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Table 3 BWS conditional logistic regression

From: Priorities when deciding on participation in early-phase gene therapy trials for Duchenne muscular dystrophy: a best–worst scaling experiment in caregivers and adult patients

Object

 

95% CI

 

B

SE B

OR

LL

UL

P

Chance of improved muscle functiona

0.00

1.00

Chance of improved lung function

−0.14

0.18

0.87

0.61

1.25

.450

Limits later use of gene therapy or CRISPR

−0.26

0.12

0.77

0.61

0.98

.033

Chance of improved heart function

−0.28

0.08

0.75

0.65

0.87

<.001

Chance of death (low risk)

−0.84

0.13

0.43

0.34

0.56

<.001

Lowest dose may be too low for benefit

−1.20

0.12

0.30

0.24

0.38

<.001

Become ineligible for future trials

−1.20

0.13

0.30

0.23

0.39

<.001

Benefit lasts about 10 years

−1.32

0.09

0.27

0.23

0.32

<.001

Chance of long hospitalization

−1.75

0.11

0.17

0.14

0.22

<.001

Chance of being in placebo group

−1.98

0.12

0.14

0.11

0.17

<.001

Two muscle biopsies required

−2.49

0.11

0.08

0.07

0.10

<.001

Interaction terms

 Improved lung function × Adults with Duchennea

 Improved lung function × Parents of children who walk

−0.94

0.19

0.39

0.27

0.57

<.001

 Improved lung function × Parents of children who use a wheelchair

−0.32

0.20

0.72

0.49

1.08

.115

  1. N = 274. Wald χ2 (12)=741.24, P < .001, R2McFadden’s = .13. B = Conditional logit regression coefficient. SE B = Robust standard error of B. OR = Odds ratio. CI = Confidence interval for odds ratios. LL = Lower limit of confidence interval. UL = Upper limit of confidence interval
  2. aReference category