Work

Baseline-dependent improvement in CF studies, plausibility of bias

Year: 2024

Type: article

Abstract: Background: pp) (Magaret et al., 2024; Stanojevic et al., 2020 and Thornton et al., 2023). Methods: pp and treatment-induced change: post-versus pre-treatment. Results: pp, at levels equivalent to those reported in large studies measuring spirometry both at home and in clinic, naive analytic approaches found spurious associations of change with baseline (Paynter et al., 2022 and Saiman et al., 2003). Type I error ranged from 31.9% to 98.3% for day-to-day variation as high as 3% to 15% relative to biological variation. Incorporating known day-to-day variation, the regression calibration approach corrected bias and controlled type I error (Chambless and Davis, 2003). Conclusion: Exaggerated ceiling effects are possible. Further studies could provide meaningful confirmation of ceiling effects in CF, perhaps reducing day-to-day variation by incorporating multiple pre- and post-treatment measurements.

Source: Contemporary Clinical Trials Communications

DOI: 10.1016/j.conctc.2024.101378

Authors: Ellen Graham, Sonya L. Heltshe, Amalia S. Magaret

Institutions: University of Washington, Seattle Children's Research Institute

Language: English

Topic: Cystic Fibrosis Research Advances

Subfield: Pulmonary and Respiratory Medicine

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: gold

Estimated APC paid: $2,350