Background/Aims: A clinical unmet need persists for medications capable of modulating the progression of primary sclerosing cholangitis (PSC). This study aimed to assess the clinical feasibility of HK-660S (beta-lapachone) in PSC. Methods: In this multicenter, randomized, double-blind, placebo-controlled, parallel-group phase 2 trial, participants were assigned in a 2:1 ratio to receive either 100 mg of HK-660S or a placebo twice daily for 12 weeks. The primary outcomes were the reduction in serum alkaline phosphatase (ALP) levels and the percentage of participants showing improvements in PSC severity, as determined by magnetic resonance cholangiopancreatography with the Anali score. Secondary endpoints included changes in liver stiffness and adverse events. Results: The analysis included 21 patients, 15 receiving HK-660S, and six receiving a placebo. Improvements in the Anali score were observed in 13.3% of the HK-660S group, with no improvements in the placebo group. HK-660S treatment resulted in a 15.2% reduction in mean ALP levels, compared to a 6.6% reduction in the placebo group. A stratified ad-hoc analysis based on baseline ALP levels showed a statistically significant response in the HK-660S group among those with ALP levels greater than twice the upper limit of normal, with a 50% responder rate (p=0.05). Additionally, 26.7% of the HK-660S group showed improvements in the enhanced liver fibrosis score, with no improvements in the placebo group. HK-660S was generally well tolerated. Conclusions HK-660S is well tolerated among patients with PSC and may improve bile duct strictures, decrease serum ALP levels, and reduce liver fibrosis (cris.nih.go.kr, Number KCT0006590).

Background/Aims: A clinical unmet need persists for medications capable of modulating the progression of primary sclerosing cholangitis (PSC). This study aimed to assess the clinical feasibility of HK-660S (beta-lapachone) in PSC. Methods: In this multicenter, randomized, double-blind, placebo-controlled, parallel-group phase 2 trial, participants were assigned in a 2:1 ratio to receive either 100 mg of HK-660S or a placebo twice daily for 12 weeks. The primary outcomes were the reduction in serum alkaline phosphatase (ALP) levels and the percentage of participants showing improvements in PSC severity, as determined by magnetic resonance cholangiopancreatography with the Anali score. Secondary endpoints included changes in liver stiffness and adverse events. Results: The analysis included 21 patients, 15 receiving HK-660S, and six receiving a placebo. Improvements in the Anali score were observed in 13.3% of the HK-660S group, with no improvements in the placebo group. HK-660S treatment resulted in a 15.2% reduction in mean ALP levels, compared to a 6.6% reduction in the placebo group. A stratified ad-hoc analysis based on baseline ALP levels showed a statistically significant response in the HK-660S group among those with ALP levels greater than twice the upper limit of normal, with a 50% responder rate (p=0.05). Additionally, 26.7% of the HK-660S group showed improvements in the enhanced liver fibrosis score, with no improvements in the placebo group. HK-660S was generally well tolerated. Conclusions HK-660S is well tolerated among patients with PSC and may improve bile duct strictures, decrease serum ALP levels, and reduce liver fibrosis (cris.nih.go.kr, Number KCT0006590).

Background/Aims: A clinical unmet need persists for medications capable of modulating the progression of primary sclerosing cholangitis (PSC). This study aimed to assess the clinical feasibility of HK-660S (beta-lapachone) in PSC. Methods: In this multicenter, randomized, double-blind, placebo-controlled, parallel-group phase 2 trial, participants were assigned in a 2:1 ratio to receive either 100 mg of HK-660S or a placebo twice daily for 12 weeks. The primary outcomes were the reduction in serum alkaline phosphatase (ALP) levels and the percentage of participants showing improvements in PSC severity, as determined by magnetic resonance cholangiopancreatography with the Anali score. Secondary endpoints included changes in liver stiffness and adverse events. Results: The analysis included 21 patients, 15 receiving HK-660S, and six receiving a placebo. Improvements in the Anali score were observed in 13.3% of the HK-660S group, with no improvements in the placebo group. HK-660S treatment resulted in a 15.2% reduction in mean ALP levels, compared to a 6.6% reduction in the placebo group. A stratified ad-hoc analysis based on baseline ALP levels showed a statistically significant response in the HK-660S group among those with ALP levels greater than twice the upper limit of normal, with a 50% responder rate (p=0.05). Additionally, 26.7% of the HK-660S group showed improvements in the enhanced liver fibrosis score, with no improvements in the placebo group. HK-660S was generally well tolerated. Conclusions HK-660S is well tolerated among patients with PSC and may improve bile duct strictures, decrease serum ALP levels, and reduce liver fibrosis (cris.nih.go.kr, Number KCT0006590).

