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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.
Abstract:
There is currently a lack of effective systemic treatment for patients with advanced pleomorphic rhabdomyosarcoma (PRMS). Although programmed death protein 1 (PD-1) inhibitors have shown efficacy in various solid tumors, their effects on PRMS have not been well established. Here, we present a case of a 12-year-old Chinese male adolescent with metastatic PRMS who benefited from the PD-1 inhibitor nivolumab. The patient initially underwent primary tumor resection but failed to respond to subsequent first-line chemotherapy and second-line pazopanib treatment. Pathological examination showed positive PD-L1 expression and tumor-infiltrating lymphocytes in the tumor tissue, and the patient was administered nivolumab as a posterior-line treatment. After attaining a clinically partial response (PR), surgical resection was performed, which was followed by adjuvant nivolumab. At the time of the submission of this manuscript, the patient achieved recurrence-free survival (RFS) lasting 45 months and counting. This is the first clinical evidence that a patient with refractory PRMS was controlled by anti-PD-1 antibody, with an RFS lasting more than 3 years. This case suggests that PD-L1 expression and T-cell infiltration could be used as potential biomarkers for PRMS immunotherapy.
Insights
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.
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.
