Influence of Co-existing Mutations on Sorafenib Maintenance Therapy After Allo-HSCT for Patients With FLT3-ITD AML

Study Overview
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Brief Summary
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The purpose of this study is to reveal the influence of co-existing mutations on the efficacy of sorafenib maintenance after allogeneic hematopoietic stem cell transplantation for patients with FLT3-ITD AML.

Detailed Description
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Internal tandem duplication of FMS-like tyrosine kinase 3 (FLT3-ITD) mutations have been reported in 20%-30% of patients with acute myeloid leukemia (AML). FLT3-ITD-positive AML patients have an inferior survival, primarily due to lower complete remission (CR) rate and higher relapse rate. Our previous studies demonstrated that post-transplantation sorafenib maintenance could improve the outcomes of FLT3-ITD-positive AML patients. However, whether other co-existing mutations influence the efficacy of post-allo-HSCT sorafenib maintenance remains unknown.

Official Title
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Influence of Co-existing Mutations on Sorafenib Maintenance Therapy After Allogeneic Hematopoietic Stem Cell Transplantation for Patients With FLT3-ITD Positive Acute Myeloid Leukemia

Conditions
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Acute Myeloid Leukemia, Acute Myeloid Leukemia With FLT3/ITD Mutation, Hematopoietic Stem Cell Transplantation

Intervention / Treatment
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* Drug: Sorafenib


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: FLT3-ITD Positive AML Allo-HSCT Recipients Exclusion Criteria: cardiac dysfunction (particularly congestive heart failure) hepatic abnormalities (bilirubin ≥ 3 mg/dL, aminotransferase> 2 times the upper limit of normal) renal dysfunction (creatinine clearance rate < 30 mL/min) Any abnormality in a vital sign (e.g., heart rate, respiratory rate, or blood pressure) Patients with any conditions not suitable for the trial (according to the investigators' decision)

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 60 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 |
| --- | --- |
| Sorafenib group<br>Patients assigned to this group received sorafenib post-transplantation. | Drug: Sorafenib<br>* The initial dose of sorafenib is 400 mg orally twice daily and is adjusted in case of suspected toxicity or resistance (dose range, 200-800 mg daily).<br>* Other names: BAY 545-9085;|
| Control group<br>Patients assigned to this group did not receive sorafenib or any other FLT3 inhibitor post-transplantation until reaching the primary outcome. | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence of leukemia relapse |  | 2 year | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Overall survival |  | 2 year | 
| Leukemia-free survival |  | 2 year | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Acute Myeloid Leukemia, FLT3-ITD, Hematopoietic Stem Cell Transplantation, sorafenib