搜索结果：Pediatric transplantation

Donation after circulatory death (DCD) is an emerging heart transplantation (HT) strategy with improved waitlist and comparable post-transplant outcomes to donation after brain death (DBD) in adults. Pediatric DCD-HT is underutilized but gaining broader adoption. We assessed the impact of DCD listing on waitlist outcomes and graft survival in pediatric HT. We queried the OPTN (Organ Procurement and Transplantation Network)/UNOS (United Network for Organ Sharing) database for all pediatric primary isolated HT candidates between January 2021 and June 2024. Waitlist outcomes were compared by final listing type to account for crossovers. Offer dynamics were assessed by average interval between offers stratified by OPTN/UNOS status. Multivariable regression modeled offer frequency as a function of DCD listing. We compared 30-day survival between recipients of DCD and DBD organs. Among 2486 candidates, 86 were initially listed eligible for DCD and 2400 for DBD. DCD candidates had higher clinical acuity than DBD candidates. DCD listing was associated with significantly shorter intervals between offers across all statuses and increased offer rate by 134% (95% CI: 92%-186%). Waitlist outcomes did not differ significantly by final listing type. There was no difference in 30-day survival between DCD and DBD recipients. DCD listing in pediatric HT is associated with a shorter interval between offers and more frequent offers. Waitlist survival was similar between groups despite DCD candidates being sicker at listing. There was no difference in 30-day survival between DCD and DBD recipients. These findings suggest that broader adoption of pediatric DCD-HT can expand access to donor hearts without compromising early post-transplant outcomes.

Anti-thymocyte globulin (ATG) remains integral to graft-versus-host disease (GvHD) prophylaxis in allogeneic hematopoietic stem cell transplantation (HSCT), yet its optimal dosing is unresolved. Excessive immunosuppression can impair immune reconstitution and increase infection-related morbidity. This review summarizes evidence on the timing, dosing, and pharmacologic modeling of rabbit ATG (Thymoglobulin) in pediatric HSCT. Early investigations demonstrated that reduced ATG exposure, achieved through lower or earlier dosing, enhanced CD4 recovery and lowered viral reactivation rates, whereas higher exposure delayed immune recovery and increased severe infections. Pharmacokinetic and pharmacodynamic analyses identified absolute lymphocyte count and body weight and graft source as key determinants of ATG disposition, supporting the development of model-based dosing (MBD) strategies that individualize exposure according to patient and graft characteristics. Across multiple clinical studies, MBD achieved superior CD4 reconstitution, significantly reduced viral reactivation, and decreased chronic GvHD and graft failure without significantly increasing acute GvHD. Survival outcomes improved in patients reaching early CD4 recovery under individualized dosing. Optimal results were associated with high ATG exposure before transplantation and low exposure afterward. Collectively, current data indicate that MBD provides a rational, safe, and effective framework to balance GvHD prevention with immune recovery in pediatric HSCT. Further studies in peripheral blood and haploidentical transplantation settings are warranted to validate and expand this approach.

Pediatric lung transplantation is a life-saving procedure for children with end-stage lung disease. As immunosuppression and treatment have improved, so too have survival outcomes; however, pediatric lung transplant recipients still face challenges, including acute rejection, infection, and long-term issues such as chronic lung allograft dysfunction (CLAD). This review details early complications following pediatric lung transplantation and provides an explorative investigation of the mechanisms underlying acute allograft dysfunction, and current prevention and treatment strategies. Focus is then given to CLAD with an overview of the subtypes of CLAD, review of recognized risk factors, challenges around diagnostics, current treatment strategies, and importantly future research goals to aid in the better identification and characterization of CLAD. As our understanding of acute and CLAD has evolved over time so have prevention and treatment strategies, including the assessment and treatment of risk factors such as infection and aspiration. The diagnosis of CLAD, however, remains extremely challenging with limited treatment options. Future research should focus on the prevention of CLAD.

