Rasch-built Pompe-specific activity scale

Instrument Name: Rasch-built Pompe-specific activity scale

Abbreviation: R-PAct

Points for Consideration:

Little psychometric data available but may be important as a disease-specific measure

Description of Tool:

The Rasch-built Pompe-specific activity scale is a PRO for adolescents aged 16 years and older which includes 18 tasks related to activities of daily living, ranging from combing hair to turn around in bed, to walk at a rapid speed, and run. Each task is rated as "0, unable to perform," "(1) able to perform, but with difficulty," and "(2) able to peforme without difficulty". Lower scores represent more disability.

Minimum Qualification Required by COA Administrator: No degree requirement

Year: 2013

Objective of Development:

To quantify the effects of Pompe disease on patient’s ability

Population of Development: Age range (therapeutic indication):

16+ years (Glycogen Storage Disease Type II)

Pediatric Population(s) in which COA has been used:

Nervous System Diseases; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Nutritional and Metabolic Diseases

COA type:

Number of Items 18

Mode of Administration:

Data Collection Mode:

Time for Completion: Not reported

Response Scales: 3-point verbal rating scale ranging from 0 "Unable to perform" to 2 "Able to perform without difficulty"

Summary of Scoring:

Available Scores:
Global score is a summed raw scored transformed to a linear 0-100 score

Weighting:
No

Score Interpretation:
Lower score = More disability

Evidence of Literature Review: None identified

Evidence of Instrument Review: Yes

Evidence of Clinical or Expert Input: Yes

Evidence of concept elicitation in target patient population: None identified

Evidence of a Saturation Grid: None identified

Evidence for Selection of Data Collection Method: None identified

Recall/Observation Period:

Evidence for Selection of Reponse Options: Yes

Evidence of cognitive interviewing of draft instrument in target patient population: Yes

Evidence of Preliminary Scoring of Items and Domains: None identified

Evidence related to respondent and administrator burden: None identified

Evidence of a Conceptual Framework: None identified

Evidence of an item-tracking matrix: None identified

Evidence related to item selection: Yes

Evidence of re-testing the final version: None identified

Internal consistency (Cronbach's alpha): None identified

Test-retest Reliability (ICC):

van der Beek (2013)
- Intraclass Correlation Coefficient (ICC): 0.95 (for patients' location)
- Was a definition of stability applied to identify stable patients: No
- Time frame or interval between the two administrations: 2-4 weeks
- Population/Disease: Patients with Pompe disease; n=44 age: 16+ years

Inter-rater/ inter-interviewer reliability (kappa):

Not applicable

Evidence of test-retest or inter-rater reliability: Yes

Known-group validity:

van der Beek (2013)
1. Measure/Groups of patients: Manual muscle weakness testing using the Medical Research Council (MRC)
- A priori hypotheses: Not stated
- Were hypotheses confirmed: Not applicable
- Results: ICC
The R-PAct summed raw score was strongly correlated to the MRC sum score: 0.82 (p Not stated)
- Population/Disease: Patients with Pompe disease; n=186 age: 16+ years

KNown-groups validity:
2. Measure/Groups of patients: Mobility groups: Ambulant without walking devices: n=69; ambulant with walking devices: n=30; partial or permanent wheelchair use: n=87
A priori hypotheses: Not stated
Were hypotheses confirmed: Not applicable
Results: ICC
The R-PAct summed raw score was significantly higher:
in patients ambulant without walking devices compared to other groups (all p<0.01) in patients ambulant with walking devices compared to patients with partial use (p=0.03) and permanent use (p<0.01) of wheelchair in patients with partial use of wheelchair compared to those with permanent use of wheelchair (p<0.01). - Population/Disease: Patients with Pompe disease; n=186, age 16+ years 3. Measure/Groups of patients: Use of ventilatory support No ventilation required : n=102 ventilation required : n=84 - A priori hypotheses: Not stated - Were hypotheses confirmed: Not applicable - Results: ICC The R-PAct summed raw score was significantly lower in patients who needed ventilation: p<0.01 - Population/Disease: Patients with Pompe disease; n=186, age 16+ years

Evidence of Translatability Assessment: None identified

Evidence related to missing data: None identified

Evidence for Selection of Recall Period: None identified

Evidence of Administration Instructions and Training Provided: None identified

Evidence of concurrent validity: None identified

Evidence of known-groups validity: Yes

Evidence of ability to detect change over time: None identified

Evidence of responder thresholds: None identified

van der Beek NA, Hagemans ML, van der Ploeg AT, van Doorn PA, Merkies IS. The Rasch-built Pompe-specific activity (R-PAct) scale. Neuromuscul Disord. 2013 Mar;23(3):256-64
Full text article: https://www.nmd-journal.com/action/showPdf?pii=S0960-8966(12)00669-4

See (PubMed results)
https://pubmed.ncbi.nlm.nih.gov/?term=(r-pact%5BTitle%2FAbstract%5D)+AND+((%222012%22%5BDate+-+Publication%5D+%3A+%223000%22%5BDate+-+Publication%5D))&sort=

None identified

Original language:
Dutch for the Netherlands

Translations:
English

None identified

Author:
Dr. N.A.M.E. (Nadine) van der Beek
Erasmus MC - Erasmus Medisch Centrum's
Gravendijkwal 230
3015 CE Rotterdam
Netherlands
E-mail: n.beek@erasmusmc.nl

Not reported

Not reported

List of items available in van der Beek et al, 2013 (Figure 2) (https://www.nmd-journal.com/action/showPdf?pii=S0960-8966(12)00669-4)