Clinical and Experimental Pediatrics最新文献

Background: Diverse factors including seizure onset age, seizure frequency, epilepsy duration, total number of antiseizure medications trialed are considered as seizures-related neurocognitive loads in children with drug-resistant focal epilepsy (DRE). However, their associations with the structural integrity of neurocognitive networks remain largely unknown.

Purpose: This study investigates a novel diffusion-weighted imaging (DWI) connectome methodology that can extract seizure-associated structural abnormality biomarkers from clinical DWI tractography, use them to classify neurocognitive impairments prior to surgery, and unveil the relationship between epilepsy-related factors and neurocognitive impairments.

Methods: Thirty-three DRE children (age: 11.8±3.3 years, 17 boys) and 29 age-matched healthy controls were enrolled to create seizure-affected networks whose edges connect epileptogenic regions to key brain regions of 6 neurocognitive networks. The deviations of local efficiency values were averaged across the seizure-affected brain regions and used as new imaging-based biomarkers quantifying the degrees of seizure-associated structural abnormalities accumulated on individual neurocognitive networks and classifying the neurocognitive impairments along with the epilepsy-related factors.

Results: Effect sizes of the proposed biomarkers for differentiating DRE from healthy controls were consistently very large across various subgroups defined by lesion types, lobar locations of epileptogenic foci, seizure frequency categories, and seizure types (i.e., Cohen d value >1.8). Compared with the epilepsy-related factors, the proposed biomarkers demonstrated superior classification accuracy for identifying neurocognitive impairments in general, verbal, and nonverbal domains. When combined with the epilepsy-related factors, the classification performance further improved, achieving an accuracy range of 90%-98% in the independent test patients. The subsequent association analysis using the proposed biomarkers as seizure-associated structural abnormality indicators demonstrated that the inclusion of such imaging indicators significantly enhances the strength of associations between epilepsy factors and neurocognitive impairments.

Conclusion: These findings offer strong potential for objectively identifying neurocognitive impairments in DRE children, supporting early, data-driven decisions for personalized interventions to mitigate long-term effects.

Background: Hematopoietic stem cell transplantation (HSCT) is a curative treatment for patients with transfusion-dependent thalassemia (TDT), and the outcomes are influenced by multiple factors.

Purpose: We retrospectively evaluated the clinical characteristics, risk factors, complications, and treatment outcomes in Thai patients aged <20 years using 30-year multicenter HSCT data. This study sought to evaluate the contributing factors affecting survival outcomes and complications, provide insights into the evolution of HSCT for TDT, and inform practice guidelines in developing countries.

Methods: The outcomes of 266 HSCT procedures from related and unrelated donors in 249 Thai patients with TDT performed from 1988 to 2016 (median follow-up, 102 months) were analyzed.

Results: The median age at HSCT was 6.9 years (range, 1-19 years). Most HSCT procedures used human leukocyte antigen-matched related donors (MRDs; 71.8%), with bone marrow serving as the primary graft source (69.5%). The thalassemia recurrence rate was 11.6%, whereas the mortality rate was 9.0%, primarily due to Gram-negative sepsis. The 5-year overall (OS) and event-free survival (EFS) were 91.3% and 81.0%, respectively. The outcomes did not differ significantly between MRDs and matched unrelated donors (MUDs: OS rate, 91.5% vs. 88.0%, P=0.52; EFS rate, 82.0% vs. 76.2%, P=0.45). Since 2000, advances in pre-HSCT transfusion, iron chelation, graft-versus-host disease prophylaxis, and supportive care have been implemented, with intravenous busulfan adopted after 2009. Over three periods (1988-1999, 2000-2009, and 2010-2016), the OS rate rose from 89.4% to 93.0% (P=0.74), and the EFS rate rose from 67.7% to 87.2% (P=0.01). Age ≤10 years was associated with better overall OS and EFS, although significance was limited to the earliest period. A multivariate analysis identified a pre-HSCT ferritin level >2,500 ng/mL, low CD34+ doses, and the use of oral busulfan conditioning as factors associated with unfavorable survival. Long-term complications, primarily endocrine disorders, affected 22.7% of survivors.

