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

Sponsor

Nanfang Hospital of Southern Medical University (Other)

Overall Status

Completed

CT.gov ID

NCT04788420

Collaborator

Peking University People's Hospital (Other), Third Affiliated Hospital, Sun Yat-Sen University (Other), Zhujiang Hospital (Other), Xiangya Hospital of Central South University (Other), First People's Hospital of Chenzhou (Other), The First Affiliated Hospital of Guangzhou Medical University (Other), Institute of Hematology & Blood Diseases Hospital (Other), The First Affiliated Hospital of Soochow University (Other), Xinqiao Hospital of Chongqing (Other), First Affiliated Hospital of Zhejiang University (Other)

456

Enrollment

Location

108

Actual Duration (Months)

4.2

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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.

Condition or Disease	Intervention/Treatment	Phase
Acute Myeloid Leukemia Acute Myeloid Leukemia With FLT3/ITD Mutation Hematopoietic Stem Cell Transplantation	Drug: Sorafenib

Detailed Description

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.

Study Design

Study Type:

Observational

Actual Enrollment :

456 participants

Observational Model:

Cohort

Time Perspective:

Retrospective

Official Title:

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

Actual Study Start Date :

Jan 1, 2012

Actual Primary Completion Date :

Jul 21, 2018

Actual Study Completion Date :

Dec 31, 2020

Arms and Interventions

Arm	Intervention/Treatment
Sorafenib group Patients assigned to this group received sorafenib post-transplantation.	Drug: Sorafenib 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). Other Names: Nexavar BAY 43-9006 BAY-673472 BAY 545-9085
Control group Patients assigned to this group did not receive sorafenib or any other FLT3 inhibitor post-transplantation until reaching the primary outcome.

Arm

Intervention/Treatment

Sorafenib group

Patients assigned to this group received sorafenib post-transplantation.

Drug: Sorafenib

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).

Other Names:

Nexavar

BAY 43-9006

BAY-673472

BAY 545-9085

Control group

Patients assigned to this group did not receive sorafenib or any other FLT3 inhibitor post-transplantation until reaching the primary outcome.

Outcome Measures

Primary Outcome Measures

Incidence of leukemia relapse [2 year]

Secondary Outcome Measures

Overall survival [2 year]
Leukemia-free survival [2 year]

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years to 60 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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)