Trial reportThe Journal of clinical endocrinology and metabolism2025
Immune Checkpoint Inhibitor Therapy for Aggressive Pituitary Neuroendocrine Tumors.
Trial report in The Journal of clinical endocrinology and metabolism, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 8 papers.
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The trial behind it
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Who cites it
8 citing papers in PubMed.
- Genetic testing in pituitary adenomas: a Pituitary Society International Consensus Statement.Nature reviews. Endocrinology · 2026Review
- Therapy for aggressive pituitary tumors and carcinomas.Reviews in endocrine & metabolic disorders · 2026Review
- Can acromegaly be controlled in all cases?Journal of neuroendocrinology · 2026Review
- Predictive capacity of mismatch repair status in the use of immune checkpoint inhibitors for the treatment of aggressive pituitary tumors and pituitary carcinomas: An illustrative case report and literature review.Surgical neurology international · 2026Article
- Targeting lineage-specific heterogeneity and hypovascular-fibrotic barriers may enable precision immunotherapy in pituitary neuroendocrine tumors.Frontiers in immunology · 2026Review
- The Pituitary Immune Environment and Immunotherapy: From Hypophysitis to Pituitary Neuroendocrine Tumors.Cells · 2025Review
- Management of Recurrent and Aggressive Non-Functioning Pituitary Adenomas.Journal of clinical medicine · 2025Review
- Rare Occurrence of Distant Metastases in Prolactinoma: Insights From 2 Cases and Review of the Literature.AACE endocrinology and diabetesArticle
Corrections and comments
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Authors and funding
17 authors.
Funding
Abstract
contextPituitary neuroendocrine tumors (PitNETs) that progress following surgery and radiotherapy are in need of additional treatment options. Responses to immune checkpoint inhibitors (ICIs) have been described; however, the feasibility of ICI therapy and biomarkers of response have not been formally assessed.
objectiveTo evaluate the activity of ICI as a treatment for PitNETs.
methodsWe performed a single-center, prospective, phase 2 trial investigating the activity of ipilimumab and nivolumab in patients with PitNETs. The primary endpoint was objective response using the iRANO response criteria. We then explored genetic biomarkers of response to ICIs in 13 patients with PitNETs who were treated with ICIs, on or outside of the trial.
resultsTen patients with a PitNET were enrolled, including 5 corticotroph, 4 lactotroph, and 1 somatotroph tumor, of which 9/10 (4 metastatic and 5 nonmetastatic) patients were evaluable for the primary endpoint. While no objective responses were observed, tumor shrinkage was seen in 2/9 patients. In a biomarker discovery cohort, comprising 7 tumors treated on trial and 6 tumors treated off trial, temozolomide hypermutation, and mismatch repair deficiency (MMRd) were associated with immunological response. In 3 tumors sequenced pre- and post-ICI treatment, we identified evidence of immunoediting, characterized by loss of MMRd and/or a decrease in tumor mutational burden.
conclusionThis study demonstrates the safety and feasibility of ICI treatment in aggressive PitNETs. We also identified MMRd and temozolomide hypermutation as potential biomarkers of response to ICI. Overall, our data suggest that ICIs might provide an additional treatment option for PitNET; this should be evaluated more broadly in future studies.
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