Conventional open thyroidectomy leaves a visible cervical scar that can adversely affect the psychosocial well-being of pediatric patients. The transoral endoscopic thyroidectomy vestibular approach (TOETVA) offers a scarless alternative, but data regarding its application in pediatric malignancies remain scarce. This study aims to evaluate the surgical, cosmetic outcomes of TOETVA in pediatric patients with differentiated thyroid carcinoma (DTC). We retrospectively reviewed consecutive pediatric patients ([Formula: see text] 18 years) who underwent TOETVA for DTC (cT1N0M0) at a high-volume center between June 2020 and June 2026. Postoperative complications and cosmetic satisfaction were evaluated. Twenty-six patients (mean age: 16.7 ± 1.9 years; 84.6% female) were enrolled. All procedures were successfully completed with a 0% conversion rate to open surgery. Twenty-four patients (92.3%) underwent thyroid lobectomy with isthmusectomy and prophylactic central neck dissection (CND) (mean operative time: 87.9 ± 9.5 min), while two (7.7%) underwent total thyroidectomy with CND (mean operative time: 132.5 min). Final histopathology confirmed papillary thyroid carcinoma in all cases. No major perioperative complications occurred. Transient adverse events included minor chin ecchymosis (11.5%), hoarseness (7.7%), and mental nerve injury (7.7%), all resolving completely within 3 months. No permanent recurrent laryngeal nerve injury, mental nerve dysfunction, or hypocalcemia was observed. Cosmetic outcomes were highly favorable, with all patients expressing excellent post-operative aesthetic satisfaction. TOETVA appears to be a safe and feasible surgical approach for carefully selected pediatric patients with DTC. In this largest reported pediatric TOETVA series to date, the procedure achieved favorable surgical and cosmetic outcomes with low complication rates. TOETVA may therefore represent an effective scarless alternative for selected pediatric thyroid cancer when performed by experienced surgeons in high-volume centers.
Describe the implementation of robotic-assisted surgery for pediatric cysts and neoplasms and characterize perioperative outcomes. A retrospective review was performed of pediatric patients undergoing robotic-assisted resection of cysts or neoplasms at a quaternary children's hospital from 2011 to 2026. Demographic, lesion, operative, and outcome data were analyzed. Thirty-eight pediatric robotic cases were reviewed. Median age was 13 years (IQR 8.25-16). Eighteen intracavitary cysts and 20 neoplasms were resected. Operations for solid neoplasms consisted of thymectomies (n = 3), mediastinal teratoma excision, lung lobectomy, partial gastrectomy, left hepatectomy, distal pancreatectomy (n = 2), pancreatic uncinate resection, pancreaticoduodenectomy, nephrectomies (2 partial, 2 radical), adrenalectomy, periadrenal mass resection, ovarian cystectomy (n = 2), and radical cystoprostatectomy. Median EBL was 15 mL (IQR 5-25) and day of discharge 1 (IQR 0-2). The median opioid dose prescribed at discharge was 0.28 mg/kg oral morphine equivalents with duration of 1 (0-3) day. Thirty-one (82%) patients experienced no complications. Major complications occurred after pancreatic resections, including hemorrhagic pancreatitis following uncinate resection and delayed biliary stenosis with ductal leak after pancreaticoduodenectomy requiring revision 1.5 years later. Robotic-assisted surgery is a safe and feasible option for selected pediatric cysts and neoplasms, offering low blood loss, short hospitalization, and minimal postoperative opioid requirements.
