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Real-world tyrosine kinase inhibitor treatment pathways, monitoring patterns and responses in patients with chronic myeloid leukaemia in the United Kingdom: The UK TARGET CML Study

Real-world tyrosine kinase inhibitor treatment pathways, monitoring patterns and responses in patients with chronic myeloid leukaemia in the United Kingdom: The UK TARGET CML Study
Real-world tyrosine kinase inhibitor treatment pathways, monitoring patterns and responses in patients with chronic myeloid leukaemia in the United Kingdom: The UK TARGET CML Study
Management of chronic myeloid leukaemia (CML) has recently undergone dramatic changes, prompting the European LeukemiaNet (ELN) to issue recommendations in 2013; however, it remains unclear whether real-world CML management is consistent with these goals. We report results of UK TARGET CML, a retrospective observational study of 257 patients with chronic-phase CML who had been prescribed a first-line TKI between 2013 and 2017, most of whom received first-line imatinib (n = 203). Although 44% of patients required ≥1 change of TKI, these real-world data revealed that molecular assessments were frequently missed, 23% of patients with ELN-defined treatment failure did not switch TKI, and kinase domain mutation analysis was performed in only 49% of patients who switched TKI for resistance. Major molecular response (MMR; BCR-ABL1IS ≤0·1%) and deep molecular response (DMR; BCR-ABL1IS ≤0·01%) were observed in 50% and 29%, respectively, of patients treated with first-line imatinib, and 63% and 54%, respectively, receiving a second-generation TKI first line. MMR and DMR were also observed in 77% and 44% of evaluable patients with ≥13 months follow-up, receiving a second-generation TKI second line. We found little evidence that cardiovascular risk factors were considered during TKI management. These findings highlight key areas for improvement in providing optimal care to patients with CML.
CML management, chronic myeloid leukaemia, molecular response, real-world study, tyrosine kinase inhibitor
0007-1048
62-74
Milojkovic, Dragana
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Cross, Nicholas
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Ali, Sahra
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Byrne, Jenny
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Campbell, Gavin
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Dignan, Fiona L.
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Drummond, Mark
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Huntly, Brian J.P.
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Marshall, Scott
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McMullin, Mary Frances
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Neelakantan, Pratap
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Raghavan, Manoj
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Sivakumaran, Muttuswamy
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Tighe, Jane
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Wandroo, Farooq
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Willis, Fenella
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Glen, Fiona
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Fildes, Louise
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Collington, Sarah J.
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Ryan, Jacqueline
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Clark, Richard E.
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Mead, Adam J.
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Milojkovic, Dragana
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Cross, Nicholas
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Ali, Sahra
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Byrne, Jenny
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Campbell, Gavin
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Dignan, Fiona L.
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Drummond, Mark
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Huntly, Brian J.P.
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Marshall, Scott
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McMullin, Mary Frances
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Neelakantan, Pratap
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Raghavan, Manoj
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Sivakumaran, Muttuswamy
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Tighe, Jane
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Wandroo, Farooq
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Willis, Fenella
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Glen, Fiona
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Fildes, Louise
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Collington, Sarah J.
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Ryan, Jacqueline
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Clark, Richard E.
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Mead, Adam J.
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Milojkovic, Dragana, Cross, Nicholas, Ali, Sahra, Byrne, Jenny, Campbell, Gavin, Dignan, Fiona L., Drummond, Mark, Huntly, Brian J.P., Marshall, Scott, McMullin, Mary Frances, Neelakantan, Pratap, Raghavan, Manoj, Sivakumaran, Muttuswamy, Tighe, Jane, Wandroo, Farooq, Willis, Fenella, Glen, Fiona, Fildes, Louise, Collington, Sarah J., Ryan, Jacqueline, Clark, Richard E. and Mead, Adam J. (2021) Real-world tyrosine kinase inhibitor treatment pathways, monitoring patterns and responses in patients with chronic myeloid leukaemia in the United Kingdom: The UK TARGET CML Study. British Journal of Haematology, 192 (1), 62-74. (doi:10.1111/bjh.16733).

Record type: Article

Abstract

Management of chronic myeloid leukaemia (CML) has recently undergone dramatic changes, prompting the European LeukemiaNet (ELN) to issue recommendations in 2013; however, it remains unclear whether real-world CML management is consistent with these goals. We report results of UK TARGET CML, a retrospective observational study of 257 patients with chronic-phase CML who had been prescribed a first-line TKI between 2013 and 2017, most of whom received first-line imatinib (n = 203). Although 44% of patients required ≥1 change of TKI, these real-world data revealed that molecular assessments were frequently missed, 23% of patients with ELN-defined treatment failure did not switch TKI, and kinase domain mutation analysis was performed in only 49% of patients who switched TKI for resistance. Major molecular response (MMR; BCR-ABL1IS ≤0·1%) and deep molecular response (DMR; BCR-ABL1IS ≤0·01%) were observed in 50% and 29%, respectively, of patients treated with first-line imatinib, and 63% and 54%, respectively, receiving a second-generation TKI first line. MMR and DMR were also observed in 77% and 44% of evaluable patients with ≥13 months follow-up, receiving a second-generation TKI second line. We found little evidence that cardiovascular risk factors were considered during TKI management. These findings highlight key areas for improvement in providing optimal care to patients with CML.

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TARGET UK BJH 13 Jan 2020 - Accepted Manuscript
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Accepted/In Press date: 18 April 2020
e-pub ahead of print date: 24 May 2020
Published date: 1 January 2021
Keywords: CML management, chronic myeloid leukaemia, molecular response, real-world study, tyrosine kinase inhibitor

Identifiers

Local EPrints ID: 439651
URI: http://eprints.soton.ac.uk/id/eprint/439651
ISSN: 0007-1048
PURE UUID: 9a5b916d-11c0-439e-b98b-acb356902d60
ORCID for Nicholas Cross: ORCID iD orcid.org/0000-0001-5481-2555

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Date deposited: 29 Apr 2020 16:30
Last modified: 17 Mar 2024 05:30

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Contributors

Author: Dragana Milojkovic
Author: Nicholas Cross ORCID iD
Author: Sahra Ali
Author: Jenny Byrne
Author: Gavin Campbell
Author: Fiona L. Dignan
Author: Mark Drummond
Author: Brian J.P. Huntly
Author: Scott Marshall
Author: Mary Frances McMullin
Author: Pratap Neelakantan
Author: Manoj Raghavan
Author: Muttuswamy Sivakumaran
Author: Jane Tighe
Author: Farooq Wandroo
Author: Fenella Willis
Author: Fiona Glen
Author: Louise Fildes
Author: Sarah J. Collington
Author: Jacqueline Ryan
Author: Richard E. Clark
Author: Adam J. Mead

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