Farmakoterapia w Psychiatrii i Neurologii

Pełna treść

2/2021 vol. 37
Artykuł przeglądowy

Perspektywy leczenia choroby Wilsona

Agnieszka Antos 1
,
Tomasz Litwin 1
,
Marta Skowrońska 1
,
Iwona Kurkowska-Jastrzębska 1
,
Anna Członkowska 1
  1. II Klinika Neurologii, Instytut Psychiatrii i Neurologii w Warszawie
Farmakoterapia w Psychiatrii i Neurologii 2021, 37 (2), 151–163
DOI: https://doi.org/10.33450/fpn.2021.06.004
Data publikacji online: 2021/12/07
Plik artykułu
04R_IPiN_FARM_2021-2C-2.pdf
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  1. Ala A, Aliu E, Schlisky ML. Prospective pilot study of single daily dose of trientine for the treatment of Wilson’s disease. Dig Dis Sci 2015; 60: 1433-1439.
  2. Beinhardt S, Leiss W, Stättermayer AF, Graziadei I, Zoller H, Stauber R et al. Long term outcomes of patients with Wilson disease in large Austrian cohort. Clin Gastroenetrol Hepatol 2014; 12: 683-689.
  3. Berzina A, Martinsone I, Svirskis S, Murovska M, Kalis M. Curcumin effect on copper transport in HepG2 cells. Medicina 2018; 54: 14.
  4. Brewer GJ, Terry CA, Aisen AM, Hill GM. Worsening of neurologic syndrome in patients with Wilson’s disease with initial penicillamine therapy. Arch Neurol 1987; 44: 490-493.
  5. Brewer G, Askari F, Lorincz MT, Carlson M, Schilsky ML, Kluin KJ et al. Treatment of Wilson disease with ammonium tetrathiomolybdate: IV. Comparision of tetrathiomolybdate and trientine in double-blind study of treatment of the neurologic presentation of Wilson disease. Arch Neurol 2006; 63: 521-527.
  6. Brewer G, Askari F, Dick RB, Sitterly J, Fink JK, Carlson M et al. Treatment of Wilson disease with tetrathiomolybdate: V. Control of free copper by tetrathiomolybdate and a comparison with trientine. Transl Res. 2009; 154(2): 70-77.
  7. Bruha R, Marecek Z, Pospisilova L, Nevsimalova S, Vitek L, Martasek P et al. Long-term follow-up of Wilson disease: natural history, treatment, mutations analysis and phenotypic correlation. Liver Int 2011: 31(1): 83-91.
  8. Chen S, Shao C, Dong T, Chai H, Xiong X, Sun D et al. Transplantation of ATP7B-transduced bone marrow mesenchymal stem cells decreases copper overload in rats. PlosOne 2014; 9(11): e111425.
  9. Czlonkowska A, Litwin T, Dusek P, Ferenci P, Lutsenko S, Medici V et al. Wilson Disease. Nat Rev Dis Primers 2018; 4(1): 21.
  10. Czlonkowska A, Tarnacka B, Litwin T, Gajda J, Rodo M. Wilson’s disease – cause of mortality in 164 patients during 1992-2003 observation period. J Neurol 2005: 252(6): 698-703.
  11. Czlonkowska A, Litwin T. Wilson disease – currently used anticopper therapy. W: Czlonkowska A, Schilsky ML (red.), Wilson Disease, Elsevier. Amsterdam 2017; 181-191.
  12. Czlonkowska A, Litwin T, Karliński M, Dziezyc K, Chabik G, Czerska M. D-penicillamine versus zinc sulfate s first-line therapy for Wilson’s Disease. Eur J Neurol 2014; 21: 599-606.
  13. European Association for Study of Liver. EASL Clinical Practice Guidelines: Wilson’s disease. J Hepatol 2012: 56(3): 671-685.
  14. Falcone E, Okafor M, Vitale N, Raibaut L, Sour A, Faller P. Extracellular Cu2+ pools and their detection: From current knowledge to next-generations probes. Coordination Chemistry Reviews 2021; 433(21).
  15. Farzaei MH, Zobeiri M, Parvizi F, El-Senduny FF, Marmouzi I, Coy-Barrera E et al. Curcumin in liver diseases: a systematic review of the cellular mechanisms of oxidative stress and clinical perspectives. Nutrients 2018; 10: 855.
  16. Ji HF, Shen L. Potential of curcumin as a multifunctional agents to combat Wilson disease. Hepatology 2010; 51(6): 2226.
  17. Kim B, Chung SJ, Shin HW. Trientine-induced neurological deterioration in patient with Wilson’s disease. J Clin Neurosci 2013; 20: 1398-1401.
  18. Krishnan N, Felice C, Rivera K, Pappin DJ, Tonks NK . DPM-1001 decreased copper levels and ameliorated deficits in a mouse model of Wilson’s disease. Genes Dev 2018; 32: 944-952.
  19. Lichtmannegger J, Leitzinger C, Wimmer R, Schmitt S, Schulz S, Kabiri Y et al. Methanobactin reverse acute liver failure in rat model of Wilson disease. J Clin Inv 2016; 126: 2721-2735.
  20. Litwin T, Dzieżyc K, Karliński M, Chabik G, Czepiel W, Czlonkowska A. Early neurological worsening in patients with Wilson’s Diseases. J Neurol Sci 2015; 355: 162-167.
