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Cancer is the second leading cause of death in the United States. A cancer cell is genetically unstable and hence can mutate faster. They can also modify their microenvironment and escape immune surveillance. The difficulties in treating cancer are further compounded by the emergence of rapid resistance to anticancer drugs. The most common ways to attain resistance in cancer cells include alteration in drug transport and metabolism, modification of drug target, elevated DNA damage response, or...

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Related Experiment Video

Updated: Jul 23, 2026

A Mouse Model of Incompletely Resected Soft Tissue Sarcoma for Testing Neoadjuvant Therapies

A Mouse Model of Incompletely Resected Soft Tissue Sarcoma for Testing Neoadjuvant Therapies

Published on: July 28, 2020

Case Report: A PD-L1-Positive Patient With Pleomorphic Rhabdomyosarcoma Achieving an Impressive Response to

Jiayong Liu¹, Peijie Liu², Fuyu Gong³

¹Key Laboratory of Carcinogenesis and Translational Research (Ministry of Education/Beijing), Department of Bone and Soft Tissue Tumor, Peking University Cancer Hospital & Institute, Beijing, China.

Frontiers in Immunology

|April 4, 2022

Summary

This case study shows a 12-year-old with advanced pleomorphic rhabdomyosarcoma (PRMS) achieved long-term remission with nivolumab, a PD-1 inhibitor. Positive PD-L1 expression and T-cell infiltration may predict treatment success in PRMS.

Keywords:

CD8 T cell PD-L1 immunotherapy pazopanib pleomorphic rhabdomyosarcoma

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Area of Science:

Oncology
Immunotherapy

Background:

Advanced pleomorphic rhabdomyosarcoma (PRMS) lacks effective systemic treatments.
Programmed death protein 1 (PD-1) inhibitors show promise in solid tumors but are not well-established for PRMS.

Observation:

A 12-year-old male with metastatic PRMS showed resistance to chemotherapy and pazopanib.
Tumor tissue exhibited positive PD-L1 expression and tumor-infiltrating lymphocytes.

Findings:

Nivolumab treatment led to a partial response, enabling surgical resection.
The patient achieved over 45 months of recurrence-free survival (RFS) with adjuvant nivolumab.
This is the first report of a refractory PRMS patient benefiting from PD-1 inhibition for over 3 years.

Implications:

PD-1 inhibitor therapy, specifically nivolumab, can be effective in refractory PRMS.
PD-L1 expression and T-cell infiltration may serve as predictive biomarkers for PRMS immunotherapy.
Further research into PD-1 inhibitors for PRMS is warranted.