# Clinical analysis of allogeneic hematopoietic stem cell transplantation for seven cases of acute myeloid leukemia with BCR::ABL1 fusion

**Authors:** 梦泽 郝, 小利 赵, 潇予 张, 圆圆 施, 明 龚, 利宁 张, 书连 陈, 嘉璘 魏, 祎 何, 四洲 冯, 明哲 韩, 尔烈 姜

PMC · DOI: 10.3760/cma.j.issn.0253-2727.2023.12.005 · Chinese Journal of Hematology · 2023-12-01

## TL;DR

This study examines the effectiveness of allogeneic stem cell transplantation in treating a rare form of acute myeloid leukemia with a specific genetic fusion.

## Contribution

The study provides clinical evidence that combining targeted therapy with stem cell transplantation improves outcomes in this rare leukemia subtype.

## Key findings

- Seven patients achieved complete molecular remission after allogeneic stem cell transplantation.
- Three-year overall survival rate was 80% with one death due to infection.
- Combining chemotherapy, TKI treatment, and transplantation improved outcomes.

## Abstract

探讨异基因造血干细胞移植（allo-HSCT）治疗急性髓系白血病伴BCR::ABL1（AML伴BCR::ABL1）患者的疗效。

纳入2012年11月至2022年1月在中国医学科学院血液病医院诊断为AML伴BCR::ABL1并接受allo-HSCT的7例患者，回顾性分析其临床资料。

7例患者起病时中位年龄35（21～40）岁，4例（57.1％）白细胞增高。7例患者BCR::ABL1融合基因均为阳性且伴有NPM1、RUNX1、ASXL1、PHF6基因突变。7例患者均接受诱导化疗联合酪氨酸酶抑制剂（TKI）靶向治疗并桥接allo-HSCT，移植后6例患者继续行TKI维持治疗。移植前6例获形态学完全缓解（CR），4例获完全分子生物学缓解（CMR），allo-HSCT后其余3例也获得CMR。1例患者移植后因感染死亡，6例无复发存活，移植后3年总生存率为（80.0±17.9）％。

诱导化疗联合TKI治疗AML伴BCR::ABL1疗效较好，桥接allo-HSCT可进一步提高CMR，移植后TKI维持治疗可能有助于改善预后。

## Linked entities

- **Genes:** BCR (BCR activator of RhoGEF and GTPase) [NCBI Gene 613], ABL1 (ABL proto-oncogene 1, non-receptor tyrosine kinase) [NCBI Gene 25], NPM1 (nucleophosmin 1) [NCBI Gene 4869], RUNX1 (RUNX family transcription factor 1) [NCBI Gene 861], ASXL1 (ASXL transcriptional regulator 1) [NCBI Gene 171023], PHF6 (PHD finger protein 6) [NCBI Gene 84295]
- **Chemicals:** TKI (PubChem CID 169447973)
- **Diseases:** acute myeloid leukemia (MONDO:0015667)

## Full-text entities

- **Genes:** RUNX1 (RUNX family transcription factor 1) [NCBI Gene 861] {aka AML1, AML1-EVI-1, AMLCR1, CBF2alpha, CBFA2, EVI-1}, NPM1 (nucleophosmin 1) [NCBI Gene 4869] {aka B23, NPM}, PHF6 (PHD finger protein 6) [NCBI Gene 84295] {aka BFLS, BORJ, CENP-31}, ASXL1 (ASXL transcriptional regulator 1) [NCBI Gene 171023] {aka BOPS, MDS}
- **Diseases:** AML (MESH:D015470), infection (MESH:D007239)
- **Species:** Homo sapiens (human, species) [taxon 9606]

## Full text

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## References

23 references — full list in the complete paper: https://tomesphere.com/paper/PMC10834871/full.md

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Source: https://tomesphere.com/paper/PMC10834871