The following is a summary of “Cultivating Patient Preferences in ALS Clinical Trials: Reliability and Prognostic Value of the Patient-Ranked Order of Function,” published in the June 2024 issue of Neurology by Eijk, et al.
The Patient-Ranked Order of Function (PROOF) is a new method for incorporating patient preferences into amyotrophic lateral sclerosis (ALS) treatment evaluation.
Researchers conducted a retrospective study to assess the reliability and prognostic value of different sets of patient-reported preferences for the PROOF endpoint.
They collected data via online surveys over 12 months using the Netherlands’ population-based registry. Patients scored functional domains of the ALS Functional Rating Scale (ALSFRS-R) and ranked each domain’s importance. Two weeks later, the questionnaire was repeated to assess test-retest reliability. Vital status was obtained from the municipal population register.
The results showed that 611 patients with ALS were monitored for survival, and 382 participated in the test-retest reliability study. All PROOF versions, with various preference sets, demonstrated excellent reliability (intraclass correlation coefficients from 0.89 [95% CI 0.87–0.91] to 0.97 [95% CI 0.97–0.98], all P<0.001), with no systematic differences between baseline and week 2 (mean rank difference range −1 to −3 [95% CI range −8 to 2], all P>0.20). Preferences on future events were more variable than those on current symptoms. All PROOF versions strongly predicted overall survival (HRs per 10th rank percentile from 0.80 to 0.83 [95% CI range 0.76–0.87], all P<0.001) and showed better survival curve separation among rank-stratified subgroups than the ALSFRS-R total score.
Investigators concluded that incorporating patient-reported preferences alongside ALSFRS-R measurement in a large cohort proved feasible without bias, suggesting the preferences offer valuable prognostic information beyond conventional measures.
Source: neurology.org/doi/10.1212/WNL.0000000000209502
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