Glioblastoma multiforme (GBM) is a rare, highly aggressive brain tumor associated with a poor outcome in both children and adults. Treatment usually involves a combination of surgical resection, chemotherapy, and radiotherapy, but ultimately it is incurable. Evidence suggests that congenital GBM may have a better prognosis with improved survival compared with GBM in older children. We describe the first known report of spontaneous resolution of a congenital GBM without any systemic therapy. A limited debulking procedure was performed at diagnosis, and the residual tumor underwent spontaneous resolution over the following 21 months. The patient remains in remission, with no tumor recurrence after 5 years of follow-up. Despite the tumor regressing, the patient has had an adverse neurologic outcome, with severe developmental delay and seizures. This case suggests that congenital GBM may be a separate biological entity much like neuroblastomas in infants, and therefore associated with better outcomes and even spontaneous resolution.
Copyright © 2016 by the American Academy of Pediatrics.