14 research outputs found
Late relapse after hematopoietic stem cell transplantation for acute leukemia: a retrospective study by SFGM-TC.
peer reviewedLate relapse (LR) after allogeneic hematopoietic stem cell transplantation (AHSCT) for acute leukemia is a rare event (nearly 4.5%) and raises the questions of prognosis and outcome after salvage therapy. We performed a retrospective multicentric study between January 1, 2010, and December 31, 2016, using data from the French national retrospective register ProMISe provided by the SFGM-TC (French Society for Bone Marrow Transplantation and Cellular Therapy). We included patients presenting with LR, defined as a relapse occurring at least 2 years after AHSCT. We used the Cox model to identify prognosis factors associated with LR. During the study period, a total of 7582 AHSCTs were performed in 29 centers, and 33.8% of patients relapsed. Among them, 319 (12.4%) were considered to have LR, representing an incidence of 4.2% for the entire cohort. The full dataset was available for 290 patients, including 250 (86.2%) with acute myeloid leukemia and 40 (13.8%) with acute lymphoid leukemia. The median interval from AHSCT to LR was 38.2 months (interquartile range [IQR], 29.2 to 49.7 months), and 27.2% of the patients had extramedullary involvement at LR (17.2% exclusively and 10% associated with medullary involvement). One-third of the patients had persistent full donor chimerism at LR. Median overall survival (OS) after LR was 19.9 months (IQR, 5.6 to 46.4 months). The most common salvage therapy was induction regimen (55.5%), with complete remission (CR) obtained in 50.7% of cases. Ninety-four patients (38.5%) underwent a second AHSCT, with a median OS of 20.4 months (IQR, 7.1 to 49.1 months). Nonrelapse mortality after second AHSCT was 18.2%. The Cox model identified the following factors as associated with delay of LR: disease status not in first CR at first HSCT (odds ratio [OR], 1.31; 95% confidence interval [CI], 1.04 to 1.64; P = .02) and the use of post-transplantation cyclophosphamide (OR, 2.23; 95% CI, 1.21 to 4.14; P = .01). Chronic GVHD appeared to be a protective factor (OR, .64; 95% CI, .42 to .96; P = .04). The prognosis of LR is better than in early relapse, with a median OS after LR of 19.9 months. Salvage therapy associated with a second AHSCT improves outcome and is feasible, without creating excess toxicity
Tout savoir sur le treat and extend : organisation et optimisation. Les Cahiers d'Ophtalmologie
National audienceLa dégénérescence maculaire liée à l’âge (DMLA) est actuellement la première cause de handicap visuel dans les pays développés chez les plus de 50 ans. Elle touche en effet environ 8% de la population française et plus de 25% des plus de 75 ans. La constatation d’une insuffisance de traitement lors d’un régime « réactif » a poussé les cliniciens à proposer d’autres types de régimes. Le « Treat and Extend » (T&E) est né de la volonté d’adapter les intervalles de retraitement des patients pour une prise en charge plus personnalisée
Fibroblasts mediate endothelium response to angiogenic cues in a newly developed 3D stroma engineered model
International audienc
Targeted panel sequencing establishes the implication of planar cell polarity pathway and involves new candidate genes in neural tube defect disorders
International audienceNeural tube defect disorders are developmental diseases that originate from an incomplete closure of the neural tube during embryogenesis. Despite high prevalence-1 out of 3000 live births-their etiology is not yet established and both environmental and genetic factors have been proposed, with a heritability rate of about 60%. Studies in mouse models as well as in human have further suggested a multifactorial pattern of inheritance for neural tube defect disorders. Here, we report results obtained from clinical diagnosis and NGS analysis of a cohort composed of 52 patients. Using a candidate gene panel approach, we identified variants in known genes of planar cell polarity (PCP) pathway, although with higher prevalence than previously reported. Our study also reveals variants in novel genes such as FREM2 and DISP1. Altogether, these results confirm the implication of the PCP genes and involve the FRAS/FREM2 complex and Sonic Hedgehog signaling as novel components in the appearance of NTDs
Inflammatory Bowel Diseases and School Absenteeism
International audienceObjectives - Inflammatory bowel diseases (IBDs) are chronic diseases which negatively affect the schooling of children. The aim is to analyze school absenteeism and its causes in children followed for IBD. Methods - A prospective multicenter study of IBD patients aged from 5 to 18 years old, from September 2016 to June 2017. Data on absenteeism and its causes were collected via a monthly questionnaire completed by patients or their family by mail. The results were compared with existing data supplied by the school authorities (497 students without IBD divided by class). Results - A total of 106 patients (62 boys), median age of 14 (5-18), were included. The global response rate was 83.1%. The patients with IBD were absent an average of 4.8% ± 5.5% of school days during the school year, against 3.2% ± 1.6% for non IBD group (P = 0.034). Digestive disorders accounted for 34% of the causes of absenteeism. Approximately 27% of the absences were due to scheduled events (hospitalizations, endoscopy, or consultations). By excluding the absences for scheduled care, the rate of school absenteeism of patients with IBD is significantly lower than that of non-IBD group. Conclusion - Children with IBD are more frequently absent from school than non-IBD group. The main cause of school absenteeism appears to be associated with the disease itself. The share of scheduled absenteeism is quite large. The organization and scheduling of the patients' care path must be a priority to maximally limit the negative impact of their disease on the patients' schooling
