Function In Pediatric Patients Research Articles

Efficacy of Nusinersen Treatment in Type 1, 2, and 3 Spinal Muscular Atrophy: Real-World Data from a Single-Center Study

Background: Spinal muscular atrophy (SMA) is an inherited neuromuscular disease characterized by progressive muscle weakness and atrophy due to the absence of the survival motor neuron 1 (SMN1) gene. SMA is classified into types 0 through 4 based on the age of symptom onset and the severity of motor function decline. Recent advances in SMA treatment, including nusinersen, onasemnogene abeparvovec, and risdiplam, have significantly improved the prognosis of SMA patients. This study evaluated the safety and efficacy of nusinersen in pediatric patients with SMA types 1, 2, and 3 in a real-world clinical setting. Methods: This prospective observational single-center study assessed the treatment effects of nusinersen in 23 pediatric patients with genetically confirmed SMA over a 22-month observation period. All the participants received intrathecal loading doses of 12 mg of nusinersen on days 1, 14, 28, and 63, followed by maintenance doses every four months. Functional assessments were conducted using the CHOP-INTEND scale. Data were collected during routine patient visits, including clinical laboratory tests and vital sign parameters, and adverse events were recorded. The inclusion criteria were defined by the national reimbursement program for nusinersen treatment in Poland. Results: Initially, 37 patients ranging from 1 month old to 18 years old were included, but 23 were ultimately observed due to changes in treatment regimens or assessment scales. The patients showed significantly improved CHOP-INTEND scores over the 22-month period. At 6 months, the average increase was 4.2 points, continuing to 17.8 points at 22 months. By the end of the study, 100% of patients showed either stabilization or improvement, with significant clinical improvements observed in several patients. Nusinersen was generally well-tolerated, with post-lumbar puncture headache and lower back pain being the most common adverse events. Conclusions: Nusinersen treatment significantly enhances motor function in pediatric patients with SMA types 1, 2, and 3. This study demonstrates the importance of early and sustained treatment, with most patients showing the continuous improvement or stabilization of motor function. These findings support the use of nusinersen as an effective therapy for SMA; however, further research is needed to understand the long-term outcomes and optimize treatment strategies.

The Effects of Sedation on Auditory Brainstem Response in Pediatric Population: A Systematic Review

Background and Aim: Auditory Brainstem Response (ABR) is crucial for evaluating auditory pathway function in pediatric patients. However, obtaining reliable ABR measurements in young children can be challenging due to their inability to stay still and quiet during the test. Sedation is frequently used to facilitate ABR testing in this population, but the most practically effective sedation method and its impact are still uncertain. This systematic review aimed to thoroughly investigate the success rates (completion of ABR in both ears) associated with different sedation techniques. Recent Findings: In recent review (2000-2022), Ten studies, identified through a comprehensive search of electronic databases, were included in the analysis. The studies reported significant variation in success rates for ABR testing with sedation, ranging from 70% to 100%. This suggests that the effectiveness of sedation may not be uniform across all situations. The included studies employed a wide variety of sedation techniques, highlighting the lack of a standardized approach in this area. Conclusion: These findings highlight the heterogeneity in sedation practices and success rates for pediatric ABR testing. This emphasizes the importance of tailoring the sedation approach to the specific needs of each child while carefully considering the potential risks associated with each sedation method. Further research is warranted to establish standardized protocols for sedation in pediatric ABR , ensuring optimal test efficacy while prioritizing patient safety. Research Square registration.Keywords: Auditory brainstem response; sedation; chloral hydrate; children; dexmedetomidine; intranasal drug administration

Changes in echocardiographic parameters after hemodialysis session in a North African pediatric population with end-stage renal disease and without known heart disease.