Epilepsy is a common pediatric neurologic disorder, and its surgical management has increased over time. To characterize longitudinal changes in patient selection, surgical approach and outcomes, we report a 30-year retrospective review at a high-volume level 4 epilepsy center. Consecutive epilepsy surgeries from 1989 to 2018 were analyzed and grouped into early (1989-2003) and late (2004-2018) 15-year eras. Demographic, clinical, radiographic, surgical, and 2-year Engel outcome data were collected and analyzed. Multivariable logistic regression assessed independent associations between era, patient characteristics, imaging findings, surgical procedures, and seizure outcomes. Of 1241 surgical records, 1128 pediatric cases were included (415 early, 713 late; 55.50% male). Patients in the late era were older at surgery (10.27 vs. 8.53 years, p < .001), and repeat surgery was more common (p < .001). Focal (OR 1.72, p = .005) and cerebral insult related (OR 2.74, p < .001) MRI abnormalities were more frequently treated in the late era. Hemispherectomies, lobectomies and corpus callosotomies were performed less often (all p ≤ .001), while focal and multilobar resections remained stable (p > .05). Use of invasive monitoring and electrical stimulation mapping declined over time (p < .001). Seizure freedom rates were stable, however, ≥ 90% seizure reduction was more common in the late era (OR 1.60, p = .038). Over three decades, pediatric epilepsy surgical volume increased, surgery shifted towards older patients, with reduced extensive resection and reduced reliance on invasive monitoring. Despite these changes, seizure freedom rates remained stable, with improved near-complete seizure control in the later era.
To compare postoperative outcomes of absorbable versus non-absorbable sutures and sutureless versus sutured incisions in pediatric cataract surgery (0-18 years). Pediatric cataract surgery requires precise wound closure to ensure postoperative stability. Identifying the most effective closure method is essential to minimize complications and the need for reintervention under general anesthesia. The review protocol was registered in the International Prospective Register of Systematic Reviews (CRD420251174549). We searched PubMed, Embase, Cochrane, and Web of Science (inception to October 2025). The primary outcome was the overall rate of postoperative complications. Meta analysis utilized Mantel-Haenszel fixed-effects model (Review Manager 5.4); certainty of evidence was assessed via Grading of Recommendations Assessment, Development and Evaluation (GRADE). Eight studies (four per comparison) evaluated suture material (321 absorbable; 430 non-absorbable) and closure technique (1,127 sutureless; 187 sutured closure). Absorbable sutures were associated with significantly lower odds of overall complications than non-absorbable sutures (OR 2.35; 95% CI 1.38 to 3.99; P=0.002; very low-certainty evidence). Specifically, 10-0 polyglactin 910 outperformed 10-0 nylon (OR 2.18; P=0.005; very low-certainty evidence), though loosening was more frequent (P=0.05). Sutureless closure was associated with significantly lower complication odds than sutured techniques (OR 0.34; 95% CI 0.23 to 0.49; P<0.00001; very low-certainty evidence). No significant differences were found for wound leakage (P=0.60) or endophthalmitis (P=0.10). Absorbable sutures and sutureless techniques are associated with a lower risk of overall complications. However, results are limited by very low-certainty and potential selection bias, as sutures are often reserved for leaking wounds. While non-absorbable sutures provide early wound security, they appear to increase long term ocular surface morbidity. Large scale RCTs are required to establish definitive surgical guidelines.
Resource-constrained programs perform epilepsy surgery under limited access to advanced imaging and neuromodulation. We describe presurgical evaluation findings, early seizure outcomes, and safety from a mixed adult-pediatric cohort. Retrospective single-center series of 22 consecutive patients meeting surgical candidacy. We captured demographics, epilepsy classification/etiology, presurgical investigations (long-term Video-EEG, MRI, selective FDG-PET), procedure type, histopathology when available, and postoperative seizure outcome (ILAE). Complications were recorded using the ILAE adverse-event taxonomy. Mean age at surgery was 21.2 ± 11.2 years; 15 (68%) were male. Epilepsy was focal in 15 (68%); structural etiologies predominated in 15 (68%). MRI was concordant with the presumed epileptogenic zone in 13 (59%). FDG-PET was obtained in 10 (45.5%) and was concordant in 7 (70%). Long-term Video-EEG (≥2 habitual seizures) was completed in 21. Mean delay to surgery was 10 years (IQR [8-15]); presurgical work-up averaged 10 months (IQR [6-15]). Procedures were resective 14 (64%), disconnective 6 (27%), and neuromodulatory 2 (9%). Histopathology was available in 16 cases, most commonly showing hippocampal sclerosis (n = 5) and focal cortical dysplasia (n = 5). At 14 months median follow-up (range 12-34), ILAE outcomes were: I 41% (9), II 14% (3), III 23% (5), IV 23% (5). Outcomes significantly differed by procedure: curative-intent (resection/disconnection) achieved ILAE I 60% (9) versus ILAE III/IV 7 after palliative-intent (corpus callosotomy/neuromodulation). No deaths or permanent deficits occurred; one corpus callosotomy case developed transient aseptic meningitis. In a resource-limited program, structured presurgical evaluation and careful selection yield resection/disconnection outcomes comparable to high-resource benchmarks, while corpus callosotomy/neuromodulation remain largely palliative. Practical, reproducible pathways may help shorten delays and improve access in similar settings.
