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Individualized Treatment of Pediatric R/R AML Based on Transcriptomic Profile and in Vitro Drug Sensitivity Test
Sponsor: The Children's Hospital of Zhejiang University School of Medicine
Summary
Acute myeloid leukemia (AML) accounts for about 15% to 20% of childhood leukemia, but the death rate accounts for about 50%. About 20-30% of children with AML did not achieve complete response (CR) after 2 induction treatments, and about 30% of children with CR had relapse within 3 years (including recurrence after hematopoietic stem cell transplantation).Relapsed/refractory (R/R) AML is a major cause of treatment failure and refractory survival. Reinduction chemotherapy for R/R-AML to obtain CR again, followed by hematopoietic stem cell transplantation, is the current treatment. At present, there is no recognized reinduction protocol, and the reinduction remission rate of R/R-AML varies greatly among different treatment regimens, ranging from 23 to 81%. Current guidelines recommend a new combination chemotherapy regimen consisting of new drugs without cross-resistance. This method selects sensitive chemotherapeutic drugs, and then forms a new combination chemotherapy regimen according to the characteristics of drugs, which is the choice of R/R-AML reinduction therapy.This study intends to conduct a clinical study on the individualized treatment of R/R AML patients through in vitro drug sensitivity test combined with patient transcriptomic characteristics.
Official title: Individualized Treatment of Pediatric Relasped and Refractory Acute Myeloid Leukemia Based on Transcriptomic Profile and in Vitro Drug Sensitivity Test
Key Details
Gender
All
Age Range
Any - 18 Years
Study Type
INTERVENTIONAL
Enrollment
60
Start Date
2024-01-01
Completion Date
2025-12-31
Last Updated
2024-07-03
Healthy Volunteers
Yes
Conditions
Interventions
Chemotherapy regiments based on the transcriptomic profile and in vitro drug sensitivity test
1. Leukemia cells from newly diagnosed and relapsed children with AML were tested for high throughput in vitro drug sensitivity and resistance. 2. Combined with multi-omics sequencing technology such as whole exome and transcriptome, the data of drug sensitivity, drug resistance and genomic characteristics of children AML patients were integrated, and the internal rules between drug sensitivity or drug resistance and molecular characteristics such as gene fusion, gene mutation and abnormal gene expression of children AML patients were deeply analyzed, and the molecular characteristics of drug sensitivity in children AML were mapped
Locations (1)
Children's Hospital of Zhejiang University School of Medicine
Hangzhou, Zhejiang, China