Children undergoing long-term hemodialysis (HD) face a reduction in life expectancy mostly due to cardiovascular mortality. Effects of HD on cardiac function have not been fully elucidated in pediatric population. This study aimed to assess HD session impact on cardiac function in pediatric patients using conventional and strain echocardiography. We performed a prospective, comparative study of echocardiographic parameters before and after single HD session in a chronic HD pediatric population. We enrolled between the 1st and 30th September 2023, all consecutive patients with end-stage renal disease (ESRD) aged up to 18 years old on maintenance HD three times weekly for at least three months. All patients underwent conventional and left ventricular (LV) longitudinal strain echocardiography in a window of 30-60 minutes before and after HD. 23 patients, 14.8 ± 2.1 years old and 47.8% male, were enrolled. Reductions in body weight and blood pressure were observed after HD, whereas heart rate increased. Significant decrease in LV and left atrial diameters and volumes after HD session were observed. Mitral peak E velocity, as well as average E/e' were significantly lower after HD. Although LV ejection fraction was unchanged, global longitudinal strain for LV was significantly reduced after dialysis (-17.3 ± 3.0% vs. -14.9 ± 2.4%, p=4.10-8). Patent deterioration in LV systolic function following HD was identified by speckle tracking echocardiography (STE). STE has the potential to unmask early myocardial dysfunction even when there is no evident alteration in conventional systolic function parameters in children with ESRD.

Outcomes of Hip Shelf Procedure in Treating Legg-Calvé-Perthes Disease

Introduction: Legg-Calvé-Perthes disease (LCPD) is a pediatric ischemic condition of the femoral head leading to necrosis. This study evaluates the hip shelf procedure's effectiveness in treating LCPD and the resulting clinical outcomes. Methods: A retrospective analysis of 17 patients treated with the hip shelf procedure at the Pediatric Orthopedics and Traumatology Department of CHU Hassan II, Fes, over ten years. Data on patient demographics, clinical presentations, radiological assessments, and surgical outcomes were analyzed. Results: The average age at consultation was 9 years, with a male predominance (88.2%). Radiological staging showed most patients in the fragmentation or reconstruction stages. Post-operative results indicated significant improvement in hip joint stability and remodeling. Conclusion: The hip shelf procedure is effective in managing LCPD, promoting favorable hip joint remodeling and function in pediatric patients. Early intervention is crucial for optimal outcomes.

Impact of general anesthesia on the echocardiographic assessment of right ventricular function in pediatric patients with pulmonary arterial hypertension.

One of the great diagnostic challenges for children with pulmonary arterial hypertension is the need for general anesthesia (GA) to enable successful right heart catheterization. Here, for the first time, we describe how echocardiographic estimates of right ventricular function and pulmonary pressures change in pediatric patients during GA.

SPA inhibits hBMSC osteogenic differentiation and M1 macrophage polarization by suppressing SETD2 in acute suppurative osteomyelitis

To clarify the impact of SETD2 on macrophage function in pediatric patients with acute suppurative osteomyelitis and to elucidate the precise underlying mechanism. To gain insights into the potential functions of SETD2, a comprehensive study was conducted utilizing a co-culture model of human bone mesenchymal stem cells (hBMSCs) and bone marrow-derived macrophages (THP-1). A range of techniques were employed, including quantitative polymerase chain reaction, western blotting, ELISA, alkaline phosphatase activity assays, alizarin red S staining, luciferase reporter gene assays, and chromatin immunoprecipitation, to unravel the intricate interactions and molecular mechanisms involving SETD2 in this system. It was observed that SETD2 expression was reduced in THP-1 cells stimulated by staphylococcal protein A (SPA). Furthermore, the downregulation of SETD2 resulted in elevated M1 macrophage polarization and glycolysis, effects that were mitigated by SPA stimulation. Notably, SPA-stimulated THP-1 cells exhibited an increase in HIF-1α expression, which exhibited an inverse correlation with SETD2 levels. Moreover, it was discovered that SETD2 functioned as a catalyst for H3K36me3 and bound to the HIF-1α gene, which, in turn, regulated HIF-1α expression. Furthermore, the suppression of HIF-1α abrogated the consequences of SETD2 downregulation on glycolysis and M1 macrophage polarization. Lastly, the study demonstrated that M1 macrophage polarization serves as a mediator for BMP4’s inhibitory effect on osteogenic differentiation of hBMSCs. This research has uncovered a previously unknown role of SETD2 in macrophages during osteomyelitis, revealing its significance in the pathogenesis of this condition. These findings suggest SETD2 as a novel target for the treatment of osteomyelitis.

