Improvement of medical care in a cohort of newborns with sickle‐cell disease in North Paris: impact of national guidelines. (7th April 2016)

• Record Type: Journal Article
• Authors: Couque, Nathalie; Girard, Delphine; Ducrocq, Rolande; Boizeau, Priscilla; Haouari, Zinedine; Missud, Florence; Holvoet, Laurent; Ithier, Ghislaine; Belloy, Marie; Odièvre, Marie‐Héléne; 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
• Is Part Of: British journal of haematology. Volume 173:Number 6(2016)
• Page Start: 927
• Page End: 937
• Publication Date: 2016-04-07
• Subjects: hydroxycarbamide -- mortality -- newborn screening -- sickle cell disease -- transfusion; Hematology -- Periodicals; Blood -- Diseases -- Periodicals; 616.15
• DOI: 10.1111/bjh.14015
• Languages: English
• ISSNs: 0007-1048

Abstract

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).