{"title":"ROTEM could be useful for lupus anticoagulant hypoprothrombinemia syndrome.","authors":"Koyo Yamada, Yuto Nakajima, Kenichi Ogiwara, Toshiyuki Sakai, Kazuyoshi Fukuda, Keiji Nogami","doi":"10.1111/ped.15773","DOIUrl":"10.1111/ped.15773","url":null,"abstract":"<p><strong>Background: </strong>Lupus anticoagulant-hypoprothrombinemia syndrome (LAHPS) is a rare disease caused by acquired factor II (FII) deficiency and lupus anticoagulant. Patients with LAHPS typically present with thrombosis and bleeding. However, little information is available on the evaluation of coagulation potential in patients with LAHPS. We examined global coagulation potentials in patients with LAHPS during the clinical course in this study.</p><p><strong>Methods: </strong>Coagulation potentials in two pediatric patients with LAHPS were assessed by measuring clotting time (CT) and clot formation time using Ca<sup>2+</sup>-triggered rotational thromboelastometry (ROTEM), CT and maximum coagulation velocity using clot waveform analysis (CWA), and lag time and peak thrombin using the thrombin generation assay (TGA). The day of admission was defined as day 0.</p><p><strong>Results: </strong>In case 1, the bleeding symptoms disappeared by day 5. However, the TGA and CWA results were markedly lower than normal, although FII activity (FII:C) returned to within the normal range by day 14. In contrast, ROTEM revealed a recovery to near-normal levels (day 14). All coagulation parameters (day 80) were within normal ranges. In case 2, coagulation potential was severely depressed until day 12, although FII:C returned to normal levels. Bleeding symptoms disappeared on day 19, and the ROTEM data revealed that the parameters were close to the normal range. The coagulation parameters in all assays were normalized on day 75.</p><p><strong>Conclusions: </strong>Recovery of coagulation potential in patients with LAHPS was slower than the recovery of FII:C. Moreover, ROTEM appeared to be clinically useful for assessing coagulation potential in patients with LAHPS.</p>","PeriodicalId":20039,"journal":{"name":"Pediatrics International","volume":"66 1","pages":"e15773"},"PeriodicalIF":1.0,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141306587","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":"Two cases of pediatric splenic torsion as a possible late complication of laparoscopic fundoplication.","authors":"Takayuki Hiraba, Hiroya Ogita, Takayuki Yokota, Sonoko Kondo, Aya Tanaka, Ryuichi Shimono, Takashi Kusaka","doi":"10.1111/ped.15810","DOIUrl":"10.1111/ped.15810","url":null,"abstract":"","PeriodicalId":20039,"journal":{"name":"Pediatrics International","volume":"66 1","pages":"e15810"},"PeriodicalIF":1.0,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142004959","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}
Tatsuki Fukuie, Hiroyuki Toyama, Mai Tanaka, Katsuyo Ohashi-Doi, Kenji Kabashima
{"title":"Post-hoc safety/efficacy analyses from pediatric delgocitinib atopic dermatitis trials.","authors":"Tatsuki Fukuie, Hiroyuki Toyama, Mai Tanaka, Katsuyo Ohashi-Doi, Kenji Kabashima","doi":"10.1111/ped.15798","DOIUrl":"10.1111/ped.15798","url":null,"abstract":"<p><strong>Background: </strong>Delgocitinib ointment is usually recommended for use in children at a concentration of 0.25%. However, there are no clear criteria for dosing, except that a 0.5% formulation may also be used, depending on symptom severity. Treatment of atopic dermatitis is based on combinations of topical corticosteroids, tacrolimus ointment, and delgocitinib ointment, but there are no reports on the safety of delgocitinib ointment when used in combination with other drugs.</p><p><strong>Methods: </strong>This is a post-hoc analysis of data from two delgocitinib ointment trials with pediatric atopic dermatitis patients. The efficacy and safety of the 0.25% and 0.5% formulations were compared. Efficacy and safety were evaluated after up to 4 and 56 weeks of treatment, respectively. The safety of delgocitinib ointment when used in combination with topical corticosteroids and/or tacrolimus ointment was investigated.</p><p><strong>Results: </strong>The dose-response relationship was examined according to baseline disease severity. The proportions of subjects with mild disease who achieved cumulative investigator's global assessment of 0 (clear) or 1 (almost clear) were 46.2% (0.25% ointment), 71.4% (0.5% ointment), and 7.7% (vehicle). For subjects with moderate to severe disease, the corresponding proportions were 19.0%, 20.0%, and 0.0%, respectively. No overall differences were seen in the safety profiles of the 0.25% and 0.5% delgocitinib ointment doses, or in the safety profiles of the two doses relating to disease severity or to concomitant use of topical corticosteroids and/or tacrolimus ointment.