Italian Journal of Pediatrics最新文献

{"title":"Activin-A urine levels correlate with radiological patterns in preterm infants complicated by intraventricular hemorrhage.","authors":"Giuseppe Lapergola, Giorgia Gasparroni, Alessandro Graziosi, Darek Gruzfeld, Bashir Moataza, Hanna Aboulgar, Hala Mufeed, Iman Iskander, Giovanni Livolti, Fabio Galvano, Gabriella Levantini, Ebe D'Adamo, Adele Patrizia Primavera, Elisabetta Barbante, Rita Salomone, Claudia D'Egidio, Chiara Strozzi, Antonio Maconi, Danilo A W Gavilanes, Ali Saber Abdelhameed, Diego Gazzolo","doi":"10.1186/s13052-025-01938-4","DOIUrl":"10.1186/s13052-025-01938-4","url":null,"abstract":"<p><strong>Background: </strong>To validate the role of Activin A in the early diagnosis and prognosis of preterm newborns at risk for intraventricular hemorrhage and neurological sequelae by means of cerebral ultrasound and magnetic resonance imaging (MRI), currently considered standard of care procedures.</p><p><strong>Methods: </strong>We conducted an observational case-control study in 46 preterm newborns, 23 with intraventricular hemorrhage (IVH group) and 23 controls matched for gestational age. Standard clinical, laboratory, cerebral ultrasound monitoring procedures and Activin A urine measurement were performed at four time-points (first void, 24, 48, 96 h) after birth. Cerebral MRI was performed at 40-42 weeks of corrected gestational age.</p><p><strong>Results: </strong>Elevated (P < 0.001, for all) Activin A levels were observed in the IVH group at all monitoring time-point. Activin A correlated (P < 0.05, for all) with intraventricular hemorrhage grade on cerebral ultrasound. At the cut-off of 0.08 pg/mL Activin A at 48-h achieved the best sensitivity, specificity, positive/negative predictive values as early predictor of an abnormal MRI pattern (area under the curve: 0.93).</p><p><strong>Conclusions: </strong>The present data showing a correlation among Activin A, cerebral ultrasound and MRI provide further support to Activin A inclusion in clinical daily management of cases at risk for intraventricular hemorrhage and adverse neurological outcome.</p>","PeriodicalId":14511,"journal":{"name":"Italian Journal of Pediatrics","volume":"51 1","pages":"85"},"PeriodicalIF":3.2,"publicationDate":"2025-03-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143669843","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Safety and efficacy of long term asfotase alfa treatment in childhood hypophosphatasia.","authors":"Debora Mariarita d'Angelo, Federico Lauriola, Luisa Silvestrini, Luigia Cinque, Marco Castori, Giulia Di Donato, Armando Di Ludovico, Saverio La Bella, Francesco Chiarelli, Cosimo Giannini, Luciana Breda","doi":"10.1186/s13052-025-01883-2","DOIUrl":"10.1186/s13052-025-01883-2","url":null,"abstract":"<p><strong>Background: </strong>Hypophosphatasia (HPP) is a rare inherited disorder characterized by a deficiency of tissue-non-specific alkaline phosphatase (TNSALP) due to loss-of-function variants of the ALPL gene. HPP is characterized by an extremely variable age of onset and clinical presentation, largely depending on the type of genetic disruption. Childhood HPP commonly presents with skeletal deformities, bone fragility, precocious tooth loss, muscle weakness and sometimes neurological implications. Laboratory tests usually document low levels of alkaline phosphatase (ALP), and radiologic investigations show peculiar bone abnormalities. Treatment with human recombinant TNSALP (asfotase alpha, Strensiq^®), available since 2015, is associated with a sudden improvement and a good safety profile.</p><p><strong>Case presentation: </strong>A previously healthy 15-month-old girl presented with progressive \"genu valgus\" and sudden limping. The patient was diagnosed with childhood HPP due to the presence of two ALPL variants, never described in compound heterozygosity: a missense variant c.571G > A, p.(Glu191Lys), and a frameshift deletion c.963delG; p.(Lys322Argfs*44), both classified as pathogenetic. The child was promptly treated with asfotase alpha, and good improvement was quickly obtained. Efficacy, safety, and good tolerance persisted after a long-term follow-up of 6 years.</p><p><strong>Conclusions: </strong>Pediatricians should consider HPP in children presenting with a suggestive clinical phenotype. Calcium-phosphorus metabolism, ALP, and vitamin B6 should always be investigated in suspected cases. Moreover, asfotase alfa represents a safe, well-tolerated, and effective drug in children with HPP.