Purpose: In this study, we evaluated 66 patients diagnosed with adenoid cystic carcinoma (ACC) enrolled in two Korean Cancer Study Group trials to investigate the response and progression patterns in recurrent and/or metastatic ACC treated with vascular endothelial growth factor receptor tyrosine kinase inhibitors (VEGFR-TKIs). Materials and Methods: We evaluated 66 patients diagnosed with ACC who were enrolled in the Korean Cancer Study Group trials. The tumor measurements, clinical data, treatment outcomes, and progression patterns of therapy were analyzed. Results: In the 66 patients (53 receiving axitinib and 13 receiving nintedanib), the disease control rate was 61%, and three patients achieved partial response. The median follow-up, median progression-free survival (PFS), overall survival, and 6-month PFS rate were 27.6%, 12.4%, and 18.1% months and 62.1%, respectively. Among 42 patients who experienced progression, 27 (64.3%) showed target lesion progression. Bone metastasis was an independent poor prognostic factor. Conclusion Overall, most patients demonstrated stable disease with prolonged PFS; however, prominent target lesion progression occurred in some patients. Thus, PFS may capture VEGFR-TKI efficacy better than the objective response rate.

Purpose: To date, there is no region-specific guideline for pediatric endoscopy training. This study aimed to illustrate the current status of pediatric endoscopy training in Asia-Pacific region and identify opportunities for improvement. Methods: A cross-sectional survey, using a standardized electronic questionnaire, was conducted among medical schools in the Asia-Pacific region in January 2024. Results: A total of 57 medical centers in 12 countries offering formal Pediatric Gastroenterology training programs participated in this regional survey. More than 75% of the centers had an average case load of <10 cases per week for both diagnostic and therapeutic endoscopies. Only 36% of the study programs employed competency-based outcomes for program development, whereas nearly half (48%) used volume-based curricula.Foreign body retrieval, polypectomy, percutaneous endoscopic gastrostomy, and esophageal variceal hemostasis, that is, sclerotherapy or band ligation (endoscopic variceal sclerotherapy and endoscopic variceal ligation), comprised the top four priorities that the trainees should acquire in the autonomous stage (unconscious) of competence. Regarding the learning environment, only 31.5% provided formal hands-on workshops/simulation training. The direct observation of procedural skills was the most commonly used assessment method. The application of a quality assurance (QA) system in both educational and patient care (Pediatric Endoscopy Quality Improvement Network) aspects was present in only 28% and 17% of the centers, respectively. Conclusion Compared with Western academic societies, the limited availability of cases remains a major concern. To close this gap, simulation and adult endoscopy training are essential. The implementation of reliable and valid assessment tools and QA systems can lead to significant development in future programs.

Despite the availability of direct-acting antivirals (DAAs) for chronic hepatitis C virus (HCV) infection in Korea, need remains for pangenotypic regimens that can be used in the presence of hepatic impairment, comorbidities, or prior treatment failure. We investigated the efficacy and safety of sofosbuvir–velpatasvir and sofosbuvir–velpatasvir–voxilaprevir for 12 weeks in HCV-infected Korean adults. Methods: This Phase 3b, multicenter, open-label study included 2 cohorts. In Cohort 1, participants with HCV genotype 1 or 2 and who were treatment-naive or treatment-experienced with interferon-based treatments, received sofosbuvir–velpatasvir 400/100 mg/day. In Cohort 2, HCV genotype 1 infected individuals who previously received an NS5A inhibitor-containing regimen ≥ 4 weeks received sofosbuvir–velpatasvir–voxilaprevir 400/100/100 mg/day. Decompensated cirrhosis was an exclusion criterion. The primary endpoint was SVR12, defined as HCV RNA < 15 IU/mL 12 weeks following treatment. Results: Of 53 participants receiving sofosbuvir–velpatasvir, 52 (98.1%) achieved SVR12. The single participant who did not achieve SVR12 experienced an asymptomatic Grade 3 ASL/ALT elevation on day 15 and discontinued treatment. The event resolved without intervention. All 33 participants (100%) treated with sofosbuvir–velpatasvir–voxilaprevir achieved SVR 12. Overall, sofosbuvir–velpatasvir and sofosbuvir–velpatasvir–voxilaprevir were safe and well tolerated. Three participants (5.6%) in Cohort 1 and 1 participant (3.0%) in Cohort 2 had serious adverse events, but none were considered treatment-related. No deaths or grade 4 laboratory abnormalities were reported. Conclusions: Treatment with sofosbuvir–velpatasvir or sofosbuvir–velpatasvir–voxilaprevir was safe and resulted in high SVR12 rates in Korean HCV patients.

