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
{"title":"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","authors":"Aziz Eghbali, Mobin Obeidinia, Hamideh Sadat Mirmohammadi, Roghayeh Rahimiafazal, Arya Shirani","doi":"10.1002/cnr2.70305","DOIUrl":null,"url":null,"abstract":"<div>\n \n \n <section>\n \n <h3> Background</h3>\n \n <p>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.</p>\n </section>\n \n <section>\n \n <h3> Case</h3>\n \n <p>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.</p>\n </section>\n \n <section>\n \n <h3> Conclusion</h3>\n \n <p>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.</p>\n </section>\n </div>","PeriodicalId":9440,"journal":{"name":"Cancer reports","volume":"8 8","pages":""},"PeriodicalIF":1.9000,"publicationDate":"2025-08-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/cnr2.70305","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Cancer reports","FirstCategoryId":"1085","ListUrlMain":"https://onlinelibrary.wiley.com/doi/10.1002/cnr2.70305","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q4","JCRName":"ONCOLOGY","Score":null,"Total":0}
引用次数: 0
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.
肺泡软部肉瘤(Alveolar soft part sarcoma, ASPS)是一种极为罕见的肿瘤,诊断时常伴有转移性疾病,预后较差。常规化疗通常对ASPS无效。然而,最近的研究表明,酪氨酸激酶抑制剂(TKIs)和免疫检查点抑制剂(ICIs)可能是有效的治疗选择。我们报告了一名7岁的IV期ASPS女孩,她最初使用帕唑帕尼(一种TKI)治疗,导致了明显的胃肠道副作用和药物依从性差。我们将她的治疗改为ICI方案,即纳武单抗和伊匹单抗的联合治疗,这导致了完全的缓解。结论本病例强调了ICIs作为儿科人群ASPS的潜在治疗选择的价值,特别是对于不能耐受tki副作用的患者。