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Usefulness of YouTube in Sharing Information about New Gene Therapy for Spinal Muscular Atrophy: A Content Analysis
Get PDFThis study aimed to objectively assess YouTube videos' quality, reliability, and information delivery capability regarding novel spinal muscular atrophy treatments. Using the keywords "nusinersen", "spinraza", "ridisplam", "evrysdi", "onasemnogene abeparvovec", and "zolgensma", we were able to retrieve and screen 360 videos before settling on a final sample of 99 on 25 September 2022. Then, two independent raters used the mDISCERN and GQS instruments to evaluate the videos' reliability and quality and the Information Delivery Capability (IDC) score to assess the videos' accuracy and patient-friendliness. The quality, reliability, and information delivery capability of the videos about the new treatment for SMA were quite heterogeneous, with an average mDISCERN, GQS, and IDC score of 3.172 ± 0.899, 2.980 ± 1.025, and 4.141 ± 1.747, respectively. In-depth analysis showed that healthcare expert videos that explained contents while showing infographic supplements had good quality, reliability, and information delivery capability. As YouTube is already a dominant media platform, the public may obtain new information about novel therapeutics for SMA through YouTube. It is necessary to consider how SMA patients and caregivers can choose trusted sources with reliable information on YouTube, and our results can provide clues. Additionally, experts should strive to provide more accurate, reliable, and patient-oriented videos.ope
Synthetic Strategies for Improving Solubility: Optimization of Novel Pyrazolo[1,5- a]pyrimidine CFTR Activator That Ameliorates Dry Eye Disease
Get PDFDry eye disease (DED) is one of the most prevalent ocular diseases but has limited treatment options. Cystic fibrosis transmembrane conductance regulator (CFTR), a major chloride channel that stimulates fluid secretion in the ocular surface, may pave the way for new therapeutic strategies for DED. Herein, we report the optimization of Cact-3, a potent CFTR activator with poor solubility, to 16d, a potent CFTR activator with suitable solubility for eye drop formulation. Notably, 16d was well distributed in target tissues including cornea and conjunctiva with minimal systemic exposure in rabbit. Topical ocular instillation of 16d significantly enhanced tear secretion and improved corneal erosion in a mouse model of DED. In addition, 16d significantly reduced mRNA expression of pro-inflammatory cytokines including IL-1β, IL-17, and TNF-α and MMP2 in cornea and conjunctiva of DED mice.ope
Early Invasive Strategy Based on the Time of Symptom Onset of Non-ST-Segment Elevation Myocardial Infarction
No full textBackground: A limitation of the current guidelines regarding the timing of invasive coronary angiography for patients with non-ST-segment elevation acute coronary syndrome is the randomization time. To date, no study has reported the clinical outcomes of invasive strategy timing on the basis of the time of symptom onset.
Objectives: The aim of this study was to investigate the effect of invasive strategy timing from the time of symptom onset on the 3-year clinical outcomes of patients with non-ST-segment elevation myocardial infarction (NSTEMI).
Methods: Among 13,104 patients from the Korea Acute Myocardial Infarction Registry-National Institutes of Health, 5,856 patients with NSTE myocardial infarction were evaluated. The patients were categorized according to symptom-to-catheter (StC) time (<48 or ≥48 hours). The primary outcome was 3-year all-cause mortality.
Results: Overall, 3,919 patients (66.9%) were classified into the StC time <48 hours group. This group had lower all-cause mortality than the group with StC time ≥48 hours (7.3% vs 13.4%; P < 0.001). The lower risk for all-cause mortality in the group with StC time <48 hours group was consistent in all subgroups. Notably, emergency medical service use (HR: 0.31; 95% CI: 0.19-0.52) showed a lower risk for all-cause mortality than no emergency medical service use (HR: 0.54; 95% CI: 0.46-0.65; P value for interaction = 0.008).
Conclusions: An early invasive strategy on the basis of StC time was associated with a decreased risk for all-cause mortality in patients with NSTEMI. Because the study was based on a prospective registry, the results should be considered hypothesis generating, highlighting the need for further research. (iCReaT Study No. C110016).restrictio
Aggressive NK Cell Leukemia with RAB29-NUCKS1 Gene Rearrangement: A Case Report
Get PDFAggressive natural killer (NK) cell leukemia (ANKL) is a rare form of leukemia that may be accompanied by various chromosomal abnormalities such as del(6)(q21q25) or del(11q). Here, we describe a case of a patient with ANKL and an RAB29-NUCKS1 rearrangement that has never been described before. An RNA fusion panel test found a gene fusion between exon 5 of RAB29 and exon 2 of NUCKS1 at chromosome 1q32.1. Additionally, reverse-transcription polymerase chain reaction and Sanger sequencing confirmed cryptic RAB29-NUCKS1 fusion. RAB29 encodes a protein that regulates exocytic and endocytic pathways. NUCKS1 encodes a chromatin-associated protein involved in DNA damage responses. Further studies will be necessary to understand the molecular pathogenesis of ANKL related to an RAB29-NUCKS1 rearrangement.ope
Clinical benefits of concomitant surgical ablation for atrial fibrillation in patients undergoing mitral valve surgery
No full textBackground: The maze procedure is the dominant concomitant surgery performed with mitral valve (MV) surgery in patients with atrial fibrillation (AF). Most clinical recommendations regarding the maze procedure depend on the individual maze expert centers.
