Abstract This abstract details the clinical outcomes of three pediatric patients diagnosed with midline pilocytic astrocytoma, highlighting their positive responses to chemotherapy. Patient 1 is a 4-year-old boy who presented with a decline in gait, left-sided hemiplegia, and vision changes. An MRI showed an heterogenous mass in the midbrain with a cystic component. Biopsy was done and pathology confirmed a juvenile pilocytic astrocytoma, WHO grade 1, with BRAF duplication fusion. He began chemotherapy with vincristine and carboplatin. MRI showed interval reduction in the midbrain tumor without evidence of growth or tumor recurrence. Patient 2 is a 3-year-old boy diagnosed with a cervicothoracic intramedullary spinal cord tumor. After biopsy and resection, pathology confirmed the diagnosis of a pilocytic astrocytoma. Given the longstanding tumor and left arm weakness, he was treated with carboplatin monotherapy to avoid neurotoxicity from vincristine. An MRI was done six months after finishing treatment and showed a decrease in the cervicothoracic lesion size. Patient 3 is a 5-year-old girl found to have an expansile medullary mass on MRI. Biopsy confirmed exophytic medullary pilocytic astrocytoma with BRAF duplication/fusion. She began carboplatin monotherapy due to vocal cord paralysis and completed treatment one year later. The MRI at the end of therapy showed a reduction in the cystic component and a stable solid component of the tumor, indicating an excellent therapeutic response. These cases underscore the effectiveness of chemotherapy in managing spinal cord and midline pilocytic astrocytoma in pediatric patients, demonstrating favorable outcomes. The vincristine and carboplatin regimen has shown promise in targeting BRAF-altered CNS tumors and may also serve as a first-line therapy for spinal cord tumors.
Valdes et al. (2025) studied this question.