Instillation de surfactant chez le prématuré en respiration spontanée : méta-analyse

peer reviewedJustification: Lors du traitement par surfactant dit moins invasif (Less invasive surfactant therapy- LIST), le produit est instillé dans la trachée par un cathéter fin alors que l’enfant respire spontanément sous CPAP. Différentes études ont donné des résultats variables mais encourageants. L’objectif de cette méta-analyse est de comparer le devenir respiratoire des prématurés traités par LIST avec celui de ceux traités par administration de surfactant par un tube endotrachéal. Méthodes : les études randomisées contrôlées (ERC) sont recherchées dans les bases de données et dans les références d’articles pertinents. Les devenirs respiratoires (dysplasie broncho-pulmonaire (DBP), décès ou DBP, échec précoce de CPAP, nécessité de ventilation invasive) et les morbidités classiques sont reprises de ces études. Pour chaque morbidité, le risque relatif (RR) des données mutualisées est calculé avec une analyse de Mantel-Haenszel à modèle d’effet aléatoire. Le RR est également calculé pour des sous-groupes établis selon l’intervention contrôle. Résultats : six ERC évaluent le LIST : 4 le comparent à l’INSURE (Intubation-Surfactant-Extubation), et les 2 autres à l’intubation (immédiate ou après maintient en CPAP) avec surfactant. Les méthodes LIST diminuent les risques de DBP (RR= 0,71 (0,52-0,99) ; nombre nécessaire à traiter NNT= 21), et de décès ou DBP (RR= 0,7 (0,58- 0,94) ; NNT= 15). L’échec précoce de CPAP et le recours à la ventilation invasive sont également réduits (RR= 0,67 (0,53-0,84) ; NNT= 8 et RR= 0,69 (0,53- 0,88) ; NNT= 6). Comparé à l’INSURE, le LIST diminue le risque combiné de décès ou DBP (RR= 0,63 (0,44-0,92) ; NNT= 11), et d’échec précoce de CPAP (RR=0,71 (0,53-0,96) ; NNT= 11). Les autres morbidités néonatales classiques sont similaires pour les différents groupes. Conclusions : une stratégie d’administration dite moins invasive de surfactant diminue les risques de morbidité respiratoire à moyen terme (DBP, décès ou DBP) et le recours à la ventilation invasive. Cette approche semble sure mais les données de suivi à long terme sont insuffisantes

Social Deprivation Is Associated With Lower Access to Pre-emptive Kidney Transplantation and More Urgent-Start Dialysis in the Pediatric Population

Introduction Socioeconomic status (SES) is recognized as an important determinant of kidney health. We aimed to evaluate the association of social deprivation with different indicators at kidney replacement therapy (KRT) initiation in the French pediatric metropolitan population. Methods All patients with end-stage kidney disease (ESKD) who started KRT before 20 years old in France between 2002 and 2015 were included. We investigated different indicators at KRT initiation, which are as follows: KRT modality (dialysis vs. pre-emptive transplantation), late referral to a nephrologist, and dialysis modality (hemodialysis [HD] vs. peritoneal dialysis [PD], urgent vs. planned start of dialysis, use of catheter vs. use of fistula for HD vascular access). An ecological index (European Deprivation Index [EDI]) was used as a proxy for social deprivation. Results A total of 1115 patients were included (males 59%, median age at dialysis 14.4 years, glomerular/vascular diseases 36.8%). The most deprived group represented 38.7% of the patients, suggesting pediatric patients with ESKD come from a more socially deprived background. The most deprived group was more likely to initiate KRT with dialysis versus kidney transplantation. Among patients on HD, the odds of starting treatment in emergency with a catheter was >2-fold higher for the most deprived compared with the least deprived children (adjusted odds ratio [aOR] 2.35, 95% CI 1.16–4.78). Conclusion Children from the most deprived area have lower access to pre-emptive transplantation, have lower access to PD, tend to be late referred to a nephrologist, and have more urgent initiation of HD with a catheter

Arguments permettant aux médecins d'évoquer le diagnostic d'AVC ou d'AIT en ambulatoire

