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Table 1 List of outcome measures

From: Efficacy of omalizumab in children, adolescents, and adults with severe allergic asthma: a systematic review, meta-analysis, and call for new trials using current guidelines for assessment of severe asthma

Outcome measures

Importance

Measure unit

Minimal clinically important difference

Mortality

Criticala

  

Exacerbation rate

Critical

1. Average reduction in the annual number of exacerbations

1. 25% (a minimum reduction of 0.5 exacerbations per year)

2. Number of patients who experience 0 exacerbations annually

2. 10 percentage points

Oral corticosteroid-maintenance treatment

Critical

1. Average %-reduction in daily dose (maintenance-treatment)

1. 20% (at least 2.5 mg prednisolone equivalent dose)

2. Percentage of patients who are discontinued oral corticosteroid-maintenance treatment

2. 5 percentage points

3. Percentage of patients who experience ≥ 50% reduction of oral corticosteroid treatment

10 percentage pointsb

Lungfunction FEV1

Important

1. Average change in lung function

1. 200 ml

2. Percentage of patients who experience an improvement of 200 ml or more

2. 15 percentage points

Asthma control

Important

Average change in asthma control. A prioritised list of scores

   ACQ 5 (Asthma Control Questionnaire)

   ACT (Asthma Control Test)

   Other similar questionnaires

ACQ: 0.5

ACT: 3

Quality of life (QoL)

Important

Average change in QoL. A prioritised list of scores

   Asthma Quality of Life Questionnaire (AQLQ)

   Other questionnaires

AQLQ: 0.5

Serious adverse events (SAEs)

Important

The added number of SAEs

5 percentage points for the added number of SAEs

Specific subgroups of SAEs, including anaphylaxis is assessed if they are distributed uniformly between the groups

No minimal clinically important difference is reported

Drop-out rate

Important

The percentage of patients who dropped out when the study was completed (difference between intention to treat population and difference between “intention to treat”-population and patients who completed the study)

10 percentage points

Sick leave

Important

Average number of sick leave days per year

5 days/year

  1. For each outcome measure, the importance is indicated, and for critical and important outcome measures the minimal clinically important difference is reported
  2. aMortality is always considered to be a critical effect goal, albeit not an effective efficacy measure in the assessment of biological drugs in severe asthma. Asthma-related death occurs rarely, and it is therefore not estimated that outcome measure will provide any relevant information. In relation to safety, it is included in outcome measure: serious adverse events (SAEs). Mortality will therefore not act as a separate outcome measure in the assessment of the therapy
  3. bThe Expert Committee defined this outcome measure after the protocol was approved as data could not be extracted for the average OCS reduction
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Allergy, Asthma & Clinical Immunology

ISSN: 1710-1492

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