Apixaban overdose in children: case report and proposed management. A brief communication from the Pediatric and Neonatal Thrombosis and Hemostasis SSC of ISTH.

Background: Direct oral anticoagulants are commonly prescribed for adults and increasingly also for children requiring anticoagulation therapy. While household medications should not be accessible to children, accidental, and intentional overdoses occur.

Key Clinical Question: How should apixaban overdose in children be managed?.

Generic PROMIS item banks in adults with hemophilia for patient-reported outcome assessment: Feasibility, measurement properties, and relevance.

Background: Legacy hemophilia-specific questionnaires are considered too long, show floor-/ceiling effects, and/or include irrelevant questions. Patient Reported Outcomes Measurement Information System (PROMIS) item banks, including Computer Adaptive Tests (CATs) and short forms, were designed for more efficient outcome assessment.

Objectives: Evaluate the feasibility, measurement properties, and relevance of seven PROMIS CATs and two short forms in patients with hemophilia.

IL-10 signaling in dendritic cells controls IL-1β-mediated IFNγ secretion by human CD4 T cells: relevance to inflammatory bowel disease.

Uncontrolled interferon γ (IFNγ)-mediated T-cell responses to commensal microbiota are a driver of inflammatory bowel disease (IBD). Interleukin-10 (IL-10) is crucial for controlling these T-cell responses, but the precise mechanism of inhibition remains unclear. A better understanding of how IL-10 exerts its suppressive function may allow identification of individuals with suboptimal IL-10 function among the heterogeneous population of IBD patients.

First Vault Expansion in Apert and Crouzon-Pfeiffer Syndromes: Front or Back?

Background: Children with Apert and Crouzon-Pfeiffer syndromes are at risk of intracranial hypertension. Until 2005, when the authors switched to occipital expansion, their institution's preferred treatment was fronto-orbital advancement. However, it was still unclear whether (1) occipitofrontal head circumference (i.

Can we predict efficacy of the ketogenic diet in children with refractory epilepsy?

Background: The ketogenic diet (KD) can be effective in reducing seizures in children. Predictors of success have not been identified yet.

Aims: To evaluate efficacy of KD treatment and to search for child- or diet-related factors that can predict its efficacy at 12 months follow-up.

Algorithm for the Management of Intracranial Hypertension in Children with Syndromic Craniosynostosis.

Background: The purpose of this study was to examine the relationship of head growth, obstructive sleep apnea, and intracranial hypertension in patients with syndromic or complex craniosynostosis, and to evaluate the authors' standardized treatment protocol for the management of intracranial hypertension in these patients.

Methods: The authors conducted a prospective observational cohort study of patients with syndromic craniosynostosis at a national referral center, treated according to a standardized protocol. Measurements included occipitofrontal head circumference, with growth arrest defined as downward deflection in occipitofrontal head circumference trajectory greater than or equal to a 0.

Quality of life in adulthood after resection of a sacrococcygeal teratoma in childhood: a Dutch multicentre study.

Objective: Children treated for sacrococcygeal teratoma (SCT) may experience functional sequelae later in life. It is not known whether SCT and associated problems affect the patient's general quality of life (QoL). In a national survey, we evaluated general QoL in adults treated for SCT during childhood and compared the results to reference values for the Dutch population.

Atypical presentation of a newborn with Apert syndrome.

Introduction: Apert syndrome is a rare syndrome characterized by a consistent phenotype including bilateral coronal suture synostosis with an enlarged anterior fontanel, midface hypoplasia, and complex symmetric syndactyly of hands and feet.

Case Report: We present a boy with Apert syndrome caused by the pathogenic c.755C > G p.

Bone marrow immunophenotyping by flow cytometry in refractory cytopenia of childhood.

Refractory cytopenia of childhood is the most common type of childhood myelodysplastic syndrome. Because the majority of children with refractory cytopenia have a normal karyotype and a hypocellular bone marrow, differentiating refractory cytopenia from the immune-mediated bone marrow failure syndrome (very) severe aplastic anemia can be challenging. Flow cytometric immunophenotyping of bone marrow has been shown to be a valuable diagnostic tool in differentiating myelodysplastic syndrome from non-clonal cytopenias in adults.

T-cell receptor Vβ skewing frequently occurs in refractory cytopenia of childhood and is associated with an expansion of effector cytotoxic T cells: a prospective study by EWOG-MDS.

Immunosuppressive therapy (IST), consisting of antithymocyte globulin and cyclosporine A, is effective in refractory cytopenia of childhood (RCC), suggesting that, similar to low-grade myelodysplastic syndromes in adult patients, T lymphocytes are involved in suppressing hematopoiesis in a subset of RCC patients. However, the potential role of a T-cell-mediated pathophysiology in RCC remains poorly explored. In a cohort of 92 RCC patients, we prospectively assessed the frequency of T-cell receptor (TCR) β-chain variable (Vβ) domain skewing in bone marrow and peripheral blood by heteroduplex PCR, and analyzed T-cell subsets in peripheral blood by flow cytometry.

Therapeutic drug monitoring of indinavir and nelfinavir to assess adherence to therapy in human immunodeficiency virus-infected children.

Introduction: Adherence to highly active antiretroviral therapy is required to obtain an optimal long term virologic response rate of HIV-1-infected children. Plasma concentrations of protease inhibitors (PIs) outside the limits of the reference values indicate nonadherence to antiretroviral therapy in adults. We studied during a 2-year follow-up period routinely taken plasma protease inhibitor concentrations to assess adherence to antiretroviral therapy in HIV-1-infected children.

Clinical and virologic response to combination treatment with indinavir, zidovudine, and lamivudine in children with human immunodeficiency virus-1 infection: a multicenter study in the Netherlands. On behalf of the Dutch Study Group for Children with HIV-1 infections.

Objective: To evaluate the clinical, immunologic, and virologic response to indinavir, zidovudine, and lamivudine in children with human immunodeficiency virus-1 (HIV-1) infection.

Study Design: Twenty-eight HIV-1-infected children (3 months to 16 years of age) with or without prior treatment with reverse-transcriptase inhibitors and a HIV-1 RNA >5000 copies/mL and/or a CD4 cell count less than the lower limit of the age-specific reference value were treated with indinavir, zidovudine, and lamivudine. Pharmacokinetics of indinavir were determined in each child.