Background
Methods
Study design
Data collected
FACT-M questionnaire
EQ-5D questionnaire
Patient interviews
Statistical analyses
Results
Patient population
Characteristicsa
| PAS (N = 70) | Interviewed patients (N = 19) |
---|---|---|
Sex, n (%) | ||
Male | 52 (74.3%) | 15 (78.9%) |
Female | 18 (25.7%) | 4 (21.1%) |
Age, years | ||
Mean (SD) | 70.2 (11.2) | 72.2 (8.2) |
Median | 73.0 | 72.0 |
95% CI | 67.5–72.9 | 68.3–76.2 |
Region, n (%) | ||
North America | 40 (57.1%) | 17 (89.5%) |
Europe | 22 (31.4%) | 2 (10.5%) |
Rest of the world | 8 (11.4%) | 0 (0.0%) |
ECOG PS at baseline (n, %) | ||
0 | 38 (54.3%) | 10 (52.6%) |
1 | 32 (45.7%) | 9 (47.4%) |
Site of primary tumor (n, %) | ||
Non-skin | 9 (12.9%) | 4 (21.1%) |
Skin | 55 (78.6%) | 14 (73.7%) |
Missing | 6 (8.6%) | 1 (5.3%) |
Tumor size at baseline (mm) | ||
n (missing) | 61 (9) | 16 (3) |
Mean (SD) | 103.7 (79.7) | 83.3 (49.9) |
Median | 83.0 | 78.5 |
95% CI | 83.3–124.1 | 56.7–109.8 |
Time since initial diagnosis (years) | ||
Mean (SD) | 2.2 (0.8) | 2.3 (0.8) |
Median | 2.0 | 2.0 |
95% CI | 2.0–2.4 | 2.0–2.7 |
Time since first metastatic disease (months) | ||
Mean (SD) | 16.9 (23.4) | 16.3 (10.4) |
Median | 9.5 | 14.0 |
95% CI | 11.4–22.5 | 11.3–21.2 |
Number of previous therapy lines (n, %) | ||
Mean (SD) | 1.5 (0.7) | 1.3 (0.5) |
Median | 1.0 | 1.0 |
95% CI | 1.3–1.6 | 1.1–1.6 |
Development of the additional MCC-specific FACT-M scores
FACT-M Subscales and Summary scores | # of items | Items/Scales | Score rangea
|
---|---|---|---|
FACT-G subscales | |||
Physical well-being (PWB) | 7 | GP1 – GP7 | 0–28 |
Social/Family well-being (SWB) | 7 | GS1 – GS7 | 0–28 |
Emotional well-being (EWB) | 6 | GE1 – GE6 | 0–24 |
Functional well-being (FWB) | 7 | GF1 – GF7 | 0–28 |
Melanoma-specific subscales | |||
Melanoma Subscale (MS) | 16 | M1-M3, B1, ITU4, An10, Hep3, C1, C6, M5, M6, ITU3, MS8, M8, M9, HI7 | 0–64 |
Melanoma surgery Scale (MSS) | 8 | M10-M17 | 0–32 |
Summary score | |||
FACT-M TOI | 30 | PWB + FWB + MS | 0–120 |
FACT-G total score | 27 | PWB + SWB + EWB + FWB | 0–108 |
FACT-M total score | 43 | PWB + SWB + EWB + FWB + MS | 0–172 |
MCC-specific scores | |||
Physical Function score (PF) | GP1, GP3, GF1, GF3, GF7, ITU4 | 0–24 | |
Psychological Impact score (PI) | GE1, GE3 - GE6, MS8 | 0–24 | |
MCC summary score | PF + PI | 0–48 |
Description of original FACT-M scale and additional MCC-specific scale scores over time
FACT-M Subscales and Summary scores | Score rangea
| Score - Mean (SD) | ||||
---|---|---|---|---|---|---|
Baseline (N = 70) | Week 7 (N = 49) | Week 13 (N = 38) | Week 19 (N = 29) | Week 25 (N = 27) | ||
FACT-G subscales | ||||||
Physical well-being (PWB) | 0–28 | 21.8 (5.69) | 21.3 (5.50) | 22.5 (4.23) | 22.3 (5.33) | 23.4 (3.53) |
Social/Family well-being (SWB) | 0–28 | 22.4 (5.04) | 21.5 (5.90) | 21.2 (4.90) | 22.7 (4.02) | 23.1 (4.37) |
Emotional well-being (EWB) | 0–24 | 17.5 (4.56) | 18.4 (4.46) | 19.0 (3.58) | 18.1 (4.29) | 19.4 (3.46) |
