Pediatric Patients Research Articles

Cost-Effectiveness of Hypoglossal Nerve Stimulation for Pediatric Severe Obstructive Sleep Apnea in Down Syndrome Patients.

To examine the cost-effectiveness of hypoglossal nerve stimulation (HGNS) implantation at an early age in simulated pediatric cohorts with Down Syndrome (DS) and severe obstructive sleep apnea (OSA). Cost-utility analysis. Hypothetical cohort. A Markov model simulated 3 pediatric cohorts with DS and OSA beginning at age 4 years until 21 years. Cohorts received HGNS implants in early childhood, late childhood, or adulthood at age 4, 13 (current FDA-approved age), or 18 years, respectively. Input model parameters were obtained from the literature and our institution. Outcomes were measured with an incremental cost-effectiveness ratio (ICER), measured in dollars per quality-adjusted life-year (QALY). Deterministic 1-way sensitivity analyses were conducted to evaluate the effects of parameter uncertainty. Results (total costs; total QALYs) across the time horizon were determined for each cohort: early implantation ($83,300.35; 15.79), late ($48,319.09; 14.98), and adult ($38,721.07; 14.55). ICERs were $48,892.47 per QALY for early vs late implantation, $43,471.15 per QALY for early vs adult implantation, and $30,959.58 per QALY for late vs adult implantation. All ICERs were below a willingness-to-pay threshold of $50,000 per QALY. Varying the discount rate and utility expectedly varied the ICERs and cost-effectiveness. Threshold analysis showed early implantation to be cost-effective for a HGNS implantation cost up to $62,230 compared to late implantation. The current study suggests HGNS is a cost-effective treatment strategy for pediatric patients with DS and severe OSA. Our findings also suggest cost-effectiveness at ages younger than 13, the current age of FDA approval.

Clinical characteristics and nomogram model for predicting the risk of recurrence of complicated urinary tract infection in pediatric patients

Complicated urinary tract infection (cUTI) has higher incidences of antibiotic resistance, recurrence, chronicity, and progression. There exist differences in the immune functions, anatomical structures and physiological functions of urinary tract system between children and adults. In addition, the immature stage of hygiene habit development and the inability to express discomfort accurately all contribute to cUTI recurrence in pediatric patients. cUTI recurrence in children can lead to serious consequences such as growth and development delay, renal scars, and progression to end-stage kidney diseases. Despite the high incidence of cUTI recurrence, no predictive model currently exists to guide targeted intervention in pediatric patients. This study aimed to establish a nomogram to predict the risk of cUTI recurrence in children, thereby facilitating targeted prevention and treatment strategies in pediatric patients. The nomogram was developed based on a retrospective cohort that included 421 pediatric patients with cUTI at West China Second University Hospital from January 2020 to August 2023. The patients were randomly divided into a training set and a validation set in a 3:1 ratio. Logistic regression analysis was used to identify risk factors and construct the nomogram for predicting the risk of cUTI recurrence, followed by validation and performance analysis. Of the 421 children with cUTI, the recurrence rate of cUTI was 68.4% (288 cases) during an average follow-up duration of 22.9 months. The nomogram comprised female gender, history of urinary tract surgery, Escherichia coli in urine culture, renal dysfunction, and vesicoureteral reflux as predictors of cUTI recurrence in pediatric patients. The model showed favorable performance with C-index values of 0.735 and 0.750 in the training dataset and the validation dataset, respectively. The decision curve analysis revealed that the nomogram might be clinically useful. The reliable nomogram would be beneficial for clinicians to identify children with high risks of cUTI recurrence for targeted intervention.

Clinical assessment of the potential use of a novel single-dose prefilled injection device for the administration of Acthar Gel in children: a narrative review.

