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Risk of progression in chronic phase-chronic myeloid leukemia patients eligible for tyrosine kinase inhibitor discontinuation: final analysis of the TFR-PRO study

Disease progression to accelerated/blast phase (AP/BP) in patients with chronic phase chronic myeloid leukemia (CP-CML) after treatment discontinuation (TD) has never been systematically reported in clinical trials. However, recent reports of several such cases has raised concern. To estimate the ri...

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Main Authors: Zambrotta, Giovanni Paolo Maria (Author) , Nicolini, Franck E. (Author) , Assouline, Sarit (Author) , Busque, Lambert (Author) , Pungolino, Ester (Author) , Abruzzese, Elisabetta (Author) , Miggiano, Maria Cristina (Author) , Elena, Chiara (Author) , Alvarez-Larran, Alberto (Author) , Triguero, Ana (Author) , Iurlo, Alessandra (Author) , Bucelli, Cristina (Author) , Cerrano, Marco (Author) , Capodanno, Isabella (Author) , Lunghi, Francesca (Author) , le Coutre, Philipp (Author) , Galimberti, Sara (Author) , Caocci, Giovanni (Author) , Maffioli, Margherita (Author) , Stagno, Fabio (Author) , Saußele, Susanne (Author) , Piazza, Rocco (Author) , Druker, Brian J. (Author) , Fava, Carmen (Author) , Guglielmana, Veronica (Author) , Colombo, Federica (Author) , Antolini, Laura (Author) , Gambacorti-Passerini, Carlo (Author)
Format: Article (Journal)
Language:English
Published: November 2023
In: American journal of hematology
Year: 2023, Volume: 98, Issue: 11, Pages: 1762-1771
ISSN:1096-8652
DOI:10.1002/ajh.27073
Online Access:Verlag, kostenfrei, Volltext: https://doi.org/10.1002/ajh.27073
Verlag, kostenfrei, Volltext: https://onlinelibrary.wiley.com/doi/abs/10.1002/ajh.27073
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Author Notes:Giovanni Paolo Maria Zambrotta, Franck E. Nicolini, Sarit Assouline, Lambert Busque, Ester Pungolino, Elisabetta Abruzzese, Maria Cristina Miggiano, Chiara Elena, Alberto Alvarez-Larran, Ana Triguero, Alessandra Iurlo, Cristina Bucelli, Marco Cerrano, Isabella Capodanno, Francesca Lunghi, Philipp le Coutre, Sara Galimberti, Giovanni Caocci, Margherita Maffioli, Fabio Stagno, Susanne Saussele, Rocco Piazza, Brian J. Druker, Carmen Fava, Veronica Guglielmana, Federica Colombo, Laura Antolini, Carlo Gambacorti-Passerini
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Summary:Disease progression to accelerated/blast phase (AP/BP) in patients with chronic phase chronic myeloid leukemia (CP-CML) after treatment discontinuation (TD) has never been systematically reported in clinical trials. However, recent reports of several such cases has raised concern. To estimate the risk of AP/BP among TD-eligible patients, we conducted TFR-PRO, a cohort retro-prospective study: 870 CP-CML patients eligible for TD formed a discontinuation cohort (505 patients) and a reference one (365 patients). The primary objective was the time adjusted rate (TAR) of progression in relation to TD. Secondary endpoints included the TAR of molecular relapse, that is, loss of major molecular response (MMR). With a median follow up of 5.5 years and 5188.2 person-years available, no events occurred in the TD cohort. One event of progression was registered 55 months after the end of TD, when the patient was contributing to the reference cohort. The TAR of progression was 0.019/100 person-years (95% CI [0.003-0.138]) in the overall group; 0.0 (95% CI [0-0.163]) in the discontinuation cohort; and 0.030 (95% CI [0.004-0.215]) in the reference cohort. These differences are not statistically significant. Molecular relapses occurred in 172/505 (34.1%) patients after TD, and in 64/365 (17.5%) patients in the reference cohort, p < .0001. Similar rates were observed in TD patients in first, second or third line of treatment. CML progression in patients eligible for TD is rare and not related to TD. Fears about the risk of disease progression among patients attempting TD should be dissipated.
Item Description:Online veröffentlicht: 30. August 2023
Gesehen am 30.01.2024
Physical Description:Online Resource
ISSN:1096-8652
DOI:10.1002/ajh.27073

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