Parents' attitudes towards varicella vaccination acceptance in France and Germany: effect of vaccine recommendation and reimbursement (a survey)

Aims: To ascertain physicians' and parents' attitudes towards varicella vaccination acceptance and to compare them between Germany (G), where routine varicella vaccination is recommended in children, and France (F), where it is not. Methods: Study design: cross-sectional pharmacoepidemiological study conducted in pediatric practice. Data included descriptions of the vaccinated children by pediatricians and descriptions of the parents’ attitudes using a self-administered questionnaire. The next five successive children, under 15 years old, vaccinated against varicella were included in the study. Results: Six hundred ninety-five pediatricians (F: 186; G: 509) and 2,593 parents (F: 664; G: 1,929) were included in the study. Initially, 7.1% of the German parents and 15.3% of the French parents were reluctant to have their children inoculated with the varicella vaccine (p < 0.0001). The main reason for their reluctance was the ‘fear of complications due to the vaccination’ in both countries (G: 60.0% vs 55.5%; p: ns). Fewer German parents thought that the varicella vaccine was too recent (5.9% vs 45.5%; p < 0.0001), and they were also less reluctant due to the cost of the vaccination (G: 11.9% vs F:22.8%; p < 0.02). In both countries, the most convincing arguments for parents who were initially reluctant were 'information on the potential seriousness of the disease,' which was reported by three-quarters of the parents (G: 70.0% vs F: 74.3%; p: ns), and 'availability of an effective, well-tolerated vaccine' (G: 59.4% vs F: 64.0%; p: ns). Conclusion: Even in the absence of an official recommendation, French parents will accept varicella vaccine to the same extent as German parents where it is advisable if they receive appropriate information about the potential severity of the disease and the efficacy and safety of the vaccine

Qualification et quantification des flux de la mine au lagon et de leurs impacts. Rapport synthétique des travaux

- Le présent document correspond au rapport synthétique du programme « Immila, Qualification et quantification des flux de la mine au lagon et de leurs impacts ».- Le texte de ce rapport synthétique est volontairement réduit pour faciliter sa lecture. Il renvoie à des paragraphes détaillés, des figures et des tableaux de données qui sont présentés dans le rapport scientifique final et le volume d’annexes (contributions complètes, consultables sur : https://cnrt.nc/bassin-versant/rapport-scientifique-lie-au-projet-immila-de-la-mine-au-lagon-edition-2020/).- Ce programme s’insère dans un ensemble de deux projets sur le « Transport solide dans les bassins versants miniers » composé de :• IMMILA, Impact de la mine au lagon, 2 volumes : Rapport scientifique et Annexes (contributions complètes) • GESTION DU PASSIF de l’activité minière et remédiation, 2 volumes : Rapport scientifique et Guide méthodologique.- Cet ensemble de projets traite des causes et des mécanismes de mobilisation des matériaux et propose des mesures de remédiation aux engravements anciens ou plus récents des rivières calédoniennes

Qualification et quantification des flux de la mine au lagon et de leurs impacts. Rapport scientifique final

- Le présent document correspond au rapport scientifique final du programme « Immila, Qualification et quantification des flux de la mine au lagon et de leurs impacts ». - Le programme s’est attaché à caractériser les impacts des activités minières (passées et actuelles) selon une approche systémique étudiant les ensembles bassins versants (en aval des mines), hydrosystèmes et système côtier. Ces impacts, qui modifient les caractéristiques des différents compartiments (versant, rivière, estuaire, lagon), génèrent également des effets sur la société. Ils peuvent amplifier les risques (sanitaires, naturels…) et la pression sur les ressources liées aux écosystèmes, et entraîner l’altération des paysages. Pour discriminer le rôle des activités minières sur d’autres sources de dégradation, la situation d’un bassin versant exploité a été comparée à celle d’un bassin versant témoin, non exploité. - L’étude des phénomènes s’est réalisée à trois échelles temporelles (temps long – quelques siècles, période récente – 50 dernières années, actuel), ce qui a permis de replacer les dynamiques actuelles dans leur trajectoire évolutive. La spatialisation croisée des informations physiques, biophysiques et sociales a été proposée pour constituer un outil innovant d’aide à la décision.- Ce programme s’insère dans un ensemble de deux projets sur le « Transport solide dans les bassins versants miniers » composé de :• IMMILA, Impact de la mine au lagon, 2 volumes : Rapport scientifique et Annexes (contributions complètes, consultables sur : https://cnrt.nc/bassin-versant/rapport-scientifique-lie-au-projet-immila-de-la-mine-au-lagon-edition-2020/) • GESTION DU PASSIF de l’activité minière et remédiation, 2 volumes : Rapport scientifique et Guide méthodologique.- Cet ensemble de projets traite des causes et des mécanismes de mobilisation des matériaux et propose des mesures de remédiation aux engravements anciens ou plus récents des rivières calédoniennes

