Repurposing diflunisal for familial amyloid polyneuropathy: A randomized clinical trial

JAMA

Volumn 310, Issue 24, 2013, Pages 2658-2667

  Berk, John L ^a   Suhr, Ole B ^b   Obici, Laura ^c   Sekijima, Yoshiki ^d   Zeldenrust, Steven R ^e   Yamashita, Taro ^f   Heneghan, Michael A ^g   Gorevic, Peter D ^j   Litchy, William J ^e   Wiesman, Janice F ^a   Nordh, Erik ^b   Corato, Manuel ^h   Lozza, Alessandro ⁱ   Cortese, Andrea ⁱ   Robinson Papp, Jessica ^j   Colton, Theodore ^k   Rybin, Denis V ^k   Bisbee, Alice B ^k   Ando, Yukio ^f   Ikeda, Shu Ichi ^d   Seldin, David C ^a   Merlini, Giampaolo ^c   Skinner, Martha ^a   Kelly, Jeffery W ^l   Dyck, Peter J ^e   more..

^a BOSTON UNIVERSITY SCHOOL OF MEDICINE   (United States)

^b UMEÅ UNIVERSITY   (Sweden)

^c UNIVERSITY OF PAVIA   (Italy)

^d SHINSHU UNIVERSITY SCHOOL OF MEDICINE   (Japan)

^e MAYO CLINIC   (United States)

^f KUMAMOTO UNIVERSITY   (Japan)

^g KING'S COLLEGE HOSPITAL NHS FOUNDATION TRUST   (United Kingdom)

^h HUMANITAS CLINICAL AND RESEARCH CENTER   (Italy)

ⁱ C MONDINO NATIONAL NEUROLOGICAL INSTITUTE   (Italy)

^j MOUNT SINAI SCHOOL OF MEDICINE   (United States)

^k BOSTON UNIVERSITY SCHOOL OF PUBLIC HEALTH   (United States)

^l SCRIPPS RESEARCH INSTITUTE   (United States)

more..

Author keywords

[No Author keywords available]

Indexed keywords

DIFLUNISAL; PLACEBO;

ADULT; ARTICLE; BODY MASS; CLINICAL EFFECTIVENESS; CONGESTIVE HEART FAILURE; CONTROLLED CLINICAL TRIAL; CONTROLLED STUDY; DISEASE ASSOCIATION; DISEASE COURSE; DOUBLE BLIND PROCEDURE; DRUG EFFICACY; DRUG HYPERSENSITIVITY; DRUG SAFETY; DRUG TOLERABILITY; FAMILIAL AMYLOID POLYNEUROPATHY; FEMALE; FUNCTIONAL ASSESSMENT; GASTROINTESTINAL HEMORRHAGE; GASTROINTESTINAL SYMPTOM; GLAUCOMA; HEADACHE; HEART DISEASE; HUMAN; KIDNEY DISEASE; KIDNEY FAILURE; MAJOR CLINICAL STUDY; MALE; MIDDLE AGED; MULTICENTER STUDY; MUSCULOSKELETAL DISEASE; NAUSEA; OUTCOME ASSESSMENT; PATIENT ASSESSMENT; PATIENT COMPLIANCE; PRIORITY JOURNAL; QUALITY OF LIFE; RANDOMIZED CONTROLLED TRIAL; SCORING SYSTEM; SENSITIVITY ANALYSIS; SURVIVAL RATE; SURVIVAL TIME; TREATMENT DURATION;

EID: 84890954073     PISSN: 00987484     EISSN: 15383598     Source Type: Journal    
DOI: 10.1001/jama.2013.283815     Document Type: Article

References (39)

