Lifetime impact of achondroplasia study in Europe (LIAISE): findings from a multinational observational study

Table 5 Correlation analysis between height/z-score and patient-reported outcomes (subset of study population)

Measure	Height^a	Height z-score
QoLISSY^b (administered to patients 5–17 years old; n = 67)
Physical score	0.387*	0.466*
Social score	0.366*	0.345*
Emotional score	0.203	0.229
Coping score	0.052	− 0.230
Beliefs score	0.159	0.324*
Future score	0.117	0.268*
Effects score	0.232	0.059
Total score	0.361*	0.394*
PedsQL^b (administered to patients 5–17 years old; n = 63)
Physical score	0.041	0.273*
Emotional score	0.054	0.100
Social score	0.200	0.177
School score	− 0.054	0.255*
Psychosocial score	0.076	0.218
Total score	0.068	0.266*
EQ-5D-5L^c (administered to patients ≥ 18 years old; n = 55)
Mobility score	− 0.039	− 0.065
Self-care score	− 0.118	− 0.167
Usual activities score	− 0.184	− 0.157
Pain/discomfort score	0.134	0.102
Anxiety/depression score	− 0.206	− 0.263*
Utility	0.083	0.143
VAS score	− 0.180	− 0.158
APPT^d (administered to patients 8–17 years old; n = 18)
Sensory score	0.292	− 0.122
Affective score	0.547*	0.156
Evaluative score	0.147	0.212
Temporal score	0.538*	0.233
Total pain score	0.641*	0.191
BPI-SF^d (administered to patients ≥ 18 years old; n = 45)
Severity score	0.078	0.079
Interference score	0.009	− 0.050
WeeFIM^d (administered to patients 5–17 years old; n = 58)
Self-care score	0.423*	0.339*
Mobility score	0.399*	0.404*
Cognition score	0.076	0.036
Total score	0.407*	0.351*

^*Indicates significant results (p < 0.05 or p < 0.01)
^aPositive cut-off value indicates a positive correlation. Negative cut-off value indicates a negative correlation. Coefficients included Pearson’s and Spearman’s. Very high correlation: 0.9─1.0; high correlation: 0.7─ < 0.9; moderate correlation: 0.5─ < 0.7; low correlation: 0.3─ < 0.5; negligible correlation: 0.0─ < 0.3. As exploratory analyses, it cannot be concluded that any p-values or statistical significance imply causality
^bSelf-assessment (if available), otherwise caregiver proxy assessment; 3/67 and 2/61 of the questionnaries for the QoLISSY and PedsQL were completed by proxies, respectively
^cA lower score on the EQ-5D-5L indicates better QoL; therefore, a negative correlation with the EQ-5D score indicates a positive association with QoL
^dProxy-assessment
APPT Adolescent Pediatric Pain Tool, BPI-SF Brief Pain Inventory-Short Form, PedsQL Pediatric quality of life inventory, QoL Quality of life, QoLISSY Quality of Life in Short Stature Youth, WeeFIM Paediatric Functional Independence Measure, VAS Visual analogue scale

ISSN: 1750-1172