Calculating the number needed to treat for trials where the outcome is time to an event
Open accessAbstract
Although many patients with disseminated nontuberculous mycobacterial disease have molecular defects in the IFN-γ/IL-12 axis, recent case reports have shown autoantibodies against IFN-γ associated with severe nontuberculous mycobacterial infections. To check this finding in an independent population, we screened 35 patients with either disseminated or pulmonary nontuberculous mycobacterial infections for whom no molecular defect was known. We identified high-titer-neutralizing anti-IFN-γ IgG in the plasma of six patients. All six patients were female, parous, of East Asian descent, and had disseminated infection, predominantly with rapidly growing mycobacteria. The anti-IFN-γ IgG had in vitro biological activity on the IFN-γ-dependent phosphorylation of STAT-1 as well as on the IFN-γ-dependent up-regulation of TNF-α and IL-12. In contrast, this anti-IFN-γ Ab had no effect on IFN-α-dependent STAT-1 phosphorylation. These patients confirm a novel syndrome linking autoimmunity and immunodeficiency.
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The number of patients who would need to be treated to prevent one event of the primary outcome over a period of 24 months was 45 on the basis of Kaplan-Meier estimates.
“The number of patients who would need to be treated to prevent one event of the primary outcome over a period of 24 months was 45 on the basis of Kaplan-Meier estimates.8”
From Semaglutide and Cardiovascular Outcomes in Patients with Type 2 Diabetes (2016) · cites this paper as [8]
The “Only survival probabilities available” section explains how to calculate a time-specific number needed to treat from Kaplan–Meier estimates. It does not report an NNT of 45 at 24 months; the Discussion table gives 6.4 at two years for its lung-cancer example. Thus the source supports the method, but not the claimed result. (LLM, conf=0.88)
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- Authors
- Altman, D. G., & Andersen, P. K.
- Affiliations
- University of Oxford (GB)
- University of Copenhagen (DK)
- Corresponding author
- Altman, D. G
- Venue
- BMJ
- Publisher
- BMJ
- Published
- 1999-12-04
- Volume
- 319
- Issue
- 7223
- Pages
- 1492-1495
- ISSN
- 0007-1447 (ISSN-L)
- 0959-8138
- 1468-5833
- PMC
- PMC1117211
- OpenAlex
- W2033209427
- Cited by
- 710
- References
- 2
- Citations by year
- 2026: 20 · 2025: 28 · 2024: 40 · 2023: 47 · 2022: 50 · 2021: 34 · 2020: 53 · 2019: 47 · 2018: 40 · 2017: 32 · 2016: 32 · 2015: 29 · 2014: 30 · 2013: 31 · 2012: 23
- FWCI
- 7.26
- Type
- journal article
- Language
- en
- Open access
- green
- www.ncbi.nlm.nih.gov
- Locations
- BMJ (publishedVersion)
- Europe PMC (PubMed Central) (publishedVersion)
- PubMed Central (publishedVersion)
- PubMed (publishedVersion)
- Research at the University of Copenhagen (University of Copenhagen) (submittedVersion)
- Funders
- Javna Agencija za Raziskovalno Dejavnost RS
- Topics
- Physical Sciences › Mathematics › Statistics and Probability › Statistical Methods in Clinical Trials
- Health Sciences › Medicine › Epidemiology › Mycobacterium research and diagnosis
- Physical Sciences › Mathematics › Statistics and Probability › Statistical Methods in Epidemiology
- Keywords
- number needed to treat, time to event, survival analysis, statistical power, sample size determination, clinical trials
- MeSH terms
- Clinical Trials as Topic, Data Collection, Prognosis, Proportional Hazards Models, Survival Analysis, Sample Size
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