This is a prospective, single-arm, open-label clinical study to evaluate the efficacy and safety of Selinexor combined with a standardized induction chemotherapy regimen (ZJCH-AML) for children with NUP98-fusion positive Acute Myeloid Leukemia (AML). NUP98-fusion positive AML is a high-risk subtype in pediatric patients, characterized by poor prognosis and high relapse rates under traditional chemotherapy. Preliminary research suggests that Selinexor, an XPO1 inhibitor, can selectively inhibit NUP98-driven leukemia cells. This study aims to determine if adding Selinexor to standardized chemotherapy can increase the complete remission rate and improve the overall survival for these specific high-risk patients.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
10
Induction Course 1: 40mg/m² orally on Days 1, 3, 8, and 10. Induction Course 2: 40mg/m² orally on Days 1 and 3.
Course 1 (HAG): Homoharringtonine (HHT) 2mg/m²/d, Cytarabine (Ara-C) 10mg/m² q12h, and G-CSF 200μg/m²/d for 14 days. Course 2 (Risk-stratified): Based on the response to Course 1, patients receive HDA (for CR), DAC+HDA (for PR), or CLAG (for NR).
Children's Hospital, Zhejiang University School of Medicine
Hangzhou, Zhejiang, China
Complete Remission (CR) Rate
Percentage of patients achieving CR after Induction Course 1 or 2. CR is defined as bone marrow blasts \<5%, ANC \> 1\\times10\^9/L, and PLT \> 80\\times10\^9/L.
Time frame: Day 14 of Induction Course 2 (approximately 2 months).
Minimal Residual Disease (MRD) Negative Rate
The proportion of patients reaching MRD negativity (threshold \< 0.1%) measured by flow cytometry or molecular methods.
Time frame: Day 14 of Induction Course 1 (approximately 28 days).
Overall Survival (OS)
The time from the date of enrollment to death from any cause.
Time frame: 1 year.
Event-Free Survival (EFS)
The time from the date of enrollment to events including treatment failure, relapse, or death.
Time frame: 1 year.
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