The International Organization for the Study of Inflammatory Bowel Disease (IOIBD) is an international scientific organization that has shaped the framework for inflammatory bowel disease (IBD) research, clinical management strategy, therapeutic development, and clinical trial methodology for more than four decades. Formally constituted in April 1981 in Lyon, France, IOIBD was created to address fundamental barriers to scientific and clinical progress in IBD, including inconsistent definitions of disease activity and outcomes across studies and countries. Since the establishment of the IOIBD Foundation for Research and Education in 1997, IOIBD has combined a highly engaged global membership of experts with structured governance, continuously active thematic clusters, and an annual rotating international meeting to deliver consensus frameworks and collaborative initiatives that translate directly to clinical practice and regulatory and translational science. Key outputs include studies of global epidemiology of IBD, the Selecting Therapeutic Targets in IBD (Selecting Therapeutic Targets in Inflammatory Bowel Disease, STRIDE) treat-to-target programs; the SPIRIT consensus initiative addressing long-term disease impact and endpoints for disease-modification trials; validated approaches to capturing disability and patient-reported outcomes; consensus guidance on nutrition and diet as modifiable and potentially disease-modifying factors; recommendations to optimize IBD clinical trial design and endpoints; reclassification of IBD initiative; rapid international guidance during the COVID-19 pandemic; and educational initiatives including topic-focused satellite symposia, the Helmsley-IOIBD Clinical Experience Exchange Program, and the Empowering Women in IBD Leadership Program (EMPOWHER). This manuscript reviews IOIBD's history, operational model, selected scientific contributions, educational mission, and evolving strategy as the field moves toward precision medicine, globalization of care, and data-intensive approaches, including artificial intelligence. International organization for the study of inflammatory bowel diseases (IOIBD) Inflammatory bowel disease (IBD), including Crohn’s disease and ulcerative colitis, faces ongoing challenges. Although treatment has improved, many patients still face delayed diagnosis, ongoing inflammation, complications, surgery, and reduced quality of life. These challenges have also made it hard for researchers and clinicians worldwide to compare studies and agree on the best ways to measure improvement. This article reviews the work of International Organization for the Study of Inflammatory Bowel Disease (IOIBD), an international network of experts founded in 1981 to help solve these problems through collaboration and shared standards. IOIBD brings together clinicians and scientists from many countries and disciplines, works year-round in focused topic groups, and meets annually to develop practical guidance and shared definitions that can be used in research studies, clinical trials, and everyday care.
To determine predictors of early mortality among pediatric patients undergoing congenital heart surgery at a tertiary care center in Saudi Arabia. This retrospective cohort study included 955 pediatric patients who underwent congenital heart surgery at King Abdulaziz University Hospital, Jeddah, between January 2017 and December 2023. Demographic, clinical, operative, and postoperative variables were collected. Overall mortality was 4.9%. Mortality was higher among neonates and infants and among patients with RACHS categories 4-6. Non-survivors had longer cardiopulmonary bypass times and more frequent delayed chest closure. Postoperative complications, including the need for extracorporeal membrane oxygenation (ECMO), depressed cardiac function, acute kidney injury, central nervous system (CNS) insult, infection, and prolonged mechanical ventilation and intensive care unit (ICU) stay, were strongly associated with mortality. In multivariate analysis, independent predictors of mortality were prolonged ICU stay, longer postoperative mechanical ventilation, postoperative ECMO support, and postoperative CNS insult. Early mortality was comparable to international benchmarks. Postoperative physiological deterioration, particularly ECMO requirement, neurological injury, prolonged ventilation, and extended ICU stay, was independently associated with death, underscoring the importance of early detection and targeted perioperative management.
