Clinical Pediatric Endocrinology最新文献_第2页

Multifaceted delineation of atrophic thyroiditis among pediatric population: An extensive literature survey. 萎缩性甲状腺炎在儿科人群中的多面描述：一项广泛的文献调查。

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Clinical Pediatric Endocrinology Pub Date : 2025-01-01 Epub Date: 2024-10-27 DOI: 10.1297/cpe.2024-0040

Sakura Motegi, Masanori Adachi, Keiko Nagahara, Tatsuyuki Ishida, Ayako Ochi, Katsumi Mizuno

{"title":"Multifaceted delineation of atrophic thyroiditis among pediatric population: An extensive literature survey.","authors":"Sakura Motegi, Masanori Adachi, Keiko Nagahara, Tatsuyuki Ishida, Ayako Ochi, Katsumi Mizuno","doi":"10.1297/cpe.2024-0040","DOIUrl":"https://doi.org/10.1297/cpe.2024-0040","url":null,"abstract":"Autoimmune hypothyroidism is categorized into Hashimoto thyroiditis (HT) and atrophic thyroiditis (AT). Although a consensus exists among Japanese endocrinologists that pediatric AT is associated with severe hypothyroidism, the question remains whether AT and HT are separate conditions. To investigate the clinical characteristics of pediatric AT, we conducted a comprehensive literature review using PubMed and ICHUSHI, a local database. We identified 54 patients (43 females), diagnosed ≤ 18 yr of age, based on 19 English- and 28 Japanese-language publications; 45 patients were Japanese. The onset of the disease typically occurs before puberty. The patients exhibited severe hypothyroidism, with median TSH level of 518.8 μIU/mL (interquartile range [IQR]: 333.0-808.6) and median Free T4 level of 0.16 ng/dL (IQR: 0.08-0.40). Common findings included a low height SD score (median -2.54 SD), low height-velocity SD score (median -3.60 SD), body mass index +1 SD (40%), delayed bone age (64%), pericardial effusion (70%), and an enlarged pituitary gland (78%). Abnormal blood test results were frequently observed, including Hb (82%), CPK (83%), AST (94%), ALT (82%), and total cholesterol (95%). Ultrasound 3D volumetry, conducted for 14 thyroid lobes, revealed 13 lobes below the 25th percentile. In conclusion, our study underscores the clinical presentation of pediatric AT, marked by severe hypothyroidism and a small thyroid gland. Nevertheless, the paucity of data on non-Japanese patients suggests a need for further research to determine if AT and HT are indeed distinct entities.","PeriodicalId":10678,"journal":{"name":"Clinical Pediatric Endocrinology","volume":"34 1","pages":"27-35"},"PeriodicalIF":1.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11701016/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142945944","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Wieacker-Wolff syndrome with hyperinsulinemic hypoglycemia successfully treated using diazoxide: A case report. 用二氮氧化合物成功治疗高胰岛素性低血糖的威克-沃尔夫综合征1例。

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Clinical Pediatric Endocrinology Pub Date : 2025-01-01 Epub Date: 2024-10-18 DOI: 10.1297/cpe.2024-0056

Satoko Kobayashi, Ayami Sato, Yumiko Chiba, Natsuho Adachi, Yu Kakimoto, Hisato Suzuki, Mamiko Yamada, Kenjiro Kosaki, Hiroyuki Tanaka