Pediatric hypertension (HTN) is a significant, growing health concern worldwide and also in Korea. Diagnosis, evaluation, and treatment of HTN in Korean children and adolescents are uncertain due to limitations in using the current international guidelines, since the recommendations by the American Academy of Pediatrics (AAP) and European Society of Hypertension (ESH) guidelines differ. Furthermore, these are guidelines for Western youth, who are racially and ethnically different from Koreans. In addition, reference blood pressure values for all pediatric age groups, which are essential for the diagnosis of HTN according to these two guidelines, are absent in Korea. Therefore, HTN guidelines for Korean children and adolescents should be established. The Korean Working Group of Pediatric Hypertension established clinical guidelines for the diagnosis, evaluation, and management of HTN in Korean children and adolescents. These guidelines were based on reported clinical evidence, expert recommendations, and AAP and ESH guidelines. The characteristics of Korean youth and the Korean medical and insurance system were considered during the establishment of the guidelines. By providing recommendations suitable for Korean youth, these guidelines will help in the prevention and management of childhood HTN, thus relieving the burden of cardiovascular disease in adulthood in Korea.

This study investigated the clinical characteristics and kidney outcomes of childhood-onset lupus nephritis (LN), and risk factors associated with prognosis. Methods: We enrolled 216 patients with histologically diagnosed LN during childhood. The Korean Society of Pediatric Nephrology organized a retrospective cohort study of childhood-onset LN in 13 major pediatric nephrology centers in South Korea. Results: The mean age at kidney biopsy was 13.2 ± 3.22 years. The main forms of presentation were nephrotic syndrome and/or hematuria in 152 patients (70.4%), and the most common histological finding was World Health Organization (WHO) class IV in 138 patients (63.9%), followed by WHO class III in 34 patients (15.7%). In the outcome analysis, the mean follow-up period of the patients was 7.8 ± 5.11 years. At last follow-up, 32 patients (14.8%) developed advanced chronic kidney disease (CKD). Male sex and failure to achieve remission at 12 months of treatment were significant risk factors for developing advanced CKD (hazard ratio of 2.57 and 2.29, respectively). Conclusion: Our study demonstrated the clinical characteristics and long-term outcomes of patients with childhood-onset LN. Male sex and failure to achieve remission in the first year of treatment were predictive of advanced CKD. Therefore, prompt awareness and close monitoring of these high-risk patients are needed, which may further improve the prognosis of children with LN.

This study aimed to develop and evaluate a multimodal deep learning model that integrates panoramic radiographs and clinical data to improve the diagnosis of medication-related osteonecrosis of the jaw (MRONJ). Materials and Methods:The study included 705 panoramic radiographs (401 MRONJ and 304 Normal) collected from Ewha Womans University Mokdong and Seoul Hospitals. Radiographs were paired with patient information, including age, sex, body mass index (BMI), type of medication, duration, route of administration, dosage, and dental risk factors. Two deep learning models were compared: a ResNet-50 model using panoramic radiographs alone and the Modified ResNet-50 model incorporating both image and clinical features. Model performance was evaluated using accuracy, precision, recall, F1-score, and area under the receiver operating characteristic curve (AUC). Lesion localization and feature importance were analyzed using Gradient-weighted Class Activation Mapping (Grad-CAM) and SHapley Additive exPlanations (SHAP). Result: Significant differences in clinical characteristics were observed between the groups; the MRONJ group was older (74.5±10.0 vs. 68.6±12.7 years, P<0.01) and had a higher proportion of females (92.5% vs. 82.2%, P<0.01). In model performance, the Modified ResNet-50 model outperformed the PR-only model. Specifically, the Modified ResNet-50 model achieved an accuracy of 75.0%, a recall of 84.3%, and an AUC of 0.843, whereas the PR-only model showed lower performance with an accuracy of 72.1%, a recall of 81.4%, and an AUC of 0.720. SHAP analysis identified prolonged medication use and specific drug types (e.g., zoledronate, P<0.01) as influential predictors. Conclusion: The proposed multimodal deep learning framework demonstrated improved diagnostic performance and interpretability compared to radiograph-only models. This approach may serve as a practical and explainable AI-assisted tool for MRONJ diagnosis in routine dental practice.

Background and Objectives: We analyzed the data of oropharyngeal squamous cell carcinoma (OPSCC) patients who had transoral surgery with/without adjuvant therapy and experienced recurrence. From the data, the treatment outcomes and prognostic factors of recurrent OPSCC were evaluated, and the predictive factors related to successful salvage treatment were identified.Subjects and Method We used data from patients who were diagnosed with OPSCC and received transoral surgery at the hospital from January 2005 to December 2019. Results: The 5-year survival rate of patients with recurrent OPSCC was 43.9%. The predictors of successful salvage treatment were adjuvant therapy and the p16 status. The 5-year survival rate following salvage treatment for patients who had recurrent OPSCC and also tested p16-positive was 64%; however, it was only 30% for patients who had recurrent OPSCC and tested p16-negative. The 5-year survival rate was 22% for patients who received adjuvant therapy and 64% for those who did not receive it. Conclusion In OPSCC patients who recurred after transoral surgery with/without adjuvant therapy, the salvage treatment success rate was 45%. In recurrent cancer, the HPV status was an important factor associated with successful salvage treatment, as the success rate of salvage treatment was remarkably high in patients who did not receive adjuvant therapy. Thus, we verified that it is crucial to conduct an initial surgery with clear margins and determine the optimal criteria for adjuvant therapy.

