Teryflunomid jako złoty standard w badaniach klinicznych w stwardnieniu rozsianym Artykuł przeglądowy
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Opublikowane:
cze 30, 2022
Słowa kluczowe:
stwardnienie rozsiane, teryflunomid, badanie kliniczne, komparator, placebo
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Abstrakt
W artykule przedstawiono wybrane cechy teryflunomidu, które spowodowały, że jest to obecnie najczęściej wybierany komparator w badaniach klinicznych w stwardnieniu rozsianym. Ilość zebranych dzięki temu informacji znacząco wzmocniła pozycję leku jako bezpiecznego i umożliwiającego uzyskanie wiarygodnych, porównywalnych danych. W skrótowy sposób opisano ewolucję badań klinicznych prowadzonych w stwardnieniu rozsianym. Zatwierdzenie w 1993 r. przez amerykańską Agencję ds. Żywności i Leków pierwszego leku wpływającego na długoterminowy przebieg rzutowo-remisyjnej postaci stwardnienia rozsianego niewątpliwie stało się kamieniem milowym w rozwoju badań klinicznych. Zastosowanie w konstrukcji badania z grupą placebo dało wtedy szansę na wykazanie siły działania leku. Stopniowo jednak, wraz z rejestracją nowych, skutecznych i ocenianych wieloma parametrami terapii, w kolejnych badaniach placebo zostało zastąpione lekami porównawczymi (komparatorami), początkowo podawanymi w iniekcjach, następnie w formie preparatów doustnych. Od 2015 r. najczęściej stosowanym w badaniach klinicznych lekiem porównawczym jest przyjmowany raz dziennie doustnie, teryflunomid (inhibitor dehydrogenazy dihydroorotanowej, DHO-DH).
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Bibliografia
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16. Kappos L, Fox RJ, Burcklen M et al. Ponesimod Compared With Teriflunomide in Patients With Relapsing Multiple Sclerosis in the Active-Comparator Phase 3 OPTIMUM Study: A Randomized Clinical Trial. JAMA Neurol. 2021; 78(5): 558-67. http://doi.org/10.1001/jamaneurol.2021.0405.
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22. Zhang Y, Yin H, Zhang D et al. Real-world outcomes of teriflunomide in relapsing-remitting multiple sclerosis: a prospective cohort study. J Neurol. 2022; 269(9): 4808-16. http://doi.org/10.1007/s00415-022-11118-7.
23. Comi G, Freedman MS, Kappos L et al. Pooled safety and tolerability data from four placebo-controlled teriflunomide studies and extensions. Mult Scler Relat Disord. 2016; 5: 97-104.
24. Ringheim GE, Wampole M, Oberoi K. Bruton’s Tyrosine Kinase (BTK) Inhibitors and Autoimmune Diseases: Making Sense of BTK Inhibitor Specificity Profiles and Recent Clinical Trial Successes and Failures. Front Immunol. 2021; 12: 662223. http://doi.org/10.3389/fimmu.2021.662223.
25. Paik J. Teriflunomide: Pediatric First Approval. Paediatr Drugs. 2021; 23(6): 609-13. http://doi.org/10.1007/s40272-021-00471-1.
26. Gärtner J, Hauser SL, Bar-Or A et al. Efficacy and safety of ofatumumab in recently diagnosed, treatment-naive patients with multiple sclerosis: Results from ASCLEPIOS I and II. Mult Scler. 2022; 28(10): 1562-75. http://doi.org/10.1177/13524585221078825.
27. Hauser SL, Bar-Or A, Cohen JA et al. ASCLEPIOS I and ASCLEPIOS II Trial Groups. Ofatumumab versus Teriflunomide in Multiple Sclerosis. N Engl J Med. 2020; 383(6): 546-57. http://doi.org/10.1056/NEJMoa1917246.
28. Steinman L, Fox E, Hartung HP et al. ULTIMATE I and ULTIMATE II Investigators. Ublituximab versus Teriflunomide in Relapsing Multiple Sclerosis. N Engl J Med. 2022; 387(8): 704-14. http://doi.org/10.1056/NEJMoa2201904.
2. Pearce JM. Historical descriptions of multiple sclerosis. Eur Neurol. 2005; 54(1): 49-53. http://doi.org/10.1159/000087387.
3. Thompson AJ, Banwell BL, Barkhof F et al. Diagnosis of multiple sclerosis: 2017 revisions of the McDonald criteria. Lancet Neurol. 2018; 17(2): 162-73. http://doi.org/10.1016/S1474-4422(17)30470-2.
4. Sorensen PS. New management algorithms in multiple sclerosis. Curr Opin Neurol. 2014; 27(3): 246-59. http://doi.org/10.1097/WCO.0000000000000096.
5. Ghezzi A. European and American Guidelines for Multiple Sclerosis Treatment. Neurol Ther. 2018; 7(2): 189-94. http://doi.org/10.1007/s40120-018-0112-1.
6. IFNB Multiple Sclerosis Study Group. Interferon beta-1b is effective in relapsing-remitting multiple sclerosis: I. Clinical results of a multicenter, randomized, double-blind, placebo-controlled trial. Neurology. 1993; 43(4): 655.
7. Galetta SL, Markowitz C, Lee AG. Immunomodulatory agents for the treatment of relapsing multiple sclerosis: a systematic review. Arch Intern Med. 2002; 162(19): 2161-9. http://doi.org/10.1001/archinte.162.19.2161.