Functional well-being (FWB) | 0–28 | 16.6 (6.37) | 16.7 (6.15) | 16.3 (5.30) | 16.6 (6.33) | 18.6 (5.50) |
Melanoma-specific subscales | ||||||
Melanoma Subscale (MS) | 0–64 | 50.5 (9.00) | 50.3 (8.77) | 51.5 (7.71) | 51.6 (8.22) | 53.9 (6.52) |
Melanoma surgery Scale (MSS) | 0–32 | 26.1 (6.10) | 25.8 (7.22) | 26.4 (5.87) | 25.9 (6.83) | 28.6 (3.42) |
Summary scales | ||||||
FACT-M TOI | 0–120 | 88.9 (19.28) | 88.3 (18.23) | 90.3 (15.21) | 90.5 (18.28) | 95.8 (14.05) |
FACT-G total score | 0–108 | 78.2 (17.71) | 77.8 (16.15) | 79.0 (12.89) | 79.7 (16.03) | 84.5 (12.89) |
FACT-M total score | 0–172 | 128.7 (25.24) | 128.1 (22.88) | 130.5 (18.96) | 131.3 (23.28) | 138.4 (17.95) |
MCC-specific scores | ||||||
Physical Function score (PF) | 0–24 | 15.2 (5.73) | 14.1 (5.58) | 14.8 (5.11) | 14.9 (5.79) | 17.1 (4.43) |
Psychological Impact score (PI) | 0–24 | 17.4 (4.75) | 18.7 (4.81) | 19.3 (3.86) | 18.3 (4.33) | 19.2 (3.34) |
MCC summary score | 0–48 | 32.6 (9.53) | 32.8 (9.07) | 34.2 (7.66) | 33.2 (8.64) | 36.3 (6.91) |
Psychometric validation in the mMCC population of both original and additional MCC-specific FACT-M scores
Internal consistency reliability
FACT-M Subscales and Summary scores | # of items | Cronbach’s alphaa
| Scale structure | ECOG PS (mean [SD]) | EQ-5D VAS | EQ-5D index | |||
---|---|---|---|---|---|---|---|---|---|
Range of item-subscale correlationsb
| Convergentc/ Divergentd validity (% of items) | 0 (N = 39) | 1 (N = 31) |
p-valuee
| Correlationb
| Correlationb
| |||
FACT-G subscales | |||||||||
Physical well-being (PWB) | 7 | 0.86 | 0.49–0.81 | 100/57 | 23.21 (5.02) | 20.10 (6.08) | 0.0221 | 0.44 | 0.75 |
Social/Family well-being (SWB) | 7 | 0.81 | 0.41–0.86 | 100/71 | 23.03 (4.78) | 21.49 (5.30) | 0.2065 | 0.15 | 0.24 |
Emotional well-being (EWB) | 6 | 0.83 | 0.53–0.75 | 100/67 | 17.97 (4.22) | 16.87 (4.95) | 0.3179 | 0.32 | 0.53 |
Functional well-being (FWB) | 7 | 0.87 | 0.49–0.82 | 100/71 | 17.41 (6.68) | 15.48 (5.90) | 0.2113 | 0.29 | 0.63 |
Melanoma-specific subscales | |||||||||
Melanoma Subscale (MS) | 16 | 0.85 | 0.24–0.85 | 75/31 | 51.64 (8.93) | 49.10 (9.02) | 0.2427 | 0.39 | 0.75 |
Melanoma surgery Scale (MSS) | 8 | 0.84 | 0.15–0.81 | 88/63 | 26.51 (6.19) | 25.58 (6.03) | 0.5290 | 0.16 | 0.51 |
Summary scales | |||||||||
FACT-M TOI | 30 | 0.94 | – | – | 92.26 (18.75) | 84.68 (19.40) | 0.1026 | 0.41 | 0.78 |
FACT-G total score | 27 | 0.94 | – | – | 81.62 (17.25) | 73.95 (17.62) | 0.0713 | 0.37 | 0.67 |
FACT-M total score | 43 | 0.96 | – | – | 133.26 (24.66) | 123.04 (25.21) | 0.0926 | 0.40 | 0.74 |
MCC-specific scores | |||||||||
Physical Function score (PF) | 6 | 0.88 | – | – | 16.38 (5.67) | 13.71 (5.54) | 0.0519 | 0.37 | 0.72 |
Psychological Impact score (PI) | 6 | 0.85 | – | – | 17.82 (4.40) | 16.87 (5.18) | 0.4101 | 0.30 | 0.52 |
MCC summary score | 12 | 0.91 | – | – | 34.21 (8.99) | 30.58 (9.94) | 0.1145 | 0.37 | 0.69 |
Construct validity
