Background Childhood-onset systemic erythematosus lupus (cSLE) is characterized by more severe disease, widespread organ involvement and higher mortality compared to adult-onset SLE. However, cSLE is largely underfunded to carry out necessary research to advance the field. Few commonly used SLE medications have been studied in children, and important knowledge gaps exist concerning epidemiology, genetics, pathophysiology and optimal treatments for cSLE. Methods In order to assess highest cSLE research priority areas, the Lupus Foundation of America (LFA) and Childhood Arthritis and Rheumatology Research Alliance (CARRA) administered a cSLE research prioritization survey to pediatric rheumatologists, dermatologists and nephrologists with expertise in lupus. Members of LFA and CARRA’s SLE Committee identified a list of cSLE research domains and developed a 17-item tiered, web-based survey asking respondents to categorize the research domains into high, medium, or low priority areas. For domains identified as high priority, respondents ranked research topics within that category. For example, for the domain of nephritis, respondents ranked importance of: epidemiology, biomarkers, long-term outcomes, quality improvement, etc. The survey was distributed to members of CARRA, Midwestern Pediatric Nephrology Consortium (MWPNC) and Pediatric Dermatology Research Alliance (PeDRA) Connective Tissue Disease group. Results The overall response rate was 256/752 (34%). The highest prioritized research domains were: nephritis, clinical trials, biomarkers, neuropsychiatric disease and refractory skin disease. Notably, nephritis, clinical trials and biomarkers were ranked in the top five by all groups. Within each research domain, all groups showed agreement in identifying the following as important focus areas: determining best treatments, biomarkers/pathophysiology, drug discovery/novel treatments, understanding long term outcomes, and refining provider reported quality measures. Conclusion This survey identified the highest cSLE research priorities among leading rheumatology, dermatology and nephrology clinicians and investigators engaged in care of children with lupus. There is a strong need for multidisciplinary collaboration moving forward, which was indicated as highly important among stakeholders involved in the survey. These survey results should be used as a roadmap to guide funding and specific research programs in cSLE to address urgent, unmet needs among this population.

e19105 Background: Patient-reported outcomes can provide symptom and function data that complement standard oncology endpoints. Frequently, trials will conclude there was no clinically meaningful detriment to health-related quality of life (HRQL) or function, even when notable toxicity is observed. It is possible that mean change from baseline analyses obscures meaningful change in subgroups experiencing symptomatic toxicity. In this study, we explore how patients’ response to a diarrhea item related to physical function (PF) and HRQL in trials submitted to US FDA. Methods: We analyzed 3 randomized, double-blind breast cancer trials (early to late line metastatic) where diarrhea was a more common AE-symptom in the treatment arm, but there was not a large detriment in the mean change from baseline for HRQL and PF. Trials included the EORTC Quality of Life Questionnaire (QLQ-C30), which captures patient-reported HRQL, symptoms, and functioning. Higher scores (range 0-100) indicate better functioning and HRQL. Symptoms were measured with a 4-point scale; not at all to very much. Descriptive statistics were used to analyze diarrhea, PF, and HRQL over time. Results: Patients reporting very much diarrhea at month 3 had worse PF and HRQL compared to patients reporting no diarrhea . The range of difference between patients who reported very much diarrhea and those with none was 8-18 points for PF across trials. For HRQL scores, the range was 13–17 points worse. This trend was also seen in the control arm and at other times. Conclusions: In this set of breast cancer trials with differences in diarrhea by arm, reporting “no meaningful difference in PF or HRQL between the arms” is insufficient and potentially misleading. A more informative interpretation is that an exploratory analysis of HRQL and PF did not show in the investigational arm; there was a greater proportion of patients reporting diarrhea on the treatment arm; and patients reporting more frequent diarrhea reported lower HRQL and PF compared to patients with no diarrhea, regardless of arm. [Table: see text]

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