TABLE 1.
Unadjusted | Adjusted | |||
---|---|---|---|---|
Variable | Categories | CARTITUDE‐1, N (%) 97 (100%) | RW cohort, N OBS (%) 336 (100%) | RW cohort, ESS a (%) 80 (100%) |
Variables used in adjustment with IPTW for the base case | ||||
Refractory status b |
Penta refractory c Triple or quad refractory d Others |
41 (42.3) 44 (45.4) 12 (12.4) |
177 (52.7) 130 (38.7) 29 (8.6) |
37 (46.3) 36 (45.5) 7 (8.2) |
ISS stage |
I II III |
61 (62.9) 22 (22.7) 14 (14.4) |
120 (35.7) e 106 (31.5) e 110 (32.7) e |
53 (66.7) 16 (20.6) 10 (12.7) |
Cytogenetic profile |
High risk f Standard risk Unknown |
23 (23.7) 68 (70.1) 6 (6.2) |
62 (18.5) 181 (53.9) 93 (27.7) |
17 (21.8) 58 (72.1) 5 (6.1) |
Time to progression on last regimen |
≤4 months >4 months |
48 (49.5) 49 (50.5) |
101 (30.1) 235 (69.9) |
40 (50.5) 40 (49.5) |
Presence of total plasmacytoma g |
No Yes |
78 (80.0) 19 (20.0) |
NR NR |
NR NR |
Number of prior LOTs |
≤4 >4 |
33 (34.0) 64 (66.0) |
65 (19.3) 271 (80.7) |
24 (29.8) 56 (70.2) |
Years since MM diagnosis |
<6 ≥6 |
45 (46.4) 52 (53.6) |
259 (77.1) 77 (22.9) |
38 (47.6) 42 (52.4) |
Age |
<65 ≥65 |
62 (63.9) 35 (36.1) |
129 (38.4) 207 (61.6) |
51 (63.2) 29 (36.8) |
Variables not used in adjustment with IPTW for the base case | ||||
Hemoglobin (g/dL) |
<12 ≥12 |
90 (92.8) 7 (7.2) |
249 (74.1) h 87 (25.9) h |
59 (74.1) 21 (25.9) |
LDH levels (units/L) |
<280 ≥280 |
85 (87.6) 12 (12.4) |
284 (84.5) i 52 (15.5) i |
67 (83.4) 13 (16.6) |
Prior stem cell transplant |
No Yes |
10 (10.3) 87 (89.7) |
137 (40.8) 199 (59.2) |
21 (24.5) 60 (75.5) |
ECOG status |
0 1 |
39 (40.2) 58 (59.8) |
98 (29.2) j 238 (70.8) j |
33 (40.8) 50 (58.5) |
Race |
White Black/African American Not reported/other |
69 (71.1) 17 (17.5) 11 (11.3) |
233 (69.3) 48 (14.3) 55 (16.4) |
57 (71.5) 13 (16.3) 10 (12.2) |
Sex |
Female Male |
40 (41.2) 57 (58.8) |
151 (44.9) 185 (55.1) |
32 (39.6) 48 (60.4) |
Type of MM |
IgG Light chain Other |
57 (58.8) 24 (24.7) 16 (16.5) |
198 (58.9) 58 (17.3) 80 (23.8) |
48 (59.5) 16 (20.3) 16 (20.2) |
ESS was rounded to whole numbers.
Refractoriness was defined as discontinuation of drug of interest within 60 days and starting a different drug in the next line or starting a new drug within 60 days after end of previous treatment (RW cohort) and by International Myeloma Working Group consensus criteria (CARTITUDE‐1) [7, 21].
Refractory to at least two IMiDs, two PIs, and an anti‐CD38 MoAB.
Refractory to two IMiDs and one PI; or two PIs and one IMiD; or two IMiDs and two PIs.
ISS stage was imputed for 96 observations in the RW cohort.
At least one of del17p, t(14;16), or t(4;14).
Includes extramedullary plasmacytomas and soft‐tissue components of bone‐based plasmacytomas [24].
Hemoglobin was imputed for 1 observation in the RW cohort.
LDH was imputed for 97 observations in the RW cohort.
ECOG was imputed for 84 observations in the RW cohort.
Abbreviations: ATT, average treatment effect in the treated; ECOG, Eastern Cooperative Oncology Group; ESS, effective sample size; IMiD, immunomodulatory drug; IPTW, inverse probability of treatment weighting; ISS, International Staging System; LDH, lactate dehydrogenase; LOTs, lines of therapy; MM, multiple myeloma; MoAB, monoclonal antibody; N OBS, number of observations; NR, not reported; PI, proteasome inhibitor; RW, real world; SMD, standardized mean difference.