Background: Infantile hemangioma (IH) is a common benign vascular tumor that occurs during infancy. Oral propranolol is used as a first-line treatment. However, standardized guidelines for evaluating treatment efficacy, particularly the appropriate timing and parameters for Doppler ultrasound (US), have not been established. This study reports on the evaluation of therapeutic efficacy of oral propranolol solution in IH patients using Doppler US, and aims to propose the appropriate timing and parameters for using Doppler US based on this experience. Methods: A retrospective analysis was conducted on 120 patients with IH who were treated with oral propranolol solution and maintained for over 6 months from May 2017 to April 2023. Doppler US evaluation of IH was performed at diagnosis, 1-2 and 6-12 months after treatment initiation, and 6 months post-therapy cessation. A complete response (CR) was identified as a reduction in vascularity along with a decrease in longest diameter (LD) or thickness of 50% or more. Recurrence was evaluated based on increased vascularity or size 6 months after treatment discontinuation. Results: Of 120 patients with IH, 82 females and 38 males were analyzed. IH was first detected at a median age of 12 days (range, 1-240 days), and treatment began at 76 days (range, 27-570 days), continuing for an average of 10.4 months (range, 5-24 months). Initial Doppler US measurements showed an LD of 2.65±1.52 cm and a thickness of 0.79±0.55 cm, with prominent vascularity. After 1-2 months of treatment, LD and thickness decreased by 12.9% and 25.9%, respectively. By 6-12 months, reductions reached 37.2% and 53.6%. CR occurred in 77 patients (64.2%) after 6-12 months of treatment. Eleven patients (9.2%) experienced a recurrence. Conclusion Doppler US is a valuable modality for evaluating the characteristics, treatment response, and recurrence of IH treated with oral propranolol.

Background: Infantile hemangioma (IH) is a common benign vascular tumor that occurs during infancy. Oral propranolol is used as a first-line treatment. However, standardized guidelines for evaluating treatment efficacy, particularly the appropriate timing and parameters for Doppler ultrasound (US), have not been established. This study reports on the evaluation of therapeutic efficacy of oral propranolol solution in IH patients using Doppler US, and aims to propose the appropriate timing and parameters for using Doppler US based on this experience. Methods: A retrospective analysis was conducted on 120 patients with IH who were treated with oral propranolol solution and maintained for over 6 months from May 2017 to April 2023. Doppler US evaluation of IH was performed at diagnosis, 1-2 and 6-12 months after treatment initiation, and 6 months post-therapy cessation. A complete response (CR) was identified as a reduction in vascularity along with a decrease in longest diameter (LD) or thickness of 50% or more. Recurrence was evaluated based on increased vascularity or size 6 months after treatment discontinuation. Results: Of 120 patients with IH, 82 females and 38 males were analyzed. IH was first detected at a median age of 12 days (range, 1-240 days), and treatment began at 76 days (range, 27-570 days), continuing for an average of 10.4 months (range, 5-24 months). Initial Doppler US measurements showed an LD of 2.65±1.52 cm and a thickness of 0.79±0.55 cm, with prominent vascularity. After 1-2 months of treatment, LD and thickness decreased by 12.9% and 25.9%, respectively. By 6-12 months, reductions reached 37.2% and 53.6%. CR occurred in 77 patients (64.2%) after 6-12 months of treatment. Eleven patients (9.2%) experienced a recurrence. Conclusion Doppler US is a valuable modality for evaluating the characteristics, treatment response, and recurrence of IH treated with oral propranolol.

Background: Infantile hemangioma (IH) is a common benign vascular tumor that occurs during infancy. Oral propranolol is used as a first-line treatment. However, standardized guidelines for evaluating treatment efficacy, particularly the appropriate timing and parameters for Doppler ultrasound (US), have not been established. This study reports on the evaluation of therapeutic efficacy of oral propranolol solution in IH patients using Doppler US, and aims to propose the appropriate timing and parameters for using Doppler US based on this experience. Methods: A retrospective analysis was conducted on 120 patients with IH who were treated with oral propranolol solution and maintained for over 6 months from May 2017 to April 2023. Doppler US evaluation of IH was performed at diagnosis, 1-2 and 6-12 months after treatment initiation, and 6 months post-therapy cessation. A complete response (CR) was identified as a reduction in vascularity along with a decrease in longest diameter (LD) or thickness of 50% or more. Recurrence was evaluated based on increased vascularity or size 6 months after treatment discontinuation. Results: Of 120 patients with IH, 82 females and 38 males were analyzed. IH was first detected at a median age of 12 days (range, 1-240 days), and treatment began at 76 days (range, 27-570 days), continuing for an average of 10.4 months (range, 5-24 months). Initial Doppler US measurements showed an LD of 2.65±1.52 cm and a thickness of 0.79±0.55 cm, with prominent vascularity. After 1-2 months of treatment, LD and thickness decreased by 12.9% and 25.9%, respectively. By 6-12 months, reductions reached 37.2% and 53.6%. CR occurred in 77 patients (64.2%) after 6-12 months of treatment. Eleven patients (9.2%) experienced a recurrence. Conclusion Doppler US is a valuable modality for evaluating the characteristics, treatment response, and recurrence of IH treated with oral propranolol.