Severe treatment-resistant asthma (STRA) in children is often sustained by type 2 inflammation and eosinophil-dependent airway disease that persists despite optimized inhaled therapy and the mitigation of modifiable factors. This review summarizes the clinical and translational evidence on monoclonal antibodies targeting the interleukin-5 (IL-5) axis (anti-IL-5 and anti-IL-5Rα) available in pediatric severe asthma. PubMed/MEDLINE was searched up to January 2026 for English-language studies in patients aged 0-18 years addressing mepolizumab and benralizumab, including randomized trials, high-quality observational studies, meta-analyses, and international guidance. Mepolizumab has the most robust pediatric data, showing consistent reductions in exacerbations and blood eosinophils, and improvements in symptom control and quality of life, with safety broadly comparable to adults. The pediatric evidence for benralizumab is more limited but shows rapid eosinophil depletion, improved outcomes in selected children, and acceptable safety; further trials are ongoing. Overall, IL-5-directed biologics represent a key add-on option for carefully selected children with severe eosinophilic asthma, while pediatric-specific predictors of response, comparative effectiveness, and standardized long-term monitoring and stopping criteria remain priorities.
Molecular profiling is now integral to the diagnosis, risk stratification, and treatment of central nervous system (CNS) tumors following the 2021 WHO Classification (WHO CNS5). However, access to molecular diagnostics remains severely limited in settings with limited resources. International outsourcing to accredited reference laboratories represents a potential bridging strategy, yet systematic data on its feasibility and clinical impact in the LMICs are lacking. We conducted a retrospective review of CNS tumor cases at King Hussein Cancer Center (KHCC), Amman, Jordan, that underwent outsourced molecular testing at the Hospital for Sick Children in Toronto, Canada, between 2021 and 2023. Four test types were ordered: medulloblastoma subgrouping by NanoString nCounter-based gene expression profiling, TruSight pan-cancer RNA sequencing, low-grade fusion gene analysis, and C19MC fluorescence in situ hybridization (FISH). For each case, we recorded turnaround time (TAT), cost, conclusive result rate, impact on diagnosis, and identification of actionable therapeutic targets. A total of 105 patients underwent outsourced molecular tumor testing (87% pediatric; median age 10 years; 54% male). Tumor types included medulloblastoma (52%), low-grade glioma (LGG, 30%), high-grade glioma (HGG, 12%), and others (6%). Of the 119 samples that reached the reference laboratory, 109 (92%) yielded a conclusive molecular result, with the highest rate in medulloblastoma (94%) and the lowest in ependymoma (33%). The median overall TAT was 26 days (range 13-140 days), and the total expenditure was 153,506 US Dollars. Molecular testing led to a change in diagnosis in 4 cases (4%): 3 major changes (reclassification of BCOR sarcoma to solitary fibrous tumor, ependymoma to pilocytic astrocytoma, and pediatric-type diffuse low-grade glioma with COL1A1::PDGFB fusion) and 1 minor change. In one additional case, a discordant molecular result (NRAS-mutant low-grade glioma) did not alter the final morphological diagnosis of high-grade glioma. Actionable targets (predominantly BRAF alterations) were identified in 28 tumors (27%), including 65% of LGGs and 54% of HGGs. International outsourcing of molecular diagnostics is a feasible and clinically impactful strategy for CNS tumor management at limited-resource settings, enabling WHO CNS5-integrated diagnoses and access to precision therapy. The TAT and cost per tumor sample are relatively acceptable given the benefits of a more accurate diagnosis and the possibility of finding a targetable alteration. Permanent solutions would require investment in regional molecular diagnostic infrastructure through in-house capacity building, laboratory networks, and international twinning programs.