{"title":"Wieacker-Wolff syndrome with hyperinsulinemic hypoglycemia successfully treated using diazoxide: A case report.","authors":"Satoko Kobayashi, Ayami Sato, Yumiko Chiba, Natsuho Adachi, Yu Kakimoto, Hisato Suzuki, Mamiko Yamada, Kenjiro Kosaki, Hiroyuki Tanaka","doi":"10.1297/cpe.2024-0056","DOIUrl":"https://doi.org/10.1297/cpe.2024-0056","url":null,"abstract":"Wieacker-Wolff syndrome (WRWF) is an X-linked genetic disorder characterized by neuromusculoskeletal abnormalities caused by loss-of-function variants of the ZC4H2 gene. Here, we report the case of a male infant with WRWF manifesting as multiple joint contractures and congenital anomalies at birth. He underwent gastrostomy to treat the gastroesophageal reflux disease, which caused mixed apnea and transient bradycardia. The patient subsequently developed hyperinsulinemic hypoglycemia (HH) and was diagnosed with dumping syndrome. Although he underwent multiple treatments, including alpha-glucosidase inhibitors (α-GI) administration, he continued to exhibit HH with seizures and loss of consciousness. Whole-exome sequencing revealed a novel missense variant of ZC4H2 [NM_018684.4: c.557T>G, p.(Met186Arg)] at Xq11.2 in both the patient and his mother. Based on these results and clinical symptoms, the patient was diagnosed with WRWF. Although WRWF is not considered a major cause of HH, we regarded it as a related complication based on previous reports. Diazoxide treatment was initiated, and the hypoglycemic attacks resolved almost entirely without any notable side effects after 18 mo. To the best of our knowledge, this is the first report of WRWF-associated HH treated with low-dose diazoxide and α-GI. Therefore, diazoxide is recommended for the treatment of WRWF-associated HH.","PeriodicalId":10678,"journal":{"name":"Clinical Pediatric Endocrinology","volume":"34 1","pages":"70-76"},"PeriodicalIF":1.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11701018/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142945231","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Idiopathic infantile hypercalcemia with a CYP24A1 variant triggered by vitamin D supplementation in fortified milk: A case report. 在强化牛奶中补充维生素D引发的具有CYP24A1变异的特发性婴儿高钙血症：一例报告。

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Clinical Pediatric Endocrinology Pub Date : 2025-01-01 Epub Date: 2024-09-09 DOI: 10.1297/cpe.2024-0049

Sota Iwafuchi, Nao Uchida, Naoya Saijo, Chisumi Sogi, Miki Kamimura, Jun Takayama, Gen Tamiya, Atsuo Kikuchi, Junko Kanno

{"title":"Idiopathic infantile hypercalcemia with a CYP24A1 variant triggered by vitamin D supplementation in fortified milk: A case report.","authors":"Sota Iwafuchi, Nao Uchida, Naoya Saijo, Chisumi Sogi, Miki Kamimura, Jun Takayama, Gen Tamiya, Atsuo Kikuchi, Junko Kanno","doi":"10.1297/cpe.2024-0049","DOIUrl":"https://doi.org/10.1297/cpe.2024-0049","url":null,"abstract":"Idiopathic infantile hypercalcemia (IIH) is characterized by hypercalcemia, nephrocalcinosis, vomiting, dehydration, and failure to thrive. It is caused by the presence of biallelic loss-of-function variants in the CYP24A1 locus. Although hypercalcemia has been linked to the consumption of vitamin D-fortified milk, no reports have documented its role in triggering IIH in patients with CYP24A1 variants. Herein, we describe a case of IIH triggered by vitamin D-fortified milk consumption in a 9-mo-old male patient carrying a CYP24A1 variant. After BCG vaccination, the patient developed a facial rash, became anorexic, appeared to be in a bad mood, and began consuming vitamin D-fortified milk instead of baby food. Blood tests showed a marked hypercalcemia (18.5 mg/dL), high 1,25-(OH)2D (98.7 pg/dL) levels, and low parathyroid hormone (PTH) (< 4.0 pg/dL) and PTHrP (< 1.0 pg/dL) levels. The calcium levels were successfully normalized after treatment with saline loading, furosemide, pamidronate, and a low-calcium milk diet. After discharge, blood calcium levels remained normal with no recurrence of symptomatic hypercalcemia, but circulating PTH levels were persistently suppressed. Renal ultrasonography at 8 yr of age revealed high medullary echogenicity and diffuse echogenic foci in both kidneys. Trio-based whole-genome sequencing identified the following biallelic pathogenic variants c.[464G>A];[1324C>T], p.[Trp155Ter];[Gln442Ter], in the CYP24A1 (NM_000782.5) locus. Unexplained hypercalcemia in infants should raise suspicions of abnormal vitamin D metabolism and CYP24A1 locus genotypic analysis can be informative in this regard.","PeriodicalId":10678,"journal":{"name":"Clinical Pediatric Endocrinology","volume":"34 1","pages":"60-65"},"PeriodicalIF":1.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11701012/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142945942","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Mortality rate and standardized mortality ratio of childhood-onset type 1 diabetes according to incidentally detected non-acute-onset or acute-onset subtype, sex, and onset age: A cohort study. 根据偶然发现的非急性发作或急性发作亚型、性别和发病年龄，儿童期发病1型糖尿病的死亡率和标准化死亡率：一项队列研究

