Kernbenson5048

INTRODUCTION Mevalonate kinase deficiency (MKD) is a rare autosomal recessive autoinflammatory disease that can lead to an inflammatory A amyloidosis (AA). METHODS To study the occurrence of AA in MKD patients we performed a systemic review of the literature and described two novel patients. RESULTS Amyloidosis occurred in 20 MKD patients, renal impairment being always the revealing symptom of AA. Although an accurate prevalence estimation is not possible since exact MKD prevalence is unknown, AA seems rare in MKD (about 6% if we estimate MKD prevalence at 300 patients worldwide). MVK gene study, available in 18 out of the 20 patients, confirmed two pathogenic mutations in all tested individuals. The most frequent genotype was V377I/I268T (n = 9/18). Retrospective search of clinical signs of MKD established, in all patients carrying MVK pathogenic mutations, a disease onset within the first four years of life. Nephrotic syndrome (n = 15), end-stage renal failure (n = 5) or both (n = 8) pointed out kidney amyloidosis. NF-κΒ 1 activator The youngest patient with renal amyloidosis was a European four-year-old girl previously misdiagnosed with PFAPA syndrome. Five patients died of AA amyloidosis despite the use of a biotherapy for two of them; kidney transplant was performed in nine individuals. Colchicine was not effective in any patient. Anti-interleukin-1 anakinra (n = 8), anti TNF etanercept (n = 7) and anti-interleukin 6 tocilizumab (n = 5) treatments were partially effective. CONCLUSION Inflammatory A amyloidosis, a rare complication of MKD, can cause death or necessitate kidney transplantation. Early diagnosis and cytokine blocking biotherapy using anti-IL1, anti-TNF or anti-IL6 agents are required to prevent terminal renal failure. OBJECTIVE To examine the onset and maintenance of efficacy of subcutaneous tanezumab for pain relief and functional improvement in difficult-to-treat patients with moderate-to-severe osteoarthritis (OA) in a 16-week dose-titration study (NCT02697773). METHODS Patients were randomized to placebo (placebo group) or tanezumab 2.5 mg at baseline and week 8 (tanezumab 2.5 mg group), or tanezumab 2.5 mg at baseline and tanezumab 5 mg at week 8 (tanezumab 2.5/5 mg group). Analyses included change from baseline in average daily index joint pain and Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC) Pain and Physical Function, and treatment responses (WOMAC Pain improvement criteria and Outcome Measures in Rheumatology-Osteoarthritis Research Society International [OMERACT-OARSI] criteria). RESULTS The 696 patients received placebo (n = 232), tanezumab 2.5 mg (n = 231), or tanezumab 2.5/5 mg (n = 233). Average daily index joint pain was statistically significantly improved within the first week (day 3-5) with tanezumab 2.5 mg compared with placebo. On first post-randomization WOMAC measurement (week 2), both tanezumab groups had statistically significant improvements compared with placebo in WOMAC Pain and Physical Function, and more tanezumab-treated patients achieved treatment response criteria (≥30%, ≥50%, or ≥70% reduction in WOMAC Pain or OMERACT-OARSI response). Efficacy was generally maintained throughout the 16-week treatment period. CONCLUSION Subcutaneous tanezumab provided statistically significant improvements compared with placebo in average daily index joint pain within the first week and WOMAC Pain and Physical Function (week 2) that were generally maintained throughout the 16-week treatment period. Tanezumab 5 mg provided only modest additional efficacy over tanezumab 2.5 mg. OBJECTIVES We aimed to identify longitudinal patterns and predictors of acute care use (emergency department [ED] visits and hospitalizations) among individuals with SLE enrolled in Medicaid, the largest U.S. public insurance. METHODS Using Medicaid data (29 states, 2000-2010) we identified 18-65-year-olds with SLE (≥3 SLE ICD-9 codes, 3rd code=index date), ≥12 months of enrollment prior to the index date and ≥24 months post. For each 90-day interval post index date, patients were assigned binary indicators (1=≥1 ED visit or hospitalization, 0=none). We used group-based trajectory models to graph patterns of overall and SLE-specific acute care use, and multinomial logistic regression models to examine predictors. RESULTS Among 40,381 SLE patients, the mean age was 40.8 (SD 11.9). Using a three-group trajectory model, 2,342 (6%) were recurrent all-cause high acute care utilizers, 12,932 (32%) moderate, 25,107 (62%) infrequent; 25% were moderate or high utilizers for SLE. There were higher odds of all-cause, recurrent acute care use (vs. infrequent) among patients with severe vs. mild SLE (OR 3.37, 95% CI 3.0-3.78), chronic pain (odds ratio [OR] 1.63, 95% CI 1.15-2.32), depression (OR 1.90 95% CI 1.74-2.09), and cardiovascular disease (OR 2.29, 95% CI 2.08-2.52). Older age, male sex and hydroxychloroquine use were associated with lower odds of recurrent overall and SLE-specific acute care use. CONCLUSION Nearly 40% of Medicaid beneficiaries with SLE are recurrent all-cause acute care utilizers; 25% have recurrent use for SLE. Modifiable factors, including outpatient management of SLE and comorbidities, may reduce avoidable acute care use. BACKGROUND Analysis of right ventricular (RV) function during the acute phase of pulmonary embolism (PE) was widely reported in the literature. However, few studies analysed its function long term after the acute phase. Our aim was to evaluate the RV function long term after a first episode of PE. METHODS In this study, we compared echocardiographic parameters of right ventricular function in 25 patients with a first episode of non-severe PE for more than six months with 25 healthy controls subject. RESULTS In the study of RV function, we noted that the mean values of the standard parameters were significantly lower in the EP group compared to the control group but their values remained within the normal range. The global RV longitudinal strain had a mean value lower than the control group statistically significant (-21±4,8% vs. -25±2,4%; P=0,28). The longitudinal strain of the free wall of the RV was altered in the EP group, however, there was no significant difference between the EP group and the control group (-19,4±16% vs.