Acthar® Gel (repository corticotropin injection; Mallinckrodt Pharmaceuticals, NJ, USA) is indicated for the treatment of myriad inflammatory disorders and is currently administered manually via a vial and syringe. The administration of Acthar via a single-dose prefilled injector (SelfJect™) is intended to simplify its subcutaneous (SC) delivery. The purpose of this review was to determine whether SelfJect is suitable for use in pediatric patients through a literature assessment of various factors, including skin depth, needle length and gauge, dosage, force required for injection, and potential harms. Infants and young children, who commonly have skin-to-muscle distances less than the minimum depth of SelfJect administration, may have risk of unintentional intramuscular (IM) injection; however, an inadvertent IM injection poses no additional risk to children because of the bioequivalence between SC and IM administration of Acthar. The needle gauge of SelfJect is acceptable for pediatric patients and aligns with the Centers for Disease Control and Prevention recommendations for SC injections. The dosage delivered by SelfJect is only appropriate for children over 2years of age. Although adolescents would likely be able to achieve the minimum force required to remove the protective cap and deliver a full dose of Acthar with SelfJect, an adult (18years of age and older) should administer SelfJect to pediatric patients. In addition to the commonly reported postmarketing adverse events (AEs) from Acthar administration (e.g., asthenic conditions, fluid retention, insomnia, headache and increased blood glucose), injection site-related AEs common to injection devices may occur with SelfJect use. The risk of needlestick injury from SelfJect is mitigated by a needle guard. In summary, this review of injection device considerations demonstrates that SelfJect is appropriate for use in the pediatric population.

--Connecting Transcriptomics with Computational Modeling to Reveal Developmental Adaptations in Pediatric Human Atrial Tissue.

Nearly 1% of babies are born with congenital heart disease (CHD) - many of whom will require heart surgery within the first few years of life. A detailed understanding of cardiac maturation can help to expand our knowledge on cardiac diseases that develop during gestation, identify age-appropriate drug therapies, and inform clinical care decisions related to surgical repair and postoperative management. Yet, to date, our knowledge of the temporal changes that cardiomyocytes undergo during postnatal development is limited. In this study, we collected right atrial tissue samples from pediatric patients (n=117) undergoing heart surgery. Patients were stratified into five age groups. We measured age-dependent adaptations in cardiac gene expression, and used computational modeling to simulate action potential and calcium transients. Enrichment of differentially expressed genes (DEGs) revealed age-dependent changes in several key biological processes (e.g., cell cycle, structural organization), cardiac ion channels, and calcium handling genes. Gene-associated changes in ionic currents exhibited age-dependent trends, with changes in calcium handling (INCX) and repolarization (IK1) most strongly associated with an age-dependent decrease in the action potential plateau potential and increase in triangulation, respectively. We observed a shift in repolarization reserve, with lower IKr expression in younger patients, a finding potentially tied to an increased amplitude of IKs that could be triggered by elevated sympathetic activation in pediatric patients. Collectively, this study provides valuable insights into age-dependent changes in human cardiac gene expression and electrophysiology, shedding light on molecular mechanisms underlying cardiac maturation and function throughout development.

Prevalence of Dental Anomalies in Pediatric Patients visiting Tertiary Care Hospital: A Panoramic Radiograph based study

Introduction: Dental anomalies are deviations from the normal development, structure, or appearance of teeth. These anomalies can affect the number, size, shape, position, and structure of teeth. Objective: This study aimed to assess the prevalence of dental anomalies in the pediatric patients visiting Kathmandu University School of Medical Sciences (KUSMS), Dhulikhel Hospital, Nepal. Methods: A descriptive cross-sectional study was conducted at KUSMS, Dhulikhel Hospital. The panoramic radiographs of the pediatric patients between 6 and 14 years of age in the archive of the hospital were assessed for the presence of dental anomalies. Results: Of the total 451 panoramic radiographs meeting the inclusion criteria, 55.9% were male and 44.1% were female. The overall prevalence of dental anomaly was 26.8 % with rotation as the most common anomaly (19.3%) followed by missing (6.7%), supernumerary teeth (4.4%), dilaceration 2.4 %, gemination (1.8%), dens-in-dente (1.1%) and peg shaped (0.9%). Conclusions: The most common dental anomaly among the pediatric patients was rotation followed by missing and supernumerary tooth.

