Refractory ultra-high-risk Choriocarcinoma achieving complete response to intensive multi-agent and intrathecal...

Background: Choriocarcinoma is a highly aggressive gestational trophoblastic neoplasm. Patients with ultra-high-risk disease (FIGO score ≥ 13) and brain metastases face cure rates of only 50-80% and require intensive multimodal therapy. Case: We present the case of a 37-year-old woman with stage IV choriocarcinoma (FIGO score 14 ultra-high-risk) who presented with lung and brain metastases. Despite an initial response to standard chemotherapy, she experienced early relapse with progressive central nervous system disease. She received salvage treatment with intensive multi-agent escalated EP chemotherapy, intrathecal methotrexate, and pembrolizumab (anti-PD-1) introduced at cycle three and stereotactic radiosurgery for residual CNS disease, achieving a complete radiological and biochemical response sustained for over one year. Conclusion: This case highlights the challenges of managing ultra-high-risk gestational trophoblastic neoplasia and underscores the importance of multidisciplinary collaboration. The complete response achieved with the addition of pembrolizumab to dose-intensified, intrathecal, and high-dose chemotherapy supports the use of immune checkpoint inhibition in the management of refractory ultra-high-risk GTN.