Pediatric Investigation最新文献

{"title":"Long-acting growth hormone in 2022.","authors":"Margaret Steiner,&nbsp;Jacklyn Frank,&nbsp;Paul Saenger","doi":"10.1002/ped4.12358","DOIUrl":"10.1002/ped4.12358","url":null,"abstract":"<p><p>After the isolation of pituitary growth hormone (GH) in 1957, this form of GH, always in limited supply, was the only drug available for the treatment of GH deficiency. In 1985, recombinant GH became available, and the modalities of GH therapies changed dramatically as the supply was unlimited. New indications for GH in pediatrics and adult medicine were developed. Treatment was daily. Now in 2021 long-acting GH (LAGH) became available the world over making GH therapy more patient-friendly and even showing slightly greater efficacy than daily GH therapy. We are now entering a new era of LAGH therapy for pediatric and adult use with new formulations of GH, which will predictably be the preferred form of GH therapy for years to come increasing adherence to GH therapy and possibly even efficacy, that is, better growth rate. The continued availability of new safety data will further solidify the use of LAGH in clinical medicine.</p>","PeriodicalId":19992,"journal":{"name":"Pediatric Investigation","volume":null,"pages":null},"PeriodicalIF":2.2,"publicationDate":"2023-01-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/42/41/PED4-7-36.PMC10030690.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9193162","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Phenomapping approach to interpreting coronary dimensions in febrile children.","authors":"Haoxun Tang, Xin Guo, Xiaolu Nie, Lin Zheng, Gang Liu, Wilfred Hing-Sang Wong, Yiu-Fai Cheung","doi":"10.1002/ped4.12361","DOIUrl":"10.1002/ped4.12361","url":null,"abstract":"<p><strong>Importance: </strong>Coronary artery dilation may occur in febrile children with and without Kawasaki disease (KD).</p><p><strong>Objective: </strong>We explored the application of unsupervised learning algorithms in the detection of novel patterns of coronary artery phenotypes in febrile children with and without KD.</p><p><strong>Methods: </strong>A total of 239 febrile children (59 non-KD and 180 KD patients), were recruited. Unsupervised hierarchical clustering analysis of phenotypic data including age, hemoglobin, white cell count, platelet count, C-reactive protein, erythrocyte sedimentation rate, albumin, alanine aminotransferase, aspartate aminotransferase, and coronary artery <i>z</i> scores were performed.</p><p><strong>Results: </strong>Using a cutoff <i>z</i> score of 2.5, the specificity was 98.3% and the sensitivity was 22.1% for differentiating non-KD from KD patients. Clustering analysis identified three phenogroups that differed in a clinical, laboratory, and echocardiographic parameters. Compared with phenogroup I, phenogroup III had the highest prevalence of KD (91%), worse inflammatory markers, more deranged liver function, higher coronary artery <i>z</i> scores, and lower hematocrit and albumin levels. Abnormal blood parameters in febrile children with <i>z</i> scores of coronary artery segments <0.5 and 0.5-1.5 was associated with increased risks of having KD to 8.7 (<i>P</i> = 0.003) and 4.4 (<i>P</i> = 0.002), respectively.</p><p><strong>Interpretation: </strong>Phenomapping of febrile children with and without KD identified useful laboratory parameters that aid the diagnosis of KD in febrile children with relatively normal-sized coronary arteries.</p>","PeriodicalId":19992,"journal":{"name":"Pediatric Investigation","volume":null,"pages":null},"PeriodicalIF":1.9,"publicationDate":"2022-12-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/51/3b/PED4-6-233.PMC9789931.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10459472","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Transcatheter pulmonary valve replacement in congenital heart diseases.","authors":"Adolphus Kai-Tung Chau","doi":"10.1002/ped4.12359","DOIUrl":"10.1002/ped4.12359","url":null,"abstract":"<p><p>Surgical repair of a variety of congenital heart diseases involves repair of the right ventricular outflow tract (RVOT) with valved or non-valved conduit to connect the right ventricle (RV) to the pulmonary artery (PA) or just patch enlargement of the native RVOT. With time, this RV-PA conduit will degenerate with deterioration of function, either causing pulmonary stenosis or pulmonary regurgitation. This RVOT dysfunction may result in RV dilation, RV dysfunction, and eventual RV failure and arrhythmias. Multiple surgical pulmonary valve replacement (PVR) is often required throughout the patient's lifetime. Patients are subjected to increased risks with each additional cardiac operation. Transcatheter PVR (TPVR) has been developed over the past two decades as a valuable non-surgical alternative to restore the RVOT and RV function, and hence reduce patients' lifetime risks related to surgery. This article will discuss the long-term results of TPVR which are demonstrated to be comparable to surgical results and the latest development of large pulmonary valves which will allow TPVR to be performed on native or larger RVOT.