Paediatric Respiratory Reviews最新文献_第10页

Evaluation of the therapeutic efficacy and tolerability of current drug treatments on the clinical outcomes of paediatric spinal muscular atrophy type 1: A systematic review 评估当前药物治疗对小儿脊髓性肌萎缩症 1 型临床结果的疗效和耐受性：系统综述

IF 5.8 3区医学

Paediatric Respiratory Reviews Pub Date : 2023-12-01 DOI: 10.1016/j.prrv.2023.06.004

Anmar Al-Taie , Aygül Köseoğlu

{"title":"Evaluation of the therapeutic efficacy and tolerability of current drug treatments on the clinical outcomes of paediatric spinal muscular atrophy type 1: A systematic review","authors":"Anmar Al-Taie , Aygül Köseoğlu","doi":"10.1016/j.prrv.2023.06.004","DOIUrl":"10.1016/j.prrv.2023.06.004","url":null,"abstract":"<div>Spinal muscular atrophy (SMA) is a severe hereditary lower motor neuron disorder characterised by degeneration of alpha motor neurons in the spinal cord, resulting in progressive weakness and paralysis of proximal muscles. A systematic literature search was carried out by using PRISMA guidelines and searching through different databases that could provide findings of evidence on the health outcomes of the approved therapies for the management of paediatric SMA type 1 regarding efficacy with follow-up in terms of motor and respiratory functions and the tolerability and incidence of adverse drug reactions (ADRs) post-treatment from real-world publications. Half of the publications (50%) had a prospective observational design. Eight studies (66.7%) assessed nusinersen, and three studies (25%) assessed onasemnogene abeparvovec with a duration of follow-up ranging from 6 months to 3 years to evaluate the motor and respiratory functions using different assessment tools, hospitalisation rates, and the tolerability and incidence of ADRs post-treatment. The three currently approved treatments for SMA type 1 provided good support and health outcomes in terms of motor function, respiratory outcomes, reduction of hospitalisations, and improvement of survival. Nevertheless, uncertainties regarding continued improvement after long-term illness and the generalizability of results are still unknown.</div>","PeriodicalId":19658,"journal":{"name":"Paediatric Respiratory Reviews","volume":"48 ","pages":"Pages 65-71"},"PeriodicalIF":5.8,"publicationDate":"2023-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10327452","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Cough medicines for children- time for a reality check 儿童止咳药--是时候对现实进行检查了

IF 5.8 3区医学

Paediatric Respiratory Reviews Pub Date : 2023-12-01 DOI: 10.1016/j.prrv.2023.08.003

Gene Clark , Dominic A. Fitzgerald , Bruce K. Rubin

{"title":"Cough medicines for children- time for a reality check","authors":"Gene Clark , Dominic A. Fitzgerald , Bruce K. Rubin","doi":"10.1016/j.prrv.2023.08.003","DOIUrl":"10.1016/j.prrv.2023.08.003","url":null,"abstract":"<div>Cough medicines have been in use for over a century to treat the common and troublesome, but often helpful, symptoms of cough in children. They contain various combinations of “anti-tussive” drugs including opioids, antihistamines, herbal preparations, mucolytics, decongestants and expectorants. Whilst theoretically attractive for symptom relief when children are suffering, as time has passed these popular over the counter medicines have been shown to lack efficacy, delay more serious underlying diagnoses, and can cause complications and sometimes death. This has resulted in clinician concerns, a citizen petition to the American Food and Drug Association in 2007, some self-regulation from manufacturers and escalating restrictions on their use from regulatory agencies across the world over the last twenty years. This article will review the protective role of cough, juxtapose the conflicting treatment goals of suppressing a dry cough and promoting expectoration for a wet cough, consider the evidence basis for prescribing cough medicines in comparison to other more specific treatments such as for asthma [beta agonists] or infection [antibiotics], regulatory interventions, and conclude with the view that over counter cough medicines should not be used in children, especially young children.</div>","PeriodicalId":19658,"journal":{"name":"Paediatric Respiratory Reviews","volume":"48 ","pages":"Pages 30-38"},"PeriodicalIF":5.8,"publicationDate":"2023-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10278955","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

An innovative strategy for personalised medicine in a CFSPID case that evolved with time 随着时间的推移，CFSPID病例中个性化药物的创新策略。

IF 5.8 3区医学

Paediatric Respiratory Reviews Pub Date : 2023-09-01 DOI: 10.1016/j.prrv.2023.06.001

