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Hematologic and molecular responses to ropeginterferon alfa-2b therapy of polycythemia vera: 48-week results from a prospective study
No full textTo prevent thrombosis in patients with polycythemia vera (PV), achieving a complete hematologic response (CHR) is highly recommended in practice. In addition, a reduced JAK2 V617F mutation burden is expected to have a disease-modifying effect, and its molecular response (MR) is currently of significant interest. This study aimed to assess the association between CHR and MR in patients with PV following treatment with ropeginterferon alfa-2b. This phase 2, single-arm, open-label, investigator-initiated trial was conducted at 16 sites in South Korea. Ninety-nine patients were treated with ropeginterferon alfa-2b subcutaneously every 2 weeks, at doses of 250 mug (week 1), 350 mug (week 3), and 500 mug (week 5), until week 48. CHRs were 27% (25/94), 46% (40/87), 56% (47/84), and 63% (51/81) at 12, 24, 36, and 48 weeks, respectively. The MR rates were 32% (28/88), 36% (29/81), 49% (38/77), and 57% (42/74) at 12, 24, 36, and 48 weeks, respectively. The Phi Coefficient for the association between CHR and MR was 0.6146 (p < .0001) at 48 weeks. In the subgroup analysis, patients with hydroxyurea resistance or intolerance, and those who were hydroxyurea-naive, had similar results in terms of the CHR. In conclusion, CHR and MR were observed to be associated in patients with PV treated with ropeginterferon
Efficacy and safety of idursulfase beta in the treatment of mucopolysaccharidosis II: A phase-3, 2-part study compared with a historical placebo cohort
No full textPURPOSE: To investigate the efficacy and safety of idursulfase beta (0.5 mg/kg weekly) in the treatment of mucopolysaccharidosis II, compared with a historical placebo from a previous idursulfase trial (TKT024). METHODS: The study comprised 2 sequential parts. In part 1, a randomized, double-blind study, idursulfase or idursulfase beta were given for 52 weeks. In the open, single-cohort part 2 study, additional participants received idursulfase beta for 52 weeks. Data from the idursulfase beta groups from parts 1 and 2 were pooled for comparisons with the historical placebo group (n = 32). The primary end point was a change in the 6-minute walk test at week 53. RESULTS: Participants in the idursulfase beta groups (n = 24) were male Asians (mean age, 12.0 years). Idursulfase beta was superior to placebo in 6-minute walk test improvement (62.2 vs 7.3 m, P < .0001). Decrease in urine glycosaminoglycan excretion (-71.13% vs 21.39%, P < .0001) and reduction in the liver (-26.67% vs -0.80%, P < .0001) and spleen volumes (-26.46% vs 7.2%, P < .0001) were significant. The safety profile of idursulfase beta was satisfactory. CONCLUSION: Idursulfase beta is a safe and effective treatment option in mucopolysaccharidosis II, addressing crucial somatic ailments presented by patients
Effects of Oral Nutrition Supplementation with or Without Multi-Domain Intervention Program on Cognitive Function and Overall Health in Older Adults: A Randomized Controlled Trial
No full textObjectives: The global rise in dementia prevalence is escalating the socioeconomic burden, prompting efforts in prevention and treatment. This study aimed to evaluate the effects of an 8-week oral nutrition supplement (ONS) program with or without a multi-domain intervention program (MIP) in patients with mild cognitive impairment. Methods: Forty-nine patients with mild cognitive impairment were divided into three groups: (1) ONS (ONS), (2) ONS+MIP (ONS+MIP), and (3) control (CON). Korean-style dementia prevention MIP was used in the ONS+MIP group. Two packs of ONS per day were provided to the ONS group during the intervention period. Cognitive function, nutritional evaluation, body composition analysis, and physical performance were measured. The paired t-test and one-way analysis of variance were used for statistical analyses. Results: A final analysis was performed on the final 46 participants. After intervention, the cognitive function test (Repeatable Battery for the Assessment of Neuropsychological Status, RBANS) scores of the ONS+MIP group were significantly increased compared to the baseline scores. However, no significant changes were observed in the ONS and CON groups. Eating behavior and food quality also improved in the ONS+MIP group, with a significant difference among the three groups. There was no significant change in body composition in all groups; however, grip strength (left hand), muscular endurance, and the total SPPB score improved in the ONS+MIP group. The total SPPB score decreased in the CON group. Conclusions: Along with ONS intake, intensive education and continuous monitoring through multi-domain interventions are important to improve cognitive function. Trial registration: Clinical Research Information Service identifier: KCT0007253
