Zhouherbert1112

To evaluate the reliability, validity, feasibility and psychometric performance of the Lupus Impact Tracker (LIT) as a patient reported outcome (PRO) measure tool in pediatric systemic lupus erythematosus (pSLE).

This is a prospective, observational, pilot study where patients aged between 12 and 25 years, fulfilling the 1997 ACR classification criteria for SLE, were enrolled. Over 3 consecutive, routine, clinical visits, the patients completed the LIT alongside the Patient-Reported Outcomes Measurement Information System-Short Forms (PROMIS-SFs), Childhood Health Assessment Questionnaire (CHAQ). Rheumatologists completed the Systemic Lupus Erythematosus Disease Activity Index 2000 (SLEDAI-2K) and the Systemic Lupus International Collaborating Clinics/American College of Rheumatology (SLICC-ACR) Damage Index. Demographic, clinical and laboratory data were also collected.

Of 46 patients enrolled, 38 patients completed 2 visits and 31 completed all 3 visits. Seventy-eight percent were female, 33% African American, 28% Asian, 15% Caucasian and 17% Hispanic. The mean (SD) age was 17.2 (2.7) years, with a mean (SD) disease duration of 4.6 (3.1) years. The mean (SD) SLEDAI-2K at enrollment was 3.54 (2.96). In the 38 patients who completed two or more visits, intra-class correlation coefficient and Cronbach alpha were calculated to be 0.70 and 0.91 respectively, signifying good reliability of LIT. The LIT showed positive correlation with CHAQ-Disability Index and majority of the PROMIS-SFs parameters. Construct validity was established against clinical disease activity (SLEDAI-2K).

The preliminary results indicate that the LIT is a reliable and valid instrument to capture PRO in p-SLE. Prospective validation with a larger, multicenter cohort is the next step.

The preliminary results indicate that the LIT is a reliable and valid instrument to capture PRO in p-SLE. Prospective validation with a larger, multicenter cohort is the next step.

Thrombotic thrombocytopenia purpura (TTP) associated with systemic lupus erythematous (SLE) (i.e., SLE-TTP) is a rare life-threatening disease often requiring intensive immunosuppressive agents, in addition to high-dose corticosteroids and plasma exchange (PEX). The optimal therapy of rituximab is unclear, but 375 mg/m

weekly for 4 weeks is the usual practice, adopted from regimens for non-Hodgkin's lymphoma. We reported two cases of refractory SLE-TTP that showed good efficacy and prognosis with combination of methylprednisolone (MP) pulse, plasma exchange and low-dose rituximab (100 mg weekly for 4 weeks) treatment.

Clinical data and treatment outcomes were reviewed of two patients diagnosed with refractory SLE-TTP at Peking Union Medical College Hospital between July 2017 and July 2018.

Both patients had SLE and presented with microangiopathic anemia and thrombocytopenia. Laboratory assays revealed high anti-nuclear antibody titers, reduced complement 3 and 4 levels, proteinuria, significantly elevated lactate dehydrogenase, schistocytes on peripheral blood smear, low ADAMTS13 activity, and the presence of ADAMTS13 inhibitor. In both patients, platelet counts remained below 50 × 10

/L after MP pulse and 6 PEXs, confirming the diagnosis of refractory SLE-TTP. Low-dose rituximab (100 mg weekly for 4 weeks) was administered in both cases, resulting in normalization of platelet counts and significant reductions in B-lymphocyte counts. No TTP relapse or SLE flare occurred during 24 months of follow-up.

Our cases confirmed the efficacy and good follow-up outcomes of low-dose rituximab treatment (100 mg weekly for 4 weeks) for refractory SLE-TTP.

Our cases confirmed the efficacy and good follow-up outcomes of low-dose rituximab treatment (100 mg weekly for 4 weeks) for refractory SLE-TTP.We sought novel approaches to improve transfection efficiencies of microRNAs (miRNAs) in platelets, and to apply these approaches to investigate the roles of miRNAs in regulating signal-activated protein translation and functional effects. We found that ex vivo human platelets support gymnosis---internalization of ectopic miRNAs following co-incubation in the absence of conventional transfection reagents or schemes---and subsequently incorporate transfected miRNA into ARGONAUTE2 (AGO2)-based RNA-induced silencing complexes (RISC). Thrombin/fibrinogen stimulation activated translation of miR-223-3p target SEPTIN2, which was suppressed by miR-223-3p transfection in an AGO2/RISC-dependent manner. Navitoclax cost Thrombin/fibrinogen-induced exosome and microvesicle generation was inhibited by miR-223-3p transfection, and this effect was reversed with a RISC inhibitor. Platelet gymnosis of naked miRNAs appeared to be mediated in part by endocytic pathways including clathrin-dependent and fluid-phase endocytosis and caveolae. These results demonstrate the ability of ex vivo platelets to internalize ectopic miRNAs by unassisted transfection, and utilize them to modulate signal-activated translation and platelet function. Our results identify new roles for miR-223-3p in extracellular vesicle generation in stimulated platelets. High-efficiency gymnotic transfection of miRNAs in ex vivo platelets may be a broadly useful tool for exploring molecular genetic regulation of platelet function.Cardiovascular events occur most frequently in the early morning. Similarly, the release of reticulated platelets (RP) by megakaryocytes has a peak in the late night and early morning. Which aspirin regimen most effectively inhibits platelets during these critical hours is unknown. Hence, the primary objective of this trial was to assess platelet function and RP levels at 8.00 AM, in stable cardiovascular (CVD) patients, during three different aspirin regimens. In this open-label randomized cross-over study subjects were allocated to three sequential aspirin regimens once-daily (OD) 80 mg morning; OD-evening, and twice-daily (BID) 40 mg. Platelet function was measured at 8.00 AM & 8.00 PM by serum Thromboxane B2 (sTxB2) levels, the Platelet Function Analyzer (PFA)-200® Closure Time (CT), Aspirin Reaction Units (ARU, VerifyNow®), and RP levels. In total, 22 patients were included. At 8.00 AM, sTxB2 levels were the lowest after OD-evening in comparison with OD-morning (p = less then 0.01), but not in comparison with BID.