Long-term safety and efficacy of tafamidis for the treatment of hereditary transthyretin amyloid polyneuropathy: results up to 6 years

Amyloid

Volumn 24, Issue 3, 2017, Pages 194-204

  Barroso, Fabio A ^a   Judge, Daniel P ^b   Ebede, Ben ^c   Li, Huihua ^c   Stewart, Michelle ^c   Amass, Leslie ^c   Sultan, Marla B ^c  

^a FLENI   (Argentina)

^b JOHNS HOPKINS UNIVERSITY   (United States)

^c PFIZER INC   (United States)

Author keywords

Amyloidosis; ATTRV30M; disease modifying drug; non ATTRV30M

Indexed keywords

GAMMA GLUTAMYLTRANSFERASE; PLACEBO; PREALBUMIN; PROTHROMBIN; TAFAMIDIS; THYROTROPIN; BENZOXAZOLE DERIVATIVE;

ADULT; ARTICLE; CONSTIPATION; CONTROLLED STUDY; DISEASE COURSE; DOUBLE BLIND PROCEDURE; DRUG EFFICACY; DRUG SAFETY; DRUG TOLERABILITY; DRUG WITHDRAWAL; FAMILIAL AMYLOID POLYNEUROPATHY; FECES INCONTINENCE; FEMALE; GAMMA GLUTAMYL TRANSFERASE BLOOD LEVEL; GASTROINTESTINAL DISEASE; GENERAL CONDITION DETERIORATION; HEADACHE; HEART DISEASE; HEART FAILURE; HUMAN; KIDNEY DISEASE; KIDNEY FAILURE; LONG TERM CARE; LYMPHOCYTE COUNT; MAJOR CLINICAL STUDY; MALE; MIDDLE AGED; NEUTROPHIL COUNT; OPEN STUDY; PERICARDIAL EFFUSION; PERIPHERAL EDEMA; PHASE 3 CLINICAL TRIAL; PLEURA EFFUSION; PRIORITY JOURNAL; PROSPECTIVE STUDY; PROTHROMBIN TIME; QT INTERVAL; QUALITY OF LIFE; RANDOMIZED CONTROLLED TRIAL; SEPSIS; SIDE EFFECT; THORAX PAIN; THYROTROPIN BLOOD LEVEL; TRANSIENT ISCHEMIC ATTACK; TREATMENT DURATION; TREATMENT OUTCOME; UREA NITROGEN BLOOD LEVEL; URINARY TRACT INFECTION; AGED; CLINICAL TRIAL; LONGITUDINAL STUDY; MULTICENTER STUDY;

ADULT; AGED; AMYLOID NEUROPATHIES, FAMILIAL; BENZOXAZOLES; FEMALE; HUMANS; LONGITUDINAL STUDIES; MALE; MIDDLE AGED; QUALITY OF LIFE;

EID: 85026547544     PISSN: 13506129     EISSN: 17442818     Source Type: Journal    
DOI: 10.1080/13506129.2017.1357545     Document Type: Article

References (37)

