Background
Methods
Study design
Study population
Patient-reported outcome assessments
Statistical analysis
Results
Study population
Sociodemographic / clinical characteristics | Study part | p-value** | ||
---|---|---|---|---|
Part A (n = 70) | Part B (n = 102) | Total (n = 172) | ||
Gender n (%) | ||||
Male | 52 (74.3%) | 69 (67.6%) | 121 (70.3%) | 0.3490 |
Female | 18 (25.7%) | 33 (32.4%) | 51 (29.7%) | |
Age (years) | ||||
Mean (SD) | 70.20 (11.19) | 72.53 (9.81) | 71.58 (10.42) | 0.1505 |
Median | 73.00 | 73.50 | 73.00 | |
Range | 33.00–88.00 | 45.00–93.00 | 33.00–93.00 | |
Pooled geographic region n (%) | ||||
North America | 40 (57.1%) | 27 (26.5%) | 67 (39.0%) | 0.0001 |
Europe | 22 (31.4%) | 63 (61.8%) | 85 (49.4%) | |
Rest of the World | 8 (11.4%) | 12 (11.8%) | 20 (11.6%) | |
ECOG PS at baseline, n (%) | ||||
ECOG PS 0 | 38 (54.3%) | 66 (64.7%) | 104 (60.5%) | 0.1697 |
ECOG PS 1 | 32 (45.7%) | 36 (35.3%) | 68 (39.5%) | |
Site of primary tumor, n (%) | ||||
Non-skin | 9 (12.9%) | 7 (6.9%) | 16 (9.3%) | 0.1097 |
Skin | 55 (78.6%) | 91 (89.2%) | 146 (84.9%) | |
Not applicable | 0 (0.0%) | 4 (3.9%) | 4 (2.3%) | |
Missing | 6 (8.6%) | 0 (0.0%) | 6 (3.5%) | |
Tumor size at baseline (mm) | ||||
n (missing) | 61 (9) | 100 (2) | 161 (11) | |
Mean (SD) | 103.69 (79.68) | 79.46 (58.48) | 88.64 (68.09) | 0.0280 |
Median | 83.00 | 64.00 | 66.00 | |
Range | 16.00–404.00 | 0.00–288.00 | 0.00–404.00 | |
Time since initial diagnosis (years) | ||||
Mean (SD) | 2.19 (0.80) | 2.27 (0.76) | 2.24 (0.78) | 0.4631 |
Median | 2.00 | 2.00 | 2.00 | |
Min - Max | 1.00–3.00 | 1.00–3.00 | 1.00–3.00 | |
Time since first metastatic disease (months) | ||||
Mean (SD) | 16.94 (23.44) | 5.46 (7.66) | 10.13 (16.98) | <.0001 |
Median | 9.49 | 2.33 | 5.73 | |
Min - Max | 1.51–156.75 | 0.36–49.58 | 0.36–156.75 |
Internal consistency reliability
FACT-M subscale, summary and MCC-specific scores | # items | Cronbach’s alpha[a] | McDonald’s coefficient omega | Scale structure | ECOG PS (mean [SD]) | |||
---|---|---|---|---|---|---|---|---|
Range of item-subscale correlations[b] | Convergent[c]/ divergent[d] validity (% of items) | 0 (n = 106) | 1 (N = 66) | p-value[e] | ||||
FACT-G subscales | ||||||||
Physical well-being | 7 | 0.84 | 0.85 | 0.53–0.71 | 100 / 57 | 24.0 (4.2) | 21.3 (5.6) | <.001 |
Social/Family well-being[f] | 6 | 0.83 | 0.85 | 0.55–0.70 | 100 / 100 | 22.5 (5.4) | 21.6 (5.7) | 0.308 |
Emotional well-being | 6 | 0.80 | 0.80 | 0.36–0.70 | 83 / 67 | 18.1 (4.1) | 16.8 (5.0) | 0.0499 |
Functional well-being | 7 | 0.89 | 0.89 | 0.52–0.77 | 100 / 71 | 18.1 (6.7) | 15.8 (6.4) | 0.028 |
Melanoma-specific subscales | ||||||||
Melanoma subscale | 16 | 0.84 | 0.86 | 0.19–0.76 | 50 / 38 | 54.3 (7.5) | 50.6 (9.0) | 0.004 |
