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).

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.

Psoriasis is a common chronic inflammatory disease in which keratinocytes proliferate abnormally due to excessive immune action. Psoriasis can be associated with various comorbidities and has a significant impact on health-related quality of life. Although many systemic treatments, including biologic agents, have been developed, topical treatment remains the main option for psoriasis management. Consequently, there is an urgent need to develop topical treatments with minimal side effects and high efficacy. Mesenchymal stem cells (MSCs) exhibit excellent immune regulation, anti-inflammatory activities, and therapeutic effects, and MSC-derived extracellular vesicles (EVs) can serve as crucial mediators of functional transfer from MSCs. Therefore, this study aimed to develop a safe and easy-to-use emulsion cream for treating psoriasis using MSC conditioned media (CM) containing EVs. We developed an enhanced Wharton’s jelly MSC (WJ-MSC) culture method through a three-dimensional (3D) culture containing exogenous transforming growth factor-β3. Using the 3D culture system, we obtained CM from WJ-MSCs, which yielded a higher EV production compared to that of conventional WJ-MSC culture methods, and investigated the effect of EV-enriched 3D-WJMSC-CM cream on psoriasis-related inflammation. Administration of the EV-enriched 3D-WJ-MSC-CM cream significantly reduced erythema, thickness, and scaling of skin lesions, alleviated imiquimod-induced psoriasiform lesions in mice, and ameliorated histopathological changes in mouse skin. The upregulated mRNA expression of inflammatory cytokines, including IL-17a, IL-22, IL-23, and IL-36, decreased in the lesions. In conclusion, we present here a new topical treatment for psoriasis using an MSC EV-enriched cream.

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.

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.