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Here is a really interesting study of what study would be required to
resolve differences in opinion about the management of ITP.

http://mdm.sagepub.com/content/31/6/805.abstract?etoc

(I can't get past the paywall, so have only read the abstract)


Michael

Dr Michael Power

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 Abstract

*Purpose.* To identify core disagreements between pediatric hematologists
who would treat children with idiopathic thrombocytopenic purpura (ITP) on
initial presentation (“Treaters”) and those who would treat by observation
(“Observers”), to determine whether each group’s preferred stance was
consistent with each individual’s detailed perceptions, and to identify key
variables in each stance. **

*Methods.* A decision model was constructed in collaboration with experts,
and a detailed questionnaire was presented to a nationally representative
committee of 25 pediatric hematologists. A full decision tree was specified
for each respondent.

*Results.* Nineteen (76%) experts responded; based on preference for
initial treatment, 9 were Treaters and 10 Observers. Of the 30
probability/effectiveness variables, 8—almost all concerning treatment
effectiveness—had at least one statistically-significant difference between
the 2 groups regarding low, best, or high estimates. To convince Observers
that treatment is effective would take a clinical trial with between 39 000
and 87 000 participants; to convince Treaters that treatment is not
effective enough, between 97 000 and 114 000 participants. Observers’
calculated numbers needed to treat (NNTs) of about 150 000 are more
consistent (*P* = 0.0023) with their elicited maximum NTTs of about 500.

*Conclusion.* Physicians not specifically trained provided enough data to
specify complete individual decision models. From the estimates provided,
no practical clinical trial could convince hematologists who would treat
children on initial presentation with ITP just to simply observe them or
could convince those who would just observe to instead treat with available
agents. Perceived burdens could be better characterized, perhaps by
including parental perceptions and preferences.