Therapeutic Role of Nusinersen on Respiratory Progression in Pediatric Patients With Spinal Muscular Atrophy Type 2 and Nonambulant Type 3.

Nusinersen has shown significant functional motor benefit in the milder types of spinal muscular atrophy (SMA). Less is known on the respiratory outcomes in patients with nusinersen-treated SMA. The aim of this study was to describe changes in respiratory function in pediatric patients with SMA type 2 and 3 on regular treatment with nusinersen within the iSMAc international cohort and to compare their trajectory with the natural history (NH) data published by the consortium in 2020. This is a 5-year retrospective observational study of pediatric SMA type 2 and nonambulant type 3 (age ≤18 years) treated with nusinersen. The primary objective was to compare the slopes of decline in forced vital capacity % predicted (FVC% pred.), FVC, and age when FVC dropped below 60% between the treated patients and a control group from the natural history cohort. Data on peak cough flow and the use of noninvasive ventilation (NIV) and cough assist were collected. Data were available for 69 treated patients, 53 were SMA type 2 and 16 type 3. The mean (SD) age at first injection was 8.5 (3.2) and 9.7 (3.7) years, respectively. The median (interquartile range) treatment duration was 1 (0.7; 1.9) and 1.2 (0.9; 1.9) years, respectively. At the time of the first nusinersen injection, 24 of 52 (46%) patients with SMA type 2 and 2 of 16 (13%) patients with SMA type 3 were on NIV. Forty-three of 53 (81%) and 4 of 16 (25%) patients used cough device. FVC% pred. in treated patients with SMA type 2 declined annually by 2.3% vs 3.9% in NH (p = 0.08) and in treated patients with type 3 by 2.6% vs 3.4% NH (p = 0.59). Patients treated reached FVC <60% later than untreated (12.1 vs 10 years, p = 0.05). A higher percentage of treated vs untreated patients maintained FVC% pred. equal/above their baseline after 12 (65% vs 36%) and 24 (50% vs 24%) months, respectively. NIV use among treated did not significantly change throughout 1-year follow-up. This study included the largest real-world cohort of pediatric patients with milder SMA types. The results suggest a positive role of nusinersen in delaying the respiratory decline in patients treated longer than 1 year when compared with natural history. Larger cohorts and longer observation are planned. This study provided Class III evidence that nusinersen slows progression for patients with SMA types 2 and 3 compared with a natural history cohort.

PCR26 Impact of Attention-Deficit/Hyperactivity Disorder Treatment Adverse Effects on Quality of Life and Functioning of Pediatric Patients and Their Parents/Caregivers

PCR26 Impact of Attention-Deficit/Hyperactivity Disorder Treatment Adverse Effects on Quality of Life and Functioning of Pediatric Patients and Their Parents/Caregivers

Comparison of cystatin C-based estimated glomerular filtration rate with measured glomerular filtration rate in a pediatric cohort of patients with chronic kidney disease.

It is essential to have an accurate assessment of the renal function of patients with chronic kidney disease to monitor, treat, and predict further development of the condition. Measurement of renal function in terms of glomerular filtration rate (GFR) requires either urine or blood sampling, but especially in children, more simple methods of measurement are preferable. The main objective of this study was to examine if the estimated GFR (eGFR) calculated with different cystatin-C-based equations was comparable to the GFR measured by a radiotracer (mGFR) in pediatric patients. In this retrospective study, 28 pediatric patients contributed with 73 pairs of measurements collected within 5 years. Bland-Altman Limits of Agreement were used to evaluate the performance and accuracy of two different cystatin-C-based estimates, the CKiDCrea-CysC and the CKiDU25 respectively, compared to an mGFR based on plasma clearance of technetium-99m-diethylenetriaminepentaacetic acid or chromium-51-ethylenediaminetetraacetic acid. Using the CKiDCrea-CysC equation, 58.9% of the datasets were within P10 and 87.7% were within P30. The mean difference was 4.8 mL/min/1.73m2 (standard deviation: 8.5 mL/min/1.73m2) and tended to overestimate GFR and thereby overrate the kidney function within the entire GFR range. Using the CKiDU25 equation, 53.4% were within P10 and 93.2% within P30. The mean difference was -2.9 mL/min/1.73m2 (standard deviation: 8.4 mL/min/1.73m2), but the difference varied with the GFR value. A cystatin-C-based eGFR provides a viable substitute for monitoring renal function in pediatric patients with chronic kidney disease. However, it has a lower accuracy than mGFR and can therefore not replace mGFR in clinical use.

