Converting EORTC QLQ-C30 scores to utility scores in the brigatinib ALTA study

Figures & data

Table 1. Summary of pertinent mapping algorithms of EORTC QLQ-C30 to EQ-5D-3L/-5L.

Reference	Estimation population	Mean observed EQ-5D (country-specific tariff used)	Regression method	No. of model variables	Model fit (adjusted R^2)	Mean QLQ-C30 global health
Jang et al.^42	Consecutive patients attending an outpatient clinic with NSCLC (n = 172)	0.76 (US)	Linear regression	15 (full model); 5 (reduced model)	0.57 (full model); 0.58 (reduced model)	65.9
Khan and Morris^44	Two national (UK) NSCLC clinical trials (TOPICAL, n = 670, recruited April 14, 2005 to April 1, 2009; SOCCAR, n = 130, recruited December 2005 to March 2010)	0.56 (TOPICAL; 3L, UK); 0.79 (SOCCAR; 3L, UK)	BB regression	15 (full model)	0.75 (TOPICAL); 0.71 (SOCCAR)	47.60 (TOPICAL); 71.36 (SOCCAR)
Khan et al.^45	Prospective (non-interventional) follow-up study in patients with NSCLC (n = 100), recruited March 2014 to July 2015	0.515 (3L, UK); 0.572 (5L)	BB	15 (full model)	0.69 (3L); 0.75 (5L)	Not reported
Longworth et al.^43	Patients from 3 studies with breast, lung, and hematological cancer (n = 771)	0.58 (3L, UK)	Response mapping	14 plus age and gender	Pseudo R^2: 0.449 (mobility); 0.392 (self-care); 0.461 (usual acts); 0.455 (pain); 0.364 (anxiety/ depression)	53
Proskorovsky et al.^56	Cohort study of patients with MM (n = 154)	0.7 (3L, UK)	Linear regression	14 (full model); 4 (trimmed model)	0.6956 (full model); 0.6941 (trimmed model)	60.1

Abbreviations. BB, Beta-binomial; EORTC QLQ-C30, European Organization for Research and Treatment of Cancer Quality-of-Life Questionnaire Core-30; EQ-5D, European Quality-of-Life-5 Dimensions; MM, multiple myeloma; NSCLC, non-small-cell lung cancer; UK, United Kingdom; US, United States.

Table 2. Patient sociodemographic and baseline clinical characteristics in the ALTA trial.

Characteristic	Total (n = 208)	Arm A (n = 105)	Arm B (n = 103)
Age, years
Mean (SD)	53.1 (13.3)	51.0 (13.2)	55.1 (13.1)
Range (Min–Max)	(18.0–82.0)	(18.0–82.0)	(20.0–81.0)
Gender
Male, n (%)	89 (42.8)	49 (46.7)	40 (38.8)
Female, n (%)	119 (57.2)	56 (53.3)	63 (61.2)
Race
White, n (%)	138 (66.3)	67 (63.8)	71 (68.9)
Black or African American, n (%)	2 (1.0)	0 (0.0)	2 (1.9)
Asian, n (%)	66 (31.7)	38 (36.2)	28 (27.2)
Other, n (%)	4 (2.0)	0 (0.0)	4 (3.9)
Geographic region
North America, n (%)	42 (20.2)	20 (19.0)	22 (21.4)
Europe, n (%)	100 (48.1)	46 (43.8)	54 (52.4)
Asia Pacific, n (%)	66 (31.7)	39 (37.1)	27 (26.2)
ECOG performance status
0, n (%)	75 (36.1)	32 (30.5)	43 (41.7)
1, n (%)	119 (57.2)	68 (64.8)	51 (49.5)
2, n (%)	14 (6.7)	5 (4.8)	9 (8.7)
Primary tumor status
IIIA, n (%)	1 (0.5)		1 (1.0)
IIIB, n (%)	4 (1.9)	3 (2.9)	1 (1.0)
IV, n (%)	203 (97.6)	102 (97.1)	101 (98.1)
Number of metastatic sites
Mean (SD)	3.5 (1.4)	3.6 (1.4)	3.4 (1.3)
Range (Min–Max)	(1.0–8.0)	(1.0–8.0)	(1.0–7.0)
Presence of brain metastasis
Yes, n (%)	145 (69.7)	74 (70.5)	71 (68.9)
No, n (%)	63 (30.3)	31 (29.5)	32 (31.1)

Abbreviations. ECOG, Eastern Cooperative Oncology Group; Max, maximum; Min, minimum; SD, standard deviation.

Figure 1. Mean utility scores over time (Khan et al.⁴⁵ and Longworth et al.⁴³). (A) Mean utility scores (Khan et al.⁴⁵; EQ-5D-5L) from baseline to cycle 35 day 1, EOT, and follow-up 30 days after last dose by treatment group (study AP26113-13-201): ITT-PRO population. (B) Mean utility scores (Longworth et al.⁴³; EQ-5D-3L) from baseline to cycle 35 day 1, EOT, and follow-up 30 days after last dose by treatment group (study AP26113-13-201): ITT-PRO population.

