Impact of baseline polyneuropathy severity on patisiran treatment outcomes in the APOLLO trial

References

• Hanna M. Novel drugs targeting transthyretin amyloidosis. Curr Heart Fail Rep. 2014;11(1):50–57.
   PubMedGoogle Scholar
• Mohty D, Damy T, Cosnay P, et al. Cardiac amyloidosis: updates in diagnosis and management. Arch Cardiovasc Dis. 2013;106(10):528–540.
   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(8):675–682.
   PubMed Web of Science ®Google Scholar
• Hawkins PN, Ando Y, Dispenzeri A, et al. Evolving landscape in the management of transthyretin amyloidosis. Ann Med. 2015;47(8):625–638.
   PubMed Web of Science ®Google Scholar
• Damy T, Judge DP, Kristen AV, et al. Cardiac findings and events observed in an open-label clinical trial of tafamidis in patients with non-Val30Met and non-Val122Ile hereditary transthyretin amyloidosis. J Cardiovasc Trans Res. 2015;8(2):117–127.
   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. 2013;34(7):520–528.
   PubMed Web of Science ®Google Scholar
• 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(1):63–76.
   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(1):11–21.
   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(1):22–31.
   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
• Gonzalez-Duarte A. Autonomic involvement in hereditary transthyretin amyloidosis (hATTR amyloidosis). Clin Auton Res. 2019;29(2):245–251.
   PubMed Web of Science ®Google Scholar
• Shin SC, Robinson-Papp J. Amyloid neuropathies. Mt Sinai J Med. 2012;79(6):733–748.
   PubMedGoogle Scholar
• Amyloidosis Research Consortium. The voice of the patient report – amyloidosis. 2016. Available from: https://www.arci.org/wp-content/uploads/2018/05/Voice-of-the-Patient.pdf, accessed July 22, 2022.
   Google Scholar
• Duncan D. With hope for a cure. 2018. Available from: http://amyloidosis.org/proactive-3/, accessed July 22, 2022.
   Google Scholar
• Mariani LL, Lozeron P, Theaudin M, French Familial Amyloid Polyneuropathies Network (CORNAMYL) Study Group, et al. Genotype-phenotype correlation and course of transthyretin familial amyloid polyneuropathies in France. Ann Neurol. 2015;78(6):901–916.
   PubMed Web of Science ®Google Scholar
• Adams D. Recent advances in the treatment of familial amyloid polyneuropathy. Ther Adv Neurol Disord. 2013;6(2):129–139.
   PubMed Web of Science ®Google Scholar
• Mitchell PM, Al-Janabi H, Richardson J, et al. The relative impacts of disease on health status and capability wellbeing: a multi-country study. PLoS One. 2015;10(12):e0143590.
   PubMed 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(7):387–404.
   PubMed Web of Science ®Google Scholar
• swissmedic. Abbreviated information for health care professionals for ONPATTRO 10 mg/5 ml, concentrate for solution for infusion (version September 2019). 2022. Available from: www.swissmedicinfo.ch, accessed July 22, 2022.
   Google Scholar
• European Medicines Agency. Summary of product characteristics: Onpattro 2 mg/mL concentrate for solution for infusion. 2018. Available from: https://www.ema.europa.eu/documents/product-information/onpattro-epar-product-information_en.pdf, accessed July 22, 2022.
   Google Scholar
• Alnylam announces approval in Brazil of ONPATTRO® for the treatment of hereditary ATTR amyloidosis with polyneuropathy [Internet]. Alnylam Pharmaceuticals Inc. 2020. Available from: https://investors.alnylam.com/press-release?id=24606, accessed July 22, 2022.
   Google Scholar
• Alnylam announces approval in Japan of ONPATTRO® for the treatment of hereditary ATTR amyloidosis with polyneuropathy [Internet]. Alnylam Pharmaceuticals Inc,; 2019. Available from: https://investors.alnylam.com/press-release?id=23886, accessed July 22, 2022.
   Google Scholar
• Alnylam Pharmaceuticals Inc. US prescribing information: ONPATTRO (patisiran) lipid complex injection, for intravenous use. 2020. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/label/2020/210922s007lbl.pdf, accessed July 22, 2022.
   Google Scholar
• CADTH. Patisiran. 2019. Available from: https://www.cadth.ca/patisiran, accessed July 22, 2022.
   Google Scholar
• Adams D, Suhr OB, Dyck PJ, et al. Trial design and rationale for APOLLO, a phase 3, placebo-controlled study of patisiran in patients with hereditary ATTR amyloidosis with polyneuropathy. BMC Neurol. 2017;17(1):181.
   PubMed Web of Science ®Google Scholar
• Suanprasert N, Berk JL, Benson MD, et al. Retrospective study of a TTR FAP cohort to modify NIS+7 for therapeutic trials. J Neurol Sci. 2014;344(1-2):121–128.
   PubMed Web of Science ®Google Scholar
• van Nes SI, Vanhoutte EK, van Doorn PA, et al. Rasch-built Overall Disability Scale (R-ODS) for immune-mediated peripheral neuropathies. Neurology. 2011;76(4):337–345.
   PubMed Web of Science ®Google Scholar
• 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(2):104–114.
   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(3):464–468.
   PubMed Web of Science ®Google Scholar
• Waddington-Cruz M, Ackermann EJ, Polydefkis M, et al. Hereditary transthyretin amyloidosis: baseline characteristics of patients in the NEURO-TTR trial. Amyloid. 2018;25(3):180–188.
   PubMed Web of Science ®Google Scholar
• Veresiu AI, Bondor CI, Florea B, et al. Detection of undisclosed neuropathy and assessment of its impact on quality of life: a survey in 25,000 Romanian patients with diabetes. J Diabetes Complications. 2015;29(5):644–649.
   PubMed Web of Science ®Google Scholar
• Adams D, Polydefkis M, González-Duarte A, patisiran Global OLE study group, et al. Long-term safety and efficacy of patisiran for hereditary transthyretin-mediated amyloidosis with polyneuropathy: 12-month results of an open-label extension study. Lancet Neurol. 2021;20(1):49–59.
   PubMed Web of Science ®Google Scholar
• Adams D, Gonzalez Duarte A, Mauricio E, et al. Global open-label extension: 24-month data of patisiran in patients with hATTR amyloidosis. Peripheral Nerve Society (PNS) Congress; June 27–30 2020. Miami, FL, USA.
   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. 2017;24(3):194–204.
   PubMed Web of Science ®Google Scholar
• Brannagan TH, Wang AK, Coelho T, NEURO-TTR open-label extension investigators, et al. Early data on long-term efficacy and safety of inotersen in patients with hereditary transthyretin amyloidosis: a 2-year update from the open-label extension of the NEURO-TTR trial. Eur J Neurol. 2020;27(8):1374–1381.
   PubMed Web of Science ®Google Scholar