메뉴 건너뛰기




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

Repurposing diflunisal for familial amyloid polyneuropathy: A randomized clinical trial

(25)  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   more..


Author keywords

[No Author keywords available]

Indexed keywords

DIFLUNISAL; PLACEBO;

EID: 84890954073     PISSN: 00987484     EISSN: 15383598     Source Type: Journal    
DOI: 10.1001/jama.2013.283815     Document Type: Article
Times cited : (549)

References (39)
  • 1
    • 0029062667 scopus 로고
    • Structure of a complex of 2 plasma proteins: Transthyretin and retinol-binding protein
    • 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.
    • (1995) Science , vol.268 , Issue.5213 , pp. 1039-1041
    • Monaco, H.L.1    Rizzi, M.2    Coda, A.3
  • 2
    • 0017824077 scopus 로고
    • Structure of prealbumin: Secondary, tertiary and quaternary interactions determined by Fourier refinement at 1.8 A
    • 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.
    • (1978) J Mol Biol , vol.121 , Issue.3 , pp. 339-356
    • Blake, C.C.1    Geisow, M.J.2    Oatley, S.J.3    Rérat, B.4    Rérat, C.5
  • 3
    • 0026675307 scopus 로고
    • Partial denaturation of transthyretin is sufficient for amyloid fibril formation in vitro
    • Colon W, Kelly JW. Partial denaturation of transthyretin is sufficient for amyloid fibril formation in vitro. Biochemistry. 1992;31(36):8654-8660.
    • (1992) Biochemistry , vol.31 , Issue.36 , pp. 8654-8660
    • Colon, W.1    Kelly, J.W.2
  • 4
    • 28244486084 scopus 로고    scopus 로고
    • The pathway by which the tetrameric protein transthyretin dissociates
    • Foss TR, Wiseman RL, Kelly JW. The pathway by which the tetrameric protein transthyretin dissociates. Biochemistry. 2005;44(47):15525-15533.
    • (2005) Biochemistry , vol.44 , Issue.47 , pp. 15525-15533
    • Foss, T.R.1    Wiseman, R.L.2    Kelly, J.W.3
  • 5
    • 77957180065 scopus 로고
    • A peculiar form of peripheral neuropathy; familiar atypical generalized amyloidosis with special involvement of the peripheral nerves
    • 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.
    • (1952) Brain , vol.75 , Issue.3 , pp. 408-427
    • Andrade, C.1
  • 6
    • 34848818004 scopus 로고    scopus 로고
    • The molecular biology and clinical features of amyloid neuropathy
    • Benson MD, Kincaid JC. The molecular biology and clinical features of amyloid neuropathy. Muscle Nerve. 2007;36(4):411-423.
    • (2007) Muscle Nerve , vol.36 , Issue.4 , pp. 411-423
    • Benson, M.D.1    Kincaid, J.C.2
  • 7
    • 0344074661 scopus 로고    scopus 로고
    • Genotypic-phenotypic variations in a series of 65 patients with familial amyloid polyneuropathy
    • 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.
    • (1998) Neurology , vol.51 , Issue.3 , pp. 708-714
    • Planté-Bordeneuve, V.1    Lalu, T.2    Misrahi, M.3
  • 8
    • 84873979913 scopus 로고    scopus 로고
    • Guideline of transthyretin-related hereditary amyloidosis for clinicians
    • Ando Y, Coelho T, Berk JL, et al. Guideline of transthyretin-related hereditary amyloidosis for clinicians. Orphanet J Rare Dis. 2013;8:31.
    • (2013) Orphanet J Rare Dis , vol.8 , pp. 31
    • Ando, Y.1    Coelho, T.2    Berk, J.L.3
  • 9
    • 70350549746 scopus 로고    scopus 로고
    • Liver transplantation for familial amyloidotic polyneuropathy: Impact on Swedish patients' survival
    • 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.
    • (2009) Liver Transpl , vol.15 , Issue.10 , pp. 1229-1235
    • Okamoto, S.1    Wixner, J.2    Obayashi, K.3
  • 10
    • 0025990675 scopus 로고
    • Biochemical effect of liver transplantation in 2 Swedish patients with familial amyloidotic polyneuropathy (FAP-met30)
    • 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.
    • (1991) Clin Genet , vol.40 , Issue.3 , pp. 242-246
    • Holmgren, G.1    Steen, L.2    Ekstedt, J.3
  • 11
    • 77955156168 scopus 로고    scopus 로고
    • Progression of transthyretin amyloid neuropathy after liver transplantation
    • Liepnieks JJ, Zhang LQ, Benson MD. Progression of transthyretin amyloid neuropathy after liver transplantation. Neurology. 2010;75(4):324-327.
    • (2010) Neurology , vol.75 , Issue.4 , pp. 324-327
    • Liepnieks, J.J.1    Zhang, L.Q.2    Benson, M.D.3
  • 12
    • 0037087692 scopus 로고    scopus 로고
    • Progression of cardiomyopathy after liver transplantation in patients with familial amyloidotic polyneuropathy, Portuguese type