Double lung transplantation (DLTx) generally provides better survival than single lung transplantation (SLTx). However, SLTx recipients may often be more deconditioned, making direct comparisons challenging. The objective of this study is to investigate the transplant outcomes in patients waitlisted for both SLTx and DLTx to minimize differences in recipient background. The United Network for Organ Sharing Database was retrospectively analyzed. The patients who were waitlisted from December 2017 to February 2023, then removed from the waiting list in the same time periods, were included in the analysis. Pediatric lung transplantation, multiorgan transplantation, and lung retransplantation were excluded. Of 4056 patients who were waitlisted for both SLTx and DLTx, 2127 patients (52.4%) received DLTx, 1351 patients (33.3%) received SLTx, and 578 patients (14.3%) were removed from waiting list without receiving lung transplantation. Median days on the waitlist were 36 days (IQR 12-93) for DLTx and 43 days (IQR 15-120) for SLTx. The lung allocation score at the time of transplantation was higher in DLTx group (50.58 &#xb1; 19.02 vs 44.67 &#xb1; 14.80, p < 0.001). DLTx recipients had significantly higher rates of prolonged intubation at 72&#xa0;hours after transplantation (33.9% vs&#xa0;19.9%, p < 0.001) and reintubation (19.3% vs&#xa0;13.0%, p < 0.001). Median post-transplant length of hospital stay was longer in DLTx group (19 [IQR 14-33] vs 15 days [IQR 11-24], p < 0.001). However, DLTx group had significantly lower mortality than SLTx group (n = 3478, log-rank test p < 0.001). Among patients waitlisted for both DLTx and SLTx, DLTx recipients had lower post-transplant mortality, despite a more complicated immediate postoperative course compared to SLTx.

Extracorporeal membrane oxygenation (ECMO) before ventricular assist device (VAD) implantation as a "double-bridge" strategy to heart transplantation is often used for the most acutely ill patients with decompensated heart failure. This cohort of patients has worse outcomes than those with primary VAD. Early crossover from ECMO to VAD has shown to have better survival in adults who are double-bridged. This study aims to evaluate the outcomes of early versus late crossover in pediatric patients who were double-bridged using the Advanced Cardiac Therapies Improving Outcomes Network registry. All patients <18 years of age in the Advanced Cardiac Therapies Improving Outcomes Network database who were double-bridged were identified. Patients were categorized into early or delayed crossover groups. Univariate and multivariate Cox regression analysis identified independent risk factors of outcomes. Kaplan-Meier and competing risk analyses assessed survival. Of 1360 patients, 334 (24%) underwent double-bridging. Median ECMO support was 6 days, leading to an early crossover group (&#x2264;6 days) of ECMO (n = 168) and a delayed crossover group (>6 days) of ECMO support (n = 166). Univariate analysis showed that patients who were double-bridged were considerably sicker than the primary VAD group. Multivariable analysis revealed that a diagnosis of dilated cardiomyopathy/myocarditis and bridge to candidacy device intent were independent predictors of outcome. Duration of ECMO support/timing of crossover (early vs late) was not independently associated with outcomes. The timing of crossover from ECMO to VAD in pediatric patients subject to the double-bridge strategy does not affect outcomes. Focus on the patient selection and reversibility of risk factors rather than the duration of ECMO support may improve outcomes in this high-risk population.

Liver transplantation (LT) is standard of care for children with end-stage liver disease, but waitlist mortality remains high, especially among infants. ABO-incompatible (ABO-I) LT offers a strategy to expand the donor pool, though concerns over antibody-mediated rejection have limited widespread use. We analyzed outcomes of ABO-I LT recipients using the Society of Pediatric Liver Transplantation (SPLIT) registry from 2011-2022, a prospective, multicenter database capturing over 75% of pediatric LTs in the United States and Canada. Clinical characteristics, post-transplant complications, and graft and patient survival were compared between matched ABO-I and ABO-compatible (ABO-C) recipients. ABO-I transplant recipients were matched 1:5 with ABO-C recipients using year of transplant, age and clinical status at the time of transplant. A center-level survey assessed institutional practices regarding ABO-I LT. Among 3,372 pediatric LT recipients, 155 received ABO-I grafts and were matched to 775 recipients of ABO-C grafts. ABO-I recipients had higher rates of ventilator support, parenteral nutrition, and ICU care at time of transplant compared to ABO-C recipients. There was no statistically significant difference in three-year graft (87.8% vs. 92.6%, p=0.06) or patient survival (93.9% vs. 96.6%, p=0.11) between ABO-I and ABO-C groups. In children &#x2264;2 years of age, there was a higher incidence of early portal venous thrombosis in the ABO-I group (8.5% vs. 3.7%, p=0.025). Survey responses revealed substantial variability in center ABO-I eligibility criteria, desensitization protocols, and immunosuppressive strategies. Outcomes for pediatric ABO-I and ABO-C LT recipients within the SPLIT registry are comparable, supporting broader implementation of ABO-I LT to reduce pediatric waitlist mortality. Variability in institutional practices underscores the need for prospective studies to inform standardized protocols and optimize outcomes.

