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HSCT 前后 TKI 治疗融合驱动型 B-ALL:五例儿科、青少年和年轻成年患者的病例系列。

Pre-and post-HSCT use of TKI therapy for fusion-driven B-ALL: A case series of five pediatric, adolescent and young adult patients.

机构信息

School of Medicine, University of California, San Francisco, California, USA.

Department of Pediatrics, Benioff Children's Hospitals, University of California, San Francisco, California, USA.

出版信息

Cancer Rep (Hoboken). 2023 Dec;6(12):e1901. doi: 10.1002/cnr2.1901. Epub 2023 Nov 7.

Abstract

BACKGROUND

The development of tyrosine kinase inhibitors (TKIs) has significantly improved survival rates among patients with Philadelphia chromosome (Ph+) B cell acute lymphoblastic leukemia (B-ALL). Ph-like B-ALL patients lack the BCR::ABL1 translocation but share gene expression profiles with Ph+ B-ALL. The role of TKIs for Ph-like patients pre- and post-hematopoietic stem cell transplantation (HSCT) is not yet clear.

CASE

Here we present five cases of pediatric, adolescent, and young adult patients who presented with Ph-like B-ALL or CML in B-ALL blast phase who were treated with personalized TKI regimens pre- and post-HSCT.

CONCLUSION

This report describes several novel Ph-like fusions as well as combinations of TKIs with chemotherapy or immunotherapy not yet reported in the pediatric population. This case series provides real-world experience highlighting the potential application of pre- and post-HSCT use of TKIs in a subset of patients with targetable fusions.

摘要

背景

酪氨酸激酶抑制剂(TKIs)的发展显著提高了费城染色体(Ph+)B 细胞急性淋巴细胞白血病(B-ALL)患者的生存率。Ph-like B-ALL 患者缺乏 BCR::ABL1 易位,但与 Ph+B-ALL 具有相似的基因表达谱。在造血干细胞移植(HSCT)前后,TKIs 对 Ph-like 患者的作用尚不清楚。

病例

本研究报告了 5 例儿科、青少年和年轻成人患者,他们患有 Ph-like B-ALL 或 B-ALL 急变期的 CML,在 HSCT 前后接受了个体化 TKI 治疗方案。

结论

本报告描述了几种新的 Ph-like 融合,以及 TKI 与化疗或免疫治疗的联合应用,这些在儿科人群中尚未报道。该病例系列提供了真实世界的经验,强调了在具有可靶向融合的亚组患者中,HSCT 前后使用 TKI 的潜在应用。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/0a9e/10728537/6abbed48387c/CNR2-6-e1901-g001.jpg

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