A Pilot Comparative Study between Creatinine- and Cystatin-C-Based Equations to Estimate GFR and Kidney Ultrasound Percentiles in Children with Congenital Anomalies of the Kidney and Urinary Tract.

Congenital anomalies affecting the kidneys present significant challenges in pediatric nephrology, needing precise methods for assessing renal function and guiding therapeutic intervention. Bedside Schwartz formula with the cystatin-C-based Full Age Spectrum formula and Chronic Kidney Disease in Children (CKiD) U 25 formula used in estimating glomerular filtration rate (eGFR) and also to assess if the eGFR in association with kidney length percentiles can be a monitoring parameter for the progression of chronic kidney disease in children with congenital anomalies of the kidney and urinary tract (CAKUT). A total of 64 pediatric patients (median age at diagnostic was 12 months with an interquartile range of 2 to 60) were diagnosed with congenital anomalies in the kidney and urinary tract between June 2018 and May 2023 at "Louis Turcanu" Emergency Hospital for Children in Timisoara, Romania. Baseline characteristics, CAKUT types, associated pathologies, CKD staging, and eGFR using creatinine and cystatin C were analyzed. The mean age at the moment of examination was 116.50 months; (65, 180). Chronic kidney disease staging revealed a predominance of patients in CKD stages G1 and A1. Analysis of eGFR methods revealed a small mean difference between eGFR estimated by creatinine and cystatin C, with a moderate-strong positive correlation observed between the eGFR and ultrasound parameters. Using cystatin-C-based formulas for eGFR, in conjunction with ultrasound measurements, may offer reliable insights into renal function in pediatric patients with congenital anomalies affecting the kidney and urinary tract. However, the economic aspect must be taken into consideration because cystatin C determination is approximately eight times more expensive than that of creatinine. An interdisciplinary approach is crucial for managing patients with CAKUT.

Impact Of Pharmacist Led Therapeutic Drug Monitoring of Vancomycin in Pediatric Cancer Patients

Aim This study aimed to evaluate the dosing regimen and therapeutic drug monitoring of vancomycin in this patient population. Method The study included 100 pediatric patients (3 months to 15 years) with various gram-positive bacterial infections. All patients received the initial vancomycin dose of 15 mg/kg every 6 hours. The researchers evaluated the incidence of achieving desired trough levels with this dosing regimen. Results The results revealed that patients between 1.0 and 5.9 years of age were less likely to achieve the desired trough levels with the initial vancomycin dose. Consequently, dose adjustments were made for patients with sub-therapeutic trough levels. These adjustments involved a 25% increase in the vancomycin dose. Importantly, the study found that the higher vancomycin dosing did not have any harmful effects on kidney function in pediatric patients, and no significant adverse effects were observed. Conclusion The dosing regimen of 15 mg/kg every 6 hours was unlikely to achieve the desired trough concentrations in pediatric patients with complicated infections and oncological diseases. The study recommends dose adjustments for the treatment of different infections in this population to increase the likelihood of reaching therapeutic steady-state concentrations of vancomycin.

The impact of unilateral pulmonary artery stenosis on right ventricular to pulmonary arterial coupling in patients with transposition of the great arteries.

