Indications and successes of intestinal transplantation in children in the 21st century: A retrospective cohort study.

Aims: To report the results and successes of intestinal transplantation (ITx) in the most active European centres, to emphasize that, although it is a difficult procedure, it should remain a therapeutic option for children with total, definitive and complicated intestinal failure when intestinal rehabilitation fails.

Methods: We retrospectively collected data about all patients less than 18 receiving an ITx from 2010 to 2022 in 8 centres, and outcomes in July 2022.

Results: ITx was performed in 155 patients, median age 6.

Association between prehospital ROX index with 30-day mortality among septic shock.

Purpose: Respiratory dysfunction is one of the most frequent symptoms observed during sepsis reflecting hypoxemia and/or acidosis that may be assessed by the ROX index (ratio of oxygen saturation by pulse oximetry/fraction of inspired oxygen to respiratory rate). This study aimed to describe the relationship between the prehospital ROX index and 30-day mortality rate among septic shock patients cared for in the prehospital setting by a mobile intensive care unit (MICU).

Methods: From May 2016 to December 2021, 530 septic shock patients cared for by a prehospital MICU were retrospectively analysed.

Glyburide confers neuroprotection against age-related macular degeneration (AMD).

Glyburide, a sulfonylurea drug used to treat type 2 diabetes, boasts neuroprotective effects by targeting the sulfonylurea receptor 1 (SUR1) and associated ion channels in various cell types, including those in the central nervous system and the retina. Previously, we demonstrated that glyburide therapy improved retinal function and structure in a rat model of diabetic retinopathy. In the present study, we explore the application of glyburide in non-neovascular ("dry") age-related macular degeneration (AMD), another progressive disease characterized by oxidative stress-induced damage and neuroinflammation that trigger cell death in the retina.

Determinants of cognitive dysfunction in adults with sickle cell-related stroke or suspected neurological morbidity.

The prognosis of sickle cell disease (SCD) in adults is determined primarily by damage to targeted organs such as the brain. Cognitive dysfunction in SCD is a common chronic neurological manifestation, but studies remain mostly descriptive in adults. The objective of this study was to better characterize the cognitive profile and the association between cognitive dysfunction and brain lesions.

Insulin-degrading enzyme inhibition increases the unfolded protein response and favours lipid accumulation in the liver.

Background And Purpose: Nonalcoholic fatty liver disease refers to liver pathologies, ranging from steatosis to steatohepatitis, with fibrosis ultimately leading to cirrhosis and hepatocellular carcinoma. Although several mechanisms have been suggested, including insulin resistance, oxidative stress, and inflammation, its pathophysiology remains imperfectly understood. Over the last decade, a dysfunctional unfolded protein response (UPR) triggered by endoplasmic reticulum (ER) stress emerged as one of the multiple driving factors.

Islet cell stress induced by insulin-degrading enzyme deficiency promotes regeneration and protection from autoimmune diabetes.

Tuning of protein homeostasis through mobilization of the unfolded protein response (UPR) is key to the capacity of pancreatic beta cells to cope with variable demand for insulin. Here, we asked how insulin-degrading enzyme (IDE) affects beta cell adaptation to metabolic and immune stress. C57BL/6 and autoimmune non-obese diabetic (NOD) mice lacking IDE were exposed to proteotoxic, metabolic, and immune stress.

Prophylactic treatment of FSGS recurrence in patients who relapsed in a previous kidney graft.

Background And Hypothesis: Recurrence of focal segmental glomerulosclerosis (FSGS) is common after kidney transplantation and is classically associated with a significant decrease in graft survival. A major risk factor is a prior history of FSGS recurrence on a previous graft. This analysis reports the impact of a prophylactic treatment of FSGS recurrence in very high-risk patients who experienced a recurrence on a previous graft.

[Pediatric myasthenia with ocular involvement].

Purpose: Myasthenia is a rare disease in children, with an estimated incidence of 1 to 5 per million children. However, the potential severity of its consequences and the existence of specific treatments require prompt diagnosis by pediatric ophthalmologists.

Methods: Retrospective review of patient records.

Expanding safety boundaries in pediatric robotic-assisted laparoscopic surgery: are we protecting our children?

Little is known about the effects of CO insufflation (CDI) on cerebral oxygen saturation (CrSO) during laparoscopy in the pediatric population. In children undergoing robotic-assisted laparoscopic pyeloplasty (RALP), we prospectively assessed the effects of CDI using standard monitoring and cerebral near-infrared spectroscopy (NIRS). We also explored whether a correlation existed between CrSO and parameters known to affect cerebral blood flow.

Efficacy and safety of odevixibat in patients with Alagille syndrome (ASSERT): a phase 3, double-blind, randomised, placebo-controlled trial.

Background: In patients with Alagille syndrome, cholestasis-associated clinical features can include high serum bile acids and severe pruritus that can necessitate liver transplantation. We aimed to evaluate the efficacy and safety of the ileal bile acid transporter inhibitor odevixibat versus placebo in patients with Alagille syndrome.

Methods: The ASSERT study was a phase 3, double-blind, randomised, placebo-controlled trial that enrolled patients at 21 medical centres or hospitals in ten countries (Belgium, France, Germany, Italy, Malaysia, the Netherlands, Poland, Türkiye, the UK, and the USA).

3D pre-operative planning and patient-specific guides for re-directional peri-acetabular osteotomy in children and adolescents.

Surgical procedures to correct hip dysplasia associated with subluxation or dislocation of the femoral head are complex. The 3D geometric abnormalities of the acetabulum and proximal femur vary across patients. We, therefore, suggest a patient-specific surgical treatment involving computer-assisted 3D planning of the peri-acetabular osteotomies, taking into account the femoral head position; 3D printing of patient-specific guides for the cuts, repositioning, and fixation; and intra-operative application of the simulated displacements with their fixation.

The chain of survival and rehabilitation for sepsis: concepts and proposals for healthcare trajectory optimization.

This article describes the structures and processes involved in healthcare delivery for sepsis, from the prehospital setting until rehabilitation. Quality improvement initiatives in sepsis may reduce both morbidity and mortality. Positive outcomes are more likely when the following steps are optimized: early recognition, severity assessment, prehospital emergency medical system activation when available, early therapy (antimicrobials and hemodynamic optimization), early orientation to an adequate facility (emergency room, operating theater or intensive care unit), in-hospital organ failure resuscitation associated with source control, and finally a comprehensive rehabilitation program.

CNS tumors with PLAGL1-fusion: beyond ZFTA and YAP1 in the genetic spectrum of supratentorial ependymomas.

A novel methylation class, "neuroepithelial tumor, with PLAGL1 fusion" (NET-PLAGL1), has recently been described, based on epigenetic features, as a supratentorial pediatric brain tumor with recurrent histopathological features suggesting an ependymal differentiation. Because of the recent identification of this neoplastic entity, few histopathological, radiological and clinical data are available. Herein, we present a detailed series of nine cases of PLAGL1-fused supratentorial tumors, reclassified from a series of supratentorial ependymomas, non-ZFTA/non-YAP1 fusion-positive and subependymomas of the young.