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Table 2 Comparison of PROMIS physical function to other tests evaluating (physical) functioning in LOPD

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

  

PROMIS Physical Function short form 20a

  

Univariable

Multivariable3

  

Regression coefficient B

(95% confidence interval)

P-value

Regression coefficient B

(95% confidence interval)

P-value

6MWD (per 1-meter increment)

0 weeks (baseline)1

0.06

(0.04, 0.07)

< 0.0001

0.04

(0.02, 0.07)

0.0006

Change from 0 to 52 weeks1

0.09

(0.05, 0.12)

< 0.0001

0.08

(0.03, 0.12)

0.0016

All measurements2

0.06

(0.05, 0.07)

< 0.0001

0.05

(0.03, 0.06)

< 0.0001

FVC (per 1% increment in predicted value)

0 weeks (baseline)1

0.20

(0.09, 0.30)

0.0004

0.10

(-0.04, 0.24)

0.1719

Change from 0 to 52 weeks1

0.05

(-0.21, 0.31)

0.7035

0.06

(-0.30, 0.42)

0.7522

All measurements2

0.14

(0.07, 0.21)

0.0001

0.08

(0.00, 0.17)

0.0490

MMT of lower extremities (per 1-point increment)

0 weeks (baseline)1

0.96

(0.61, 1.30)

< 0.0001

0.67

(0.23, 1.11)

0.0034

Change from 0 to 52 weeks1

0.15

(-0.33, 0.63)

0.5349

0.08

(-0.45 0.61)

0.7626

All measurements2

0.26

(0.10, 0.43)

0.0023

0.11

(-0.07 0.29)

0.2401

GSGC test score (per 1-point increment)

0 weeks (baseline)1

-1.31

(-1.72, -0.90)

< 0.0001

-0.78

(-1.47, -0.09)

0.0278

Change from 0 to 52 weeks1

-0.28

(-1.01, 0.45)

0.4482

-0.30

(-1.30, 0.70)

0.5527

All measurements2

-0.48

(-0.74, -0.21)

0.0005

-0.19

(-0.52 0.14)

0.2489

R-PAct (per 1-point increment)

0 weeks (baseline)1

1.81

(1.57, 2.04)

< 0.0001

1.72

(1.36, 2.08)

< 0.0001

Change from 0 to 52 weeks1

1.07

(0.65, 1.50)

< 0.0001

1.03

(0.52, 1.53)

0.0001

All measurements2

1.19

(1.05, 1.34)

< 0.0001

1.23

(1.04, 1.41)

< 0.0001

  1. Bold values are those that show statistical significance
  2. 6MWD: 6-minute walk distance; FVC: forced vital capacity; GSGC: gait, stairs, Gowers’ maneuver, chair; LOPD: late-onset Pompe disease; MMT: manual muscle test; R-PAct: Rasch-built Pompe-specific Activity scale
  3. 1Regression coefficients were estimated using uni- and multivariable linear regression models (for unadjusted and adjusted model, respectively)
  4. 2Regression coefficients were estimated using mixed-effects linear regression models, adjusting for repeated measures within individuals
  5. 3Regression variables included were treatment (cipaglucosidase alfa plus miglustat or alglucosidase alfa plus placebo), age (continuous), sex (male or female), race (White, Asian, other), enzyme-replacement therapy prior to study (naïve or experienced), and body mass index (continuous) at baseline