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Table 2 Numbers required to power an augmentation trial

From: Health status decline in α-1 antitrypsin deficiency: a feasible outcome for disease modifying therapies?

Parameter

No. needed per arm to detect 25% reduction in decline

SGRQ total score

Whole group

8577

Rapid FEV1 decliners

5039

FEV1 (% predicted)

Whole group

1516

Rapid FEV1 decliners

86

Kco (% predicted)

Whole group

569

Rapid Kco decliners

77

  1. The number that is required in each arm with total SGRQ score or lung function as the outcome based on patients with a diagnosis of COPD or only those with known rapid decline in physiology as indicated
  2. FEV1 Forced Expiratory Volume in 1 Second, Kco Transfer Coefficient for carbon monoxide, SGRQ St George’s Respiratory Questionnaire