Comparative efficacy of neoadjuvant short-course versus long-course radiotherapy-based regimens with or without immunotherapy for locally advanced pMMR rectal cancer: a systematic review and network meta-analysis.

Journal: BMC medicine

This publication reports a network meta-analysis of randomized trials in proficient MMR locally advanced rectal cancer, comparing four neoadjuvant strategies:

  • Short-course radiotherapy (SCRT)-based regimen alone
  • SCRT-based regimen plus immune checkpoint inhibitor
  • Long-course chemoradiotherapy (LCRT)-based regimen alone
  • LCRT-based regimen plus immune checkpoint inhibitor

Methods:

  • Systematic search: Systematic search of major databases up to September 20, 2025
  • Inclusion criteria: Randomized controlled trials where ICIs were added to SCRT- or LCRT-based neoadjuvant therapy and compared to the same platform without ICIs
  • Analysis: Frequentist random-effects network meta-analysis
  • Co-primary endpoints:
    • Curative-intent response: Composite of pathologic complete response (pCR) or clinical complete response managed with Watch-and-Wait
    • Grade ≥3 treatment-related adverse events (TRAEs)
  • Key secondary endpoint: pCR rate
  • Treatment ranking: Treatments ranked using SUCRA probabilities

Key results (7 RCTs, 1132 patients):

  • Curative-intent response:
    • SCRT + ICI had the highest probability of being the most effective regimen (SUCRA 98.5%).
    • SCRT + ICI vs SCRT alone: RR 1.82 (95% CI 1.27–2.60).
    • SCRT + ICI vs LCRT alone: RR 2.23 (95% CI 1.33–3.76).
    • SCRT + ICI vs LCRT + ICI: Numerically better but not statistically significant, RR 1.63 (95% CI 0.88–3.02).
    • LCRT + ICI vs LCRT alone: Numerical improvement, RR 1.37 (95% CI 0.98–1.90), not definitively significant.
  • pCR:
    • Treatment ranking for pCR was consistent with the curative-intent response findings, favoring SCRT + ICI.
  • Safety:
    • Adding ICIs to either SCRT- or LCRT-based regimens did not significantly increase grade ≥3 TRAEs.
    • No significant safety difference between SCRT + ICI and LCRT + ICI (RR 0.87; 95% CI 0.38–2.04).

Clinical implication:

  • Among current neoadjuvant platforms for pMMR LARC, SCRT combined with immunotherapy appears to provide the highest likelihood of curative-intent response without a clear trade-off in high-grade toxicity.
  • The authors conclude that SCRT is a particularly promising backbone for integration with ICIs and advocate for future phase III trials directly comparing SCRT- versus LCRT-based immunotherapy strategies, with a focus on long-term survival and organ preservation outcomes.

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