EOLP-04. EXTRA-NEURAL DISSEMINATION OF MYC-ACTIVATED INFANT MEDULLOBLASTOMA DESPITE MULTIMODAL THERAPY

Abstract

Abstract Extra neural metastases of infant medulloblastoma portend poor prognosis. Previous reports were in patients receiving treatment with radiation alone or patients with ventriculoperitoneal shunt (VPS). Recently with multimodal therapy, reports of extra-neural medulloblastoma are rarely reported. We report two patients with stage M3 MYC-Activated Infant Medulloblastoma who received aggressive therapy yet developed extra-neural relapses. The first case was a 2-year-old male who underwent gross total resection, which was complicated by hydrocephalus requiring VPS placement. He completed treatment as per ACNS0334 and achieved a near-complete response. Next, he received four cycles of five drug anti-angiogenic therapy followed by 2 cycles of expanded access therapy with I-3F8 antibody. He progressed both in brain and spinal cord, then received palliative craniospinal irradiation with boost. Two months following radiation, he died of disease with significant pancytopenia and hypoechoic nodules throughout the liver, suggestive of disseminated disease. Our second patient is a five-year-old girl, who was diagnosed at age two. She achieved a CR after treatment as per ACNS0334. She then enrolled on a clinical trial with Difluoromethylornithine (DFMO) for three months. On initial disease evaluation, she had progression in brain and spine. She underwent palliative radiation with craniospinal irradiation and boost with concurrent Indoximod on phase 2 clinical trial. Following radiation, she received compassionate use indoximod and temadar for 24 months and remained in CR. Three months after stopping therapy, she progressed to left orbit, pelvis and ribs. She is currently receiving palliative care. MYC-activated medulloblastoma has a very poor outcome and requires multimodal including radiation to achieve remission. Despite aggressive therapy, disease is highly evasive and can spread extracranially to multiple areas including bones, bone marrow, liver, and abdomen. Novel therapies and more exhaustive surveillance regiments need to be developed to limit extracranial dissemination and achieve more durable remissions.