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Updated: Jul 2, 2025
A Mouse Model of Incompletely Resected Soft Tissue Sarcoma for Testing Neoadjuvant Therapies
Published on: July 28, 2020
Contemporary preclinical mouse models for pediatric rhabdomyosarcoma: from bedside to bench to bedside
Illya Martynov1,2, Lajwanti Dhaka1, Benedikt Wilke1
1Department of Pediatric Surgery and Urology, University Hospital Giessen-Marburg, Marburg, Germany.
Background:
Rhabdomyosarcoma (RMS) is the most common pediatric soft-tissue malignancy, characterized by high clinicalopathological and molecular heterogeneity. Preclinical in vivo models are essential for advancing our understanding of RMS oncobiology and developing novel treatment strategies. However, the diversity of scholarly data on preclinical RMS studies may challenge scientists and clinicians. Hence, we performed a systematic literature survey of contemporary RMS mouse models to characterize their phenotypes and assess their translational relevance.
Methods:
We identified papers published between 01/07/2018 and 01/07/2023 by searching PubMed and Web of Science databases.
Results:
Out of 713 records screened, 118 studies (26.9%) were included in the qualitative synthesis. Cell line-derived xenografts (CDX) were the most commonly utilized (n = 75, 63.6%), followed by patient-derived xenografts (PDX) and syngeneic models, each accounting for 11.9% (n = 14), and genetically engineered mouse models (GEMM) (n = 7, 5.9%). Combinations of different model categories were reported in 5.9% (n = 7) of studies. One study employed a virus-induced RMS model. Overall, 40.0% (n = 30) of the studies utilizing CDX models established alveolar RMS (aRMS), while 38.7% (n = 29) were embryonal phenotypes (eRMS). There were 20.0% (n = 15) of studies that involved a combination of both aRMS and eRMS subtypes. In one study (1.3%), the RMS phenotype was spindle cell/sclerosing. Subcutaneous xenografts (n = 66, 55.9%) were more frequently used compared to orthotopic models (n = 29, 24.6%). Notably, none of the employed cell lines were derived from primary untreated tumors. Only a minority of studies investigated disseminated RMS phenotypes (n = 16, 13.6%). The utilization areas of RMS models included testing drugs (n = 64, 54.2%), studying tumorigenesis (n = 56, 47.5%), tumor modeling (n = 19, 16.1%), imaging (n = 9, 7.6%), radiotherapy (n = 6, 5.1%), long-term effects related to radiotherapy (n = 3, 2.5%), and investigating biomarkers (n = 1, 0.8%). Notably, no preclinical studies focused on surgery.
Conclusions:
This up-to-date review highlights the need for mouse models with dissemination phenotypes and cell lines from primary untreated tumors. Furthermore, efforts should be directed towards underexplored areas such as surgery, radiotherapy, and biomarkers.
Insights
This review of pediatric rhabdomyosarcoma (RMS) mouse models found cell line-derived xenografts are common, but models with dissemination and primary tumor cell lines are needed. Future research should explore surgery, radiotherapy, and biomarkers.
Area of Science:
- Oncology
- Preclinical Research
- Translational Medicine
Background:
- Rhabdomyosarcoma (RMS) is a common pediatric soft-tissue cancer with significant heterogeneity.
- Preclinical in vivo models are crucial for understanding RMS biology and developing treatments.
- A comprehensive understanding of existing RMS models is needed due to data diversity.
Purpose of the Study:
- To systematically survey contemporary RMS mouse models.
- To characterize their phenotypes and assess translational relevance.
- To identify gaps in current preclinical research for RMS.
Main Methods:
- Systematic literature search of PubMed and Web of Science databases.
- Inclusion of studies published between July 1, 2018, and July 1, 2023.
- Qualitative synthesis of 118 selected studies.
Main Results:
- Cell line-derived xenografts (CDX) were the most frequent (63.6%), followed by patient-derived xenografts (PDX) and syngeneic models (11.9% each).
- Subcutaneous xenografts were more common than orthotopic models; few studies investigated disseminated RMS.
- Drug testing and tumorigenesis were primary research areas; surgery was notably absent.
Conclusions:
- There is a need for RMS mouse models that better represent tumor dissemination.
- Developing models using cell lines from primary, untreated tumors is essential.
- Further research should focus on surgery, radiotherapy, and biomarker investigation in preclinical RMS studies.
