Modified Performance-Oriented Mobility Assessment-Gait

Instrument Name: Modified Performance-Oriented Mobility Assessment-Gait

Abbreviation: MPOMA-G

Points for Consideration:

Has been used in more recent FDA approvals

Description of Tool:

The Modified Performance-Oriented Mobility Assessment-Gait (MPOMA-G) is a ClinRO developed to detect clinically significant impairments in gait in videos of children aged 5-15 years with hypophosphatasia. It is composed of 8 items assessing gait.

Minimum Qualification Required by COA Administrator: MA or BA

Comment:

The m-POMA-G was based on the POMA gait observations. These were initially developed from the Fall Risk Index.

Year: 2018

Objective of Development:

To detect clinically significant impairments in gait in videos of children with hypophosphatasia

Population of Development: Age range (therapeutic indication):

5-15 years (Hypophosphatasia)

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 8

Mode of Administration:

Data Collection Mode:

Time for Completion: Not reported

Response Scales: Categorical rating scale. Depending on the item either 2 or 3 behavioral definitions.

Summary of Scoring:

Available scores: Global score ranging from 0 to 12

Weighting: No

Score Interpretation: Higher score = Better gait performance

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: None identified

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: None identiifed

Evidence of re-testing the final version: None identified

Internal consistency (Cronbach's alpha): None identified

Test-retest Reliability (ICC):

Not applicable

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

Phillips (2018)
Intra-rater reliability
- Intraclass Correlation Coefficient (ICC): 0.76 (p< 0.001) - Was a definition of stability applied to identify stable patients: No - Time frame or interval between the two administrations: 8 months - Population/Disease: Children and adolescents 5-15 years old with hypophosphatasia; n= 14

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

Concurrent validity (convergent, divergent):

Phillips (2018)
- Correlation coefficient used: Pearson's correlation coefficient
- Measure: Pediatric Outcomes Data Collection Instrument (PODCI) Sports/Physical Functioning and Transfer/Basic Mobility domains; Childhood Health Assessment Questionnaire-Disability Index (CHAQ-DI)
- Results:
- There were significant correlations between mPOMA-G scores and PODCI Sports/Physical Functioning domain scores in both, infantile HPP (r= 0.78) and childhood HPP (r= 0.65); p< 0.0001 for all results - There were significant correlations between mPOMA-G scores and PODCI Transfer/Basic Mobility domain scores in both, infantile HPP (r= 0.72; p= 0.00002) and childhood HPP (r= 0.57; p= 0.0001) - There were significant correlations between mPOMA-G scores and CHAQ-DI scores in both, infantile HPP (r= 0.86) and childhood HPP (r= 0.72); p< 0.0001 for all results - Population/Disease: Children with hypophosphatasia (age range 5-15 years); Infantile HPP: n= 5 (21 observations); Childhood HPP: n= 8 (39 observations)

Known-group validity:

Phillips (2018)
- Measure/Groups of patients: Distance walked as measured by the 6-Minute Walk Test
- A priori hypotheses: Not stated
- Were hypotheses confirmed: Not applicable
- Results: Pearson correlation coefficient. There were significant correlations between mPOMA-G scores and distance walked in both, infantile HPP (r= 0.83) and childhood HPP (r= 0.70); p< 0.0001 for all results - Population/Disease: Children with hypophosphatasia (age range 5-15 years); Infantile HPP: n= 5 (28 observations); Childhood HPP: n= 8 (51 observations)

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: Yes

Evidence of concurrent validity: Yes

Evidence of known-groups validity: Yes

Evidence of ability to detect change over time: None identified

Ability to detect change (Responsiveness):

None identified

Evidence of responder thresholds: None identified

Phillips D, Griffin D, Przybylski T, Morrison E, Reeves AL, Vallee M, Fujita KP, Madson KL. Development and validation of a modified performance-oriented mobility assessment tool for assessing mobility in children with hypophosphatasia. J Pediatr Rehabil Med. 2018;11(3):187-192 (Full text article: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6294582/pdf/prm-11-prm170523.pdf)

Grieco JC, Romero B, Flood E, Cabo R, Visootsak J. A Conceptual Model of Angelman Syndrome and Review of Relevant Clinical Outcomes Assessments (COAs). Patient. 2019 Feb;12(1):97-112
Full text article: https://www.ncbi.nlm.nih.gov/pmc/articles/pmid/29987743/

See PubMed results: https://pubmed.ncbi.nlm.nih.gov/?term=%22mPOMA-G%22&sort=date)

Yes

Strensiq, Asfotase Alfa (FDA, 2015)
Results:
Juvenile-Onset HPP
Skeletal Manifestations: All 8 treated patients were rated as responders by Month 54 of treatment. The mean duration between the baseline and last RGI-C assessments for control patients was 56 months (range was 8 to 95 months). At last assessment, 2/32 (6%) of control patients were rated as responders.
Eight of 20 (40%) patients with juvenile-onset HPP experienced new fractures during the course of treatment. There were insufficient data to assess the effect of STRENSIQ on fractures.
Gait/Mobility: Step length improved by at least 1 point in either foot in 6/8 patients compared to 1/6 (17%) control patients. The proportion of patients who had 6MWT percent predicted values within the Normal range for age, sex, and height-matched peers increased from 0/8 patients at baseline to 6/6 patients (100%) by Month 48 and all 6 were also able to walk longer distances at this time point compared to baseline.
Long-Term Extension Trials in Juvenile-Onset HPP
At last assessment, 7 patients with available 6MWT results had maintained improvements in gait/mobility

Original: English for the USA

Translations:
None identified

N/A

Author and Contact:
Dawn Phillips
Evidera Inc.
Bethesda
MD
USA;
UNC Division of Physical Therapy
Chapel Hill
NC
USA
E-mail: DawnPhillipspt@gmail.com

Copyright: According to the author, Dawn Philipps, I believe the copyright would belong to Alexion with a patent that only limits application to Hypophosphatasia.

CoU:
*Free access according to Dawn Philipps
* for further information, please contact directly Dr. Dawn Phillips

Not reported

Available in Table 1 in Phillips D, 2018