2D speckle tracking echocardiography and comparison with cardiac magnetic resonance in children with acute myocarditis

BackgroundCardiac magnetic resonance (CMR) plays a major diagnostic role in acute myocarditis (AM) in children as biopsy is rarely performed in this age group. Contribution of standard echocardiography (ECHO) is limited in AM, but speckle tracking echocardiography (STE) quantitatively characterizes myocardial function, with good sensitivity for detecting subclinical left ventricular (LV) dysfunction and regional kinetics disorders beyond the site of inflammation. This work aimed to evaluate the diagnostic potential of STE as compared with CMR findings in pediatric patients with AM.MethodsThe study was conducted during 2022–2023. Troponin, electrocardiography, ECHO with STE, and CMR with early and late enhancement were performed on each patient. Affected heart segments were analyzed by both STE and CMR, and the correlation of the two methods was assessed.ResultsDuring the study period, 20 children were diagnosed with AM [14 boys, 6 girls; mean age 12 years (median 14)]. On ECHO, three patients had a deviation in LV biometry, and four patients had a mild systolic function disorder. STE showed at least one affected cardiac segment in all patients, most often the inferolateral segment (16/20; 80%). Of the 20 patients, STE showed a reduction in LV global longitudinal strain in 13 (65%) patients. In all patients, CMR identified an inflammatory focus, most frequently inferolateral (15/20; 75%). The strongest accordance between STE and CMR was observed for the involvement of anterolateral segments (k = 0.88) and the weakest for inferoseptal damage (k = 0.4).ConclusionsSTE can provide important diagnostic information in pediatric patients with AM. This modality supports the detection of early regional edema and subclinical myocardial dysfunction and can determine the impairment severity. STE is non-invasive and repeatable without the need for special patient preparation or for general anesthesia.

Prognostic Effects of Operation Age for Pediatric Patients with Supravalvar Aortic Stenosis.

The appropriate age for surgical repair of asymptomatic congenital supravalvular aortic stenosis (SVAS) is still unknown. The purpose of this research was to assess the safety and effectiveness of various operation ages when managing SVAS. Consecutive asymptomatic SVAS pediatric patients in the Beijing Fuwai and Yunnan Fuwai hospitals over a period of 18 years were retrospectively analyzed. Patients were classified as follows: age <2.0 years (y) (n = 84), 2.0-5.0 y (n = 72), and >5.0 y (n = 92). The primary safety endpoint was in-hospital death or extracorporeal membrane oxygenation (ECMO) needed. The primary effectiveness outcome was re-operation or restenosis during follow-up. To calculate the hazard ratios (HR), Cox regression with inverse probability of treatment weighted was utilized. At the time of surgery, the median age of the 248 patients that were included was 4 y (interquartile range (IQR): 1.8-6.5). For the primary safety outcome, 7 (8.3%) patients in the age <2.0 y group had in-hospital death or ECMO needed, while no patients in the age 2.0-5.0 y and age>5.0 y groups (p = 0.001). The median follow-up was 25.5 months (IQR: 7.0-59.0). Compared with the age 2.0-5.0 y group, the age <2.0 y group and age >5.0 y group had a higher risk of re-operation or restenosis (age <2.0 y, HR = 3.27, 95% CI 1.25-8.60; age >5.0 y, HR = 2.87, 95% CI 1.19-6.91). Asymptomatic children with SVAS without other cardiovascular abnormalities should be considered for delayed surgical intervention until 2 years of age, and then surgery should be conducted as soon as possible. Children with severe symptoms should undergo surgery immediately, regardless of age. ChiCTR2300067851, https://www.chictr.org.cn/showproj.html?proj=177491.

Malnutrition‐related cardiomyopathy in a pediatric patient with autism spectrum disorder

AbstractWe present a case of a 5‐year‐old male patient with history of autism spectrum disorder and chronic malnutrition secondary to an extremely restrictive diet who presented with life‐threatening anemia and heart failure secondary to malnutrition‐related cardiomyopathy. This case highlights the importance of recognizing nutritional deficiencies in high‐risk individuals and screening for nutritional deficiencies in at‐risk children. Furthermore, it underscores the need for early recognition and intervention as malnutrition‐related cardiomyopathy can have significant morbidity.

