Patient-Reported Outcomes

3. Reasons to Measure PROs

Exercise 2

For each surrogate in the following table, identify the patient-important outcomes (PIOs) with which there is a putative causal link. When you make a correct match a summary of results of randomized trials will appear. These results disappointed investigators by showing that their understanding of biology may be terribly flawed: compelling biological rationale failed to predict impact on patient-important outcomes, sometimes disastrously.

Drag the following outcomes into the PIO column below which you think was the trial result. A PIO may apply to more than one surrogate.

The outcomes are: QOL impairment, vertebral and longbone fractures; QOL impairment, coronary events; Death; Dyspnea and Fatigue; Renal Failure

The answers are in the last column of the table.

Intervention Surrogate Endpoint Patient-Important Outcomes (Answers)
Milrinone, amrinone, ibopamine, epoprotonol, vesnarinone for patients with heart failure Measures of cardiac function Death
Hormone replacement therapy for post-menopausal women High-Density Lipoprotein and ultrasound-measured vessel wall thickening QOL impairment, coronary events
Fluoride for women with osteoporosis Bone density QOL impairment, vertebral and longbone fractures
Encainide and flecainide for cardiac arrhythmias Arrhythmia suppression Death
Rosiglitazone for diabetes Serum glucose or Hemoglobin A1C Renal Failure
Torcetrapib for prevention of coronary events HDL QOL impairment, coronary events
Nitric oxide for critically ill patients with acute respiratory distress syndrome (ARDS) Oxygenation Death
Theophylline for asthma Spirometric measures of lung function Dyspnea and Fatigue