# Case Report: Rapid response to gemtuzumab-ozogamicin in a pediatric patient with refractory systemic mastocytosis with AML1::ETO+ acute myeloid leukemia

**Authors:** Song Xue, Man Chen, Hui-Peng Sun, Tong Wang, Li-Na Zhang, Xing-Yu Cao

PMC · DOI: 10.3389/fimmu.2025.1566805 · 2025-04-09

## TL;DR

A 6-year-old child with a rare and resistant form of leukemia showed quick improvement after treatment with gemtuzumab-ozogamicin.

## Contribution

This case report highlights the potential of gemtuzumab-ozogamicin as a salvage therapy for refractory systemic mastocytosis with AML1::ETO+ AML.

## Key findings

- Gemtuzumab-ozogamicin induced rapid remission in a pediatric patient with refractory systemic mastocytosis and AML.
- Standard salvage treatments failed, but gemtuzumab-ozogamicin was effective in this rare case.

## Abstract

This article describes a 6-year-old patient diagnosed with “systemic mastocytosis with AML1::ETO+ AML”, he experience refractory disease during the course of treatment and salvage treatment was ineffective. The patient was administered gemtuzumab-ozogamicin therapy, resulting in rapid remission.

## Linked entities

- **Genes:** RUNX1 (RUNX family transcription factor 1) [NCBI Gene 861], RUNX1T1 (RUNX1 partner transcriptional co-repressor 1) [NCBI Gene 862]
- **Diseases:** systemic mastocytosis (MONDO:0016586), acute myeloid leukemia (MONDO:0015667)

## Full-text entities

- **Diseases:** AML (MESH:D015470), systemic mastocytosis (MESH:D034721)
- **Species:** Homo sapiens (human, species) [taxon 9606]

## Figures

3 figures with captions in the complete paper: https://tomesphere.com/paper/PMC12014624/full.md

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Source: https://tomesphere.com/paper/PMC12014624