Compounded tacrolimus suspensions are frequently used in pediatric kidney transplant recipients who cannot reliably swallow capsules or who receive medication via a gastrostomy tube. Clinicians at our center observed unusually flat mini-pharmacokinetic (mini-PK) profiles in patients receiving liquid tacrolimus. We retrospectively analyzed 13 tacrolimus mini-PK profiles (C0, C1, C2, C4) from 7 pediatric kidney transplant recipients receiving compounded liquid tacrolimus, measured by LC-MS/MS in routine clinical care. As a contemporaneous comparator measured by the same assay, we assembled mini-PK profiles from pediatric recipients receiving capsule tacrolimus at our center (December 2024 onward). A historical capsule cohort from Berlin measured with Abbott Tacrolimus II immunoassay was used only as external context. Contemporary LC-MS/MS capsule profiles showed the expected early post-dose rise, whereas liquid profiles remained comparatively flat across the 0-4&#x2009;h window. In the historical capsule cohort, higher trough concentrations were associated with lower Cmax/Cmin ratios, consistent with a trough-dependent change in apparent PK shape. In this Quality Improvement (QI) analysis, pediatric patients receiving compounded liquid tacrolimus demonstrated flatter mini-PK profiles than contemporaneous capsule recipients when evaluated within the same analytical platform. These findings support closer attention to PK shape (not trough alone) in clinically complex children requiring liquid formulations and motivate prospective studies that control for gastrointestinal comorbidity, co-medications, and administration route.

Background/Objectives: Immunocompromised children undergoing chemotherapy or allogeneic hematopoietic stem cell transplantation (HSCT) for hematologic disorders face a high risk of serious, life-threatening infections caused by multidrug-resistant (MDR) bacteria. Cefiderocol is a novel siderophore cephalosporin, indicated for use in adult patients with MDR Gram-negative infections. Clinical data in immunocompromised children are limited. To report a multicenter real-life experience from the Infection Working Group of the Italian Pediatric Hematology and Oncology Association (IWG-AIEOP) on the use of cefiderocol in treating pediatric onco-hematologic patients with severe, high-risk infections. Methods: Multicenter retrospective collection of infectious episodes treated with cefiderocol, from January 2021 to December 2024, in patients 18 years or younger, after treatment for malignancies or undergoing HSCT in the AIEOP network, part of a prospective, observational study on the etiology and outcome of febrile episodes among 24 AIEOP centers (code NCT06419426). Results: Fifteen episodes of MDR, life-threatening Gram-negative infections treated with cefiderocol in 13 pediatric onco-hematologic patients were collected. There were eight males and five females, mainly affected by acute leukemia (six lymphoblastic and four myeloid, three other hematologic malignancies). The median age was 11.1 years (range 1-17.4 years), and the median weight was 37.8 kg (range 8-65). Bloodstream infection occurred in 10 of 15 episodes. Pseudomonas aeruginosa, Klebsiella pneumoniae, and Stenotrophomonas maltophilia were isolated in 11, 3, and 1 episodes, respectively. Notably, 11 of 15 isolated pathogens carried a metallo-beta-lactamase (MBL) gene (Verona integron-encoded, VIM, n = 10; New Delhi, NDM, n = 1). All patients achieved infection resolution and were alive and infection-free 90 days after infection onset. Conclusions: Cefiderocol was well tolerated and showed encouraging, favorable clinical outcomes, without serious adverse effects.

