Vnitřní lékařství 4/2020
PŘEHLEDOVÉ ČLÁNKY Súčasné možnosti liečby BCR‑ABL1 pozitívnej chronickej myelocytovej leukémie u dospelých pacientov 224 | VNITŘNÍ LÉKAŘSTVÍ / Vnitř Lék 2020; 66(4): 214–224 / www.casopisvnitrnilekarstvi.cz Záver Ešte pred vyše 3 desaťročiami bola CML fatálnym ochorením s me‑ diánom prežívania 4–6 rokov, s progresiou do blastovej fázy, ktorej prognóza je krajne nepriaznivá. Vývoj liečebných možností od alo‑ génnych transplantácií krvotvorných buniek v 90. rokoch 20. storočia smerom k cielenej terapii TKI po roku 2000 spôsobil prevrat v liečebnej stratégii, keďže zásadne došlo k predĺženiu celkového prežívania ako aj k zlepšeniu prognózy pacientov. V súčasnej modernej ére liečby CML dochádza len zriedka k progresii ochorenia do blastovej krízy, väčšina pacientov veľmi dobre reaguje na TKI, no problémom môže byť vznik nežiaducich účinkov dlhodobo podávanej terapie, ktoré môžu v niek‑ torých prípadoch limitovať adekvátne dávkovanie liekov. Na základe odporúčaní odborných spoločností ELN z roku 2013, ESMO z roku 2017 a NCCN pre rok 2019 máme možnosť v 1. línii liečby zvážiť u novodiagnostikovaného pacienta imatinib, dasatinib, nilotinib alebo bosutinib (14, 16, 55). Výber TKI závisí od stratifikácie rizika CML u individuálneho pacienta, jeho veku, komorbidít ako aj profilu nežiaducich účinkov zvažovaného TKI. Imatinib je lacnejší než druhogeneračné TKI a k dispozícii sú už generiká imatinibu, ktoré môžu znížiť ekonomické náklady na inak finančné náročnú dlhodobú terapiu v mnohých krajinách. Výzvou do budúcnosti je veľmi aktuálna problematika možnosti tzv. TFR, ktorá je nádejou aj pre mnohých pacientov s CML na možné vyliečenie ochorenia bez užívania TKI. LITERATÚRA 1. O’Brien S, Berman E, Moore JO et al. NCCN Task Force report: tyrosine kinase inhibitor therapy selection in the management of patients with chronic myelogenous leukemia. J Natl Compr Cancer Netw 2011; 9: (Suppl. 2): S1–S25. 2. Hehlmann R, Hochhaus A, Baccarani M European Leukemia Net. Chronic myeloid leu‑ kaemia. Lancet 2007; 370: 342–350. 3. Bower H, Björkholm M, Dickman PW et al. Life expectancy of patients with chronic myeloid leukemia approaches the life expectancy of the general population. J Clin On‑ col 2016; 34: 2851–2857. 4. Sokal JE, Cox EB, Baccarani M et al. Prognostic discrimination in „good‑risk“ chronic gra‑ nulocytic leukemia. Blood 1984; 63: 789–799. 5. Žáčková D Tyrozinkinázové inhibitory v léčbě starších pacientů s chronickou myeloid‑ ní leukémií - editorial. Vnitř Lék 2015; 61: 760–761. 6. Hochhaus A, Larson RA, Guilhot MD et al. Long‑Term Outcomes of Imatinib Treatment for Chronic Myeloid Leukemia. N Engl J Med 2017; 376: 917–927. 7. Kantarjian H, Shah NP, Hochhaus A et al. Dasatinib versus imatinib in newly diagnosed chronic‑phase chronic myeloid leukkemia. N Engl J Med 2010; 362: 2260–2270. 8. Hehlmann R, Lauseker M, Saußele S et al. Assesment of imatinib as first‑line treatment of chronic myeloid leukemia: 10-year survival results of the randomized CML study IV and impact of non‑CML determinants. Leukemia 2017; 31: 2398–2406. 