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Clinical Pediatric Endocrinology Pub Date : 2025-01-01 Epub Date: 2024-09-16 DOI: 10.1297/cpe.2024-0050

Hiroshi Yokomichi, Mie Mochizuki, Shigeru Suzuki, Yoshiya Ito, Tomoyuki Hotsubo, Nobuo Matsuura

引用次数: 0

Understanding the burden faced by families of children living with Type 1 diabetes mellitus in Indonesia: A multidimensional study on the financial, social, and psychosocial aspects. 了解印度尼西亚1型糖尿病儿童家庭所面临的负担：一项关于经济、社会和心理社会方面的多维研究

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Clinical Pediatric Endocrinology Pub Date : 2025-01-01 Epub Date: 2024-12-02 DOI: 10.1297/cpe.2024-0071

Muhammad Faizi, Zi T La, Helena A Puteri, Vahira Waladhiyaputri, Gassani Amalia, Harjoedi A Tjahjono, Nur Rochmah, Ghaisani Fadiana, Yuni Hisbiyah, Rayi K Perwitasari, Fadilah Mutaqin, Khairunnisa, Irfan A Salim, Achmad Y Heryana, Aman B Pulungan

{"title":"Understanding the burden faced by families of children living with Type 1 diabetes mellitus in Indonesia: A multidimensional study on the financial, social, and psychosocial aspects.","authors":"Muhammad Faizi, Zi T La, Helena A Puteri, Vahira Waladhiyaputri, Gassani Amalia, Harjoedi A Tjahjono, Nur Rochmah, Ghaisani Fadiana, Yuni Hisbiyah, Rayi K Perwitasari, Fadilah Mutaqin, Khairunnisa, Irfan A Salim, Achmad Y Heryana, Aman B Pulungan","doi":"10.1297/cpe.2024-0071","DOIUrl":"https://doi.org/10.1297/cpe.2024-0071","url":null,"abstract":"Type 1 diabetes mellitus (T1DM) is a lifelong disorder that affects all aspects of the lives of children and their families. A Health Needs Assessment (HNA) survey was conducted at two diabetes camps in Batu, East Java, and Parung, West Java, to evaluate the challenges and burdens faced by families of children living with T1DM in Indonesia. A total of forty-one respondents, comprising parents/caregivers, participated in the HNA. Most respondents had to pay for diabetes-related expenses, such as insulin (31.7%), self-monitoring blood glucose (31.7%), needles and syringes (63.4%), travel expenses (97.6%), and additional laboratory examinations (24.4%). The majority of the children in this study attended school (97.6%) and most liked going to school (95%). Diabetes camps were reported to be very helpful (95.1%) for gaining more knowledge and social support within the community. A family-centered approach focusing on community support and individualized solutions is required to strengthen support, share resources, increase knowledge, and ultimately improve the quality of life of children and families living with T1DM.","PeriodicalId":10678,"journal":{"name":"Clinical Pediatric Endocrinology","volume":"34 1","pages":"45-53"},"PeriodicalIF":1.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11701017/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142945247","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Newborn screening for congenital adrenal hyperplasia: Utility of liquid chromatography with tandem mass spectrometry as a secondary test. 新生儿先天性肾上腺增生筛检：液相色谱串联质谱作为二次测试的效用。