8. Hauser SL, Cree BAC. Treatment of Multiple Sclerosis: A Review. Am J Med. 2020; 133(12): 1380-90.e2. http://doi.org/10.1016/j.amjmed.2020.05.049.
9. Agencja Badań Medycznych. Słownik (access: 3.10.2022).
10. Lublin FD. New multiple sclerosis phenotypic classification. Eur Neurol. 2014; 72(suppl 1): 1-5. http://doi.org/10.1159/000367614.
11. Sharma S, Mathur AG, Pradhan S et al. Fingolimod (FTY720): First approved oral therapy for multiple sclerosis. J Pharmacol Pharmacother. 2011; 2(1): 49-51. http://doi.org/10.4103/0976-500X.77118.
12. Genzyme. FDA approves Genzyme’s Aubagio® (teriflunomide), a once-daily, oral treatment for relapsing multiple sclerosis [media release]. 2012 (access: 3.10.2022).
13. Genzyme. Genzyme announces EMA accepts oral teriflunomide marketing application for treatment of multiple sclerosis [media release]. 2012 (access: 3.10.2022).
14. Foroughipour M, Gazeran S. Effectiveness and side effects of dimethyl fumarate in multiple sclerosis after 12 months of follow up: An Iranian clinical trial. Iran J Neurol. 2019; 18(4): 154-8.
15. Wray S, Bergh FT, Wundes A et al Efficacy and Safety Outcomes with Diroximel Fumarate After Switching from Prior Therapies or Continuing on DRF: Results from the Phase 3 EVOLVE-MS-1 Study. Adv Ther. 2022; 39(4): 1810-31. http://doi.org/10.1007/s12325-022-02068-7.
16. Kappos L, Fox RJ, Burcklen M et al. Ponesimod Compared With Teriflunomide in Patients With Relapsing Multiple Sclerosis in the Active-Comparator Phase 3 OPTIMUM Study: A Randomized Clinical Trial. JAMA Neurol. 2021; 78(5): 558-67. http://doi.org/10.1001/jamaneurol.2021.0405.
17. O’Connor P, Wolinsky JS, Confavreux C et al. TEMSO Trial Group. Randomized trial of oral teriflunomide for relapsing multiple sclerosis. N Engl J Med. 2011; 365(14): 1293-303. http://doi.org/10.1056/NEJMoa1014656.
18. Confavreux C, O’Connor P, Comi G et al. TOWER Trial Group. Oral teriflunomide for patients with relapsing multiple sclerosis (TOWER): a randomised, double- blind, placebo-controlled, phase 3 trial. Lancet Neurol. 2014; 13(3): 247-56. http://doi.org/10.1016/S1474-4422(13)70308-9.
19. Comi G, Freedman MS, Meca-Lallana JE et al. Prior treatment status: impact on the efficacy and safety of teriflunomide in multiple sclerosis. BMC Neurol. 2020; 20(1): 364. http://doi.org/10.1186/s12883-020-01937-4.
20. Scott LJ. Teriflunomide: A Review in Relapsing-Remitting Multiple Sclerosis. Drugs. 2019; 79(8): 875-86. http://doi.org/10.1007/s40265-019-01135-8.
21. Miller AE. Teriflunomide in multiple sclerosis: an update. Neurodegener Dis Manag. 2017; 7(1): 9-29. http://doi.org/10.2217/nmt-2016-0029.
22. Zhang Y, Yin H, Zhang D et al. Real-world outcomes of teriflunomide in relapsing-remitting multiple sclerosis: a prospective cohort study. J Neurol. 2022; 269(9): 4808-16. http://doi.org/10.1007/s00415-022-11118-7.
23. Comi G, Freedman MS, Kappos L et al. Pooled safety and tolerability data from four placebo-controlled teriflunomide studies and extensions. Mult Scler Relat Disord. 2016; 5: 97-104.
24. Ringheim GE, Wampole M, Oberoi K. Bruton’s Tyrosine Kinase (BTK) Inhibitors and Autoimmune Diseases: Making Sense of BTK Inhibitor Specificity Profiles and Recent Clinical Trial Successes and Failures. Front Immunol. 2021; 12: 662223. http://doi.org/10.3389/fimmu.2021.662223.
25. Paik J. Teriflunomide: Pediatric First Approval. Paediatr Drugs. 2021; 23(6): 609-13. http://doi.org/10.1007/s40272-021-00471-1.
26. Gärtner J, Hauser SL, Bar-Or A et al. Efficacy and safety of ofatumumab in recently diagnosed, treatment-naive patients with multiple sclerosis: Results from ASCLEPIOS I and II. Mult Scler. 2022; 28(10): 1562-75. http://doi.org/10.1177/13524585221078825.
27. Hauser SL, Bar-Or A, Cohen JA et al. ASCLEPIOS I and ASCLEPIOS II Trial Groups. Ofatumumab versus Teriflunomide in Multiple Sclerosis. N Engl J Med. 2020; 383(6): 546-57. http://doi.org/10.1056/NEJMoa1917246.
28. Steinman L, Fox E, Hartung HP et al. ULTIMATE I and ULTIMATE II Investigators. Ublituximab versus Teriflunomide in Relapsing Multiple Sclerosis. N Engl J Med. 2022; 387(8): 704-14. http://doi.org/10.1056/NEJMoa2201904.