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REVIEW

Practical Guidance for the Use of Patisiran in the Management of Polyneuropathy in Hereditary Transthyretin-Mediated Amyloidosis

, &
Pages 973-981 | Received 05 Aug 2023, Accepted 13 Nov 2023, Published online: 26 Nov 2023

References

  • Ando Y, Adams D, Benson MD, et al. Guidelines and new directions in the therapy and monitoring of ATTRv amyloidosis. Amyloid. 2022;29(3):143–155. doi:10.1080/13506129.2022.2052838
  • Ines M, Coelho T, Conceicao I, Duarte-Ramos F, de Carvalho M, Costa J. Epidemiology of transthyretin familial amyloid polyneuropathy in Portugal: a nationwide study. Neuroepidemiology. 2018;51(3–4):177–182. doi:10.1159/000490553
  • Agbor-Etang BB, Okafor HE, Farber-Eger EH, Wells QS. Low prevalence of clinically apparent cardiac amyloidosis among carriers of transthyretin V122I variant in a large electronic medical record. Am J Med. 2021;134(2):e98–e100. doi:10.1016/j.amjmed.2020.06.031
  • Kozlitina J, Garg S, Drazner MH, et al. Clinical implications of the amyloidogenic V122I transthyretin variant in the general population. J Card Fail. 2022;28(3):403–414. doi:10.1016/j.cardfail.2021.09.015
  • 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. doi:10.1093/brain/75.3.408
  • Holmgren G, Costa PM, Andersson C, et al. Geographical distribution of TTR met30 carriers in northern Sweden: discrepancy between carrier frequency and prevalence rate. J Med Genet. 1994;31(5):351–354. doi:10.1136/jmg.31.5.351
  • 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. doi:10.1185/03007995.2012.754348
  • Ueda M, Yamashita T, Misumi Y, Masuda T, Ando Y. Origin of sporadic late-onset hereditary ATTR Val30Met amyloidosis in Japan. Amyloid. 2018;25(3):143–147. doi:10.1080/13506129.2018.1531842
  • Dispenzieri A, Coelho T, Conceicao I, et al. Clinical and genetic profile of patients enrolled in the Transthyretin Amyloidosis Outcomes Survey (THAOS): 14-year update. Orphanet J Rare Dis. 2022;17(1):236. doi:10.1186/s13023-022-02359-w
  • Benson MD, Teague SD, Kovacs R, Feigenbaum H, Jung J, Kincaid JC. Rate of progression of transthyretin amyloidosis. Am J Cardiol. 2011;108(2):285–289. doi:10.1016/j.amjcard.2011.03.040
  • 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. doi:10.1212/WNL.0000000000001870
  • Carroll A, Dyck PJ, de Carvalho M, et al. Novel approaches to diagnosis and management of hereditary transthyretin amyloidosis. J Neurol Neurosurg Psychiatry. 2022;93(6):668–678. doi:10.1136/jnnp-2021-327909
  • Madhani A, Sabogal N, Massillon D, et al. Clinical penetrance of the transthyretin V122I variant in older black patients with heart failure: the SCAN-MP (screening for cardiac amyloidosis with nuclear imaging in minority populations) study. J Am Heart Assoc. 2023;12(15):e028973. doi:10.1161/JAHA.122.028973
  • Stangou AJ, Hawkins PN, Heaton ND, et al. Progressive cardiac amyloidosis following liver transplantation for familial amyloid polyneuropathy: implications for amyloid fibrillogenesis. Transplantation. 1998;66(2):229–233. doi:10.1097/00007890-199807270-00016
  • Berk JL, Suhr OB, Obici L, et al. Repurposing diflunisal for familial amyloid polyneuropathy: a randomized clinical trial. JAMA. 2013;310(24):2658–2667. doi:10.1001/jama.2013.283815
  • 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. doi:10.1212/WNL.0b013e3182661eb1
  • Maurer MS, Schwartz JH, Gundapaneni B, et al. Tafamidis treatment for patients with transthyretin amyloid cardiomyopathy. N Engl J Med. 2018;379(11):1007–1016. doi:10.1056/NEJMoa1805689
  • Pederson T. Obituary: paul C. Zamecnik (1912–2009). Nature. 2009;462(7272):423. doi:10.1038/462423a
  • Stephenson ML, Zamecnik PC. Inhibition of Rous sarcoma viral RNA translation by a specific oligodeoxyribonucleotide. Proc Natl Acad Sci U S A. 1978;75(1):285–288. doi:10.1073/pnas.75.1.285
  • Caruthers MH. Gene synthesis machines: DNA chemistry and its uses. Science. 1985;230(4723):281–285. doi:10.1126/science.3863253
  • Bennett CF. Therapeutic antisense oligonucleotides are coming of age. Annu Rev Med. 2019;70:307–321. doi:10.1146/annurev-med-041217-010829
  • Crooke ST, Baker BF, Crooke RM, Liang XH. Antisense technology: an overview and prospectus. Nat Rev Drug Discov. 2021;20(6):427–453. doi:10.1038/s41573-021-00162-z