9. Kantarjian HM, Giles F, Bhalla K et al. Nilotinib is effective in patients with chronic mye‑ loid leukemia in chronic phase after imatinib resistance or intolerance: 24-month follow ‑up results. Blood 2011; 117: 1141–114. 10. Klamová H, Žižková H, Burda P et al. Současné trendy v léčbě a diagnostice chronické myeloidní leukemie. Transfuze Hematol dnes 2017; 23: (Suppl. 1): S34–S46. 11. Etienne G, Guilhot J, Rea D et al. Long‑Term Follow‑Up of the French Stop Imatinib (STIM1) Study in Patients With Chronic Myeloid Leukemia. J Clin Oncol 2017; 35: 298–305. 12. Saussele S, Richter J, Gilhot J et al. Discontinuation of tyrosine kinase inhibitor therapy in chronic myeloid leukaemia (EURO‑SKI): a prespecified interim analysis of a prospective, multicentre, non‑randomised, trial. Lancet Oncol 2018; 19: 747–757. 13. Réa D, Nicolini FE, Tulliez M et al. Discontinuation of dasatinib or nilotinib in chronic myeloid leukemia: interim analysis of the STOP 2G‑TKI study. Blood 2017; 129: 846–854. 14. Baccarani M, Deininger MW, Rosti G et al. European LeukemiaNet Recommendati‑ ons for the management of chronic myeloid leukemia 2013. Blood 2013; 122: 872–884. 15. Alhuraiji A, Kantarjian H, Boddu P et al. Prognostic significance of additional chromozomal abnormalities at the time of diagnosis in patients with chronicmyeloid leukemia treatedwith frontline tyrosine kinase inhibitors. Am J Hematol. 2018; 93: 84–90. 16. Radich JP, Deininger M, Abboud CN et al. Chronic Myeloid Leukemia, Version 1.2019, NCCN Clinical Practice Guidelines in Oncology. J Natl Vanc Netw 2018; 16: 1108–1135. 17. Cohen MH, Williams G, Johnson G et al. Approval summary for imatinib mesylate cap‑ sules in the treatment of chronic myelogenous leukemia. Clin Cancer Res 2002; 8: 935–942. 18. Guilhot J, Baccarani M, Clark RE et al. Definitions, methodological and statictical issues for phase 3 clinical trials in chronic myeloid leukemia: a proposal by the European Leuke‑ mia Net. Blood 2012; 119: 5963–5971. 19. Hehlman R, Berger U, Pfirrmann M et al. Drug treatment is superior to allografting as first‑line therapy in chronic myeloid leukemia. Blood 2007; 109: 4686–4692. 20. Gratwohl A, Pfirrmann M, Zander A et al. Long‑term outcome of patients with newly diagnosed chronic myeloid leukemia: a randomized comparison of stem cell transplan‑ tation with drug treatment. Leukemia 2016; 30: 562–569. 21. Westin JR, Kurzrock R It’s about time: lessons for solid tumors from chronic myeloge‑ nous leukemia therapy. Mol Cancer Ther 2012; 11: 2549–2555. 22. Mayer J, Klamová H, Žáčková D et al. Imatinib v první linii léčby nemocných s nově diagnotikovanou chronickou myeloidní leukemií v chronické fázi. Transfuze Hematol dnes 2008; 14: 150–158. 23. Hughes T, White D Which TKI? An embarrassment of riches for chronic myeloid leu‑ kemia patients. Hematology 2013; 2013: 168–175. 24. O’Brien SG, Guilhot F, Larson RA et al. Imatinib compared with interferon and low ‑dose cytarabine for newly diagnosed chronic‑phase chronic myeloid leukemia. N Engl J Med 2003; 348: 994–1004. 25. Breccia M, Cannella L, Frustraci A et al. Pleural‑pericardial effusion as uncommon com‑ plication in CML patients treated with Imatinib. Eur J Haematol 2005; 74: 89–90. 