Medical therapy for refractory pituitary adenomas

Introduction: Refractory pituitary adenomas are those that have progressed following standard of care treatments. Medical therapy options for these challenging tumors are limited. Purpose: To review the landscape of tumor directed medical therapies and off-label investigational approaches for refractory pituitary adenomas. Methods: Literature on medical therapies for refractory adenomas was reviewed. Results: The established first-line medical therapy for refractory adenomas is temozolomide, which importantly may increase survival, but clinical trial data are still needed to clearly establish its efficacy, identify biomarkers of response, and clarify eligibility and outcome criteria. Other therapies for refractory tumors have only been described in case reports and small case series. Conclusion: There are currently no approved non-endocrine medical therapies for refractory pituitary tumors. There is an urgent need for identifying effective medical therapies and studying them in multi-center clinical trials.