title: 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).
authors.affiliation: Génétique Moléculaire et Biochimie, AP-HP, Hôpital Robert-Debré, Paris, France.
authors.forename: Nathalie
authors.initials: N
authors.lastname: Couque
authors.affiliation: Unité d'Epidémiologie Clinique, AP-HP, Hôpital Robert-Debré, Paris, France.|Univ Paris Diderot, Sorbonne Paris Cité, Paris, France.
authors.forename: Delphine
authors.initials: D
authors.lastname: Girard
authors.affiliation: Génétique Moléculaire et Biochimie, AP-HP, Hôpital Robert-Debré, Paris, France.
authors.forename: Rolande
authors.initials: R
authors.lastname: Ducrocq
authors.affiliation: Unité d'Epidémiologie Clinique, AP-HP, Hôpital Robert-Debré, Paris, France.
authors.forename: Priscilla
authors.identifier: 0000-0003-4573-4617
authors.initials: P
authors.lastname: Boizeau
authors.affiliation: Service d'Hématologie Pédiatrique, AP-HP, Hôpital Robert-Debré, Paris, France.|Centre de Référence de la Drépanocytose, Paris, France.
authors.forename: Zinedine
authors.initials: Z
authors.lastname: Haouari
authors.affiliation: Service d'Hématologie Pédiatrique, AP-HP, Hôpital Robert-Debré, Paris, France.|Centre de Référence de la Drépanocytose, Paris, France.
authors.forename: Florence
authors.initials: F
authors.lastname: Missud
authors.affiliation: Service d'Hématologie Pédiatrique, AP-HP, Hôpital Robert-Debré, Paris, France.|Centre de Référence de la Drépanocytose, Paris, France.
authors.forename: Laurent
authors.initials: L
authors.lastname: Holvoet
authors.affiliation: Service d'Hématologie Pédiatrique, AP-HP, Hôpital Robert-Debré, Paris, France.|Centre de Référence de la Drépanocytose, Paris, France.
authors.forename: Ghislaine
authors.initials: G
authors.lastname: Ithier
authors.affiliation: Hôpital Robert Ballanger, Aulnay sous Bois, France.
authors.forename: Marie
authors.initials: M
authors.lastname: Belloy
authors.affiliation: AP-HP, Hôpital Louis Mourier, Colombes, France.
authors.forename: Marie-Héléne
authors.initials: MH
authors.lastname: Odièvre
authors.affiliation: Hôpital de Gonesse, Gonesse, France.
authors.forename: Michel
authors.initials: M
authors.lastname: Benemou
authors.affiliation: AP-HP, Hôpital Jean Verdier, Bondy, France.
authors.forename: Patricia
authors.initials: P
authors.lastname: Benhaim
authors.affiliation: Centre Hospitalier René Dubos, Pontoise, France.
authors.forename: Brigitte
authors.initials: B
authors.lastname: Retali
authors.affiliation: Centre Hospitalier Victor Dupouy, Argenteuil, France.
authors.forename: Philippe
authors.initials: P
authors.lastname: Bensaid
authors.affiliation: Hôpital Simone Veil, Montmorency, France.
authors.forename: Brigitte
authors.initials: B
authors.lastname: Monier
authors.affiliation: AP-HP, Hôpital Necker-Enfants Malades, Paris, France.
authors.forename: Valentine
authors.initials: V
authors.lastname: Brousse
authors.affiliation: Centre Hospitalier Intercommunal de Saint-Denis, Saint-Denis, France.
authors.forename: Roger
authors.initials: R
authors.lastname: Amira
authors.affiliation: Centre Hospitalier de Bry-sur-Marne, Bry-sur-Marne, France.
authors.forename: Christine
authors.initials: C
authors.lastname: Orzechowski
authors.affiliation: AP-HP, Hôpital Armand Trousseau, Paris, France.
authors.forename: Emmanuelle
authors.initials: E
authors.lastname: Lesprit
authors.affiliation: CHI des Portes de l'Oise, Beaumont-sur-Oise, France.
authors.forename: Laurent
authors.initials: L
authors.lastname: Mangyanda
authors.affiliation: Centre Hospitalier de Marne-la-Vallée, Jossigny, France.
authors.forename: Nathalie
authors.initials: N
authors.lastname: Garrec
authors.affiliation: Génétique Moléculaire et Biochimie, AP-HP, Hôpital Robert-Debré, Paris, France.|Univ Paris Diderot, Sorbonne Paris Cité, Paris, France.|Inserm UMR S1134, Paris, France.
authors.forename: Jacques
authors.initials: J
authors.lastname: Elion
authors.affiliation: Unité d'Epidémiologie Clinique, AP-HP, Hôpital Robert-Debré, Paris, France.|Univ Paris Diderot, Sorbonne Paris Cité, Paris, France.|Inserm 1123, Paris, France.
authors.forename: Corinne
authors.initials: C
authors.lastname: Alberti
authors.affiliation: Univ Paris Diderot, Sorbonne Paris Cité, Paris, France.|Service d'Hématologie Pédiatrique, AP-HP, Hôpital Robert-Debré, Paris, France.|Institut Universitaire d'Hématologie (EA3518), Université Paris Diderot, Paris, France.
authors.forename: André
authors.initials: A
authors.lastname: Baruchel
authors.affiliation: Service d'Hématologie Pédiatrique, AP-HP, Hôpital Robert-Debré, Paris, France.|Centre de Référence de la Drépanocytose, Paris, France.|Inserm 1123, Paris, France.
authors.forename: Malika
authors.initials: M
authors.lastname: Benkerrou
mesh_terms: D056586:Acute Chest Syndrome / Q000209:etiology; D000755:Anemia, Sickle Cell / Q000150:complications / Q000188:drug therapy / Q000401:mortality; D002648:Child; D002675:Child, Preschool; D015331:Cohort Studies; D005260:Female; D005500:Follow-Up Studies; D019983:Guideline Adherence; D006801:Humans; D006918:Hydroxyurea / Q000627:therapeutic use; D007231:Infant, Newborn; D008297:Male; D010297:Paris; D058996:Quality Improvement / Q000592:standards*; D012189:Retrospective Studies; D020521:Stroke / Q000209:etiology; D013789:Thalassemia
chemical_list: D006918:Hydroxyurea
keywords: hydroxycarbamide; mortality; newborn screening; sickle cell disease; transfusion