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Updated: Aug 24, 2026
Tumorsphere Derivation and Treatment from Primary Tumor Cells Isolated from Mouse Rhabdomyosarcomas
Published on: September 13, 2019
Exploring drug sensitivity guided individualized treatment in pediatric spindle cell/sclerosing rhabdomyosarcoma
Yi-Tian Chang1,2, Yuan Zhang1, Yu-Tong Zhang1
1Department of Pediatric Oncology, Children's Medical Center, The First Hospital of Jilin University, Changchun, Jilin, China.
Purpose:
Spindle cell and sclerosing rhabdomyosarcoma (sRMS/scRMS) are rare variants accounting for 5%-10% of all RMS cases. This study aimed to evaluate the feasibility of using drug sensitivity assay to guide real time treatment decisions in children with sRMS/scRMS.
Methods:
We retrospectively analyzed 14 consecutive patients diagnosed with sRMS/scRMS between January 2020 and December 2024. All patients initially received two cycles of VAC (vincristine/dactinomycin/cyclophosphamide). If the tumor response is stable or progressive, subsequent treatment is tailored based on drug sensitivity assay. The primary endpoint was feasibility of drug sensitivity assay guided decision making. Secondary endpoints included objective response rate (ORR), PFS, and OS.
Results:
Drug sensitivity assay revealed uniform platinum sensitivity across all patients. After two cycles of VAC, 13 of 14 patients exhibited either stable or progressive disease, suggesting limited efficacy of the VAC regimen in this cohort. These 13 patients were subsequently transitioned to a platinum containing regimen guided by drug sensitivity assay, following which an objective response was observed in all cases. The 5-year PFS and OS were 36.92% (95% CI, 11.82-61.33) and 53.85% (95% CI, 31.70-82.93), respectively. Compared with the historical median survival of 9 months for relapsed/refractory RMS, the outcomes in our cohort appeared to be somewhat longer, suggesting a potential clinical benefit. The approach proved to be feasible in all 14 patients.
Conclusion:
Our findings suggest that drug sensitivity assay may represent a feasible and potentially useful tool for informing real time treatment decisions in children with sRMS/scRMS. The incorporation of such testing into the therapeutic algorithm for this chemotherapy resistant sarcoma variant may offer a means to individualize treatment and possibly improve prognostic outcomes. However, given the exploratory nature and limited sample size of this study, these observations should be interpreted with caution, and larger prospective studies are needed to confirm their validity.
Insights
Drug sensitivity assays are feasible for guiding treatment in spindle cell/sclerosing rhabdomyosarcoma (sRMS/scRMS). This approach showed promise in tailoring chemotherapy for children with this rare sarcoma variant.
Area of Science:
- Pediatric Oncology
- Sarcoma Research
- Clinical Trial Design
Background:
- Spindle cell and sclerosing rhabdomyosarcoma (sRMS/scRMS) are rare subtypes of rhabdomyosarcoma.
- These variants constitute 5%-10% of all rhabdomyosarcoma cases.
- Effective treatment strategies for sRMS/scRMS remain an area of active investigation.
Purpose of the Study:
- To assess the feasibility of employing drug sensitivity assays for real-time treatment adjustments in pediatric sRMS/scRMS.
- To evaluate the potential of personalized medicine in managing this rare sarcoma subtype.
Main Methods:
- Retrospective analysis of 14 pediatric patients with sRMS/scRMS.
- Initial treatment with two cycles of vincristine/dactinomycin/cyclophosphamide (VAC).
- Subsequent treatment tailored by drug sensitivity assay for patients with stable or progressive disease.
Main Results:
- Drug sensitivity assays consistently indicated platinum sensitivity in all patients.
- 13 out of 14 patients showed stable or progressive disease after VAC, highlighting limited efficacy.
- All 13 patients receiving platinum-based therapy guided by assay demonstrated objective responses.
- Five-year progression-free survival (PFS) and overall survival (OS) were 36.92% and 53.85%, respectively.
- The drug sensitivity assay-guided approach was feasible in all patients.
Conclusions:
- Drug sensitivity assays offer a feasible method for guiding treatment decisions in pediatric sRMS/scRMS.
- Personalized treatment strategies based on drug sensitivity testing may improve outcomes for this chemotherapy-resistant sarcoma.
- Larger prospective studies are warranted to validate these findings due to the study's exploratory nature and small sample size.