OmegaROP-2 prospective study: Expression of placental fatty acid receptors in preterm newborns with retinopathy of prematurity
Abstract Background Incomplete vascularization of the retina in preterm infants carries a risk of retinopathy of prematurity (ROP). Progress in neonatal resuscitation in developing countries has led to the survival of an increasing number of premature infants, resulting in an increased rate of ROP and consequently in visual disability. Strategies to reduce ROP involve optimizing oxygen saturation, nutrition, and normalizing factors such as insulin-like growth factor 1 and n-3 long-chain polyunsaturated fatty acids (LC-PUFA). Our previous study, OmegaROP, showed that there is an accumulation or retention of docosahexaenoic acid (DHA) in mothers of infants developing ROP, suggesting abnormalities in the LC-PUFA placental transfer via fatty acid transporting proteins. The present study aims to better understand the LC-PUFA transport dysfunction in the fetoplacental unit during pregnancy and to find a novel target for the prevention of ROP development. Methods The study protocol is designed to evaluate the correlation between the expression level of placental fatty acid receptors and ROP occurrence. This ongoing study will include 100 patients: patients giving birth before 29 weeks of gestational age (GA) and patients with full-term pregnancies. Recruitment is planned for over 46 months. Maternal and cord blood samples as well as placental tissue samples will be taken following delivery. ROP screening will be performed using wide-field camera imaging according to the International Classification of ROP consensus statement. Discussion The results of this study will have a tangible impact on public health. Indeed, if we show a correlation between the expression level of placental omega-3 receptors and the occurrence of ROP, it would be an essential step in discovering novel pathophysiological mechanisms involved in this retinopathy. Trial registration: 2020-A03253-3
Oligogenic inheritance in neural tube defects: major involvment of primary cilia, planar cell polarity and extracellular matrix genes
International audienc
Study protocol of OmegaROP-2 prospective study: expression of placental fatty acid receptors in preterm newborns with retinopathy of prematurity
Correspondence to: [email protected] audienceBackground Incomplete vascularization of the retina in preterm infants carries a risk of retinopathy of prematurity (ROP). Progress in neonatal resuscitation in developing countries has led to the survival of an increasing number of premature infants, resulting in an increased rate of ROP and consequently in visual disability. Strategies to reduce ROP involve optimizing oxygen saturation, nutrition, and normalizing factors such as insulin-like growth factor 1 and n-3 long-chain polyunsaturated fatty acids (LC-PUFA). Our previous study, OmegaROP, showed that there is an accumulation or retention of docosahexaenoic acid (DHA) in mothers of infants developing ROP, suggesting abnormalities in the LC-PUFA placental transfer via fatty acid transporting proteins. The present study aims to better understand the LC-PUFA transport dysfunction in the fetoplacental unit during pregnancy and to find a novel target for the prevention of ROP development. Methods The study protocol is designed to evaluate the correlation between the expression level of placental fatty acid receptors and ROP occurrence. This ongoing study will include 100 mother-infant dyads: mother-infant dyads born before 29 weeks of gestational age (GA) and mother-infant dyads with full-term pregnancies. Recruitment is planned over a period of 46 months. Maternal and cord blood samples as well as placental tissue samples will be taken following delivery. ROP screening will be performed using wide-field camera imaging according to the International Classification of ROP consensus statement. Discussion The results of this study will have a tangible impact on public health. Indeed, if we show a correlation between the expression level of placental omega-3 receptors and the occurrence of ROP, it would be an essential step in discovering novel pathophysiological mechanisms involved in this retinopathy. Trial registration NCT04819893
Effects of topical docosahexaenoic acid on postoperative fibrosis in an animal model of glaucoma filtration surgery
