T cell malignancies after CAR T cell therapy in the DESCAR-T registry.

The risk of T cell malignancies after chimeric antigen receptor (CAR) T cell therapy is a concern, although the true incidence remains unclear. Here we analyzed the DESCAR-T registry database, encompassing all pediatric and adult patients with hematologic malignancies who received CAR T cell therapy in France since 1 July 2018. Of the 3,066 patients included (2,536 B cell lymphoma, 162 B cell acute lymphoblastic leukemia (ALL) and 368 multiple myeloma), 1,680 (54.

[Vaccination of children and adolescents treated for acute leukemia, excluding HSCT recipients: Recommendations of the French Society for Childhood and Adolescent Cancer and Leukemia (SFCE)].

Children and adolescents who are being treated or have been treated for acute leukemia have a secondary immunodeficiency linked to chemotherapy, resulting in an increased risk of infections. Some of which can be prevented by vaccination but its effectiveness is not optimal during chemotherapy. Upon cessation of chemotherapy, the time required for immune reconstitution varies from three months to more than a year, depending on lymphocyte subpopulations, the patient's age, and the intensity of the treatment received.

Characteristics and Popularity of Videos of Abusive Head Trauma Prevention: Systematic Appraisal.

Background: Numerous strategies for preventing abusive head trauma (AHT) have been proposed, but controlled studies failed to demonstrate their effectiveness. Digital tools may improve the effectiveness of AHT prevention strategies by reaching a large proportion of the adult population.

Objective: This study aimed to describe the characteristics of videos of AHT prevention published on the internet, including their quality content, and to study their association with popularity.

Health-related quality of life, social and psychological well-being of 109 adult patients with genetic lipodystrophy.

Introduction: Lipodystrophy syndromes are rare diseases characterized by a generalized or partial lipoatrophic morphotype and metabolic complications. Data on health-related quality of life and impact of genetic lipodystrophy on social or psychological well-being are lacking.

Patients And Methods: Patients with genetic lipodystrophy were recruited throughout the French national reference network for rare diseases of insulin secretion and insulin sensitivity.

Discrimination against adolescents with chronic diseases: a systematic review.

Unlabelled: Discrimination is a social construct that discredits individuals based on attributes deemed socially undesirable. Adolescence is a period of transition where individuals acquire skills, values, and experiences that prepare them for adulthood. Adverse experiences during adolescence could particularly affect these acquisitions.

Treatment of septic arthritis of the hip in children.

Septic arthritis of the hip (SAH) in children is a common pediatric ailment that must be diagnosed immediately as proper treatment is needed to ensure good outcomes. It mostly affects children less than 2 years of age. The causative bacteria depend on age.

Endocrine management of transgender adolescents: Expert consensus of the french society of pediatric endocrinology and diabetology working group.

Introduction: Requests for hormonal transition in minors are increasing. To date, there is no national recommendation to guide these practices in France. Therefore, the SFEDP (French Society of Pediatric Endocrinology and Diabetology) has commissioned a group of experts to draft the first national consensus on this topic.

A community-based peer-facilitated psychological and social support model to improve retention in care among Cameroonian adolescents perinatally infected with human immunodeficiency virus: A randomized controlled trial.

Background: Psychological and social support for adolescents living with HIV remains undocumented and unaddressed in Central Africa. This study aimed at assessing effectiveness of a peer-facilitated community-based support model in improving retention in care among adolescents living with HIV and attending care in Chantal Biya Foundation, Yaounde, Cameroon.

Materials And Methods: We conducted an analysis of adolescents aged 10-19 years old, perinatally infected with HIV, on follow-up in the Day Care Unit of a reference hospital in Yaounde, Cameroon, and enrolled in the IAS-CIPHER-2021/1214-ATE-SMAVI, a individually randomized controlled trial.

Home-Based Connected Devices Combined With Statistical Process Control for the Early Detection of Respiratory Exacerbations by Patients With Cystic Fibrosis: Pilot Interventional Study With a Pre-Post Design.

Background: Currently, patients with cystic fibrosis do not routinely monitor their respiratory function at home.

Objective: This study aims to assess the clinical validity of using different connected health devices at home to measure 5 physiological parameters to help prevent exacerbations on a personalized basis from the perspective of patient empowerment.

Methods: A multicenter interventional pilot study including 36 patients was conducted.

[Antibiotic treatment of invasive meningococcal infections].

ANTIBIOTIC TREATMENT OF INVASIVE MENINGOCOCCAL INFECTION. Invasive meningococcal infections (IMI) are extremely severe pathologies that justify very early antibiotic therapy to limit complications and death. Three different situations may arise: 1) clinical suspicion of purpura fulminans in the pre-hospital setting, 2 confirmed or strongly suspected IMI, 3) post-exposure chemoprophylaxis of a patient's contacts.

