AML, Adult
Conditions
Brief summary
Following the results of the phase 1b and the phase 3 studies, Venetoclax/Azacitidine (VEN/AZA) was available in France for newly diagnosed AML patients ineligible-IC patients through the early access program the so-called ATU program. Venetoclax (VEN) has been available in France through the ATU since Feb 2021 and through the current post-ATU schema from the point of marketing authorization approval and up to the pending publication of reimbursement and price. Between February 15, 2021, and June 30, 2021, 285 requests for ATU were made to the pharmaceutical company (Abbvie) and led to the initiation of treatment of more than 230 patients. At the end of ATU period, all these 230 ATU patients continued to be treated by VEN/AZA as part of the current post-ATU period. Healthcare professionals and health care decision makers need real world data to better understand the benefit/risk profile of treatment. Early access to treatment in France is close to real-life setting condition.
Interventions
None listed
Sponsors
Acute Leukemia French Association
French Innovative Leukemia Organisation
Study design
Observational model
COHORT
Time perspective
CROSS_SECTIONAL
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Adult patients * treatment with VEN-AZA for newly diagnosed AML and ineligible to intensive chemotherapy * Treatment in the named-patients program (ATU)
Exclusion criteria
* Treatment with VEN-AZA for previously treated AML * Prior treatment for preexisting hematological malignancies other that AML, including AZA is not an
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| characterize the overall response rate, in patients treated by Venetoclax/Azacitidine (VEN/AZA), in real life cohort | 18 months (since Cycle 1 Day 1) | 1. Rate of complete remission and complete remission with incomplete hematologic recovery (CR/CRi), 2. Rate of morphological leukemia free state (MLFS) 3. Rate of CR/CRi rate with negative minimal residual disease (MRD) assessed by flow cytometry (FCT) and/or molecular techniques if available |
| characterize the overal survival (OS) in patients treated by Venetoclax/Azacitidine (VEN/AZA), in real life cohort | 18 months (since Cycle 1 Day 1) | rate of OS according to the 2022 European LeukemiaNet (ELN) |
| characterize the event free survival (EFS) in patients treated by Venetoclax/Azacitidine (VEN/AZA), in real life cohort | 18 months (since Cycle 1 Day 1) | rate of EFS according to the 2022 European LeukemiaNet (ELN) |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| characterize the incidence and relatedness of serious adverse events (SAE), in patients treated by Venetoclax/Azacitidine (VEN/AZA), in real life | 18 months (since Cycle 1 Day 1) | description of grade 3/4 SAE and death according to CTCAE v5. |
| describe the management of Venetoclax/Azacitidine (VEN/AZA) in a real -life cohort of patient. | 18 months (since Cycle 1 Day 1) | daily dose and duration of Venetoclax and Azacitidine description of Venetoclax and Azacitidine dose modification description of Venetoclax and Azacitidine duration |
Countries
France
Contacts
Primary ContactAriane MINEUR
Outcome results
None listed