A Prospective Study of NUDT15/TPMT Multi-gene Combined Guidance on 6-MP Dosage During Maintenance Therapy in Childhood Acute Lymphoblastic Leukemia
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 发起方
- 入组人数
- 110
- 试验地点
- 1
- 主要终点
- Cumulative Incidence of Grade 3 or Higher Neutropenia
研究概览
简要总结
This is a prospective, single-center clinical study to evaluate the safety and efficacy of a personalized 6-mercaptopurine (6-MP) dosing strategy guided by NUDT15 and TPMT genotypes in children with Acute Lymphoblastic Leukemia (ALL) during maintenance therapy. The study compares this gene-guided strategy with historical controls to assess if it reduces the incidence of Grade ≥3 neutropenia and infection events.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 1 Year 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosed with ALL and scheduled for maintenance therapy. Plan to receive oral 6-MP. NUDT15 and TPMT genotyping results available. Baseline liver and kidney function within acceptable limits (ALT/AST ≤ 2.5xULN, etc.).
- •Signed informed consent. -
排除标准
- •Down syndrome. Relapsed/refractory disease before maintenance. Prior hematopoietic stem cell transplantation. Severe organ dysfunction or uncontrolled infection.
研究组 & 干预措施
Genotype-guided Dosing Group
All participants will receive 6-MP maintenance therapy with dosage adjustments based on their NUDT15 and TPMT genotype status.
干预措施: NUDT15/TPMT genotype-guided 6-MP dosing (Drug)
结局指标
主要结局
Cumulative Incidence of Grade 3 or Higher Neutropenia
时间窗: From the start of maintenance therapy up to Month 12
Defined according to NCI CTCAE v5.0 criteria. Grade ≥3 neutropenia is defined as an Absolute Neutrophil Count (ANC) \< 1000/mm³. The measure reports the percentage of participants who experience at least one episode of Grade ≥3 neutropenia.
次要结局
- Incidence of Severe Infection and Febrile Neutropenia (FN)(Up to 12 months from the start of maintenance therapy)
- Relative Dose Intensity (RDI) of 6-Mercaptopurine (6-MP)(Up to 12 months from the start of maintenance therapy)
研究者
xiatian
Attending Physician
The Children's Hospital of Zhejiang University School of Medicine