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Results From a French Temporary Utilization Authorization of First-line Acute Myeloid Leukemia (AML) Patients Ineligible for Intensive Chemotherapy (IC), Treated With Venetoclax Azacitidine

Results From a French Temporary Utilization Authorization of First-line Acute Myeloid Leukemia (AML) Patients Ineligible for Intensive Chemotherapy (IC), Treated With Venetoclax Azacitidine

Recruiting
18 years and older
All
Phase N/A

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Overview

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.

Eligibility

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 exclusion criteria
    • HYDROXYCARBAMIDE given for AML is not an exclusion criteria
    • AZA started before VEN for AML is not an exclusion criteria
  • Opposition to data collection

Study details
    AML
    Adult

NCT06263387

French Innovative Leukemia Organisation

14 October 2025

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