Cohort profile: Multicenter Networks for Ideal Outcomes of Rare Pediatric Endocrine and Metabolic Diseases in Korea (OUTSPREAD study)

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.

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Publication Details

Journal
Annals of Pediatric Endocrinology & Metabolism
Published
2024-12-30
DOI
https://doi.org/10.6065/apem.2448272.136
Primary Topic
Sexual Differentiation and Disorders
Type
article
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article

Cohort profile: Multicenter Networks for Ideal Outcomes of Rare Pediatric Endocrine and Metabolic Diseases in Korea (OUTSPREAD study)

Hae Soon Kim, Aram Yang, Yong Hee Hong, Sejin Kim et al.
Annals of Pediatric Endocrinology & Metabolism
Sexual Differentiation and Disorders
article

Cohort profile: Multicenter Networks for Ideal Outcomes of Rare Pediatric Endocrine and Metabolic Diseases in Korea (OUTSPREAD study)

Hae Soon Kim, Aram Yang, Yong Hee Hong, Sejin Kim, Han Hyuk Lim, Hyo‐Kyoung Nam, Jung-Eun Moon, Moon Bae Ahn, Sochung Chung, Chong Kun Cheon, Hae Sang Lee, Eungu Kang, Junghwan Suh, Sung Yoon Cho, Seung Yang, Kye Shik Shim, Shin-Hye Kim, Eun-Gyong Yoo, Heon‐Seok Han, Ji Eun Lee, Jae Hyun Kim, Eun Young Kim, Se Young Kim, Won Kyoung Cho, Jin‐Ho Choi, Jin‐Kyung Kim, Min Sun Kim, Yun Jeong Lee, Seong Hwan Chang, Young Ah Lee, Yena Lee
article en

Abstract

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.

Annals of Pediatric Endocrinology & MetabolismVol. 29(6)
Ulsan College (KR), Ewha Womans University (KR), Seoul National University (KR), Yonsei University (KR), Chungnam National University (KR), Korea University (KR), Soonchunhyang University (KR), Korea University Medical Center (KR), Bundang Jesaeng Hospital (KR), Kyungpook National University Hospital (KR), Asan Medical Center (KR), Samsung Medical Center (KR), Seoul National University Bundang Hospital (KR), Ansan University (KR), University of Ulsan (KR), Daegu Catholic University Medical Center (KR), Konkuk University Medical Center (KR), The Catholic University of Korea St. Vincent's Hospital (KR), Chonnam National University Hospital (KR), Chosun University Hospital (KR), Kangbuk Samsung Hospital (KR), Seoul National University Children's Hospital (KR), Inje University Sanggye Paik Hospital (KR), Keimyung University Dongsan Medical Center (KR), Jeonbuk National University Hospital (KR), CHA University Bundang Medical Center (KR), Hanyang University Seoul Hospital (KR), Chungnam National University Hospital (KR), Pusan National University Yangsan Hospital (KR), Kyung Hee University Hospital at Gangdong (KR), Inha University Hospital (KR), Hallym University Sacred Heart Hospital (KR), Chungbuk National University Hospital (KR), Pusan National University (KR), Keimyung University (KR), Ajou University (KR), Bucheon University (KR), Jeonbuk National University (KR), Sungkyunkwan University (KR), Catholic University of Korea (KR), Daegu University (KR)
Good health and well-being
Openalex Percentile: Top 79%
Sexual Differentiation and Disorders
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