physical function-short form 20a  (ProMIS Neurosciences)

Journal: Journal of Patient-Reported Outcomes

Article Title: Switching treatment to cipaglucosidase alfa plus miglustat positively affects patient-reported outcome measures in patients with late-onset Pompe disease

doi: 10.1186/s41687-024-00805-w

Figure Lengend Snippet: Patient-level responder analysis ( A ) and group-level analysis ( B ) of PROMIS physical and fatigue domains and R-PAct outcomes in ERT-experienced patients. Patients with a change from baseline >0 in PROMIS Physical Function or R-PAct, or <0 for PROMIS Fatigue, were classed as ‘responders.’ *For the Fatigue endpoint, a negative change from the baseline value indicated a better health outcome. alg alglucosidase alfa, CFBL change from baseline, CI confidence interval, cipa cipaglucosidase alfa, ERT enzyme replacement therapy, LS least squares, mig miglustat, pbo placebo, PROMIS Patient-Reported Outcome Measurement Information System, R-Pact Rasch-built Pompe-specific Activity

Article Snippet: PROs evaluated included the Subject’s Global Impression of Change (SGIC), Patient-Reported Outcomes Measurement Information System (PROMIS) Physical Function Short Form 20a, PROMIS Fatigue Short Form 8a, Rasch-built Pompe-specific Activity (R-PAct), and European Quality of Life-5 Dimensions 5 Response Levels (EQ-5D-5L).

Techniques: Activity Assay

Journal: Orphanet Journal of Rare Diseases

Article Title: Measuring health related quality of life (HRQoL) in Lysosomal Storage Disorders (LSDs): a rapid scoping review of available tools and domains

doi: 10.1186/s13023-024-03256-0

Figure Lengend Snippet: PROMs included in review

Article Snippet: PROMIS - Physical Function Short Form 20a [ ] , , 1 , N , Physical function , N.

Techniques: Activity Assay, Clinical Proteomics

Journal: Orphanet Journal of Rare Diseases

Article Title: Minimal clinically important differences in six-minute walking distance in late-onset Pompe disease

doi: 10.1186/s13023-024-03156-3

Figure Lengend Snippet: Anchor-based MCIDs for 6MWD (% predicted) for subgroups of patients with baseline 6MWD < 150 m, < 300 m, < 400 m, < 450 m, and ≥ 450 m. Abbreviations: 6MWD 6-min walk distance, m meter, FVC forced vital capacity, FVC ≥ 3% MCID based on patients with an FVC (% predicted) change from baseline of ≥ 3% at Week 52, MCID minimal clinically important difference, PROMIS Patient-Reported Outcome Measures Information System, PROMIS ≥ 0 MCID based on patients with a PROMIS Physical Function short form 20a (PF) change from baseline of ≥ 0 points at Week 52, PROMIS ≥ 2 MCID based on patients with a PROMIS PF change from baseline of ≥ 2 points at Week 52, PROMIS ≥ 4 MCID based on patients with a PROMIS PF change from baseline of ≥ 4 points at Week 52, SGIC Subject’s Global Impression of Change, SGIC ≥ 4 MCID based on patients with an SGIC of ≥ 4 points at Week 52, SGIC ≥ 5 MCID based on patients with an SGIC of ≥ 5 points at Week 52, SGIC = 4 MCID based on patients with an SGIC of = 4 points at Week 52, SGIC = 5 MCID based on patients with an SGIC of = 5 points

Article Snippet: In this study, answers were grouped into 3 categories: improving (patient scored ‘somewhat improved’, ‘much improved’, or ‘very much improved’); stable (patient scored ‘no change’); or declining (patient scored ‘somewhat worse’, ‘much worse’, or ‘very much worse’). (v) PROMIS® Physical Function short form 20a [ , ]: (PROMIS PF): This score is obtained after the patient completes a questionnaire with 20 questions on physical function, which the patient can score from unable to do (1) to be able to do without any difficulties or limitations (5).

Techniques:

Journal: Journal of Patient-Reported Outcomes

Article Title: Validation of the Patient-Reported Outcomes Measurement Information System (PROMIS ® ) physical function questionnaire in late-onset Pompe disease using PROPEL phase 3 data

doi: 10.1186/s41687-024-00686-z

Figure Lengend Snippet: Summary of outcome measurements collected in PROPEL

Article Snippet: Fig. 1 Correlation between PROMIS Physical Function short form 20a scores and other outcome measures, adjusted model. P-values are determined by multivariable linear regression.

Techniques:

Journal: Journal of Patient-Reported Outcomes

Article Title: Validation of the Patient-Reported Outcomes Measurement Information System (PROMIS ® ) physical function questionnaire in late-onset Pompe disease using PROPEL phase 3 data

doi: 10.1186/s41687-024-00686-z

Figure Lengend Snippet: Comparison of PROMIS physical function to other tests evaluating (physical) functioning in LOPD

Article Snippet: Fig. 1 Correlation between PROMIS Physical Function short form 20a scores and other outcome measures, adjusted model. P-values are determined by multivariable linear regression.

Techniques: Comparison

Journal: Journal of Patient-Reported Outcomes

Article Title: Validation of the Patient-Reported Outcomes Measurement Information System (PROMIS ® ) physical function questionnaire in late-onset Pompe disease using PROPEL phase 3 data

doi: 10.1186/s41687-024-00686-z

Figure Lengend Snippet: Correlation between PROMIS Physical Function short form 20a scores and other outcome measures, adjusted model. P-values are determined by multivariable linear regression. 6MWD: 6-minute walk distance; FVC: forced vital capacity; GSGC: gait, stairs, Gowers’ maneuver, chair; MMT: manual muscle test; N: number of patients; R: Pearson’s correlation coefficient; R-PAct: Rasch-built Pompe-specific Activity scale

Article Snippet: Fig. 1 Correlation between PROMIS Physical Function short form 20a scores and other outcome measures, adjusted model. P-values are determined by multivariable linear regression.

Techniques: Activity Assay