Observational Study for the Evaluation of Incidence of Systemic Mastocytosis in t(8;21) Acute Myeloid Leukemia

Study Overview
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Brief Summary
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The observational study aimed at evaluating the incidence of systemic mastocytosis associated with t(8;21) AML in patients with de novo t(8;21) AML and their responses to first induction, and the prognosis from standard therapy.

Detailed Description
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This is a multicenter, retrospective and prospective, observational study that aims to collect clinical information on patients with systemic mastocytosis associated with t(8;21) AML from September 2022 to August 2023. No intervention is expected. The purpose of this study is to identify and characterize the patients with systemic mastocytosis associated with t(8;21) AML, t(8;21) AML without systemic mastocytosis, and OSM (Oligo-mastocytic SM) with associated t(8;21) AML. In order to estimate the incidence of systemic mastocytosis associated with t(8;21) AML, a survey will be sent every month to all participating sites to collect the number of all diagnoses of systemic mastocytosis associated with t(8;21) AML, t(8;21) AML without systemic mastocytosis, and OSM with associated t(8;21) AML. All patients will be followed until August 2025 in order to have at least 2 years of observation.

Official Title
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A Retrospective and Prospective Multicenter Observational Study for the Evaluation of Incidence of Systemic Mastocytosis With Associated t(8;21) Acute Myeloid Leukemia in t(8;21) Acute Myeloid Leukemia

Conditions
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Systemic Mastocytosis With AHNMD, Acute Myeloid Leukemia With T(8;21)(Q22;Q22)

Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Male or female, Age (years) >= 5; Newly diagnosed as t(8;21) AML patients according to World Health Organization (WHO) classification; Patients who sign the informed consent must have the ability to understand and be willing to participate in the study and sign the informed consent. Exclusion Criteria: The t(8;21) AML patients with SM have been diagnosed already; Patients with drug abuse or long-term alcoholism that affected the evaluation of trial results; Patients were deemed unsuitable for enrolment by the investigator.

Ages Eligible for Study
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Minimum Age: 5 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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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Systemic Mastocytosis with associated t(8;21) AML<br>SM and AML were diagnosed according to the 5th edition WHO classification criteria. | |
| The t(8;21) AML without Systemic Mastocytosis<br>The t(8;21) AML patients do not have associated Systemic Mastocytosis according to the 5th edition WHO classification criteria and no AML1-ETO clone was detected in mast cells. | |
| OSM (Oligo-mastocytic SM) with associated t(8;21) AML<br>The t(8;21) AML patients do not have associated Systemic Mastocytosis according to the 5th edition WHO classification criteria, but AML1-ETO clones were detected in mast cells. | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence of Systemic Mastocytosis associated with t(8;21) Acute Myeloid Leukemia | Evaluation of the incidence of systemic mastocytosis associated with t(8;21) AML in patients with de novo t(8;21) AML. The incidence of systemic mastocytosis associated with t(8;21) AML will be evaluated by means of the number of diagnosis of systemic mastocytosis associated with t(8;21) AML on the number of all diagnoses of de novo t(8;21) AML between September 2022 and August 2023. | at 1 year | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Hematological characteristics of all the t(8;21) Acute Myeloid Leukemia | The neutrophils, eosinophils, basophils, and mast cells in WBC classification (%); The proliferative degree in sections of BM (%); The ratio of mast cells in BM smear and FCM (%); The ratio of expression of CD25, CD2, and CD30 in FCM (%); The quantification of the AML1-ETO gene fusions (%). | at 1 year | 
| Responses to the first induction therapy of all the t(8;21) Acute Myeloid Leukemia | The rate of eligible complete remission (CR) patients(%); The rate of eligible CR with incomplete hematologic recovery (CRi) patients(%); The rate of eligible morphologic leukemia-free state (MLFS) patients(%); The rate of eligible partial remission (PR) patients(%); The rate of eligible no response (NR) patients(%); The rate of eligible CR without MRD patients(%); The rate of eligible overall response rate (ORR) patients(%). | at 1 year | 
| Incidence of transplantation of all the t(8;21) Acute Myeloid Leukemia | The outcome of transplant in three groups of patients with systemic mastocytosis associated with t(8;21) AML, t(8;21) AML without systemic mastocytosis, and OSM with associated t(8;21) AML. | at 1 year | 
| Survival Distribution of all the t(8;21) Acute Myeloid Leukemia | Overall survival (months): Measured the time from enrollment to the date of the last follow-up or death; Leukemia-free survival (months): Measured the time from the date of attaining CR1 until the first relapse, death, or the final follow-up day. | at 2 years |