Background

First line treatment for prolactinoma patients is with a dopamine agonist (DA). Endoscopic trans-sphenoidal resection is reserved for patients with severe side effects or a DA resistant prolactinoma. Most patients need prolonged DA treatment, which often causes side effects. Surgery has a good safety profile and high chance of remission and may thus deserve a more prominent place in the treatment of prolactinomas.

Earlier this year, we have started a multicenter observational cohort study, aimed to inventory several patient-specific outcomes, including health-related quality of life (HRQoL) and disease remission. However, there are no randomized clinical trials (RCTs) that have compared the treatment strategies for prolactinomas. The hypothesis for the current study is that early or upfront surgical resection is superior to DA treatment for HRQoL and remission rate in patients with a non-invasive prolactinoma of limited size.

Study Design

This multicenter RCT will compare endoscopic trans-sphenoidal surgery to standard care in 3 groups of prolactinoma patients at different phases in the disease course: (1) newly diagnosed, treatment naïve patients; (2) patients who have had DA treatment for 4-6 months; and (3) patients who have a persisting prolactinoma after DA treatment for >2 years.

The intervention group will be referred to a neurosurgical expertise center to receive personalized surgical consultation with a multidisciplinary team, designed to guide patients in their treatment choice (continue with surgery or return to DA treatment). The control group will receive standard care (DA treatment) and follow up with their own physician.

At least 200 patients are needed per subgroup to assess our main endpoints, HRQoL at 12 months (during DA treatment vs post-surgery) and remission after 3 years (1-year DA withdrawal vs post-surgery).

For both our cohort study and RCT, we are building a collaborative network containing endocrinologists, gynaecologists and neurosurgeons throughout the Netherlands and abroad. For more information, see www.prolactinoom.nl.