The gold-standard for the diagnosis and management of biliary complications, particularly biliary strictures in pediatric liver transplant (LT) recipients is percutaneous transhepatic cholangiogram (PTC) followed by biliary drainage (PTBD). Despite the established role of PTBD in managing biliary complications in pediatric LT recipients, limited data exist on post-procedural infection rates and optimal antibiotic prophylaxis strategies. The primary aim was to analyze the complications of PTBD in pediatric LT recipients. The secondary aim was to analyze factors associated with PTBD complications. We retrospectively reviewed the medical records of children having undergone LT between August 2004 and October 2020 in our center. Patients who developed a biliary complication treated by PTBD were selected. We then compared PTBD patients with complications to those without. Eleven patients underwent 35 percutaneous transhepatic biliary drainage (PTBD) sessions. Biliary atresia was the most frequent indication for LT in 6/11 (54%) children. Ten (10/11; 91%) received a left lateral segment, and all had undergone biliary-enteric anastomosis. Anastomotic stricture was the most common finding on PTC in 22/35 (63%) sessions. 25/35 (71%) PTBD were conducted using one-day antibiotic prophylaxis and 10/35 (29%) using extended-24-h antibiotherapy. Of 35 PTBD sessions, 17/35 (49%) experienced complications, primarily infectious (40%; 14/35). We compared different antibioprophylaxis regimens in 14 PTBD with infection. There was a trend to a lower incidence of infections in the extended-24-h antibiotherapy group (10%; 1/10) compared to the one-day antibiotic prophylaxis group (52%; 13/25) with a relative risk of 0.19 (0.03-1.28). PTBD is associated with a high rate of post-procedure infectious complications. These complications result in a high burden and morbidity. There was a trend to an association between extended-24-h antibiotherapy and the absence of infectious complications after PTBD.

Gastrointestinal graft-versus-host disease (GI GVHD) following hematopoietic stem cell transplant is typically managed with medical therapy, but surgery and angioembolization may be warranted in selected cases with life-threatening complications. Data on surgical and interventional radiology treatment in pediatric patients are limited. We retrospectively reviewed data on pediatric patients diagnosed with complicated intestinal GVHD between 2010 and 2024. Complicated GI GVHD was defined as biopsy-proven GI GVHD associated with one or more symptoms of surgical interest such as severe bleeding, bowel stricture or obstruction, pneumatosis intestinalis (PI), and intestinal perforation. Need for surgery and interventional radiology, GVDH-related mortality, chronic intestinal failure (CIF) rates, and postoperative morbidity were described. Eighteen patients met the inclusion criteria. Fourteen patients (78%) presented severe bleeding, eight patients (44%) with obstruction, and three patients (17%) with PI. Five cases required invasive procedures: four patients underwent surgery for obstruction, while one with severe bleeding had angioembolization of a mesenteric vessel. The remaining cases were managed conservatively. The most frequent complication after surgery was found to be CIF (80%). In the whole cohort, overall mortality was 67%, mortality directly related to complicated GI GVHD was 22%, and the prevalence of CIF was 78%. In pediatric patients affected by complicated GI GVHD, uncontrolled bleeding and critical stricture causing bowel obstruction are indications for angioembolization and surgery, while PI can be treated conservatively. Morbidity and mortality are high in this cohort of patients, highlighting the severity of this condition and the limited role of invasive procedures as emergency life-saving therapy.

Brain death in organ donors may trigger tissue injury that can negatively influence transplant outcome. We analyzed plasma metabolomic profiles in heart transplant donors and tested whether any donor metabolite may predict the risk of acute cellular rejection after heart transplantation. Plasma samples from 83 heart transplant donors and 20 healthy volunteers were profiled using quantitative targeted metabolomics of 102 metabolites. Plasma samples from another set of 48 heart transplant donors were used for validation. The plasma levels of 24 metabolites representing 24% of the targeted metabolites were significantly altered in brain-dead heart transplant donors, compared with healthy controls. Alterations in the purine metabolism pathway were most prominent: adenine, xanthosine, allantoin, xanthine, and inosine monophosphate were upregulated, whereas adenosine monophosphate and adenosine were downregulated, indicating an energy metabolism shift and oxidative stress. Donor plasma glycine levels predicted the risk of acute cellular rejection (concordance = 0.74, area under the curve = 0.75; P < .01) in both the training and the validation cohort within 1-year after transplantation. Donor plasma glycine levels moderately correlated with other plasma metabolites linked to collagen formation and extracellular matrix organization. Targeted metabolomics revealed altered purine metabolism in brain-dead heart transplant donors, suggesting increased energy demand and oxidative stress. Donor plasma glycine was identified as a risk predictor for acute cellular rejection after heart transplantation and may serve as a donor-side biomarker to guide posttransplant risk stratification and monitoring.

