To evaluate the survival of patients with sickle cell disease (SCD) recorded in Belgian SCD Registry and to assess impact disease-modifying treatments (DMT).The created 2008 included eight centers. All available data were retrospectively encoded database. After until 2012, all prospectively for already registered as well newly diagnosed subjects. Data from neonatal screening or diagnosis (first contact) last follow-up death. diagnosis, demography, outcome data.We collected 469 over a 5,110...

Despite improvements in medical management, sickle cell disease (SCD) remains associated with severe morbidity and decreased survival. Allogeneic haematopoietic stem transplantation (HSCT) the only curative approach. We report outcome of 50 consecutive children SCD that received HSCT our unit between November 1988 April 2013. The source was bone marrow (n = 39), cord blood 3), 7) peripheral cells 1). All patients had ≥1 manifestation: 37 presented recurrent vaso-occlusive crises/acute chest...

For better characterizing the effect of anti-CD20 therapy, we analysed use rituximab in Belgian patients experiencing auto-immune haemolytic anaemia (AIHA) and immune thrombocytopenic purpura (ITP).We performed a retrospective multicentric analysis with AIHA ITP treated Belgium.Haematological departments were invited to fill questionnaire about patient disease characteristics.All ITP, both primary secondary other diseases, who received one or more courses during their course included....

Isolated hypoparathyroidism is an uncommon metabolic disorder characterized by hypocalcemia and hyperphosphatemia, with absent or low levels of PTH. It may present as apparently sporadic be transmitted in families a genetic trait. Mutations the calcium-sensing receptor gene preproPTH have been reported occasional cases, mutation parathyroid-specific transcription factor GCMB has one familial case. We report second family isolated mutation. The patients were two siblings from asymptomatic,...

Our objective is to provide the clinical characteristics, uropathogen frequencies, and antimicrobial resistance rates of first urinary tract infection (UTI) diagnosed in febrile Belgian children. The ability noninvasive ultrasound detect renal abnormalities vesicoureteral reflux (VUR) these patients was also assessed.We prospectively followed (median, 20 months) 209 children treated for UTI. Renal (US) voiding cystourethrography examinations were performed all patients.Among children, 63%...

Objectives. Chronic inflammation in juvenile idiopathic arthritis interferes with linear growth and bone mass acquisition. We prospectively evaluated compared evolution of acquisition body composition MTX-resistant polyarticular-course JIA (polyJIA) patients started on etanercept recently diagnosed polyJIA MTX monotherapy. Methods. Sixteen were given add-on etanercept, eight MTX. Patients at baseline 1, 6, 12 18 months respect to disease activity, growth, BMD composition. Results. Baseline...

A neonatal haemoglobinopathy screening programme was implemented in Brussels more than a decade ago and Liège 5 years ago; the adapted to local situation.Neonatal for haemoglobinopathies universal, performed using liquid cord blood an isoelectric focusing technique. All samples with abnormalities underwent confirmatory testing. Major minor were reported. Affected children referred specialist centre. central database which all results stored available accessible care workers. clinical monitor...

Changes in several components of the clotting system are well documented sickle cell disease (SCD) patients. However, whether global hemostatic potential these patients is altered still unclear. Calibrated automated thrombogram(®) method thrombin generation (TG) was used to characterize 83 SCD children (75 SS, 6 SC, and 2 Sβ (thal)) at steady-state as compared with 50 controls same range age. TG triggered using 1 pM tissue factor 4 μM phospholipids without thrombomodulin. Thirteen were also...

Introduction: Autoimmune pancreatitis (AIP) is a rare and distinct condition in children compared to adults. Despite being based on adult diagnostic criteria, several differences have been reported, making pediatric AIP challenging diagnosis. Case Presentation: A 14-year-old adolescent presented the emergency department with abdominal pain, vomiting, jaundice. combination of biological radiological assessments led diagnosis AIP. The patient experienced swift complete recovery without...

