Long-term Survival (>13 Years) in a Child With Recurrent Diffuse Pontine Gliosarcoma

Abstract

Pediatric gliosarcoma (GS) is a rare variant of glioblastoma multiforme. The authors describe the case of an unusual pontine location of GS in a 9-year-old boy who was initially diagnosed with low-grade astrocytoma (LGA) that was successfully controlled for 4 years. Subsequently, his brain tumor transformed into a GS. Prior treatment of his LGA included subtotal tumor resection 3 times, standard radiation therapy, and Gamma Knife procedure twice. His LGA was also treated with a standard chemotherapy regimen of carboplatin and vincristine, and his GS with subtotal resection, high-dose cyclophosphamide, and thiotepa with stem cell rescue and temozolomide. Unfortunately, he developed disseminated disease with multiple lesions and leptomeningeal involvement including a tumor occupying 80% of the pons. Upon presentation at our clinic, he had rapidly progressing disease. He received treatment with antineoplastons (ANP) A10 and AS2-1 for 6 years and 10 months under special exception to our phase II protocol BT-22. During his treatment with ANP his tumor stabilized, then decreased, and, ultimately, did not show any metabolic activity. The patient’s response was evaluated by magnetic resonance imaging and positron emission tomography scans. His pathology diagnosis was confirmed by external neuropathologists, and his response to the treatment was determined by central radiology review. He experienced the following treatment-related, reversible toxicities with ANP: fatigue, xerostomia and urinary frequency (grade 1), diarrhea, incontinence and urine color change (grade 2), and grade 4 hypernatremia. His condition continued to improve after treatment with ANP and, currently, he complains only of residual neurological deficit from his previous surgery. He achieved a complete response, and his overall and progression-free survival is in excess of 13 years. This report indicates that it is possible to obtain long-term survival of a child with a highly aggressive recurrent GS with diffuse pontine involvement with a currently available investigational treatment.

Complete Response and Long-Term Survival (>20 Years) of a Child with Tectal Glioma: A Case Report

“…Surgery and biopsy were not advised in this case since some reports indicated rapid tumor growth after surgical intervention [6,7]. Based on prior experience in the treatment of both benign and aggressive pediatric gliomas, the patient was admitted to clinical study according to Protocol BT-09 [4,5,8]. He accomplished durable CR with long-term survival (over 20 years from tumor diagnosis).…”

“…This report suggests that it is possible to obtain long-term overall survival in patients suffering from tectal glioma with an aggressive course of the disease. The results of phase II studies of ANP are described in a number of articles [8,9,10]. …”

Long-Term Survival Over 21 Years and Pathologically Confirmed Complete Response in Pediatric Anaplastic Astrocytoma: A Case Report

“…ANP A10 capsules contain 500 mg of 3-phenylacetylamino-2,6-piperidinedione, which is metabolized in the body to 4:1 ratio of PG, and isoPG and AS2-1 capsules contain 500 mg of 4:1 ratio of PN and PG [6]. Interim results of the phase II studies have already been reported as well as case reports of long-term survival of HGG and diffuse intrinsic pontine glioma (DIPG) [3,[7][8][9][10][11][12][13][14][15][16]. Here, we discuss a case of very long survival (over 21 years) of PAA with pathologically-confirmed complete response (CR).…”

A Phase II Study of Antineoplastons A10 and AS2-1 in Children with High-Grade Glioma. Final Report (Protocol BT-06), and Review of Recent Trials

“…AS2-1 is a formulation with a 4:1 ratio of phenylacetate sodium (PN) and PG [6]. Initial encouraging clinical responses in the treatment of HGG led to the design of this single-arm Phase II study to assess the efficacy and safety of ANP therapy in children diagnosed with HGG [6]- [11].…”