Niemann-Pick Disease, type C (NPC) is a fatal, neurodegenerative, lysosomal storage disorder. It is a rare disease with broad phenotypic spectrum and variable age of onset. These issues make it difficult to develop a universally accepted clinical outcome measure to assess urgently needed therapies. To this end, clinical investigators have defined emerging, disease severity scales. The average time from initial symptom to diagnosis is approximately 4 years. Further, some patients may not travel to specialized clinical centers even after diagnosis. We were therefore interested in investigating whether appropriately trained, community-based assessment of patient records could assist in defining disease progression using clinical severity scores. In this study we evolved a secure, step wise process to show that pre-existing medical records may be correctly assessed by non-clinical practitioners trained to quantify disease progression. Sixty-four undergraduate students at the University of Notre Dame were expertly trained in clinical disease assessment and recognition of major and minor symptoms of NPC. Seven clinical records, randomly selected from a total of thirty seven used to establish a leading clinical severity scale, were correctly assessed to show expected characteristics of linear disease progression. Student assessment of two new records donated by NPC families to our study also revealed linear progression of disease, but both showed accelerated disease progression, relative to the current severity scale, especially at the later stages. Together, these data suggest that college students may be trained in assessment of patient records, and thus provide insight into the natural history of a disease.

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3 Background: At Hospice Africa Uganda (HAU), patients' spiritual well-being is assessed in depth upon admission, but there are no rigorous assessment tools for regular spiritual distress screening in this setting. Our prior research demonstrated the “Spirit 8” assessment too cumbersome for routine use by HAU staff. The new “Spirit 3” includes three core questions thought to be most clinically relevant. The aims are to assess the feasibility of the Spirit 3 in routine screening for spiritual distress in hospice and to understand whether HAU staff value using the Spirit 3 in routine clinical practice. Methods: Between June 2014 and February 2015, the Spirit 3 was administered to consenting patients for four consecutive visits. The scale ranges from 3 to 15 with 15 representing an ideal state of spiritual well-being. For any patient unable to complete all assessments, a reason was recorded. Any staff intervention performed was recorded. Results: 49 patients were enrolled in the study and completed the first assessment. 41 (84%) of those patients had untreatable, metastatic cancer and 8 (16%) had HIV/AIDS. Twenty patients (41%) completed the second assessment; 11 the third (23%); and 8 patients (16%) completed all 4 assessments. There were no partially completed assessments. Eight patients died prior to the second assessment; the remainder were missed by research staff during clinical care and thus lost to follow-up. At 86% of visits, a staff member performed an intervention to address the reported spiritual distress. Upon study completion, 75% of the HAU staff wished to use the Spirit 3 in routine clinical care. Conclusion: Almost half of living patients completed the second assessment (49%). Attrition was due to difficulty in flagging charts rather than any barriers in acceptance by patients or staff. The majority of HAU staff wished to continue using the Spirit 3 suggesting feasibility in clinical practice. AUTHORS' DISCLOSURES OF POTENTIAL CONFLICTS OF INTEREST: No COIs from the authors.