Acute Myeloid Leukemia, Chronic Myelomonocytic Leukemia, Myelodysplastic Syndrome
Conditions
Brief summary
Efficacy and Safety of Selinexor Combined with Azacitidine as Maintenance Therapy in High-Risk Myeloid Neoplasms Patients Post-Transplantation: A Single-Center, Single-Arm, Exploratory Study
Detailed description
Treatment period: From the time of transplantation, after screening by inclusion and exclusion criteria, patients who meet the criteria are enrolled and given maintenance therapy with Selinexor in combination with azacitidine (Selinexor: 40 mg/weekly, administered on D1; azacitidine: 50 mg/m2\*5d; every 28 days for a cycle of treatment, for at least one year of medication, or until progression of the disease, or until the development of intolerable toxicity, whichever comes first)
Interventions
DRUGSelinexor
maintenance therapy with Selinexor combined with Azacitidine in patients with high-risk myeloid neoplasms after transplantation
Sponsors
Institute of Hematology & Blood Diseases Hospital, China
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
16 Years to 75 Years
Healthy volunteers
No
Inclusion criteria
with pre-transplant MRD-positive AML, i.e., patients who meet one of the following criteria: 1. Age≥ 16 years old; any gender; 2. High-risk MDS (IPSS-R or/and IPSS-M high-risk and above); 3. High relapse risk AML, including relapsed refractory AML and patients with pre-transplant MRD-positive AML, i.e., patients who meet one of the following criteria: (1)Refractory AML is defined as meeting one of the following conditions 1. Primary cases that have failed to respond to 2 courses of standard regimen chemotherapy; 2. CR was followed by consolidation and intensive treatment with no recurrence within 12 months; 3. It recurred 12 months later, but conventional chemotherapy was ineffective; 4. 2 or more relapses; 5. Extramedullary leukemia persists. (2)Relapsed AML i.e., re-initialization of leukemic cells in the peripheral blood after complete remission (CR) or bone marrow primitive cells \>0.050 (except for other reasons such as bone marrow reconstitution after consolidation chemotherapy) or extramedullary infiltration of leukemic cells. (3)Positive pre-transplant MRD, i.e., one of the following conditions is met: 1. Proportion of abnormal myeloid cells \>0.01% by pre-transplant flow assay; 2. Positive pre-transplantation molecular biology-related tests. (4)AML with poor prognosis (according to the Chinese Guidelines for the Diagnosis and Treatment of Adult Acute Myeloid Leukemia (Non-Acute Promyelocytic Leukemia) 2023 Edition) 4.Meets WHO diagnostic criteria for staging CMML; 5.MDS or CMML transforms AML.
Exclusion criteria
Patients with any of the following are not eligible for enrollment in this study: 1. Patients who relapsed within 3 months of transplantation or during maintenance therapy, including hematologic, molecular genetic, cytogenetic relapses, and extramedullary relapses. 2. Known positive serology for HIV or active hepatitis B virus (HBV) and hepatitis C virus (HCV); 3. Requirements for mental illness or other conditions that preclude cooperation with study treatment and monitoring; 4. Patients who are pregnant or who are unable to use appropriate contraception during treatment; 5. Suspected hypersensitivity to the experimental drug or any of its excipients; 6. Active heart disease, defined as one or more of the following: （1）History of uncontrolled or symptomatic angina; （2）Myocardial infarction less than 6 months from study entry; （3）History of tardive dyskinesia requiring medication or clinically significant symptoms; （4）Uncontrolled or symptomatic congestive heart failure (\> NYHA class 2) （5）Ejection fraction is below the lower limit of the normal range. 7. Those deemed unsuitable for enrollment by the investigator.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 2-year recurrence free survival rate (RFS) | 2 years | the rate at which a patient has been on the drug for at least 1 year without morphological relapse or death of the subject from the transplantation date (whichever comes first) |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| 2-year non-relapse mortality rate (NRM) | 2 years | incidence of death from causes other than AML relapse/progression from the date of transplantation to the date of the subject's death |
| 2-year survival rate (OS) | 2 years | the 2-year survival rate from transplantation to death from any causes |
| Median overall survival (OS) | 2 years | The time from transplantation to death from any causes |
| Cumulative incidence of acute graft-versus-host disease (GVHD) | 2 years | Cumulative incidence of grade II-IV acute GVHD at 6 months after enrollment. Acute GVHD will be graded based on the diagnostic and severity scores used by the Blood and Bone Marrow Transplantation Clinical Trial Network (BMTCTN). |
Countries
China
Contacts
Primary ContactErlie jiang
Outcome results
None listed