{"title":"Sirolimus treatment for intractable vascular anomalies (SIVA): An open-label, single-arm, multicenter, prospective trial.","authors":"Michio Ozeki, Saori Endo, Shiho Yasue, Ryuta Asada, Akiko M Saito, Hiroya Hashimoto, Shigeru Ueno, Shoji Watanabe, Motoi Kato, Kyoichi Deie, Shunsuke Nosaka, Mikiko Miyasaka, Akihiro Umezawa, Kentaro Matsuoka, Mototoshi Kato, Tatsuo Kuroda, Takanobu Maekawa, Satoshi Hirakawa, Taizo Furukawa, Shigehisa Fumino, Tatsuro Tajiri, Junkichi Takemoto, Naonori Kawakubo, Akihiro Fujino","doi":"10.1111/ped.70002","DOIUrl":"10.1111/ped.70002","url":null,"abstract":"<p><strong>Background: </strong>Intractable vascular anomalies (VAs), including vascular tumors and venous, lymphatic, and mixed malformations, often have severe symptoms and a poor prognosis, highlighting the need for new treatments. We conducted a prospective trial of sirolimus (tablet and granule forms) for the treatment of VAs.</p><p><strong>Methods: </strong>In this open-label, single-arm, multicenter trial across four Japanese institutions, patients with VAs received oral sirolimus daily, targeting a trough concentration of 5-15 ng/mL. We evaluated response rates (radiological volume changes in lesions), skin lesions, performance status, respiratory function, visceral symptoms (bleeding, pain), laboratory data, quality of life, and safety at 12, 24, and 52 weeks.</p><p><strong>Results: </strong>Thirteen patients with VAs were treated with sirolimus. Seven patients (53.8%; 95% confidence interval: 25.1%-80.8%) showed a partial radiological response at 24 weeks, with no complete responses, and 61.5% had a partial response by 12 weeks, with little subsequent change in patients who had stable disease thereafter. Improvements in skin lesions, blood coagulation, and activities of daily living were noted. Common adverse events included stomatitis, dermatitis, diarrhea, and fever.</p><p><strong>Conclusions: </strong>Sirolimus may reduce VA tissue volume and potentially improve symptoms and activities of daily living in patients with VAs.</p>","PeriodicalId":20039,"journal":{"name":"Pediatrics International","volume":"67 1","pages":"e70002"},"PeriodicalIF":1.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11937875/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143710744","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":"Conspicuous short stature induced by chronic functional constipation: A case report.","authors":"Ryo Matsuoka, Kenta Watanabe, Naoe Akiyama","doi":"10.1111/ped.70047","DOIUrl":"https://doi.org/10.1111/ped.70047","url":null,"abstract":"","PeriodicalId":20039,"journal":{"name":"Pediatrics International","volume":"67 1","pages":"e70047"},"PeriodicalIF":1.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144209134","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Emre Sarıkaya, Leyla Kara, Ebru Suman Gök, Uğur Berber, Ülkü Gül Şiraz, Nihal Hatipoglu
{"title":"Post-pandemic insights: Increased insulin resistance without dysglycemia in youths with overweight and obesity.","authors":"Emre Sarıkaya, Leyla Kara, Ebru Suman Gök, Uğur Berber, Ülkü Gül Şiraz, Nihal Hatipoglu","doi":"10.1111/ped.70068","DOIUrl":"https://doi.org/10.1111/ped.70068","url":null,"abstract":"<p><strong>Background: </strong>During the pandemic, lifestyle changes such as curfews, physical distancing, and increased screen time may have contributed to weight gain and obesity, raising the risk of dysglycemia in children and adolescents. The purpose of this study was to reveal the change in oral glucose tolerance test (OGTT) results of pediatric patients and adolescents before and after the declaration of the pandemic.</p><p><strong>Methods: </strong>Anthropometric measurements, physical examination findings, and laboratory results of patients with overweight or obesity who underwent the OGTT in 1-year periods before and after the pandemic declaration (2019 vs. 2021) in a tertiary single pediatric endocrinology center were obtained from the archive.