Cancer Rep (Hoboken). 2025 Aug;8(8):e70305. doi: 10.1002/cnr2.70305.
ABSTRACT
BACKGROUND: Alveolar soft part sarcoma (ASPS) is an extremely rare neoplasm that often presents with metastatic disease at diagnosis, leading to a poor prognosis. Conventional chemotherapy is generally ineffective against ASPS. Recent studies, nonetheless, suggest that tyrosine kinase inhibitors (TKIs) and immune checkpoint inhibitors (ICIs) may be effective treatment options.
CASE: We present a seven-year-old girl with stage IV ASPS in whom initial therapy with pazopanib, a TKI, had resulted in significant gastrointestinal side effects and poor drug compliance. We switched her treatment to an ICI regimen, a combination of nivolumab and ipilimumab, which led to a complete response.
CONCLUSION: This case highlights the value of ICIs as potential treatment options for ASPS in the pediatric population, especially in patients who cannot tolerate TKIs’ side effects.
PMID:40817777 | DOI:10.1002/cnr2.70305