1. Ando Y, Coelho T, Berk JL, et al. Guideline of transthyretin-related hereditary amyloidosis for clinicians. Orphanet J Rare Dis. 2013;8:31–49.
2. Sekijima Y., Transthyretin (ATTR) amyloidosis:clinical spectrum, molecular pathogenesis and disease-modifying treatments. J Neurol Neurosurg Psychiatry. 2015;86:1036–1043.
3. Coelho T, Maurer MS, Suhr OB., THAOS - The Transthyretin Amyloidosis Outcomes Survey:initial report on clinical manifestations in patients with hereditary and wild-type transthyretin amyloidosis. Curr Med Res Opin. 2013;29:63–76.
4. Adams D, Coelho T, Obici L, et al. Rapid progression of familial amyloidotic polyneuropathy:a multinational natural history study. Neurology. 2015;85:675–682.
5. Mariani LL, Lozeron P, Théaudin M, et al. Genotype-phenotype correlation and course of transthyretin familial amyloid polyneuropathies in France. Ann Neurol. 2015;78:901–916.
6. Rowczenio DM, Noor I, Gillmore JD, et al. Online registry for mutations in hereditary amyloidosis including nomenclature recommendations. Hum Mutat. 2014;35:E2403–24E2412.
7. Plante-Bordeneuve V., Update in the diagnosis and management of transthyretin familial amyloid polyneuropathy. J Neurol. 2014;261:1227–1233.
8. Adams D, Théaudin M, Cauquil C, et al. FAP neuropathy and emerging treatments. Curr Neurol Neurosci Rep. 2014;14:1–12.
9. Conceição IM, González-Duarte A, Obici L, et al. Red-flag symptom clusters in transthyretin familial amyloid polyneuropathy”. J Peripher Nerv Syst. 2016;21:5–9.
10. Schmidt H, Waddington-Cruz M, Botteman MF, et al. Estimating the global prevalence of transthyretin familial amyloid polyneuropathy. Muscle Nerve. 2017; submitted
11. Azoulay D, Samuel D, Castaing D, et al. Domino liver transplants for metabolic disorders:experience with familial amyloidotic polyneuropathy. J Am Coll Surg. 1999;189:584–593.
12. Ericzon BG, Wilczek HE, Larsson M, et al. Liver transplantation for hereditary transthyretin amyloidosis:after 20 years still the best therapeutic alternative? Transplantation. 2015;99:1847–1854.
13. Suhr OB, Larsson M, Ericzon BG, et al. Survival after transplantation in patients with mutations other than Val30Met:extracts from the FAP World Transplant Registry. Transplantation. 2016;100:373–381.
14. Adams D., Recent advances in the treatment of familial amyloid polyneuropathy. Ther Adv Neurol Disord. 2013;6:129–139.
15. Berk JL, Suhr OB, Obici L, et al. Repurposing diflunisal for familial amyloid polyneuropathy:a randomized clinical trial. JAMA. 2013;310:2658–2667.
16. MedlinePlus. Diflunisal. [Internet]. Bethesda (MD):US National Library of Medicine; 2015 Sep 15 [cited 2017 July 7]. Available from:https://www.nlm.nih.gov/medlineplus/druginfo/meds/a684037.html.
17. European Medicines Agency. Vyndaqel (tafamidis):European Public Assessment Report (EPAR). [Internet]. London (England):European Medicines Agency; 2016 Feb 8 [cited 2017 July 7]. Available from:http://www.ema.europa.eu/ema/index.jsp?curl=pages/medicines/human/medicines/002294/human_med_001498.jsp&mid=WC0b01ac058001d124.
18. Japan Pharmaceuticals and Medical Devices Agency. Vyndaqel (tafamidis):report on the deliberation results of the regulatory review. [Internet]. Tokyo (Japan):Evaluation and Licensing Division, Pharmaceutical and Food Safety Bureau, Ministry of Health, Labour and Welfare 22 August 2013 [cited 2017 July 7]. Available from:http://www.pmda.go.jp/files/000153750.pdf#page=2&r=s&r=s.
19. Waddington Cruz M, Benson MD., A review of tafamidis for the treatment of transthyretin-related amyloidosis. Neurol Ther. 2015;4:61–79.
20. Coelho T, Merlini G, Bulawa CE, et al. Mechanism of action and clinical application of tafamidis in hereditary transthyretin amyloidosis. Neurol Ther. 2016;5:1–25.
21. Coelho T, Maia LF, Martins da Silva A, et al. Tafamidis for transthyretin familial amyloid polyneuropathy:a randomized, controlled trial. Neurology 2012;79:785–792.
22. Coelho T, Maia LF, da Silva AM, et al. Long-term effects of tafamidis for the treatment of transthyretin familial amyloid polyneuropathy. J Neurol. 2013;260:2802–2814.
23. Waddington Cruz M, Amass L, Keohane D, et al. Early intervention with tafamidis provides long-term (5.5-year) delay of neurologic progression in transthyretin hereditary amyloid polyneuropathy. Amyloid 2016;23:178–183.
24. Merlini G, Plante-Bordeneuve V, Judge DP, et al. Effects of tafamidis on transthyretin stabilization and clinical outcomes in patients with non-Val30Met transthyretin amyloidosis. J Cardiovasc Trans Res. 2013;6:1011–1020.
25. Dyck PJ, Litchy WJ, Lehman KA, et al. Variables influencing neuropathic endpoints:the Rochester Diabetic Neuropathy Study of Healthy Subjects. Neurology 1995;45:1115–1121.
26. Vinik EJ, Hayes RP, Oglesby A, et al. The development and validation of the Norfolk QOL-DN, a new measure of patients' perception of the effects of diabetes and diabetic neuropathy. Diabetes Technol Ther. 2005;7:497–508.
27. Vinik EJ, Vinik AI, Paulson JF, et al. Norfolk QOL-DN:validation of a patient reported outcome measure in transthyretin familial amyloid polyneuropathy. J Peripher Nerv Syst. 2014;19:104–114.
28. Suhr O, Danielsson A, Holmgren G, et al. Malnutrition and gastrointestinal dysfunction as prognostic factors for survival in familial amyloidotic polyneuropathy. J Intern Med. 1994;235479–485.
29. Coelho T, Vinik A, Vinik EJ, et al. Clinical measures in transthyretin familial amyloid polyneuropathy. Muscle Nerve. 2017;55:323–332.
30. Coutinho P, Martins da Silva A, Lopes Lima J, et al. Forty years of experience with type I amyloid neuropathy. Review of 483 cases. In:Glenner GG, Pinho e Costa P, Falcao de Freitas A, editors. Amyloid and amyloidosis. Amsterdam (Netherlands):Excerpta Medica; 1980. p. 88–98.
31. D'Agostino RB., Sr.The delayed-start study design. N Engl J Med. 2009;361:1304–1306.
32. Liu-Seifert H, Andersen SW, Lipkovich I, et al. A novel approach to delayed-start analyses for demonstrating disease-modifying effects in Alzheimer's disease. PLoS One. 2015;10:1–16.
33. Suhr OB, Conceicao IM, Karayal ON, et al. Post hoc analysis of nutritional status in patients with transthyretin familial amyloid polyneuropathy:impact of tafamidis. Neurol Ther. 2014;3:101–112.
34. Li H, Gundapaneni B, Schwartz J, et al. Impact of baseline neurologic score on disease progression in transthyretin familial amyloid polyneuropathy. International Society of Amyloidosis–XV International Symposium on Amyloidosis; 3–7 July 2016; Uppsala, Sweden, Abstract No. PB2, p. 222.
35. Cortese A, Vita G, Luigetti M, et al. Monitoring effectiveness and safety of Tafamidis in transthyretin amyloidosis in Italy:a longitudinal multicenter study in a non-endemic area. J Neurol. 2016;263:916–924.
36. Plante-Bordeneuve V, Gorram F, Salhi H, et al. Long-term treatment of transthyretin familial amyloid polyneuropathy with tafamidis:a clinical and neurophysiological study. J Neurol. 2017;264:268–276.
37. Ando Y, Sekijima Y, Obayashi K, et al. Effects of tafamidis treatment on transthyretin (TTR) stabilization, efficacy, and safety in Japanese patients with familial amyloid polyneuropathy (TTR-FAP) with Val30Met and non-Val30Met:a phase III, open-label study. J Neurol Sci. 2016;362:266–271.