Clinical outcomes in children with sickle cell disease living in England: a neonatal cohort in East London.
OBJECTIVE
We investigated outcomes in a UK neonatal cohort as a benchmark for care of children with sickle cell disease (SCD).


METHODS
Two-hundred and fifty-two children (180 with hemoglobin [Hb] SS, 64 with HbSC, and 8 with HbS/beta thalassemia), identified during 1983-2005 by universal birth screening in East London, were followed in a hospital and community-based program which included penicillin V prophylaxis from 3 months of age, 23-valent pneumococcal polysaccharide vaccine from 1993, conjugate pneumococcal vaccine from 2002 and transcranial Doppler screening from 1991.


RESULTS
At the end of 2005, there were 2158 patient years of observation. The median age of the patients was 7.8 (interquartile range 3.3-13.0) years, and 2.8% of those enrolled had been lost to follow-up. The estimated survival of children with HbSS at 16 years was 99.0% (95% confidence interval, CI, 93.2 to 99.9%) and pneumococcal sepsis rate was 0.3 (95% CI 0.1-0.8) episodes per 100 patient-years. The risk of overt stroke was 4.3% (95%CI 1.5 to 11.4%) and could be further reduced by transcranial Doppler screening from infancy and transfusing all children with high-risk scans. No deaths, strokes or episodes of pneumococcal sepsis were observed in children with HbSC or HbS/beta thalassemia. The mortality rates from HbSS were significantly lower than those in other reported cohorts.


CONCLUSIONS
Mortality in childhood SCD can virtually be eliminated in a well-resourced health service setting linking community-based care with a specialized, hospital-based center. SCD continues to cause substantial morbidity from acute complications and chronic organ damage. We recommend setting up of clinical networks to optimize the management of SCD.
Telfer
Paul
P
Department of Paediatric Haematology and Oncology, Royal London Hospital, Whitechapel Road, London E1 1BB. paul.telfer@bartsandthelondon.nhs.uk
Coen
Pietro
P
Chakravorty
Subarna
S
Wilkey
Olu
O
Evans
Jane
J
Newell
Heather
H
Smalling
Beverley
B
Amos
Roger
R
Stephens
Adrian
A
Rogers
David
D
Kirkham
Fenella
F
D000755:Anemia, Sickle Cell / Q000150:complications / Q000453:epidemiology* / Q000401:mortality / Q000628:therapy; D015331:Cohort Studies; D019058:Community Networks / Q000592:standards*; D004739:England; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D008131:London; D011787:Quality of Health Care / Q000706:statistics & numerical data*; D015996:Survival Rate; D016896:Treatment Outcome