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Improvement of medical care in a cohort of newborns with sickle‐cell disease in North Paris: impact of national guidelines
Author(s) -
Couque Nathalie,
Girard Delphine,
Ducrocq Rolande,
Boizeau Priscilla,
Haouari Zinedine,
Missud Florence,
Holvoet Laurent,
Ithier Ghislaine,
Belloy Marie,
Odièvre MarieHéléne,
Benemou Michel,
Benhaim Patricia,
Retali Brigitte,
Bensaid Philippe,
Monier Brigitte,
Brousse Valentine,
Amira Roger,
Orzechowski Christine,
Lesprit Emmanuelle,
Mangyanda Laurent,
Garrec Nathalie,
Elion Jacques,
Alberti Corinne,
Baruchel André,
Benkerrou Malika
Publication year - 2016
Publication title -
british journal of haematology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.907
H-Index - 186
eISSN - 1365-2141
pISSN - 0007-1048
DOI - 10.1111/bjh.14015
Subject(s) - medicine , cohort , disease , pediatrics , cohort study , intensive care medicine , family medicine
Summary 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).