</p>","PeriodicalId":19992,"journal":{"name":"Pediatric Investigation","volume":null,"pages":null},"PeriodicalIF":1.9,"publicationDate":"2022-12-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/b3/10/PED4-6-280.PMC9789934.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10821627","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Optical coherence tomography of the pulmonary arteries in children with congenital heart diseases: A systematic review.","authors":"Ling Sun, Qiuping Jiang, Yumei Xie, Shushui Wang, Zhiwei Zhang","doi":"10.1002/ped4.12353","DOIUrl":"10.1002/ped4.12353","url":null,"abstract":"<p><strong>Importance: </strong>Optical coherence tomography (OCT) is a high-resolution intravascular imaging tool and has shown promise for providing real-time quantitative and qualitative descriptions of pulmonary vascular structures <i>in vivo</i> in adult pulmonary hypertension (PH), while not popular in pediatric patients with congenital heart diseases (CHD).</p><p><strong>Objective: </strong>The aim of this review is to summarize all the available evidence on the use of OCT for imaging pulmonary vascular remodeling in pediatric patients.</p><p><strong>Methods: </strong>We conducted the systematic literature resources (Cochran Library database, Medline via PubMed, EMBASE, and Web of Knowledge) from January 2010 to December 2021 and the search terms were \"PH\", \"child\", \"children\", \"pediatric\", \"OCT\", \"CHD\", \"pulmonary vessels\", \"pulmonary artery wall\". Studies in which OCT was used to image the pulmonary vessels in pediatric patients with CHD were considered for inclusion.</p><p><strong>Results: </strong>Five studies met the inclusion criteria. These five papers discussed the study of OCT in the pulmonary vasculature of different types of CHD, including common simple CHD, complex cyanotic CHD, and Williams-Beuren syndrome. In biventricular anatomy, pulmonary vascular remodeling was primarily reflected by pulmonary intima thickening from two-dimensional OCT. In single-ventricle anatomy, due to the state of hypoxia, the morphology of pulmonary vessels was indirectly reflected by the number and shape of nourishing vessels from three-dimensional OCT.</p><p><strong>Interpretation: </strong>OCT may be an adequate imaging procedure for the demonstration of pulmonary vascular structures and provide additional information in pediatric patients.</p>","PeriodicalId":19992,"journal":{"name":"Pediatric Investigation","volume":null,"pages":null},"PeriodicalIF":1.9,"publicationDate":"2022-11-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/64/4a/PED4-6-264.PMC9789933.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10459473","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Bath-plug technique for the endoscopic management of cerebrospinal fluid leaks in children.","authors":"Xiaojian Yang, Lixing Tang, Pengpeng Wang, Ming Ge, Wei Zhang, Xiao Xiao, Yang Han, Wentong Ge","doi":"10.1002/ped4.12354","DOIUrl":"10.1002/ped4.12354","url":null,"abstract":"<p><strong>Importance: </strong>The safety and efficacy of the bath-plug technique for the closure of cerebrospinal fluid (CSF) leaks in children remain unknown.</p><p><strong>Objective: </strong>We undertook this study to ascertain whether the bath-plug technique was safe and effective for the repair of CSF leaks.</p><p><strong>Methods: </strong>We retrospectively reviewed patients who underwent endoscopic repair of CSF leaks with the fat graft as a plug-in at Beijing Children's Hospital from March 2016 to May 2020. Demographic data, medical history, defect sites and sizes, interventions, and clinical outcomes were analyzed. One representative clinical case was additionally selected to highlight the procedure and the healing process.