Complete Response to Nivolumab and Ipilimumab After Pazopanib Discontinuation in a Seven-Year-Old Girl With Alveolar Soft Part Sarcoma: A Case Report and Literature Review.

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.