A Retrospective Epidemiologic Registry to Gain Insight Into the Characteristics and Prognosis of AML Patients According to the Routinely Used Genetic and Biologic Markers

Study Overview
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Brief Summary
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This is a retrospective, translational, epidemiologic, multicenter, non-interventional study (No EPA study) to provide insights into disease epidemiology, disease biology, treatment regimens, and clinical outcomes of patients with acute myeloblastic leukemia (AML) in routine clinical practice according to their molecular markers. The primary objective of the study is to describe the use of the main molecular markers (FLT3 and NPM1) in the real-life according of the type of AML, treating institution, patients' characteristics, and disease status.

Detailed Description
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This is a retrospective, translational, epidemiologic, multicenter, non-interventional study (No EPA study) to provide insights into disease epidemiology, disease biology, treatment regimens, and clinical outcomes of patients with AML in routine clinical practice according to their molecular markers. Once it has been confirmed that all selection criteria for the study have been met and informed consent has been obtained, the subject will be considered enrolled in the study and the investigator can proceed to collect data from their medical record by completing a case report (CRF). The study contemplates the retrospective collection of data from disease diagnosis to the start of the study. Only data obtained before the start of the study will be collected in order to ensure they are retrospective in nature. The study will be conducted following the requirements contained in the Declaration of Helsinki (Fortaleza, Brazil 2013) and in accordance with the current Spanish legislation with regard to conducting observational studies (Ministerial Order SAS/3470/2009). The primary objective of the study is to describe the use of the main molecular markers (FLT3 and NPM1) in the real-life according of the type of AML, treating institution, patients' characteristics, and disease status. This project will be conducted in the Spanish PETHEMA cooperative group, constituted by 60 institutions and seven main central laboratories with extensive technological capacity. All patients will be included in the ongoing PETHEMA epidemiologic registry of AML with the purpose to collect a large number, additional and non-pre-existent clinical data, including first-line and salvage treatment schedules and outcomes of each patient. For this study the PETHEMA AML registry will be enlarged seeking for new cases not previously reported. The data base information will be updated emphasizing for the capture of new data on molecular screening tests performed on a routine basis (FLT3, NPM1, and others). For these purpose, information will be systematically requested from the main PETHEMA laboratories. Therapies and clinical outcome data will be retrospectively collected. This is a one-step study without intervention, in which all analyses will be performed at the end of the database completion.

Official Title
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A Retrospective Epidemiologic Registry to Gain Insight Into the Characteristics and Prognosis of AML Patients According to the Routinely Used Genetic and Biologic Markers

Conditions
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Acute Myeloid Leukemia

Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: All adult patients with AML (excluding APL) according to the WHO criteria (2008), regardless of the treatment administered by their treating physician. AML at diagnosis and at relapse or refractoriness. Patients from institutions participating in the ongoing PETHEMA AML registry. Patients with information about the molecular screening including FLT3 with or without NPM1 mutations (including positive/negative or not performed). Ability to give informed consent before the study initiation. Death patients and patients who are no longer contactable or lost to follow-up will be excluded from consent requirement. Exclusion Criteria: Pediatric patients. Acute promyelocytic leukemia.

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 |
| --- | --- | --- |
| Percentage of patients with molecular alterations (FLT3 and NPM1 mutations). | Percentage of each of the standard screening panel molecular alterations studied in the AML patients (FLT3 and NPM1) by AML type, by center, by patient's characteristics and the disease status. | Baseline | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Overall response rate. | Percentage of patients achieving response in each group of the baseline alterations studied in the AML patients (FLT3 and NPM1) . | Throughout the study period. Approximately 1 year | 
| Description of initial and later treatments in each baseline alterations studied (FLT3 and NPM1) in each AML type. | Frequency of initial and later type of treatments in each baseline alterations studied (FLT3 and NPM1) in each AML type. | Baseline and throughout the study period. Approximately 1 year | 
| Molecular response rate. | Percentage of patients achieving molecular response in each group of the baseline alterations studied in the AML patients (FLT3 and NPM1) . | Throughout the study period. Approximately 1 year | 
| Description of biology of disease at relapse or refractoriness by molecular characteristics. | Percentage of AML type at relapse or refractoriness in each baseline alterations studied (FLT3 and NPM1). | Baseline and throughout the study period. Approximately 1 year | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Relapsed/refractory Acute Myeloid Leukemia, De novo Acute Myeloid Leukemia