The Alisklamp® Nero M is a novel metallic disposable circumcision device allowing thermocautery-assisted excision, immediate device removal, and standardized mucosal cuff length. This study evaluated its real-world safety profile and complication rates in a large pediatric cohort. This retrospective study included 6000 boys aged 1 month to 12 years circumcised using the Alisklamp® Nero M between November 2020 and November 2025. Procedures were performed by a single experienced surgeon using a standardized local anesthesia technique. Postoperative follow-up was conducted through a structured smartphone-based telemedicine protocol until complete wound healing. Primary and secondary outcomes were overall complication and revision surgery rates, respectively. The mean patient age was 3.14 ± 3.13 years. No patients were lost to follow-up. A total of 130 complications (2.16%) occurred, predominantly wound dehiscence (0.95%) and postoperative bleeding (0.58%). Revision surgery was required in only 10 patients (0.16%) due to persistent bleeding (n = 5) or secondary phimosis (n = 5). Remaining complications were managed conservatively, yielding complete mucocutaneous healing in all patients. Circumcision with the Alisklamp® Nero M demonstrated low overall complication and exceptionally low revision surgery rates. Standardized excision, immediate device removal, and structured telemedicine follow-up support its safety, feasibility, and practicality in routine pediatric care.
Background: Neonatal open-heart surgery induces profound oxidative stress, yet its perioperative dynamics remain incompletely characterized. This study quantified urinary 8-hydroxy-2'-deoxyguanosine (8-OHdG) and 8-isoprostane as markers of oxidative damage, and plasma α1-microglobulin (A1M) as an endogenous antioxidant, while exploring the influence of pre- and intraoperative factors. Methods: In a prospective cohort of 40 term neonates with critical congenital heart defects undergoing open-heart surgery, serial urinary and plasma samples were collected perioperatively. Biomarker concentrations were analyzed using mixed-effects regression models to assess associations with postnatal age, sex, preoperative oxygenation, oxygen surge at bypass initiation, and cell-free hemoglobin in the prime solution. Results: Urinary 8-OHdG and 8-isoprostane increased following bypass separation; 8-OHdG remained elevated through postoperative days 0-2, while 8-isoprostane returned toward baseline by day 1. Plasma A1M declined at bypass initiation, recovered to preoperative levels at separation, and rose thereafter. Females exhibited higher A1M concentrations throughout. Longer time to surgery was associated with greater preoperative oxidative stress, and lower preoperative arterial pO2 correlated with increased 8-isoprostane at bypass separation. No statistically significant associations were identified between intraoperative variables and biomarker levels, although the study was designed to be a hypothesis generating study and not powered to detect modest intraoperative effects. Conclusions: This exploratory study delineates distinct perioperative trajectories of oxidative stress and antioxidant response in neonates undergoing open-heart surgery, with surgical timing, preoperative hypoxemia, and sex emerging as relevant associations. No statistically significant associations were identified between intraoperative variables and biomarker levels; however, the study was hypothesis-generating in design and not powered to detect modest intraoperative effects.
Pediatric head and neck rhabdomyosarcoma (HNRMS) is well characterized in Western cooperative group trials, but real-world outcomes from resource-constrained MENA settings are poorly documented. Whether the locally advanced-stage presentation typical of Middle Eastern referral centers precludes outcomes equivalent to international benchmarks is unknown. We conducted a retrospective analysis of pediatric patients younger than 18 years with non-metastatic HNRMS diagnosed between January 2001 and May 2025. We extracted demographic, pathological, treatment, and outcome data. Risk stratification and treatment planning followed COG protocols throughout, with local control decisions made via multidisciplinary team assessment. Among 98 patients with HNRMS, 77 (79%) had non-metastatic disease and were included in the analysis. The male-to-female ratio was 1:1, with a median age at diagnosis of 5.9 years (range, 0.2-18). The parameningeal region (n = 31, 40%) and the orbital region (n = 20, 26%) were the most common primary sites. Histology was embryonal in 51 patients (66%), and tumor size exceeded 5 cm in 55 patients (71%). Sixty patients (78%) were classified as intermediate risk and 93.5% were IRS Clinical Group III. All patients received chemotherapy, with 32% receiving maintenance therapy. Local excision was performed in 15 patients (19%), and 71 patients (92%) received radiotherapy. Twenty-three patients (30%) experienced relapse or progression, with 74% presenting with local recurrence or progression. The median follow-up time was 30.7 months (range, 2.6-294). The 5-year event-free survival (EFS) and overall survival (OS) were 66.1% (55.4%-79%) and 70.1% (59.1-83.1%), respectively. Subgroup analysis demonstrated variable 5-year OS rates according to primary site: orbital (91.7 ± 8%), parameningeal (51.2 ± 10.6%), and other HN primaries (76.1 ± 9.4%). Despite 93.5% IRS Clinical Group III and 71% tumors >5 cm-rates substantially exceeding Western cooperative group series-5-year OS of 70.1% was achieved. Parameningeal location was the only factor significantly associated with adverse survival outcomes in this cohort, driven by local failure. These findings demonstrate that outcomes equivalent to international benchmarks are attainable in a resource-constrained MENA setting, and identify local control intensification for parameningeal disease as the priority unmet need.