Music Therapy for Pain and Anxiety Reduction in Pediatric Patients Undergoing Procedures: A Systematic Review and Meta-Analysis Protocol

Introduction: Many children undergo invasive and noninvasive procedures in the pediatric medical setting, which can be associated with pain and anxiety. Both pharmacological and nonpharmacological interventions are currently being used to minimize pain and anxiety in pediatric patients in the medical setting. Music therapy is an evidence-based, cost-effective, and nonpharmacological service administered by a board-certified music therapist which may reduce pediatric pain and anxiety during procedures. We will undertake a systematic review and meta-analysis that will comprehensively analyze the quantitative publications that detail the effects of music therapy on pediatric pain and anxiety for patients undergoing invasive or noninvasive medical procedures. Method: This protocol is reported according to the Preferred Reporting Items for Systematic Reviews and Meta-Analyses Protocols (PRISMA-P) statement and is registered in the International Prospective Register of Systematic Reviews (Prospero; CRD42023474635 ). A three-step, iterative search strategy will include (1) an initial scoping search, (2) development and execution of a comprehensive search strategy, and (3) a search for additional publications. Procedures for disagreements among reviewers and the article screening process are outlined. Statistical analyses including analysis of outcomes and sensitivity analysis has been determined. A plan for critical appraisal of sources using the Grading Recommendations Assessment, Development, and Evaluation (GRADE) and quality of evidence using the Outcome Reporting Bias in Trials (ORBIT) of evidence is also present in this protocol. Conclusion: This protocol is presented with the intention to execute and publish results of this systematic review and meta-analysis to help inform clinical practice and future research.

Безопасность применения двухкомпонентного препарата моноклональных антител к SARS-CoV-2 (тиксагевимаб + цилгавимаб) для доконтактной профилактики новой коронавирусной инфекции у детей с ревматическими заболеваниями

New coronavirus infection (COVID-19) is an acute respiratory disease caused by the SARS-CoV-2 coronavirus. Tixagevimab+cilgavimab has been developed for the prevention and treatment of COVID-19. The purpose of this research was to evaluate the safety of the use of the two-component drug of monoclonal antibodies to SARS-CoV-2 (tixagevimab+cilgavimab) for pre-exposure prophylaxis of the new coronavirus infection in children with rheumatic diseases (RDs). Materials and methods used: a single-center retrospective study with prospective stage had been carried out that included 300 pediatric patients aged 12 to 18 y/o with RDs. The safety of pre-exposure prophylaxis of COVID-19 was assessed based on the dynamics of clinical and laboratory parameters as well as the total indices of RD activity. The effectiveness of pre-exposure prophylaxis was assessed based on recording the incidence of the new coronavirus infection. Safety and effectiveness were assessed after 3, 6, and 9 months after the administration of tixagevimab+cilgavimab drug. Results: no statistically significant increase in RDs activity was registered during 9 months of observation after pre-exposure prophylaxis of COVID-19. On the contrary, in patients with various types of juvenile arthritis, systemic lupus erythematosus and juvenile ankylosing spondylitis during the observation period against the background of antirheumatic therapy, a statistically significant decrease in the activity indices was registered - JADAS71 (p&lt;0.001, p=0.002, p=0.030), SELENA-SLEDAI (p&lt;0.001, p=0.027, p=0.570) and JSpADA (p=0.009, p=0.205, p=1.000), respectively; in patients with juvenile dermapolymyositis, systemic sclerosis and systemic vasculitis - a tendency to decrease in activity indices - DAS (p=0.002, p=0.050, p=0.193), J4S (p=0.840, p=0.720, p=1.000), PVAS (p=0.120, p=0.230, p=1.000 for the new and p=0.360, p=1.000, p=0.860 for the constant attributes), respectively. The new coronavirus infection was suffered by 6/300 (2%) patients within 9 months after pre-exposure prophylaxis. Conclusion: pre-exposure prophylaxis of COVID-19 with the two-component drug of monoclonal antibodies to SARS-CoV-2 (tixagevimab+cilgavimab) may be considered as both effective and safe and can therefore be suggested to pediatric patients with RDs.

Peadiatric amputation from traditional bone setters’ gangrene in Ibadan: a malady

