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Apatinib and temozolomide in children with recurrent ependymoma: A case report
Shuangshuang Zhao1, Zhipeng Shen2, Juan Li1
1Department of Radiation Oncology, Hangzhou Cancer Hospital, Hangzhou, Zhejiang, P.R. China.
Rationale:
Outcomes remain poor in children with recurrent ependyomams (rEPNs), despite advances in surgery and radiotherapy. Systemic therapeutic options are limited, given the low response to chemotherapy and targeted drugs. There is an urgent need for efective pharmacotherapy. Apatinib is a multitarget tyrosine kinase inhibitor, which has been reported to exhibit broad antitumor profiles. However, its effects on rEPNs have not been reported thus far.
Patient Concerns:
We present a 5-year-old recurrent ependyomam patient benefting from apatinib and temozolomide. The patient was diagnosed with ependyomam in January 2016 and treated with surgery and radiotherapy. After surgery, the patient walked with an mild unsteady gait. He was diagnosed with recurrence in November 2018 following which he was treated with reoperation, reirradiation and chemotherapy (etopside, cisplatin, and temozolomide [TMZ]). The patients increased gait instability in April 2019.
Diagnoses:
Magnetic resonance imaging (MRI) showed progression of the disease. The lession at the left edge of the fourth ventricle and cerebellar peduncles was significantly increased.
Interventions:
The patient was administer TMZ (200 mg/m2/d, d1-5, 28 days as a cycle) + apatinib (250 mg, every other day). Twelve cycle of TMZ and apatinib were given.
Outcomes:
The tumor significantly shrank during the patient received TMZ and apatinib. After 9 months of medication, MRI revealed a nearly complete response However, the tumor progressed on May 5, 2020. From the beginning of the application of TMZ and apatinib, the progression-free survival was 1 year and the survival time was 19 months. Grade 1 leukocytopenia was observed without other adverse effects.
Conclusion:
Apatinib and temozolomide treatment with mild side effects may be a new option for children with recurrent ependyomams.
Insights
Apatinib combined with temozolomide showed a nearly complete response in a child with recurrent ependymoma, offering a potential new treatment option. This combination therapy demonstrated a 1-year progression-free survival with manageable side effects.
Area of Science:
- Pediatric Oncology
- Neuro-oncology
- Pharmacotherapy
Background:
- Recurrent ependymomas (rEPNs) in children have poor outcomes with limited systemic treatment options.
- Apatinib, a tyrosine kinase inhibitor, has shown broad antitumor activity but its efficacy in rEPNs is unknown.
- There is a critical need for effective pharmacotherapy for pediatric rEPNs.
Observation:
- A 5-year-old patient with recurrent ependymoma experienced disease progression after surgery and radiotherapy.
- Magnetic resonance imaging (MRI) confirmed disease progression in the fourth ventricle and cerebellar peduncles.
- The patient presented with increased gait instability.
Findings:
- Combination therapy with apatinib and temozolomide (TMZ) led to significant tumor shrinkage.
- After 9 months, MRI showed a near-complete response, with a progression-free survival of 1 year.
- The patient experienced grade 1 leukocytopenia as the only adverse effect.
Implications:
- Apatinib and TMZ combination therapy represents a potential novel treatment strategy for pediatric recurrent ependymoma.
- This regimen may offer improved outcomes with a manageable side effect profile.
- Further investigation is warranted to establish the role of apatinib in treating pediatric brain tumors.
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