Page 318 - Haematologica Vol. 109 - July 2024

LETTER TO THE EDITOR
PRO scores remained generally consistent with baseline ones through cycle 6 in both treatment groups (Figure 1). Consistent with this, mean change from baseline in PRO scores through cycle 6 was also largely unchanged (Online Supplementary Figure S1).
Overall, there was no difference in the proportion of pa- tients with improvements or stable or worsening PRO measures over time between treatment groups, as exem- plified by a snapshot at cycle 6 (Figure 2). Most patients had improved or stable PRO measures at cycle 6. The proportions of patients with improved PRO measures at cycle 6 ranged from 20% to 39% in the melflufen group and 11% to 33% in the pomalidomide group, and the pro- portions with stable PRO measures ranged from 26% to
72% for melflufen and from 37% to 67% for pomalidomide. In contrast, the proportion of patients with worsening PRO measures was generally low, particularly for physical functioning (melflufen: 13% and pomalidomide: 15%), emo- tional functioning (8% and 20%), disease symptoms (10% and 20%), and side effects of treatment (13% and 26%). Because a TTP <36 months after previous ASCT has been shown to be associated with worse outcomes in patients treated with melflufen and dexamethasone,5 PRO assess- ments were compared between the target and non-target populations within the melflufen group.
Mean PRO scores at baseline with melflufen and dexa- methasone were similar between the target and non-target populations and trended similar to those in the overall
Table 1. Baseline characteristics of patients with and without patient-reported outcomes assessments.a
Patients with PRO assessments
Melflufen group Pomalidomide group Overall N=77 N=81 N=158
Patients without PRO assessmentsb N=310
Age in years, median (range) 69 (41-91) 67 (44-82) 68 (41-91)
67 (39-87)
Age group, N (%)
< 65 years 27 (35) 27 (33) 54 (34)
118 (38)
65-74 years 34 (44) 39 (48) 73 (46)
154 (50)
≥ 75 years 16 (21) 15 (19) 31 (20)
38 (12)
Male, N (%) 46 (60) 41 (51) 87 (55)
178 (57)
Race, N (%)
White 70 (91) 69 (85) 139 (88)
284 (92)
All other races 5 (6) 11 (14) 16 (10)
11 (4)
Missing 2 (3) 1 (1) 3 (2)
15 (5)
ECOG PS, N (%)
0 34 (44) 35 (43) 69 (44)
104 (34)
1 37 (48) 43 (53) 80 (51)
173 (56)
2 6 (8) 3 (4) 9 (6)
33 (11)
EORTC QLQ-C30 multi-item scales, mean score (range)c
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Global health status/QoL 63.8 (16.7-100) 64.3 (16.7-100) 64.0 (16.7-100)
Physical functioning 72.4 (20-100) 74.2 (33.3-100) 73.3 (20-100)
Emotional functioning 81.0 (0-100) 79.8 (16.7-100) 80.4 (0-100)
EORTC QLQ-C30 symptom scales, mean score (range)d
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Fatigue 35.1 (0-100) 32.6 (0-100) 33.8 (0-100)
Pain 30.2 (0-100) 28.7 (0-100) 29.5 (0-100)
EORTC MY20 score, mean (range)e
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Disease symptoms 24.7 (0-83.3) 22.6 (0-88.9) 23.6 (0-88.9)
Side effects of treatment 16.1 (0-73.3) 16.1 (0-60) 16.1 (0-73.3)
EQ-5D-3L VAS score, mean (range)f 64.0 (20-100) 66.9 (9-100) 65.5 (9-100)
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aPatients from OCEAN enrolled after protocol v4.1 who did not complete the PRO questionnaire were excluded from the analysis. bBaseline characteristics in the PRO analysis population and in patients not included in the PRO analysis population were comparable, except for a higher proportion of patients in the PRO analysis population having an ECOG PS score of 0 (44% vs. 34%). cEORTC QLQ-C30 global health status/QoL, emotional functioning, and physical functioning scores range from 0 to 100; a higher score indicates better function.9 dEORTC QLQ-C30 fatigue and pain symptoms range from 0 to 100; a higher score indicates greater severity of symptoms.10 eEORTC MY20 disease symptoms and side effects of treatment scores range from 0 to 100; a higher score indicates greater severity of symptoms.10 fEQ-5D-3L VAS scores range from 0 to 100; a higher score indicates a better health state (0 equals death; 100 equals perfect health).11 PRO: patient-reported outcome; melflufen: melphalan flufenamide; ECOG PS: Eastern Cooperative Oncology Group performance status; EORTC: European Organi- zation for Research and Treatment of Cancer; QLQ-C30: Core Quality of Life Questionnaire; QoL: quality of life; MY20: Multiple Myeloma Mod- ule; EQ-5D-3L: European Quality of Life 5 Dimensions 3 Level Version; VAS: visual analog scale.
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