J. Twynam-Perkins , A. Fall , J.W. Lefferts , D.S. Urquhart

引用次数: 0

Domiciliary management of infants and children with chronic respiratory diseases 患有慢性呼吸道疾病的婴儿和儿童的户籍管理。

IF 5.8 3区医学

Paediatric Respiratory Reviews Pub Date : 2023-09-01 DOI: 10.1016/j.prrv.2023.04.001

Henry J. Rozycki , Sailesh Kotecha

引用次数: 0

Airway epithelial development and function: A key player in asthma pathogenesis? 气道上皮的发育和功能：哮喘发病机制的关键因素？

IF 5.8 3区医学

Paediatric Respiratory Reviews Pub Date : 2023-09-01 DOI: 10.1016/j.prrv.2023.04.005

Grigorios Chatziparasidis , Andrew Bush , Maria Rafailia Chatziparasidi , Ahmad Kantar

引用次数: 0

Variable and evolving degrees of CFTR dysfunction: Implications for diagnosis and clinical management CFTR功能障碍的变化和发展程度：对诊断和临床管理的意义。

IF 5.8 3区医学

Paediatric Respiratory Reviews Pub Date : 2023-09-01 DOI: 10.1016/j.prrv.2023.07.001

A. Shawcross, J.C. Davies, R. Pabary

引用次数: 0

Airway clearance in patients with neuromuscular disease 神经肌肉疾病患者的气道清除率。

IF 5.8 3区医学

Paediatric Respiratory Reviews Pub Date : 2023-09-01 DOI: 10.1016/j.prrv.2023.02.002

Alexander Ilan Gipsman , Nicole Christine Lapinel , Oscar Henry Mayer

{"title":"Airway clearance in patients with neuromuscular disease","authors":"Alexander Ilan Gipsman , Nicole Christine Lapinel , Oscar Henry Mayer","doi":"10.1016/j.prrv.2023.02.002","DOIUrl":"10.1016/j.prrv.2023.02.002","url":null,"abstract":"<div>Airway clearance is a critical component of both maintenance of respiratory health and management of acute respiratory illnesses. The process of effective airway clearance begins with the recognition of secretions in the airway and culminates in expectoration or swallowing. There are multiple points on this continuum at which neuromuscular disease causes impaired airway clearance. This can result in an otherwise mild upper respiratory illness progressing unabated from an easily managed condition to a severe, life-threatening lower respiratory illness requiring intensive therapy for patient recovery. Even during periods of relative health, airway protective mechanisms can be compromised, and patients may have difficulty managing average quantities of secretions. This review summarizes airway clearance physiology and pathophysiology, mechanical and pharmacologic treatment modalities, and provides a practical approach for managing secretions in patients with neuromuscular disease. Neuromuscular disease is an umbrella term used to describe disorders that involve dysfunction of peripheral nerves, the neuromuscular junction, or skeletal muscle. Although this paper specifically reviews airway clearance pertaining to those with neuromuscular diseases (e.g., muscular dystrophy, spinal muscular atrophy, myasthenia gravis), most of its content is relevant to the management of patients with central nervous system disorders such as chronic static encephalopathy caused by trauma, metabolic or genetic abnormalities, congenital infection, or neonatal hypoxic-ischemic injury.</div>","PeriodicalId":19658,"journal":{"name":"Paediatric Respiratory Reviews","volume":"47 ","pages":"Pages 33-40"},"PeriodicalIF":5.8,"publicationDate":"2023-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9076576","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 1

Cystic Fibrosis screen positive, inconclusive diagnosis (CFSPID) to Cystic Fibrosis: Detecting disease with serial assessments 囊性纤维化筛查阳性，非结论性诊断（CFSPID）囊性纤维化：用系列评估检测疾病。

IF 5.8 3区医学

Paediatric Respiratory Reviews Pub Date : 2023-09-01 DOI: 10.1016/j.prrv.2023.06.002

J. Manzoor, D.A. Hughes

引用次数: 0

The United States’ decision to mask children as young as two for COVID-19 has been extended into 2023 and beyond: The implications of this policy 美国为新冠肺炎两岁儿童戴口罩的决定已延长至2023年及以后：这项政策的影响。

IF 5.8 3区医学

Paediatric Respiratory Reviews Pub Date : 2023-09-01 DOI: 10.1016/j.prrv.2023.04.004

Tracy Beth Høeg , Sebastián González-Dambrauskas , Vinay Prasad