Conclusion: Our results broaden the donor pool by demonstrating comparable outcomes between MRD and MUD transplantation. Optimizing pretransplant care, such as regular pre-HSCT transfusion, adjusting conditioning intensity, and improving posttransplant supportive care, may mitigate age-related risks in older recipients.

Shared decision-making (SDM), which has become a core principle of patient-clinician communication, emphasizes respect for autonomy, partnership, and transparency. In neonatal practice-where decisions often carry profound ethical and emotional weight-SDM helps align medical recommendations with the family's values, fosters trust, and may reduce moral distress for both parents and clinicians. However, in Korea, the systematic recognition and formalization of SDM remains limited, underscoring the need for practical guidance and institutional support in neonatal and pediatric care. This review discusses key principles of SDM and illustrates their application in real clinical communication through hypothetical neonatal case vignettes. SDM is best understood not as a single model but as a continuum shaped by the balance between medical judgment and parental values. Drawing on Opel's stepwise framework, SDM is most appropriate when more than one medically reasonable option exists and value considerations meaningfully influence the choice. Physicians must also remain aware of personal biases that can shape how options are framed and discussed. Building on this conceptual foundation, this paper describes how meaningful SDM can be practiced using the 4 conversational phases outlined by Madrigal and Kelly: (1) acknowledging the need for a decision and inviting participation; (2) sharing information while listening; (3) uncovering values, hopes, and fears; and (4) reaching a balanced and ethically grounded decision. Through clinical examples, these phases demonstrate how empathy, pacing, and reflective dialogue can transform information exchange into shared moral reasoning. Finally, structural and cultural barriers continue to hinder the implementation of SDM in Korea, including time constraints, limited training, and restrictive legal frameworks. Policy reform, education, and team-based strategies are essential to support its broader adoption. SDM ultimately offers an ethically robust and relational framework for navigating complex, value-laden decisions in neonatal and pediatric care that is grounded in communication that recognizes bias and engages families as true partners.

Background: Malignant vasovagal syncope (VVS) is characterized by cardiac arrest lasting more than 3 seconds during a syncope episode or head-up tilt test (HUTT). We aim to conduct a risk assessment for potential malignant VVS before the HUTT by using economic, simple and convenient demographic data, in order to prevent adverse outcomes for pediatric VVS.

Purpose: To explore the correlation between demographic factors and pediatric malignant VVS, and verify the value of these factors in early risk assessment for malignant VVS before HUTT, so as to optimize test safety and reduce adverse events.

Methods: We conducted a retrospective analysis of the clinical data of 3,734 children who were initially diagnosed with VVS due to unexplained syncope and presyncope. Finally, 122 children who met the diagnostic criteria for malignant VVS were included in the malignant VVS group, and 661 children who did not meet the criteria during the same period were matched as the control group. By analyzing demographic data and other factors, we attempted to clarify the association between these factors and malignant VVS.

Results: Linear relationship: age and body mass index (BMI) have independent protective effects on malignant VVS. For every 1-year increase in age and every 1 kg/m2 increase in BMI, the risk of malignant VVS decreases by 12% and 9%, respectively. Nonlinear relationship: When the age is <12.9 years old, for every additional year of age, the risk of malignant VVS decreases by 20%. For ages 12.9 years and above, the efficacy is not significant. There is no significant nonlinear relationship between BMI and malignant VVS.

Conclusion: Age and BMI are independent protective factors for pediatric malignant VVS. Before the age of 12.9 years, the incidence of malignant VVS gradually decreases with the increase in age, and thereafter there is no significant impact.

Background: The survival rate of pediatric acute lymphoblastic leukemia (ALL) currently exceeds 90% in high-income settings, shifting the focus to its long-term effects. Kidney injury, acute kidney injury (AKI), and chronic kidney disease (CKD) are increasingly recognized associated conditions; however, the determinants of CKD in pediatric ALL remain poorly defined.