Background: Infantile hemangioma (IH) is a common benign vascular tumor that occurs during infancy. Oral propranolol is used as a first-line treatment. However, standardized guidelines for evaluating treatment efficacy, particularly the appropriate timing and parameters for Doppler ultrasound (US), have not been established. This study reports on the evaluation of therapeutic efficacy of oral propranolol solution in IH patients using Doppler US, and aims to propose the appropriate timing and parameters for using Doppler US based on this experience. Methods: A retrospective analysis was conducted on 120 patients with IH who were treated with oral propranolol solution and maintained for over 6 months from May 2017 to April 2023. Doppler US evaluation of IH was performed at diagnosis, 1-2 and 6-12 months after treatment initiation, and 6 months post-therapy cessation. A complete response (CR) was identified as a reduction in vascularity along with a decrease in longest diameter (LD) or thickness of 50% or more. Recurrence was evaluated based on increased vascularity or size 6 months after treatment discontinuation. Results: Of 120 patients with IH, 82 females and 38 males were analyzed. IH was first detected at a median age of 12 days (range, 1-240 days), and treatment began at 76 days (range, 27-570 days), continuing for an average of 10.4 months (range, 5-24 months). Initial Doppler US measurements showed an LD of 2.65±1.52 cm and a thickness of 0.79±0.55 cm, with prominent vascularity. After 1-2 months of treatment, LD and thickness decreased by 12.9% and 25.9%, respectively. By 6-12 months, reductions reached 37.2% and 53.6%. CR occurred in 77 patients (64.2%) after 6-12 months of treatment. Eleven patients (9.2%) experienced a recurrence. Conclusion Doppler US is a valuable modality for evaluating the characteristics, treatment response, and recurrence of IH treated with oral propranolol.

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.

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.

The Committee of Central Precocious Puberty of Korean Pediatrics and Adolescents of the Korean Society of Pediatric Endocrinology has newly developed evidence-based 2022 clinical practice guidelines for central precocious puberty in Korean children and adolescents. These guidelines provide the grade of recommendations, which includes both the strength of recommendations and the level of evidence. In the absence of sufficient evidence, recommendations are based on expert opinion. These guidelines have been revised and supplement the previous guidelines "Clinical Guidelines for Precocious Puberty 2011," and are drawn from a comprehensive review of the latest domestic and international research and the grade of recommendation appropriate to the domestic situation. This review summarizes the newly revised guidelines into 8 key questions and 27 recommendations and consists of 4 sections: screening, diagnosis, treatment, and long-term outcome of central precocious puberty.

Functional bladder disorders including overactive bladder and interstitial cystitis may induce problems in many other parts of our body such as brain and gut. In fact, diagnosis is often less accurate owing to their complex symptoms. To have correct diagnosis of these diseases, we need to understand the pathophysiology behind overlapped clinical presentation. First, we focused on reviewing literatures that have reported the link between bladder and brain, as the patients with bladder disorders frequently accompanied mood disorders such as depression and anxiety. Second, we reviewed literatures that have described the relationship between bladder and gut. There exist many evidences of patients who suffered from both bladder and intestinal diseases, such as irritable bowel syndrome and inflammatory bowel disease, at the same time. Furthermore, the interaction between brain and gut, well-known as brain-gut axis, might be a key factor that could change the activity of bladder and vice versa. For example, the affective disorders could alter the activity of efferent nerves or autonomic nervous system that modulate the gut itself and its microbiota, which might cause the destruction of homeostasis in bladder eventually. In this way, the communication between bladder and brain-gut axis might affect permeability, inflammation, as well as infectious etiology and dysbiosis in bladder diseases. In this review, we aimed to find an innovative insight of the pathophysiology in the functional bladder disorders, and we could provide a new understanding of the overlapped clinical presentation by elucidating the pathophysiology of functional bladder disorders.

In children, prolonged primary Epstein-Barr virus (EBV) infection or reactivation may lead to the development of T or natural killer cell lymphoproliferative disorders, classified as chronic active EBV infection (CAEBV). CAEBV is a prolonged systemic illness lasting beyond three months, with elevated EBV DNA levels in the serum or affected tissue and a lack of underlying immunodeficiency. Notably, EBV is the most common virus that induces secondary hemophagocytic lymphohistiocytosis (HLH), and Coronavirus disease 2019 (COVID-19) has been reported to trigger HLH. We present the case of a 20-year-old Korean male who was diagnosed with CAEBV and developed HLH after being infected with COVID-19. The patient met 7 out of 8 criteria for HLH. Following treatment with corticosteroids and intravenous immunoglobulin, the patient recovered from HLH without additional chemotherapy. HLH treatment should be tailored to each patient based on clinical presentation and disease severity.Prolonged and regular evaluation for developing HLH or lymphoma is necessary for patients with CAEBV.