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Table 1 Study endpoints

From: The Salford Lung Study protocol: a pragmatic, randomised phase III real-world effectiveness trial in asthma

Endpoint Definitions
Primary endpoint The primary efficacy analysis population is defined as all ITT patients who have an ACT total score of <20 at baseline
The percentage of patients who have either an ACT total score of ≥20 or an increase from baseline of ≥3 at week 24 (6th month) assessment
Secondary efficacy endpoints • All contacts are any encounter the patient may have with a doctor or nurse or other healthcare professionals working as part of the NHS (including telephone calls). Contacts with the NHS or hospitalisation are defined as exacerbation-related contacts if these contacts were a direct result of an acute worsening of asthma symptoms. Contacts are defined to be asthma-related as per GP/investigator-defined normal clinical practice. These contacts do not include protocol-defined study-related visits/contacts
• A prescription of systemic corticosteroid or antibiotics is defined as exacerbation-related if the reason the drug was given, in whole or in part, was to treat an acute worsening of asthma symptoms
• A severe asthma exacerbation is defined as deterioration of asthma requiring the use of systemic corticosteroids (tablets, suspension or injection) or antibiotics, an inpatient hospitalisation, or emergency department visit due to asthma that required systemic corticosteroids or antibiotics. Exacerbation-related hospitalisation includes hospitalisation that is prolonged as a result of an asthma exacerbation
• Percentage of patients who have either an ACT total score of ≥20 or an increase from baseline of ≥3 in ACT total score at weeks 12, 40 and 52
• Percentage of patients with ACT total score ≥20 at weeks 12, 24, 40 and 52
• Mean change from baseline in ACT total score at weeks 12, 24, 40 and 52
• All/asthma-related primary or secondary care contacts
• Mean annual rate of severe asthma exacerbations
• Number of salbutamol inhalers (adjusted to equivalence of 200 actuations) dispensed from study-enrolled community pharmacies over the entire treatment period
• Time to discontinuation or modification of initial therapy (i.e. therapy the patient is randomised to at visit 2)
• Percentage of patients who have an increase from baseline of ≥0.5 in AQLQ(s) total score at week 52
• Percentage of patients who have an increase from baseline of ≥0.5 in AQLQ(s) environmental stimuli domain score at week 52
Safety endpoints • An ADR is any untoward medical occurrence in a patient temporally associated with the use of a medicinal product, for which there is a reasonable possibility that the untoward occurrence is causally related to the medicinal product
• An SAE is any untoward medical occurrence that, at any dose: results in death; is life-threatening; requires hospitalisation or prolongation of existing hospitalisation; results in disability/incapacity; is a congenital anomaly/birth defect; subject to medical or scientific judgement, may jeopardise the patient or may require medical or surgical intervention to prevent one of the other outcomes listed
• Incidence of SAEs of pneumonia during the study
• Time to first SAE of pneumonia
• Deaths due to serious events of pneumonia
• Frequency and type of SAEs
• Frequency and type of ADRs
Other efficacy endpoints • Increase from baseline of ≥0.5 in AQLQ(s) refers to: total score at week 24; individual domain scores at week 24; individual domain scores (symptoms, activity, limitations and emotional function) at week 52
• WPAI: asthma at week 52 refers to the following categories: percentage of work time missed due to asthma: percentage of impairment while working due to asthma; percentage of overall work impairment due to asthma; percentage of activity impairment due to asthma
• Mean change from baseline in individual question scores for ACT at weeks 12, 24, 40 and 52
• Mean change from baseline in total score and domain scores of AQLQ(s) at weeks 24 and 52
• Percentage of patients who have an increase from baseline of ≥0.5 in AQLQ(s)
• WPAI: asthma at week 52
• Health status using the EQ-5D at week 52
• Adherence with study medication based on analysis of medications (prescribed, dispensed and collected) during the study
• Use of the MARS-A at week 52
  1. ACT Asthma Control Test, ADR adverse drug reaction, AQLQ Asthma Quality of Life Questionnaire, EQ-5D EuroQol questionnaire, GP general practitioner, ITT intent-to-treat, MARS-A Medication Adherence Report Scale for Asthma, NHS National Health Service, SAE serious adverse event, WPAI Work Productivity and Activity Impairment Questionnaire