Purpose: To quantify the burden of AKI during induction and CKD in children with ALL, estimate CKD-free survival, and identify clinical predictors of CKD.

Methods: This retrospective cohort at a single university-affiliated tertiary center included patients aged 2-18 years with ALL who completed ≥3 months of follow-up. AKI was classified by Kidney Disease: Improving Global Outcomes serum-creatinine criteria, while CKD was defined as a glomerular filtration rate <90 mL/min/1.73 m² for ≥3 months. CKD-free survival was estimated using the Kaplan-Meier method. Associations with time to CKD were assessed using the Cox proportional hazards model.

Results: Of 113 children (median age, 5.6; interquartile range [IQR], 3.8-9.4 years), AKI occurred during induction in 49 (43.4%). Leukemic kidney infiltration (LKI) was more frequently noted in patients with versus without AKI (P=0.01). Over 644 patient-years of follow-up (median, 5.1; IQR, 2.9-8.3 years), 15 (13.3%) developed CKD (stage 2 [n=12], stage 3 [n=3]). The 1-, 3-, and 5-year CKD-free survival rates were 99.1%, 95.3%, and 94.1%, respectively. In multivariate models, age was independently associated with CKD (adjusted hazard ratio [aHR], 1.28 per year; 95% confidence interval [CI], 1.04-1.57; P=0.02), whereas the incidence of LKI did not reach significance (aHR, 2.93; 95% CI, 0.87-9.89; P=0.08).

Conclusion: AKI commonly developed during induction. An older age at diagnosis was the principal independent predictor of CKD development. The age effect demonstrated a linear risk gradient rather than a conventional dichotomous ≥10-year threshold. A LKI was associated with AKI and suggestive of subsequent CKD. These results suggest that older children may benefit from intensive kidney surveillance and supportive care. Multicenter prospective studies are warranted to refine the prevention strategies.

Background: Febrile neutropenia (FN) remains a leading cause of morbidity and mortality in pediatric patients with high-risk hematologic disorders, particularly in low- and middle-income countries (LMICs), where antimicrobial resistance limits treatment options. Granulocyte transfusion (GT) is considered an adjunctive therapy; however, pediatric data from LMICs are limited.

Purpose: This study evaluates the effectiveness, timing, and safety of GT in a real-world LMIC setting.

Methods: This 15-year retrospective cohort study included pediatric patients (≤18 years) with severe neutropenic infections treated at a national tertiary referral center in Thailand in 2009-2023. The patients received GT plus antimicrobial therapy or antimicrobial therapy alone. The primary outcome measure was 30-day survival. The analyses included multivariate logistic regression, Cox regression, propensity score matching (PSM), and inverse probability of treatment weighting (IPTW).

Results: Among the 54 patients (26 GT recipients; 28 controls), GT was associated with improved 30-day survival in the full cohort (odds ratio [OR], 0.105; 95% confidence interval [CI], 0.016-0.700; P=0.020). IPTW confirmed this association (OR, 0.099; P=0.001), with consistent results in the PSM analysis (OR, 0.157; P=0.028). In the high-risk hematologic condition subgroup (n=48), GT was associated with increased survival (95.2% vs. 61.9%; hazard ratio [HR], 0.105; P=0.034). GT also accelerated the resolution of fever (HR, 2.24; P=0.028), FN recovery (HR, 2.35; P=0.017), and absolute neutrophil count recovery (HR, 2.10; P=0.047). No serious transfusion-related adverse events were observed.

Conclusion: GT was associated with improved survival and faster clinical recovery in pediatric patients with FN. These real-world LMIC data support its use as a feasible adjunctive therapy and warrant prospective validation.

