School of Medicine, University of California, San Francisco, California, USA.
Department of Pediatrics, Benioff Children's Hospitals, University of California, San Francisco, California, USA.
Cancer Rep (Hoboken). 2023 Dec;6(12):e1901. doi: 10.1002/cnr2.1901. Epub 2023 Nov 7.
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.
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.
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.
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。
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。
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。
