[Cystic fibrosis - A look at Belgium in 2022]. / Mucoviscidose : un regard sur la situation en Belgique en 2022.

Cystic fibrosis care is expensive. In Belgium, its financial support is not provided by powerful charities but by the national health system, which also sponsors the Belgian Cystic Fibrosis Registry. Recent data allow to better evaluate the quality of care for patients with cystic fibrosis in our country. Overall, it is high but varies from one centre to another. Similarly, use of the main symptomatic treatments is heterogeneous. Access to lung transplantation is one of the fluidest in the world. However, Belgium was one of the last medicalised countries to implement a neonatal screening programme for cystic fibrosis. It also lags behind in regard of the reimbursement of modulators of the CTFR gene function. This is especially detrimental for the lack of reimbursement of a recent highly effective combination of three modulators. The cost of this triple therapy is opaque and far too high. However, its effectiveness is impressive and, in the long term, around 90 % of Belgian patients with cystic fibrosis are expected to greatly benefit from it.

La prise en charge de la mucoviscidose est coûteuse. En Belgique, elle n'est pas financée par de puissantes organisations caritatives, mais par le système de santé national, qui parraine également le Registre Belge de la Mucoviscidose. Des données récentes permettent de mieux situer la qualité des soins prodigués aux patients atteints de mucoviscidose dans notre pays. Globalement, elle est élevée, mais varie d'un centre à l'autre. De même, l'utilisation des principaux traitements symptomatiques est hétérogène. L'accès à la transplantation pulmonaire est l'un des plus fluides au monde. La Belgique a été l'un des derniers pays médicalisés à mettre en place un programme de dépistage néonatal de la mucoviscidose. Elle est également à la traîne en ce qui concerne le remboursement des modulateurs de la fonction du gène CTFR. Ceci est particulièrement préjudiciable pour le remboursement d'une récente combinaison de trois d'entre eux, hautement efficace. Le coût de cette trithérapie est opaque et beaucoup trop élevé, mais l'impact médical de ce traitement est impressionnant et, à terme, 90 % des patients belges devraient en bénéficier grandement.

Profile of pediatric Crohn's disease in Belgium.

AIM: A Belgian registry for pediatric Crohn's disease, BELCRO, was created. This first report aims at describing disease presentation and phenotype and determining associations between variables at diagnosis and registration in the database. METHODS: Through a collaborative network, children with previously established Crohn's disease and newly diagnosed children and adolescents (under 18 y of age) were recruited over a 2 year period. Data were collected by 23 centers and entered in a database. Statistical association tests analyzed relationships between variables of interest at diagnosis. RESULTS: Two hundred fifty-five patients were included. Median age at diagnosis was 12.5 y (range: 1.6-18 y); median duration of symptoms prior to diagnosis was 3 m (range: 1-12 m). Neonatal history and previous medical history did not influence disease onset nor disease behavior. Fifty three % of these patients presented with a BMI z-score < -1. CRP was an independent predictor of disease severity. Steroids were widely used as initial treatment in moderate to severe and extensive disease. Over time, immunomodulators and biological were prescribed more frequently, reflecting a lower prescription rate for steroids and 5-ASA. A positive family history was the sole significant determinant for earlier use of immunosuppression. CONCLUSION: In Belgium, the median age of children presenting with Crohn's disease is 12.5 y. Faltering growth, extensive disease and upper GI involvement are frequent. CRP is an independent predictive factor of disease activity. A positive family history appears to be the main determinant for initial treatment choice.

An unusual case of anti-Borrelia burgdorferi immunoglobulin G seroconversion caused by administration of intravenous gammaglobulins.

Administration of gammaglobulins to individuals without specific anti-Borrelia burgdorferi antibodies may lead to immunoglobulin G (IgG) conversion as detected by enzyme-linked immunosorbent assay (ELISA). In some cases however, complementary techniques such as Western blot or avidity will be of prime importance in distinguishing the start of an infection from the passive immunization induced by the gammaglobulins. In all cases, the key element before reaching conclusions in relation to any of these investigations remains the confrontation between the clinical context and the biological findings. This is the scenario that has been followed in our observation.

[Congenital toxoplasmosis and seroconversion at the end of pregnancy: clinical observations]. / Toxoplasmoses congénitales et séroconversions de fin de grossesse: observations cliniques.

We report seven cases of subclinical congenital toxoplasmosis secondary to maternal primary infections. Mothers were infected between two and four weeks prior to delivery. The diagnostic criteria of congenital infections included: IgM antibody (Ab) (1 case); IgM and IgA Ab (1 case); a real IgG seroconversion in the neonatal and postnatal samples (3 cases); persistence of IgG Ab beyond 6 months post-delivery (2 cases). A treatment was initiated, including a combination of pyrimethamine + sulfadiazine (6 cases); trimethoprim + sulfamethoxazole (1 case). This retrospective study suggests that it is important to screen the non-immune pregnant women until delivery. We confirmed the usefulness of a combination of isotypes of antibodies for the accurate assessment of congenital infection. Finally, infected infants have to be treated and monitored clinically and immunologically during the first year of life.

Thrombosis and embolism in long-term central venous access for parenteral nutrition.

Although the use of silicone catheters for long-term central venous access is widespread, little is known about the incidence of pulmonary thromboembolic complications. We studied clinical events, lung perfusion scans, and echocardiographic screens in 34 children and adolescents with gut failure who had received cyclical parenteral nutrition for 2 months to 9 years. Major thrombosis and/or embolism was identified in 12 patients and 4 died as a consequence. Actuarial survival free from thrombosis was 53% at 5 years (95% Cl, 30-77%). Survival free from fatal pulmonary thromboembolic events was 74% at 5 years (48-99%). 3 patients required surgery to remove right atrial thrombus or pulmonary emboli. Major right atrial thrombosis and pulmonary embolism are common and potentially fatal complications of parenteral nutrition by long-term venous access in childhood. Anticoagulation is recommended.