Diagnosis
Anaplastic ependymoma, status post-surgical resection.
Patient:
3-year-old child, Uzbekistan
Physician:
Prof. Dr. Marc Remke, Chief of the Center for Pediatric Neuro-Oncology, University Hospital Saarland
Expert Opinion by Prof. Dr. Marc Remke
At present, we recommend treatment with temozolomide at a dosage of 150–200 mg/m² of body surface area, administered orally for 5 consecutive days, followed by a 28-day rest period. The initial treatment duration is planned for one year.
We are uncertain whether the child will be able to swallow tablets. If oral intake proves difficult, the medication can be prepared as an oral suspension and administered via a feeding tube.
This temozolomide regimen aims to strike a balance between therapeutic efficacy and tolerability, particularly when compared with more aggressive chemotherapy options. If robust clinical evidence existed demonstrating that intensive chemotherapy significantly improves outcomes in this setting, we would certainly consider recommending it.
Temozolomide offers a relatively straightforward treatment approach, often preserving a good quality of life with limited side effects. Maintaining a high quality of life during therapy is of utmost importance in pediatric care.
Unfortunately, as confirmed by our radiation oncology colleagues, re-irradiation—including with proton beam therapy—is not feasible in this case. Although re-irradiation is generally a valuable and effective local treatment option for recurrent ependymoma—especially when complete tumor resection cannot be achieved to ensure long-term control—it requires delivery of a sufficiently high radiation dose (typically >50 Gy) to be effective.
In this patient, both the primary and recurrent tumors are located in a highly sensitive anatomical region extending from the medulla oblongata down to the spinal cord at the C1 level. Delivering a curative radiation dose in this area carries an unacceptably high risk of severe neurological complications, particularly paraplegia. Therefore, definitive radiotherapy is not considered safe here.
Our radiation oncology team has indicated that, at this time, only palliative radiotherapy could be offered—but this would not provide meaningful long-term benefit for the child.
Meanwhile, tumor cells sent for cell culture ("PDX" or patient-derived xenograft modeling) are continuing to grow in the laboratory. We anticipate receiving the results of this analysis in the near future. Should these findings yield actionable insights, we will be prepared to adjust our treatment recommendations accordingly.