Unilateral pulmonary artery (PA) stenosis is common in the transposition of the great arteries (TGA) after arterial switch operation (ASO) but the effects on the right ventricle (RV) remain unclear. To assess the effects of unilateral PA stenosis on RV afterload and function in pediatric patients with TGA-ASO. In this retrospective study, eight TGA patients with unilateral PA stenosis underwent heart catheterization and cardiac magnetic resonance (CMR) imaging. RV pressures, RV afterload (arterial elastance [Ea]), PA compliance, RV contractility (end-systolic elastance [Ees]), RV-to-PA (RV-PA)coupling (Ees/Ea), and RV diastolic stiffness (end-diastolic elastance [Eed]) were analyzed and compared to normal values from the literature. In all TGA patients (mean age 12 ± 3 years), RV afterload (Ea) and RV pressures were increased whereas PA compliance was reduced. RV contractility (Ees) was decreased resulting in RV-PA uncoupling. RV diastolic stiffness (Eed) was increased. CMR-derivedRV volumes, mass, and ejection fraction were preserved. Unilateral PA stenosis results in an increased RV afterload in TGA patients after ASO. RV remodeling and function remain within normal limits when analyzed by CMR but RV pressure-volume loop analysis shows impaired RV diastolic stiffness and RV contractility leading to RV-PA uncoupling.

415 Cryopreserved Human Umbilical Cord in the Treatment of Pediatric Tethered Cord Syndrome

INTRODUCTION: Tethered cord syndrome (TCS) is characterized by neurological, gastrointestinal/genitourinary, and musculoskeletal dysfunction attributable to spinal cord traction. It is estimated that 20-50% of children with open and closed neural tube defects repaired after birth will require surgery to detether the spinal cord. The cryopreserved human umbilical cord (HUC) patch has regenerative, anti-inflammatory, and anti-scarring properties. In animal models, when HUC is used as a meningeal patch it allows regeneration of the arachnoid layer, prevents spinal cord tethering, and improves spinal cord function after in utero spina bifida repair. We hypothesized that this would benefit pediatric patients requiring detethering. METHODS: Serial patients with clinical and radiographic evidence of a tethered cord were enrolled following informed consent. At the time of surgery they underwent spinal cord detethering with neuromonitoring. At the time of closure a HUC graft (Clarix 1k) was placed as a dural underlay, the dura was closed primarily, and a second graft was placed as a dural overlay and sealed with tissue sealant. Serial neurologic assessments and urologic assessments were obtained. MRIs are performed annually or as indicated clinically. RESULTS: Four patients have been detethered with use of HUC as a dural underlay and overlay (2 lipomyelomeningocele, 1 lipoma/diastematomyelia, 1 tethered cord). One patient remains neurointact, with resolution of mild neurogenic bladder. Another patient has gained sensation of lower extremities, sensation to catheterization, with resolution of neurogenic bowel and bladder. One has gained sensation to catheterization, with resolution of neurogenic bowel and bladder. The fourth patient is stable. CONCLUSIONS: Early use of HUC as a dural underlay and overlay during closure for tethered cord surgery shows promise in improving spinal cord function in pediatric patients.

Novel and deleterious nucleotide variations in the HAND1 gene probably affect miRNA target sites and protein function in pediatric patients with congenital heart disease.

Congenital heart disease (CHD) is the most prevalent developmental defect and principal cause of infant mortality and affects cardiac and large blood vessel structures in approximately 1% of live births worldwide. To date, numerous studies have related critical genetic dysfunctions to the pathogenesis of CHDs. However, the genetic basis underlying CHD remains largely unknown. In the present study, we investigated the association of nucleotide variations in coding and noncoding regions of the HAND1 gene with the risk of CHD. The HAND1 gene, encoding a helix-loop-helix transcription factor, is particularly relevant for mechanisms underlying CHD since it plays a significant role in heart development. The genomic DNA of 150 unrelated pediatric patients with CHD was screened by PCR-SSCP and direct sequencing. Four novel and heterozygous missense mutations were identified in the first exon, with three causing amino acid substitutions (p.Val149Met, p.Tyr142His, and p.Leu146Met). In-silico analysis also indicated their deleterious impact on protein structure and function. In addition, we identified five novel nucleotide variants in the 3'UTR region (c.*461, c.*342, c.*529, c.*448, c.*593), potentially altering the target sites of miRNAs. These changes include the loss of certain target sites and the acquisition of new ones. These findings confirm the phenotypic association between CHDs and HAND1 mutations and can pave the way for developing new preventive and therapeutic strategies.

