Natural History of Medullary Thyroid Cancer to Inform Advanced Disease Management

PRIMARY OBJECTIVES:

I. To describe the natural history of medullary thyroid cancer (MTC) using a comprehensive battery of demographic, clinical, pathologic, and genotypic variables collected in our existing multi-institutional patient registry.

Ia. To describe the demographic, clinical, pathologic, and genotypic variables associated with each phase of disease.

Ib. To identify those variables which are predictors of progression to the most advanced phases of disease that require small molecule therapy, including commercially approved and experimental agents.

II. To characterize patients’ experience with the different phases of MTC through a systematic evaluation of patient-reported outcomes (PROs).

IIa. To describe clinical, psychosocial, economic, and physical well-being variables and the subsequent impact on quality of life associated with each phase of disease.

IIb. To evaluate longitudinal changes in clinical, psychosocial, economic, and physical well-being variables between and within all phases of MTC, and the subsequent impact they have on quality of life.

III. To evaluate the association of selected biometric and patient-reported outcomes on adherence, change, and discontinuation of approved targeted therapies and drugs being tested within clinical trial.

OUTLINE: This is an observational study.

AIM I & II: Patients complete questionnaires over 10-40 minutes 2-4 times per year about health and how finances and quality of life effect experience with disease.

AIM III: Patients complete a questionnaire over 2 minutes and undergo blood pressure measurements every day for up to 12 weeks.

The questionnaire data gets paired with the collected medical record history that is maintained in the Registry.