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). 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.

Central nervous system complications and management in sickle cell disease

“…42 In a French cohort, 2.8% of children with SCD (7 of 251) had either an intracranial hemorrhage or an unruptured aneurysm. 43,44 Risk factors for cerebral hemorrhage are poorly defined, but include hypertension associated with the use of corticosteroids 40 or phenylephrine, 45 recent transfusion, 40 splenic sequestration, 46 and hyperviscosity associated with higher hemoglobin levels. 16 Cerebral hemorrhage in adults is commonly related to aneurysm formation (see supplemental Figure 1C-D).…”

Cyclophosphamide improves engraftment in patients with SCD and severe organ damage who undergo haploidentical PBSCT

“…Furthermore, the transplant approach in children should be sufficiently safe to justify providing transplants for a patient population in which 99% are expected to reach 18 years of age. 59,60 Such an approach can serve to maximize the chance that a successful transplant will truly improve not only the quality but also the quantity of life.…”

Sickle Cell Disease

“…1,9 In high-income countries, this increase largely reflects gains in life expectancy among affected persons as a result of interventions such as newborn screening, penicillin prophylaxis, primary stroke prevention, and hydroxyurea treatment (Table 1). 14 Life expectancy has improved significantly in high-income countries over the past 40 years, with childhood mortality now close to that in the general population 15 and an observed median survival of more than 60 years. 16 Despite these remarkable achievements, life expectancy for patients with sickle cell disease is reduced by about 30 years, even with the best medical care, and the quality of life is often poor.…”