Hepatic venous outflow obstruction (HVOO) is a serious complication after liver transplantation, and endovascular/vascular intervention&#xa0;is an effective treatment option. Most IR procedures for HVOO are performed via transjugular or transfemoral venous approaches, while the use of the percutaneous transhepatic approach has rarely been reported. Here, we report a case of percutaneous transhepatic hepatic vein stent placement for HVOO after living donor liver transplantation (LDLT). A woman in her 50s who had undergone living donor liver transplantation two years ago was admitted to our hospital with weight gain and massive ascites. Contrast-enhanced computed tomography (CT) suggested HVOO, which was confirmed by CT arterial portography. Because selective catheterization of the hepatic vein via transjugular or transfemoral approach was unsuccessful, an ultrasound-guided percutaneous transhepatic approach using a two-step puncture technique with a 21-gauge fine needle was performed. Balloon venoplasty, followed by placement of two self-expanding stents, was performed across the stenotic segment. Hepatic venous outflow was restored immediately without procedure-related complications. Follow-up imaging demonstrated an improvement in hepatic congestion and sustained stent patency. This case highlighted percutaneous transhepatic hepatic vein stent placement as a useful alternative treatment for posttransplant HVOO when standard venous access is difficult.

Introduction: Asthma is one of the most common chronic diseases in childhood and represents a major global public health concern due to its high prevalence, healthcare burden, and impact on quality of life. Pediatric asthma is characterized by clinical and biological heterogeneity, reflected in variable airflow limitations and distinct inflammatory endotypes. Conventional diagnostic tools do not fully capture the metabolic mechanisms underlying lung function impairment and disease variability. Aim: This narrative review aims to synthesize evidence published linking metabolomic and breathomic signatures to lung function parameters in children with asthma. Methods: We searched PubMed, Scopus, and Google Scholar using predefined keywords including pediatric asthma, metabolomics, breathomics, volatile organic compounds, exhaled breath condensate, and lung function. The search covered publications from January 2015 to January 2026. Earlier studies were included when necessary for the conceptual or methodological context. We included human studies evaluating metabolomic or breathomic profiles in children (&#x2264;18 years) and reporting associations with lung function, severity, endotypes, or exacerbations. Duplicate records, adult-only studies, animal models, non-English publications, and conference abstracts without full data were excluded. Results: Alterations in lipid and sphingolipid metabolism, oxidative stress pathways, and purine metabolism were associated with airflow limitation and reduced FEV1. Breathomic analyses revealed associations between volatile profiles, small airway dysfunction, and inflammatory patterns. However, findings remain heterogeneous across biological matrices and analytical platforms. Conclusions: Metabolomic and breathomic profiling may complement conventional lung function assessment by providing additional mechanistic insight into pediatric asthma heterogeneity. Standardized methodologies, longitudinal validation, and integration within multi-omics approaches are required before routine clinical implementation.

Living donor liver transplantation (LDLT) has become an important therapeutic option for children with selected inherited metabolic and genetic cholestatic liver diseases (IM-GCLDs).However, evidence on disease-specific outcomes across different diagnostic categories remains limited, and we therefore conducted a single-center retrospective study with contemporaneous non-IM-GCLD pediatric LDLT recipients as a comparator to better contextualize transplant-related outcomes and disease-specific benefits. Among 21 children with IM-GCLDs, the median follow-up was 21 months; two patients died (one perioperatively from disseminated intravascular coagulation and one at 21 months from pneumonia-related multiorgan failure), and all others are alive with functioning grafts. Disease-specific manifestations, including neuropsychiatric symptoms, portal hypertension, metabolic crises, cholestasis, hyperbilirubinemia, and hyperammonemia, improved or resolved in almost all survivors.At 6 months after LDLT, in children&#x2009;<10 years, mean weight- and height-for-age Z-scores increased from -0.48 to 0.43 and from -0.76 to -0.01; in children&#x2009;&#x2265;10 years, mean height Z-scores increased from -1.49 to -0.53 while BMI Z-scores showed no significant change. Overall survival did not differ significantly between IM-GCLDs and non-IM-GCLD indications. Living donor liver transplantation in children with IM-GCLDs not only improves survival but also confers disease-specific benefits, including recovery of neurologic function, metabolic stabilization, relief of portal hypertension and cholestasis, and catch-up growth. These findings support LDLT as an important therapeutic option for IM-GCLDs, while diagnosis-tailored perioperative assessment and long-term management remain essential given the phenotypic heterogeneity.