Objective: The purpose of this study was to evaluate the clinical benefits of the maze procedure during MV surgery in a national cohort.
Methods: Using the national health claims database established by the National Health Insurance Service of South Korea, data on subjects with AF who had undergone MV surgery from 2009 to 2017 were reviewed. The outcomes of interest were mortality; occurrence of ischemic or hemorrhagic stroke; hospitalization for bleeding events; and the composite of death, cerebrovascular accident, and major bleeding. Propensity score (PS) matching was performed for baseline adjustment.
Results: Among 9501 subjects, the maze procedure was performed in 5508 (58.0%). In the PS-matched cohort (3376 pairs), the risk of the composite event was significantly lower in the maze group (hazard ratio [HR] 0.799; 95% confidence interval [CI] 0.731-0.873) than in the nonmaze group. The superiority of the maze procedure was similar for individual clinical events, including death (HR 0.795; 95% CI 0.711-0.889); ischemic stroke (HR 0.788; 95% CI 0.67-0.926); and major bleeding (HR 0.749; 95% CI 0.627-0.895), but not for hemorrhagic stroke (HR 0.984; 95% CI 0.768-1.262). In subgroup analyses of the composite events, these benefits were consistent among subjects aged ≥70 years or <70 years, surgery type (replacement vs repair), MV pathologies, and subjects with CHA2DS2-VASc score ≥4 or <4.
Conclusion: The addition of the maze procedure during MV surgery provided protective effects in the composite outcome of interest.restrictio
Impact of physiological parameters on the parotid gland fat fraction in a normal population
Get PDFQuantifying physiological fat tissue in the organs is important to further assess the organ's pathologic status. This study aimed to investigate the impact of body mass index (BMI), age, and sex on the fat fraction of normal parotid glands. Patients undergoing magnetic resonance imaging (MRI) of iterative decomposition of water and fat with echo asymmetry and least squares estimation (IDEAL-IQ) due to non-salivary gland-related disease were reviewed. Clinical information of individual patients was categorized into groups based on BMI (under/normal/overweight), age (age I/age II/age III), and sex (female/male) and an inter-group comparison of the fat fraction values of both parotid glands was conducted. Overall, in the 626 parotid glands analyzed, the fat fraction of the gland was 35.80%. The mean fat fraction value increased with BMI (30.23%, 35.74%, and 46.61% in the underweight, normal and overweight groups, respectively [p < 0.01]) and age (32.42%, 36.20%, and 41.94% in the age I, II, and III groups, respectively [p < 0.01]). The fat content of normal parotid glands varies significantly depending on the body mass and age regardless of sex. Therefore, the patient's age and body mass should be considered when evaluating fatty change in the parotid glands in imaging results.ope
A Comparative Analysis of Bi-Portal Endoscopic Spine Surgery and Unilateral Laminotomy for Bilateral Decompression in Multilevel Lumbar Stenosis Patients
Get PDFThe clinical and radiological results before and after surgery were compared and analyzed for patients with multilevel lumbar stenosis who underwent bi-portal endoscopic spine surgery (BESS) and microscopic unilateral laminotomy for bilateral decompression (ULBD). We retrospectively identified 47 and 49 patients who underwent BESS and microscopic ULBD, respectively, who were diagnosed with multi-level lumbar stenosis. Clinical outcomes were evaluated using the visual analog scale score for both back and leg pain, and medication (pregabalin) use and Oswestry Disability Index (ODI) scores for overall treatment outcomes were used pre-operatively and at the final follow-up. Radiological outcomes were evaluated as the percentage of dura expansion volume, and percentage preservation of both facets and both lateral recess angles. The follow-up period of patients was about 17.04 months in the BESS group and about 16.90 months in the microscopic ULBD group. The back and leg visual analog scale (VAS) scores and average pregabalin use decreased more significantly in the BESS group than in the microscopic ULBD group (each p-value 0.0443, <0.001, 0.0378). All radiological outcomes were significantly higher in the BESS group than in the ULBD group. The change in ODI in two-level spinal stenosis showed a significantly higher value in the BESS group compared to the microscopic ULBD group (p-value 0.0335). Multilevel decompression with the BESS technique in multiple spinal stenosis is an adequate technique as it shows better clinical and radiological results than microscopic ULBD during a short-term follow-up period.ope
Altered gut microbiota in individuals with episodic and chronic migraine
Get PDFEmerging evidence reveals a close association between gut microbiota and human neurological disorders. The present study aimed to assess whether the composition of gut microbiota in participants with episodic migraine (EM) and chronic migraine (CM) was altered in comparison to that of the controls. This study was a cross-sectional, case-control study. The gut microbiota were evaluated by the partial, targeted sequencing of the 16S rRNA V3-V4 region. This study enrolled 42 and 45 participants with EM and CM, respectively, and 43 controls. Alpha and beta diversities revealed no significant difference among the three groups; however, the microbiota composition at the class, order, family, and genus levels differed significantly between EM and the control, CM and the control, and the EM and CM groups. Moreover, higher composition of PAC000195_g was significantly associated with a lower headache frequency among the five genera that exhibited significantly different microbiota composition in EM and CM. Agathobacter revealed a significant negative association with severe headache intensity. The findings of the present study provide evidence of altered gut microbiota in EM and CM. These findings will help in understanding the course and treatment of migraine.ope
Impact of frailty on early rhythm control outcomes in older adults with atrial fibrillation: A nationwide cohort study
Get PDFPurpose: Rhythm-control therapy administered early following the initial diagnosis of atrial fibrillation (AF) has superior cardiovascular outcomes compared to rate-control therapy. Frailty is a key factor in identifying older patients' potential for improvement after rhythm-control therapy. This study evaluated whether frailty affects the outcome of early rhythm-control therapy in older patients with AF.
