1. Improvement of medical care in a cohort of newborns with sickle-cell disease in North Paris: impact of national guidelines.
- Author
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Couque N, Girard D, Ducrocq R, Boizeau P, Haouari Z, Missud F, Holvoet L, Ithier G, Belloy M, Odièvre MH, Benemou M, Benhaim P, Retali B, Bensaid P, Monier B, Brousse V, Amira R, Orzechowski C, Lesprit E, Mangyanda L, Garrec N, Elion J, Alberti C, Baruchel A, and Benkerrou M
- Subjects
- Acute Chest Syndrome etiology, Child, Child, Preschool, Cohort Studies, Female, Follow-Up Studies, Humans, Hydroxyurea therapeutic use, Infant, Newborn, Male, Paris, Retrospective Studies, Stroke etiology, Thalassemia, Anemia, Sickle Cell complications, Anemia, Sickle Cell drug therapy, Anemia, Sickle Cell mortality, Guideline Adherence, Quality Improvement standards
- 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.)
- Published
- 2016
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