Effectiveness of pharmacotherapy in asthmatic preschool children

Abstract

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.

Compliance with Dysphagia Recommendations by Carers of Adults with Intellectual Impairment

“…There is evidence that the more severe the symptoms are, the more likely proxies are to comply [13][14][15][16]. In this study 11/13 dependent clients had both oral and pharyngeal dysphagia (Table 1), i.e., more severe dysphagia.…”

“…The questions required a yes/no response and then further comments were invited. The areas included in the questionnaire were based on the information suggested to be relevant by the literature [3,[10][11][12][13][14][15][16][17][18][19][20]22].…”

“…Compliance with medical guidelines ranges widely from 30% to 98% and depends on parental education, symptom severity, observation of equipment use, provision of information sheets and specialist training, caregiver beliefs in the benefits of treatment, and ongoing therapist support [15][16][17][18]. It is difficult to draw conclusions because studies investigate different conditions and use different methodologies, but the themes have been used as a basis to inform the design of this pilot study.…”

In young children, persistent wheezing is associated with bronchial bacterial infection: a retrospective analysis

“…From clinical experience and scientific reports, it is apparent that preschool wheezers are a heterogeneous group of which only part will benefit from the use of ICS [8][9][10]. Several authors recommend an extensive work-up in ICS non-responders, including gastrooesophageal reflux and allergy testing, sweat chloride test and bronchoscopy with bronchoalveolar lavage (FOB + BAL) to evaluate for underlying structural anomalies, comorbidities, underlying genetic disorders and an infectious etiology [2,5,6,9,10].…”

Investigation of young children with severe recurrent wheeze: any clinical benefit?

“…Prescribing therapy targeted at the most likely cause of wheezing is therefore the most common approach in managing these patients, often resulting in therapeutic trials of asthma treatment, such as bronchodilators, inhaled corticosteroids and montelukast [6]. However, in a proportion of patients with recurrent wheeze, symptoms are refractory to conventional asthma therapies [7] and the safety and efficacy of more unusual treatments has not been tested [8], resulting in referral to a tertiary centre for further assessment. Furthermore, there have been very few attempts to delineate the underlying disease in this difficult group of patients.…”