S. Goswami1, B. J. Peipert1, S. E. Yount3,4, C. Sturgeon1 1Feinberg School Of Medicine – Northwestern University,Department Of Surgery,Chicago, IL, USA 3Feinberg School Of Medicine – Northwestern University,Department Of Medical Social Sciences,Chicago, IL, USA 4Feinberg School Of Medicine – Northwestern University,Department Of Psychiatry And Behavioral Sciences,Chicago, IL, USA
Introduction: The physical and psychosocial morbidity of multiple endocrine neoplasia type 1 (MEN-1) is ill-defined. Due to lack of genotype-phenotype relationships, heterogeneity in clinical manifestations, and infrequent surgical cures, there is controversy regarding optimal management. Thus it is important to understand how disease and treatment-related factors relate to patient-reported outcomes (PROs) such as health-related quality of life (HRQOL). We hypothesized that disease and treatment burden negatively impacts HRQOL in adults with MEN-1.
Methods: Adults ≥18 years with MEN-1 (n=174) were recruited through an MEN-1 support group. An online survey on demographics, disease features, and treatment history was administered, and respondents were grouped and compared based on their answers. The PROMIS-29 instrument was used to evaluate HRQOL in 7 domains. T scores generated from MEN-1 survey responses were compared to United States (US) normative data using a 1-sample t-test. The Mann-Whitney U test was used for subgroup analysis of categorical variables and linear regression was performed on continuous variables. Holms-Bonferroni was used to correct for multiple comparisons. Data are reported as mean T scores ± standard deviation.
Results: Adults with MEN-1 reported scores reflecting poorer physical, mental, and social well-being than US normative data in all 7 domains (p<0.001): anxiety (61.1±10.2), depression (57.1±10.5), fatigue (60.7±11.7), pain interference (55.6±11.1), physical function (44.7±9.5), sleep disturbance (57.2±9.0), and social function (44.9±10.6). Respondents with recurrent hyperparathyroidism (42%) reported worse scores than those without recurrence in 5 of 7 domains: anxiety (63.2±8.6 vs 58.1±10.0, p<0.01), depression (58.5±8.9 vs 54.3±11.3, p<0.05), fatigue (63.2±10.8 vs 57.9±11.9, p<0.05), pain interference (58.8±9.8 vs 53.0±12.0, p<0.01) and social functioning (43.3±9.3 vs 48.2±11.4, p<0.01). Requirement of prescription medication for MEN-1 (73%) was associated with worse PRO scores across all 7 domains (p<0.05). Increased frequency of specialty doctor appointments was associated with higher levels of anxiety, depression and pain interference and lower levels of physical and social functioning (r = 0.19-0.28, p<0.05). Traveling > 50 miles for specialty care was associated with greater anxiety (p<0.01). Increased frequency of hospitalization was associated with higher anxiety (r=0.26, p=0.001). No significant association was found between presence of pancreatic tumors or extent of pancreatic surgery and HRQOL. PRO scores were independent of age, sex, and ethnicity.
Conclusion: This study is the first to explore PROs in MEN-1. Adults with MEN-1 report lower HRQOL than US normative data in all 7 PROMIS-29 domains. Factors associated with lower HRQOL include recurrent hyperparathyroidism, increased travel distance and frequency of specialty appointments, hospitalization frequency, and requirement of prescription medications.