</p><p><strong>Results: </strong>OGTT was performed on 146 (43.8% boys) before the pandemic declaration and 246 (37.3% boys) consecutive patients (68% increase) after the pandemic declaration. While there were no substantial variations in body mass index (BMI), BMI standard deviations (SD), glucose, glycosylated hemoglobin (HbA1c), and lipid profile, distinct changes were observed in insulin, homeostatic model assessment of insulin resistance (HOMA-IR), calcium, vitamin D, OGTT 0- and 30-min glucose, as well as ≥30-min and total insulin values.</p><p><strong>Conclusion: </strong>This study reveals a significant rise in insulin resistance diagnoses in youths with overweight and obesity undergoing OGTT post-pandemic, while dysglycemia rates remained stable. These findings highlight the need to assess insulin levels alongside glucose in OGTTs for this population, as results may not apply to the broader pediatric group.</p>","PeriodicalId":20039,"journal":{"name":"Pediatrics International","volume":"67 1","pages":"e70068"},"PeriodicalIF":1.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144209136","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Evaluation of cardiac function in patients with SMA after treatment with onasemnogene abeparvovec.","authors":"Akihisa Horigome, Satoshi Takasago, Reiko Arakawa, Katsuyoshi Shimozawa, Masao Kaneshige, Keiji Goishi, Hideko Uryu, Junko Yamanaka, Hiroyuki Shichino, Ayumi Mizukami","doi":"10.1111/ped.70019","DOIUrl":"10.1111/ped.70019","url":null,"abstract":"<p><strong>Background: </strong>Spinal muscular atrophy (SMA) is a recessively inherited neurological disease resulting in motor neuron disorder. Onasemnogene abeparvovec is a gene replacement therapy used to treat patients with SMA. Cardiac toxicity was observed in animal studies on this therapy, and elevated cardiac troponin I levels were observed in clinical trials; however, the clinical importance of these findings is unknown. Therefore, this study aimed to reveal the cardiac toxicity of onasemnogene abeparvovec through careful investigation of cardiac function using cardiac strain analysis, which can detect early subtle abnormalities.</p><p><strong>Methods: </strong>This study included patients with SMA treated with onasemnogene abeparvovec between June 2020 and November 2020. Echocardiography, including peak global longitudinal strain (GLS), and other laboratory test results were evaluated.</p><p><strong>Results: </strong>Case 1 showed a relative GLS decrease of 14.5% compared with that at the baseline (GLS reduction from -22.1% to -18.9%), elevation of N-terminal prohormone B-type natriuretic peptide levels from 227 pg/mL to 494 pg/mL, and elevated liver enzyme concentrations after gene replacement therapy without reduction of the left ventricular ejection fraction.</p><p><strong>Conclusions: </strong>Subclinical myocardial dysfunction after infusion of onasemnogene abeparvovec was suggested by careful investigation of cardiac function. Cardiac toxicity may correlate with liver impairment following gene replacement therapy. Long-term studies that allow for a higher number of patients and more extended observation periods should be conducted to confirm the cardiac toxicity of onasemnogene abeparvovec.</p>","PeriodicalId":20039,"journal":{"name":"Pediatrics International","volume":"67 1","pages":"e70019"},"PeriodicalIF":1.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144038037","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Hayato Ogura, Hidetsugu Asano, Shuji Kitahara, Eiji Hirakawa, Ken Masamune
{"title":"Impact of non-contact respiratory rate monitoring in infants: Effects of posture, movement, and sensor placement.","authors":"Hayato Ogura, Hidetsugu Asano, Shuji Kitahara, Eiji Hirakawa, Ken Masamune","doi":"10.1111/ped.70097","DOIUrl":"https://doi.org/10.1111/ped.70097","url":null,"abstract":"<p><strong>Background: </strong>Premature infants are at high risk for medical adhesive-related skin injuries. It is crucial to establish reliable methods for non-contact monitoring. This study aimed to identify the optimal piezoelectric sensor placement (chest, abdomen, and hips) for respiratory rate estimation in each body position (supine, lateral, and prone) and demonstrate that body movements affect respiratory estimation accuracy.