Sacral nerve stimulation effect on colonic motility in pediatric patients.

Sacral nerve stimulation (SNS) is a minimally invasive surgical procedure used to treat refractory constipation in children. While its efficacy in improving symptoms has been studied, its effect on colonic motor function remains unclear. This case series explores SNS's impact on colonic motor function in pediatric patients with idiopathic constipation, using high-resolution colonic manometry (HRCM). Four pediatric patients with chronic idiopathic constipation underwent SNS placement for intractable symptoms and were subsequently evaluated via HRCM. Clinical characteristics, comorbidities, treatment regimens, and outcomes were reviewed. HRCM was conducted during the SNS-off and SNS-on phases. The motility index (MI) was measured during the SNS-off (fasting and postprandial) and SNS-on phases. Four pediatric patients aged 8 to 21 years met the inclusion criteria. In three patients, SNS-induced high-amplitude propagating contractions (HAPCs) were noted, and in one patient, low-amplitude propagating contractions (LAPCs) were noted. In one patient, propagating contractions were induced only when SNS was turned on. MI changes with SNS-on were variable among different patients with an increase in MI in two patients after turning SNS on and a decrease in the other two compared with baseline. Adverse effects following SNS placement remained minimal across all cases. This case series is the first to report SNS effects on colonic motility evaluated by HRCM in pediatrics. We demonstrate that propagating colonic contractions are promptly induced when SNS is turned on. Although the initial effects of SNS on colonic motility were observable, additional investigation is necessary to comprehend the fundamental mechanisms and long-term effectiveness of SNS in pediatric patients.

Cardiac Function in Pediatric Patients with MIS-C Using Speckle Tracking and Conventional Echocardiography: A Longitudinal, Single-Center Study.

Cardiovascular involvement in Multisystem Inflammatory Syndrome in Children (MIS-C), a potential consequence of coronavirus disease-2019 (COVID-19), is common. Conventional transthoracic echocardiography (TTE) provides primary data on the function of the left and right ventricles, while Speckle Tracking Echocardiography (STE) is more sensitive. This study aims to assess longitudinal cardiac function using STE in these patients. This longitudinal study was conducted from late 2021 to early 2022 at Imam Hossein Children's Hospital, Isfahan. Cardiac function was assessed by STE at the time of diagnosis and again two months later. Demographics, clinical characteristics, ECG interpretations, imaging studies, and serum cardiac marker levels were collected. Thirty-five pediatric patients with a mean age of 5.1 years (range: 4 months to 17 years) were included and prospectively followed. Twenty-nine of them, comprising 14 males (48.3%) and 15 females (51.7%), underwent STE and were compared with 29 healthy age- and sex-matched children. Factors related to adverse events included reduced myocardial function, enlarged left atrium or ventricle, and mitral regurgitation (MR). Patients with comorbidities affecting strain measurements were excluded from the strain analyses. A significant difference was observed between the groups in regional strains in the basal and apical septal and middle lateral regions. Global strain rate (GLS) and strain rates were not significantly different but were still lower than the control group. Twenty percent of patients had abnormal GLS but normal left ventricular ejection fraction (LVEF). All patients exhibited reduced segmental myocardial strain in at least one segment. Four out of 26 recovered patients without comorbidities had abnormal GLS at follow-up, despite normal LVEF. STE proves more useful than conventional echocardiography in patients with MIS-C, revealing subclinical cardiac injury in the acute and post-acute phases.

Impulse oscillometry assessment of respiratory function in pediatric patients with a history of COVID-19.

