Purpose: This multicenter, open-label, phase II trial evaluated the efficacy and safety of bortezomib combined with dexamethasone for the treatment of relapsed/refractory cutaneous T-cell lymphoma (CTCL) in previously treated patients across 14 institutions in South Korea. Materials and Methods: Between September 2017 and July 2020, 29 patients with histologically confirmed CTCL received treatment, consisting of eight 4-week cycles of induction therapy followed by maintenance therapy, contingent upon response, for up to one year. The primary endpoint was the proportion of patients achieving an objective global response. Results: Thirteen of the 29 patients (44.8%) achieved an objective global response, including two complete responses. The median progression-free survival (PFS) was 5.8 months, with responders showing a median PFS of 14.0 months. Treatment-emergent adverse events were generally mild, with a low incidence of peripheral neuropathy and hematologic toxicities. Despite the trend toward shorter PFS in patients with higher mutation burdens, genomic profiling before and after treatment showed no significant emergence of new mutations indicative of disease progression. Conclusion This study supports the use of bortezomib and dexamethasone as a viable and safe treatment option for previously treated CTCL, demonstrating substantial efficacy and manageability in adverse effects. Further research with a larger cohort is suggested to validate these findings and explore the prognostic value of mutation profiles.

Purpose: This multicenter, open-label, phase II trial evaluated the efficacy and safety of bortezomib combined with dexamethasone for the treatment of relapsed/refractory cutaneous T-cell lymphoma (CTCL) in previously treated patients across 14 institutions in South Korea. Materials and Methods: Between September 2017 and July 2020, 29 patients with histologically confirmed CTCL received treatment, consisting of eight 4-week cycles of induction therapy followed by maintenance therapy, contingent upon response, for up to one year. The primary endpoint was the proportion of patients achieving an objective global response. Results: Thirteen of the 29 patients (44.8%) achieved an objective global response, including two complete responses. The median progression-free survival (PFS) was 5.8 months, with responders showing a median PFS of 14.0 months. Treatment-emergent adverse events were generally mild, with a low incidence of peripheral neuropathy and hematologic toxicities. Despite the trend toward shorter PFS in patients with higher mutation burdens, genomic profiling before and after treatment showed no significant emergence of new mutations indicative of disease progression. Conclusion This study supports the use of bortezomib and dexamethasone as a viable and safe treatment option for previously treated CTCL, demonstrating substantial efficacy and manageability in adverse effects. Further research with a larger cohort is suggested to validate these findings and explore the prognostic value of mutation profiles.

Purpose: This multicenter, open-label, phase II trial evaluated the efficacy and safety of bortezomib combined with dexamethasone for the treatment of relapsed/refractory cutaneous T-cell lymphoma (CTCL) in previously treated patients across 14 institutions in South Korea. Materials and Methods: Between September 2017 and July 2020, 29 patients with histologically confirmed CTCL received treatment, consisting of eight 4-week cycles of induction therapy followed by maintenance therapy, contingent upon response, for up to one year. The primary endpoint was the proportion of patients achieving an objective global response. Results: Thirteen of the 29 patients (44.8%) achieved an objective global response, including two complete responses. The median progression-free survival (PFS) was 5.8 months, with responders showing a median PFS of 14.0 months. Treatment-emergent adverse events were generally mild, with a low incidence of peripheral neuropathy and hematologic toxicities. Despite the trend toward shorter PFS in patients with higher mutation burdens, genomic profiling before and after treatment showed no significant emergence of new mutations indicative of disease progression. Conclusion This study supports the use of bortezomib and dexamethasone as a viable and safe treatment option for previously treated CTCL, demonstrating substantial efficacy and manageability in adverse effects. Further research with a larger cohort is suggested to validate these findings and explore the prognostic value of mutation profiles.

Background and Objectives: This study aimed to comprehensively assess the factors influencing hearing ability in patients with vestibular schwannoma, focusing on tumor size, location, primary complaint, and contralateral hearing threshold.Subjects and Method This was a retrospective analysis of 50 patients diagnosed with vestibular schwannoma by magnetic resonance imaging (MRI) at Nowon Eulji Medical Center. Tumor size and location were evaluated using a 3-T MRI system, and pure-tone threshold and speech discrimination were retrieved. To investigate the relationship between tumor size and hearing, we conducted a multiple regression analysis with several variables, including tumor size, tumor location, patient age, and gender. Results: When patients were categorized into groups according to their primary complaint at initial presentation, no significant differences were observed in pure tone thresholds but different speech discrimination were detected among the groups. When patients were classified according to the tumor location, the extrameatal group exhibited lower pure-tone thresholds and higher speech discrimination scores than the intrameatal group. Multiple regression analysis indicated that differences in pure tone thresholds and speech discrimination scores between affected and unaffected sides were more pronounced in cases with smaller tumor sizes and extrameatal tumor locations, emphasizing the greater impact of location over size. Conclusion Using both tumor size and location, it is possible to predict the differences in pure-tone thresholds and speech discrimination between the affected and unaffected sides. Extrameatal invasion of the tumor appears to be the most important factor among them.