A4GALT-targeting siRNA lipid nanoparticles ameliorate Fabry disease phenotype: Greater efficacy in endothelial cells than in podocytes
No full textIn this study, we explore the therapeutic feasibility of globotriaosylceramide (Gb3) synthase (A4GALT)-specific siRNA-loaded polyhistidine (pHis)-incorporated lipid nanoparticles (HLNPs) for Fabry disease (FD). HLNPs were developed to deliver siRNAs targeting A4GALT using a microfluidic device, with pHis aiding in endosome escape. The therapy was tested on GLA-knockout human-induced pluripotent-stem-cell-derived endothelial cells (GLA-KO-hiPSC-ECs) and podocytes (GLA-KO-hiPSC-PCs). GLA-KO-hiPSCs-ECs or -PCs, upon differentiation, were treated with A4GALT-siRNA-HLNP. Successful intracellular uptake of A4GALT-siRNA-HLNP was confirmed through fluorescence and electron microscopy in both cell types. A4GALT-siRNA-HLNP treatment confirmed both cell types' stability at 5 mug/mL. Increased Gb3 deposition and zebra body formation were detected in both cell types, but A4GALT-siRNA-HLNP treatment attenuated these FD phenotypes, demonstrating reduced expression of A4GALT through western blot analysis. RNA sequencing analysis revealed that the expression of transcripts associated with FD was restored by A4GALT-siRNA-HLNP treatment in GLA-KO-hiPSCs-ECs, whereas in GLA-KO-hiPSCs-PCs, this effect was relatively less pronounced. Suppression of A4GALT via siRNA/HLNP treatment significantly rescued FD phenotypes especially in EC, presenting a novel therapeutic approach for FD
A comparison of soft tissue outcomes in skeletal Class II malocclusion: Anterior segmental osteotomy vs. non-surgical orthodontic treatment
No full textOBJECTIVE: Temporary anchorage devices (TADs) have considerably reduced the need for anterior segmental osteotomy (ASO) in patients with Class I malocclusion. Most previous studies have been published before the widespread use of TADs, thus warranting new guidelines for determining the optimal approach for surgery and orthodontic treatment. This study aimed to establish guidelines on the choice between ASO and non-ASO (NASO) based on soft tissue considerations. METHODS: Sixty-seven patients diagnosed with skeletal Class II malocclusion were divided into the ASO (n = 31) and NASO (n = 36) groups. Cephalometric analyses were used to compare the initial and final records to assess the effect of treatment on soft tissues. The interlabial gap, upper lip anterior to the E-line, lower lip anterior to the E-line, H-angle, upper lip to the nasion-perpendicular line, and nasolabial angle were evaluated. In particular, a proportional difference indicator between the upper and lower lips relative to the pogonion angle between the facial plane and CK line was presented, followed by statistics analyses. Statistical significance was set at P < 0.05. RESULTS: Both groups demonstrated normal proportions of the upper and lower lips; however, significant differences favoring ASO over NASO in terms of soft tissue changes were observed for several variables. CONCLUSIONS: ASO is advised if the required adjustment for the upper and lower lips is -4.0 mm and -5.0 mm, respectively. For modifications of -2.0 mm, NASO is preferred. This study provides clinical guidelines on the choice between ASO and NASO based on the required lip movement measurements
Risk factors and impact of hypertriglyceridemia in preterm infants under 32 weeks of gestation: optimizing intravenous lipid emulsion infusion rates–a single center retrospective study
No full textINTRODUCTION: Intravenous lipid emulsions are essential for parenteral nutrition in preterm infants. However, hypertriglyceridemia (HTG) is a common complication that can lead to severe complications if left untreated. This study analyzed factors influencing HTG and the effects of reduced intravenous lipid emulsion dosages. METHODS: A retrospective cohort study involving premature infants born at less than 32 weeks of gestation and admitted to Ajou University Hospital between 2017 and 2022 was conducted. Infants with documented triglyceride levels within the first 10 days of life were included. The risk factors for HTG and the time to normalize triglyceride levels after intravenous lipid dose reduction were evaluated. RESULTS: HTG was diagnosed in 104 infants. Significant risk factors included lower birth weight (OR = 0.75; 95% CI, 0.66-0.85; p < 0.001), treated for patent ductus arteriosus (OR = 2.47; 95% CI, 1.30-4.69; p = 0.006), intravenous lipid emulsion intake (OR = 1.79; 95% CI, 1.25-2.55; p = 0.001), and serum glucose level (OR = 1.01; 95% CI, 1.00-1.02; p = 0.023). The average triglyceride level in the HTG group was 287.8 mg/dl, with intravenous