M. Man-son-hing et al., Patient preference-based treatment thresholds and recommendations: A comparison of decision-analytic modeling with the probability-tradeoff technique, MED DECIS M, 20(4), 2000, pp. 394-403
Background. Decision analysis (DA) and the probability-tradeoff technique (
PTOT) are patient preference-based methods of determining optimal therapy f
or individuals. Using aspirin therapy for the primary prevention of stroke
and myocardial infarction (MI) in elderly persons as an example, the object
ive of this study was to determine whether group-level treatment thresholds
and individual-revel treatment recommendations derived using PTOT are iden
tical to those of DA incorporating the patients' own values. Methods. Perso
ns in a pilot study of the efficacy of aspirin in the prevention of stroke
and MI were asked to participate. Participant values and utilities for pert
inent health states (e.g., minor and major stroke, MI, major bleeding episo
de) were determined. Then, in three hypothetical clinical situations in whi
ch the chance of stroke or MI was varied, PTOT was used to directly determi
ne treatment thresholds for aspirin therapy (i.e., the smallest reduction i
n MI or stroke risk for which participants would be willing to take aspirin
). Using DA modeling, with the same probabilities of events as in the PTOT
exercise and incorporating participants' own values, treatment thresholds f
or the three clinical situations were determined. The thresholds determined
by the two approaches were compared. Finally, based on these treatment thr
esholds, using the best estimates of the efficacy of aspirin to prevent fir
st-time stroke and MI, PTOT and DA treatment recommendations for individual
participants were compared. Results. The 42 participants reported that a m
ajor stroke was the least desirable health state, followed by MI, minor str
oke, and major bleeding. The minimum risk reduction required to take aspiri
n was greater for MI prevention compared with stroke prevention. For the tw
o clinical situations in which the hypothetical efficacy of aspirin to prev
ent stroke was varied, treatment thresholds for the PTOT Versus DA approach
es differed (p < 0.04), but this difference was not significant (p = 0.19)
for the MI-based clinical situation. Using the best estimate of the efficac
y of aspirin to prevent first-time stroke and MI, PTOT and DA treatment rec
ommendations whether or not to take aspirin were discordant for 38% of part
icipants (16 of 42) (2 < 0.001). Conclusions. Patient preference-based grou
p-level treatment thresholds and individual-level treatment recommendations
can differ significantly depending on whether PTOT or DA is used, apparent
ly because the two emphasize different aspects of the decision-making proce
ss. DA theory assumes that effective therapeutic decision making should max
imize both quality and quantity of life; with PTOT, the emphasis for effect
ive clinical decision making allows patients to be fully engaged in the pro
cess, thus hopefully leading to fully informed decisions that may result in
satisfaction and compliance.