Survival among children and adults with sickle cell disease in Belgium: Benefit from hydroxyurea treatment
Corresponding Author
Phu Quoc Lê MD
Hemato-Oncology Unit, Hôpital Universitaire des Enfants Reine Fabiola, U.L.B., Brussels, Belgium
Correspondence to: Phu Quoc Lê, Department of Hemato-oncology, Hôpital Universitaire des Enfants Reine Fabiola, 15 Avenue Jean Joseph Crocq, 1020 Brussels, Belgium.
E-mail: [email protected]
Search for more papers by this authorBéatrice Gulbis MD, PhD
Department of Clinical Chemistry, Hôpital Erasme, U.L.B., Brussels, Belgium
Search for more papers by this authorLaurence Dedeken MD
Hemato-Oncology Unit, Hôpital Universitaire des Enfants Reine Fabiola, U.L.B., Brussels, Belgium
Search for more papers by this authorSophie Dupont MD
Hemato-Oncology Unit, Cliniques Universitaires Saint Luc, Brussels, Belgium
Search for more papers by this authorAnna Vanderfaeillie MD
Department of Pediatrics, Hôpital Saint-Pierre, U.L.B., Brussels, Belgium
Search for more papers by this authorCatherine Heijmans MD
Hemato-Oncology Unit, Hôpital Universitaire des Enfants Reine Fabiola, U.L.B., Brussels, Belgium
Department of Pediatrics, Hôpital de Jolimont, La Louvière, Belgium
Search for more papers by this authorSophie Huybrechts MD
Hemato-Oncology Unit, Hôpital Universitaire des Enfants Reine Fabiola, U.L.B., Brussels, Belgium
Search for more papers by this authorChristine Devalck MD
Hemato-Oncology Unit, Hôpital Universitaire des Enfants Reine Fabiola, U.L.B., Brussels, Belgium
Search for more papers by this authorAndré Efira MD
Hemato-Oncology Unit, Hôpital Universitaire Brugmann, U.L.B., Brussels, Belgium
Search for more papers by this authorMarie-Françoise Dresse MD, PhD
Hemato-Oncology Unit, Hôpital Régional de la Citadelle, ULg, Liège, Belgium
Search for more papers by this authorLaurence Rozen
Laboratory of Hematology, CHU-Brugmann, Hôpital Universitaire des Enfants Reine Fabiola, U.L.B., Brussels, Belgium
Search for more papers by this authorFleur Samantha Benghiat MD, PhD
Hemato-Oncology Unit, Hôpital Erasme, U.L.B., Brussels, Belgium
Search for more papers by this authorAlina Ferster MD
Hemato-Oncology Unit, Hôpital Universitaire des Enfants Reine Fabiola, U.L.B., Brussels, Belgium
Search for more papers by this authorCorresponding Author
Phu Quoc Lê MD
Hemato-Oncology Unit, Hôpital Universitaire des Enfants Reine Fabiola, U.L.B., Brussels, Belgium
Correspondence to: Phu Quoc Lê, Department of Hemato-oncology, Hôpital Universitaire des Enfants Reine Fabiola, 15 Avenue Jean Joseph Crocq, 1020 Brussels, Belgium.
E-mail: [email protected]
Search for more papers by this authorBéatrice Gulbis MD, PhD
Department of Clinical Chemistry, Hôpital Erasme, U.L.B., Brussels, Belgium
Search for more papers by this authorLaurence Dedeken MD
Hemato-Oncology Unit, Hôpital Universitaire des Enfants Reine Fabiola, U.L.B., Brussels, Belgium
Search for more papers by this authorSophie Dupont MD
Hemato-Oncology Unit, Cliniques Universitaires Saint Luc, Brussels, Belgium
Search for more papers by this authorAnna Vanderfaeillie MD
Department of Pediatrics, Hôpital Saint-Pierre, U.L.B., Brussels, Belgium
Search for more papers by this authorCatherine Heijmans MD
Hemato-Oncology Unit, Hôpital Universitaire des Enfants Reine Fabiola, U.L.B., Brussels, Belgium
Department of Pediatrics, Hôpital de Jolimont, La Louvière, Belgium
Search for more papers by this authorSophie Huybrechts MD
Hemato-Oncology Unit, Hôpital Universitaire des Enfants Reine Fabiola, U.L.B., Brussels, Belgium
Search for more papers by this authorChristine Devalck MD
Hemato-Oncology Unit, Hôpital Universitaire des Enfants Reine Fabiola, U.L.B., Brussels, Belgium
Search for more papers by this authorAndré Efira MD
Hemato-Oncology Unit, Hôpital Universitaire Brugmann, U.L.B., Brussels, Belgium
Search for more papers by this authorMarie-Françoise Dresse MD, PhD
Hemato-Oncology Unit, Hôpital Régional de la Citadelle, ULg, Liège, Belgium
Search for more papers by this authorLaurence Rozen
Laboratory of Hematology, CHU-Brugmann, Hôpital Universitaire des Enfants Reine Fabiola, U.L.B., Brussels, Belgium
Search for more papers by this authorFleur Samantha Benghiat MD, PhD
Hemato-Oncology Unit, Hôpital Erasme, U.L.B., Brussels, Belgium
Search for more papers by this authorAlina Ferster MD
Hemato-Oncology Unit, Hôpital Universitaire des Enfants Reine Fabiola, U.L.B., Brussels, Belgium
Search for more papers by this authorAbstract
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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