Concurrent validity and responsiveness of the Peabody Developmental Motor Scales-2 in infants and children with Pompe disease undergoing enzyme replacement therapy
Bibliographic record
Abstract
Purpose: To examine the responsiveness of the PDMS-2 in children diagnosed with Pompe disease who had different levels of functional mobility. To examine the concurrent validity between the PDMS-2 and the Alberta Infant Motor Scale (AIMS), the Pediatric Evaluation of Disability Inventory (PEDI) and the Pompe PEDI (PPEDI) in children diagnosed with Pompe disease. Methods: A secondary analysis was completed of the Genzyme Corporation Pompe efficacy trials for Myozyme. The children were divided into two functional groups, independent ambulators (group 1), and children who required use of assistive devices to ambulate or were unable to ambulate (group 2). Results: A significant difference was present between the mean PDMS-2 subtest and Gross Motor Composite percentage scores at baseline and at week 52 when the whole group was combined. Responsiveness by the factor of Time and Function revealed Locomotion subtest statistically significant mean percentage change within both functional groups from baseline to week 52 and between groups at both time periods. No significant within group or between group differences were seen for the Stationary subtest. The Object Manipulation subtest demonstrated significant change within group one from baseline to week 52 and between the functional presentations at week 52.The age equivalent correlations between the PDMS-2 and AIMS indicate a good to excellent relationship for all subtests except the Object Manipulation subtest. A four-month range in age equivalent scores was necessary to achieve 100% agreement between the AIMS and the PDMS-2. Correlations between the PDMS-2, Locomotion and Stationary subtest and PPEDI for all age groups were in the moderate to good and good to excellent range. Non-significant correlations were found for the Reflex and Object Manipulation subtest. Conclusions: The PDMS-2 gross motor subtests were responsive to change in a heterogeneous group of children diagnosed with Pompe disease. Responsiveness concerns were identified in the Object Manipulation and Stationary subtest when the children were divided into two different functional groups. A stronger relationship was present between the PDMS-2 and AIMS age equivalent scores than the percentile scores. Motor capacity on the PDMS-2 reflects actual performance in the home and community environment as measured by the PPEDI.
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How this classification was reachedexpand
Full frame machine prediction
Teacher imitationNot calibrated prevalence, not ground truth. Human validation pending. The Gemma side is a direct model label for every work in the frame, read from the title-only record. The Codex side is a classifier learned from the 10,348 direct Codex labels and calibrated to design-weighted sample rates; fields without enough sample support carry no Codex call. Candidate is the union of the two sides; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels.
Distilled classifier scores by category (both heads)
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.002 | 0.014 |
| Meta-epidemiology (narrow) | 0.000 | 0.000 |
| Meta-epidemiology (broad) | 0.001 | 0.001 |
| Bibliometrics | 0.001 | 0.001 |
| Science and technology studies | 0.000 | 0.000 |
| Scholarly communication | 0.001 | 0.000 |
| Open science | 0.000 | 0.001 |
| Research integrity | 0.000 | 0.001 |
| Insufficient payload (model declined to judge) | 0.001 | 0.000 |
Machine scores (provisional)
The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.
Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.
score_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from itClassification
machine, unvalidatedMachine predicted; a candidate call from one source (direct Gemma or distilled Codex), not a consensus.
How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".