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Clinical Pediatric Endocrinology Pub Date : 2025-01-01 Epub Date: 2024-11-25 DOI: 10.1297/cpe.2024-0069

Toshihiro Tajima

{"title":"Newborn screening for congenital adrenal hyperplasia: Utility of liquid chromatography with tandem mass spectrometry as a secondary test.","authors":"Toshihiro Tajima","doi":"10.1297/cpe.2024-0069","DOIUrl":"https://doi.org/10.1297/cpe.2024-0069","url":null,"abstract":"In Japan, newborn screening (NBS) for congenital adrenal hyperplasia (CAH) began in 1989. NBS is useful for early diagnosis and preventing gender misidentification, however, it has a higher false positive rate for CAH compared to other diseases detected by neonatal screening. Recently, it has become clear that using liquid chromatography with tandem mass spectrometry (LC-MS/MS) for second-tier testing reduces false positive rates and repeat blood sampling. LC-MS/MS commonly measures cortisol (F), androstenedione (A4), 11-deoxycortsiol (11DOF), 21-deoxycortisol (21DOF), and 17-hydroxyprogesterone (17OHP) levels. The ratios for (21DOF+17OHP)/F and (17OHP+A4)/F have been used to establish cut-off values for the second-tier test. In Japan, the recall rate is reduced using the 11DOF/17OHP ratio as well as the ratios for (21DOF+17OHP)/F and (17OHP+A4)/F for the second-tier test. Currently, second-tier testing using LC-MS/MS for CAH neonatal screening is unfeasible in all regions of Japan due to equipment costs, however, it will hopefully be available nationwide in the future.","PeriodicalId":10678,"journal":{"name":"Clinical Pediatric Endocrinology","volume":"34 1","pages":"13-18"},"PeriodicalIF":1.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11701021/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142945945","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Factors predictive of serum cortisol in pediatric patients with acute physiological stress: a cohort study. 急性生理应激患儿血清皮质醇的预测因素：一项队列研究。

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Clinical Pediatric Endocrinology Pub Date : 2025-01-01 Epub Date: 2024-11-11 DOI: 10.1297/cpe.2024-0048

Shogo Akahoshi, Marie Mitani-Konno, Taku Murakami, Hiroshi Hayashi, Yoshihiko Morikawa, Yusuke Hagiwara, Osamu Saito, Hiroshi Hataya, Yukihiro Hasegawa

{"title":"Factors predictive of serum cortisol in pediatric patients with acute physiological stress: a cohort study.","authors":"Shogo Akahoshi, Marie Mitani-Konno, Taku Murakami, Hiroshi Hayashi, Yoshihiko Morikawa, Yusuke Hagiwara, Osamu Saito, Hiroshi Hataya, Yukihiro Hasegawa","doi":"10.1297/cpe.2024-0048","DOIUrl":"https://doi.org/10.1297/cpe.2024-0048","url":null,"abstract":"Measuring cortisol is crucial for assessing adrenal function in patients under stress; however, its value can fluctuate owing to various clinical factors. This study aimed to identify predictors of cortisol levels in pediatric patients with acute physiological stress. Children who were urgently admitted to the general ward or pediatric intensive care unit for acute illness or postoperative care were enrolled, while those with suspected adrenal function abnormalities or on current steroid therapy were excluded. Cortisol was measured in serum samples collected within 72 h of registration and its association with clinical factors was explored. A total of 397 samples from 217 patients were analyzed between August and November 2021 showing a median cortisol level of 375 nmol/L (interquartile range: 190-646 nmol/L). Multiple regression analysis with a mixed-effects model identified the following predictors of higher cortisol levels: heart rate z-score (+43.8 nmol/L/point), body temperature (+42.3 nmol/L/°C), Pediatric Early Warning System score (+44.3 nmol/L/point), age 3-6 yr (+68.8 nmol/L vs. < 1 yr), elapsed time < 4 h (+130.9 nmol/L vs. 4-12 h), and sampling time 6-10 AM (+96.4 nmol/L vs. 10 AM-2 PM). These variables independently predicted cortisol levels in pediatric patients during acute physiological stress.","PeriodicalId":10678,"journal":{"name":"Clinical Pediatric Endocrinology","volume":"34 1","pages":"36-44"},"PeriodicalIF":1.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11701019/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142945940","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Solitary median maxillary central incisor syndrome caused by 22q11.2 microdeletion. 22q11.2微缺失引起的上颌中切牙孤立综合征。