TOURS-BU Médecine (372612103) / SudocPARIS-BIUM (751062103) / SudocSudocFranceF

Cathéters pour instillation moins invasive de SURFACTANT : une étude de simulation

peer reviewedIntroduction et objectifs : l’instillation trachéale de surfactant par un cathéter fin (Less invasive surfactant therapy- LIST) chez le prématuré sous CPAP permet de diminuer la morbidité respiratoire. Plusieurs cathéters sont décrits à cette fin : une sonde oro-gastrique insérée avec (LISA-Köln, K) ou sans pince de Maggil (Take Care- Ankara, A), un cathéter veineux de 13 cm (MIST- Hobart, H), un cathéter d’angiographie de 30 cm (Stockholm, S) ou un cathéter ombilical fixé à un stylet d’intubation utilisé localement (Liège, L). L’objectif de l’étude est d’évaluer l’efficacité de ces techniques en prenant l’INSURE (Intubation-Surfactant-Extubation) comme référence. Intervention : 20 néonatologues travaillant dans 4 services ayant des stratégies d’administration du surfactant différentes ont participé. Ils ont simulé ces 6 techniques sur deux têtes d’intubation de difficulté croissante. L’efficacité de l’intervention est évaluée par le taux d’échec et la durée de procédure mesurée sur vidéo. Chaque intervenant apprécie la facilité d’utilisation sur une échelle de 1 à 9 (Difficile> facile). Résultats : Pour le premier modèle, les durées médianes de procédure pour Köln et Ankara sont allongées [K: 21s (IQR 17-24); A: 23s (15-42); H: 10s (8-16); S: 12s (10-22); L (10-20); INSURE: 14s (11-21); p<.0001]. Pour le second modèle, seul Liège permet une durée de procédure similaire à l’INSURE [K: 32s (25-44); A: 39s (27-95); H: 34s (27-46); S: 37s (29-42); L: 24s (15-35); INSURE: 24s (17-32); p<.002]. Les taux d’échec des méthodes LIST sont similaires entre eux (de 3 à 8/ 40 essais), mais supérieurs à celui de l’INSURE (0/40). Köln et Ankara sont considérés comme plus difficiles [scores de facilité : K: 5 (4-6); A: 3 (2-4); H: 6,5 (6-7); S: 7 (4-8); L: 8 (6,5-8); INSURE: 7 (6-8); p<.001]. Conclusions : les cathéters plus rigides sont plus efficaces et perçus comme plus simples d’utilisation. L’insertion d’un cathéter guidé et incurvé pourrait être plus rapide dans les cas difficiles

Surfactant instillation in spontaneously breathing preterm infants: a systematic review and meta-analysis.

Less invasive surfactant therapies (LIST) use surfactant instillation through a thin tracheal catheter in spontaneously breathing infants. This review and meta-analysis investigates respiratory outcomes for preterm infants with respiratory distress syndrome treated with LIST rather than administration of surfactant through an endotracheal tube. Randomised controlled trial (RCT) full texts provided outcome data for bronchopulmonary dysplasia (BPD), death or BPD, early CPAP failure, invasive ventilation requirements and usual neonatal morbidities. Relative risks (RR) from pooled data, with subgroup analyses, were obtained from a Mantel-Haenszel analysis using a random effect model. Six RCTs evaluated LIST: 4 vs InSurE and 1 each vs delayed or immediate intubation for surfactant. LIST resulted in decreased risks of BPD (RR = 0.71 [0.52-0.99]; NNT = 21), death or BPD (RR = 0.74 [0.58-0.94]; NNT = 15) and early CPAP failure or invasive ventilation requirements (RR = 0.67 [0.53-0.84]; NNT = 8 and RR = 0.69 [0.53-0.88]; NNT = 6). Compared to InSurE, LIST decreased the risks of BPD or death (RR = 0.63 [0.44-0.92]; NNT = 11) and of early CPAP failure (RR = 0.71 [0.53-0.96]; NNT = 11). Common neonatal morbidities were not different. CONCLUSIONS: Respiratory management with LIST decreases the risks of BPD and BPD or death, and the need for invasive ventilation. This strategy appears safe, but long-term follow-up is lacking. WHAT IS KNOWN: • Initial management of preterm infants with CPAP decreases the risk of death or BPD, but many still require surfactant or invasive ventilation. • Surfactant can be instilled through a tracheal thin catheter while the infant breathes on CPAP, but improvement in BPD is inconsistent between studies. What is New: • Less invasive surfactant therapy (LIST) strategies decrease the risks of BPD, of death or BPD, and of CPAP failure compared to strategies where surfactant is administered through an endotracheal tube. • LIST strategies decrease the risks of the composite outcome of BPD or death and of early CPAP failure when compared to "intubation-surfactant-extubation" approaches

Economic evaluation of biosimilars for reimbursement purposes – what, when, how?