This systematic review and meta-analysis synthesised global prevalence, regimens, guideline adherence, and factors associated with antibiotic prescribing in dental implant surgery. MEDLINE, Scopus, and Web of Science were systematically searched from January 2010 to October 2025. Dentist surveys, patient-record studies, and register-based studies were included. Prevalence was pooled using random-effects meta-analysis with Freeman-Tukey transformation; regimen and appropriateness were narratively synthesised. Risk of bias and evidence certainty were assessed using JBI and GRADE tools. Thirty-nine studies (7266 dentists; 137,207 patients) were included. Pooled prevalence of routine antibiotic prescribing was 61.3% (95% CI: 53.4-68.8%; I²=96.8%) with extreme heterogeneity (24.9-88.8%). Combined pre- and post-operative regimens predominated (52.6%), typically involving 5-7-day post-operative courses. Amoxicillin was the most frequent choice (50.9%). Prescribing increased with procedural complexity, reaching 97.3% for compromised cases. Guideline-concordant prescribing was low (1-28%), with a declining temporal trend. Overall evidence certainty was very low. Antibiotic prescribing for implant surgery varied widely but was often high, frequently exceeding evidence-based indications. Post-operative regimens predominated despite no demonstrated benefit over a single pre-operative dose. Reducing unnecessary antibiotic use in implant surgery may require procedure-specific international guidelines and targeted stewardship, though more robust evidence is needed to determine effective strategies.
This study aims to describe the technical details of laser-assisted endoscopic pilonidal sinus treatment (LEPSIT), a method used for the first time in pediatric pilonidal sinus disease (PSD), and to report early-stage clinical outcomes in a pediatric cohort. Clinical data of children under 18 years of age who underwent LEPSIT for PSD between November 2021 and July 2023 were retrospectively analyzed. Of the 45 patients included, 19 (42.2%) were female, and 26 (57.8%) were male; the mean age at surgery was 15.2 ± 3.1 years, and the mean body mass index was 25.1 ± 3.2. Of the 45 patients, 35 presented with an acute pilonidal abscess at initial evaluation and had undergone abscess drainage prior to LEPSIT. Six patients had been previously operated on for pilonidal sinus disease at an external center prior to LEPSIT. Mean procedure duration was 30 ± 7.2 min. The mean visual analog scale (VAS) score at 6 h was 1.8, 24 h was 1,1 and the mean analgesic use was 1 ± 0.3 days. Successful treatment was achieved in 41 patients (91.1%) after a single procedure; 4 patients (8.9%) had recurrence, all of whom achieved cure after a second LEPSIT. Improvement was ultimately achieved in all 45 patients. Mean follow-up was 32.4 ± 11.3 months. LEPSIT is an easy-to-administer, minimally painful (mean VAS score: 1.1), and minimally invasive treatment that shortens hospital stay and enables rapid return to daily activities. It is also a safe and well-tolerated option for recurrent PSD.