Background and Objectives: We analyzed the treatment results and prognostic factors of stage IV oral tongue squamous cell carcinoma (OTSCC) patients and explored the existence of subgroups with distinctive prognoses. In addition, the outcome of salvage therapy was analyzed in recurrent cases, and the survival rates and prognostic factors were investigated.Subjects and Method This study was conducted on patients who were diagnosed with OTSCC and underwent surgery at our hospital between June 2005 and January 2020. A total of 144 patients with stage IV OTSCC was enrolled. Results: A total of 64 recurrences, local (6), regional (21), distant metastasis (33), and locoregional (4), occurred. Seventy-five patients died because of disease progression during the course of study. The 5-year recurrence-free survival rate was 54.5%, and the 5-year disease-specific survival rate was 49.2%. Surgical margins, lymphovascular invasion (LVI), T classification, and lymph nodes (LNs) metastasis exhibited significant correlation with mortality. LVI and advanced T were statistically important factors for predicting distant metastasis. The treatment outcome of the T4N0 patient group without LN metastasis fared the best, while the treatment outcome of the T4N1-3 patient group with advanced T and N findings was the worst. Conclusion The major type of treatment failure in stage IV OTSCC patients was distant metastasis, and the related predictors of distant metastasis were LVI and advanced T. In the stage IV OTSCC patient group, there were subgroups with distinct prognosis according to the combination of T and N classification. The T4N0 group had the best survival rate, and the T4N1-3 group had the worst prognosis.

Background: This study aimed to validate the Geriatric Trauma Outcome Score (GTOS) for predicting mortality associated with trauma in older Korean adults and compare the GTOS with the Trauma and Injury Severity Score (TRISS). Methods: This study included patients aged ≥65 years who visited the Chungbuk National University Hospital Regional Trauma Center between January 2016 and December 2022. We used receiver operating characteristic curves and calibration plots to assess the discrimination and calibration of the scoring systems. Results: Among 3,053 patients, the median age was 77 years, and the mortality rate was 5.2%. The overall GTOS-predicted mortality and 1–TRISS were 5.4% (interquartile range [IQR], 3.7–9.5) and 4.7% (IQR, 4.7–4.7), respectively. The areas under the curves (AUCs) of 1–TRISS and GTOS for the total population were 0.763 (95% confidence interval [CI], 0.719–0.806) and 0.794 (95% CI, 0.755–0.833), respectively. In the Glasgow Coma Scale (GCS) ≤12 group, the in-hospital mortality rate was 27.5% (79 deaths). The GTOS-predicted mortality and 1–TRISS in this group were 18.6% (IQR, 7.5–34.7) and 26.9% (IQR, 11.9–73.1), respectively. The AUCs of 1–TRISS and GTOS for the total population were 0.800 (95% CI, 0.776–0.854) and 0.744 (95% CI, 0.685–0.804), respectively. Conclusion The GTOS and TRISS demonstrated comparable accuracy in predicting mortality, while the GTOS offered the advantage of simpler calculations. However, the GTOS tended to underestimate mortality in patients with GCS ≤12; thus, its application requires care in such cases.

Rare endocrine diseases are complex conditions that require lifelong specialized care due to their chronic nature and associated long-term complications. In Korea, a lack of nationwide data on clinical practice and outcomes has limited progress in patient care. Therefore, the Multicenter Networks for Ideal Outcomes of Pediatric Rare Endocrine and Metabolic Disease (OUTSPREAD) study was initiated. This study involves 30 centers across Korea. The study aims to improve the long-term prognosis of Korean patients with rare endocrine diseases by collecting comprehensive clinical data, biospecimens, and patient-reported outcomes to identify complications and unmet needs in patient care. Patients with childhood-onset pituitary, adrenal, or gonadal disorders, such as craniopharyngioma, congenital adrenal hyperplasia (CAH), and Turner syndrome were prioritized. The planned enrollment is 1,300 patients during the first study phase (2022–2024). Clinical, biochemical, and imaging data from diagnosis, treatment, and follow-up during 1980–2023 were retrospectively reviewed. For patients who agreed to participate in the prospective cohort, clinical data and biospecimens will be prospectively collected to discover ideal biomarkers that predict the effectiveness of disease control measures and prognosis. Patient-reported outcomes, including quality of life and depression scales, will be evaluated to assess psychosocial outcomes. Additionally, a substudy on CAH patients will develop a steroid hormone profiling method using liquid chromatography-tandem mass spectrometry to improve diagnosis and monitoring of treatment outcomes. This study will address unmet clinical needs by discovering ideal biomarkers, introducing evidence-based treatment guidelines, and ultimately improving long-term outcomes in the areas of rare endocrine and metabolic diseases.