Question

Difficulty: MediumMeasures of Association and Impact

Investigators recently concluded a Phase III clinical study assessing a new monoclonal antibody against interleukin-5 (IL-5). The trial enrolled adults with severe, uncontrolled eosinophilic asthma to determine the drug's efficacy in preventing disease exacerbations. A total of 2,0002,000 participants were randomly assigned to receive either the IL-5 inhibitor or a matching placebo. The primary endpoint was the incidence of at least one severe asthma exacerbation requiring an emergency department visit or hospitalization over a 1212-month period.

Data analysis revealed the following:
- Among the 1,2001,200 participants who received the IL-5 inhibitor, 108108 experienced a severe exacerbation.
- Among the 800800 participants who received the placebo, 112112 experienced a severe exacerbation.

Based on these findings, calculate the number needed to treat (NNT) to prevent one additional severe asthma exacerbation. (Enter your answer as a whole number)

Answer: 20 patients

Answer

20
The Number Needed to Treat (NNT) is calculated as 1/Absolute Risk Reduction (ARR)1 / \text{Absolute Risk Reduction (ARR)}. First, calculate the event rates for both groups. The Control Event Rate (CER) is 112/800=0.14112 / 800 = 0.14 (14%14\%). The Experimental Event Rate (EER) is 108/1200=0.09108 / 1200 = 0.09 (9%9\%). The ARR is the difference between these two rates: 0.14−0.09=0.050.14 - 0.09 = 0.05 (5%5\%). Finally, the NNT is the inverse of the ARR: 1/0.05=201 / 0.05 = 20. This means that 2020 patients must be treated with the IL-5 inhibitor for one year to prevent one additional severe asthma exacerbation.

Step-by-Step Solution

1
Calculate the Control Event Rate (CER)
CER = 112 / 800 = 0.14
CER represents the baseline risk of an exacerbation in the placebo group.
2
Calculate the Experimental Event Rate (EER)
EER = 108 / 1200 = 0.09
EER represents the risk of an exacerbation in the treatment group.
3
Calculate the Absolute Risk Reduction (ARR)
ARR = 0.14 - 0.09 = 0.05
ARR measures the absolute difference in risk between the control and experimental groups.
4
Calculate the Number Needed to Treat (NNT)
NNT = 1 / 0.05 = 20
NNT is the inverse of the ARR, representing how many patients must receive the intervention to prevent one adverse event.

Key Concept

Number Needed to Treat (NNT) Calculation
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