# Mitoxantrone hydrochloride liposome combined with cytarabine for treating pediatric acute myeloid leukemia with RUNX1∷MTG16 fusion gene: a case report and literature review

## TL;DR

A 13-year-old boy with a rare genetic form of leukemia achieved remission using a specific chemotherapy combination before a successful stem cell transplant.

## Contribution

Reports a novel treatment approach for pediatric AML with RUNX1∷MTG16 fusion gene using mitoxantrone hydrochloride liposome and cytarabine.

## Key findings

- The MA chemotherapy regimen induced complete remission and cleared genetic mutations temporarily.
- RUNX1∷MTG16 and FLT3-ITD mutations reappeared during consolidation therapy.
- The patient survived after receiving a matched sibling umbilical cord blood stem cell transplant.

## Abstract

回顾性分析中国医学科学院血液病医院2023年10月收治的1例急性髓系白血病（AML）伴RUNX1∷MTG16患儿，男性，13岁，因“乏力20 d”就诊，骨髓细胞形态学可见17.0％原始细胞，染色体核型46,XY,t（16;21）（q24;q22），RUNX1∷MTG16融合基因阳性，合并FLT3-ITD突变，诊断为AML（伴RUNX1∷MTG16）。经盐酸米托蒽醌脂质体联合阿糖胞苷（MA）方案诱导化疗达到完全缓解，序贯MA方案1个疗程后RUNX1∷MTG16融合基因及FLT3-ITD突变均转阴。但继续巩固强化治疗中融合基因及突变均转为阳性，行同胞全相合脐血造血干细胞移植存活。