</p><p><strong>Results: </strong>A total of 18 pediatric patients were included in this study. The group was composed of 11 boys and seven girls, aged from 5 to 123 months. The etiologies included congenital CSF leaks (<i>n</i> = 9) and head trauma (<i>n</i> = 9). Among all patients, 12 fistulas (66.7%) were located at the cribriform plate area, two (11.1%) at the roof of the ethmoid sinuses, two (11.1%) in the sphenoid sinus, and two (11.1%) at the frontal sinus. The maximum diameters of fistulas ranged from 5 to 20 mm, with a median value of 8 mm. Encephaloceles were identified in 14 (77.8%) patients. No hydrocephalus was recognized. All CSF leaks were successfully repaired with a bath-plug technique. Follow-up ranged from 50 to 70 months. No surgical complications were encountered in any patient.</p><p><strong>Interpretation: </strong>Bath-plug technique is safe and reliable for the endoscopic management of CSF leaks in children. Meticulous peri-operative preparations are important for pediatric patients.</p>","PeriodicalId":19992,"journal":{"name":"Pediatric Investigation","volume":null,"pages":null},"PeriodicalIF":1.9,"publicationDate":"2022-11-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/64/df/PED4-7-23.PMC10030683.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9188397","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Extracardiovascular injury complications in Kawasaki disease.","authors":"Jie Liu, Yuqin Huang, Suyuan Qin, Danyan Su, Bingbing Ye, Yusheng Pang","doi":"10.1002/ped4.12355","DOIUrl":"10.1002/ped4.12355","url":null,"abstract":"<p><strong>Importance: </strong>Patients with Kawasaki disease (KD) experience various extracardiovascular injury complications, which may affect their outcomes.</p><p><strong>Objective: </strong>To investigate the incidence and clinical characteristics of extracardiovascular complications in children with KD.</p><p><strong>Methods: </strong>The clinical data of patients diagnosed with KD in the First Affiliated Hospital of Guangxi Medical University from January 2003 to January 2021 were reviewed. The clinical characteristics and extracardiovascular complications were compared among patients stratified by age, intravenous immunoglobulin (IVIG) therapy responsiveness, and coronary status.</p><p><strong>Results: </strong>A total of 511 patients with KD were included, 357 (69.9%) were aged 1-5 years. Children aged <1 year (21.5%) and boys (70.8%) were more likely to have coronary artery lesions (CALs). The incidence of incomplete KD was lowest in 1-5-year-old patients (19.6%). Involvement of the hematological system gradually decreased with age (<1 year, 51.8%; 1-5 years, 36.7%; >5 years, 29.5%), whereas the involvement of the joints gradually increased with age (<1 year, 2.7%; 1-5 years, 6.2%; >5 years, 20.5%). Nervous system involvement was more common in IVIG non-responders (15.7% [13/83] <i>vs</i>. 5.4% [23/428], <i>P</i> = 0.001). However, there were no significant differences in extracardiovascular injury complications between patients with or without CALs.</p><p><strong>Interpretation: </strong>KD can involve multiple organ injuries as well as cardiovascular complications, and nervous systerm involvement may be more common in patients unresponsive to IVIG.</p>","PeriodicalId":19992,"journal":{"name":"Pediatric Investigation","volume":null,"pages":null},"PeriodicalIF":1.9,"publicationDate":"2022-11-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/b4/be/PED4-6-241.PMC9789935.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10459474","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Allogeneic hematopoietic stem cell transplantation with the modified myeloablative conditioning regimen for children with chronic active Epstein-Barr virus infection.","authors":"Yanhui Luo, Ang Wei, Bin Wang, Guanghua Zhu, Rui Zhang, Chenguang Jia, Yan Yan, Xuan Zhou, Jun Yang, Maoquan Qin, Tianyou Wang","doi":"10.1002/ped4.12350","DOIUrl":"10.1002/ped4.12350","url":null,"abstract":"<p><strong>Importance: </strong>Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is considered the only effective treatment for chronic active Epstein-Barr virus infection (CAEBV). The clinical efficacy and safety of allo-HSCT with different conditioning regimens in children with CAEBV remain unclear.</p><p><strong>Objective: </strong>To evaluate the effectiveness and safety of allo-HSCT with the modified myeloablative conditioning (MAC) regimen for children with CAEBV and also the factors affecting the outcomes.</p><p><strong>Methods: </strong>We retrospectively analyzed children with CAEBV who underwent allo-HSCT with the modified MAC regimen at Beijing Children's Hospital, Capital Medical University from October 2016 to June 2021. Data related to the clinical manifestations, engraftment, and outcome were extracted from the medical records.