Conducting pituitary care: innovation and standardization in a rare disease

Abstract

This thesis outlines an effort to standardise management and outcome measurement in the rare disease field of pituitary tumours. Moreover, it is an effort to improve patient-centred care in this field.

In this thesis a novel way of representing outcome is proposed, Outcome Squares. These squares integrate the achievement of treatment goal and treatment safety. They can aid in patient counselling, in this case for pituitary surgery. Moreover, they can be used to analyse the quality of provided care and compare outcomes between time periods or treatment facilities.

Furthermore, perioperative diagnostics surrounding pituitary surgery are analysed. A pathophysiology-based definition and treatment algorithm for postoperative diabetes insipidus are formalised. The diagnostics of postoperative adrenal insufficiency was critically reviewed, and an argument is made against the routine use of the expensive CRH-test. An overview is presented of the pattern of visual field defects in pituitary tumours and the pitfalls in diagnosing the tumour as cause of these defects. Finally, a first analysis of the new network of centres that provide care for rare endocrine diseases in the European Union, Endo-ERN, is provided. It also presents the view of rare endocrine disease patients in this network on unmet needs in research on their disease.