</p><p><strong>Conclusions: </strong>These analyses indicate that after up to 4 weeks of treatment, delgocitinib 0.5% ointment may be more effective than the 0.25% dose for mild atopic dermatitis, and that after up to 56 weeks of treatment, delgocitinib is well tolerated in a pediatric trial population when used as prescribed in combination with topical corticosteroids and/or tacrolimus ointment.</p>","PeriodicalId":20039,"journal":{"name":"Pediatrics International","volume":"66 1","pages":"e15798"},"PeriodicalIF":1.0,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11580369/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142381472","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":"Handling of human milk to prevent acquired cytomegalovirus infection in Japanese neonatal intensive care units: The first nationwide survey.","authors":"Kana Saito, Kosuke Tanaka, Ryota Nakagawa, Junichi Ozawa, Mitsuhiro Haga, Naoyuki Miyahara, Kazuhiko Kabe, Fumihiko Namba","doi":"10.1111/ped.15728","DOIUrl":"10.1111/ped.15728","url":null,"abstract":"<p><strong>Background: </strong>Human milk (HM) has been proven to provide immunological and nutritional advantages to neonates; however, acquired cytomegalovirus (CMV) infection can be associated with raw HM. In Japan, there are no standardized guidelines concerning HM handling. This cross-sectional survey was performed to reveal specific trends in HM handling in neonatal intensive care units (NICUs) in Japan.</p><p><strong>Methods: </strong>A questionnaire was sent to 255 NICUs participating in the Japanese Neonatologist Association in May 2020. It involved HM handling practices, such as maternal screening, pasteurization, storage, and the workforce.</p><p><strong>Results: </strong>Of 255 NICUs, 174 (67.8%) responded to the survey. Maternal CMV screening was carried out in 37 units (22.2%), and CMV inactivation in HM was performed in 44 units (26.5%). For CMV inactivation, a freeze-thawing method was employed in about 90% of units. In 70% of units providing CMV inactivation, CMV inactivation was conducted regardless of bodyweight and corrected gestational age of infants until the infants' discharge. Acquired CMV infection in preterm neonates was observed in 43 units (25.7%) in the survey period.</p><p><strong>Conclusion: </strong>A wide range of HM handling practices are used in Japanese NICUs. A national guideline for handling HM in NICUs should be created to promote the infection control of CMV.</p>","PeriodicalId":20039,"journal":{"name":"Pediatrics International","volume":"66 1","pages":"e15728"},"PeriodicalIF":1.4,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140336469","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":"Clinical characteristics and risk factors for kidney involvement in children with immunoglobulin A vasculitis.","authors":"Boonyapohn Chatpaitoon, Pornpimol Rianthavorn, Ankanee Chanakul, Parichat Khaosut","doi":"10.1111/ped.15781","DOIUrl":"10.1111/ped.15781","url":null,"abstract":"<p><strong>Background: </strong>Immumoglobulin A (IgA) vasculitis (IgAV), formerly known as Henoch-Schönlein purpura (HSP), is a self-limiting systemic vasculitis in children. Kidney involvement is associated with a long-term unfavorable outcome and can lead to significant morbidity. This study was conducted to describe the clinical and laboratory characteristics of childhood IgAV with kidney involvement and to identify risk factors associated with IgAV nephritis (IgAVN).</p><p><strong>Methods: </strong>This was an ambidirectional descriptive study of 77 children with IgAV. All demographic data, clinical features, and laboratory tests were collected from electronic medical records from January 2010 to December 2022. Risk factors for kidney involvement in IgAV were assessed using multivariate logistic regression. Kaplan-Meier survival analysis was used to calculate the time to commencement of kidney involvement.</p><p><strong>Results: </strong>Twenty-five children (32.4% of the IgAV patients) developed IgAVN. The common findings in IgAV with kidney involvement were microscopic hematuria (100%), nephrotic range proteinuria (44%), and non-nephrotic range proteinuria (40%). Multivariate logistic regression showed that age greater than 10 years (adjusted hazard ratio, AHR 4.66; 95% confidence interval, CI, 1.91-11.41; p = 0.001), obesity (body mass index, BMI, z-score ≥ +2 standard deviations, SDs) (AHR 3.59; 95% CI 1.41-9.17; p = 0.007), and hypertension at onset (AHR 4.78; 95% CI 1.76-12.95; p = 0.002) were associated significantly with kidney involvement. During follow up, most IgAV patients developed nephritis within the first 9 months.</p><p><strong>Conclusion: </strong>Age greater than 10 years, obesity, and hypertension at presentation were predictive factors for IgAVN. Our study emphasized that IgAV patients with risk factors should be closely monitored for at least 1 year after the onset of the disease.</p>","PeriodicalId":20039,"journal":{"name":"Pediatrics International","volume":"66 1","pages":"e15781"},"PeriodicalIF":1.0,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141306544","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}