Background and Objectives: This study aimed to comprehensively assess the factors influencing hearing ability in patients with vestibular schwannoma, focusing on tumor size, location, primary complaint, and contralateral hearing threshold.Subjects and Method This was a retrospective analysis of 50 patients diagnosed with vestibular schwannoma by magnetic resonance imaging (MRI) at Nowon Eulji Medical Center. Tumor size and location were evaluated using a 3-T MRI system, and pure-tone threshold and speech discrimination were retrieved. To investigate the relationship between tumor size and hearing, we conducted a multiple regression analysis with several variables, including tumor size, tumor location, patient age, and gender. Results: When patients were categorized into groups according to their primary complaint at initial presentation, no significant differences were observed in pure tone thresholds but different speech discrimination were detected among the groups. When patients were classified according to the tumor location, the extrameatal group exhibited lower pure-tone thresholds and higher speech discrimination scores than the intrameatal group. Multiple regression analysis indicated that differences in pure tone thresholds and speech discrimination scores between affected and unaffected sides were more pronounced in cases with smaller tumor sizes and extrameatal tumor locations, emphasizing the greater impact of location over size. Conclusion Using both tumor size and location, it is possible to predict the differences in pure-tone thresholds and speech discrimination between the affected and unaffected sides. Extrameatal invasion of the tumor appears to be the most important factor among them.

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.

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: This study aimed to comprehensively assess the factors influencing hearing ability in patients with vestibular schwannoma, focusing on tumor size, location, primary complaint, and contralateral hearing threshold.Subjects and Method This was a retrospective analysis of 50 patients diagnosed with vestibular schwannoma by magnetic resonance imaging (MRI) at Nowon Eulji Medical Center. Tumor size and location were evaluated using a 3-T MRI system, and pure-tone threshold and speech discrimination were retrieved. To investigate the relationship between tumor size and hearing, we conducted a multiple regression analysis with several variables, including tumor size, tumor location, patient age, and gender. Results: When patients were categorized into groups according to their primary complaint at initial presentation, no significant differences were observed in pure tone thresholds but different speech discrimination were detected among the groups. When patients were classified according to the tumor location, the extrameatal group exhibited lower pure-tone thresholds and higher speech discrimination scores than the intrameatal group. Multiple regression analysis indicated that differences in pure tone thresholds and speech discrimination scores between affected and unaffected sides were more pronounced in cases with smaller tumor sizes and extrameatal tumor locations, emphasizing the greater impact of location over size. Conclusion Using both tumor size and location, it is possible to predict the differences in pure-tone thresholds and speech discrimination between the affected and unaffected sides. Extrameatal invasion of the tumor appears to be the most important factor among them.

Background and Objectives: This study aimed to comprehensively assess the factors influencing hearing ability in patients with vestibular schwannoma, focusing on tumor size, location, primary complaint, and contralateral hearing threshold.Subjects and Method This was a retrospective analysis of 50 patients diagnosed with vestibular schwannoma by magnetic resonance imaging (MRI) at Nowon Eulji Medical Center. Tumor size and location were evaluated using a 3-T MRI system, and pure-tone threshold and speech discrimination were retrieved. To investigate the relationship between tumor size and hearing, we conducted a multiple regression analysis with several variables, including tumor size, tumor location, patient age, and gender. Results: When patients were categorized into groups according to their primary complaint at initial presentation, no significant differences were observed in pure tone thresholds but different speech discrimination were detected among the groups. When patients were classified according to the tumor location, the extrameatal group exhibited lower pure-tone thresholds and higher speech discrimination scores than the intrameatal group. Multiple regression analysis indicated that differences in pure tone thresholds and speech discrimination scores between affected and unaffected sides were more pronounced in cases with smaller tumor sizes and extrameatal tumor locations, emphasizing the greater impact of location over size. Conclusion Using both tumor size and location, it is possible to predict the differences in pure-tone thresholds and speech discrimination between the affected and unaffected sides. Extrameatal invasion of the tumor appears to be the most important factor among them.

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.