Question

Difficulty: MediumMeasures of Disease Frequency and Association

A multicenter research group conducts a randomized controlled trial to evaluate the cardiovascular benefits of a novel medication in adults with a history of severe hypertriglyceridemia. A total of 5,0005,000 eligible participants are enrolled and assigned in a 1:11:1 ratio to receive either the active medication or a visually identical placebo. Participants are followed closely for 55 years, and the primary endpoint is the occurrence of a major adverse cardiovascular event (MACE). At the conclusion of the trial, the researchers compile the following data:

GroupTotal ParticipantsDeveloped MACE
Active Medication2,5002,500150150
Placebo2,5002,500250250

Based on the provided data, what is the number of patients who need to be treated with the active medication for 55 years to prevent one additional major adverse cardiovascular event?

  1. A
    44
  2. B
    1010
  3. C
    1717
  4. 2525Answer
  5. E
    5050

Answer

The correct value is 2525.
The Number Needed to Treat (NNT) is the number of patients who must receive a specific intervention to prevent one additional adverse outcome. It is calculated by taking the reciprocal of the Absolute Risk Reduction (ARR). First, calculate the event rate in the control group: 250/2,500=0.10250 / 2,500 = 0.10 (10%10\%). Next, calculate the event rate in the treatment group: 150/2,500=0.06150 / 2,500 = 0.06 (6%6\%). The ARR is the difference between these two rates: 0.10−0.06=0.040.10 - 0.06 = 0.04. The inverse of 0.040.04 (1/0.041 / 0.04) is 2525. Therefore, 2525 patients must be treated to prevent one MACE.

Step-by-Step Solution

1
Determine the Control Event Rate (CER) in the placebo group.
CER=2502500=0.10CER = \frac{250}{2500} = 0.10 (10%10\%).
This establishes the baseline risk of the outcome without the intervention.
2
Determine the Experimental Event Rate (EER) in the active medication group.
EER=1502500=0.06EER = \frac{150}{2500} = 0.06 (6%6\%).
This establishes the risk of the outcome when receiving the novel medication.
3
Calculate the Absolute Risk Reduction (ARR).
ARR=CER−EER=0.10−0.06=0.04ARR = CER - EER = 0.10 - 0.06 = 0.04 (4%4\%).
ARR measures the absolute difference in risk attributable to the intervention.
4
Calculate the Number Needed to Treat (NNT).
NNT=1ARR=10.04=25NNT = \frac{1}{ARR} = \frac{1}{0.04} = 25.
NNT represents the number of patients that must receive the treatment to prevent one adverse event.

Key Concept

Number Needed to Treat (NNT)
Estimated Time:1m 15s
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