1. Monaco HL, Rizzi M, Coda A. Structure of a complex of 2 plasma proteins: transthyretin and retinol-binding protein. Science. 1995;268(5213):1039-1041.
2. Blake CC, Geisow MJ, Oatley SJ, Rérat B, Rérat C. Structure of prealbumin: secondary, tertiary and quaternary interactions determined by Fourier refinement at 1.8 A. J Mol Biol. 1978;121(3):339-356.
3. Colon W, Kelly JW. Partial denaturation of transthyretin is sufficient for amyloid fibril formation in vitro. Biochemistry. 1992;31(36):8654-8660.
4. Foss TR, Wiseman RL, Kelly JW. The pathway by which the tetrameric protein transthyretin dissociates. Biochemistry. 2005;44(47):15525-15533.
5. Andrade C. A peculiar form of peripheral neuropathy; familiar atypical generalized amyloidosis with special involvement of the peripheral nerves. Brain. 1952;75(3):408-427.
6. Benson MD, Kincaid JC. The molecular biology and clinical features of amyloid neuropathy. Muscle Nerve. 2007;36(4):411-423.
7. Planté-Bordeneuve V, Lalu T, Misrahi M, et al. Genotypic-phenotypic variations in a series of 65 patients with familial amyloid polyneuropathy. Neurology. 1998;51(3):708-714.
8. Ando Y, Coelho T, Berk JL, et al. Guideline of transthyretin-related hereditary amyloidosis for clinicians. Orphanet J Rare Dis. 2013;8:31.
9. Okamoto S, Wixner J, Obayashi K, et al. Liver transplantation for familial amyloidotic polyneuropathy: impact on Swedish patients' survival. Liver Transpl. 2009;15(10):1229-1235.
10. Holmgren G, Steen L, Ekstedt J, et al. Biochemical effect of liver transplantation in 2 Swedish patients with familial amyloidotic polyneuropathy (FAP-met30). Clin Genet. 1991;40(3):242-246.
11. Liepnieks JJ, Zhang LQ, Benson MD. Progression of transthyretin amyloid neuropathy after liver transplantation. Neurology. 2010;75(4):324-327.
12. Olofsson BO, Backman C, Karp K, Suhr OB. Progression of cardiomyopathy after liver transplantation in patients with familial amyloidotic polyneuropathy, Portuguese type. Transplantation. 2002;73(5):745-751.
13. Hammarström P, Schneider F, Kelly JW. Trans-suppression ofmisfolding in an amyloid disease. Science. 2001;293(5539):2459-2462.
14. Hammarström P, Wiseman RL, Powers ET, Kelly JW. Prevention of transthyretin amyloid disease by changing protein misfolding energetics. Science. 2003;299(5607):713-716.
15. Coelho T, Chorao R, Sousa A, Alves I, Torres MF, Saraiva MJ. Compound heterozygotes of transthyretin Met30 and transthyretin Met119 are protected from the devastating effects of familial amyloid polyneuropathy. Neuromuscul Disord. 1996;6(S20):27.
16. Sekijima Y, Dendle MA, Kelly JW. Orally administered diflunisal stabilizes transthyretin against dissociation required for amyloidogenesis. Amyloid. 2006;13(4):236-249.
17. Coelho T, Maia LF, Martins da Silva A, et al. Tafamidis for transthyretin familial amyloid polyneuropathy: a randomized, controlled trial. Neurology. 2012;79(8):785-792.
18. Johnson SM, Wiseman RL, Sekijima Y, Green NS, Adamski-Werner SL, Kelly JW. Native state kinetic stabilization as a strategy to ameliorate protein misfolding diseases: a focus on the transthyretin amyloidoses. Acc Chem Res. 2005;38(12):911-921.
19. 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(5):545-552.
20. Dyck PJ, Davies JL, Litchy WJ, O'Brien PC. Longitudinal assessment of diabetic polyneuropathy using a composite score in the Rochester Diabetic Neuropathy Study cohort. Neurology. 1997;49(1):229-239.
21. McDougall AJ, McLeod JG. Autonomic neuropathy, II: specific peripheral neuropathies. J Neurol Sci. 1996;138(1-2):1-13.
22. Ferlini A, Romeo G, Tassinari CA, Saraiva MJ, Costa PP, Salvi F. Discrimination of peripheral polyneuropathies caused by TTR variant or diabetes in the same pedigree through protein studies. Adv Neurol. 1988;48:201-208.
23. Diabetic polyneuropathy in controlled clinical trials: Consensus Report of the Peripheral Nerve Society. Ann Neurol. 1995;38(3):478-482.
24. Apfel SC, Schwartz S, Adornato BT, et al; rhNGF Clinical Investigator Group. Efficacy and safety of recombinant human nerve growth factor in patients with diabetic polyneuropathy: a randomized controlled trial. JAMA. 2000;284(17):2215-2221.
25. Dyck PJ, Bushek W, Spring EM, et al. Vibratory and cooling detection thresholds compared with other tests in diagnosing and staging diabetic neuropathy. Diabetes Care. 1987;10(4):432-440.
26. Service FJ, Daube JR, O'Brien PC, et al. Effect of blood glucose control on peripheral nerve function in diabetic patients. Mayo Clin Proc. 1983;58(5):283-289.
27. Ziegler D, Ametov A, Barinov A, et al. Oral treatment with alpha-lipoic acid improves symptomatic diabetic polyneuropathy: the SYDNEY 2 trial. Diabetes Care. 2006;29(11):2365-2370.
28. Dyck PJ, Low PA, Windebank AJ, et al. Plasma exchange in polyneuropathy associated with monoclonal gammopathy of undetermined significance. N Engl J Med. 1991;325(21):1482-1486.
29. Dyck PJ, Daube J, O'Brien P, et al. Plasma exchange in chronic inflammatory demyelinating polyradiculoneuropathy. N Engl J Med. 1986;314(8):461-465.
30. Dyck PJ, Litchy WJ, Kratz KM, et al. A plasma exchange vs immune globulin infusion trial in chronic inflammatory demyelinating polyradiculoneuropathy. Ann Neurol. 1994;36(6):838-845.
31. Dyck PJ, Litchy WJ, Lehman KA, Hokanson JL, Low PA, O'Brien PC. Variables influencing neuropathic endpoints: the Rochester Diabetic Neuropathy Study of Healthy Subjects. Neurology. 1995;45(6):1115-1121.
32. Dyck PJ, Overland CJ, Low PA, et al. "Unequivocally abnormal" vs "usual" signs and symptoms for proficient diagnosis of diabetic polyneuropathy: Cl vs N Phys Trial. Arch Neurol. 2012;69(12):1609-1614.
33. Seldin DC, Anderson JJ, Sanchorawala V, et al. Improvement in quality of life of patients with AL amyloidosis treated with high-dose melphalan and autologous stem cell transplantation. Blood. 2004;104(6):1888-1893.
34. 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(5):479-485.
35. Suhr OB, Holmgren G, Steen L, et al. Liver transplantation in familial amyloidotic polyneuropathy: follow-up of the first 20 Swedish patients. Transplantation. 1995;60(9):933-938.
36. Tashima K, Ando Y, Terazaki H, et al. Outcome of liver transplantation for transthyretin amyloidosis: follow-up of Japanese familial amyloidotic polyneuropathy patients. J Neurol Sci. 1999;171(1):19-23.
37. Diggle PJ, Heagerty P, Liang KY, Zegger SL. Analysis of Longitudinal Data. 2nd ed. Oxford, England: Oxford University Press; 2002.
38. Committee on National Statistics. The Prevention and Treatment of Missing Data in Clinical Trials. Washington, DC: National Academies Press; 2010.
39. Rubin DB. Multiple Imputation for Nonresponse in Surveys. New York, NY: John Wiley & Sons Inc; 1987.