Figure 2. Mean utility change scores over time (Khan et al.⁴⁵ and Longworth et al.⁴³). (A) Mean change in utility scores (Khan et al.⁴⁵; EQ-5D-5L) from baseline to cycle 35 day 1, EOT, and follow-up 30 days after last dose by treatment group (study AP26113-13-201): ITT-PRO population. (B) Mean change in utility scores (Longworth et al.⁴³; EQ-5D-5L) from baseline to cycle 35 day 1, EOT, and follow-up 30 days after last dose by treatment group (study AP26113-13-201): ITT-PRO population.

Table 3. Change from baseline in utility scores by visit and treatment group.

n, Mean (SD)	Baseline utility score (primary method: Khan et al.^45; EQ-5D-5L)							Baseline utility score (secondary method: Longworth et al.^43; EQ-5D-3L)
	Total sample		Treatment group					Total sample		Treatment group
			Arm A		Arm B					Arm A		Arm B
BL to Cycle 2	196	0.09^a (0.28)	100	0.14^a (0.29)		96	0.04 (0.25)	197	0.07^a (0.23)	100	0.12^a (0.25)	97	0.02 (0.20)
BL to Cycle 3	182	0.09^a (0.28)	91	0.12^a (0.28)		91	0.06 (0.27)	182	0.07^a (0.23)	91	0.11^a (0.25)	91	0.04 (0.21)
BL to Cycle 4	168	0.11^a (0.27)	82	0.16^a (0.27)		86	0.06^a (0.26)	168	0.09^a (0.22)	82	0.13^a (0.24)	86	0.04 (0.19)
BL to Cycle 5	164	0.12^a (0.24)	80	0.15^a (0.26)		84	0.08^a (0.22)	164	0.09^a (0.22)	80	0.13^a (0.25)	84	0.05^a (0.18)
BL to Cycle 6	160	0.10^a (0.25)	78	0.15^a (0.26)		82	0.05 (0.23)	160	0.08^a (0.21)	78	0.13^a (0.23)	82	0.03 (0.18)
BL to Cycle 7	152	0.10^a (0.26)	76	0.14^a (0.28)		76	0.06^a (0.23)	152	0.08^a (0.23)	76	0.13^a (0.25)	76	0.03 (0.21)
BL to Cycle 8	148	0.10^a (0.28)	72	0.15^a (0.29)		76	0.05 (0.26)	149	0.08^a (0.25)	72	0.12^a (0.27)	77	0.03 (0.23)
BL to Cycle 9	144	0.07^a (0.26)	69	0.11^a (0.27)		75	0.04 (0.25)	144	0.06^a (0.22)	69	0.10^a (0.23)	75	0.03 (0.21)
BL to Cycle 10	133	0.07^a (0.26)	64	0.12^a (0.24)		69	0.03 (0.26)	134	0.06^a (0.23)	64	0.10^a (0.23)	70	0.02 (0.22)
BL to Cycle 11	130	0.06^a (0.27)	60	0.13^a (0.27)		70	−0.00 (0.26)	130	0.05^a (0.24)	60	0.10^a (0.24)	70	0.00 (0.23)
BL to Cycle 12	125	0.06^a (0.25)	60	0.10^a (0.26)		65	0.02 (0.24)	125	0.05^a (0.22)	60	0.08^a (0.24)	65	0.02 (0.19)

^ap-value < 0.05 based on one-sample t-test of utility change score against null hypothesis of no change (0).

Abbreviations. BL, baseline; EQ-5D-3L, European Quality-of-Life-3 Dimensions; EQ-5D-5L, European Quality-of-Life-5 Dimensions; SD, standard deviation.

Table 4. Change from baseline in utility scores by ECOG performance status results.

	ECOG performance status				p-value
	Worsen (progressive disease)		No change or improvement (stable)
	n	LS mean (SE)	n	LS mean (SE)
Change in utility score (Primary Method: Khan et al.^45; EQ-5D-5L)
Baseline to Cycle 3 Day 1	18	−0.05 (0.07)	163	0.11 (0.02)	0.0281
Baseline to Cycle 5 Day 1	7	0.09 (0.09)	156	0.12 (0.02)	0.8050
Change in utility score (Secondary Method: Longworth et al.^43; EQ-5D-3L)
Baseline to Cycle 3 Day 1	18	−0.04 (0.06)	163	0.08 (0.02)	0.0360
Baseline to Cycle 5 Day 1	7	0.04 (0.08)	156	0.09 (0.02)	0.5399

Analysis of covariance adjusted for age and gender; p-value: Comparison between worsen and no change/improvement.

Note. For ECOG performance status, categories range from Grade 0 (fully active) to Grade 5 (dead); changes from baseline in performance status grade were categorized as worsening (progressive disease) and no change or improvement (stable).

Abbreviations. ECOG, Eastern Cooperative Oncology Group; EQ-5D-3L, European Quality-of-Life-5 Dimensions; EQ-5D-5L, European Quality-of-Life-5 Dimensions; LS, least-squares; SE, standard error.

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Data availability

The ALTA study data (NCT02094573) are proprietary and owned by Millennium Pharmaceuticals, Inc.