Comparison between tafamidis and liver transplantation as first-line therapy for hereditary transthyretin amyloidosis

References

• Rapezzi C, Arbustini E, Caforio AL, et al. Diagnostic work-up in cardiomyopathies: bridging the gap between clinical phenotypes and final diagnosis. A position statement from the ESC Working Group on myocardial and pericardial diseases. Eur Heart J. 2013;34:1448–1458.
   PubMed Web of Science ®Google Scholar
• Rapezzi C, Quarta CC, Obici L, et al. Disease profile and differential diagnosis of hereditary transthyretin-related amyloidosis with exclusively cardiac phenotype: an Italian perspective. Eur Heart J [Internet]. 2012 [cited 2012 Oct 16]; Available from: http://eurheartj.oxfordjournals.org/content/early/2012/06/28/eurheartj.ehs123.
   Web of Science ®Google Scholar
• Adams D, Koike H, Slama M, et al. Hereditary transthyretin amyloidosis: a model of medical progress for a fatal disease. Nat Rev Neurol. 2019;15:387–404.
   PubMed Web of Science ®Google Scholar
• Adams D, Samuel D, Goulon-Goeau C, et al. The course and prognostic factors of familial amyloid polyneuropathy after liver transplantation. Brain J Neurol. 2000;123(Pt 7):1495–1504.
   Google Scholar
• Ericzon B-G, Wilczek HE, Larsson M, et al. Liver transplantation for hereditary transthyretin amyloidosis: after 20 years still the best therapeutic alternative? Transplantation. 2015;1.
   Google Scholar
• 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.
   PubMed Web of Science ®Google Scholar
• Benson MD, Waddington-Cruz M, Berk JL, et al. Inotersen treatment for patients with hereditary transthyretin Amyloidosis. N Engl J Med. 2018;379:22–31.
   PubMed Web of Science ®Google Scholar
• Adams D, Gonzalez-Duarte A, O’Riordan WD, et al. Patisiran, an RNAi therapeutic, for hereditary transthyretin amyloidosis. N Engl J Med. 2018;379:11–21.
   PubMed Web of Science ®Google Scholar
• Gillmore JD, Gane E, Taubel J, et al. CRISPR-Cas9 in vivo gene editing for transthyretin amyloidosis. N Engl J Med. 2021;385:493–502.
   PubMed Web of Science ®Google Scholar
• Ando Y, Coelho T, Berk JL, et al. Guideline of transthyretin-related hereditary amyloidosis for clinicians. Orphanet J Rare Dis. 2013;8:31.
   PubMed Web of Science ®Google Scholar
• Adams D, Suhr OB, Hund E, et al. First European consensus for diagnosis, management, and treatment of transthyretin familial amyloid polyneuropathy. Curr Opin Neurol. 2016;29:S14–S26.
   PubMed Web of Science ®Google Scholar
• Maurer MS, Schwartz JH, Gundapaneni B, et al. Tafamidis treatment for patients with transthyretin amyloid cardiomyopathy. N Engl J Med. 2018;379:1007–1016.
   PubMed Web of Science ®Google Scholar
• Gundapaneni BK, Sultan MB, Keohane DJ, et al. Tafamidis delays neurological progression comparably across Val30Met and non-Val30Met genotypes in transthyretin familial amyloid polyneuropathy. Eur J Neurol. 2018;25:464–468.
   PubMed Web of Science ®Google Scholar
• Barroso FA, Judge DP, Ebede B, et al. Long-term safety and efficacy of tafamidis for the treatment of hereditary transthyretin amyloid polyneuropathy: results up to 6 years. Amyloid Int J Exp Clin Investig Off J Int Soc Amyloidosis. 2017;24:194–204.
   PubMed Web of Science ®Google Scholar
• Sultan MB, Gundapaneni B, Schumacher J, et al. Treatment with tafamidis slows disease progression in early-stage transthyretin cardiomyopathy. Clin Med Insights Cardiol. 2017;11:1179546817730322.
   Web of Science ®Google Scholar
• Coelho T, Inês M, Conceição I, et al. Natural history and survival in stage 1 Val30Met transthyretin familial amyloid polyneuropathy. Neurology. 2018;91:e1999–e2009.
   PubMed Web of Science ®Google Scholar
• Rowczenio DM, Noor I, Gillmore JD, et al. Online registry for mutations in hereditary amyloidosis including nomenclature recommendations. Hum Mutat. 2014;35:E2403–E2412.
   PubMed Web of Science ®Google Scholar
• Ponikowski P, Voors AA, Anker SD, et al. 2016 ESC Guidelines for the diagnosis and treatment of acute and chronic heart failurethe task force for the diagnosis and treatment of acute and chronic heart failure of the European society of cardiology (ESC)developed with the special contribution of the heart failure association (HFA) of the ESC. Eur Heart J. 2016;37:2129–2200.