</p>","PeriodicalId":14511,"journal":{"name":"Italian Journal of Pediatrics","volume":"51 1","pages":"86"},"PeriodicalIF":3.2,"publicationDate":"2025-03-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143669845","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Prevalence and factors associated with hypertension among children attending pre-schools in Dar es Salaam, Tanzania.","authors":"Jida Said, Nahya Salim, Peter P Kunambi, Francis Furia","doi":"10.1186/s13052-025-01841-y","DOIUrl":"10.1186/s13052-025-01841-y","url":null,"abstract":"<p><strong>Background: </strong>Childhood hypertension has become a public health problem due to its increasing prevalence and complications; the high prevalence is noted to mirror an increase in obesity among children. Hypertension in children is frequently undiagnosed due to challenges in getting appropriate cuff sizes and interpretation of the readings. Several studies have been carried out among children however; most of available information is focused on hypertension among older children and adolescents.</p><p><strong>Methods: </strong>A cross sectional study was conducted in 2 districts of Dar es Salaam region namely Ilala and Kinondoni from October to November 2020. Children aged 2-5 years attending pre-schools in these districts meeting the inclusion criteria and whose parent/guardian signed informed consent were included. Questionnaires were used to collect socio-demographic characteristics; anthropometric and three oscillometric single- occasion blood pressure measurements were taken. The average blood pressure was compared to the standard charts for age and sex provided by the American Academy of Pediatrics 2017 to determine the blood pressure category of the child.</p><p><strong>Results: </strong>A total of 1083 children fulfilled the eligibility criteria and were enrolled into the study, 51.3% (556/1083) of participants were males and the median age was 4 years (IQR 3-5). Blood pressures for 252 (23.3%) participants were in the high blood pressure range (19.8% with elevated blood pressure and 3.5% with hypertension). No significant gender difference was observed among those with high blood pressure. Factors that were noted to be significantly associated with elevated blood pressure included low birth weight (p = 0.036), increasing age (p = 0.032) and body mass index (p < 0.001).</p><p><strong>Conclusion: </strong>High prevalence of elevated blood pressure in this population of pre-school aged children is alarming. Low birth weight, increasing age and body mass index were significantly associated with elevated blood pressure.</p>","PeriodicalId":14511,"journal":{"name":"Italian Journal of Pediatrics","volume":"51 1","pages":"84"},"PeriodicalIF":3.2,"publicationDate":"2025-03-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11921718/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143663489","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Clinical characteristics and impact of exchange transfusion in infant pertussis with extreme leukocytosis.","authors":"Yuanyuan Wu, Chuan Gan","doi":"10.1186/s13052-025-01933-9","DOIUrl":"10.1186/s13052-025-01933-9","url":null,"abstract":"<p><strong>Background: </strong>Extreme leukocytosis in pertussis is a rare condition, and without effective interventions to reduce white blood cell counts, the mortality rate can approach 100%. The clinical characteristics of these patients and the application of exchange transfusion (ET) in their management are not yet clear.</p><p><strong>Methods: </strong>This retrospective study examines the clinical characteristics and impact of ET in infant pertussis with extreme leukocytosis.</p><p><strong>Results: </strong>We have included six infant pertussis patients with extreme leukocytosis, all of whom were female and underwent ET. Two patients survived, while four died. The surviving patients were relatively older at disease onset compared to those who died, and all three unvaccinated patients died. All patients required admission to the pediatric intensive care unit, presenting with fever, whooping cough, cyanosis, severe pneumonia, and respiratory failure. Pulmonary consolidation, cardiovascular failure, and pulmonary hypertension (PH) were also common, especially among those who died. Hypoglycemia and seizures were rare. Acute-phase proteins, such as C-reactive protein and procalcitonin, were elevated to varying degrees. ET effectively reduced peripheral blood leukocytes; however, a significant increase in leukocytes was observed 1-2 days after the first ET in the deceased patients.