Background: Pediatric amputations resulting from gangrene pose a significant medical challenge, especially in resource-limited settings. In Nigeria, traditional bone setters (TBS) often serve as the first point of contact for musculoskeletal injuries, but their practices can lead to severe complications, including gangrene. Our hospital manages many of such cases. This study aims to explore the epidemiology, clinical presentation, and outcomes of pediatric patients who developed gangrene requiring amputation following TBS treatment, with the goal of informing public health strategies and improving pediatric care quality. Methods: The study is a retrospective study which seek to extract the data from the hospital record all the patient who underwent amputation due to gangrene after initial TBS treatment. All the case notes were retrieved and data was extracted and analyzed using descriptive statistics. The study adhered to ethical standards and anonymized data to protect patient confidentiality, ensuring the study's integrity. Results: The study involved 33 pediatric patients aged 8.3 years, with a significant gender disparity. Most patients were from the Yoruba ethnic group and practiced Islam. 87.9% of parents had no formal education. Gangrene was most common in the left hand, and falls were the leading cause of initial injury. The average surgery time was 81.2 minutes, with a 13-day hospital stay. Post-operative complications occurred in 43.5% of patients, with surgical site infections (SSI) being the most common. Only 8.7% received occupational therapy. Conclusions: The study highlights post-operative care challenges in pediatric amputations, including high complications and limited access to services. It suggests targeted interventions, infection control, and public health campaigns to improve outcomes. Future research should include long-term follow-up studies.

Случай тяжелого пищевого ботулизма у ребенка: трудности диагностики и тактика терапии

Practitioners’ alertness of botulism in children decreases due to the rarity of its cases. Early diagnosis is based on clinical symptoms though the clinical manifestations may also escalate rapidly and further lead to misdiagnosis. Patients with suspected botulism require immediate hospitalization due to the high risk for paralysis of respiratory muscles and the need for mechanical ventilation. The timely administration of the antitoxin is crucial. However, maximum allowable time of its administration counting from the onset is still a subject to discussion among physicians. Authors represent a clinical case of late diagnosis of severe foodborne botulism in a 5 y/o pediatric patient with the development of tetraplegia, paralysis of cranial nerves, mechanical ventilation for 148 days, the beginning of motor recovery on the 10th day and antitoxin administration on the 18th day counting from the onset of paralysis that all let to the full motor recovery after 20 months. The difficulties of diagnosis in the acute period and both laboratory and instrumental studies are described. The questions of the duration and the necessity of the administration of botulinum antitoxin in this particular clinical case of late diagnosis are discussed as well.

Пути оптимизации антропометрических параметров у детей с фармакорезистентной эпилепсией, получающих кетогенную диетотерапию

Ketogenic diet (KD) whilst being an effective method of diet therapy for drug-resistant epilepsy (DRE) is nevertheless accompanied by a significant number of side effects that include the possibility for the delayed physical development in pediatric patients. The problem is multifactorial and is primarily associated with the non-physiological nature of the ketogenic diet itself. Foreign studies on the KD effect on the physical development in children are contradictory and there are none domestic ones. The purpose of this research was to study the dynamics of anthropometric parameters in children with DRE against the background of KD and to assess the possibilities of the alimentary factor in its improvement. Materials and methods used: single-center retrospective cohort study had been carried out on the basis of the Russian Federal Research Centre for Nutrition, Biotechnology and Food Safety (Moscow, Russia) in 2016-2024. The study included patients with an established diagnosis of DRE against the background of KD aged 1 to 18 y/o. Patients were monitored for 24 months as follows: quarterly during the first year and semiannually during the second year of observation, using anthropometric, clinical-laboratory and clinical-instrumental research methods. Results: by the 3rd month of KD, out of 174 pediatric patients, the diet was effective in 105 (60%); by 12th month, 100% control over attacks was achieved in 11 (6%). Analysis of anthropometric parameters made it possible to establish that at the initial point of observation, in accordance with the z-score of the body mass index (BMI), 108 (64%) had normal nutritional status, 22 (13%) had malnutrition and 40 (24%) were overweight/obese. When studying the dynamics of weight and height indicators of children who were on KD throughout the observation period, it was found that the median BMI z-score statistically significantly changed upward (p=0.047), which was largely due to growth retardation, with a decrease in the height/age z-score throughout the observation period (p&lt;0.001). It was found that the protein component of the diet, as well as the level of ketonemia, have a significant impact on the dynamics of the BMI z-score in KD patients. The amount of protein in the diet at 6% to 7% of the energy value contributed to the maintenance of optimal growth parameters in children within this research. Conclusion: the significance of the BMI indicator in assessing the nutritional status over KD decreases against the background of growth retardation. Taking into account the value of the protein/energy indicator when calculating nutrition helps in maintaining of the physical development rate in such patients.

