Dataset Information

Evaluating longitudinal therapy effects via the North Star Ambulatory Assessment.

ABSTRACT:

Introduction/aims

In comparative studies, treatment effects are typically evaluated at a specific time point. When data are collected periodically, an alternative, clinically meaningful approach could be used to assess the totality of treatment effects. We applied a well-developed analytical procedure for evaluating longitudinal treatment effects using North Star Ambulatory Assessment (NSAA) data for illustration.

Methods

The NSAA comprises 17 scorable items/outcomes that measure changes in motor function. Using NSAA data from the published ataluren phase 3, randomized, placebo-controlled trial (NCT01826487), cumulative counts of failures to perform each item (transition from 2/1 [able/impaired] to 0 [unable]) were collected at specified time points for each patient over 48 wk. Treatment group-wise mean cumulative item failure count curves were constructed, comparing ataluren versus placebo and deflazacort versus prednisone/prednisolone among placebo-treated patients. The steeper the curve, the worse the outcome. A clinically meaningful summary of the between-group difference was provided for each comparison.

Results

The curve was uniformly steeper for placebo than ataluren after 16 wk and for prednisone/prednisolone than deflazacort after 8 wk. The two curves in each comparison continued to diverge thereafter, indicating sustained treatment benefits over time. Using a unique analytical approach, cumulative failure rates were reduced, on average, by 27% for ataluren versus placebo (rate ratio, 0.73; 95% confidence interval [CI], 0.55-0.97; p = .027) and 28% for deflazacort versus prednisone/prednisolone (rate ratio, 0.72; 95% CI, 0.53-0.96; p = .028).

Discussion

Unlike fixed-time analyses, this analytical approach enabled demonstration of cumulative, longitudinal treatment effects over time using repeatedly measured NSAA observations.

SUBMITTER: McDonald CM

PROVIDER: S-EPMC9290940 | biostudies-literature | 2021 Nov

REPOSITORIES: biostudies-literature

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Publications

Evaluating longitudinal therapy effects via the North Star Ambulatory Assessment.

McDonald Craig M CM Wei Lee-Jen LJ Flanigan Kevin M KM Elfring Gary G Trifillis Panayiota P Muntoni Francesco F

Muscle & nerve 20210902 5

<h4>Introduction/aims</h4>In comparative studies, treatment effects are typically evaluated at a specific time point. When data are collected periodically, an alternative, clinically meaningful approach could be used to assess the totality of treatment effects. We applied a well-developed analytical procedure for evaluating longitudinal treatment effects using North Star Ambulatory Assessment (NSAA) data for illustration.<h4>Methods</h4>The NSAA comprises 17 scorable items/outcomes that measure ...[more]

PMID: 34383312

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Dataset Information

Evaluating longitudinal therapy effects via the North Star Ambulatory Assessment.

Introduction/aims

Methods

Results

Discussion

Publications

Evaluating longitudinal therapy effects via the North Star Ambulatory Assessment.

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