Premium
Survival among children and adults with sickle cell disease in Belgium: Benefit from hydroxyurea treatment
Author(s) -
Lê Phu Quoc,
Gulbis Béatrice,
Dedeken Laurence,
Dupont Sophie,
Vanderfaeillie Anna,
Heijmans Catherine,
Huybrechts Sophie,
Devalck Christine,
Efira André,
Dresse MarieFrançoise,
Rozen Laurence,
Benghiat Fleur Samantha,
Ferster Alina
Publication year - 2015
Publication title -
pediatric blood and cancer
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.116
H-Index - 105
eISSN - 1545-5017
pISSN - 1545-5009
DOI - 10.1002/pbc.25608
Subject(s) - medicine , pediatrics , cohort , hematopoietic stem cell transplantation , disease , young adult , mortality rate , hematopoietic cell , cohort study , haematopoiesis , stem cell , biology , genetics
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.