lunes, 18 de abril de 2016

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

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



 2016 Apr 7. doi: 10.1111/bjh.14015. [Epub ahead of print]

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

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).
© 2016 John Wiley & Sons Ltd.

KEYWORDS:

hydroxycarbamide; mortality; newborn screening; sickle cell disease; transfusion

PMID:
 
27062606
 
[PubMed - as supplied by publisher]

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