Methods: From the Korean National Health Insurance Service database (2005-2015), we collected 20,611 populations aged ≥65 years undergoing rhythm- or rate-control therapy initiated within 1 year of AF diagnosis. Participants were emulated by the EAST-AFNET4 trial, and stratified into non-frail, moderately frail, and highly frail groups based on the hospital frailty risk score (HFRS). A composite outcome of cardiovascular-related mortality, myocardial infarction, hospitalization for heart failure, and ischemic stroke was compared between rhythm- and rate-control.
Results: Early rhythm-control strategy showed a 14% lower risk of the primary composite outcome in the non-frail group [weighted incidence 7.3 vs. 8.6 per 100 person-years; hazard ratio (HR) 0.86, 95% confidence interval (CI) 0.79-0.93, p < 0.001] than rate-control strategy. A consistent trend toward a lower risk of early rhythm-control was observed in the moderately frail (HR 0.91, 95% CI 0.81-1.02, p = 0.09) and highly frail (HR 0.93, 95% CI 0.75-1.17, p = 0.55) groups.
Conclusion: Although the degree attenuated with increasing frailty, the superiority of cardiovascular outcomes of early rhythm-control in AF treatment was maintained without increased risk for safety outcomes. An individualized approach is required on the benefits of early rhythm-control therapy in older patients with AF, regardless of their frailty status.ope
The Reclassification of Patients With Previously Diagnosed Eosinophilic Granulomatosis With Polyangiitis Based on the 2022 ACR/EULAR Criteria for Antineutrophil Cytoplasmic Antibody-Associated Vasculitis
No full textObjective: The American College of Rheumatology (ACR) and the European Alliance of Associations for Rheumatology (EULAR) have proposed the 2022 classification criteria for eosinophilic granulomatosis with polyangiitis (EGPA). This study applied the 2022 ACR/EULAR criteria to Korean patients with previously diagnosed EGPA to investigate the concordance rate between the 2022 ACR/EULAR criteria and the old criteria for EGPA.
Methods: In total, 51 patients with EGPA who met the 1990 ACR criteria, the 2007 European Medicines Agency algorithm, and the 2012 Chapel Hill Consensus Conference definitions were reclassified based on the 2022 ACR/EULAR criteria.
Results: Of 51 patients, 44 (86.3%) were reclassified as having EGPA according to the 2022 ACR/EULAR criteria. Among the 7 patients who failed to meet the 2022 ACR/EULAR criteria, 3 patients were reclassified as having microscopic polyangiitis (MPA) and 1 was reclassified as having granulomatosis with polyangiitis (GPA) based on the 2022 ACR/EULAR criteria; as well, 3 patients were reclassified as having unclassifiable vasculitis. Moreover, 6 patients who met the 2022 ACR/EULAR criteria for EGPA simultaneously met the criteria for MPA based on the 2022 ACR/EULAR criteria for MPA, and 1 who met the criteria for EGPA simultaneously met the criteria for GPA based on the 2022 ACR/EULAR criteria for GPA.
Conclusion: The concordance rate between the 2022 ACR/EULAR criteria for EGPA and the old criteria was 86.3%. The most important factor in the failure to reclassify patients as having EGPA was the exclusion of nonfixed pulmonary infiltrates in the 1990 ACR criteria for EGPA. We cautiously suggest reconsidering nonfixed pulmonary infiltrates in cases reclassified as unclassifiable vasculitis. Further, additional classification strategies are needed for patients who simultaneously satisfy both antineutrophil cytoplasmic antibody-associated vasculitis subtypes.restrictio