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Povzetek: Background The majority of children with primary immune thrombocytopenia (ITP) follow a benign, self-limiting course. However, early identification of children at risk for chronic disease, timely recognition of alternative diagnoses that may mimic ITP at onset, and selection of patients for observation alone remain important clinical challenges. Methods This retrospective single-center study included patients aged ≤18 years who were evaluated for ITP between 2009 and 2024. Demographic, clinical, laboratory, and therapeutic data were collected at diagnosis and during follow-up. Factors associated with chronic ITP were assessed using logistic regression, providing odds ratios with 95% confidence intervals. Kaplan-Meier analysis was used to describe time to first bleeding complication according to initial management. Results A total of 271 patients were included (43.5% female; median age 4 years). Of these, 240 were ultimately diagnosed with ITP, while 31 received an alternative final diagnosis. In multivariate logistic regression, chronic ITP was independently associated with older age (OR 1.08; p=0.017), absence of a preceding infection or vaccination (OR 0.45; p=0.029), and higher platelet count at diagnosis (OR 1.03, p<0.0001). Compared with patients with final ITP, those with alternative causes of thrombocytopenia were older (p=0.034), had lower bleeding grades (p=0.037), higher MCV, and lower total leukocyte, lymphocyte, monocyte, and eosinophil counts at onset (all p<0.05). Patients receiving upfront pharmacological therapy were younger and presented with higher bleeding grades and lower platelet counts than those managed with observation. Bleeding complications were more frequent with upfront pharmacological therapy, whereas those in the watch-and-wait group tended to be more severe. Conclusion Our results suggest that readily available data may help identify children at risk for chronic disease and those whose thrombocytopenia is attributable to a cause other than ITP. Initial treatment decisions appeared to be driven primarily by clinical presentation.
Ključne besede: Alternative diagnoses, chronicity, complications, immune thrombocytopenia, pediatrics, watch- and-wait management
Objavljeno v DiRROS: 23.07.2026; Ogledov: 276; Prenosov: 157
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Povzetek: Autism spectrum disorder (ASD) has been associated with alterations in the gut microbiota and its metabolites, particularly short-chain fatty acids (SCFAs) and microbiota-derived tryptophan catabolites, which may influence neurodevelopment through immune and epigenetic mechanisms. We investigated whether stool SCFAs and tryptophan-pathway metabolites differ between children with ASD and typically developing controls, and whether these metabolites associate with ASD severity and systemic biochemical signatures. In this cross-sectional study, we analyzed stool samples from 229 children (160 with ASD, 69 controls) with complete SCFA and tryptophan-metabolite data, while urine metabolomics data were available for a subset and were used for exploratory stool–urine integration analyses. Children with ASD and controls were similar in age, but the ASD group had a higher proportion of males. Absolute concentrations of individual SCFAs, total SCFAs, and derived indices were broadly comparable between groups; nominal differences in propionate/acetate ratio and caproate did not remain significant after false discovery rate correction. Similarly, stool tryptophan-pathway metabolites reported as ng/a.u. based on the NanoDrop-derived proxy (tryptophan, kynurenine, indole-3-acetic, indole-3-lactic, indole-3-propionic, indole-3-aldehyde, N-acetyl-tryptophan, serotonin, melatonin, tryptamine) and functional ratios (kynurenine/tryptophan, indole-derived/tryptophan, serotonin/tryptophan) showed no robust ASD–control differences; N-acetyl-tryptophan was nominally higher in ASD but did not survive multiple-testing correction. In the ASD subgroup with available Childhood Autism Rating Scale (CARS) data (n = 34), SCFA and tryptophan indices showed only weak, non-significant correlations with global ASD severity. In contrast, correlation analyses revealed two coherent metabolic modules, i.e., an SCFA block with very strong internal correlations among individual SCFAs and total SCFAs and a tryptophan block with strong correlations between metabolites and their normalized ratios, while cross-module correlations were modest. These results indicate that stool SCFA and microbiota-derived tryptophan profiles do not robustly distinguish ASD from controls in this cohort, but they form stable metabolic modules compatible with microbiome–epigenome frameworks.