While coronavirus disease 2019 (COVID-19) is generally considered to exhibit a less severe clinical course in children than in adults, studies have demonstrated that respiratory symptoms can endure for more than 3 months following infection in at least one-third of pediatric cases. The present study evaluates the respiratory functions of children aged 3-15 years within 3-6 months of their recovery from COVID-19 using impulse oscillometry (IOS) and compares them with the values of healthy children. Included in this prospective cross-sectional study were 63 patients (patient group) aged 3-15 years who contracted COVID-19 between December 2021 and May 2022, as well as 57 healthy children as a control group, matched for age and sex. The demographic, clinical, and laboratory data of the patients were recorded, and respiratory function was assessed based on airway resistance (zR5, zR20, R5-20) and reactance (zX5, zX20, reactance area[AX], resonant frequency [Fres]) using an IOS device. There were no significant differences in the age, weight, height, and body weight z score values of the two groups (p > .05). While the zR5 and R5-20 levels of the patient group were higher (p = .008 and p < .001, respectively) than those of the controls, the zR20, AX, and Fres values did not differ significantly between the groups (p > .05). The parameters indicating the reactance, including zX5 and zX20, were significantly lower in the patient group than in the control group (p = .028 and p < .001, respectively). Total and peripheral airway resistances were found to be elevated in children who had recovered from COVID-19 in the preceding 3-6 months.

Supplementary Respiratory Therapy Improves Pulmonary Function in Pediatric Patients with Cerebral Palsy: A Systematic Review and Meta-Analysis

Background: Despite medical advances, individuals with cerebral palsy (CP) face significant respiratory challenges, leading to heightened hospitalization rates and early mortality among this population. We hypothesize that integrating supplementary respiratory therapy into standard rehabilitation will result in significant improvements in pulmonary function, enhanced respiratory muscle strength, and an overall increase in the quality of life among pediatric patients with CP. Methods: A systematic search of literature across five databases was conducted, and random-effects meta-analyses were performed to assess the impact of supplementary respiratory therapy on (a) pulmonary function: forced vital capacity (FVC), forced expiratory volume in 1 s (FEV1), FVC/FEV1 ratio, peak expiratory flow (PEF), and (b) respiratory muscle strength: maximal inspiratory and expiratory pressure (MIP, MEP), and (c) quality of life. Certainty of evidence was determined by the GRADE assessment. Results: Analysis of data from 11 eligible randomized controlled trials revealed clinically meaningful changes in pulmonary function. We found a relevant mean difference (MD) in absolute PEF of 0.50 L/s (95% confidence interval (CI): 0.19; 0.82 p = 0.0107). The certainty of the evidence ranged from moderate to high. Conclusions: This study presents current evidence on the impact of various supplementary respiratory therapies for CP patients classified under gross motor function classification level I–IV, demonstrating clinically meaningful improvements in pulmonary function and respiratory muscle strength. These improvements suggest the potential for an enhanced quality of life. Our findings hold the promise of serving as a foundational reference for potential revisions to conventional rehabilitation care, incorporating supplementary respiratory therapy.

Specific Alterations in Brain White Matter Networks and Their Impact on Clinical Function in Pediatric Patients With Thoracolumbar Spinal Cord Injury.

The alternation of brain white matter (WM) network has been studied in adult spinal cord injury (SCI) patients. However, the WM network alterations in pediatric SCI patients remain unclear. To evaluate WM network changes and their functional impact in children with thoracolumbar SCI (TSCI). Prospective. Thirty-five pediatric patients with TSCI (8.94 ± 1.86 years, 8/27 males/females) and 34 age- and gender-matched healthy controls (HCs) participated in this study. 3.0 T/DTI imaging using spin-echo echo-planar and T1-weighted imaging using 3D T1-weighted magnetization-prepared rapid gradient-echo sequence. Pediatric SCI patients were evaluated for motor and sensory scores, injury level, time since injury, and age at injury. The WM network was constructed using a continuous tracing method, resulting in a 90 × 90 matrix. The global and regional metrics were obtained to investigate the alterations of the WM structural network. topology. Two-sample independent t-tests, chi-squared test, Mann-Whitney U-test, and Spearman correlation. Statistical significance was set at P < 0.05. Compared with HCs, pediatric TSCI patients displayed decreased shortest path length (Lp = 1.080 ± 0.130) and normalized Lp (λ = 5.020 ± 0.363), and increased global efficiency (Eg = 0.200 ± 0.015). Notably, these patients also demonstrated heightened regional properties in the orbitofrontal cortex, limbic system, default mode network, and several audio-visual-related regions. Moreover, the λ and Lp values negatively correlated with sensory scores. Conversely, nodal efficiency values in the right calcarine fissure and surrounding cortex positively correlated with sensory scores. The age at injury positively correlated with node degree in the left parahippocampal gyrus and nodal efficiency in the right posterior cingulate gyrus. Reorganization of the WM networks in pediatric SCI patients is indicated by increased global and nodal efficiency, which may provide promising neuroimaging biomarkers for functional assessment of pediatric SCI. 2 TECHNICAL EFFICACY: Stage 5.

