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 Publisher Pubmed



A Eghbali AZIZ ; M Obeidinia MOBIN ; Hs Mirmohammadi Hamideh SADAT ; R Rahimiafazal ROGHAYEH ; A Shirani ARYA
Source: Cancer Reports Published:2025

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. © 2025 Elsevier B.V., All rights reserved.