Ključne besede: autism spectrum disorder, gut microbiota, short-chain fatty acids, tryptophan metabolism, indole metabolites, kynurenine pathway, epigenetics, microbiome–epigenome interaction, metabolomics, pediatrics
Objavljeno v DiRROS: 05.05.2026; Ogledov: 370; Prenosov: 279
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Povzetek: Context: GH treatment in children with idiopathic short stature (ISS) can be controversial, and analyses comparing responses to children with GH deficiency (GHD) are limited. Objective: To compare the effectiveness and safety of GH treatment in children with ISS and GHD, including those reaching near adult height (NAH). Methods: This post hoc analysis of the NordiNet International Outcome Study (2006-2016) and the American Norditropin Studies: Web-Enabled Research Program (2002-2016) included children with ISS or GHD who initiated treatment aged <18 years. The safety analysis set had birthdate and GH exposure information. The effectiveness analysis set was GH-naïve with valid baseline information. GH exposure, effectiveness, and safety outcomes were analyzed annually for ≤10 years. Results: The safety analysis set included 3816 children with ISS and 22 858 with GHD. The effectiveness analysis set comprised 18 405 children (ISS: 2684; GHD: 15 721), 1856 of whom reached NAH (ISS: 230; GHD: 1626). Average dose of GH was higher for children with ISS vs children with GHD but mean duration of treatment was shorter. At NAH, height SD score (mean [SD]) was −1.21 (1.09) and −0.90 (1.20) for children with ISS and GHD, respectively, whereas change in height SD score (mean [SD]) from baseline to 10 years was 1.21 (0.86) and 1.45 (1.09). Incidence of adverse reactions was similar across indications, with no new safety signals. Conclusion: GH treatment over 5 to 10 years effectively increased height in children with ISS and children with GHD, including those who reached NAH, with a favorable benefit-risk profile.
Ključne besede: comparative outcomes, growth hormon treatment, growth hormon deficiency, pediatrics
Objavljeno v DiRROS: 31.03.2026; Ogledov: 516; Prenosov: 341
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Povzetek: Image-guided percutaneous abscess drainage (IPAD) is an effective, minimally invasive technique to manage infected abdominal fluid collections in children. It is the treatment of choice in cases where surgery is not immediately required due to another coexisting indication. The skills and equipment needed for this procedure are widely available. IPAD is typically guided by ultrasound, fluoroscopy, computed tomography, or a combination thereof. Abscesses in hard-to-reach locations can be drained by intercostal, transhepatic, transgluteal, transrectal, or transvaginal approaches. Pediatric IPAD has a success rate of over 80% and a low complication rate.
Ključne besede: appendicitis, children, pediatrics, Seldinger technique, percutaneous abscess drainage
Objavljeno v DiRROS: 13.01.2026; Ogledov: 651; Prenosov: 413
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Povzetek: Despite widely and regularly used therapy asthma in children is not fully controlled. Recognizing the complexity of asthma phenotypes and endotypes imposed the concept of precision medicine in asthma treatment. By applying machine learning algorithms assessed with respect to their accuracy in predicting treatment outcome, we have successfully identified 4 distinct clusters in a pediatric asthma cohort with specific treatment outcome patterns according to changes in lung function (FEV1 and MEF50), airway inflammation (FENO) and disease control likely affected by discrete phenotypes at initial disease presentation, differing in the type and level of inflammation, age of onset, comorbidities, certain genetic and other physiologic traits. The smallest and the largest of the 4 clusters- 1 (N = 58) and 3 (N = 138) had better treatment outcomes compared to clusters 2 and 4 and were characterized by more prominent atopic markers and a predominant allelic (A allele) effect for rs37973 in the GLCCI1 gene previously associated with positive treatment outcomes in asthmatics. These patients also had a relatively later onset of disease (6 + yrs). Clusters 2 (N = 87) and 4 (N = 64) had poorer treatment success, but varied in the type of inflammation (predominantly neutrophilic for cluster 4 and likely mixed-type for cluster 2), comorbidities (obesity for cluster 2), level of systemic inflammation (highest hsCRP for cluster 2) and platelet count (lowest for cluster 4). The results of this study emphasize the issues in asthma management due to the overgeneralized approach to the disease, not taking into account specific disease phenotypes.
Ključne besede: asthma, allergy and immunology, pediatrics, machine learning, treatment outcome, phenotypes, childhood asthma, clustering
Objavljeno v DiRROS: 16.08.2021; Ogledov: 2511; Prenosov: 1673
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