</p><p><strong>Methods: </strong>Respiratory rates were estimated for 30 infants admitted to the Growing Care Unit for each body position. Body positions, movements (categorized by the Neonatal Behavioral Assessment Scale), and sensor placements were recorded and analyzed for their effects on accuracy. Estimation accuracy was defined as the percentage of time during which the error between the estimated and reference (respiratory rate displayed on the monitor) was within 5 breaths/min.</p><p><strong>Results: </strong>The most improved estimation accuracies were achieved in the supine position with the chest sensor, in the lateral position with the abdominal sensor. The prone position was limited to a single case, thus rendering it inappropriate to draw comparisons. Accuracy declined progressively as the infants' state changed from sleeping to crying. Moreover, substantial reductions in accuracy were observed during full-body movements (quiet and active alert state) as well as during arm movements (drowsy and quiet alert state).</p><p><strong>Conclusion: </strong>This study demonstrated that the accuracy of non-contact respiratory rate estimation using piezoelectric sensors is highly impacted by body position, movement, and sensor placement. Optimizing the algorithm to systematically account for these variables is essential for improving the robustness and accuracy of non-contact vital sign monitoring.</p>","PeriodicalId":20039,"journal":{"name":"Pediatrics International","volume":"67 1","pages":"e70097"},"PeriodicalIF":1.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144249080","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Task-oriented game therapy enhances upper extremity functions for children with cerebral palsy.","authors":"Mehmet Sönmez, Sena Sönmez, Musa Eymir","doi":"10.1111/ped.70116","DOIUrl":"https://doi.org/10.1111/ped.70116","url":null,"abstract":"<p><strong>Background: </strong>This study aimed to compare the Fruit Ninja® game (FNG) therapy combined with standard physiotherapy (PT) to standard PT with sham-FNG therapy in children with Cerebral palsy (CP) in terms of hand functions, grip strength, and range of motion (ROM).</p><p><strong>Method: </strong>Twenty children with CP were randomized to the experimental group (EG) and the sham group (SG). Both groups received standard PT for 45-min sessions, three times a week for 6 weeks. The EG additionally performed FNG training for 20-min sessions, three times a week for 6 weeks. The SG watched the FNG demonstration video and tried to perform the movements on a flat table by tracking the video for the same duration as the EG.</p><p><strong>Results: </strong>All baseline values were comparable (p > 0.05). The post-trial within-group analysis showed significant improvements in some variables (for EG: dominant-hands' grip strength, wrist extension, and radial deviation, non-dominant-hands' ulnar deviation, and both hands' 9-HPT score, Box and Block test (BBT) score, and wrist flexion; for SG: dominant-hands' grip strength and ulnar deviation, and both hands' BBT score; for all, p < 0.05) in both groups. EG showed statistically significant improvement in hand functions compared to SG (for all; p < 0.05).</p><p><strong>Conclusion: </strong>The FNG therapy offers beneficial results for hand functioning in the rehabilitation of children with CP.</p>","PeriodicalId":20039,"journal":{"name":"Pediatrics International","volume":"67 1","pages":"e70116"},"PeriodicalIF":1.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144267024","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"A case of pulmonary embolism occurring with cardiopulmonary arrest after catheter ablation.","authors":"Hiroki Ishii, Yu Matsumura, Tadahiro Yoshikawa","doi":"10.1111/ped.15858","DOIUrl":"https://doi.org/10.1111/ped.15858","url":null,"abstract":"","PeriodicalId":20039,"journal":{"name":"Pediatrics International","volume":"67 1","pages":"e15858"},"PeriodicalIF":1.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143692889","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}