This is a multicentre ambispective cohort study involving French patients who have started or are receiving for less than 6 months a treatment with ponatinib. This study aims at better qualifying the ponatinib benefit-risk balance in real life and in relation with CML patients' therapeutic history.
Study Type
OBSERVATIONAL
Enrollment
120
For participants in chronic myeloid leukemia in chronic (CP-CML) phase: Proportion of participants who achieve a major molecular response after the initiation of study treatment
Chronic myeloid leukemia (CML) response criteria as defined by the European LeukemiaNet Recommendations for Management of Chronic Myeloid Leukemia (Hochhaus et al Leukemia 2020).
Time frame: from 24-60 months
For participants in chronic myeloid leukemia in accelerated phase (AP-CML) or chronic myeloid leukemia in blast phase (BP-CML): Proportion of participants who achieve a complete hematologic response
CML response criteria as defined by the European LeukemiaNet Recommendations for Management of Chronic Myeloid Leukemia.
Time frame: from 24-60 months
Proportion of participants in CP-CML phase who achieved complete hematologic response
CML response criteria as defined by the European LeukemiaNet Recommendations for Management of Chronic Myeloid Leukemia.
Time frame: from 24-60 months
Proportion of participants in AP and BP phases who achieved major (complete + partial) cytogenetic response
CML response criteria as defined by the European LeukemiaNet Recommendations for Management of Chronic Myeloid Leukemia.
Time frame: from 24-60 months
Proportion of participants who achieved major molecular response and/or depth molecular response: (MR4 or MR4.5 or MR5)
CML response criteria as defined by the European LeukemiaNet Recommendations for Management of Chronic Myeloid Leukemia.
Time frame: from 24-60 months
Duration of response
Duration of CML response criteria as defined by the European LeukemiaNet Recommendations for Management of Chronic Myeloid Leukemia.
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CHU SUD Reunion GHSR
Saint-Pierre, Reunion, France
CHU Amiens-Picardie- Site SUD
Amiens, France
CHU D'Angers
Angers, France
CH Annecy
Annecy, France
Centre Hospitalier Argenteuil
Argenteuil, France
Centre Hospitalier D'Avignon
Avignon, France
CH Beziers
Béziers, France
Institut Bergonie
Bordeaux, France
Cabinet D'hematologie De La Clinique Du Parc
Castelnau-le-Lez, France
CH William Morey
Chalon-sur-Saône, France
...and 30 more locations
Time frame: from 24-60 months
Time to progression to AP-CML or BP-CML (for those participants not in AP-CML or BP-CML)
Time to progression to AP-CML defined as follows: Blasts in blood or marrow 15%-29%, or blasts plus promyelocytes in blood or marrow \> 30%, with blasts \< 30%; basophils in blood ≥ 20%; persistent thrombocytopenia (\< 100 × 10\^9/L) unrelated to therapy; clonal chromosome abnormalities in Ph1 cells (CCA/Ph1), major route, on treatment. Time to progression to BP-CML defined as follows: Blasts in blood or marrow ≥ 30%; extramedullary blast proliferation, apart from spleen.
Time frame: from 24-60 months
Dose reduction (after response) in each cohort
Includes level of response at the time of dose reduction and maintenance of response after dose reduction.
Time frame: from 24-60 months
Time to response
Time to CML response criteria as defined by the European LeukemiaNet Recommendations for Management of Chronic Myeloid Leukemia.
Time frame: from 24-60 months
Rate of progression to accelerated phase (AP-) or blast phase (BP-) CML
Rate of progression to AP- or BP-CML as defined in European LeukemiaNet (ELN) criteria.
Time frame: from 24-60 months
Progression-free survival (PFS)
Survival without any progression to AP or BP according to ELN criteria.
Time frame: from 24-60 months
Overall survival (OS)
Overall survival defined according to ELN criteria.
Time frame: from 24-60 months
Rate of adverse events
Adverse event is any untoward medical occurrence in a patient or clinical study subject administered a medicinal (investigational or non-investigational) product. An adverse event does not necessarily have a causal relationship with the treatment.
Time frame: from 24-60 months
Rate of discontinuation due to adverse events in each dose cohort
Adverse event is any untoward medical occurrence in a patient or clinical study subject administered a medicinal (investigational or non-investigational) product. An adverse event does not necessarily have a causal relationship with the treatment.
Time frame: from 24-60 months
Dose reductions (prior to response) in each dose cohort
Reduction in the dose of Iclusig.
Time frame: from 24-60 months
Dose interruptions in each dose cohort
Interruption of Iclusig treatment.
Time frame: from 24-60 months