Psychometric performance of the CFQ-R-8D compared to the EQ-5D-3L and SF-6D in people with cystic fibrosis

Mukuria, Clara; Rowen, Donna; Acaster, Sarah; McGarry, Lisa J.; Lou, Yiyue; Sosnay, Patrick R.; Quittner, Alexandra L.

doi:10.1186/s41687-024-00697-w

Journal of Patient-Reported Outcomes

Table 3 Responsiveness of generic and condition-specific measures by change in ppFEV₁ and severity group from baseline to follow up^a−c

From: Psychometric performance of the CFQ-R-8D compared to the EQ-5D-3L and SF-6D in people with cystic fibrosis

		Change in CFQ-R-8D					Change in EQ-5D-3L or SF-6D					Change in CFRSD
Variable	Groups	N	Mean	SD	SRM	ES	N	Mean	SD	SRM	ES	N	Mean	SD	SRM	ES
EQ-5D-3L trials
ppFEV₁—baseline to 24 weeks^d,e	Improvement	396	0.024	0.10	0.23	0.22	396	0.019	0.14	0.14	0.25	–	–	–	–	–
	No change	243	0.001	0.09	0.01	0.23	243	−0.016	0.13	−0.12	0.06	–	–	–	–	–
	Deterioration	267	−0.023	0.11	−0.21		267	−0.024	0.15	−0.16		–	–	–	–
ppFEV₁ severity group—baseline to 24 weeks^d	Improvement	120	0.041	0.10	0.41	0.39	120	0.035	0.13	0.28	0.30	–	–	–	–	–
	No change	723	0.000	0.11	0.00	0.28	723	−0.008	0.14	−0.06	0.05	–	–	–	–	–
	Deterioration	63	−0.029	0.10	−0.28		63	−0.015	0.16	−0.09		–	–	–	–
Pulmonary exacerbations (≥ 4)	No	829	0.011	0.10	0.11	0.59	829	0.006	0.13	0.05	0.37	–	–	–	–	–
Pulmonary exacerbations (≥ 4)	Yes	116	−0.051	0.14	−0.49		116	−0.046	0.20	−0.32		–	–	–	–
SF-6D trial
ppFEV₁—baseline to 24 weeks^d,e	Improvement	155	0.030	0.09	0.33	0.40	155	0.026	0.11	0.23	0.23	155	3.79	10.50	0.36	0.38
	No change	112	−0.010	0.11	−0.09	−0.06	112	0.002	0.10	0.02	0.09	112	−0.28	9.58	−0.03	0.04
	Deterioration	128	−0.004	0.10	−0.04		128	−0.007	0.09	−0.08		128	−0.74	11.34	−0.07
ppFEV₁ severity group—baseline to 24 weeks^d	Improvement	39	0.027	0.09	0.31	0.19	39	0.056	0.13	0.42	0.48	39	4.87	9.36	0.52	0.36
	No change	320	0.008	0.10	0.08	0.24	320	0.006	0.10	0.06	0.26	320	0.98	10.73	0.09	0.20
	Deterioration	36	−0.016	0.11	−0.15		36	−0.021	0.08	−0.28		36	−1.14	11.29	−0.10
Pulmonary exacerbations (≥ 4)	No	357	0.012	0.10	0.12	0.42	357	0.014	0.11	0.13	0.50	357	1.57	10.67	0.15	0.24
Pulmonary exacerbations (≥ 4)	Yes	48	−0.031	0.12	−0.30		48	−0.039	0.10	−0.37		48	−1.02	11.67	−0.09

CFQ-R-8D indicates Cystic Fibrosis Questionnaire–Revised–8 Dimensions; CFRSD: Cystic Fibrosis Respiratory Symptom Diary; ES: effect sizes; ppFEV₁: percent predicted forced expiratory volume in 1 s; SF-6D: Short Form 6 dimensions; SRM: standardized response mean
^a Pooled treatment and placebo arms
^b Change in ppFEV₁ severity group was defined as movement between the following groups: ppFEV₁ < 40%, ≥ 40 to < 70%, and ≥ 70%
^c Small SRM/ES ≥ 0.2 to < 0.5; medium SRM/ES ≥ 0.5 to < 0.8; large SRM/ES ≥ 0.8
^d 24 weeks indicates follow-up at 24 weeks
^e For ppFEV₁, an improvement was defined as an increase of ≥ 2 percentage points, no change was defined as a change of < ± 2 percentage points, and deterioration was defined as a decrease of ≥ 2 percentage points

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