Cardiopulmonary function in paediatric post-COVID-19: a controlled clinical trial

Recently, the importance of post-COVID-19 in children has been recognized in surveys and retrospective chart analysis. However, objective data in the form of cardiopulmonary exercise test as performed in adults suffering from this condition are still lacking. This study aimed to investigate the cardiopulmonary effects of post-COVID-19 on children and adolescents. In this cross-sectional study (the FASCINATE study), children fulfilling the criteria of post-COVID-19 and an age- and sex-matched control group underwent cardiopulmonary exercise testing on a treadmill and completed a questionnaire with regard to physical activity before, during and after the infection with SARS-CoV-2. We were able to recruit 20 children suffering from post-COVID-19 (mean age 12.8 ± 2.4 years, 60% females) and 28 control children (mean age 11.7 ± 3.5 years, 50% females). All participants completed a maximal treadmill test with a significantly lower V˙O2peak\\documentclass[12pt]{minimal} \\usepackage{amsmath} \\usepackage{wasysym} \\usepackage{amsfonts} \\usepackage{amssymb} \\usepackage{amsbsy} \\usepackage{mathrsfs} \\usepackage{upgreek} \\setlength{\\oddsidemargin}{-69pt} \\begin{document}$$\\dot{{\ ext{V}}}{{\ ext{O}}}_{2}{\ ext{peak}}$$\\end{document} in the post-COVID-19 group (37.4 ± 8.8 ml/kg/min vs. 43.0 ± 6.7 ml/kg/min. p = 0.019). This significance did not persist when comparing the achieved percentage of predicted V˙O2peak\\documentclass[12pt]{minimal} \\usepackage{amsmath} \\usepackage{wasysym} \\usepackage{amsfonts} \\usepackage{amssymb} \\usepackage{amsbsy} \\usepackage{mathrsfs} \\usepackage{upgreek} \\setlength{\\oddsidemargin}{-69pt} \\begin{document}$$\\dot{{\ ext{V}}}{{\ ext{O}}}_{2}{\ ext{peak}}$$\\end{document}. There were no significant differences for oxygen pulse, heart rate, minute ventilation or breathing frequency. Conclusion: This is the first study to investigate post-COVID-19 in children using the cardiopulmonary exercise test. Although there was a significantly reduced V˙O2peak\\documentclass[12pt]{minimal} \\usepackage{amsmath} \\usepackage{wasysym} \\usepackage{amsfonts} \\usepackage{amssymb} \\usepackage{amsbsy} \\usepackage{mathrsfs} \\usepackage{upgreek} \\setlength{\\oddsidemargin}{-69pt} \\begin{document}$$\\dot{{\ ext{V}}}{{\ ext{O}}}_{2}{\ ext{peak}}$$\\end{document} in the post-COVID-19 group, this was not true for the percent of predicted values. No pathological findings with respect to cardiac or pulmonary functions could be discerned. Deconditioning was the most plausible cause for the experienced symptoms. Trial registration: clinicaltrials.gov, NCT054445531, Low-field Magnetic Resonance Imaging in Pediatric Post Covid-19—Full Text View—ClinicalTrials.gov.What is Known:• The persistence of symptoms after an infection with SARS-CoV 2, so-called post-COVID-19 exists also in children.• So far little research has been conducted to analyze this entity in the pediatric population.What is New:• This is the first study proving a significantly lower cardiopulmonary function in pediatric patients suffering from post-COVID-19 symptoms.• The cardiac and pulmonary function appear similar between children suffering from post-COVID-19 and those who don’t, but the peripheral muscles seem affected.