[Diagnosis of invasive Neisseria meningitidis infections].

DIAGNOSIS OF INVASIVE NEISSERIA MENINGITIDIS INFECTIONS. Invasive meningococcal infections are unpredictable, difficult to diagnose and extremely serious, with a high risk of death and sequelae in survivors. They primarily affect subjects with no underlying pathology, but risk factors have been identified.

Patient follow-up after discharge from the paediatric intensive care unit: A scoping review.

Background: Most children admitted to a paediatric intensive care unit (PICU) now survive because of improvements in care. Many studies have identified the psychological, functional, cognitive and social impact of PICU admission on a child and their family. However, expert recommendations on follow-up are lacking.

The expanding field of genetic developmental and epileptic encephalopathies: current understanding and future perspectives.

Recent advances in genetic testing technologies have revolutionised the identification of genetic abnormalities in early onset developmental and epileptic encephalopathies (DEEs). In this Review, we provide an update on the expanding landscape of genetic factors contributing to DEEs, encompassing over 800 reported genes. We focus on the cellular and molecular mechanisms driving epileptogenesis, with an emphasis on emerging therapeutic strategies and effective treatment options.

[Development and validation of a tool for the systematic identification of social vulnerabilities in cancer patients: the DEFCO tool].

Introduction: Literature suggests that patients from deprived backgrounds are less likely to adhere to their treatments, continue to expose themselves to risk factors and, as a result, have poorer health outcomes. It is therefore crucial to identify these vulnerable populations early on, in order to provide them with tailored and reinforced care. The primary aim of this research is to construct and validate a systematic screening tool for identifying patients at highest risk of social vulnerability due to deprivation, through the use of psychometric techniques.

End-of-life medical decisions in French overseas departments: results of a retrospective survey.

Background: French laws governing end-of-life medical practices forbid euthanasia and affirm patients' right to deep and continuous sedation until death. Cultural traditions and disparities in health care provision, as in overseas France, could limit the enforcement of such laws and modify end-of-life medical practices.

Aim: This research aims to describe end-of-life medical decisions in overseas France and to compare with those described in mainland France.

Laparoscopic Treatment of Wilms' Tumor: Criteria of SIOP-UMBRELLA Protocol may be Updated.

Introduction: Total nephrectomies for the treatment of Wilms' tumor (WT) are more and more performed by laparoscopy, although indications for this approach following the UMBRELLA guidelines are currently very restrictive. The purpose of this study was to assess the compliance to the criteria of the UMBRELLA protocol for minimally invasive approach of WT.

Methods: This retrospective multicenter study included children operated on by laparoscopic total nephrectomy for suspected WT before 2020.

Dominantly acting variants in ATP6V1C1 and ATP6V1B2 cause a multisystem phenotypic spectrum by altering lysosomal and/or autophagosome function.

The vacuolar H-ATPase (V-ATPase) is a functionally conserved multimeric complex localized at the membranes of many organelles where its proton-pumping action is required for proper lumen acidification. The V-ATPase complex is composed of several subunits, some of which have been linked to human disease. We and others previously reported pathogenic dominantly acting variants in ATP6V1B2, the gene encoding the V1B2 subunit, as underlying a clinically variable phenotypic spectrum including dominant deafness-onychodystrophy (DDOD) syndrome, Zimmermann-Laband syndrome (ZLS), and deafness, onychodystrophy, osteodystrophy, intellectual disability, and seizures (DOORS) syndrome.

Adult patients treated for bladder exstrophy at a young age What are their current demands?

Introduction: Bladder exstrophy-epispadias complex is a rare condition that necessitates numerous surgical procedures during a patient's youth to achieve adequate urine storage and continence. This study aimed to identify the specific needs and functional challenges faced by adults who underwent pediatric bladder exstrophy reconstructions and assess the management of these issues in an adult population.

Methods: A retrospective chart review was conducted for all bladder exstrophy complex patients who underwent surgery at a young age and were subsequently referred to our center between 2005 and 2020.

Phase 3 Trial of Crinecerfont in Pediatric Congenital Adrenal Hyperplasia.

Background: Children with classic congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency require treatment with glucocorticoids, usually at supraphysiologic doses, to address cortisol insufficiency and reduce excess adrenal androgens. However, such treatment confers a predisposition to glucocorticoid-related complications. In 2-week phase 2 trials, patients with CAH who received crinecerfont, a new oral corticotropin-releasing factor type 1 receptor antagonist, had decreases in androstenedione levels.

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.