Case Report: Should IGF-1R targeted therapy be revisited in Ewing sarcoma? a report of long-term complete response and review of the literature

作者：Georgios M. Stergiopoulos, Brittany L. Siontis, Safia K. Ahmed, Judith Jebastin Thangaiah, Matthew T. Houdek, Thanh P. Ho, Scott H. Okuno, Steven I. Robinson · 发表于：Frontiers in Oncology · 年份：2025 · DOI：10.3389/fonc.2025.1667628 · 被引用次数：2 · 研究领域：Sarcoma Diagnosis and Treatment、Growth Hormone and Insulin-like Growth Factors、Pituitary Gland Disorders and Treatments

Introduction: Ewing sarcoma (ES) is a malignancy that mostly affects adolescents and young adults, with relapse or refractory cases posing major therapeutic challenges. Its unique transcriptional profile offers multiple targetable pathways, including the insulin-like growth factor-1 (IGF-1) receptor (IGF-1R) pathway. Case Report: We present the case of a 42-year-old female with recurrent ES with pulmonary metastases who, after progressing on anti-IGF-1R monotherapy with figitumumab (CP-751,871, NCT00560235), achieved complete remission in a phase I clinical trial (NCT00976508) that combined figitumumab IGF-1R-inhibition with growth hormone receptor antagonist pegvisomant. The patient has remained in long-term remission (>10 years) since the discontinuation of both agents and has not received any additional therapeutic interventions. Literature Review: We reviewed PubMed and the ClinicalTrials.gov database to identify clinical trials employing IGF-1R-targeted therapies in patients with ES and identified 24 relevant studies treating 723 patients with anti-IGF-1R therapy. Conclusion: This case represents the first report to our knowledge of patient outcomes following IGF-1R and growth hormone inhibition combination. The impressive response observed highlights the clinical synergy of this combination which warrants further clinical exploration as well as the potential of IGF-1R inhibition for ES. Additionally, this case suggests that targeted therapy discontinuation might be an o...