Immunoglobulin superfamily (IgSF) domains are conserved structures present in many proteins eukaryotes and prokaryotes. These well-capable of facilitating sequence variation, which is most clearly illustrated by the variable regions immunoglobulins (Igs) T cell receptors (TRs). We studied an antibody-deficient patient suffering from recurrent respiratory infections with impaired antibody responses to vaccinations. Patient's B cells showed Ca2+ influx upon stimulation anti-IgM lacked...

Objectives To compare the outcomes of sickle cell disease patients diagnosed through neonatal screening with those who were not. Methods In an observational multicenter study in Belgium, 167 screened and 93 unscreened analyzed for a total 1116 958 patient-years follow-up, respectively. Both groups compared propensity score analysis, matched on three covariates (gender, genotype, central Africa origin). Bonferroni correction was applied all comparisons. Results Kaplan–Meier estimates survival...

The aim of the Patient preferences to Assess Value IN Gene therapies (PAVING) study was investigate trade-offs that adult Belgian people with haemophilia (PWH) A and B are willing make when choosing between prophylactic factor replacement therapy (PFRT) gene therapy.

ABSTRACT This study assessed the efficacy of oral care education among nursing home staff members to improve health residents. Nursing support (NHSSMs) in group received at baseline between a pretest and posttest. NHSSMs’ knowledge was measured using 20‐item test baseline, posteducation, 6‐month follow‐up. Residents’ again follow‐up Modified Plaque Index (PI) Gingival (GI). Among who (n = 32), posttest statistically significantly increased from level ( p < .05). Thirty‐nine control...

BACKGROUND Chronic transfusion in sickle cell disease (SCD) remains the gold standard therapy for stroke prevention and patients with severe despite adequate hydroxyurea treatment. The aim of our study was to assess safety efficacy automated red blood exchange (aRBX) SCD previously treated manual (MET). Costs related chelation overtime were evaluated. STUDY DESIGN AND METHODS Beginning January 2012, children who weighed 30 kg or more on MET could switch aRBX. Clinical, biological,...

Background Asparaginase (Asp) and corticosteroid (CS) treatment in patients with acute lymphoblastic leukaemia (ALL) is associated an increased risk of thrombotic events. Objective Characterization global haemostatic phenotypes ALL during Asp therapy. Procedure Thrombin generation (TG) was monitored platelet-poor plasma 56 children treated for a B lineage (36 native, 20 PEG Asp) using 1 pM tissue factor 4 μM phospholipids, without thrombomodulin. Protein C activity (PC), free protein S (PS),...

Introduction In the management of juvenile idiopathic arthritis (JIA), there is a lack diagnostic and prognostic biomarkers. This study assesses use serum calprotectin (sCal) as marker to monitor disease activity, classification prognosis tool response treatment or risk flares in patients with JIA. Methods Eighty-one JIA from CAP48 multicentric cohort were included this study, well 11 non-paediatric healthy controls. An ELISA method was used quantify sCal commercial kit. Results Patients an...

Abstract Introduction Increased thrombin generation ( TG ) was described in sickle cell disease SCD children. The aim of this study to characterize at the individual level and assess its relationship with age, hemolysis, transcranial Doppler velocity TCD ), hydroxyurea treatment. Patients methods triggered platelet‐poor plasma using tissue factor phospholipids addition thrombomodulin 97 steady state 80 control controls were aged from 2 20 years, they distributed four categories age: [2–5],...

For centuries, writers have recorded their observations on pica. Nevertheless the association of pica with sickle cell disease (SCD) was poorly documented.Cross-sectional evaluation performed SCD children and caregivers attending outpatient clinic who were invited to complete questionnaires assessing behavior pica.Out 55 children, 31(56.4%) reported practicing regularly. Substances ingested by patients covered a broad spectrum. Compared non-pica group, subjects younger had lower hemoglobin...