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Prolonged response to carboplatin in an infant with brain stem glioma
P M Zeltzer1, K Epport, M D Nelson
1Department of Hematology/Oncology, Children's Hospital of Los Angeles, CA 90054-0700.
Insights
A 15-month-old child with brain stem glioma showed a prolonged response to carboplatin chemotherapy, avoiding radiation therapy (XRT). This suggests chemotherapy without XRT may be viable for young patients with this tumor.
Area of Science:
- Pediatric Oncology
- Neuro-oncology
- Radiation Oncology
Background:
- Brain stem gliomas in children typically have a poor prognosis, with a mean survival of 15 months post-radiation therapy (XRT).
- Radiation therapy in infants poses risks of neurotoxicity to the developing brain, complicating treatment decisions.
- Standard treatment protocols often involve radiation, necessitating exploration of alternative approaches for this vulnerable population.
Observation:
- A 15-month-old infant with biopsy-proven, progressive brain stem glioma was treated with carboplatin chemotherapy.
- The patient received 24 monthly courses of carboplatin without radiation therapy.
- Clinical and radiographic assessments were used to monitor disease progression and treatment response.
Findings:
- The patient achieved a durable response lasting over 39 months.
- Clinical improvement was noted within 3 months, with radiographic evidence of response confirmed by magnetic resonance imaging at 10 months.
- Carboplatin chemotherapy demonstrated minimal toxicity in this young patient.
- Longitudinal neuropsychological assessments showed continued improvement, though some motor deficits persisted.
Implications:
- Chemotherapy without radiation therapy presents a promising alternative for infants with brain stem gliomas, potentially mitigating long-term neurodevelopmental effects.
- Cervico-medullary astrocytoma in young children may represent a suitable clinical context for future trials investigating chemotherapy-exclusive treatment strategies.
- This case highlights the potential for effective disease control and improved quality of life by adapting treatment paradigms for pediatric brain tumors.
Abstract:
Adults and children with brain stem gliomas have a mean survival time of 15 months after radiation therapy (XRT). Infants with this tumor present additional complexities for treatment because of possible neurotoxicity of the radiation to the developing brain. We report a 15-month-old child with biopsy-proven brain stem glioma with clinical and radiographic evidence of disease progression. She was treated with 24 monthly courses of carboplatin without radiation therapy and has had a 39+ month response. The clinical response started after 3 months and the radiographic evidence was documented at 10 months by magnetic resonance imaging. The toxicity was minimal. Longitudinal neuropsychological assessment demonstrated continued improvement at 36 months post diagnosis but with some motor functioning below expected age levels. Cervico-medullary astrocytoma in a young patient may be the appropriate clinical setting for future trials of chemotherapy without XRT.