Case Report: Management of a BCR-ABL1-positive high-risk rhabdomyosarcoma patient using tyrosine kinase inhibitors

Rhabdomyosarcoma represents a prevalent type of soft tissue sarcoma encountered in pediatric patients. Despite multimodal intensified therapies encompassing surgical intervention, chemotherapy, and radiotherapy, the prognosis for patients with high-risk rhabdomyosarcoma remains notably unfavorable. To date, no definitive and efficacious molecularly targeted therapies have been established. This report describes the first documented case of a rhabdomyosarcoma patient harboring a positive <i>BCR-ABL1</i> fusion gene. At the time of initial diagnosis, the patient presented with a primary tumor in the right thigh and extensive metastatic involvement affecting both lungs, pleura, mediastinum, pelvic cavity, and the right inguinal region, resulting in the classification of the case as high-risk. In addition to conventional multimodal therapy, early intervention using tyrosine kinase inhibitors was implemented, leading to the achievement of an early complete response.

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Journal
Frontiers in Medicine
Published
2025-10-13
DOI
https://doi.org/10.3389/fmed.2025.1693688
Primary Topic
Chronic Myeloid Leukemia Treatments
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article
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article

Case Report: Management of a BCR-ABL1-positive high-risk rhabdomyosarcoma patient using tyrosine kinase inhibitors

Yu Liu, Dapeng Jiang, Anan Zhang, Fan Li et al.
Frontiers in Medicine
Chronic Myeloid Leukemia Treatments
article

Case Report: Management of a BCR-ABL1-positive high-risk rhabdomyosarcoma patient using tyrosine kinase inhibitors

Yu Liu, Dapeng Jiang, Anan Zhang, Fan Li, Yumin Zhong, Min Xu, Minzhi Yin, Yali Han
article en

Abstract

Rhabdomyosarcoma represents a prevalent type of soft tissue sarcoma encountered in pediatric patients. Despite multimodal intensified therapies encompassing surgical intervention, chemotherapy, and radiotherapy, the prognosis for patients with high-risk rhabdomyosarcoma remains notably unfavorable. To date, no definitive and efficacious molecularly targeted therapies have been established. This report describes the first documented case of a rhabdomyosarcoma patient harboring a positive <i>BCR-ABL1</i> fusion gene. At the time of initial diagnosis, the patient presented with a primary tumor in the right thigh and extensive metastatic involvement affecting both lungs, pleura, mediastinum, pelvic cavity, and the right inguinal region, resulting in the classification of the case as high-risk. In addition to conventional multimodal therapy, early intervention using tyrosine kinase inhibitors was implemented, leading to the achievement of an early complete response.

Frontiers in MedicineVol. 12
Shanghai Jiao Tong University (CN), Shanghai Children's Medical Center (CN)
Openalex Percentile: Top 63%
Chronic Myeloid Leukemia Treatments
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