</p><p><strong>Results: </strong>The cohort comprised 41 patients (24 males, 17 females) with a median transplantation age of 92.6 (60.4, 120.7) months and a median follow-up time of 28.2 (15.3, 40.2) months. Four patients (9.8%) died, among which three died from primary disease relapse, and one died from grade IV acute graft-versus-host diseases (aGVHD) after stopping treatment. The 3-year overall survival (OS) and 3-year event-free survival (EFS) rates were 88.8% ± 5.4% and 85.0% ± 5.7%, respectively. The 3-year OS and EFS did not significantly differ between the patients with hemophagocytic lymphohistiocytosis (HLH) and the patient without HLH (87.7% ± 6.8% <i>vs</i>. 91.7% ± 8.0%, <i>P</i> = 0.790; 85.0% ± 6.9% <i>vs</i>. 84.6% ± 10.0%, <i>P</i> = 0.921), or among the patients with complete remission, partial remission, and activity disease before HSCT (all <i>P</i> > 0.05). Multivariate analysis showed that grade III-IV aGVHD was a risk factor for mortality (Hazards ratio: 11.65, 95% confidence interval: 1.00, 136.06; <i>P</i> = 0.050).</p><p><strong>Interpretation: </strong>Allo-HSCT with the modified MAC regimen is safe and effective for pediatric CAEBV. This treatment benefits patients with HLH or active disease. Patients with Grade III-IV aGVHD may be associated with worse outcomes.</p>","PeriodicalId":19992,"journal":{"name":"Pediatric Investigation","volume":null,"pages":null},"PeriodicalIF":1.9,"publicationDate":"2022-11-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/04/8d/PED4-6-250.PMC9789936.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10821628","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Is it worth already in the first step exceeding the 3-g protein limit for the oral food challenge for food protein-induced enterocolitis syndrome?","authors":"Francesco Mastellone, Giulia Bersani, Dario Sinatti, Mariannita Gelsomino, Stefano Miceli Sopo","doi":"10.1002/ped4.12351","DOIUrl":"10.1002/ped4.12351","url":null,"abstract":"","PeriodicalId":19992,"journal":{"name":"Pediatric Investigation","volume":null,"pages":null},"PeriodicalIF":1.9,"publicationDate":"2022-11-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/7b/d7/PED4-7-54.PMC10030697.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9193158","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Sitting intolerance: A new disease entity in children and adolescents.","authors":"Yumeng Gao, Hongfang Jin, Junbao Du","doi":"10.1002/ped4.12352","DOIUrl":"10.1002/ped4.12352","url":null,"abstract":"","PeriodicalId":19992,"journal":{"name":"Pediatric Investigation","volume":null,"pages":null},"PeriodicalIF":1.9,"publicationDate":"2022-11-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/31/bd/PED4-6-299.PMC9789932.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10821626","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Newborn screening for genetic disorders: Current status and prospects for the future.","authors":"Si Ding, Lianshu Han","doi":"10.1002/ped4.12343","DOIUrl":"10.1002/ped4.12343","url":null,"abstract":"<p><p>Newborn screening (NBS) is a public health service aimed at identifying infants with severe genetic disorders, thus providing effective treatment early enough to prevent or ameliorate the onset of symptoms. Current NBS uses biochemical analysis of dried blood spots, predominately with time-resolved fluorescence immunoassay and tandem mass spectrometry, which produces some false positives and false negatives. The application of enzymatic activity-based testing technology provides a reliable screening method for some disorders. Genetic testing is now commonly used for secondary or confirmatory testing after a positive result in some NBS programs. Recently, next-generation sequencing (NGS) has emerged as a robust tool that enables large panels of genes to be scanned together rapidly. Rapid advances in NGS emphasize the potential for genomic sequencing to improve NBS programs. However, some challenges still remain and require solution before this is applied for population screening.</p>","PeriodicalId":19992,"journal":{"name":"Pediatric Investigation","volume":null,"pages":null},"PeriodicalIF":1.9,"publicationDate":"2022-10-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/d1/58/PED4-6-291.PMC9789938.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10455182","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}