Background: Bronchopulmonary dysplasia (BPD) is the most prevalent chronic lung disease in very preterm infants; however, conventional classifications have limited ability to predict severity before 36 weeks' postmenstrual age (PMA). A new Japanese classification, based on small for gestational age (SGA), bubbly/cystic chest radiographic findings, and chorioamnionitis (CAM), was proposed to enable earlier risk stratification. However, its validation in homogeneous cohorts is warranted.

Purpose: This study aimed to examine the association between this new Japanese classification and severe BPD development at 36 weeks' PMA in a secondary analysis of a randomized controlled trial (RCT).

Methods: A retrospective secondary analysis of a multicenter, double-blind RCT of inhaled corticosteroids in 12 tertiary neonatal intensive care units in Japan (2006-2009) was performed. Infants with a birth weight (BW)<1,000 g and requiring mechanical ventilation were enrolled. Of those 211 infants, 194 survivors were analyzed. Severe BPD was defined by National Institute of Child Health and Human Development criteria as requiring supplemental oxygen (fraction of inspired O2 >0.30) or positive pressure ventilation at 36 weeks' PMA. Logistic regression analyses were adjusted for gestational age, BW, sex, Apgar score, maternal steroid use, respiratory distress syndrome, and patent ductus arteriosus.

Results: Among the 194 infants, 25 were SGA, 45 had bubbly/cystic findings, and 86 had CAM. Severe BPD occurred in 80 infants. A multivariate analysis identified SGA (adjusted odds ratio [aOR], 3.32; 95% confidence interval [CI], 1.16-9.48; P=0.032) and bubbly/cystic findings (aOR, 10.88; 95% CI, 4.43-26.72; P<0.01) as independent risk factors. Compared with type II (non-CAM, no bubbly/cystic findings), type I (bubbly/cystic only: aOR, 6.21; 95% CI, 1.93-14.36) and type III (CAM plus bubbly/cystic: aOR, 15.32; 95% CI, 2.48-46.32) were significantly associated with severe BPD.

Conclusion: The new Japanese classification demonstrated that SGA and bubbly/cystic findings at day 28 independently predicted severe BPD. Early stratification using this classification may facilitate the early identification of high-risk infants for targeted interventions.

Background: Thrombocytopenia in preterm infants born to mothers with systemic lupus erythematosus (SLE) is poorly characterized, despite its potential link to adverse outcomes. Our understanding of platelet dynamics, risk factors, and clinical outcomes in this population is limited, necessitating further investigation.

Purpose: This study aimed to characterize the incidence, timing, and severity of thrombocytopenia in this population; to identify associated maternal and neonatal risk factors; and to evaluate its association with adverse outcomes.

Methods: We included 154 preterm infants born to mothers with SLE who were admitted to Shanghai Children's Medical Center within 24 hours of birth between 2014 and 2024. Logistic regression was used to identify risk factors and outcomes associated with neonatal thrombocytopenia.

Results: Thrombocytopenia (platelet count < 150 ×109/L) occurred in 32.5% of infants, and severe form (< 50 ×109/L) occurred in 4.6%. The condition peaked on postnatal days 4-5, and 16% of affected infants required intervention. Late preterm infants (adjusted odds ratio [aOR], 0.15; 95% confidence interval [CI], 0.05-0.42), moderate preterm infants (aOR, 0.26; 95% CI, 0.09-0.76), and maternal hydroxychloroquine use (aOR, 0.19; 95% CI, 0.07-0.52) were protective factors. In contrast, maternal hypertensive disorders of pregnancy (HDP; aOR, 3.41; 95% CI, 1.06-10.93) increased the risk. Infants with thrombocytopenia had significantly higher risks of intracranial hemorrhage (aOR, 4.27; 95% CI, 1.65-11.00) and late-onset sepsis (aOR, 11.00; 95% CI, 1.23-98.14).

Conclusion: Preterm infants exposed to maternal SLE frequently developed thrombocytopenia, but most cases were self-limited. Key risk modulators included gestational age, maternal HDP, and hydroxychloroquine use. Furthermore, thrombocytopenia was significantly associated with neonatal morbidity.