Neoadjuvant Immunotherapy-Based Systemic Treatment in MMR-Deficient or MSI-High Rectal Cancer: Case Series

Rahel Demisse, Neha Damle, Edward Kim, Jun Gong, Marwan Fakih, Cathy Eng, Leslie Oesterich, Madison McKenny, Jingran Ji, James Liu, Ryan Louie, Kit Tam, Sepideh Gholami, Wissam Halabi, Arta Monjazeb, Farshid Dayyani, May Cho

Research output: Contribution to journalArticlepeer-review

11 Scopus citations


Treatment options for locally advanced rectal cancer have continued to consist largely of chemotherapy, chemoradiation, and/or surgical resection. For patients who are unable to undergo these therapeutic modalities or who do not to experience a response to them, treatment options are limited. We report 3 cases of mismatch repair-deficient (dMMR) locally advanced adenocarcinoma of the rectum that showed significant response with neoadjuvant immunotherapy-based systemic treatment. The first patient was not eligible for standard therapy because of a history of radiotherapy to the prostate with concurrent comorbidities and therefore received single-agent pembrolizumab. The second patient did not respond to total neoadjuvant chemoradiation and subsequently received combined nivolumab and ipilimumab. The third patient had a known family history of Lynch syndrome and presented with locally advanced rectal cancer and a baseline carcinoembryonic antigen level of 1,566 ng/mL. She was treated using neoadjuvant pembrolizumab and FOLFOX (folinic acid, fluorouracil, oxaliplatin). In this small series, we suggest that single-agent and combined-modality neoadjuvant immunotherapy/chemotherapy appear to be safe and effective treatment options for patients with (dMMR) locally advanced rectal cancer. Our findings encourage further studies to investigate the role of neoadjuvant immunotherapy as a viable treatment strategy in this population.

Original languageEnglish (US)
Pages (from-to)798-804
Number of pages7
JournalJournal of the National Comprehensive Cancer Network : JNCCN
Issue number7
StatePublished - Jul 1 2020

ASJC Scopus subject areas

  • Oncology


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