Objective: Bronchopulmonary dysplasia (BPD) is a significant respiratory disorder in premature infants, and its prevalence remains high at 35% to 40% of very low gestational age (<28 weeks gestation). The aim of this study is to analyze the respiratory outcomes of BPD in neonatal intensive care unit as well as respiratory outcomes over a corrected age of 18 to 24 months using 3 different definitions of BPD (National Institutes of Health [NIH] 2001, National Institute of Child Health and Human Development [NICHD] 2018 and Jensen 2019). Methods: We conducted a study on infants under 32 weeks of gestation between 2012 and 2021 at Ajou university hospital. First, we compared the incidence of BPD, mortality, and length of hospital stays. To evaluate long-term respiratory outcomes, the number of re-admissions and prescriptions due to respiratory problems were analyzed. Results: NIH 2001 showed an incidence of 281 (40.0%), NICHD 2018 showed 139 (19.7%), and Jensen 2019 showed 137 (19.5%). In grade III of Jensen 2019, it demonstrated the highest severity with mortality rate of 29.4% and an average length of hospital stay of 42.5 weeks. Also, it was confirmed that the period of use of invasive ventilator was the longest at 87.8±60.3 days. In the analysis of readmission and prescription counts, grade III showed statistically significant higher occurrences in both NICHD 2018 and Jensen 2019 than NIH 2001. Conclusion The latest definitions of BPD have demonstrated to better represent both short-term and long-term respiratory severity in premature infants less than 32 weeks.

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.

Objective: Bronchopulmonary dysplasia (BPD) is a significant respiratory disorder in premature infants, and its prevalence remains high at 35% to 40% of very low gestational age (<28 weeks gestation). The aim of this study is to analyze the respiratory outcomes of BPD in neonatal intensive care unit as well as respiratory outcomes over a corrected age of 18 to 24 months using 3 different definitions of BPD (National Institutes of Health [NIH] 2001, National Institute of Child Health and Human Development [NICHD] 2018 and Jensen 2019). Methods: We conducted a study on infants under 32 weeks of gestation between 2012 and 2021 at Ajou university hospital. First, we compared the incidence of BPD, mortality, and length of hospital stays. To evaluate long-term respiratory outcomes, the number of re-admissions and prescriptions due to respiratory problems were analyzed. Results: NIH 2001 showed an incidence of 281 (40.0%), NICHD 2018 showed 139 (19.7%), and Jensen 2019 showed 137 (19.5%). In grade III of Jensen 2019, it demonstrated the highest severity with mortality rate of 29.4% and an average length of hospital stay of 42.5 weeks. Also, it was confirmed that the period of use of invasive ventilator was the longest at 87.8±60.3 days. In the analysis of readmission and prescription counts, grade III showed statistically significant higher occurrences in both NICHD 2018 and Jensen 2019 than NIH 2001. Conclusion The latest definitions of BPD have demonstrated to better represent both short-term and long-term respiratory severity in premature infants less than 32 weeks.

Purpose: Nonambulatory pediatric patients may have low bone mineral density (BMD) and increased risk of pathologic fractures. Though bisphosphonate therapy is the mainstream medical intervention in these children, clinical data regarding this treatment are limited. Therefore, this study aimed to evaluate the effectiveness and safety of bisphosphonate therapy in such children. Methods: We conducted a retrospective study of 21 nonambulatory children (Gross Motor Function Classification System level V) with BMD z-score ≤ -2.0 who were treated with intravenous pamidronate for at least 1 year. These patients received pamidronate every 4 months at a dose of 1.0 to 3.0 mg/kg for each cycle and had regular follow-ups for at least 1 year. The main outcome measures were changes in BMD, risk rate of fracture, biochemical data, and adverse events. Results: The average duration of pamidronate treatment was 2.0±0.9 years, and the mean cumulative dose of pamidronate according to body weight was 7.7±2.5 mg/kg/yr. After treatment, the mean lumbar spine bone mineral content, BMD, and height-for-age-z-score-adjusted BMD z-score (BMDhazZ) significantly improved. The relative risk of fracture after treatment was 0.21 (p=0.0032), suggesting that pamidronate treatment reduced fracture incidence significantly. The increase in the average dose per body weight in each cycle significantly increased the changes in BMDhazZ. Conclusion Pamidronate treatment improved the bone health of nonambulatory children with low bone density without any significant adverse events. Independent of cumulative dosage and duration of treatment, the effectiveness of pamidronate increased significantly with an increase in the average dose per body weight in subsequent cycles.

Objective: Bronchopulmonary dysplasia (BPD) is a significant respiratory disorder in premature infants, and its prevalence remains high at 35% to 40% of very low gestational age (<28 weeks gestation). The aim of this study is to analyze the respiratory outcomes of BPD in neonatal intensive care unit as well as respiratory outcomes over a corrected age of 18 to 24 months using 3 different definitions of BPD (National Institutes of Health [NIH] 2001, National Institute of Child Health and Human Development [NICHD] 2018 and Jensen 2019). Methods: We conducted a study on infants under 32 weeks of gestation between 2012 and 2021 at Ajou university hospital. First, we compared the incidence of BPD, mortality, and length of hospital stays. To evaluate long-term respiratory outcomes, the number of re-admissions and prescriptions due to respiratory problems were analyzed. Results: NIH 2001 showed an incidence of 281 (40.0%), NICHD 2018 showed 139 (19.7%), and Jensen 2019 showed 137 (19.5%). In grade III of Jensen 2019, it demonstrated the highest severity with mortality rate of 29.4% and an average length of hospital stay of 42.5 weeks. Also, it was confirmed that the period of use of invasive ventilator was the longest at 87.8±60.3 days. In the analysis of readmission and prescription counts, grade III showed statistically significant higher occurrences in both NICHD 2018 and Jensen 2019 than NIH 2001. Conclusion The latest definitions of BPD have demonstrated to better represent both short-term and long-term respiratory severity in premature infants less than 32 weeks.