title: Survival among children and adults with sickle cell disease in Belgium: Benefit from hydroxyurea treatment.
abstract: 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).
  
  
  METHODS
  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).
  
  
  CONCLUSIONS
  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.
authors.affiliation: Hemato-Oncology Unit, Hôpital Universitaire des Enfants Reine Fabiola, U.L.B., Brussels, Belgium.
authors.forename: Phu Quoc
authors.initials: PQ
authors.lastname: Lê
authors.affiliation: Department of Clinical Chemistry, Hôpital Erasme, U.L.B., Brussels, Belgium.
authors.forename: Béatrice
authors.initials: B
authors.lastname: Gulbis
authors.affiliation: Hemato-Oncology Unit, Hôpital Universitaire des Enfants Reine Fabiola, U.L.B., Brussels, Belgium.
authors.forename: Laurence
authors.initials: L
authors.lastname: Dedeken
authors.affiliation: Hemato-Oncology Unit, Cliniques Universitaires Saint Luc, Brussels, Belgium.
authors.forename: Sophie
authors.initials: S
authors.lastname: Dupont
authors.affiliation: Department of Pediatrics, Hôpital Saint-Pierre, U.L.B., Brussels, Belgium.
authors.forename: Anna
authors.initials: A
authors.lastname: Vanderfaeillie
authors.affiliation: Hemato-Oncology Unit, Hôpital Universitaire des Enfants Reine Fabiola, U.L.B., Brussels, Belgium.|Department of Pediatrics, Hôpital de Jolimont, La Louvière, Belgium.
authors.forename: Catherine
authors.initials: C
authors.lastname: Heijmans
authors.affiliation: Hemato-Oncology Unit, Hôpital Universitaire des Enfants Reine Fabiola, U.L.B., Brussels, Belgium.
authors.forename: Sophie
authors.initials: S
authors.lastname: Huybrechts
authors.affiliation: Hemato-Oncology Unit, Hôpital Universitaire des Enfants Reine Fabiola, U.L.B., Brussels, Belgium.
authors.forename: Christine
authors.initials: C
authors.lastname: Devalck
authors.affiliation: Hemato-Oncology Unit, Hôpital Universitaire Brugmann, U.L.B., Brussels, Belgium.
authors.forename: André
authors.initials: A
authors.lastname: Efira
authors.affiliation: Hemato-Oncology Unit, Hôpital Régional de la Citadelle, ULg, Liège, Belgium.
authors.forename: Marie-Françoise
authors.initials: MF
authors.lastname: Dresse
authors.affiliation: Laboratory of Hematology, CHU-Brugmann, Hôpital Universitaire des Enfants Reine Fabiola, U.L.B., Brussels, Belgium.
authors.forename: Laurence
authors.initials: L
authors.lastname: Rozen
authors.affiliation: Hemato-Oncology Unit, Hôpital Erasme, U.L.B., Brussels, Belgium.
authors.forename: Fleur Samantha
authors.initials: FS
authors.lastname: Benghiat
authors.affiliation: Hemato-Oncology Unit, Hôpital Universitaire des Enfants Reine Fabiola, U.L.B., Brussels, Belgium.
authors.forename: Alina
authors.initials: A
authors.lastname: Ferster
mesh_terms: D000293:Adolescent; D000328:Adult; D000367:Age Factors; D064591:Allografts; D000755:Anemia, Sickle Cell / Q000401:mortality* / Q000628:therapy*; D000986:Antisickling Agents / Q000008:administration & dosage*; D001530:Belgium / Q000453:epidemiology; D001803:Blood Transfusion; D002648:Child; D002675:Child, Preschool; D016208:Databases, Factual; D005260:Female; D005500:Follow-Up Studies; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D006918:Hydroxyurea / Q000008:administration & dosage*; D007223:Infant; D007231:Infant, Newborn; D008297:Male; D012189:Retrospective Studies; D015996:Survival Rate
chemical_list: D000986:Antisickling Agents; D006918:Hydroxyurea
keywords: hydroxyurea; mortality; sickle cell disease; survival