Italian Observational Study of Patients With Acute Myeloid Leukemia Treated With Small Molecule Inhibiting BCL-2

Study Overview
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Brief Summary
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This is a multi-center retrospective observational study. Every patient with Acute Myeloid Leukemia (AML) treated with anti-B-cell lymphoma 2 (BCL2) treatment outside clinical trial from 1st January 2015 up to 01 April 2019 may be included in this study. No additional drug/procedures/patient visits in comparison with the usual clinical practice are planned for the study. The decision to treat patient with ant-BCL2 inhibitors is made by the physician based on his clinical judgment, independently from the decision to include the patient in this study.

Detailed Description
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In phase 1-2 studies, anti BCL-2 treatment has shown evidence of anti-leukemic activity as single agent and in combination and proved to be particularly effective in providing a deep response, with an acceptable safety profile. Since 2015 anti-BCL2 treatment has been available in other indications and in off-label use in Italy. In this non-interventional retrospective study, toxicity, effectiveness and costs assessment data will be collected from patients with AML, to improve the knowledge about anti-BCL2 treatment in clinical practice. Collecting and analyzing data from a large unbiased patient-set receiving anti-BCL2, would enlarge our knowledge on therapies inhibiting BCL2. This is a multi-center retrospective observational study. Every patient with AML treated with anti-BCL2 treatment outside clinical trial from 1st January 2015 up to 01 April 2019 may be included in this study. No additional drug/procedures/patient visits in comparison with the usual clinical practice are planned for the study. The decision to treat patient with ant-BCL2 inhibitors is made by the physician based on his clinical judgment, independently from the decision to include the patient in this study. As this study is intended to be purely observational (not interventional), the patient's medical records will be the source of all data to be recorded. No additional procedures/patient visits should be planned in the study with respect to clinical practice. Clinical data (treatment, survival, adverse events) will be collected for patients enrolled.

Official Title
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Italian Observational Study of Patients With Acute Myeloid Leukemia Treated With Small Molecule Inhibiting B-cell Lymphoma 2 (BCL-2)

Conditions
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Acute Myeloid Leukemia

Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Patient with AML according to World Health Organization (WHO) 2016 classification Patient who have received any anti-BCL-2 treatment as single agent or in combination with other drugs from 1 Jan 2015 to 1 Apr 2019 outside clinical trials Exclusion Criteria: • Patient who have received any anti-BCL-2 treatment within a clinical trials

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence of Adverse Events of grade 3 and 4 (NCTCAE version 5.0) | To evaluate the toxicity profile of the therapy with anti-BCL-2 in patients with AML. | up to 18 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Overall Survival | to describe the clinical outcomes in terms of Overall Survival (OS) defined as the number of days between the first study drug administration and death from any cause | 18 months | 
| Disease Free Survival (DFS) | to describe Disease Free Survival, defined as the number of days between the first study drug administration and any event including disease progression or death from any cause (both median and restricted mean) whichever occurs first. | 18 months | 
| Complete Remission | to describe the Complete Remission (CR), in terms of proportions, in response to therapy | 18 months | 
| Complete Remission with incomplete hematologic recovery | to describe Complete Remission with incomplete hematologic recovery (CRi) in terms of proportions, in response to therapy | 18 months | 
| Minimal Residual Disease | to describe Minimal Residual Disease, in terms of proportions, in response to therapy | 18 months | 
| Incidence of successful bridge to allogeneic transplant | to describe the incidence of successful bridge to allogeneic transplant, in terms of proportions, in response to therapy | 18 months | 
| Collection of number of hospitalizations | to describe healthcare resource utilization in terms of number of hospitalizations per patient | 18 months | 
| Collection of days of hospitalizations | to describe healthcare resource utilization in terms of days of hospitalizations per patient | 18 months | 
| Collection of number of clinical visits per patient | to describe healthcare resource utilization in terms of number of clinical visits per patient | 18 months | 
| Collection of number of accesses Day Hospital per patient | to describe healthcare resource utilization in terms of number of accesses in Day Hospital per patient | 18 months | 
| Collection of number of accesses in Emergency Care Units per patient | to describe healthcare resource utilization in terms of number of accesses in Emergency Care Units per patient | 18 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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AML, BCL-2, anti BCL2 inhibitors, clinical practice