Purpose: Renal tumors account for approximately 7% of all childhood cancers. These include Wilms tumor (WT), clear cell sarcoma of the kidney (CCSK), malignant rhabdoid tumor of the kidney (MRTK), renal cell carcinoma (RCC), congenital mesoblastic nephroma (CMN) and other rare tumors. We investigated the epidemiology of pediatric renal tumors in Korea. Materials and Methods: From January 2001 to December 2015, data of pediatric patients (0–18 years) newly-diagnosed with renal tumors at 26 hospitals were retrospectively analyzed. Results: Among 439 patients (male, 240), the most common tumor was WT (n=342, 77.9%), followed by RCC (n=36, 8.2%), CCSK (n=24, 5.5%), MRTK (n=16, 3.6%), CMN (n=12, 2.7%), and others (n=9, 2.1%). Median age at diagnosis was 27.1 months (range 0-225.5) and median follow-up duration was 88.5 months (range 0-211.6). Overall, 32 patients died, of whom 17, 11, 1, and 3 died of relapse, progressive disease, second malignant neoplasm, and treatment-related mortality. Five-year overall survival and event free survival were 97.2% and 84.8% in WT, 90.6% and 82.1% in RCC, 81.1% and 63.6% in CCSK, 60.3% and 56.2% in MRTK, and 100% and 91.7% in CMN, respectively (p < 0.001). Conclusion The pediatric renal tumor types in Korea are similar to those previously reported in other countries. WT accounted for a large proportion and survival was excellent. Non-Wilms renal tumors included a variety of tumors and showed inferior outcome, especially MRTK. Further efforts are necessary to optimize the treatment and analyze the genetic characteristics of pediatric renal tumors in Korea.

Purpose: Acute promyelocytic leukemia (APL) is a rare disease in children and there are some different characteristics between children and adult. We aimed to evaluate incidence, clinical characteristics and treatment outcomes of pediatric APL in Korea. Materials and Methods: Seventy-nine pediatric APL patients diagnosed from January 2009 to December 2016 in 16 tertiary medical centers in Korea were reviewed retrospectively. Results: Of 801 acute myeloid leukemia children, 79 (9.9%) were diagnosed with APL. The median age at diagnosis was 10.6 years (range, 1.3 to 18.0). Male and female ratio was 1:0.93. Thirty patients (38.0%) had white blood cell (WBC) count greater than 10×109/L at diagnosis. All patients received induction therapy consisting of all-trans retinoic acid and chemotherapy. Five patients (6.6%) died during induction chemotherapy and 66 patients (86.8%) achieved complete remission (CR) after induction chemotherapy. The causes of death were three intracranial hemorrhage, one cerebral infarction, and one sepsis. Five patients (7.1%) suffered a relapse during or after maintenance chemotherapy. The estimated 4-year event-free survival and overall survival (OS) rates were 82.1%±4.4%, 89.7%±5.1%, respectively. The 4-year OS was significantly higher in patients with initial WBC < 10×109/L than in those with initial WBC ≥ 10×109/L (p=0.020). Conclusion This study showed that the CR rates and survival outcomes in Korean pediatric APL patients were relatively good. The initial WBC count was the most important prognostic factor and most causes of death were related to serious bleeding in the early stage of treatment.

Background: Treatment remains a challenge in massive and irreparable rotator cuff tears (RCTs), and superior capsular reconstruction (SCR) has become an increasingly popular choice. The objective of this study was to evaluate clinical and radiological outcomes after SCR using an Achilles tendon allograft in irreparable massive RCTs. Methods: From December 2015 to March 2018, 11 patients (mean age, 66.3 ± 5.8 years) with irreparable massive RCTs who underwent SCR using an Achilles tendon allograft were enrolled in this study. The range of motion (ROM), visual analog scale (VAS), clinical scores, muscle strength, and acromiohumeral distance (AHD) were measured preoperatively and at 3, 6, and 12 months, and final follow-up postoperatively. Magnetic resonance imaging (MRI) was performed preoperatively and at 6 months postoperatively to assess the global fatty degeneration index and graft failure. Ultrasonography was also conducted preoperatively and at 3, 6, and 12 months, and final follow-up postoperatively to assess graft continuity. Results: The mean follow-up period was 27.6 months (range, 24–32 months). The shoulder ROM at final follow-up increased significantly in forward flexion (p = 0.023), external rotation (p = 0.018), internal rotation (p = 0.016), and abduction (p = 0.011). All patients showed improvement in VAS score (p = 0.005) and clinical scores (p < 0.001) compared with the preoperative state. Pseudoparalysis improved in all patients. The AHD was 3.88 mm (± 1.21 mm) preoperatively, 7.75 mm (± 1.52 mm, p = 0.014) at 6 months postoperatively, and 6.37 mm (± 1.72 mm, p = 0.031) at final follow-up. Graft removal and synovectomy were performed in 1 patient who developed postoperative infections. Radiological failure on follow-up MRI occurred in 2 patients at 6 and 12 months postoperatively, respectively. Conclusions SCR using an Achilles tendon allograft in irreparable massive RCTs achieved functional and clinical improvement. The use of Achilles tendon allograft also has the advantages of short operation time without donor site morbidity, sufficient thickness, and robustness; therefore, this allograft can be a useful graft for SCR.