International audiencePurpose The aim of this study was to evaluate docosahexaenoic acid (DHA) as a potential antifibrotic agent after glaucoma filtration surgery (GFS) in rats. Methods A total of 36 10-week-old Brown Norway rats underwent GFS. Animals were equally divided into three groups: a control group, a DHA group and a mitomycin C (MMC) group. Intraocular pressure (IOP) was measured using a dynamic rebound tonometer, and a photograph of the surgical site was taken on days 1, 3, 7, 10, 14 and 17. The incorporation of DHA into fibroblasts was evaluated by gas chromatography. The expression of alfa-smooth muscle actin (α-SMA) and Smad proteins was assessed by Western blotting. Results IOP decreased after surgery in animals from the three groups on day 1 after surgery. Over time, IOP remained lower in the DHA and MMC groups than in the control group (median [interquartile range] 8.0 [7.0–8.0] and 8.0 [7.3–8.0] mmHg vs. 9.0 [8.0–9.0] mmHg, respectively; p < 0.001). Bleb area in the DHA and MMC groups remained larger than that of the control group from day 7 to day 14 (3.9 [2.9–5.2] and 3.5 [2.3–4.4] mm2 vs. 2.3 [2.0–2.8] mm2, respectively; p = 0.0021). We did not observe any change in DHA concentrations in the fibroblasts of the DHA group compared with the other groups. Conclusion The impact of DHA on IOP and bleb area was similar to that of MMC. The mechanisms of action of DHA in rat eye fibroblasts deserve further investigation
Sciences participatives et restitution
On appelle « sciences participatives » les programmes scientifiques visant à répondre directement ou indirectement à des demandes de la société civile, et qui associent les diverses parties prenantes dans la construction d’un objet, dans son exploration, et dans les décisions qui peuvent en dériver. L’objet de ce dossier est de présenter, d’un point de vue avant tout méthodologique, un éventail de recherches participatives divergeant dans leurs modalités et dans leurs finalités. On distinguera d’abord celles dont l’objectif est d’abord d’accroître les connaissances scientifiques (articles de Couvet & Teyssèdre, Legrand, et Kohler et al.) ; puis celles qui répondent à des demandes locales (gestion des risques – Hardy ; mise en place de filières horticoles – Correia et al) ; enfin, un regard extérieur posé sur les dynamiques participatives à l’échelle micro-locale, dans leur impact sur l’organisation d’une micro-société (article de Stoll et Folhes). Dans la mesure où ce dossier se veut une présentation ample de la démarche participative, on ne s’étonnera pas d’en voir les trois premiers articles consacrés à la dimension française de la question. Quant au champ privilégié par ce type de recherche, il se situe à l’interface de la nature et de la société. Il répond à une demande forte de prise en compte de l’environnement au niveau global, demande qui vient buter, localement, sur des dynamiques sociales et économiques difficiles à maîtriser. L’intervention d’équipes scientifiques apparaît alors comme révélateur d’un état des lieux et comme médiateur entre environnement et société. Chamamos “ciências participativas” os programas científicos que visam responder diretamente ou indiretamente às necessidades da sociedade civil, e que associam as diversas partes envolvidas na construção de um objeto, na sua exploração e nas decisões que podem dele derivar. O objetivo deste dossiê é apresentar, de um ponto de vista antes de tudo metodológico, um leque de pesquisas participativas, divergindo nas suas finalidades e modalidades. Distinguiremos a princípio estas pesquisas cujo objetivo primeiro é aumentar os conhecimentos científicos (artigos de Couvet & Teyssèdre, Legrand, et Kohler et al.); em seguida os que tratam as necessidades locais (gestão de riscos – Hardy; implementação de filiais hortícolas – Correia et al) e, finalmente, um olhar exterior voltado sobre as dinâmicas participativas na escala micro local, no impacto sobre a organização de uma micro sociedade (artigo de Stoll e Folhes). Na medida em que este dossiê pretende uma apresentação ampla da iniciativa participativa, não nos surpreenderemos ao ver os três primeiros artigos consagrados à dimensão francesa da questão. Quanto ao campo privilegiado por este tipo de pesquisa, situa-se na interface da natureza e da sociedade, porquanto corresponde a uma forte necessidade de considerar o meio ambiente em nível global, pedido que vem bater, localmente, a dinâmicas sociais e econômicas difíceis de dominar. A intervenção de equipes científicas aparece então como reveladora de uma situação multifacetada e como mediadora entre meio ambiente e sociedade. We call "participative sciences" the scientific programs which aim is to answer directly or indirectly demands of the civil society, and which associate the diverse stakeholders in the construction of an object, in its exploration, and in the decisions which can derive from it. The object of this special issue is to present, from a methodological point of view, a range of participative researches diverging in their modalities and their purposes. We shall first distinguish those which primary objective is to increase scientific knowledge (Couvet and Teyssèdre; Legrand; and Kohler et al.); then those who answer local demands (risk management, Hardy; implementation of horticultural commercial networks, Correia et al); finally, an outside look put on the dynamics of participation in a micro-local scale, considering their impact on the organization of a micro-society (Stoll and Folhes). This issue aims to be an ample presentation of participative approaches. The field favored by participative research is situated in the interface of nature and society. It wishes to respond to the necessity of considering the environment at a global level, a demand that comes to locally stumble over social and economic dynamics which need to be negotiated. The intervention of scientific teams appears then as revelation of a current situation and as mediator between environment and society