  • Chi X, Gatti P, Papoian T. Safety of antisense oligonucleotide and siRNA-based therapeutics. Drug Discov Today. 2017;22(5):823–833. doi:10.1016/j.drudis.2017.01.013
  • Urits I, Swanson D, Swett MC, et al. A review of patisiran (ONPATTRO(R)) for the treatment of polyneuropathy in people with hereditary transthyretin amyloidosis. Neurol Ther. 2020;9(2):301–315. doi:10.1007/s40120-020-00208-1
  • Food and Drug Administration 2018. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/label/2018/210922s000lbl.pdf. ONPATTRO-Prescribing Information.
  • Akinc A, Maier MA, Manoharan M, et al. The Onpattro story and the clinical translation of nanomedicines containing nucleic acid-based drugs. Nat Nanotechnol. 2019;14(12):1084–1087. doi:10.1038/s41565-019-0591-y
  • Suzuki Y, Ishihara H. Difference in the lipid nanoparticle technology employed in three approved siRNA (Patisiran) and mRNA (COVID-19 vaccine) drugs. Drug Metab Pharmacokinet. 2021;41:100424. doi:10.1016/j.dmpk.2021.100424
  • 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. doi:10.1056/NEJMoa1716153
  • Coelho T, Adams D, Silva A, et al. Safety and efficacy of RNAi therapy for transthyretin amyloidosis. N Engl J Med. 2013;369(9):819–829. doi:10.1056/NEJMoa1208760
  • Kozma GT, Shimizu T, Ishida T, Szebeni J. Anti-PEG antibodies: properties, formation, testing and role in adverse immune reactions to PEGylated nano-biopharmaceuticals. Adv Drug Deliv Rev. 2020;154–155:163–175. doi:10.1016/j.addr.2020.07.024
  • Suzuki T, Suzuki Y, Hihara T, et al. PEG shedding-rate-dependent blood clearance of PEGylated lipid nanoparticles in mice: faster PEG shedding attenuates anti-PEG IgM production. Int J Pharm. 2020;588:119792. doi:10.1016/j.ijpharm.2020.119792
  • Zhang X, Goel V, Attarwala H, Sweetser MT, Clausen VA, Robbie GJ. Patisiran pharmacokinetics, pharmacodynamics, and exposure-response analyses in the phase 3 APOLLO trial in patients with hereditary transthyretin-mediated (hATTR) amyloidosis. J Clin Pharmacol. 2020;60(1):37–49. doi:10.1002/jcph.1480
  • Suhr OB, Coelho T, Buades J, et al. Efficacy and safety of patisiran for familial amyloidotic polyneuropathy: a phase II multi-dose study. Orphanet J Rare Dis. 2015;10:109. doi:10.1186/s13023-015-0326-6
  • Coelho T, Adams D, Conceicao I, et al. A phase II, open-label, extension study of long-term patisiran treatment in patients with hereditary transthyretin-mediated (hATTR) amyloidosis. Orphanet J Rare Dis. 2020;15(1):179. doi:10.1186/s13023-020-01399-4
  • Dyck PJB, Gonzalez-Duarte A, Obici L, et al. Development of measures of polyneuropathy impairment in hATTR amyloidosis: from NIS to mNIS + 7. J Neurol Sci. 2019;405:116424. doi:10.1016/j.jns.2019.116424
  • Adams D, Polydefkis M, Gonzalez-Duarte A, 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. doi:10.1016/S1474-4422(20)30368-9
  • Lin H, Merkel M, Hale C, Marantz JL. Experience of patisiran with transthyretin stabilizers in patients with hereditary transthyretin-mediated amyloidosis. Neurodegener Dis Manag. 2020;10(5):289–300. doi:10.2217/nmt-2020-0020
  • European Medicine Agency 2018. Available from: https://www.ema.europa.eu/en/medicines/human/EPAR/onpattro. Onpattro-European public assessment report.
  • Solomon SD, Adams D, Kristen A, et al. Effects of Patisiran, an RNA interference therapeutic, on cardiac parameters in patients with hereditary transthyretin-mediated amyloidosis. Circulation. 2019;139(4):431–443. doi:10.1161/CIRCULATIONAHA.118.035831
  • National Library of Medicine. APOLLO-B: a study to evaluate patisiran in participants with transthyretin amyloidosis with cardiomyopathy (Attr amyloidosis with cardiomyopathy) 2019. Available from: https://clinicaltrials.gov/study/NCT03997383.
  • Yamamoto S, Wilczek HE, Nowak G, et al. Liver transplantation for familial amyloidotic polyneuropathy (FAP): a single-center experience over 16 years. Am J Transplant. 2007;7(11):2597–2604. doi:10.1111/j.1600-6143.2007.01969.x
  • Coutinho P. Amyloid and Amyloidosis. Excerpta Medica. Amsterdam. 1980;1980:1.
  • National Institutes of Health (NIH) Office of Dietary Supplements (ODS). Vitamin A and Carotenoids 2022. Available from: https://ods.od.nih.gov/factsheets/VitaminA-Consumer/.
  • Food and Drug Administration. List of pregnancy exposure registries; 2023. Available from: https://www.fda.gov/science-research/womens-health-research/list-pregnancy-exposure-registries. Accessed November 14, 2023.