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).

Hydroxyurea prescription, availability and use for children with sickle cell disease in Italy: Results of a National Multicenter survey

“…Our national survey performed in pediatric hematological reference centers reveals an increase in the number of children on HU treatment in recent years, after national guidelines dissemination. The proportion of children on HU (40%) is quite high compared to published reports in Europe that remain lower …”

“…The age at diagnosis is high, surely due to the lack of a national newborn screening program, but nevertheless children start HU at a mean age that is remarkably high compared to other countries (i.e. 3.7 years in France) . Since the majority of patients began HU after the publication of the Italian National Guidelines, there was probably unawareness on HU indications and lack of confidence on its use among pediatric hematologists and the change in clinical attitude could be due to guidelines dissemination as it has already happened in the AIEOP network .…”

Cyclophosphamide improves engraftment in patients with SCD and severe organ damage who undergo haploidentical PBSCT

“…Furthermore, the transplant approach in children should be sufficiently safe to justify providing transplants for a patient population in which 99% are expected to reach 18 years of age. 59,60 Such an approach can serve to maximize the chance that a successful transplant will truly improve not only the quality but also the quantity of life.…”

Survival in adults with sickle cell disease in a high-income setting

“…In 1960, it was described as a "disease of childhood" 1 whereas 25 years later, the Cooperative Study of Sickle Cell Disease reported that 85% of hemoglobin SS (HbSS) patients lived to adulthood. More recently, the estimate is 99% in London, 2 97% in Paris, 3 and 94% in the United States. 4 Survival estimates have continued to improve; in 1994, the median survival for patients with HbSS/Sb 0 thalassemia was estimated at 42 to 48 years, 5 increasing to 53 to 58 years in Jamaica in 2001 6 and 58 years in the United States in 2014.…”