Background: Treatment remains a challenge in massive and irreparable rotator cuff tears (RCTs), and superior capsular reconstruction (SCR) has become an increasingly popular choice. The objective of this study was to evaluate clinical and radiological outcomes after SCR using an Achilles tendon allograft in irreparable massive RCTs. Methods: From December 2015 to March 2018, 11 patients (mean age, 66.3 ± 5.8 years) with irreparable massive RCTs who underwent SCR using an Achilles tendon allograft were enrolled in this study. The range of motion (ROM), visual analog scale (VAS), clinical scores, muscle strength, and acromiohumeral distance (AHD) were measured preoperatively and at 3, 6, and 12 months, and final follow-up postoperatively. Magnetic resonance imaging (MRI) was performed preoperatively and at 6 months postoperatively to assess the global fatty degeneration index and graft failure. Ultrasonography was also conducted preoperatively and at 3, 6, and 12 months, and final follow-up postoperatively to assess graft continuity. Results: The mean follow-up period was 27.6 months (range, 24–32 months). The shoulder ROM at final follow-up increased significantly in forward flexion (p = 0.023), external rotation (p = 0.018), internal rotation (p = 0.016), and abduction (p = 0.011). All patients showed improvement in VAS score (p = 0.005) and clinical scores (p < 0.001) compared with the preoperative state. Pseudoparalysis improved in all patients. The AHD was 3.88 mm (± 1.21 mm) preoperatively, 7.75 mm (± 1.52 mm, p = 0.014) at 6 months postoperatively, and 6.37 mm (± 1.72 mm, p = 0.031) at final follow-up. Graft removal and synovectomy were performed in 1 patient who developed postoperative infections. Radiological failure on follow-up MRI occurred in 2 patients at 6 and 12 months postoperatively, respectively. Conclusions SCR using an Achilles tendon allograft in irreparable massive RCTs achieved functional and clinical improvement. The use of Achilles tendon allograft also has the advantages of short operation time without donor site morbidity, sufficient thickness, and robustness; therefore, this allograft can be a useful graft for SCR.

The purpose of this study was to investigate trends in dental sedation of Korean children and adolescents.
A retrospective study was conducted on patients under 20 years of age who received dental treatment under sedation using National Health Insurance Service-National Sample Cohort Database from 2002 to 2015. Based on the 1 million standard data, there were 436 cases of dental treatment under sedation in 2002, but 4002 cases in 2015, showing a trend increasing every year. The 3 - 5 year old group accounted for the largest portion (54.2%), while the 6 - 8 year old group increased recently. Nitrous oxide inhalation sedation is the most commonly used, accounting for 45.9% in 2002, but increased to 89.5% in 2015. Combination of nitrous oxide inhalation sedation, chloralhydrate and hydroxyzine was the most common, accounting for 5.7% in 2002, but decreased to 2.9% in 2015.
There is a trend to use the sedation method more safely and in a way that reduces side effects.

Purpose: Effectiveness and safety of clofarabine (one of the treatment mainstays in pediatric patients with relapsed/refractory acute lymphoblastic leukemia [ALL]) was assessed in Korean pediatric patients with ALL to facilitate conditional coverage with evidence development. Materials and Methods: In this multicenter, prospective, observational study, patients receiving clofarabine as mono/combination therapy were followed up every 4-6 weeks for 6 months or until hematopoietic stem cell transplantation (HSCT). Response rates, survival outcomes, and adverse events were assessed. Results: Sixty patients (2-26 years old; 65% B-cell ALL, received prior ≥ 2 regimen, 68.3% refractory to previous regimen) were enrolled and treated with at least one dose of clofarabine; of whom 26 (43.3%) completed 6 months of follow-up after the last dose of clofarabine. Fifty-eight patients (96.7%) received clofarabine combination therapy. Overall remission rate (complete remission [CR] or CR without platelet recovery [CRp]) was 45.0% (27/60; 95% confidence interval [CI], 32.4 to 57.6) and the overall response rate (CR, CRp, or partial remission [PR]) was 46.7% (28/60; 95% CI, 34.0 to 59.3), with 11 (18.3%), 16 (26.7%), and one (1.7%) patients achieving CR, CRp, and PR, respectively. The median time to remission was 5.1 weeks (95% CI, 4.7 to 6.1). Median duration of remission was 16.6 weeks (range, 2.0 to 167.6 weeks). Sixteen patients (26.7%) proceeded to HSCT. There were 24 deaths; 14 due to treatment-emergent adverse events. Conclusion Remission with clofarabine was observed in approximately half of the study patients who had overall expected safety profile; however, there was no favorable long-term survival outcome in this study.