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A series of qualitative studies conducted by the OMERACT Myositis Working Group identified pain interference, fatigue, and physical function as highly important life impact domains for adults with idiopathic inflammatory myositis (IIM). In this study, our goal was to assess the responsiveness and minimal important difference of PROMIS pain interference (6a), fatigue (7a), and physical function (8b). Adults with IIM from USA, Netherlands, Korea, Sweden, and Australia with two "clinical" visits were enrolled in this prospective study. Anchor questions on a Likert scale were collected at baseline, and manual muscle testing (MMT), physician and patient reported global disease activity, and PROMIS instruments were collected at both visits. Responsiveness was assessed with i) ANOVA, ii) paired t -test, effect size and standardized response mean, and iii) Pearson correlation. Minimal important difference (MID), minimal important change (MIC) and minimal detectable change (MDC) values were calculated. 114 patients with IIM (median age 60, 60 % female) completed both visits. Changes in PROMIS instruments were significantly different among anchor categories. Patients who reported improvement had a significant improvement in their PROMIS scores with at least medium effect size, while patients who reported worsening and stability did not show a significant change with weak effect size. PROMIS instruments had weak to moderate correlations with MMT, patient and physician global disease activity. MID was approximately 2–3 points for Pain Interference and 3–4 points for Fatigue and Physical Function forms based on the method used. MIC was approximately 4–5 for improvement of all the instruments, while MDC was 1.7–2 points for Pain Interference and Physical Function and 3.2–3.9 for Fatigue. This study provides evidence towards the responsiveness of the PROMIS instruments in a large international prospective cohort of adults with IIM supporting their use as PROMs in adult myositis. [ABSTRACT FROM AUTHOR]

Summary Introduction There is a lack of prospective studies that include a selected population of patients with primary non-refluxing megaureter (PM). Thus, a longitudinal observational study was designed to follow from birth a selected population of children with PM; all were antenatally diagnosed. In this paper, the outcomes observed in the first year of life are presented. Objective The primary aim was to follow the natural history of PM. The secondary aim was to monitor the onset of any potential complications such as urinary tract infections (UTIs), need for hospitalization and need for surgical correction. Study design All children with antenatally diagnosed PM, born between January 2007 and December 2013, were prospectively followed with observational management: renal ultrasonography and clinical evaluation on a 3-month basis; urinalysis and culture in case of symptoms; and mercaptoacetyltriglycine (MAG3) nuclear scan once older than 1 month. Children presenting at birth with mild urinary tract dilatation were included in Group A; those with moderate-to-severe dilatation were included in Group B. Continuous antibiotic prophylaxis (CAP) was administered to Group B. Results Forty-seven children (44 males, three females) with 58 PM were included in the study. The participants and their corresponding outcomes are shown in the summary Table. The presence of obstruction at renogram was a significant predictor of UTIs and hospitalization. Discussion The strengths of this study were its prospective nature and its very consistent population. A limitation was the lack of control groups. The results regarding the negligible incidence of complications in Group A and the residual incidence of febrile UTIs (20%) and hospitalization (17%) in Group B, even with CAP, are in line with previous literature. In contrast, there was a higher risk of UTIs observed in children aged older than 6 months. Conclusions Resolution or improvement is expected in all cases of PM with mild postnatal dilatation, and close to 60% of those with moderate or severe dilatation. Surgery is rarely performed on children younger than 1 year of age. It is safe to observe children with mild urinary tract dilatation without CAP, because the incidence of UTIs is negligible. In those presenting with moderate or severe urinary tract dilatation, despite CAP, a residual incidence of UTIs is seen, and symptomatic patients often require hospitalization. However, UTIs are well tolerated and do not seem to modify outcome. Cases showing obstruction on the MAG3 scan seem to be at higher risk of UTIs and hospitalization. Table Outcomes and events of interest during the first year of life, in the overall population and in the two groups. Group Participants n Primary non-refluxing megaureter n Outcome at 1 year of age Events of interest Resolved n (%) Improved n (%) Stable n (%) Worsened n (%) Afebrile UTIs (%) Febrile UTIs (%) Hospitalization (%) Surgery (%) A + B 47 58 11 (19.0) 30 (51.7) 14 (24.1) 3 (5.2) 3 (6.4) 7 (14.9) 6 (12.8) 2 (4.2) A 13 17 6 (35.3) 11 (64.7) – – 2 (15.4) – – – B 34 41 5 (12.2) 19 (46.3) 14 (34) 3 (7.3) 1 (2.9) 7 (20.6) 6 (17.6) 2 (5.9) [ABSTRACT FROM AUTHOR]