A case of platelet transfusion refractoriness in a CD36 ‐negative patient with acute myeloid leukemia: Diagnostic and therapeutic management

International audienc

Association between prophylactic angiotensin-converting enzyme inhibitors and overall survival in Duchenne muscular dystrophy—analysis of registry data

International audienceAims: To estimate the effect of prophylactic angiotensin-converting enzyme inhibitors (ACEi) on survival in Duchenne muscular dystrophy (DMD).Methods and results: We analysed the data from the French multicentre DMD Heart Registry (ClinicalTrials.gov: NCT03443115). We estimated the association between the prophylactic prescription of ACEi and event-free survival in 668 patients aged 8 to 13 years, with normal left ventricular function, using (i) a Cox model with intervention as a time-dependent covariate, (ii) a propensity-based analysis comparing ACEi treatment vs. no treatment, and (iii) a set of sensitivity analyses. The study outcomes were overall survival and hospitalizations for heart failure (HF) or acute respiratory failure. Among the 668 patients included in the DMD Heart Registry, 576 (mean age 6.1 ± 2.8 years) were eligible for this study, of whom 390 were treated with ACEi prophylactically. Death occurred in 53 patients (13.5%) who were and 60 patients (32.3%) who were not treated prophylactically with ACEi, respectively. In a Cox model with intervention as a time-dependent variable, the hazard ratio (HR) associated with ACEi treatment was 0.49 [95% confidence interval (CI) 0.34-0.72] and 0.47 (95% CI 0.31-0.17) for overall mortality after adjustment for baseline variables. In the propensity-based analysis, 278 patients were included in the treatment group and 834 in the control group, with 18.5% and 30.4% 12-year estimated probability of death, respectively. ACEi were associated with a lower risk of death (HR 0.39; 95% CI 0.17-0.92) and hospitalization for HF (HR 0.16; 95% CI 0.04-0.62). All other sensitivity analyses yielded similar results.Conclusion: Prophylactic ACEi treatment in DMD was associated with a significantly higher overall survival and lower rates of hospitalization for HF

Amelogenesis imperfecta: Next-generation sequencing sheds light on Witkop’s classification

Amelogenesis imperfecta (AI) is a heterogeneous group of genetic rare diseases disrupting enamel development (Smith et al., Front Physiol, 2017a, 8, 333). The clinical enamel phenotypes can be described as hypoplastic, hypomineralized or hypomature and serve as a basis, together with the mode of inheritance, to Witkop’s classification (Witkop, J Oral Pathol, 1988, 17, 547–553). AI can be described in isolation or associated with others symptoms in syndromes. Its occurrence was estimated to range from 1/700 to 1/14,000. More than 70 genes have currently been identified as causative.Objectives: We analyzed using next-generation sequencing (NGS) a heterogeneous cohort of AI patients in order to determine the molecular etiology of AI and to improve diagnosis and disease management.Methods: Individuals presenting with so called “isolated” or syndromic AI were enrolled and examined at the Reference Centre for Rare Oral and Dental Diseases (O-Rares) using D4/phenodent protocol (www.phenodent.org). Families gave written informed consents for both phenotyping and molecular analysis and diagnosis using a dedicated NGS panel named GenoDENT. This panel explores currently simultaneously 567 genes. The study is registered under NCT01746121 and NCT02397824 (https://clinicaltrials.gov/).Results: GenoDENT obtained a 60% diagnostic rate. We reported genetics results for 221 persons divided between 115 AI index cases and their 106 associated relatives from a total of 111 families. From this index cohort, 73% were diagnosed with non-syndromic amelogenesis imperfecta and 27% with syndromic amelogenesis imperfecta. Each individual was classified according to the AI phenotype. Type I hypoplastic AI represented 61 individuals (53%), Type II hypomature AI affected 31 individuals (27%), Type III hypomineralized AI was diagnosed in 18 individuals (16%) and Type IV hypoplastic-hypomature AI with taurodontism concerned 5 individuals (4%). We validated the genetic diagnosis, with class 4 (likely pathogenic) or class 5 (pathogenic) variants, for 81% of the cohort, and identified candidate variants (variant of uncertain significance or VUS) for 19% of index cases. Among the 151 sequenced variants, 47 are newly reported and classified as class 4 or 5. The most frequently discovered genotypes were associated with MMP20 and FAM83H for isolated AI. FAM20A and LTBP3 genes were the most frequent genes identified for syndromic AI. Patients negative to the panel were resolved with exome sequencing elucidating for example the gene involved ie ACP4 or digenic inheritance.Conclusion: NGS GenoDENT panel is a validated and cost-efficient technique offering new perspectives to understand underlying molecular mechanisms of AI. Discovering variants in genes involved in syndromic AI (CNNM4, WDR72, FAM20A … ) transformed patient overall care. Unravelling the genetic basis of AI sheds light on Witkop’s AI classification