Drainage of middle cranial fossa epidural abscess through mastoidectomy: Our experience and review of the literature

Background: Otitis media (OM) can uncommonly lead to intracranial complications. Epidural abscesses represent a large proportion of cases; however, literature regarding the optimal surgical management of otogenic epidural abscesses is sparse. Favorably located epidural abscesses may be amenable to drainage through a transmastoid approach because the tegmen mastoideum lies immediately inferior to the middle cranial fossa (MCF). Case Description: We report 3 pediatric patients with OM complicated by epidural abscesses of the MCF. The ages ranged from 3 to 6 years old, with 2 females and 1 male. All 3 patients had acute mastoiditis with an abscess of the MCF ranging from 1.6 cm to 6.3 cm at the largest dimension. All patients underwent canal wall-up mastoidectomy with the evacuation of the MCF abscess through a small window, 0.7 × 0.7 cm or less, created in the tegmen mastoideum. All patients were successfully treated through this approach and had complete resolution of their infection on follow-up imaging. There were no postoperative temporal lobe encephaloceles. Conclusion: This is one of the few descriptions of the treatment of an otogenic epidural abscess of the MCF through a transmastoid approach. Collaboration with neurosurgery is vital because their familiarity with the intracranial space helped to guide the accurate direction of dissection. This highlights the importance of a multidisciplinary approach in the treatment of epidural intracranial abscess of the MCF through this approach. The risk of postoperative temporal lobe encephalocele was minimized due to the small tegmen defect size.

Pediatric Burn Care for Burn Injury: Outcomes by Timing of Referral Using a U.S. Single-Center Retrospective Cohort, 2005-2019.

Burn centers offer specialized care to pediatric patients with burn injuries. However, data about outcome in relation to timing of transfer are limited. We examined these timings and outcomes among burn-injured pediatric patients. Single-center, retrospective cohort study of U.S. national and international practice. Shriners ESO Trauma Burn Registry, 2005-2019. Patients 0-17 years old admitted to a pediatric burn center with 10-80% total body surface area (TBSA) burn between 2005 and 2019. None. The cohort of 761 patients was subcategorized according to timing of referral: early presentation (EP) (0-7 d), delayed presentation (DP) (8-60 d), and very delayed presentation (VDP) (> 60 d). Primary outcomes included hospital length of stay (LOS), ICU LOS, percent TBSA (%TBSA)/LOS ratio, mortality, and disposition. Dichotomous outcomes were analyzed using logistic regression and continuous outcomes were compared using median regression, both utilizing multivariable analysis adjusting for a priori confounders and presented as adjusted coefficients (ACs) with 95% CI. Delayed referral (DP and VDP) was associated with higher %TBSA burn and with fewer referrals from high-income countries. Compared with EP, VDP was associated with higher LOS/%TBSA (AC, 0.2 [0.01-0.4]). On univariate analysis, in comparison with EP, DP and VDP were associated with higher ICU (DP 16 [6-29]; VDP 8 [0-18]) and hospital LOS (DP 34 [21-55]; VDP 32 [18-58]). Overall, ten of 761 patients died (1.3%), and we found no association between timing of referral and mortality. Also, in 751 survivors, 635 patients (84.6%) were discharged home without home health, and we found no association with timing of referral on multivariable analysis. In this report, we have reviewed our single-center, international burn-injured pediatric cohort, operating in the United States (2005-2019). We find that referral with VDP as opposed to EP was associated with increased LOS/%TBSA.

Relative Bioavailability Studies With Mitapivat: Formulation and Food Effect Assessments in Healthy Subjects.

Pyruvate kinase (PK) deficiency is a rare, hereditary, hemolytic anemia caused by mutations in the PKLR gene encoding the PK enzyme. Mitapivat (previously designated AG-348) is a first-in-class, oral, allosteric activator of PK. We report results from 5 Phase 1 trials in healthy adults to characterize and compare mitapivat pharmacokinetics across different formulations and analyze food effects on mitapivat bioavailability (Studies 1-5). Pharmacokinetic assessments were peak exposure, total exposure, time to maximum plasma concentration of mitapivat, and relative bioavailability (where appropriate). Plasma total exposure of mitapivat was similar in the fasted and fed (high-fat meal or different soft foods) states after capsule, tablet, and pediatric granule formulations. Although mitapivat administration with food reduced the rate of mitapivat absorption (delay in time to maximum plasma concentration; reduction in maximum concentration) versus dosing under fasted conditions, this was not considered clinically relevant, given the lack of effect on total mitapivat exposure. Consequently, the administration instructions for mitapivat relating to food state that "patients may take mitapivat tablets with or without food." These findings will continue to inform clinical studies and development of mitapivat in adult and pediatric patients with hemolytic anemias and may help inform healthcare professionals on mitapivat dosing/administration recommendations in clinical practice.

