Anna Vanderfaeillie

Survival among children and adults with sickle cell disease in Belgium: Benefit from hydroxyurea treatment.

To evaluate the survival of patients with sickle cell disease (SCD) recorded in the Belgian SCD Registry and to assess the impact of disease‐modifying treatments (DMT).

An Estimation of the Incidence and Demographic Picture of the Major Hemoglobinopathies in Belgium (From a Confidential Inquiry)

An estimation of the incidence and demographic picture of the major hemoglobinopathies in Belgium has been approached through a confidential inquiry sent to 228 pediatric and adult hematological departments. Forty-two percent of responses showed that 417 patients are known in Belgium: 83% with sickle cell disease, 13% with β-thalassemia (β-thal) major, 2% with β-thal intermedia, and 1% with Hb H disease. Twenty-five percent of the sickle cell disease patients and 54% of those suffering from a β-thal major were older than 20 years. Three hospitals ensure the follow-up of 70% of the patients and are situated in Brussels, Belgium; a follow-up of less than 20 patients was reported at 21 centers. These results confirm that sickle cell disease is the major hemoglobinopathy in Belgium; it concerns mostly pediatricians but adult hematologists are also confronted with these pathologies. Therefore, it is necessary to implement integrated programs of prevention and treatment.

Effect of N-acetylcysteine on pain in daily life in patients with sickle cell disease: a randomised clinical trial

N.J.S. collected and analysed the data and wrote the manuscript; H.K. designed the study and treated patients; E.J., J.E.C, D.A.T, M.E.R., N.D., Y.A., M.K., P.K., W.G.W. and C.D.D. treated patients; C.B., D.M. and M.A.R. collected and analysed the data; F.R. designed the study, collected and analysed the data, treated patients and wrote the manuscript. All authors approved the final version of the manuscript.

Neonatal screening improves sickle cell disease clinical outcome in Belgium.

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 sickle cell disease patients were analyzed for a total of 1116 and 958 patient-years of follow-up, respectively. Both groups were compared with propensity score analysis, with patients matched on three covariates (gender, genotype, and central Africa origin). Bonferroni correction was applied for all comparisons. Results Kaplan–Meier estimates of survival without bacteremia were significantly higher in the screened group than the unscreened group (94.47%; [95% CI, 88.64–97.36%] versus 83.78% [95% CI, 72.27–90.42%]), p = 0.032. Non-significant differences between both groups were reported for survival without acute chest syndrome, acute anemia, cerebral complication, severe infection, and vaso-occlusive crisis. Significantly lower hospitalization rate and days per 100 patient-years were observed in the screened compared with the unscreened group (0.27 vs. 0.63 and 1.25 vs. 2.82, p = 0.0006 and <0.0001). Conclusion These data confirm the benefit of a neonatal screening programme in reducing bacteremia and hospitalization.