Melanoma surgery scale | 8 | 0.82 | 0.83 | 0.20–0.77 | 75 / 75 | 27.0 (5.3) | 24.4 (7.1) | 0.007 |
Summary scales | ||||||||
FACT-M TOI | 30 | 0.94 | – | – | – | 96.4 (16.7) | 88.1 (19.5) | 0.003 |
FACT-G Total score | 27 | 0.93 | – | – | – | 82.8 (16.2) | 75.5 (18.1) | 0.007 |
FACT-M Total score | 43 | 0.95 | – | – | – | 137.0 (22.3) | 126.6 (25.8) | 0.006 |
MCC-Specific scores | ||||||||
Physical function score (PF) | 6 | 0.89 | 0.89 | 0.61–0.76 | 100 / 100 | 17.2 (5.5) | 14.3 (5.9) | 0.001 |
Psychological impact score (PI) | 6 | 0.84 | 0.85 | 0.55–0.72 | 100 / 100 | 18.4 (4.3) | 17.0 (5.2) | 0.064 |
MCC summary score | 12 | 0.90 | – | – | – | 35.5 (8.7) | 31.3 (10.0) | 0.004 |
Convergent and divergent validity using multi-trait analysis
Construct validity using confirmatory factor analysis
Clinical/known groups validity
Concurrent validity
Score at baseline | EQ-5D mobility | EQ-5D self-care | EQ-5D usual activity | EQ-5D pain/ discomfort | EQ-5D anxiety/ depression | EQ VAS | EQ-5D Index |
---|---|---|---|---|---|---|---|
FACT-G subscales | |||||||
Physical well-being | − 0.43** | − 0.39 | − 0.64 | − 0.68 | − 0.43 | 0.59 | 0.65 |
Social/Family well-being | −0.21 | − 0.14 | − 0.19 | − 0.12 | − 0.24 | 0.22 | 0.22 |
Emotional well-being | − 0.31 | − 0.27 | − 0.45 | −0.41 | − 0.59 | 0.52 | 0.48 |
Functional well-being | −0.46 | −0.33 | − 0.63 | −0.51 | − 0.52 | 0.56 | 0.61 |
Melanoma-specific subscales | |||||||
Melanoma subscale* | −0.49 | −0.41 | − 0.70 | −0.65 | − 0.49 | 0.63 | 0.68 |
Melanoma surgery scale | −0.38 | −0.42 | − 0.53 | −0.45 | − 0.26 | 0.45 | 0.50 |
Summary scales | |||||||
FACT-M TOI | −0.50 | −0.41 | − 0.72 | −0.66 | − 0.53 | 0.65 | 0.71 |
FACT-G Total score | −0.45 | −0.35 | − 0.60 | −0.54 | − 0.55 | 0.59 | 0.62 |
FACT-M Total score | −0.49 | −0.39 | − 0.67 | −0.60 | − 0.56 | 0.64 | 0.67 |
MCC-specific scores | |||||||
Physical function (PF) | −0.56 | −0.40 | − 0.73 | −0.59 | − 0.47 | 0.57 | 0.68 |
Psychological impact (PI) | −0.30 | −0.26 | − 0.45 | −0.43 | − 0.58 | 0.50 | 0.48 |
MCC summary score | −0.49 | −0.38 | − 0.67 | −0.57 | − 0.57 | 0.60 | 0.66 |
Ability of the FACT-M scores to detect change over time
FACT-M subscale, summary and MCC-specific scores | Percent change in tumor size; mean (effect size) | Change in EQ VAS (MID = 7); mean (effect size) | ||||||
---|---|---|---|---|---|---|---|---|
Reduction ≤ − 30%[a] (N = 47) | Reduction > − 30% to ≤0 (N = 14) | Increase > 0% (N = 44) | p-value[b] | Improved ≥7 points (n = 35) | Stable > − 7 to < 7 (n = 56) | Worsened ≤ − 7 points (n = 30) | p-value[b] | |
FACT-G subscales | ||||||||
Physical well-being | −0.02 (− 0.01) | −0.50 (− 0.11) | − 1.86 (− 0.36) | 0.101 | 0.46 (0.10) | −0.54 (− 0.11) | −3.17 (− 1.03) | < 0.001 |
Social/Family well-being | 0.47 (0.09) | − 0.21 (− 0.06) | − 1.55 (− 0.24) | 0.209 | 0.72 (0.15) | − 1.19 (− 0.21) | − 0.13 (− 0.02) | 0.272 |