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Clinical Pediatric Endocrinology Pub Date : 2025-01-01 Epub Date: 2024-09-12 DOI: 10.1297/cpe.2024-0024

Hirohito Shima, Akinobu Miura, Sayaka Kawashima, Ikumi Umeki, Chisumi Sogi, Dai Suzuki, Yusuke Takezawa, Ryo Sato, Natsuko Arai-Ichinoi, Miki Kamimura, Ikuma Fujiwara, Mika Adachi, Aya Yamada, Hiroshi Kawame, Atsuo Kikuchi, Junko Kanno

{"title":"Solitary median maxillary central incisor syndrome caused by 22q11.2 microdeletion.","authors":"Hirohito Shima, Akinobu Miura, Sayaka Kawashima, Ikumi Umeki, Chisumi Sogi, Dai Suzuki, Yusuke Takezawa, Ryo Sato, Natsuko Arai-Ichinoi, Miki Kamimura, Ikuma Fujiwara, Mika Adachi, Aya Yamada, Hiroshi Kawame, Atsuo Kikuchi, Junko Kanno","doi":"10.1297/cpe.2024-0024","DOIUrl":"https://doi.org/10.1297/cpe.2024-0024","url":null,"abstract":"Solitary median maxillary central incisor (SMMCI) syndrome, the mildest form of the holoprosencephaly spectrum, is a rare anomaly characterized by the presence of a single midline central incisor in both the deciduous and permanent dentitions. Affected individuals can present with additional midline defects beyond dental findings. The 22q11.2 deletion syndrome (22q11.2 DS) arises from heterozygous microdeletions on chromosome 22q11.2, with breakpoints frequently located in eight clusters of low-copy repeats (LCR22A-H). Herein, we report an atypical case of 22q11.2 microdeletion in a male patient with SMMCI and additional features including hypothyroidism, ventricular septal defect, and several facial anomalies. The telomeric breakpoint was located in a segmental duplication 0.5 Mb distal to LCR22D, whereas the centromeric breakpoint was within LCR22C. Both segmental duplications shared a high level of sequence identity (97.2%), indicating the possibility of non-allelic homologous recombination (NAHR). This report supports the critical role of NAHR in the formation of rearrangements between regions other than LCR blocks and establishes a clinical association between 22q11.2 microdeletion and SMMCI.","PeriodicalId":10678,"journal":{"name":"Clinical Pediatric Endocrinology","volume":"34 1","pages":"54-59"},"PeriodicalIF":1.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11701020/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142945946","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Complex equilibrium involving aldosterone underlies the pathophysiology of renovascular hypertension. 涉及醛固酮的复杂平衡是肾血管性高血压病理生理的基础。

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Clinical Pediatric Endocrinology Pub Date : 2025-01-01 Epub Date: 2024-09-25 DOI: 10.1297/cpe.2024-0041