This study reviewed pediatric cases managed by the Aeromedical Evacuation Squadron (AMES) of the Japan Air Self-Defense Force and analyzed patient characteristics. Pediatric transportation cases (n = 34) between 2006 and 2023 were reviewed. Data on patient age, main disease, transportation purpose and distance, and use of mechanical ventilators or extracorporeal membrane oxygenation (ECMO) were obtained by referring to the records. The average (standard deviation) patient age was 5.7 (5.8) years (range: 0-16 years), and 17 patients (50%) were younger than 1 year of age. Furthermore, 10 (58.8%) of these 17 children were younger than 7 months of age and 1 child was under 1 month of age. The most common diseases in the overall patient population were cardiovascular diseases (CVDs, n = 18) and respiratory diseases (RDs, n = 14). The purposes of transportation in cases of 17 patients with CVDs and 3 patients with RDs were the implantation of a ventricular assist device and lung transplantation, respectively. The average transportation distance was 453.7 (218.6) (range: 176.9-962.8) miles or 730.2 (351.8) (range: 284.7-1,549.5) km, and in 8 cases, the transportation distance was > 600 miles. Of the patients, 29 (85.3%) were fitted with a ventilator, of whom 8 received ECMO (6 with CVDs and 2 with RDs). In all cases, physicians from the transporting hospitals were on board. There were no cases of cardiac arrest during the transportation. AMES plays an important role, especially in the long-distance transportation of critically ill children.

X-linked hyper-IgM syndrome (XHIGM) is a rare immunodeficiency for which allogeneic hematopoietic stem cell transplantation (HSCT) remains the only curative option, yet large pediatric Chinese datasets are scarce. We retrospectively analyzed 71 boys with genetically confirmed CD40L deficiency who underwent allogeneic HSCT between 2009 and 2020 at eight centers in China. Median age at HSCT was 3 years (range, 0.6-17), with 74.6% transplanted before age 5. Donors were mismatched unrelated (42.3%), matched unrelated (33.8%), matched sibling (18.3%), and haploidentical (5.6%). Peripheral blood was the predominant stem cell source (67.6%), followed by cord blood (31.0%). Myeloablative conditioning was used in 95.8%. Median times to neutrophil and platelet engraftment were 12 and 14 days, respectively. Primary graft failure occurred in 5.6%. Acute GVHD developed in 60.6% (grade III-IV, 11.3%), and chronic GVHD in 25.4% (none-severe). CMV and EBV-DNAemia occurred in 56.3% and 39.4%, respectively; post-transplant lymphoproliferative disorder developed in 4.2%. Other complications included sinusoidal obstruction syndrome and hemorrhagic cystitis (7.0% each). At last follow-up, 9 patients (12.1%) had died, mainly from infections (8.5%). At a median follow-up of 7.8 years, the estimated 10-year overall, disease-free, and GVHD-free survival rates were 85.91%, 64.79%, and 77.14%, respectively. Survival was superior in younger patients, those with lower body weight, and well-matched related or unrelated donors. Immune reconstitution showed early NK recovery with progressive T and B cell restoration over one year. These data support HSCT as effective long-term therapy for XHIGM and highlight benefits of early timing and optimal HLA matching.

