Phu Quoc Lê1, Béatrice Gulbis2, Laurence Dedeken1, Sophie Dupont3, Anna Vanderfaeillie4, Catherine Heijmans1,5, Sophie Huybrechts1, Christine Devalck1, André Efira6, Marie-Françoise Dresse7, Laurence Rozen8, Fleur Samantha Benghiat9, Alina Ferster1. 1. Hemato-Oncology Unit, Hôpital Universitaire des Enfants Reine Fabiola, U.L.B., Brussels, Belgium. 2. Department of Clinical Chemistry, Hôpital Erasme, U.L.B., Brussels, Belgium. 3. Hemato-Oncology Unit, Cliniques Universitaires Saint Luc, Brussels, Belgium. 4. Department of Pediatrics, Hôpital Saint-Pierre, U.L.B., Brussels, Belgium. 5. Department of Pediatrics, Hôpital de Jolimont, La Louvière, Belgium. 6. Hemato-Oncology Unit, Hôpital Universitaire Brugmann, U.L.B., Brussels, Belgium. 7. Hemato-Oncology Unit, Hôpital Régional de la Citadelle, ULg, Liège, Belgium. 8. Laboratory of Hematology, CHU-Brugmann, Hôpital Universitaire des Enfants Reine Fabiola, U.L.B., Brussels, Belgium. 9. Hemato-Oncology Unit, Hôpital Erasme, U.L.B., Brussels, Belgium.
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). 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.
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.
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