA; 2010.
10. Jacobson DR, Gorevic PD, Buxbaum JN. A homozygous transthyretin variant associated with senile systemic amyloidosis: evidence for a late-onset disease of genetic etiology. Am J Hum Genet. July 1990;47:127-136.
11. Jacobson DR, Pastore RD, Yaghoubian R, et al. Variant-sequence transthyretin (isoleucine 122) in late-onset cardiac amyloidosis in black Americans. N Engl J Med. 1997;336:466-473.
12. Suhr OB, Herlenius G, Friman S, Ericzon BG. Liver transplantation for hereditary transthyretin amyloidosis. Liver Transpl. 2000;6:263-276.
13. Pilato E, Dell'Amore A, Botta L, Arpesella G. Combined heart and liver transplantation for familial amyloidotic neuropathy. Eur J Cardiothorac Surg. 2007;32:180-182.
14. Liepnieks JJ, Zhang LQ, Benson MD. Progression of transthyretin amyloid neuropathy after liver transplantation. Neurology. 2010;75:324-327.
15. Hammarstrom P, Wiseman RL, Powers ET, Kelly JW. Prevention of transthyretin amyloid disease by changing protein misfolding energetics. Science. 2003;299:713-716.
16. Alhamadsheh MM, Connelly S, Cho A, et al. Potent kinetic stabilizers that prevent transthyretin-mediated cardiomyocyte proteotoxicity. Sci Transl Med. 2011;3:97ra81.
17. Sekijima Y, Dendle MA, Kelly JW. Orally administered diflunisal stabilizes transthyretin against dissociation required for amyloidogenesis. Amyloid. 2006;13:236-249.
18. Tojo K, Sekijima Y, Kelly JW, Ikeda S. Diflunisal stabilizes familial amyloid polyneuropathy-associated transthyretin variant tetramers in serum against dissociation required for amyloidogenesis. Neurosci Res. 2006;56:441-449.
19. Johnson SM, Connelly S, Fearns C, et al. The transthyretin amyloidoses: from delineating the molecular mechanism of aggregation linked to pathology to a regulatory-agency-approved drug. J Mol Biol. 2012 Jan 5. [Epub ahead of print].
20. Miller SR, Sekijima Y, Kelly JW. Native state stabilization by NSAIDs inhibits transthyretin amyloidogenesis from the most common familial disease variants. Lab Invest. 2004;84:545-552.
21. Adamski-Werner SL, Palaninathan SK, Sacchettini JC, Kelly JW. Diflunisal analogues stabilize the native state of transthyretin. Potent inhibition of amyloidogenesis. J Med Chem. 2004;47:355-374.
22. Berk JL, Dyck PJ, Obici L, et al. The diflunisal trial: update on study drug tolerance and disease progression. Amyloid. 2011;18(suppl 1):191-192.
23. Kearney PM, Baigent C, Godwin J, et al. Do selective cyclo-oxygenase-2 inhibitors and traditional non-steroidal anti-inflammatory drugs increase the risk of atherothrombosis? Meta-analysis of randomised trials. BMJ. 2006;332:1302-1308.
24. Epstein M. Non-steroidal anti-inflammatory drugs and the continuum of renal dysfunction. J Hypertens Suppl. 2002;20:S17-S23.
25. Marnett LJ, Kalgutkar AS. Cyclooxygenase 2 inhibitors: discovery, selectivity and the future. Trends Pharmacol Sci. 1999;20:465-469.
26. Wallace JL. Pathogenesis of NSAID-induced gastroduodenal mucosal injury. Best Pract Res Clin Gastroenterol. 2001;15:691-703.
27. Mukherjee D, Nissen SE, Topol EJ. Risk of cardiovascular events associated with selective COX-2 inhibitors. JAMA. 2001;286:954-959.
28. Page J, Henry D. Consumption of NSAIDs and the development of congestive heart failure in elderly patients: an underrecognized public health problem. Arch Intern Med. 2000;160:777-784.
29. Suhr O, Danielsson A, Holmgren G, Steen L. Malnutrition and gastrointestinal dysfunction as prognostic factors for survival in familial amyloidotic polyneuropathy. J Intern Med. 1994;235:479-485.
30. Murtagh B, Hammill SC, Gertz MA, et al. Electrocardiographic findings in primary systemic amyloidosis and biopsy-proven cardiac involvement. Am J Cardiol. 2005;95:535-537.
31. Connors LH, Doros G, Sam F, et al. Clinical features and survival in senile systemic amyloidosis: comparison to familial transthyretin cardiomyopathy. Amyloid. 2011;18(suppl 1):152-154.
32. Ruberg FL, Maurer MS, Judge DP, et al. Prospective evaluation of the morbidity and mortality of wild-type and V122I mutant transthyretin amyloid cardiomyopathy: the transthyretin amyloidosis cardiac study (TRACS). Am Heart J. 2012 (in press).
33. Russo C, Green P, Maurer MS, eds. Comparison of V12I and senile cardiac amyloidosis: differences in clinical features and outcomes. 13th International Symposium on Amyloid; 2012; Groningen, Netherlands.
34. Berk JL, Suhr OB, Sekijima YS, et al., eds. The diflunisal trial: study accrual and drug tolerance. VIIIth International Symposium on Familial Amyloidotic Polyneuropathy; 2011 November 20-23; Kumamoto, Japan.