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

Gulbis, Béatrice; Ferster, Alice; Vermylen, Christiane; Dresse, Marie-Françoise; Vanderfaeillie, Anna; Delannoy, André; Labarque, Veerle; Philippet, Pierre; Kentos, Alain; Sztern, B; B, Deprijck; Vertongen, Françoise

doi:10.1080/03630260802004400

Cited by 7 publications

(6 citation statements)

References 8 publications

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“…In the absence of a national‐based neonatal screening program and/or official registration based on diagnosis, the total number of SCD living in Belgium is unknown. A national inquiry performed in 2008 estimated the total number of patient with severe hemoglobinopathy to be 417 from whom 83% were sickle cell patients . Our data concern thus a large majority of regularly monitored patients living in Belgium.…”

Section: Discussionmentioning

confidence: 90%

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

Lê

Gulbis

Dedeken

et al. 2015

Pediatric Blood & Cancer

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145

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Objective 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). Method The Registry created in 2008 included patients of eight centers. All available data in 2008 were retrospectively encoded in the database. After 2008 and until 2012, all data were recorded prospectively for already registered patients as well as newly diagnosed subjects. Data were registered from neonatal screening or from diagnosis (first contact) until last follow‐up or death. Data included diagnosis, demography, and outcome data. Results We collected data from 469 patients over a 5,110 patient years (PY) follow‐up period. The global mortality rate was low (0.25/100 PY), although 13 patients died (2.8%) and was similar between children, adolescents (10–18 years), and young adults (P = 0.76). Out of the cohort, 185 patients received hydroxyurea at last follow‐up (median duration of treatment: 10.3 years), 90 underwent hematopoietic stem cell transplantation (HSCT), 24 were chronically transfused, and 170 had never had any DMT. Hydroxyurea showed significant benefit on patients outcome as reflected by a lower mortality rate compared to transplanted individuals or people without DMT (0.14, 0.36, and 0.38 per 100 PY, respectively) and by higher Kaplan–Meier estimates of 15 year survival (99.4%) compared to HSCT (93.8%; P = 0.01) or no DMT groups (95.4%; P = 0.04). Conclusion SCD mortality in Belgium is low with no increase observed in young adults. Patients treated with hydroxyurea demonstrate a significant benefit in survival when compared to those without DMT or transplanted. Pediatr Blood Cancer © 2015 Wiley Periodicals, Inc.

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Section: Discussionmentioning

confidence: 90%

“…A national inquiry performed in 2008 estimated the total number of patient with severe hemoglobinopathy to be 417 from whom 83% were sickle cell patients. [42] Our data concern thus a large majority of regularly monitored patients living in Belgium.…”

Section: Discussionmentioning

confidence: 98%

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

Lê

Gulbis

Dedeken

et al. 2015

Pediatric Blood & Cancer

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145

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“…15 In some European countries, the prevalence of β-thalassemia and other major hemoglobinopathies is increasing due to migration, and major hemoglobinopathies are now the most common genetic rare disease in Europe (Table 3). 7,13,15,16,[32][33][34][35][36][37][38][39][40][41][42] As of 2007, new cases of hemoglobinopathies occurred at a comparable frequency throughout Northern, Western, and Southern Europe. 33 Although birth rates are decreasing in the general population, affected birth rates remain relatively high among some at-risk migrant groups.…”

Section: Pre Valen Ce Of β -Thal a Ss Emiamentioning

confidence: 99%

Changing patterns in the epidemiology of β‐thalassemia

Kattamis

Forni

Aydınok

et al. 2020

European J of Haematology

195

114

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β-thalassemias are a heterogeneous group of hereditary hemoglobinopathies characterized by defects in the β-globin chain of hemoglobin and autosomal recessive inheritance. Homozygous or compound heterozygous forms have an imbalance in the production of α-and non-α-globin chains, resulting in ineffective erythropoiesis and decreased production of normal hemoglobin A. 1 Patients with β-thalassemia major have severe chronic hemolytic anemia and require regular blood transfusions from early childhood. 1-3 Chronic blood transfusion therapy is typically combined with iron chelation therapy (ICT) to prevent complications due to iron overload, such as cardiac morbidity, liver disease, and endocrine dysfunction. 1-3 Patients with β-thalassemia intermedia have symptoms in between carriers and those with β-thalassemia major: Anemia is often moderate, but patients may still have morbidity due to ineffective erythropoiesis and hemolysis, including ulcers, pulmonary hypertension, and pain. Some patients require occasional blood transfusions, although less frequently than patients with β-thalassemia major. 1,2 Historically, the prevalence of β-thalassemia has been highest in the Mediterranean region, the Middle East, and Southeast Asia and lowest in Northern Europe and North America. 4 Due to migration patterns, β-thalassemia is increasingly more common in non-endemic regions,

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“…The registry currently includes patients from six adult and two pediatric centers, of which two are secondary and six are tertiary university care centers. Results published in 2008 8 estimated that about 75–80% of SCD patients living in Belgium were enrolled. Patient data before 2008 were retrieved from medical charts and retrospectively encoded in the database.…”

Section: Methodsmentioning

confidence: 99%

Neonatal screening improves sickle cell disease clinical outcome in Belgium

et al. 2017

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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.

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

Abstract: Ⅺ 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

Cited by 7 publications

References 8 publications

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

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

Changing patterns in the epidemiology of β‐thalassemia

Neonatal screening improves sickle cell disease clinical outcome in Belgium

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