</p><p><strong>Conclusion: </strong>Extreme hyperleukocytosis is more commonly observed in young female children with pertussis. Younger age, unvaccinated status, and the presence of concurrent heart failure and PH may be associated with a poor prognosis. ET can effectively reduce peripheral blood leukocytes, but a rapid leukocyte rebound post-ET may be indicative of impending death.</p>","PeriodicalId":14511,"journal":{"name":"Italian Journal of Pediatrics","volume":"51 1","pages":"82"},"PeriodicalIF":3.2,"publicationDate":"2025-03-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11921730/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143656658","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"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 for mucopolysaccharidosis patients: a single-center experience and assessment of quality of life.","authors":"Wen Zhang, Yonglan Huang, Xueying Su, Xiaoyuan Zhao, Huiying Sheng, Cuili Liang, Minyan Jiang, Chunhua Zeng, Yanna Cai, Yunting Lin, Yongxian Shao, Sha Liu, Hua Jiang, Li Liu","doi":"10.1186/s13052-025-01919-7","DOIUrl":"10.1186/s13052-025-01919-7","url":null,"abstract":"<p><strong>Background: </strong>Allogeneic hematopoietic stem cell transplantation (HSCT) has proven to be a viable treatment option for patients with mucopolysaccharidoses (MPS). We investigate the efficacy and improvements in the quality of life of HSCT in pediatric patients with MPS.</p><p><strong>Methods: </strong>A retrospective analysis of transplantation data from 46 cases of MPS from a single institution in China was conducted.</p><p><strong>Results: </strong>The cohort of 46 patients included 9 cases of MPS I, 16 cases of MPS II, 15 cases of MPS IVA and 6 cases of MPS VI. The median age at diagnosis was 2.59 years. The median age at transplantation was 3.80 years. The median follow-up time was 3.1 years (range, 0.8-8.1 years) and 43 patients were alive. The incidence of grades II to IV aGVHD was 17.4%, wherein the incidence of grades III and IV aGVHD was 4.3%. The incidence of moderate-to-severe cGVHD was 6.5%. GAGs urinary excretion decreased and enzyme activity levels reached normal. After HSCT, multiple bone dysplasia, upper-airway obstruction and recurrent otitis media were significantly improved; vision, corneal clouding, cardiovascular disease, hepatosplenomegaly and hydrocephalus were improved or remained stable; neurological symptoms were improved or remained stable in most patients but progressed in others; the patients with MPS IH/S and MPS II reached nearly normal growth rate of height and weight. Meanwhile, the patients with MPS IH, MPS IVA and MPS VI remained poor growth after HSCT. The Activities of Daily Living (ADL) scores were improved in most patients with MPS. ADL scores in patients with severe phenotypes were lower than health control subjects and patients with attenuated phenotypes.</p><p><strong>Conclusions: </strong>HSCT is a good therapeutic option for MPS and improves the quality of life of patients. MPS patients with attenuated phenotypes provide a better outcome in ADL after HSCT.</p>","PeriodicalId":14511,"journal":{"name":"Italian Journal of Pediatrics","volume":"51 1","pages":"83"},"PeriodicalIF":3.2,"publicationDate":"2025-03-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11921649/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143657186","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Proteomic analysis of premature umbilical cord blood and its relationship with bronchopulmonary dysplasia.","authors":"Jia Chen, Yuanye He, Ying Liu, Zhiwei Guo, Longli Yan, Xiaotao Jiang, Weiwei Gao","doi":"10.1186/s13052-025-01926-8","DOIUrl":"10.1186/s13052-025-01926-8","url":null,"abstract":"<p><strong>Background: </strong>Bronchopulmonary dysplasia (BPD) frequently occurs in preterm infants, causing significantly impaired lung function and increased mortality rates. Studies on plasma protein levels can facilitate early detection of BPD, enabling prompt intervention and a decrease in mortality.</p><p><strong>Methods: </strong>We conducted a prospective observational study involving proteomic sequencing of plasma samples from 19 preterm infants. Our analysis included principal component analysis, volcano plots, heatmap analysis, enrichment analysis, and receiver operating characteristic (ROC) analysis.