Acute Myeloid Leukemia in Children
Conditions
Keywords
Acute myeloid leukemia, relapsed/refractory, transcriptomic sequencing, drug sensitivity test
Brief 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.
Interventions
OTHERChemotherapy 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
Sponsors
College of Pharmaceutical Science at Zhejiang University
The Children's Hospital of Zhejiang University School of Medicine
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
No minimum to 18 Years
Healthy volunteers
Yes
Inclusion criteria
\- (1) Refractory recurrent acute myeloid leukemia (AML), and after second-line reinduction therapy, such as C+HAG did not reach complete CR.Criteria for complete response, refractory, and recurrence are as follows. (2) \<18 years old; (3) The child had good organ function, could tolerate chemotherapy, and had a physical strength score of 0-3 (WHO standard); (4) Understand the research procedures and voluntarily sign written informed consent.
Exclusion criteria
\- (1) Acute promyelocytic leukemia, chronic myelogenous leukemia, acute mixed cell leukemia or known central nervous system leukemia; (2) AML associated with congenital syndromes such as Down syndrome, Fanconi anemia, Bloom syndrome, Cole's syndrome, or congenital aplastic anemia; (3) secondary to immunodeficiency or positive for human immunodeficiency virus (HIV); (4) Cardiac and renal function were obviously abnormal, and left ventricular ejection fraction was \<50%. (5) There is active systemic infection; (6) any medical history or concomitant condition that the investigator believes would impair the subject's safe completion of the study; (7) The investigator considers that the subject is medically unfit to receive the investigational drug or is unfit for any other reason; (8) a known or suspected allergy to the subject drug or to any drug administered in connection with this test;
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Complete remission rate | 4 weeks | Complete remission rate after the course of individualized treatment |
| Event free survival | one year, five years | The patient's event free survival at one and five years after treatment |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Overall Survival | one year, five years | The patient's overall survival at one and five years after treatment |
Countries
China
Contacts
Primary ContactXiaojun Xu, MD
Backup ContactMeidan Ying, PhD
Outcome results
None listed