de Jongste J C, Janssens H M, Van der Wouden J
Department of Pediatrics, Division Pediatric Respiratory Medicine, Erasmus University and University Hospital/Sophia Children's Hospital, Rotterdam, The Netherlands.
Allergy. 2002;57 Suppl 74:42-7. doi: 10.1034/j.1398-9995.57.s74.6.x.
The term "effectiveness" relates to the question of whether or not a certain treatment works in practice. Usually, such a treatment was first evaluated under tightly controlled conditions in selected patient populations, and the potential benefits were shown. There is, however, a great difference between the efficacy of a given treatment, indicating its optimal therapeutic action in controlled trials, and its effectiveness when applied to a less well-defined population of patients in daily practice. This is especially relevant for asthma in young children, where many factors are responsible for the difference. Among these are, first of all, the heterogeneity of the wheezing phenotype. Other factors include the compliance with prescribed treatments, as determined by the attitude of doctors and parents towards such treatment, the ease of administration and the perceived effects and side effects. Also, the performance of different inhaler devices may be insufficient for a good, reliable dose deposition in young children in daily life. As a result, the current treatment guidelines for preschool children with recurrent wheeze are probably too optimistic in assuming that inhaled treatment is most effective and feasible at all ages. We propose careful re-evaluation of such recommendations in a first-line setting resembling daily life as closely as possible, and consideration of oral treatments as well. Also, we need methods to separate the different phenotypes within the group of recurrently wheezing preschool children to optimize targeting of asthma treatment to those who have ongoing airway inflammation.
“有效性”一词涉及某种治疗方法在实际应用中是否有效的问题。通常，这种治疗方法首先在选定的患者群体中，在严格控制的条件下进行评估，并显示出潜在的益处。然而，特定治疗方法的疗效（表明其在对照试验中的最佳治疗作用）与在日常实践中应用于定义不太明确的患者群体时的有效性之间存在很大差异。这对于幼儿哮喘尤为重要，其中许多因素导致了这种差异。首先，这些因素包括喘息表型的异质性。其他因素包括对规定治疗的依从性，这取决于医生和家长对这种治疗的态度、给药的难易程度以及感知到的效果和副作用。此外，在日常生活中，不同吸入器装置的性能可能不足以在幼儿中实现良好、可靠的剂量沉积。因此，目前针对反复喘息的学龄前儿童的治疗指南可能过于乐观地认为吸入治疗在所有年龄段都是最有效和可行的。我们建议在尽可能贴近日常生活的一线环境中仔细重新评估此类建议，并考虑口服治疗方法。此外，我们需要方法来区分反复喘息的学龄前儿童群体中的不同表型，以便将哮喘治疗优化针对那些存在持续性气道炎症的儿童。