</p><p><strong>Results: </strong>Infants with BPD were characterized by increased levels of lipopolysaccharide (LPS)-binding protein (LBP), X-ray repair cross-complementing protein 6 (XRCC6), GLI pathogenesis-related 1 (GLIPR1), Golgi membrane Protein 1(GOLM1), immunoglobulin kappa variable (IGKV1-5), and immunoglobulin kappa variable 1-33 (IGKV1-33) in cord blood. Additionally, gene pathway analysis revealed a significant correlation between the pathways associated with these genes and BPD, particularly pathways involved in the immune system, innate immune system, neutrophil degranulation, prion diseases, regulation of the actin cytoskeleton, and the MAPK signaling. The proteins amine oxidase copper containing 3 (AOC3) and H4 clustered histone 6 (H4C6) were diagnostically significant. Additionally, H4C6 was negatively correlated with intraventricular haemorrhage and patent ductus arteriosus, and positively correlated with antenatal steroid administration. AOC3 was also positively correlated with antenatal steroid use.</p><p><strong>Conclusions: </strong>Our findings suggest that the development of BPD is associated with changes in the plasma proteome of preterm infants. Specifically, the levels of AOC3 and H4C6 in the bloodstream could serve as biomarkers for the early detection of BPD in preterm infants. Furthermore, we found that GOLM1, lipopolysaccharide (LPS)-binding protein, XRCC6, and the contribution of neutrophil degranulation may play a crucial role in the development of therapies for BPD.</p>","PeriodicalId":14511,"journal":{"name":"Italian Journal of Pediatrics","volume":"51 1","pages":"81"},"PeriodicalIF":3.2,"publicationDate":"2025-03-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11917046/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143648446","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Analysis of risk factors and establishment of predictive models for neonatal necrotizing enterocolitis: a retrospective study.","authors":"Keqin Liu, Jinjin Guo, Yaqi Zhu, Jixin Yang, Yanwei Su","doi":"10.1186/s13052-025-01930-y","DOIUrl":"10.1186/s13052-025-01930-y","url":null,"abstract":"<p><strong>Background: </strong>Necrotizing enterocolitis (NEC) is a leading gastrointestinal condition in preterm infants, characterized by significant morbidity and mortality. Early recognition of risk factors is crucial for its prevention and prediction. This study focuses on identifying factors that contribute to the development of NEC in neonates.</p><p><strong>Methods: </strong>A case-control study that looked back at 144 newborns hospitalized to a Wuhan hospital between January 2010 and March 2023 for NEC was carried out. Over the same period, another 144 children without NEC were identified and selected as the non-NEC group for comparison, following a 1:1 pairing ratio. The relevant data from these two groups of newborns were compared. Univariate analysis was conducted using T-tests or χ² tests, followed by multivariate logistic regression to determine independent risk factors and develop a clinical prediction model.</p><p><strong>Results: </strong>A total of 288 neonates (144 NEC and 144 non-NEC) were enrolled. The independent risk variables for NEC, as shown by the multivariate logistic regression analysis (p < 0.05), were Small for Gestational Age (SGA), neonatal sepsis, neonatal hyperbilirubinemia, and non-human milk (HM) feeding. Furthermore, ROC (receiver operating characteristic) analysis showed that the AUC (area under the curve) of the Logistic regression model predicting the effect of neonatal necrotizing enterocolitis was 0.746, suggesting a high level of discriminative ability in differentiating efficacy. This model can be instrumental in facilitating early identification of infants prone to developing NEC in clinical settings.</p><p><strong>Conclusion: </strong>In conclusion, the risk factors associated with newborn NEC include SGA, neonatal sepsis, and non-HM feeding. Newborn hyperbilirubinemia may potentially serve as a protective factor against NEC.</p>","PeriodicalId":14511,"journal":{"name":"Italian Journal of Pediatrics","volume":"51 1","pages":"80"},"PeriodicalIF":3.2,"publicationDate":"2025-03-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11909941/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143633951","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Amino acid variants in the HLA-DQA1 and HLA-DQB1 molecules explain the major association of variants with relapse status in pediatric patients with steroid-sensitive nephrotic syndrome.","authors":"Hui Yin, Sijie Yu, Xuelan Chen, Haiping Yang, Mo Wang, Qiu Li, Han Chan","doi":"10.1186/s13052-025-01913-z","DOIUrl":"10.1186/s13052-025-01913-z","url":null,"abstract":"<p><strong>Background: </strong>Management of patients with steroid-sensitive nephrotic syndrome (SSNS) is challenging because of frequent relapses. Causal variants in the human leukocyte antigen (HLA) class II region that are associated with relapse remain undetermined.