   PubMedGoogle Scholar
• Algalarrondo V, Antonini T, Théaudin M, et al. Cardiac dysautonomia predicts long-term survival in hereditary transthyretin amyloidosis after liver transplantation. JACC Cardiovasc Imaging. 2016;9:1432–1441.
   PubMed Web of Science ®Google Scholar
• Adams D, Coelho T, Obici L, et al. Rapid progression of familial amyloidotic polyneuropathy: a multinational natural history study. Neurology. 2015;85:675–682.
   PubMed Web of Science ®Google Scholar
• Denier C, Ducot B, Husson H, et al. A brief compound test for assessment of autonomic and sensory-motor dysfunction in familial amyloid polyneuropathy. J Neurol. 2007;254:1684–1688.
   PubMed Web of Science ®Google Scholar
• Consensus statement on indications for liver transplantation: Paris, June 22–23, 1993. Hepatol Baltim Md. 1994;20:63S–68S.
   Google Scholar
• Algalarrondo V, Antonini T, Théaudin M, et al. Cause of death analysis and temporal trends in survival after liver transplantation for transthyretin familial amyloid polyneuropathy. Amyloid Int J Exp Clin Investig Off J Int Soc Amyloidosis. 2018;25:253–260.
   PubMed Web of Science ®Google Scholar
• D’Agostino RB. Propensity score methods for bias reduction in the comparison of a treatment to a non-randomized control group. Stat Med. 1998;17:2265–2281.
   PubMed Web of Science ®Google Scholar
• D’Agostino RB, D’Agostino RB. Estimating treatment effects using observational data. JAMA. 2007;297:314–316.
   PubMed Web of Science ®Google Scholar
• Little RJ, Rubin DB. Causal effects in clinical and epidemiological studies via potential outcomes: concepts and analytical approaches. Annu Rev Public Health. 2000;21:121–145.
   PubMed Web of Science ®Google Scholar
• Klein JP, Moeschberger ML. Survival analysis: techniques for censored and truncated data. Springer Science & Business Media; 2005.
   Google Scholar
• Faries DE, Obenchain R, Haro JM, et al. Analysis of observational health care data using SAS. SAS Institute; 2014.
   Google Scholar
• Barlow WE. Robust variance estimation for the case-cohort design. Biometrics. 1994;1064–1072.
   Google Scholar
• Fine JP, Gray RJ. A proportional hazards model for the subdistribution of a competing risk. J Am Stat Assoc. 1999;94:496–509.
   Web of Science ®Google Scholar
• Algalarrondo V, Antonini T, Théaudin M, et al. Prediction of long-term survival after liver transplantation for familial transthyretin amyloidosis. J Am Coll Cardiol. 2015;66:2154–2156.
   PubMed Web of Science ®Google Scholar
• Bézard M, Kharoubi M, Galat A, et al. Natural history and impact of treatment with tafamidis on major cardiovascular outcome-free survival time in a cohort of patients with transthyretin amyloidosis. Eur J Heart Fail. 2021;23:264–274.
   PubMed Web of Science ®Google Scholar
• Rappley I, Monteiro C, Novais M, et al. Quantification of transthyretin kinetic stability in human plasma using subunit exchange. Biochemistry. 2014;53:1993–2006.
   PubMed Web of Science ®Google Scholar
• Cho Y, Baranczak A, Helmke S, et al. Personalized medicine approach for optimizing the dose of tafamidis to potentially ameliorate wild-type transthyretin amyloidosis (cardiomyopathy). Amyloid Int J Exp Clin Investig Off J Int Soc Amyloidosis. 2015;22:175–180.
   PubMed Web of Science ®Google Scholar
• Damy T, Garcia-Pavia P, Hanna M, et al. Efficacy and safety of tafamidis doses in the tafamidis in transthyretin cardiomyopathy clinical trial (ATTR-ACT) and long-term extension study. Eur J Heart Fail. 2021;23:277–285.
   PubMed Web of Science ®Google Scholar
• Beirao I, Lobato L, Costa PMP, et al. Kidney and anemia in familial amyloidosis type I. Kidney Int. 2004;66:2004–2009.
   PubMed Web of Science ®Google Scholar
• Asahara K, Ando Y, Tanaka Y, et al. Secondary hypoplastic anemia in patients with familial amyloidotic polyneuropathy. Acta Haematol. 1993;90:130–135.
   PubMed Web of Science ®Google Scholar
• Beirao I, Lobato L, Costa PMP, et al. Liver transplantation and anemia in familial amyloidosis ATTR V30M. Amyloid Int J Exp Clin Investig Off J Int Soc Amyloidosis. 2007;14:33–37.
   PubMed Web of Science ®Google Scholar
• Ikeda T, Masuda T, Ueda M, et al. Unwanted road to anaemia in transthyretin familial amyloid polyneuropathy may continue irrespective of tafamidis treatment. Ann Clin Biochem. 2018;4563218754587.
   Web of Science ®Google Scholar