Arterial Ischemic Stroke in Pediatric Patients: A Tertiary Hospital Experience

Arterial Ischemic Stroke in Pediatric Patients: A Tertiary Hospital Experience

Дефицит лизосомной кислой липазы: клиническая патофизиология и возможности ферментозаместительной терапии

Lysosomal acid lipase deficiency (LALD) is a rare disease of lysosomal storage inherited in an autosomal recessive manner and caused by damaging mutations in the LIPA gene encoding the synthesis of the enzyme lysosomal acid lipase (LAL). Authors represent basic information about LALD, describe its typical clinical and laboratory manifestations, features of enzyme replacement therapy with Sebelipase alfa (sold under the brand name KANUMA®), which is a recombinant form of the enzyme LAL, outcomes and prognosis of the disease in children. Modern methods for the LALD detection and an algorithm for diagnosing the disease in patients with hypertransaminasemia are discussed as well. A clinical case report of LALD with proven effectiveness of treatment with Sebelipase alfa (KANUMA®) in a pediatric patient are given.

Indications, outcomes, and complications of orthopaedic implant removal: a prospective observational study

Background: Orthopaedic implant removal is a common procedure, but its indications and outcomes remain debated. This study aimed to examine the indications, outcomes, and complications of implant removal in a tertiary care center. Methods: A prospective observational study was conducted on 66 patients undergoing implant removal between February 2022 and February 2024. Patient demographics, indications for removal, time to removal, and complications were recorded. Follow-ups were conducted at 6 weeks, 3 months, and 6 months post-removal. Results: The study included 47 males and 19 females, with 74% adults and 26% paediatric patients. Pain was the most common indication for removal (42%), followed by elective removal (36%). Most implants (33%) were removed after 2 years. Complications were minimal, with 4.5% incomplete removals and 1.5% failure to remove. No intraoperative fractures or postoperative infections were observed. Patients generally reported improvement in symptoms or psychological well-being following implant removal. Conclusion: Implant removal should be approached selectively, with careful consideration of indications and potential complications. Pain relief and improved function were common outcomes, but risks such as incomplete removal should be considered. The study supports individualized decision-making in implant management, emphasizing the need for thorough preoperative planning and skilled surgical execution.

The protective effect and immunomodulatory ability of orally administrated Lacticaseibacillus rhamnosus GG against Mycoplasma pneumoniae infection in BALB/c mice.

Mycoplasma pneumoniae represents one of the significant etiologies of community-acquired pneumonia in pediatric patients. However, clinical treatment of M. pneumoniae infection in children has encountered challenges due to the escalating resistance to quinolones. Numerous studies have highlighted the potential of probiotic lactobacillus administration in boosting immune responses to bacterial and viral respiratory infections. In this study, the protective efficacy of pre-oral administration of Lacticaseibacillus rhamnosus GG (LGG), Limosilactobacillus reuteri F275, Lactiplantibacillus plantarum NCIMB 8826, L. plantarum S1 or L. plantarum S2 was evaluated in the BALB/c mice model; it was observed that among these five strains of lactobacillus, the supplementation of LGG exhibited the most significant protective effect against M. pneumoniae infection. Moreover, when administered orally, both live LGG and heat-inactivated LGG have demonstrated efficacy in reducing the burden of M. pneumoniae in the lungs and alleviating pulmonary inflammation. Oral supplementation with LGG resulted in the inhibition of neutrophil recruitment into the lungs and increased recruitment of alveolar macrophages in M. pneumoniae-infected mice. Additionally, LGG supplementation led to increased production of IL-10 and secretory IgA (sIgA), while suppressing the levels of IL-1β, IL-6, IL-17A, and TNF-α in the lungs of mice infected with M. pneumoniae. The data suggests that supplementation with LGG can modulate immune responses, decrease pathogen load, and alleviate inflammatory injury in the lungs of M. pneumoniae-infected mice.