Chronic Myeloid Leukemia
Conditions
Keywords
Chronic myeloid leukemia, Iclusig, ponatinib
Brief summary
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.
Interventions
None listed
Sponsors
Incyte BioSciences France
Study design
Observational model
COHORT
Time perspective
OTHER
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Presenting a CML in any phase. * Having initiated for less than six months a treatment with ponatinib. * The ability to understand the requirements of the study and to comply with the study data collection procedures.
Exclusion criteria
* Patients previously treated with investigational ponatinib (within a clinical trial). * Patients receiving an investigational agent. * Patients who are pregnant and/or breastfeeding. * Patients with contraindications for Ponatinib according to Summary of Products Characteristics.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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 | from 24-60 months | Chronic myeloid leukemia (CML) response criteria as defined by the European LeukemiaNet Recommendations for Management of Chronic Myeloid Leukemia (Hochhaus et al Leukemia 2020). |
| 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 | from 24-60 months | CML response criteria as defined by the European LeukemiaNet Recommendations for Management of Chronic Myeloid Leukemia. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Proportion of participants who achieved major molecular response and/or depth molecular response: (MR4 or MR4.5 or MR5) | from 24-60 months | CML response criteria as defined by the European LeukemiaNet Recommendations for Management of Chronic Myeloid Leukemia. |
| Duration of response | from 24-60 months | Duration of CML response criteria as defined by the European LeukemiaNet Recommendations for Management of Chronic Myeloid Leukemia. |
| Time to progression to AP-CML or BP-CML (for those participants not in AP-CML or BP-CML) | from 24-60 months | 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. |
| Dose reduction (after response) in each cohort | from 24-60 months | Includes level of response at the time of dose reduction and maintenance of response after dose reduction. |
| Time to response | from 24-60 months | Time to CML response criteria as defined by the European LeukemiaNet Recommendations for Management of Chronic Myeloid Leukemia. |
| Rate of progression to accelerated phase (AP-) or blast phase (BP-) CML | from 24-60 months | Rate of progression to AP- or BP-CML as defined in European LeukemiaNet (ELN) criteria. |
| Proportion of participants in CP-CML phase who achieved complete hematologic response | from 24-60 months | CML response criteria as defined by the European LeukemiaNet Recommendations for Management of Chronic Myeloid Leukemia. |
| Overall survival (OS) | from 24-60 months | Overall survival defined according to ELN criteria. |
| Rate of adverse events | from 24-60 months | 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. |
| Rate of discontinuation due to adverse events in each dose cohort | from 24-60 months | 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. |
| Dose reductions (prior to response) in each dose cohort | from 24-60 months | Reduction in the dose of Iclusig. |
| Dose interruptions in each dose cohort | from 24-60 months | Interruption of Iclusig treatment. |
| Progression-free survival (PFS) | from 24-60 months | Survival without any progression to AP or BP according to ELN criteria. |
| Proportion of participants in AP and BP phases who achieved major (complete + partial) cytogenetic response | from 24-60 months | CML response criteria as defined by the European LeukemiaNet Recommendations for Management of Chronic Myeloid Leukemia. |
Countries
France
Outcome results
None listed