    • 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.
    • (2002) Transplantation , vol.73 , Issue.5 , pp. 745-751
    • Olofsson, B.O.1    Backman, C.2    Karp, K.3    Suhr, O.B.4
  • 13
    • 0035964955 scopus 로고    scopus 로고
    • Trans-suppression ofmisfolding in an amyloid disease
    • Hammarström P, Schneider F, Kelly JW. Trans-suppression ofmisfolding in an amyloid disease. Science. 2001;293(5539):2459-2462.
    • (2001) Science , vol.293 , Issue.5539 , pp. 2459-2462
    • Hammarström, P.1    Schneider, F.2    Kelly, J.W.3
  • 14
    • 0037473750 scopus 로고    scopus 로고
    • Prevention of transthyretin amyloid disease by changing protein misfolding energetics
    • Hammarström P, Wiseman RL, Powers ET, Kelly JW. Prevention of transthyretin amyloid disease by changing protein misfolding energetics. Science. 2003;299(5607):713-716.
    • (2003) Science , vol.299 , Issue.5607 , pp. 713-716
    • Hammarström, P.1    Wiseman, R.L.2    Powers, E.T.3    Kelly, J.W.4
  • 15
    • 0002270675 scopus 로고    scopus 로고
    • Compound heterozygotes of transthyretin Met30 and transthyretin Met119 are protected from the devastating effects of familial amyloid polyneuropathy
    • 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.
    • (1996) Neuromuscul Disord , vol.6 , Issue.S20 , pp. 27
    • Coelho, T.1    Chorao, R.2    Sousa, A.3    Alves, I.4    Torres, M.F.5    Saraiva, M.J.6
  • 16
    • 33751082387 scopus 로고    scopus 로고
    • Orally administered diflunisal stabilizes transthyretin against dissociation required for amyloidogenesis
    • Sekijima Y, Dendle MA, Kelly JW. Orally administered diflunisal stabilizes transthyretin against dissociation required for amyloidogenesis. Amyloid. 2006;13(4):236-249.
    • (2006) Amyloid , vol.13 , Issue.4 , pp. 236-249
    • Sekijima, Y.1    Dendle, M.A.2    Kelly, J.W.3
  • 17
    • 84862222705 scopus 로고    scopus 로고
    • Tafamidis for transthyretin familial amyloid polyneuropathy: A randomized, controlled trial
    • 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.
    • (2012) Neurology , vol.79 , Issue.8 , pp. 785-792
    • Coelho, T.1    Maia, L.F.2    Martins Da Silva, A.3
  • 18
    • 28244502156 scopus 로고    scopus 로고
    • Native state kinetic stabilization as a strategy to ameliorate protein misfolding diseases: A focus on the transthyretin amyloidoses
    • 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.
    • (2005) Acc Chem Res , vol.38 , Issue.12 , pp. 911-921
    • Johnson, S.M.1    Wiseman, R.L.2    Sekijima, Y.3    Green, N.S.4    Adamski-Werner, S.L.5    Kelly, J.W.6
  • 19
    • 2942599707 scopus 로고    scopus 로고
    • Native state stabilization by NSAIDs inhibits transthyretin amyloidogenesis from the most common familial disease variants
    • 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.
    • (2004) Lab Invest , vol.84 , Issue.5 , pp. 545-552
    • Miller, S.R.1    Sekijima, Y.2    Kelly, J.W.3
  • 20
    • 0030753606 scopus 로고    scopus 로고
    • Longitudinal assessment of diabetic polyneuropathy using a composite score in the Rochester Diabetic Neuropathy Study cohort
    • 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.
    • (1997) Neurology , vol.49 , Issue.1 , pp. 229-239
    • Dyck, P.J.1    Davies, J.L.2    Litchy, W.J.3    O'Brien, P.C.4
  • 21
    • 0029884027 scopus 로고    scopus 로고
    • Autonomic neuropathy, II: Specific peripheral neuropathies
    • McDougall AJ, McLeod JG. Autonomic neuropathy, II: specific peripheral neuropathies. J Neurol Sci. 1996;138(1-2):1-13.
    • (1996) J Neurol Sci , vol.138 , Issue.1-2 , pp. 1-13
    • McDougall, A.J.1    McLeod, J.G.2
  • 22
    • 0023704327 scopus 로고
    • Discrimination of peripheral polyneuropathies caused by TTR variant or diabetes in the same pedigree through protein studies
    • 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.
    • (1988) Adv Neurol , vol.48 , pp. 201-208
    • Ferlini, A.1    Romeo, G.2    Tassinari, C.A.3    Saraiva, M.J.4    Costa, P.P.5    Salvi, F.6
  • 23
    • 0029112095 scopus 로고
    • Diabetic polyneuropathy in controlled clinical trials: Consensus Report of the Peripheral Nerve Society
    • Diabetic polyneuropathy in controlled clinical trials: Consensus Report of the Peripheral Nerve Society. Ann Neurol. 1995;38(3):478-482.
    • (1995) Ann Neurol , vol.38 , Issue.3 , pp. 478-482
  • 24