Background Recurrence of focal segmental glomerulosclerosis (rFSGS) remains a major complication and a challenge to study treatment efficacy due to lack of granular data in a sufficient sample size. Aggregated data from electronic health records can provide such data. Methods We applied computational phenotypes to data from 11 large pediatric health systems in the USA, to identify treatments used and remission outcomes in children with rFSGS after renal transplantation. Additional data were collected by chart review. We performed both linear and non-linear multivariable Cox regression analyses with penalized splines to allow for time-varying predictors. Based on effect sizes from the hazard ratios, we then calculated a sample size needed for a future randomized clinical trial. Results Plasmapheresis was used in 101/107 (94%) patients, followed by anti-CD20 agents in 84 (78%), Low-Density-Lipoprotein (LDL)-apheresis in 22 (20%) and CTLA4Igs in 8 (7%). In linear multivariable models, complete remission was associated with more plasmapheresis sessions. In non-linear models, more doses or sessions of all the above treatments were associated with complete remission or any remission (partial or complete). Penalized spline curves for complete or any remission showed greatest yield within 5 doses of anti-CD20 agents but increasing yield with more doses/sessions of CTLA4Igs or LDL-apheresis. Based on observed hazard ratios, a prospective randomized trial of plasmapheresis vs LDL-apheresis would require 155 participants to have 80% power. Conclusions Increased doses/sessions or additional therapies for rFSGS associated with more favorable outcomes. Non-linear modelling identified when further increases did not improve outcomes.

Purpose: The purpose of this study was to evaluate the feasibility of magnetic resonance imaging (MRI)-based radiomics derived from routine T2-weighted imaging for initial risk stratification in pediatric neuroblastoma (NB) and to explore its potential role as a noninvasive adjunct to established clinical and molecular classification systems. Methods: In this retrospective, single-center pilot study, 45 children with newly diagnosed NB (2015-2024) were analyzed. Primary tumors were manually segmented on baseline axial T2-weighted MRI. A total of 107 Image Biomarker Standardization Initiative (IBSI)-compliant radiomic features were extracted. Supervised machine learning classifiers (Random Forest, XGBoost) and dimensionality reduction approaches (principal component analysis [PCA], linear discriminant analysis [LDA]) combined with K-means clustering were evaluated. Model performance was assessed using stratified cross-validation and an independent test set. Reporting adhered to the Checklist for Evaluation of Radiomics Research (CLEAR). Results: Fifteen patients (33%) were classified as high-risk (HR) and 30 (67%) as non-high-risk (NHR) according to International Neuroblastoma Risk Group (INRG) criteria. The highest classification performance was achieved using LDA followed by K-means clustering, with a test accuracy of 77.8%, sensitivity of 64.7%, and specificity of 85.7%. Radiomic classification agreed with conventional risk stratification in 77.8% of cases. The analysis relied exclusively on T2-weighted imaging, supporting workflow feasibility without requiring contrast administration or advanced MRI sequences. Conclusions: In this single-center pilot study, T2-weighted MRI radiomics demonstrated feasibility for noninvasive initial risk stratification in pediatric NB. Although limited by sample size and the lack of external validation, these findings support further multicenter investigations of radiomics as an adjunctive imaging biomarker during early diagnostic evaluation.

Retrospective studies showed a reduction of non-anastomotic strictures (NAS) after cDCD liver transplantation using normothermic regional perfusion (NRP). In the absence of an RCT, a systematic radiological follow-up of recipients is warranted to validate these findings. This single-center retrospective study (01/2015-01/2024) includes all consecutive cDCD liver transplantations performed after A-NRP at a single center. Recipients were included in a prospective radiological follow-up protocol with magnetic resonance cholangiopancreatography (MRCP) to identify NAS, which were defined as strictures of intra- or extra-hepatic bile ducts in the absence of arterial thrombosis. Seventy-one NRP cDCD LTs were included in the study of which 66 (93%) underwent a follow-up MRCP after a median of 180 days. We diagnosed 1 asymptomatic radiological NAS (1.4%) with minor severity (grade E) not requiring treatment. The rate of symptomatic anastomotic biliary complications was 30% (n=21). After a median follow-up of 39 months, we recorded no graft loss related to biliary complications. One- and 5-year graft and recipient survival rates were 94.4% and 85.9%, and 95.8% and 84.6% respectively. This single center 10-year experience with A-NRP for cDCD LT and a prospective MRCP follow-up of recipients shows <2% of NAS with no graft loss related to biliary complications.