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Purpose <p>This study aimed to evaluate the role and optimal timing of radiotherapy following intensive chemotherapy in infants with medulloblastoma (MB), and to explore whether molecular subtyping may inform radiotherapy timing strategies.</p> Methods <p>We conducted a retrospective cohort study including 105 children aged &lt; 3 years with MB treated at Beijing Shijitan Hospital, Capital Medical University, between 2013 and 2023. Clinical data were collected to compare clinical characteristics and survival outcomes across different radiotherapy strategies.</p> Results <p>Among 105 infants with MB, 67 (63.8%) were classified as SHH, 18 (17.1%) as Group 3, and 20 (19.0%) as Group 4, with no WNT cases identified. The SHH subgroup demonstrated significantly better survival than the Non-SHH subgroup (comprising Group 3 and Group 4), with 3-year overall survival (OS) of 80.2% versus 46.0% and progression-free survival (PFS) of 62.6% versus 30.4% (both <i>P</i> &lt; 0.001). Patients were stratified into chemotherapy-only, upfront radiotherapy, and salvage radiotherapy groups based on treatment timing. In SHH patients, upfront radiotherapy did not confer additional survival benefit over chemotherapy alone (<i>P</i> = 0.658), whereas salvage radiotherapy was associated with longer observed OS (<i>P</i> = 0.005). In contrast, in the Non-SHH subgroup, chemotherapy alone was associated with a high progression rate (85.7%), and radiotherapy was associated with improved survival outcomes.</p> Conclusions <p>Real-world data demonstrate distinct differences between SHH and Non-SHH infant MBs in survival outcomes and radiotherapy strategies. The SHH subtype showed favorable survival with systemic chemotherapy, whereas the Non-SHH subtype exhibited limited disease control with chemotherapy alone, suggesting a more critical role for radiotherapy in this population.</p>

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Real-world outcomes of radiotherapy timing in infants with medulloblastoma by molecular subtype: a single-center retrospective cohort study

  • 期刊论文

  • 发表日期：

    2026年7月30日

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  • Yi-fan Liu,
  • Xiang-chen Li,
  • Jin Zhang,
  • Jia-hao Niu,
  • Shu-xu Du,
  • Yong-ji Tian

摘要

Purpose

This study aimed to evaluate the role and optimal timing of radiotherapy following intensive chemotherapy in infants with medulloblastoma (MB), and to explore whether molecular subtyping may inform radiotherapy timing strategies.

Methods

We conducted a retrospective cohort study including 105 children aged < 3 years with MB treated at Beijing Shijitan Hospital, Capital Medical University, between 2013 and 2023. Clinical data were collected to compare clinical characteristics and survival outcomes across different radiotherapy strategies.

Results

Among 105 infants with MB, 67 (63.8%) were classified as SHH, 18 (17.1%) as Group 3, and 20 (19.0%) as Group 4, with no WNT cases identified. The SHH subgroup demonstrated significantly better survival than the Non-SHH subgroup (comprising Group 3 and Group 4), with 3-year overall survival (OS) of 80.2% versus 46.0% and progression-free survival (PFS) of 62.6% versus 30.4% (both P < 0.001). Patients were stratified into chemotherapy-only, upfront radiotherapy, and salvage radiotherapy groups based on treatment timing. In SHH patients, upfront radiotherapy did not confer additional survival benefit over chemotherapy alone (P = 0.658), whereas salvage radiotherapy was associated with longer observed OS (P = 0.005). In contrast, in the Non-SHH subgroup, chemotherapy alone was associated with a high progression rate (85.7%), and radiotherapy was associated with improved survival outcomes.

Conclusions

Real-world data demonstrate distinct differences between SHH and Non-SHH infant MBs in survival outcomes and radiotherapy strategies. The SHH subtype showed favorable survival with systemic chemotherapy, whereas the Non-SHH subtype exhibited limited disease control with chemotherapy alone, suggesting a more critical role for radiotherapy in this population.