source: British Journal of Haematology

year: 2016

authors: 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 N1, Elion J, Alberti C, Baruchel A, Benkerrou M

summary/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).

organization: AP-HP, Hôpital Robert-Debré, Paris; Univ Paris Diderot, Sorbonne Paris Cité; Centre de Référence de la Drépanocytose, Paris; Hôpital Robert Ballanger, Aulnay sous Bois; AP-HP, Hôpital Louis Mourier, Colombes; Hôpital de Gonesse; AP-HP, Hôpital Jean Verdier, Bondy; Centre Hospitalier René Dubos, Pontoise; Centre Hospitalier Victor Dupouy, Argenteuil; Hôpital Simone Veil, Montmorency; AP-HP, Hôpital Necker-Enfants Malades, Paris; Centre Hospitalier Intercommunal de Saint-Denis; Centre Hospitalier de Bry-sur-Marne; AP-HP, Hôpital Armand Trousseau, Paris; CHI des Portes de l'Oise, France; Centre Hospitalier de Marne-la-Vallée, Jossigny; Inserm UMR S1134, Paris; Inserm 1123, Paris; Institut Universitaire d'Hématologie (EA3518), Université Paris Diderot

DOI: 10.1111/bjh.14015

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