Real-world data (RWD) from nonrandomized clinical settings, such as registries and observational cohorts, can be used to address research questions for which randomized controlled trials (RCTs) are not feasible or appropriate. However, these data are susceptible to important sources of bias, including confounding by indication and immortal time bias. Target trial emulation (TTE) has emerged as a framework to improve the quality, credibility, and reproducibility of observational causal inference by aligning the design and analysis of observational studies with the structure of a hypothetical RCT. Appropriate application of the TTE framework requires careful specification of key design elements. Recent methodological guidance documents and reporting guidelines now provide practical support for researchers seeking to specify, conduct, and report TTE studies. Although TTE is increasingly used in rheumatology, its application in pediatric rheumatology remains limited despite the expanding availability of RWD. This review details the most recent methodological developments and guidelines, outlining the core concepts needed to apply the framework appropriately. The aim of this review is also to summarize recent applications in adult and pediatric immune-mediated diseases, and to explore the opportunities and limitations of this approach with a specific focus on pediatric rheumatology.
Our aim was to identify the potential predictors of liver transplant in patients with autoimmune hepatitis based on data at initial presentation. We retrospectively evaluated records of children diagnosed with autoimmune hepatitis between 2000 and 2025. We excluded 21 patients with de novo autoimmune hepatitis (n =5) and incomplete data (n = 16). We compared demographic, clinical, biochemical, and histopathological data in 2 study groups: transplanted (n = 9) and not transplanted (n = 81). Of 90 included patients, 63 (70% ) were female. Median age at diagnosis was 115 months (range, 8-215 months). Among included patients, 70 had type 1 autoimmune hepatitis, 13 had type 2 autoimmune hepatitis, and 7 had seronegative autoimmune hepatitis. Nine patients underwent liver transplant (5 with type 1 autoimmune hepatitis, 4 with type 2 autoimmune hepatitis). Rate of transplant was significantly higher in patients with type 2 autoimmune hepatitis (P = . ⁰³ ). Compared with the nontransplant group, patients in the transplant group were more likely to have type 2 autoimmune hepatitis; higher gamma-glutamyl transferase, bilirubin, international normalized ratio, and immunoglobulin levels; and lower albumin and platelet levels. In addition, this group was more likely to have relapsing disease and moderate-to-severe fibrosis at diagnosis. Multivariate analysis showed that moderate-to-severe fibrosis and total bilirubin level were significant predictors of liver transplant (odds ratio: 21.84, 95%CI, 1.34-355.25; P = . ⁰³⁰ and odds ratio: 1.22, 95% CI, 1.02-1.47; P = . ⁰²⁹, respectively). Autoimmune hepatitis type 2 was not found to be associated with outcome (P = . ⁸³⁹). ). Systematic risk assessment at diagnosis is important to identify high-risk patients who may benefit from close monitoring, early escalation of therapy, and timely referral for transplant evaluation. Tailoring management strategies based on these predictive factors may improve long-term outcomes in pediatric autoimmune hepatitis.
Anorectal malformations (ARM) are rare congenital anomalies requiring complex surgical correction. Due to low caseload and heterogeneous cohorts, prior studies failed to establish whether higher hospital caseloads improve outcomes. This study applies the IQWiG V24-07 framework to evaluate whether indirect evidence from other surgical procedures can inform a potential volume-outcome relationship in ARM surgery. An interdisciplinary expert panel conducted a formal consensus using three steps: (1) identifying suitable source populations; (2) systematically comparing these with the ARM population using 12 IQWiG criteria; and (3) evaluating transferability through structured discussion and voting. Adult rectal resection was unanimously selected as evidence source population due to robust evidence of a volume-outcome relationship and relevant surgical commonalities, including deep pelvic dissection. Of the 12 transferability criteria, consensus was reached in six. Differences in comorbidities, concomitant treatment, and setting were judged irrelevant for transferability. Differences in sociodemographic and disease-specific characteristics were considered estimable in their impact. Divergent ratings were observed for diagnosis, intervention, endpoints, specialization, and follow-up care. Short-term technical outcomes such as anastomotic leakage were deemed transferable, whereas mortality was considered non-informative in ARM. Functional outcomes required subtype-specific interpretation. This structured analysis supports the existence of a volume-outcome relationship for ARM surgery.