Muhammet Furkan Korkmaz, Ahmet Karacan, Adalet Mehmedali, Şefika Elmas Bozdemir, Didar Arslan, Arzu Oto, Murat Tutanç
{"title":"Immature granulocytes as an early clinical predictor in children with respiratory syncytial virus bronchiolitis.","authors":"Muhammet Furkan Korkmaz, Ahmet Karacan, Adalet Mehmedali, Şefika Elmas Bozdemir, Didar Arslan, Arzu Oto, Murat Tutanç","doi":"10.1111/ped.15840","DOIUrl":"10.1111/ped.15840","url":null,"abstract":"<p><strong>Background: </strong>Immature granulocytes can be measured easily in a complete blood count by new automated hemolytic analyzers and have recently been studied as bio-markers in many infectious/inflammatory diseases. This study aims to investigate whether immature granulocyte percentage (IG%) would enable greater discrimination than conventionally utilized laboratory values in terms of early clinical prediction in instances with respiratory syncytial virus (RSV) bronchiolitis.</p><p><strong>Methods: </strong>A prospective observational cohort study involved 149 individuals with RSV bronchiolitis. Complete blood count (including IG%), C-reactive protein, and procalcitonin (PCT) assays were performed.</p><p><strong>Results: </strong>Sixty-two (42%) RSV bronchiolitis patients responded well to outpatient therapy; 60 (40%) were admitted to the hospital, and 27 (18%) required pediatric intensive care unit (PICU) follow up. Inpatients had a higher platelet count than outpatients, and PICU patients had a higher platelet lymphocyte ratio (PLR). Pediatric intensive care unit patients had greater PCT levels than outpatients, and their IG% was higher than that of both inpatient and outpatient groups. In the receiver operating characteristic analysis examining the usefulness of parameters in predicting PICU hospitalization, PLR, PCT, and IG% produced statistically significant findings, with IG% having the highest area under the curve (0.730).</p><p><strong>Conclusions: </strong>Immature granulocyte percentage was the biomarker that best differentiated PICU patients from other patient groups when compared with traditional acute phase reactants. Immature granulocytes may help to determine the severity of infection/inflammation at an early stage of the disease.</p>","PeriodicalId":20039,"journal":{"name":"Pediatrics International","volume":"66 1","pages":"e15840"},"PeriodicalIF":1.0,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142854905","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":"Splenomegaly and progressive neurologic involvement: Think about Niemann-Pick type C disease.","authors":"Merve Yoldas Celik, Havva Yazici, Fehime Erdem, Ayse Yuksel Yanbolu, Ebru Canda, Ebru Sezer, Sema Kalkan Ucar, Huseyin Onay, Ferda Ozkinay, Eser Yildirim Sozmen, Mahmut Coker","doi":"10.1111/ped.15832","DOIUrl":"https://doi.org/10.1111/ped.15832","url":null,"abstract":"<p><strong>Background: </strong>Niemann-Pick type C (NPC) disease is a lysosomal storage disease with visceral organ involvement and neurological and psychiatric symptoms. This study presents the clinical and laboratory findings of NPC cases involving three novel variants.</p><p><strong>Methods: </strong>The clinical and laboratory findings were reviewed retrospectively between February 2006 and December 2022.</p><p><strong>Results: </strong>There were nine females and five males. The median age of diagnosis of the patients was 5 months (range: 1 month to 38 years). The clinical phenotypes of the patients were early infantile (n = 7), late infantile (n = 4), juvenile (n = 2), and adult (n = 1). The visceral findings were splenomegaly (100%), hepatomegaly (78.6%), neonatal jaundice (42.8%), and hepatic failure (14.3%). The neurological findings were developmental delay (71.4%), seizures (50%), vertical supranuclear gaze palsy (28.6%), ataxia (28.6%), dysarthria (28.6%), dysphagia (21.4%), dystonia (21.4%). Cataplexy (14.3%) and psychiatric manifestations (14.3%) were recorded in juvenile and adult-onset patients. Three novel variants were detected (p.Asn524LysfsTer39, p.Val1023Phe, and p.Asn906Tyr). Miglustat treatment was received by 64.3% of patients but had no clear effect on the disease course. The median duration of use was 3 years (with a range from 1-13 years). Miglustat's adverse effects were low platelet count (35.7%) and diarrhea (7.1%).</p><p><strong>Conclusions: </strong>Niemann-Pick type C disease should be considered in the presence of visceral organ involvement and progressive neurological findings. It is essential to support the diagnosis with laboratory and genetic analysis.</p>","PeriodicalId":20039,"journal":{"name":"Pediatrics International","volume":"66 1","pages":"e15832"},"PeriodicalIF":1.0,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142854996","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}