Immune Checkpoint Inhibitor Therapy for Aggressive Pituitary Neuroendocrine Tumors
Authors' conclusion
This 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.
Study context
- Population
- Patients with aggressive pituitary neuroendocrine tumors (PitNETs) (5 corticotroph, 4 lactotroph, 1 somatotroph)9 evaluable
- Participants
- 10 patients
- Outcome
- Objective response using the iRANO response criteria
Methodological detailssingle-center, prospective
- Single-center
- Prospective
Main finding
As reported in the abstract
Objective response (iRANO criteria)
Tumor shrinkage
2/9 patients
Observational data: associations, not effects.
Further findings
Tumor shrinkage
- Tumor shrinkage
- 2/9 patients
Genetic biomarkers of response (biomarker discovery cohort, 7 on-trial and 6 off-trial tumors, n = 13)
- Evidence of immunoediting (loss of MMRd and/or decrease in tumor mutational burden) in tumors sequenced pre- and post-ICI
- 3 tumors
- Temozolomide hypermutation and MMRd were associated with immunological response
Original publication
Immune Checkpoint Inhibitor Therapy for Aggressive Pituitary Neuroendocrine Tumors. J Clin Endocrinol Metab. Published 20 March 2025. doi:10.
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