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Treatment goals are focused on getting CML under control1 while preserving/improving QOL2

Getting CML under control

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Potential to attempt TFR2

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Improve survival2

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Achieve and maintain milestones1

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Prevent disease progression1

Preserving/Improving QOL

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Improve or maintain QOL2

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Minimize treatment-related toxicities3

  • Specific treatment goals are related to individual patient factors, and they may vary or evolve over time and by line of therapy4
  • Achievement of milestones (hematologic, cytogenetic, and molecular)5 may be linked to outcomes, including reduced progression1 and prolonged survival2

 

Goals in newly diagnosed or patients with fewer treatments

  • TFR2 and rapid and durable achievement of milestones (EMR, DMR, MMR)1

 

Goals in older patients or with multiple treatments

  • Maintaining QOL with less-aggressive milestone goals6

 

Deeper molecular responses are associated with improved outcomes7

  • Getting CML under control

MMR (0.1% BCR::ABL1IS) as a predictor of PFS and OS1

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QOL, Quality of life; CML, Chronic myeloid leukemia; TFR, treatment-free remission; EMR, Early molecular response; DMR, Deep molecular response; ABL1, Abelson protein tyrosine kinase 1; BCR, breakpoint cluster region; CCyR, complete cytogenetic response; CHR, complete hematologic response; IS, International Scale; MMR, major molecular response (BCR::ABL1IS 0.1%); MR4, BCR::ABL1IS 0.01%; MR4·5, BCR::ABL1IS 0.0032%; OS, overall survival; PFS, progression-free survival; TFR, treatment-free remission. a BCR::ABL1IS 10% is roughly equivalent to CHR. b BCR::ABL1IS <1% is roughly equivalent to CCyR.

References

  1. National Comprehensive Cancer Network® (NCCN®), NCCN Guidelines Version 1.2026 Chronic Myeloid Leukemia. Available at: https://www.nccn.org/professionals/physician_gls/pdf/cml.pdf . Last accessed 10/9/2025

  2. Hochhaus A, Baccarani M, Silver RT, Schiffer C, Apperley JF, Cervantes F, Clark RE, Cortes JE, Deininger MW, Guilhot F, Hjorth-Hansen H. European LeukemiaNet 2020 recommendations for treating chronic myeloid leukemia. Leukemia. 2020 Apr;34(4):966-84

  3. Khoury HJ, Williams LA, Atallah E, Hehlmann R. Chronic myeloid leukemia: what every practitioner needs to know in 2017. American Society of Clinical Oncology Educational Book. 2017 Jan 1;37:468-79

  4. Saglio G, Jabbour E. First-line therapy for chronic phase CML: selecting the optimal BCR-ABL1-targeted TKI. Leukemia & lymphoma. 2018 Jul 3;59(7):1523-38.

  5. Leukemia & Lymphoma Society® (LLS). Chronic myeloid leukemia. Treatment outcomes. Available at: https://bloodcancerunited.org/blood-cancer/leukemia/chronic-myeloid-leukemia-cml/treatment . Last accessed 10/9/2025

  6. Castagnetti F, Binotto G, Capodanno I, Billio A, Calistri E, Cavazzini F, Crugnola M, Gozzini A, Gugliotta G, Krampera M, Lucchesi A. Making treatment-free remission (TFR) easier in chronic myeloid leukemia: fact-checking and practical management tools. Targeted oncology. 2021 Nov;16(6):823-38.

  7. Mahon FX, Etienne G. Deep molecular response in chronic myeloid leukemia: the new goal of therapy?. Clinical Cancer Research. 2014 Jan 15;20(2):310-22.

  8. Baccarani M, Saglio G, Goldman J, Hochhaus A, Simonsson B, Appelbaum F, Apperley J, Cervantes F, Cortes J, Deininger M, Gratwohl A. Evolving concepts in the management of chronic myeloid leukemia: recommendations from an expert panel on behalf of the European LeukemiaNet. Blood. 2006 Sep 15;108(6):1809-20.

  9. Hochhaus A, Saussele S, Rosti G, Mahon FX, Janssen JJ, Hjorth-Hansen H, Richter J, Buske C. Chronic myeloid leukaemia: ESMO Clinical Practice Guidelines for diagnosis, treatment and follow-up. Annals of Oncology. 2017 Jul 1;28:iv41-51

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