We report the 5-year analysis of tisagenlecleucel in 79 pediatric and young adult patients with relapsed or refractory (r/r) B-cell ALL (B-ALL) from the global phase II ELIANA trial (ClinicalTrials.gov identifier: NCT02435849), with a median follow-up of 79.4 months. Tisagenlecleucel was administered as a single infusion with dosing normalized by weight in patients ≤50 kg. Key long-term end points included relapse-free survival (RFS), overall survival (OS), and safety. Censoring included loss to follow-up, withdrawal of consent, new anticancer therapy (± stem cell transplantation [SCT]), and death. The estimated 5-year RFS among responders (n = 70) with and without inclusion of SCT in censoring for new anticancer therapies was 47.3% and 51.0%, respectively. The median time to B-cell recovery was not reached with censoring for all further anticancer therapies, including SCT. The median OS was not reached. The estimated OS at 5 years was 55.0% and 62.4% with and without inclusion of SCT in censoring for new anticancer therapies, respectively. In total, 17 responders received a postinfusion SCT, 14 while still in complete remission. No new or unexpected adverse events were reported. These findings continue to support the potential of tisagenlecleucel as definitive therapy for many heavily pretreated pediatric and young adult patients with r/r B-ALL.
Artificial intelligence (AI) is revolutionising medicine. The aim of this study was to detail its use, opinions, knowledge, and concerns in rheumatology and paediatric rheumatology. A web-based survey open to all professionals working in the field was developed by the Emerging EULAR Network (EMEUNET) and disseminated between March and July 2025 in collaboration with other international rheumatology societies (AFLAR, ArLAR, CARRA, PAFLAR, PANLAR). The survey was divided into 4 sections: (i) participants' characteristics, (ii) AI use and applications, (iii) opinions and knowledge, and (iv) concerns, needs, and expectations. Overall, 461 responses were collected from 59 countries. Respondents were mostly physicians who completed their training (316, 68.7%) and were based in Europe (170, 36.9%). Most participants (397, 86.7%) used AI for medical purposes, especially large language models (385, 83.7%) for grammar correction and brainstorming. Although there was broad optimism about its use (366, 79.6%), self-reported practical skills were predominantly basic or still in development (346, 75.1%), and knowledge was rarely defined as strong or expert-level (63, 13.7%). Concerns focused on ethics (314, 69%), lack of trust (316, 69.5%), and insufficient training (270, 59.3%). Disparities emerged across geographic regions in use, knowledge, and practical skills. AI is widely used and positively perceived in rheumatology, despite limited knowledge and practical skills, and regional disparities. Addressing gaps in ethics, transparency, and insufficient training through targeted education and implementation strategies will be essential to ensure an equitable and effective integration into clinical and research practice.
Traditional preoperative fasting guidance (6-4-2) aims to reduce aspiration risk but frequently produces prolonged fasting and discomfort in children. We evaluated whether progressively liberalizing the clear-liquid fasting policy reduces fasting time and improves the perioperative experience. We conducted a prospective, four-phase interventional quality-improvement study at a tertiary hospital in rural Kenya (11/2021-12/2022). Phase 0 followed standard 6-4-2 guidelines; Phase 1 shortened clear-liquid fasting to 1 h; Phase 2 permitted a fixed volume of water until transfer to theatre; Phase 3, unrestricted water. The primary outcome was clear-liquid fasting time. Secondary outcomes were thirst, hunger, parental satisfaction, preoperative anxiety, and aspiration events. Among 253 children, median clear-liquid fasting time decreased: 14.0 h (Phase 0) to 1.7 h (Phase 3) (p < 0.001). Anxiety decreased significantly (mYPAS 35.4 to 22.9; p < 0.001) and thirst declined. Parental satisfaction was higher but did not reach statistical significance (8.0 to 9.0; p = 0.06). One confirmed aspiration occurred in Phase 1 (7.2-h fasting time). Reducing preoperative fasting improved comfort and lowered anxiety. No increase in clinically significant aspiration events was observed, although the study was not powered for rare events. An unrestricted clear-liquid protocol appears feasible and beneficial.