  21. Litwin T, Dusek P, Czlonkowska A. Neurological manifestations in Wilson’s disease possible treatment options for symptoms. Expert Opin on Orphans Drugs 2016; 4(7): 719-728.
  22. Litwin T, Dzieżyc K, Czlonkowska A. Wilson disease-treatment perspectives. Ann Transl Med, 2019; Suppl 2; S68.
  23. Masełbas W, Czlonkowska A, Litwin T, Niewada M. Persistence with treatment for Wilson disease: a retrospective study. BMC Neurology 2019; 19: 278.
  24. Merle U, Encke J, Tuma S, Volkmann M, Naldini L, Stremmel W. Lentiviral gene transfer ameliorates disease progression in Long-Evans cinnamon rats: an animal model for Wilson disease. Scand J Gastroenterol 2006; 12: 2239-2242.
  25. Murillo O, Luqui DM, Gazquez C, Martinez-Espartosa D, Navarro-Blasco I, Monreal JI et al. Long-term correction of Wilson’s disease in a murine model by gene therapy. J Hepatol 2016; 64: 419-426.
  26. Park SM, Vo K, Lallier M, Cloutier AS, Brochu P, Alvarez F, et al. Hepatocyte transplantation in the Long Evans Cinnamon rat model of Wilson’s disease. Cell Transplantation 2006; 15: 13-22.
  27. Peña-Quintana L, Llarena M, Reyes-Suárez D, Aldámiz-Echevarria L . Profile of sodium phenylbutyrate granules for the treatment of urea-cycle disorders: patient perspectives. Patient Prefer Adherence 2017; 11: 1489-1496.
  28. Poujois A, Trocello JM, Djebrani-Oussedik N, Poupon J, Collet C, Girardot-Tinant N et al. Exchangeable copper: a reflection of the neurological severity in Wilson’s disease. Eur J Neurol 2017; 24: 154-160.
  29. Pujol M, Cuillel M, Renaudet O, Lebrun C, Charbonnier P, Cassio D et al. Hepatocyte targeting and intracellular copper chelation by a thiol-containing glycocyclopeptide. J Am Chem Soc 2011; 133: 286-296.
  30. Roberts E, Schilsky M, American Association for Study of Liver Diseases (AASLD). Diagnosis and treatment of Wilson’s disease an update. AASLD Practice Guidelines. Hepatology 2008; 47: 2089-2111.
  31. Roy-Chowdhury J, Schilsky ML. Gene therapy of Wilson disease: a „golden” opportunity using rAAV on the 50th anniversary of the discovery of the virus. J Hepatol 2016; 64: 265-267.
  32. Rupp C, Weiss KH. Tetrathiomolibdate. W: Weis KH, Schilsky M (red.), Wilson disease. Pathogenesis, molecular mechanism, diagnosis, treatment and monitoring. 2019, Elsevier, 197-201.
  33. Sauer V, Siaj R, Stöppeler S, Bahde R, Spiegel HU, Köhler G et al. Repeated transplantation of hepatocytes prevents fulminant hepatitis in a rat model of Wilson’s disease. Liver Trnsplantation 2012; 18: 248-259.
  34. Schilsky ML. Long-term outcome for Wilson disease: 85% good. Clin Gastroenterol Hepatol 2014; 12: 690-691.
  35. Spincemaille P, Pham DH, Chandhok G, Verbeek J, Zibert A, Libbrecht L et al. The plant decapeptide OSIP108 prevents copper-induced toxicity in various models for Wilson disease. Toxicology and Applied Pharmacology 2014; 280: 345-351.
  36. Svetel M, Pekmezović T, Petrović I, Tomić A, Kresojević N, Jesić R et al. Long-term outcome in Serbian patients with Wilson disease. Eur J Neurol 2009; 16: 852-857.
  37. Tremmel R, Uhl P, Helm F, Wupperfeld D, Sauter M, Mier W et al. Delivery of copper-chelating trientine (TETA) to the central nervous system by surface modified liposomes. International Journal of Pharmaceutics 2016; 512: 87-95.
  38. Uerlings R, Moreno D, Murillo O, Gazquez C, Hernández-Alcoceba R, González-Aseguinolaza G et al. Brain copper storage after genetic long-term correction in a mouse model of Wilson disease. Neurol Genet 2018; 4(3):e243.
  39. Weiss KH, Stremmel W. Clinical considerations for an effective medical therapy in Wilson’s disease. Ann N Y Sci 2014; 1315: 81-85.
  40. Weiss KH, Askari FK, Czlonkowska A, Ferenci P, Bronstein JM, Bega D et al. Bis-choline tetrathiomolybdate in patients with Wilson’s disease: an open label, multicentre, phase 2 study. Lancet Gastroenterol Hepatol 2017; 2: 869-876.
  41. Van den Berghe PVE, Stapelbroek JM, Kriegera E, de Bie P, van de Graaf SFJ, de Groot REA et al. Reduced expression of ATP7B affected by Wilson disease-causing mutations is rescued by pharmacological folding chaperones 4-phenylbutrate and curcumin. Hepatology 2009; 50: 1783-1795.
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