Survival among children and adults with sickle cell disease in Belgium: Benefit from hydroxyurea treatment.
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.
['Lê|Phu Quoc|PQ|', 'Gulbis|Béatrice|B|', 'Dedeken|Laurence|L|', 'Dupont|Sophie|S|', 'Vanderfaeillie|Anna|A|', 'Heijmans|Catherine|C|', 'Huybrechts|Sophie|S|', 'Devalck|Christine|C|', 'Efira|André|A|', 'Dresse|Marie-Françoise|MF|', 'Rozen|Laurence|L|', 'Benghiat|Fleur Samantha|FS|', 'Ferster|Alina|A|']
D000293:Adolescent D000328:Adult D000367:Age Factors D064591:Allografts D000755:Anemia, Sickle Cell D000986:Antisickling Agents D001530:Belgium 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 D007223:Infant D007231:Infant, Newborn D008297:Male D012189:Retrospective Studies D015996:Survival Rate
survival sickle cell disease hydroxyurea mortality