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Purpose <p>Advanced or recurrent ependymoma lacks effective systemic therapies. Temozolomide (TMZ) is widely used in gliomas, but its role in ependymoma remains unclear.</p> Methods <p>PubMed and Embase were systematically searched for relevant studies. Outcomes including complete response (CR), partial response (PR), stable disease (SD), progressive disease (PD), progression-free survival (PFS), overall survival (OS), and adverse events (AEs) were extracted for further analysis. Sensitivity analysis was performed to determine whether the results were stable and objective. In this paper, the data were analyzed by Stata version 15.0.</p> Results <p>19 studies involving 265 patients were enrolled in this meta-analysis. In terms of tumor response, the pooled CR, PR, SD, and PD were 0.037 (95% CI: 0.000-0.124), 0.082 (95% CI: 0.008–0.195), 0.275 (95% CI: 0.131–0.440) and 0.711 (95% CI: 0.557–0.848), respectively. With regard to survival analysis, the pooled PFS and OS were 5.05 months (95% CI: 3.16–6.93 months) and 4.6 months (95% CI: 1.70–7.51 months), respectively. The most common treatment-related adverse events of temozolomide were leukopenia (0.312, 95% CI: 0.163–0.478), hypohemia (0.290, 95% CI: 0.19–0.40), and thrombocytopenia (0.191, 95% CI: 0.004–0.491).</p> Conclusions <p>TMZ shows modest efficacy and manageable toxicity in advanced or recurrent ependymoma, offering a viable treatment option in this challenging population. Prospective studies and biomarker-guided strategies are needed to optimize outcomes.</p>

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Efficacy and Safety of Temozolomide for the Treatment of Advanced or Recurrent Ependymoma: A Meta-Analysis

  • 期刊论文

  • 发表日期：

    2026年2月5日

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  • Rong Zhang,
  • Binyan Liu,
  • Zhuang Kang,
  • Shenglan Li,
  • Yingfan Sun,
  • Jiachen Wang,
  • Xin Yang,
  • Shuo Yin,
  • Feng Chen,
  • Wenbin Li

摘要

Purpose

Advanced or recurrent ependymoma lacks effective systemic therapies. Temozolomide (TMZ) is widely used in gliomas, but its role in ependymoma remains unclear.

Methods

PubMed and Embase were systematically searched for relevant studies. Outcomes including complete response (CR), partial response (PR), stable disease (SD), progressive disease (PD), progression-free survival (PFS), overall survival (OS), and adverse events (AEs) were extracted for further analysis. Sensitivity analysis was performed to determine whether the results were stable and objective. In this paper, the data were analyzed by Stata version 15.0.

Results

19 studies involving 265 patients were enrolled in this meta-analysis. In terms of tumor response, the pooled CR, PR, SD, and PD were 0.037 (95% CI: 0.000-0.124), 0.082 (95% CI: 0.008–0.195), 0.275 (95% CI: 0.131–0.440) and 0.711 (95% CI: 0.557–0.848), respectively. With regard to survival analysis, the pooled PFS and OS were 5.05 months (95% CI: 3.16–6.93 months) and 4.6 months (95% CI: 1.70–7.51 months), respectively. The most common treatment-related adverse events of temozolomide were leukopenia (0.312, 95% CI: 0.163–0.478), hypohemia (0.290, 95% CI: 0.19–0.40), and thrombocytopenia (0.191, 95% CI: 0.004–0.491).

Conclusions

TMZ shows modest efficacy and manageable toxicity in advanced or recurrent ependymoma, offering a viable treatment option in this challenging population. Prospective studies and biomarker-guided strategies are needed to optimize outcomes.