Complete response to pembrolizumab in a paediatric patient with Lynch syndrome-associated colorectal cancer: a case report and review of immunotherapy-related bowel obstruction.

IF 2.8 Q2 Pharmacology, Toxicology and Pharmaceutics

Drugs in Context Pub Date : 2026-04-10 eCollection Date: 2026-01-01 DOI:10.7573/dic.2026-1-5

Antonio Ruggiero, Fernando Fuccillo, Palma Maurizi, Alberto Romano, Dario Talloa, Stefano Mastrangelo, Michele Basso, Vito Briganti, Maria Cristina Giustiniani, Roberto Persiani, Giorgio Attinà

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引用次数: 0

Abstract

Colorectal cancer (CRC) is extremely rare in paediatrics with aggressive histopathology and advanced presentation. Whilst pembrolizumab has shown efficacy in adult microsatellite instability-high or mismatch repair deficiency CRC, paediatric data remain scarce. A 12-year- old boy with Lynch syndrome-associated CRC developed severe cardiac toxicity after initial chemotherapy. Treatment was switched to off-label pembrolizumab, resulting in a complete response after 24 cycles. Despite good tolerability, the patient developed a delayed bowel stricture at the tumour site. After surgery, there was no pathological evidence of residual adenocarcinoma. This case demonstrates the potential of pembrolizumab in paediatric CRC, highlighting the importance of molecular-guided rather than age-restricted therapy selection.

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派姆单抗在Lynch综合征相关结直肠癌患儿中的完全缓解：免疫治疗相关肠梗阻的病例报告和回顾

结直肠癌（CRC）是极为罕见的儿科侵袭性组织病理学和晚期表现。虽然pembrolizumab已显示对成人微卫星不稳定性高或错配修复缺陷CRC有效，但儿科数据仍然很少。一名患有Lynch综合征相关性结直肠癌的12岁男孩在初始化疗后出现严重的心脏毒性。治疗转为标签外派姆单抗，24个周期后完全缓解。尽管耐受性良好，但患者在肿瘤部位出现了延迟性肠狭窄。术后病理未见腺癌残留。该病例证明了派姆单抗在儿童CRC中的潜力，强调了分子引导而不是年龄限制治疗选择的重要性。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Drugs in Context Medicine-Medicine (all)

CiteScore

5.90

自引率

0.00%

发文量

审稿时长

9 weeks

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