lipid doses of 2.7 g/kg/day. Reducing intravenous lipid doses by 0.27 +/- 0.46 g/kg/day shortened the triglyceride normalization time to 4.9 +/- 5.0 days and reduced the risk of retinopathy of prematurity stage 2 or higher (OR = 0.59; 95% CI, 0.40-0.88; p = 0.011). The fat overload infusion rate, which induces HTG, was 0.332 cc/kg/h (1.6 g/kg/day). DISCUSSION: Lower birth weight, treated patent ductus arteriosus, serum glucose levels, and higher intravenous lipid levels are significant risk factors for HTG. Adjusting the lipid dose aids in triglyceride normalization and reducing severe retinopathy of prematurity risk. Careful monitoring and management of intravenous lipid emulsion infusion rates are crucial to prevent HTG
Author Correction: A randomized controlled trial of a digital lifestyle intervention involving postoperative patients with colorectal cancer
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Survey of Perioperative Practices in Gastric Cancer Surgery for Establishing an Enhanced Recovery After Surgery Program Across 10 Tertiary Hospitals in South Korea
No full textPURPOSE: This study aimed to identify the current perioperative management practices for gastric cancer surgery in South Korea and develop a consensus-driven Enhanced Recovery After Surgery (ERAS) protocol for a multicenter randomized controlled trial (RCT). MATERIALS AND METHODS: A survey was conducted with 20 principal investigators, comprising one gastric surgeon and one anesthesiologist each, from 10 tertiary hospitals in South Korea who participated in a planned multicenter RCT. The survey included 41 questions regarding ERAS implementation and department-specific perioperative management practices. The responses were analyzed using descriptive statistics, and the findings were used to develop a consensus-driven ERAS protocol. RESULTS: A total of 20 participants completed the survey. Most respondents estimated the overall compliance rate with ERAS protocols for gastric cancer surgery to be 30%-50%. A major barrier to ERAS implementation is limited personnel resources. The survey revealed significant variability in perioperative practices-particularly in postoperative oral feeding protocols and expected discharge dates-across institutions. Most institutions practice water fasting from midnight before surgery, and the adoption rate of multimodal opioid-sparing analgesia is low. Based on these findings, an ERAS protocol was established through collaborative discussions to shorten perioperative fasting periods and implement multimodal opioid-sparing analgesia. CONCLUSIONS: This study revealed significant variability in the perioperative management of gastric cancer surgery in South Korea. A consensus-driven ERAS protocol was established to standardize care and promote functional recovery. Its feasibility and effectiveness should be evaluated in an upcoming multicenter RCT
Role of Emergent Endoscopic Evaluation for Corrosive Injuries: Should We Do It or Not?
No full textBACKGROUND/AIM: Although emergency endoscopic evaluation of patients presenting with corrosive ingestion may facilitate prognosis prediction, the role of early endoscopic evaluation remains unclear. Therefore, we aimed to select patients who required emergency endoscopic evaluation based on the initial admission information and to investigate the factors associated with Zargar grade >/=2b in patients with corrosive ingestion. PATIENTS AND METHODS: We retrospectively reviewed patients with corrosive ingestion who visited the emergency department between 2010 and 2023. We investigated short- and long-term endoscopic outcomes after corrosive ingestion. Using logistic regression analysis, we investigated the factors associated with endoscopic mucosal damage of Zargar grade >/=2b. RESULTS: A total of 211 patients were enrolled. The most common reason for and type of corrosive ingestion were suicidal intent (n=138, 65.4%) and alkaline substrates (n=181, 85.5%), respectively. The median (interquartile range, IQR) of endoscopy time after admission was 455 min (235-890 min), and 92.9% (n=196) of patients underwent endoscopic evaluation within 48 h. At the initial endoscopic evaluation, 47.9% (n=101) of the patients had no mucosal injury. Stricture was observed in 2.4% (n=5) during the follow-up period, and three (1.4%) deaths occurred. In multivariate logistic analysis, bleeding events [odds ratio (OR)=12.2, 95% confidence interval (CI)=1.619-164.581, p=0.024] and leukocytosis (OR=2.9, 95%CI=1.138-7.331, p=0.023) were significant risk factors for Zargar grade >/=2b. However, the amount ingested was not revealed to be a risk factor for Zargar grade >/=2b. CONCLUSION: Mucosal injury was mild in most patients admitted to the emergency department after corrosive ingestion, regardless of the amount ingested