M. N. Mongelli1, I. Helenowski1, S. Yount1, C. Sturgeon1 1Feinberg School Of Medicine – Northwestern University,Chicago, IL, USA
Introduction: Patient-Reported Outcomes (PROs) are being measured in many chronic disease states to inform decisions about intervention and management of disease while minimizing patient suffering and side effects. There is a paucity of data on health-related quality of life (HRQOL) and PROs in patients with Multiple Endocrine Neoplasia Type-2 (MEN-2). We hypothesized that HRQOL in MEN-2 patients would be lower than that of the general United States (US) population, but similar to that of patients suffering from other chronic diseases.
Methods: Adults ≥ 18 years were recruited from an online MEN support group and completed the PROMIS-29 questionnaire (n=46). Responses were converted into t-scores for each of the seven health domains. T-scores of PROMIS domains were compared to US normative data using a one-sample t-test. PROMIS scores for other conditions, including low back pain, cancer, congestive heart failure (CHF), chronic obstructive pulmonary disease (COPD), major depressive disorder, rheumatoid arthritis (RA), neuroendocrine tumors (NET), primary hyperparathyroidism (PHPT), and MEN-1 were obtained through literature review. T-scores for health domains were compared to other conditions using a Wilcoxon signed-rank test.
Results: The mean age was 46.1 years and the average time since diagnosis was 14.1 years. Compared to the US normative population, MEN-2 patients reported statistically significantly more anxiety (56.1±11.2, p=.001), depression (54.6±11.2, p=.008), fatigue (61.0±10.4, p<.001), pain interference (55.2±11.1, p =.003), and sleep disturbance (57.0±3.7, p<.001), as well as significantly decreased physical functioning (44.6±9.5, p<.001) and ability to participate in social roles (45.8±9.7, p=.005). MEN-2 patients reported statistically significantly greater fatigue than patients with cancer (p<.0001), COPD (p=.01), RA (p=.0001), NET (p=.0007), and PHPT (p<.0001), greater pain interference than patients with PHPT (p<.0001), and improved physical functioning compared to patients with low back pain (p<.0001), CHF (p<.0001), and COPD (p=.0002).
Conclusion: PROs may be a valuable tool to inform the management of patients with MEN-2. Our study is the first to use the PROMIS-29 metric to directly compare PROs between MEN-2 and other chronic conditions. Individuals with MEN-2 reported worse HRQOL in all 7 domains compared to normative data. There is a pattern of increased fatigue among MEN-2 patients compared to many other chronic conditions, even though MEN-2 patients report greater physical functioning than other chronic diseases. Prospective longitudinal evaluation of PROs in MEN-2 should be conducted in order to identify treatments associated with the highest HRQOL.