%0 Journal Article
%T Improvement of medical care in a cohort of newborns with sickle-cell disease in North Paris: impact of national guidelines.
%A Couque N
%A Girard D
%A Ducrocq R
%A Boizeau P
%A Haouari Z
%A Missud F
%A Holvoet L
%A Ithier G
%A Belloy M
%A Odièvre MH
%A Benemou M
%A Benhaim P
%A Retali B
%A Bensaid P
%A Monier B
%A Brousse V
%A Amira R
%A Orzechowski C
%A Lesprit E
%A Mangyanda L
%A Garrec N
%A Elion J
%A Alberti C
%A Baruchel A
%A Benkerrou M
%J Br J Haematol
%V 173
%N 6
%D 06 2016
%M 27062606
%F 8.615
%R 10.1111/bjh.14015
%X We conducted a retrospective study on newborns with sickle-cell disease (SCD), born 1995-2009, followed in a multicentre hospital-based network. We assessed patient outcomes, medical care and compliance with the national guidelines published in December 2005. Data from 1033 patients (742 SS/Sβ°-thalassaemia) with 6776 patient-years of follow-up were analysed (mean age 7·1 ± 3·9 years). SCD-related deaths (n = 13) occurred only in SS-genotype patients at a median age of 23·1 months, mainly due to acute anaemia (n = 5, including 2 acute splenic sequestrations) and infection (n = 3). Treatment non-compliance was associated with a 10-fold higher risk of SCD-related death (P = 0·01). Therapeutic intensification was provided for all stroke patients (n = 12), almost all patients with abnormal transcranial Doppler (TCD) (n = 76) or with >1 acute chest syndrome/lifetime (n = 64) and/or ≥3 severe vaso-occlusive crises/year (n = 100). Only 2/3 of patients with baseline haemoglobin <70 g/l received intensification, mainly for other severity criteria. Overall, hydroxycarbamide was under-prescribed, given to 2/3 of severe vaso-occlusive patients and 1/3 of severely anaemic patients. Nevertheless, introduction of the on-line guidelines was concomitant with an improvement in medical care in the 2006-2009 cohort with a trend towards increased survival at 5 years, from 98·3% to 99·2%, significantly increased TCD coverage (P = 0·004) and earlier initiation of intensification of therapy (P ≤ 0·01).