</p><p><strong>Methods: </strong>We collected a cohort of East Asian individuals comprising 206 pediatric patients with SSNS and 435 healthy controls from Southwest China. Ninety children with steroid-sensitive nephrotic syndrome without relapse (SSNSWR) and 116 children with steroid-dependent and/or frequent relapse nephrotic syndrome (SDNS/FRNS) were genotyped using Sanger sequencing. We then measured the transcriptional level, allele expression imbalance (AEI) and functional proteins of HLA-DQA1 and HLA-DQB1 in different stages of SDNS/FRNS.</p><p><strong>Results: </strong>rs1464545187 in ANKRD36 was associated with an approximately 1.69-fold greater risk for SSNSWR (P = 0.04; 95% confidence interval [CI], 1.05-2.72). Clustered risk variants in HLA-DQA1 and HLA-DQB1 were significantly associated with SDNS/FRNS (rs1047989: P = 2.26E-07, odds ratio [OR] = 2.25, 1.65-3.05; rs9273471: P = 5.45E-05, OR = 1.84, 1.37-2.46; HLA-DQB1*06:02: P = 0.017, OR = 0.19, 0.04-0.77). The genotype distributions of rs1047989, 2:171713702, rs1049123, rs9273471, and HLA-DQB1*06:02 in patients with SSNS were significantly different from those in healthy controls. rs1047989 (HLA-DQA1) was significantly associated with a greater number of infections at relapse in SDNS/FRNS patients (P = 0.045, OR = 6.79, 95% CI: 1.29-168.52). Flow cytometry showed that the proportion of cells expressing HLA-DQA1⁺/DQB1⁺ (HLA-DQA1⁺, P = 0.0046; HLA-DQB1⁺, P = 0.0045) was lowest in the relapse stage. In addition, the mRNA levels of HLA-DQA1 and HLA-DQB1 were significantly greater in the relapse group than in the remission group (HLA-DQA1, P = 0.03; HLA-DQB1, P = 0.002). No significant AEIs were detected in the different stages of SDNS/FRNS. The rs1047989 variant is likely to affect the structure and stability of HLA-DQA1.</p><p><strong>Conclusion: </strong>rs1464545187 is a risk locus for SSNSWR but not SDNS/FRNS in Chinese children. Functional variations in HLA-DQA1 and HLA-DQB1 are implicated in regulating the immune response of SSNS patients, which may explain the typical triggering of SDNS/FRNS onset by infections.</p>","PeriodicalId":14511,"journal":{"name":"Italian Journal of Pediatrics","volume":"51 1","pages":"79"},"PeriodicalIF":3.2,"publicationDate":"2025-03-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11909919/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143633950","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Unmet needs of Italian centers for pediatric diabetes care: analysis of a survey among pediatric diabetologists facing the national screening program for Type 1 Diabetes.","authors":"Marco Marigliano, Roberto Franceschi, Enza Mozzillo, Valentina Tiberi, Monica Marino, Giada Boccolini, Malgorzata Wasniewska, Maria Elizabeth Street, Maria Rosaria Licenziati, Riccardo Bonfanti, Felice Citriniti, Giuseppe D'Annunzio, Maria Carolina Salerno, Valentino Cherubini","doi":"10.1186/s13052-025-01854-7","DOIUrl":"10.1186/s13052-025-01854-7","url":null,"abstract":"<p><strong>Backgrounds: </strong>The incidence of Type 1 Diabetes (T1D) in children and adolescents is increasing by 3-4% per year. Children and adolescents with T1D (CwD) should receive person-centered, specialized treatment from a multidisciplinary team to ensure appropriate care. Italy is the first to implement a countrywide T1D screening program, which will raise the need for funding for specialized pediatric care. The study aims to update the organization of the Italian Centers for pediatric diabetes care.</p><p><strong>Methods: </strong>In 2022, members of the 59 Italian Centers following CwD were invited to complete an email survey regarding the Centers' organization, characteristics, and activities. The questionnaire included information on responders, department organization, team composition, activities, and the organizational structures: department, ambulatory care services (AC), simple operational units (UOS), simple departmental operational units (UOSd), and complex operational units (UOC).