Improvement of medical care in a cohort of newborns with sickle-cell disease in North Paris: impact of national guidelines.

Couque N1, Girard D2,3, Ducrocq R1, Boizeau P2, Haouari Z4,5, Missud F4,5, Holvoet L4,5, Ithier G4,5, Belloy M6, Odièvre MH7, Benemou M8, Benhaim P9,Retali B10, Bensaid P11, Monier B12, Brousse V13, Amira R14, Orzechowski C15, Lesprit E16, Mangyanda L17, Garrec N18, Elion J1,3,19, Alberti C2,3,20,Baruchel A3,4,21, Benkerrou M4,5,20.

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Abstract

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).