# Efficacy and safety of venetoclax-cytarabine-homoharringtonine-based cytoreductive therapy before allogeneic hematopoietic stem cell transplantation in refractory/relapsed acute myeloid leukemia with RUNX1::RUNX1T1: a retrospective study

**Authors:** 子璐 张, 承伟 金, 肃 李, 璐祥 王, 佳瑜 黄, 传和 江, 海洋 陆, 晓霞 胡

PMC · DOI: 10.3760/cma.j.cn121090-20250312-00133 · Chinese Journal of Hematology · 2026-02-01

## TL;DR

A new treatment combining venetoclax, cytarabine, and homoharringtonine before a stem cell transplant shows promise for a rare type of leukemia.

## Contribution

A novel pre-transplant therapy for RUNX1::RUNX1T1 fusion AML is shown to be safe and effective.

## Key findings

- All five patients achieved molecular remission after the treatment.
- No patients had detectable residual disease after six months.
- All patients remained disease-free during follow-up.

## Abstract

回顾性分析2023年3月至2024年12月在上海交通大学医学院附属瑞金医院血液科接受含维奈克拉、阿糖胞苷和高三尖杉酯碱减瘤预处理桥接异基因造血干细胞移植（allo-HSCT）的5例伴RUNX1::RUNX1T1融合基因复发难治急性髓系白血病（AML）患者的临床结局。5例患者诊断至allo-HSCT中位时间为315（217～560）d，移植后6个月所有患者RUNX1::RUNX1T1融合基因定量转阴，中位转阴时间移植后2（1～6）个月。中位随访时间625（372～1 010）d，所有患者无病生存，无流式细胞术或分子学可测量残留病阳性事件，初步提示含维奈克拉、阿糖胞苷和高三尖杉酯碱减瘤预处理作为伴RUNX1::RUNX1T1融合基因复发难治AML移植前桥接方案安全有效。

## Linked entities

- **Genes:** RUNX1 (RUNX family transcription factor 1) [NCBI Gene 861], RUNX1T1 (RUNX1 partner transcriptional co-repressor 1) [NCBI Gene 862]
- **Chemicals:** venetoclax (PubChem CID 49846579), cytarabine (PubChem CID 6253), homoharringtonine (PubChem CID 285033)
- **Diseases:** acute myeloid leukemia (MONDO:0015667)

## Full-text entities

- **Genes:** BCL2L1 (BCL2 like 1) [NCBI Gene 598] {aka BCL-XL/S, BCL2L, BCLX, Bcl-X, PPP1R52}, KIT (KIT proto-oncogene, receptor tyrosine kinase) [NCBI Gene 3815] {aka C-Kit, CD117, MASTC, PBT, SCFR}, CD34 (CD34 molecule) [NCBI Gene 947], ABL1 (ABL proto-oncogene 1, non-receptor tyrosine kinase) [NCBI Gene 25] {aka ABL, BCR-ABL, CHDSKM, JTK7, bcr/abl, c-ABL}, MYC (MYC proto-oncogene, bHLH transcription factor) [NCBI Gene 4609] {aka MRTL, MYCC, bHLHe39, c-Myc}, BAX (BCL2 associated X, apoptosis regulator) [NCBI Gene 581] {aka BCL2L4}, MCL1 (MCL1 apoptosis regulator, BCL2 family member) [NCBI Gene 4170] {aka BCL2L3, EAT, MCL1-ES, MCL1L, MCL1S, Mcl-1}, BCL2 (BCL2 apoptosis regulator) [NCBI Gene 596] {aka Bcl-2, PPP1R50}, CD33 (CD33 molecule) [NCBI Gene 945] {aka CD33rSiglec, SIGLEC-3, SIGLEC3, p67}, NFKB1 (nuclear factor kappa B subunit 1) [NCBI Gene 4790] {aka CVID12, EBP-1, KBF1, NF-kB, NF-kB1, NF-kappa-B1}
- **Diseases:** HHT (MESH:D013683), HID (MESH:C566528), aGVHD,2 (MESH:D020803), AML (MESH:D015470), (8;21 (OMIM:614172)
- **Chemicals:** venetoclax (MESH:C579720), cytarabine (MESH:D003561), homoharringtonine (MESH:D000077863)
- **Species:** Homo sapiens (human, species) [taxon 9606]

## Full text

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## References

28 references — full list in the complete paper: https://tomesphere.com/paper/PMC13008556/full.md

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Source: https://tomesphere.com/paper/PMC13008556