FACT-M Subscales and Summary scores | Comparison of change in score from baseline to Week 7 Percent change in tumor size (mean change in score [ES[a]]) | MIDs assessed at Week 7 | Published MIDs in melanoma population | ||||||
---|---|---|---|---|---|---|---|---|---|
Change in tumor size | 0.2 x SDBL
| 0.5 x SDBL
| SEM[d]
| Range(min-max) | |||||
Reduction ≥ 30%[b]
(N = 14) | Reduction between 0-30% (N = 7) | Increase >0% (N = 16) |
p-value[c]
| ||||||
FACT-G subscales | |||||||||
Physical well-being (PWB) | 0.21 (0.05) | -1.00 (-0.18) | -1.75 (-0.31) | 0.631 | 1.14 | 2.85 | 2.15 | 0.21 - 2.85 | 2.00-3.00[e]
|
Social/Family well-being (SWB) | -0.49 (-0.10) | -0.45 (-0.12) | -1.74 (-0.28) | 0.624 | 1.01 | 2.52 | 2.21 | -0.49 - 2.52 | - |
Emotional well-being (EWB) | 1.07 (0.22) | 1.29 (0.20) | -0.25 (-0.09) | 0.596 | 0.91 | 2.28 | 1.87 | 0.91 - 2.28 | 2.00[e]
|
Functional well-being (FWB) | 2.64 (0.42) | -0.14 (-0.02) | -2.81 (-0.42) | 0.005 | 1.27 | 3.19 | 2.26 | 1.27 - 3.19 | 2.00-3.00[e]
|
Melanoma-specific subscales | |||||||||
Melanoma Subscale (MS) | 2.71 (0.26) | -2.43 (-0.36) | -2.94 (-0.42) | 0.051 | 1.80 | 4.50 | 3.46 | 1.80 - 4.50 | 2.00-4.00[f]
|
Melanoma surgery scale (MSS) | 2.07 (0.34) | -3.00 (-1.02) | -3.13 (-0.49) | 0.036 | 1.22 | 3.05 | 2.46 | 1.22 - 3.05 | 1.00-2.00[f]
|
Summary scales | |||||||||
FACT-M TOI | 5.57 (0.31) | -3.57 (-0.21) | -7.50 (-0.42) | 0.038 | 3.86 | 9.64 | 4.84 | 3.86 - 9.64 | 5.00-9.00[f]
|
FACT-G total score | 3.44 (0.21) | -0.31 (-0.02) | -6.55 (-0.35) | 0.155 | 3.54 | 8.85 | 4.24 | 3.44 - 8.85 | 3.00-7.00[e]
|
FACT-M total score | 6.15 (0.26) | -2.74 (-0.12) | -9.49 (-0.38) | 0.074 | 5.05 | 12.62 | 5.26 | 5.05 - 12.62 | - |
MCC-Specific scores | |||||||||
Physical Function score (PF) | 0.71 (0.14) | -1.86 (-0.33) | -2.13 (-0.34) | 0.153 | 1.15 | 2.87 | 1.88 | 0.71 - 2.87 | - |
Psychological Impact score (PI) | 0.79 (0.15) | 2.29 (0.39) | 0.31 (0.10) | 0.609 | 0.95 | 2.38 | 1.98 | 0.79 - 2.38 | - |
MCC summary score | 1.50 (0.17) | 0.43 (0.04) | -1.81 (-0.21) | 0.530 | 1.91 | 4.76 | 2.91 | 1.50 - 4.76 | - |
Ability to detect change and MID
FACT-M Subscales and Summary scores | Percentage of responders[a] at Week 7 by MID calculation method | |||
---|---|---|---|---|
SEM[b]
| Anchor[c]
| Minimum threshold | Maximum threshold | |
FACT-G subscales | ||||
Physical well-being (PWB) | 18.4 | 32.7 | 32.7 | 18.4 |
Social/Family well-being (SWB) | 22.4 | 53.1 | 53.1 | 18.4 |
Emotional well-being (EWB) | 34.7 | 34.7 | 44.9 | 26.5 |
Functional well-being (FWB) | 30.6 | 30.6 | 34.7 | 22.4 |
Melanoma-specific subscales | ||||
Melanoma Subscale (MS) | 22.4 | 26.5 | 34.7 | 20.4 |
Melanoma surgery scale (MSS) | 22.4 | 22.4 | 30.6 | 20.4 |
Summary scales | ||||
FACT-M TOI | 24.5 | 22.4 | 30.6 | 14.3 |
FACT-G total score | 30.6 | 34.7 | 34.7 | 20.4 |
FACT-M total score | 28.6 | 28.6 | 28.6 | 14.3 |
MCC-Specific scores | ||||
Physical Function score (PF) | 18.4 | 26.5 | 26.5 | 12.2 |
Psychological Impact score (PI) | 38.8 | 51.0 | 51.0 | 26.5 |
MCC summary score | 24.5 | 30.6 | 30.6 | 18.4 |