Masanori Adachi, Takanari Fujii, Ayako Ochi, Tatsuyuki Ishida, Sakura Motegi, Keiko Nagahara, Katsumi Mizuno

{"title":"Complex equilibrium involving aldosterone underlies the pathophysiology of renovascular hypertension.","authors":"Masanori Adachi, Takanari Fujii, Ayako Ochi, Tatsuyuki Ishida, Sakura Motegi, Keiko Nagahara, Katsumi Mizuno","doi":"10.1297/cpe.2024-0041","DOIUrl":"https://doi.org/10.1297/cpe.2024-0041","url":null,"abstract":"The mechanisms underlying the maintenance of hypertension in renovascular hypertension (RVH) are not well understood. To test the current concept of RVH pathophysiology, circulating aldosterone levels in clinical cases were investigated through a literature survey of pediatric cases. Fifty-four patients with documented aldosterone levels were identified. Of these, 42 patients (78%) were assigned to the high renin (HR) group and the rest to the low-normal renin (LR) group. Patients in the HR group were more likely to have unilateral lesions (35/42) than those in the LR group (6/12). In the LR group (corresponding to volume-dependent RVH), 50% (6/12) of patients had elevated aldosterone levels, indicating that the equilibrium between renin and aldosterone shifted towards aldosterone dominance. In the HR group (corresponding to renin-dependent RVH), aldosterone levels were much higher, with 76% (32/42) of patients exceeding the reference range and 14 patients developing hypokalemia. These results are consistent with the notion that pressure natriuresis allows continuous aldosterone action in renin-dependent RVH. In conclusion, the aldosterone status observed in the clinical cases is in agreement with the current understanding of the pathophysiology of RVH, in which a complex equilibrium state involving renin, angiotensin-II, pressure natriuresis, and aldosterone exists.","PeriodicalId":10678,"journal":{"name":"Clinical Pediatric Endocrinology","volume":"34 1","pages":"19-26"},"PeriodicalIF":1.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11701014/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142945938","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Current understanding and perspectives on growth and long-acting GH therapy in Japan. 日本生长发育和长效生长激素治疗的现状和观点。

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Clinical Pediatric Endocrinology Pub Date : 2025-01-01 Epub Date: 2024-10-26 DOI: 10.1297/cpe.2024-0058

Yukihiro Hasegawa, Kento Ikegawa, Marie Mitani-Konno, Daisuke Ariyasu, Naoko Amano

{"title":"Current understanding and perspectives on growth and long-acting GH therapy in Japan.","authors":"Yukihiro Hasegawa, Kento Ikegawa, Marie Mitani-Konno, Daisuke Ariyasu, Naoko Amano","doi":"10.1297/cpe.2024-0058","DOIUrl":"https://doi.org/10.1297/cpe.2024-0058","url":null,"abstract":"From the perspective of clinical pediatric endocrinology, progress in molecular biology over the past few decades has improved our understanding of growth physiology. This progress was particularly conspicuous in the early era of Sanger-based sequencing and has continued into the current era of next-generation sequencing. The first half of this review summarizes the current understanding about growth. The latter half discusses the development of long-acting GH (LAGH), an important, recent topic in pediatric endocrinology, with a focus on the Japanese market. LAGH is administered weekly over several years in some developed countries as treatment for GH deficiency (GHD). In Japan, somatrogon and somapacitan are available, and lonapegsomatropin has completed a clinical trial. As per recent meta-analyses, these three products were not inferior to conventional, daily GH therapy for short-term growth in pediatric GHD. Although LAGH is promising, some concerns remain. Well-designed clinical research is needed to expand the indications for LAGH to other conditions, such as idiopathic short stature. Finally, future research on orally administered agents may open new avenues for the treatment of short stature.","PeriodicalId":10678,"journal":{"name":"Clinical Pediatric Endocrinology","volume":"34 1","pages":"1-12"},"PeriodicalIF":1.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11701010/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142945939","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0