Background: Limited previous research and guidelines on the design of economic evaluation for biosimilars have led to unresolved methodological questions on how to assess biosimilars. Objectives: We want to raise awareness of and explore methodological issues for the economic evaluation of biosimilars. Methods: We relied on a literature review, exploratory interviews, and our experiences. Results and Conclusions: In the majority of cases in which reimbursement for a biosimilar is sought, it will not be necessary to conduct an economic evaluation, given that the reference product is already reimbursed and standard of care. If the latter is not the case, a full economic evaluation of the biosimilar versus standard of care is needed. This might also be needed in the case of differences in administration form or adherence (for example, due to a nocebo effect) and to take into account value-added services. The entry of biosimilars and of next-generation biological products should trigger a re-assessment of the entire product class. HTA bodies and reimbursement agencies should provide clear guidance on how to assess the value of a biosimilar in each of these circumstances.status: publishe

Les cathéters rigides accélèrent l'administration moins invasive de surfactant à des mannequins.

Aim: Different catheters can be used for less invasive surfactant therapy (LIST): feeding tubes inserted with or without Magill forceps, different angiocatheters and centre specific devices, such as umbilical catheters affixed to a stylet. This study compared the effectiveness of LIST devices and endotracheal tubes (ETT). Methods: Video recordings of 20 neonatologists simulating different LIST techniques on two manikin heads were analysed. Procedural effectiveness was evaluated by the duration of procedures and failure rates. Ease of use was scored. Results: The median procedure time for the Neonatal Intubation Trainer was significantly longer with feeding tubes without Maggil forceps. For the more difficult ALS Baby Trainer, successful procedures lasted a median of 24 (17-32) seconds with ETT, 24 (15-36) seconds with stylet-guided catheters and 34 (27-46) seconds and 37 (29-42) seconds with 13cm and 30cm angiocatheters, respectively. Both methods using feeding tubes were statistically slower than ETT intubation, lasting 32 (25-44) seconds and 39 (27-95) seconds with or without Maggil forceps. Failure rates (7-20%) were no different between the LIST methods. Techniques using feeding tubes were rated as more difficult. Conclusion: Only rigid or stylet-guided catheters required tracheal catheterisation times similar to those of endotracheal intubation and neonatologists found them easier.  KEY NOTES • This manikin study used video recordings of 20 neonatologists to compare the effectiveness of devices for less invasive surfactant therapy (LIST) and endotracheal tubes. • The durations of tracheal catheterisation for LIST using rigid or stylet-guided catheters were no different from those obtained with endotracheal tubes, but feeding tubes with or without Maggil forceps required longer procedures. • Neonatologists found rigid or stylet-guided catheters easier to use

Economic evaluation of biosimilars for reimbursement purposes - what, when, how?

Background: Limited previous research and guidelines on the design of economic evaluation for biosimilars have led to unresolved methodological questions on how to assess biosimilars. Objectives: We want to raise awareness of and explore methodological issues for the economic evaluation of biosimilars. Methods: We relied on a literature review, exploratory interviews, and our experiences. Results and Conclusions: In the majority of cases in which reimbursement for a biosimilar is sought, it will not be necessary to conduct an economic evaluation, given that the reference product is already reimbursed and standard of care. If the latter is not the case, a full economic evaluation of the biosimilar versus standard of care is needed. This might also be needed in the case of differences in administration form or adherence (for example, due to a nocebo effect) and to take into account value-added services. The entry of biosimilars and of next-generation biological products should trigger a re-assessment of the entire product class. HTA bodies and reimbursement agencies should provide clear guidance on how to assess the value of a biosimilar in each of these circumstances