    • 0034328085 scopus 로고    scopus 로고
    • Efficacy and safety of recombinant human nerve growth factor in patients with diabetic polyneuropathy: A randomized controlled trial
    • rhNGF Clinical Investigator Group
    • 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.
    • (2000) JAMA , vol.284 , Issue.17 , pp. 2215-2221
    • Apfel, S.C.1    Schwartz, S.2    Adornato, B.T.3
  • 25
    • 0023277450 scopus 로고
    • Vibratory and cooling detection thresholds compared with other tests in diagnosing and staging diabetic neuropathy
    • 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.
    • (1987) Diabetes Care , vol.10 , Issue.4 , pp. 432-440
    • Dyck, P.J.1    Bushek, W.2    Spring, E.M.3
  • 26
    • 0020576641 scopus 로고
    • Effect of blood glucose control on peripheral nerve function in diabetic patients
    • 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.
    • (1983) Mayo Clin Proc , vol.58 , Issue.5 , pp. 283-289
    • Service, F.J.1    Daube, J.R.2    O'Brien, P.C.3
  • 27
    • 33845499323 scopus 로고    scopus 로고
    • Oral treatment with alpha-lipoic acid improves symptomatic diabetic polyneuropathy: The SYDNEY 2 trial
    • 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.
    • (2006) Diabetes Care , vol.29 , Issue.11 , pp. 2365-2370
    • Ziegler, D.1    Ametov, A.2    Barinov, A.3
  • 28
    • 0026052403 scopus 로고
    • Plasma exchange in polyneuropathy associated with monoclonal gammopathy of undetermined significance
    • 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.
    • (1991) N Engl J Med , vol.325 , Issue.21 , pp. 1482-1486
    • Dyck, P.J.1    Low, P.A.2    Windebank, A.J.3
  • 29
    • 0022613235 scopus 로고
    • Plasma exchange in chronic inflammatory demyelinating polyradiculoneuropathy
    • 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.
    • (1986) N Engl J Med , vol.314 , Issue.8 , pp. 461-465
    • Dyck, P.J.1    Daube, J.2    O'Brien, P.3
  • 30
    • 0028080407 scopus 로고
    • A plasma exchange vs immune globulin infusion trial in chronic inflammatory demyelinating polyradiculoneuropathy
    • 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.
    • (1994) Ann Neurol , vol.36 , Issue.6 , pp. 838-845
    • Dyck, P.J.1    Litchy, W.J.2    Kratz, K.M.3
  • 31
    • 0029073983 scopus 로고
    • Variables influencing neuropathic endpoints: The Rochester Diabetic Neuropathy Study of Healthy Subjects
    • 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.
    • (1995) Neurology , vol.45 , Issue.6 , pp. 1115-1121
    • Dyck, P.J.1    Litchy, W.J.2    Lehman, K.A.3    Hokanson, J.L.4    Low, P.A.5    O'Brien, P.C.6
  • 32
    • 84871246506 scopus 로고    scopus 로고
    • "Unequivocally abnormal" vs "usual" signs and symptoms for proficient diagnosis of diabetic polyneuropathy: Cl vs N Phys Trial
    • 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.
    • (2012) Arch Neurol , vol.69 , Issue.12 , pp. 1609-1614
    • Dyck, P.J.1    Overland, C.J.2    Low, P.A.3
  • 33
    • 4444363999 scopus 로고    scopus 로고
    • Improvement in quality of life of patients with AL amyloidosis treated with high-dose melphalan and autologous stem cell transplantation
    • 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.
    • (2004) Blood , vol.104 , Issue.6 , pp. 1888-1893
    • Seldin, D.C.1    Anderson, J.J.2    Sanchorawala, V.3
  • 34
    • 0028357375 scopus 로고
    • Malnutrition and gastrointestinal dysfunction as prognostic factors for survival in familial amyloidotic polyneuropathy
    • 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.
    • (1994) J Intern Med , vol.235 , Issue.5 , pp. 479-485
    • Suhr, O.1    Danielsson, A.2    Holmgren, G.3    Steen, L.4
  • 35
    • 0028788659 scopus 로고
    • Liver transplantation in familial amyloidotic polyneuropathy: Follow-up of the first 20 Swedish patients
    • 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.
    • (1995) Transplantation , vol.60 , Issue.9 , pp. 933-938
    • Suhr, O.B.1    Holmgren, G.2    Steen, L.3
  • 36
    • 0032724681 scopus 로고    scopus 로고
    • Outcome of liver transplantation for transthyretin amyloidosis: Follow-up of Japanese familial amyloidotic polyneuropathy patients
    • 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.
    • (1999) J Neurol Sci , vol.171 , Issue.1 , pp. 19-23
    • Tashima, K.1    Ando, Y.2    Terazaki, H.3


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.