What are minimal important changes for asthma measures in a clinical trial?
In this study, the perceptions of asthmatics to change in their disease was associated with observed changes in clinical asthma measures, in order to identify the threshold where changes in clinical asthma measures are perceivable by patients. The study included 281 asthmatic patients, aged 18-63 yrs, in a randomized, placebo-controlled clinical trial of a leukotriene antagonist. Changes were related in: 1) asthma symptom scores; 2) inhaled beta-agonist use; 3) forced expiratory volume in one second (FEV1); and 4) peak expiratory flow (PEF) to a global question that queried overall change in asthma since starting the study drug. Additional analyses examined differences in the group reporting minimal improvement by treatment (active treatment versus placebo), sex and age groups. The average minimal patient perceivable improvement for each measure was: 1) -0.31 points for the symptom score on a scale of 0-6; 2) -0.81 puffs x day(-1) for inhaled beta-agonist use; 3) 0.23 L for FEV1; and 4) 18.79 L x min(-1) for PEF. In general placebo-treated patients and older patients, who reported minimal improvement, experienced less mean improvement from baseline than active-treated patients and younger patients, who reported minimal improvement. Determining the minimal patient perceivable improvement value for a measure may be helpful to interpret changes. However, interpretation should be carried out cautiously when reporting a single value as a clinically important change.
Santanello
N C
NC
Merck Research Laboratories, West Point, PA, USA.
Zhang
J
J
Seidenberg
B
B
Reiss
T F
TF
Barber
B L
BL
D000085:Acetates / Q000627:therapeutic use*; D000280:Administration, Inhalation; D000293:Adolescent; D000318:Adrenergic beta-Agonists / Q000008:administration & dosage; D000328:Adult; D000367:Age Factors; D000368:Aged; D001249:Asthma / Q000188:drug therapy / Q000503:physiopathology*; D002940:Circadian Rhythm; D003521:Cyclopropanes; D004311:Double-Blind Method; D015991:Epidemiologic Measurements; D005260:Female; D006801:Humans; D020024:Leukotriene Antagonists / Q000627:therapeutic use*; D008168:Lung / Q000187:drug effects / Q000503:physiopathology*; D008297:Male; D008875:Middle Aged; D011804:Quinolines / Q000627:therapeutic use*; D012129:Respiratory Function Tests; D012720:Severity of Illness Index; D012737:Sex Factors; D016037:Single-Blind Method; D013440:Sulfides
D000085:Acetates; D000318:Adrenergic beta-Agonists; D003521:Cyclopropanes; D020024:Leukotriene Antagonists; D011804:Quinolines; D013440:Sulfides; C093875:montelukast