BCR-ABL Positive Acute Lymphoblastic Leukemia
Conditions
Keywords
acute lymphoblastic leukemia, ponatinib, tyrosine kinase inhibitor
Brief summary
The purpose of this study is to evaluate the effectiveness and safety of ponatinib (Iclusig®) in patients with BCR-ABL positive acute lymphoblastic leukemia (ALL) in standard clinical practice in Europe.
Interventions
None listed
Sponsors
Incyte Biosciences International Sàrl
Study design
Observational model
CASE_ONLY
Time perspective
OTHER
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Adult patients with BCR-ABL (Ph+) positive ALL who are initiating Iclusig®, or for whom Iclusig® was initiated after January 2015. * Patients who have the ability to understand the requirements of the study and provide written informed consent to comply with the study data collection procedures.
Exclusion criteria
* Patients previously treated with investigational ponatinib. * Patients who are pregnant and/or breastfeeding. * Concurrent treatment with another tyrosine kinase inhibitor for Ph+ALL.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Complete hematological remission (CR) rate | 6 months | CR defined as leukemic cells not detectable by light microscopy in bone marrow (BM), peripheral blood (PB), or cerebrospinal fluid (CSF) (BM \< 5% blasts). |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Duration of best molecular response (MolCR) | Up to 12 months | MolCR defined as complete molecular remission/MRD negativity. |
| CR rate | 1, 3, 9, and 12 months | CR defined as leukemic cells not detectable by light microscopy in BM, PB, or CSF (BM \< 5% blasts). |
| Time to CR | Up to 12 months | Defined as time from enrollment to first CR. |
| Minimal residual disease (MRD) level | 3, 6, 9, and 12 months | Minimal residual disease level. |
| Best MRD (MolR) level rate | 12 months | MolR defined as molecular/MRD response, less than MolCR. |
| Time to best MRD (MolR) level | Up to 12 months | MolR defined as molecular/MRD response, less than MolCR. |
| Duration of molecular response | Up to 12 months | Measured by MRD log reduction (MolR). |
| Time to death | Up to 12 months | Defined as time from enrollment to death due to any cause. |
| Prescribed dose | Up to 12 months | Prescribed dose of Iclusig® in milligrams. |
| Daily average dose | Up to 12 months | The average daily dose of Iclusig® in milligrams. |
| Number of serious adverse events (SAEs) | 12 months | Defined as an adverse event that results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability or incapacity, is a congenital anomaly/birth defect, or is an important medical event. |
| Number of adverse events of special interest (AESI) | 12 months | AESI defined as vascular occlusive events, including arterial and venous events, as defined in the protocol. |
| Amount of hospital days | 12 months | Defined as overnight stay(s), each night in the hospital will be counted as 1 day. |
| Time to progression | Up to 12 months | Defined as the time to molecular relapse or hematological relapse. |
Countries
Czechia, France, Germany
Outcome results
None listed