Emotional well-being | 1.38 (0.35) | 0.79 (0.14) | 0.23 (0.05) | 0.292 | 2.37 (0.63) | 0.99 (0.20) | −2.03 (− 0.65) | <.0001 |
Functional well-being | 0.06 (0.01) | − 0.43 (− 0.06) | − 2.16 (− 0.30) | 0.164 | 0.34 (0.05) | − 0.91 (− 0.12) | −2.24 (− 0.40) | 0.165 |
Melanoma-specific subscales | ||||||||
Melanoma subscale | 1.64 (0.21) | −2.64 (− 0.44) | − 2.32 (− 0.27) | 0.006 | 1.74 (0.24) | − 0.18 (− 0.02) | −4.63 (− 0.69) | <.0001 |
Melanoma surgery scale | 1.50 (0.36) | −1.79 (− 0.48) | −1.16 (− 0.15) | 0.043 | 0.69 (0.12) | 0.02 (0.02) | − 0.67 (− 0.09) | 0.619 |
Summary scales | ||||||||
FACT-M TOI | 1.68 (0.10) | − 3.57 (− 0.23) | − 6.34 (− 0.32) | 0.012 | 2.54 (0.16) | − 1.63 (− 0.08) | −10.04 (− 0.76) | < 0.001 |
FACT-G Total score | 1.89 (0.13) | − 0.36 (− 0.02) | − 5.34 (− 0.29) | 0.026 | 3.90 (0.26) | − 1.65 (− 0.09) | −7.56 (− 0.56) | 0.001 |
FACT-M Total score | 3.53 (0.17) | −3.00 (− 0.14) | − 7.65 (− 0.30) | 0.009 | 5.64 (0.27) | −1.83 (− 0.07) | −12.20 (− 0.67) | <.0001 |
MCC-Specific scores | ||||||||
Physical function score (PF) | −0.30 (− 0.06) | −1.64 (− 0.32) | −2.18 (− 0.33) | 0.100 | −0.09 (− 0.01) | −0.95 (− 0.15) | −3.60 (− 0.79) | 0.002 |
Psychological impact score (PI) | 1.40 (0.32) | 1.21 (0.22) | 0.30 (0.07) | 0.393 | 2.94 (0.71) | 0.98 (0.20) | −2.10 (−0.62) | <.0001 |
MCC summary score | 1.11 (0.14) | −0.43 (− 0.04) | −1.89 (− 0.19) | 0.129 | 2.86 (0.33) | 0.04 (0.00) | −5.70 (− 0.91) | <.0001 |
MID thresholds
FACT-M subscale and summary scores | 0.2*SDBL | 0.5*SDBL | SEM[a] | Anchor[b] | Range (min-max)[c] | Range rounded (min-max)[d] | Published MIDs for FACT-M[e] |
---|---|---|---|---|---|---|---|
FACT-G subscales | |||||||
Physical well-being | 0.99 | 2.47 | 1.95 | 0.46 | 0.46–2.47 | 1–3 | 2–3 † |
Social/Family well-being | 1.09 | 2.74 | 2.26 | 0.72 | 0.72–2.74 | 1–3 | |
Emotional well-being | 0.90 | 2.24 | 2.02 | 2.37 | 0.90–2.37 | 1–3 | 2† |
Functional well-being | 1.33 | 3.33 | 2.25 | 0.34 | 0.34–3.33 | 1–4 | 2–3† |
Melanoma-specific subscales | |||||||
Melanoma subscale | 1.65 | 4.13 | 3.31 | 1.74 | 1.65–4.13 | 2–5 | 2–4 ¥ |
Melanoma surgery scale | 1.23 | 3.08 | 2.63 | 0.69 | 0.69–3.08 | 1–3 | 1–2 ¥ |
Summary scales | |||||||
FACT-M Trial Outcome Index (TOI) | 3.40 | 8.50 | 4.30 | 2.54 | 2.54–8.50 | 3–9 | 5–9 ¥ |
FACT-G Total score | 3.44 | 8.60 | 4.59 | 3.90 | 3.44–8.60 | 4–9 | 5–7 † |
FACT-M Total score | 4.83 | 12.08 | 5.61 | 5.64 | 4.83–12.08 | 5–12 | |
MCC-Specific scores | |||||||
FACT-M Physical function score | 1.15 | 2.88 | 1.93 | −0.09 | 1.15–2.88 | 2–3 | |
FACT-M Psychological impact score | 0.94 | 2.35 | 1.90 | 2.94 | 0.94–2.94 | 1–3 | |
FACT-M MCC summary score | 1.88 | 4.71 | 2.95 | 2.86 | 1.88–4.71 | 2–5 |