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Changes in chronic myeloid leukemia treatment modalities and outcomes after introduction of second-generation tyrosine kinase inhibitors as first-line therapy: a multi-institutional retrospective study by the CML Cooperative Study Group

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Abstract

This study investigated changes in treatment modalities and outcomes of chronic myeloid leukemia in the chronic phase (CP-CML) after the approval of second-generation tyrosine kinase inhibitors (2G-TKIs) for first-line therapy. Patients were grouped into those who underwent TKI therapy up to December 2010 (imatinib era group, n = 185) and after January 2011 (2G-TKI era group, n = 425). All patients in the imatinib era group were initially treated with imatinib, whereas patients in the 2G-TKI era group were mostly treated with dasatinib (55%) or nilotinib (36%). However, outcomes including progression-free survival, overall survival, and CML-related death (CRD) did not differ significantly between groups. When stratified by risk scores, the prognostic performance of the ELTS score was superior to that of the Sokal score. Even though both scoring systems predicted CRD in the imatinib era, only the ELTS score predicted CRD in the 2G-TKI era. Notably, the outcome of patients classified as high-risk by ELTS score was more favorable in the 2G-TKI era group than in the imatinib era group. Thus, expanding treatment options may have improved patient outcomes in CP-CML, particularly in patients classified as high-risk by ELTS score.

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The study participants did not give written consent to share their data publicly, so the research data is unavailable.

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Authors

Contributions

N.I., E.I., M.I., T.N., E.S., T(Tomoiku).T., M.T., H.F., M.A., Y.H., and T.K. contributed to the conception of the work. N.I., E.I., Y.K., N.W., M.I., H.N., T.N., E.S., T(Takayuki).T., T.M., T(Tomoiku).T., M.T., H.F., and T.K. accumulated patient data. N.I. analyzed data and wrote the manuscript, and all other authors revised and interpretation of the study results. Finally, all authors agreed and gave final approval of the manuscript for submission.

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Correspondence to Noriyoshi Iriyama.

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Conflict of interest

N.I. received honoraria and speaker fees from Bristol-Myers Squibb, Novartis Pharma K.K., Otsuka Pharmaceutical and Pfizer Inc. E.I. received honoraria from Bristol-Myers Squibb, Novartis Pharma K.K. and Otsuka Pharmaceutical. M.T. received honoraria and speaker fees from Pfizer Inc. and Bristol-Myers Squibb. T(Tomoiku).T. and Y.H. received honoraria from Bristol-Myers Squibb and Novartis Pharma K.K. T.K. received honoraria and speaker fees from Bristol-Myers Squibb, Novartis Pharma K.K. and Pfizer Inc. The remaining authors declare no competing financial interests to declare.

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12185_2024_3758_MOESM1_ESM.jpg

Supplementary Figure 1. Prognostic predictability of ELTS score in patients initially treated with second-generation tyrosine kinase inhibitors. The event-free survival (EFS, A), progression-free survival (PFS, B), overall survival (OS, C), and chronic myeloid leukemia-related death (CRD, D) were stratified according to ELTS score (low, n = 260; intermediate, n = 97; high, n = 14). Supplementary file1 (JPG 390 KB)

Supplementary file2 (DOCX 27 KB)

Supplementary file3 (DOCX 23 KB)

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Iriyama, N., Iwanaga, E., Kimura, Y. et al. Changes in chronic myeloid leukemia treatment modalities and outcomes after introduction of second-generation tyrosine kinase inhibitors as first-line therapy: a multi-institutional retrospective study by the CML Cooperative Study Group. Int J Hematol (2024). https://doi.org/10.1007/s12185-024-03758-4

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