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Objective: To compare 12-month post-treatment health-related quality of life (HR-QoL) and symptom severity (SS) changes among patients with symptomatic uterine fibroids (SUF) not seeking fertility and undergo a hysterectomy, abdominal myomectomy (AM), or uterine artery embolization (UAE). Materials and Methods: The Comparing Options for Management: Patient-Centered Results for Uterine Fibroids (COMPARE-UF) Registry is a multi-institutional prospective observational cohort study of patients treated for SUF. A subset of 1465 women 31-45 years of age, who underwent either hysterectomy (n = 741), AM (n = 446), or UAE (n = 155) were included in this analysis. Demographics, fibroid history, and symptoms were obtained by baseline questionnaires and at 1 year post-treatment. Results were stratified by all treatments and propensity score weighting to adjust for differences in baseline characteristics. Results: Women undergoing UAE reported the lowest baseline HR-QoL and highest SS scores (mean = 40.6 [standard deviation (SD) = 23.8]; 62.3 [SD = 24.2]) followed by hysterectomy (44.3 [24.3]; 59.8 [SD = 24.1]). At 12 months, women who underwent a hysterectomy experienced the largest change in both HR-QoL (48.7 [26.2]) and SS (51.9 [25.6]) followed by other uterine-sparing treatments. Propensity score weighting revealed all treatments produced substantial improvement, with hysterectomy patients reporting the highest HR-QoL score (92.0 [17.8]) compared with myomectomy (86.7 [17.2]) and UAE (82.6 [21.5]) (p

Background Effectiveness of rituximab in pediatric idiopathic nephrotic syndrome suggests that B cells play a pathogenic role. We tested safety and efficacy of the B-cell-modulating agent belimumab in frequently relapsing nephrotic syndrome (FRNS). Methods An open-label, prospective, single-arm pilot study (EUDRACT 2017-003839-11) was designed to treat 10 children with FRNS with i.v. belimumab for 12 months. Prednisone was tapered/stopped. Safety, number of relapses, cumulative prednisone dose and B-cell subset 'levels' are referred to both B cell subset and immunoglobulin. Results Five patients were enrolled, and four reached the primary 6-month endpoint. Of these, two completed the 12-month endpoint. Three patients experienced [greater than or equal to]2 relapses while on belimumab, requiring additional immunosuppression. Compared to the 6 months before belimumab treatment, the mean number of relapses (1.4 vs. 2, p=0.21) and the mean cumulative prednisone dose (1.86 vs. 2.62 g/m.sup.2, p=0.17) were not significantly reduced during the 6 months on belimumab. This study was terminated by the steering committee after the interim evaluation because belimumab failed to show clear benefits to counterbalance the inconvenience of monthly i.v. infusion. During follow-up, total and mature-naïve B cells decreased, while no change in memory B-cells was observed. Serum immunoglobulins remained stable. No infusion reaction was observed. Conclusions Short-term treatment with belimumab in pediatric FRNS was well tolerated. The number of patients was too small to draw conclusions on efficacy. Nonetheless, we did not observe clear improvements. The burden of monthly in-hospital i.v. infusions outweighed potential benefits. Persistence of circulating memory B cells supports their pathogenic role in the disease. Graphical abstract A higher resolution version of the Graphical abstract is available as Supplementary information., Author(s): Marina Vivarelli [sup.1] [sup.2] , Manuela Colucci [sup.2] , Antonio Gargiulo [sup.1] , Chiara Bettini [sup.1] , Anna Lo Russo [sup.3] , Francesco Emma [sup.1] [sup.2] Author Affiliations: (1) [...]

Background In the clinical practice, prednisone (PDN) dose in children is often prescribed using the patient weight, despite dose calculation using body surface area (BSA) is assumed to be preferable, because it parallels better with PDN metabolism in human subjects. Methods Calculations based on body weight (W) carry the risk of underdosing, particularly in young children. Conversely, BSA estimation requires knowing the patient height, which is not always available, and more complex calculations. Results To overcome these limitations, we have developed linear equations allowing approximating the BSA-based dose using only the patient weight in kilogram. To this end, we have used anthropomorphic data from 754 pediatric patients and have validated the proposed equations with a prospective cohort of 77 children with steroid sensitive nephrotic syndrome. The equation estimating a dose of 60 mg/m.sup.2 was [2 x W + 8] and the equation estimating a dose of 40 mg/m.sup.2 was [W + 11]. Conclusions Both equations performed very well and predicted reliably the BSA-based dose with an average error of 3.4% and 2.2%, respectively., Author(s): Francesco Emma [sup.1] , Giovanni Montini [sup.2] [sup.3] , Antonio Gargiulo [sup.1] Author Affiliations: (Aff1) 0000 0001 0727 6809, grid.414125.7, Department of Pediatric Subspecialties, Division of Nephrology, Bambino Gesù [...]