26. Hochhaus A, Baccarani M, Deininger M et al. Dasatinib induces durable cytogenetic responses in patients with chronic myelogenous leukemia in chronic phase with resis‑ tance or intolerance to imatinib. Leukemia 2008; 22: 1200–1206. 27. Shah NP, Kim DW, Kantarjian H et al. Potent, transient inhibition of BCR‑ABL with dasatinib 100 mg daily achieves rapid and durable cytogenetic responses and high transformation‑free survival rates in chronic phase chronic myeloid leukemia pati‑ ents with resistance, suboptimal response or intolerance to imatinib. Haematologi‑ ca 2010; 95: 232–240. 28. Cortes JE, Saglio G, Kantarjian HM et al. Final 5-year study results of DASISION: The da‑ satinib versus imatinib study in treatment‑naïve chronic myeloid patients trial. J Clin On‑ col 2016; 34: 2333–2340. 29. Breccia M, Stagno F, Luciano L et al. Dasatinib first‑line: Multicentric Italian experience outside clinical trials. Leuk Res 2016; 40: 24–29. 30. Shah NP, Rousselot P, Schiffer Ch et al. Dasatinib in imatinib‑resistant or intolerant chronic‑phase, chronic myeloid leukemia patients: 7-year follow‑up of study CA180-034. Am J Hematol 2016; 91: 869–874. 31. Saglio G, KimDW, Issaragrisil S et al. Nilotinib verzus imatinib for newly diagnosed chro‑ nic myeloid leukemia. N Engl J Med 2010; 362: 2251–2259. 32. Saglio G, Hochhaus A, Hughes TP et al. ENESTnd Update: nilotinib (NIL) vs imatinib(IM) in patients (PTS) with newly diagnosed chronic myeloid leukemia in chronic phase (CML ‑CP) and the impact of early molecular response (EMR) and Sokal risk at diagnosis on long ‑term outcomes. Blood 2013; 122: abstract 92. 33. Hughes TP, Saglio G, Kantarjian HM et al. Early molecular response predicts outcomes in patients with chronic myeloid leukemia in chronic phase treated with frontline niloti‑ nib or imatinib. Blood 2014; 123: 1353-1360. 34. Hochhaus A, Saglio G, Larson RA et al. Nilotinib is associated with a reduced inciden‑ ce of BCR‑ABL mutations vs imatinib in patients with newly diagnosed chronic myeloid leukemia in chronic phase. Blood 2013; 121: 3703–3708. 35. Castagnetti F, Breccia M, Gugliotta G et al. Nilotinib 300 mg twice daily: an academic single‑arm study of newly diagnosed chronic phase chronic myeloid leukemia patients. Haematologica 2016; 101: 1200–1207. 36. Cortes JE, Kantarjian HM, Brümmendorf TH et al. Safety and efficacy of bosutinib (SKI- 606) in chronic phase Philadelphia chromosome‑positive chronic myeloid leukemia pa‑ tients with resistance or intolerance to imatinib. Blood 2011; 118: 4567–4576. 37. Cortes JE, Gambacorti‑Passerini C, Deininger MW et al. Bosutinib versus imatinib for newly diagnosed chronic myeloid leukemia: results from the randomized BFORE trial. J Clin Oncol 2018; 36: 231–237. 38. Cortes JE, Khoudry HJ, Kantarjian HM et al. Long‑term bosutinib for chronic phase chronic myeloid keukemia after failure of imatinib plus dasatinib and/or nilotinib. Am J Hematol 2016; 91: 1206-1214. 39. O’Hare T, Shakespeare WC, Zhu X et al. AP24534, a pan‑BCR/ABL inhibitor for chronic myeloid leukemia, potently imhibits the T315I mutant and overcomes mutation‑based resistance. Cancer Cell 2009; 16: 401–412. Další literatura u autorky a na www.casopisvnitrnilekarstvi.cz
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