Measuring Disease Activity in Adults With SLE
Measuring Disease Activity in Adults With SLE
The judgment of whether a patient with SLE is better or worse is a central question in patient management. After the failure of several clinical trials of biologic therapy in SLE, the management of SLE today remains an art rather than a science. The objective is to improve disease state or at least fend off its deterioration, be accurate in defining disease activity and flare state, and employ evidence-based and clinically meaningful response criteria measured with valid and reproducible instruments that are sensitive to change and responsive to a patient's concerns.
Pitfalls in lupus disease activity measures have had a significant impact on the interpretation of study outcomes. Many of the clinical trials were overpowered or underpowered and had complex and multiple outcome measures. Devised in the 1990s, these metrics were never intended for use in clinical trials. This underlines the importance for improving these instruments and optimizing on the composite indices for ascertainment of disease activity. Nonetheless, in the absence of a biomarker-based gold standard against which to gauge improvement or flare, selection of SLE disease endpoints should be defined and tailored to the outcome of interest. Moreover, the exact choice of disease activity-measuring instruments should be governed by the purpose for which they are required in clinical research. They should be simple, reliable, and valid with reduced administrative burden, which may integrate elements for enhanced responsiveness to patient concerns by using the platform by the published FDA guidance document. It is essential to make certain that disease activity measures are being applied consistently and uniformly through proper and simple training, given the potential complexity of patients with different SLE manifestations.
Conclusions
The judgment of whether a patient with SLE is better or worse is a central question in patient management. After the failure of several clinical trials of biologic therapy in SLE, the management of SLE today remains an art rather than a science. The objective is to improve disease state or at least fend off its deterioration, be accurate in defining disease activity and flare state, and employ evidence-based and clinically meaningful response criteria measured with valid and reproducible instruments that are sensitive to change and responsive to a patient's concerns.
Pitfalls in lupus disease activity measures have had a significant impact on the interpretation of study outcomes. Many of the clinical trials were overpowered or underpowered and had complex and multiple outcome measures. Devised in the 1990s, these metrics were never intended for use in clinical trials. This underlines the importance for improving these instruments and optimizing on the composite indices for ascertainment of disease activity. Nonetheless, in the absence of a biomarker-based gold standard against which to gauge improvement or flare, selection of SLE disease endpoints should be defined and tailored to the outcome of interest. Moreover, the exact choice of disease activity-measuring instruments should be governed by the purpose for which they are required in clinical research. They should be simple, reliable, and valid with reduced administrative burden, which may integrate elements for enhanced responsiveness to patient concerns by using the platform by the published FDA guidance document. It is essential to make certain that disease activity measures are being applied consistently and uniformly through proper and simple training, given the potential complexity of patients with different SLE manifestations.
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