Soru

Zorluk: OrtaMeasures of Association and Impact

To evaluate the efficacy of a novel immunomodulator in preventing clinical relapses among patients with relapsing-remitting multiple sclerosis, researchers conduct a phase III, placebo-controlled, double-blind trial. A total of 2,0002,000 enrolled patients are randomized in a 1:1 ratio to receive either the novel immunomodulator or a placebo. After two years of continuous follow-up, 140140 patients in the placebo arm and 9595 patients in the intervention arm experienced at least one clinical relapse. In their primary analysis, the investigators report the absolute risk reduction alongside a 95% confidence interval of 0.0210.021 to 0.0690.069.

Determine the number of patients that must be treated with the novel immunomodulator to prevent one additional clinical relapse, and appropriately characterize the statistical significance based on the reported absolute risk reduction confidence interval.

  1. 23 patients; the finding is statistically significant because the confidence interval for the risk difference excludes 0.Cevap
  2. B
    22 patients; the finding is statistically significant because the confidence interval for the risk difference excludes 0.
  3. C
    23 patients; the finding is statistically significant because the confidence interval for the risk difference excludes 1.
  4. D
    22 patients; the finding is statistically significant because the confidence interval for the risk difference excludes 1.
  5. E
    4 patients; the finding is statistically significant because the confidence interval for the risk difference excludes 0.

Cevap

23 patients; the finding is statistically significant because the confidence interval for the risk difference excludes 0.
The correct answer accurately calculates the NNT as 23 and correctly identifies 0 as the null value for an absolute risk difference. First, the ARR is determined by finding the difference in event rates: 140/1000140/1000 (14%14\%) in the placebo group minus 95/100095/1000 (9.5%9.5\%) in the intervention group, yielding an ARR of 4.5%4.5\% (0.0450.045). The NNT is the inverse of the ARR (1/0.045=22.221 / 0.045 = 22.22). In clinical biostatistics, NNT must always be rounded up to the next highest whole number to guarantee that the desired clinical benefit (preventing one relapse) is achieved, making the correct NNT 23. Furthermore, because ARR is an absolute difference, the null value indicating no effect is 0. The reported 95% CI of 0.0210.021 to 0.0690.069 does not cross 0, indicating a statistically significant reduction in risk.

Adım Adım Çözüm

1
Calculate the Control Event Rate (CER) and Experimental Event Rate (EER).
CER = 140/1000=0.14140 / 1000 = 0.14 (14%14\%), EER = 95/1000=0.09595 / 1000 = 0.095 (9.5%9.5\%).
Incidence rates for both groups must be derived from the raw patient data before calculating comparative measures.
2
Calculate the Absolute Risk Reduction (ARR).
ARR = CER - EER = 0.14−0.095=0.0450.14 - 0.095 = 0.045 (4.5%4.5\%).
ARR represents the absolute difference in the risk of the primary outcome between the placebo and intervention groups.
3
Calculate the Number Needed to Treat (NNT).
NNT = 1/ARR=1/0.045=22.221 / \text{ARR} = 1 / 0.045 = 22.22. Rounding up yields 2323.
NNT is the inverse of the ARR. It must always be rounded up to the next whole number to guarantee the prevention of one additional adverse event.
4
Interpret the statistical significance of the confidence interval.
The 95% CI (0.0210.021 to 0.0690.069) excludes 00.
For an absolute risk difference (such as ARR), the null value indicating no treatment effect is 0. Because the entire interval sits above 0, the finding is statistically significant.

Anahtar Kavram

Calculating Number Needed to Treat (NNT) and Interpreting Confidence Intervals for Absolute Risk
Bu soruyu puanla