</p><p><strong>Results: </strong>The data collected referred to the year 2022. According to the results, 21,318 people with diabetes were treated. Of these, 19,643 subjects (92.1%) have T1D (16,672 were CwD), 387 (1,8%) have Type 2 Diabetes, and 1,288 (6,1%) have other forms of diabetes. Compared to the 2012 survey, a 13% decrease (from 68 to 59 Centers) in the number of pediatric Centers caring for CwD was observed with a parallel increase of total (+ 6.6%) and average (+ 22%) number of CwD per Center. The estimated prevalence of T1D has increased (1.4 vs. 1.7 per 1,000 CwD-2012 vs. 2022). A reduction in numbers for AC (-22%) and UOS (-35%) was observed, whereas UOSd/UOC increased by 50%. Almost 35% of the dietitians and 40% of the psychologists were not permanent members of the multidisciplinary diabetes team.</p><p><strong>Conclusions: </strong>The observed decrease in the overall number of pediatric diabetes Centers, the reduction in specialized and dedicated HCPs, and the concurrent increase in the number of treated CwD in the last ten years indicate an alarming situation for pediatric diabetes treatment in Italy. Furthermore, the projected rise in CwD due to the National T1D screening program emphasizes the need for increased resources for specialized pediatric care of CwD at all stages.</p>","PeriodicalId":14511,"journal":{"name":"Italian Journal of Pediatrics","volume":"51 1","pages":"77"},"PeriodicalIF":3.2,"publicationDate":"2025-03-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11907787/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143623798","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Analysis of factors affecting long-term quality of life in children on home mechanical ventilation: a 4-year prospective observational cohort study.","authors":"Yingying Zhang, Yan Qin, Pan Liu, Yuxin Liu, Zhengzheng Zhang, Yun Jin, Guoping Lu, Jing Hu, Weiming Chen","doi":"10.1186/s13052-025-01920-0","DOIUrl":"10.1186/s13052-025-01920-0","url":null,"abstract":"<p><strong>Background: </strong>Home mechanical ventilation improves survival of critically ill children but partially affects quality of life. Studies in China have more often analyzed the risk factors for death from prolonged mechanical ventilation in hospitalized children while less attention has been paid to children with home mechanical ventilation. This study aimed to describe the quality of life of children with home mechanical ventilation and the influencing factors.</p><p><strong>Methods: </strong>It was a prospective cohort study. The cohort population was children undergoing prolonged mechanical ventilation in the Pediatric Intensive Care Unit, with the outcome of whether they were alive or dead at 1-year follow-up after discharge. Standardized scores for quality of life were calculated using TNO-AZL Children's Health-Related Quality of Life and TNO-AZL Preschool Children Quality of Life. Multiple linear regression was used to analyze the factors affecting the quality of life.</p><p><strong>Results: </strong>A total of 106 children were included, and 11 children (10.38%) died within one year after discharge. The mean age was 8.26 ± 4.10 years, and the hospitalization days was 68.46 ± 34.23. Child self-care had a significant effect on the risk of death one year after discharge, with higher Barthel self-care scores associated with a lower risk of death. There was a statistically significant difference in quality-of-life scores between the non-home and home mechanical ventilation groups, whereas tracheotomy or not had no effect. 81.57% of the surviving children with home mechanical ventilation were placed on invasive mechanical ventilation, with a mean ventilation duration of 19.94 h/d and a pressure-controlled mode primarily. Home invasive mechanical ventilation, age, and Barthel self-care score were independent influences on children's quality of life scores.</p><p><strong>Conclusion: </strong>The long-term survival rate of children who transitioned to home mechanical ventilation in Shanghai, China, was higher than the international average. Most children were discharged to home invasive mechanical ventilation, decreasing their quality of life. It needs to continue standardizing the post-discharge management procedures and explore how to better transition to home non-invasive mechanical ventilation. It's necessary to describe the parents' quality of life with home mechanical ventilated children and its impact on child outcomes.</p>","PeriodicalId":14511,"journal":{"name":"Italian Journal of Pediatrics","volume":"51 1","pages":"78"},"PeriodicalIF":3.2,"publicationDate":"2025-03-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11908011/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143624810","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}