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CARVYKTI - LocoMMotion (MMY4001) Study

Last Updated: 07/08/2024

SUMMARY

LocoMMotion was a prospective, noninterventional, multinational study evaluating the efficacy and safety of current, real-life, standard-of-care (SOC) antimyeloma treatments in patients with relapsed/refractory multiple myeloma (RRMM) who received ≥3 prior lines of therapy (LOT).¹^-⁵
- At a median follow-up of 26.4 months (95% confidence interval [CI], 25.0-28.1), the overall response rate (ORR) among the 248 enrolled patients was 31.9% (95% CI, 26.1-38.0).⁴
- The median progression-free survival (PFS) was 4.6 months (95% CI, 3.9-5.6), and the median overall survival (OS) was 13.8 months (95% CI, 10.8-17.0).⁴
- The median duration of treatment was 4.0 months (range, 0.1-33.6) and the median duration of response (DOR) was 7.4 months (95% CI, 4.9-11.1).⁴
- An adverse event (AE) was reported in 86.7% of patients during the index LOT. The most commonly reported AEs were cytopenias.⁴
- A total of 158 patients (63.7%) died during the study, predominantly due to progressive disease (PD).⁴
Efficacy outcomes were evaluated in key subgroups of patients participating in the LocoMMotion study after a median follow-up of 16 months.⁶
- The ORR ranged from 11.8% to 43.9% across all subgroups. The characteristics associated with worse efficacy outcomes included tripleclass refractory, penta-drug exposed, penta-drug refractory, Eastern Cooperative Oncology Group performance status (ECOG PS) ≥1, poor renal function (creatinine clearance ≤60 mL/min), International Staging System (ISS) stage II-III, presence of extramedullary plasmacytomas, high lactate dehydrogenase (LDH; >245 U/L), bone marrow plasma cells ≥60%, and high-risk cytogenetics.
An analysis of health-related quality of life (HRQoL) was conducted in patients in the LocoMMotion study.⁷
- In the overall population, the least square (LS) mean changes from baseline during SOC treatment showed minimal improvement in HRQoL, primarily worsening during subsequent treatment.
- Patients who achieved very good partial response (≥VGPR) during SOC treatment had a greater improvement in patient-reported outcome (PRO) scores, including LS mean change for pain score.

CLINICAL DATA

LocoMMotion (MMY4001; clinicaltrials.gov identifier: NCT04035226) was a prospective, noninterventional, multinational study evaluating the efficacy and safety of current, real-life, SOC antimyeloma treatments in patients with RRMM who received ≥3 prior LOT.¹^-⁵

Study Design/Methods

The study was conducted in the United States and 9 European countries (Germany, France, Spain, Italy, United Kingdom, Poland, Russia, the Netherlands, and Belgium).²
The study design is summarized in Figure: LocoMMotion Study Design.

LocoMMotion Study Design¹^-⁵

Abbreviations: CD, cluster of differentiation; CBR, clinical benefit rate; CR, complete response; DOR, duration of response; ECOG PS, Eastern Cooperative Oncology Group Performance Status; IMWG, International Myeloma Working Group; mAb, monoclonal antibody; MM, multiple myeloma; MR, minimal response; MRD, minimal residual disease; ORR, overall response rate; OS, overall survival; PD, progressive disease; PFS, progression-free survival; PFS2, time to progression on the next line of subsequent antimyeloma therapy or death, whichever occurs first; PI, proteasome inhibitor; PR, partial response; PRO, patient-reported outcome; sCR, stringent complete response; SOC, standard of care; VGPR, very good partial response.
^aProgressive disease/responses were assessed by response review committee. Responses with subsequent LOT were subject to investigator assessment.
^bCBR is defined as the percentage of patients with clinical benefit. CBR = ORR (sCR + CR + VGPR + PR) + MR.

Results

Patient Characteristics

Overall, 248 patients were included in the study, all of whom received a median of 4 prior LOT (range, 2-13).³ The baseline patient characteristics are summarized in Table: Baseline Patient Demographics and Characteristics.
The index LOT included 91 unique treatment regimens. A total of 65.3% of patients (n=162) received a combination of ≥3 drugs and 2.4% of patients had a stem cell transplant (SCT).⁴ SOC regimens are summarized in Table: Antimyeloma SOC Treatments.

Baseline Patient Demographics and Characteristics²^,³^,⁸

Patient Characteristic	Total (N=248)
Median age, years (range)	68 (41-89)
Sex, male, n (%)	135 (54.4)
Geographic region, n (%)
US	23 (9.3)
Europe	225 (90.7)
Race^a, n (%)
White	182 (73.7)
Black or African American	5 (2.0)
Other	1 (0.4)
Unknown/not reported	59 (23.9)
Baseline ECOG PS^b, n (%)
0	65 (26.2)
1	179 (72.2)
2	3 (1.2)
3	1 (0.4)
Median years since initial MM diagnosis (range)	6.3 (0.3-22.8)
Median number of prior LOT (range)	4 (2-13)
Prior LOT^c, n (%)
2	16 (6.5)
3	48 (19.4)
4	62 (25.0)
≥5	122 (49.2)
ISS stage at enrollment^d, n (%)
I	70 (32.3)
II	69 (31.8)
III	78 (35.9)
Presence of extramedullary plasmacytomas^c, n (%)
Yes	33 (13.3)
No	215 (86.7)
Type of measurable disease^c, n (%)
Serum only	123 (49.6)
Serum and urine	19 (7.7)
Urine only	22 (8.9)
Serum free light chain	82 (33.1)
Not evaluable	2 (0.8)
Previous stem cell transplant, n (%)	160 (64.5)
Creatinine clearance^c, n (%)
≤60 mL/min	94 (40.0)
>60 mL/min	141 (60.0)
Triple-class exposed^c, n (%)	248 (100)
Refractory status, n (%)
Any PI	197 (79.4)
Any immunomodulatory drugs	233 (94.0)
Any anti-CD38 mAb	229 (92.3)
Triple-class refractory^e	182 (73.4)
Penta-drug refractory^f	43 (17.3)
Refractory to last line of prior therapy, n (%)	230 (92.7)
Abbreviations: CD, cluster of differentiation; ECOG PS, Eastern Cooperative Oncology Group performance status; ISS, International Staging System; LOT, lines of therapy; mAb, monoclonal antibody; MM, multiple myeloma; PI, proteasome inhibitor; US, United States. ^aN=247. ^bScreening ECOG PS scores were 0 or 1 only. ^cBased on a median follow-up of 11 months. ^dN=217. ^ePI, immunomodulatory drug, and anti-CD38 mAB. ^f2 PIs, 2 immunomodulatory drugs and anti-CD38 mAb.

Antimyeloma SOC Treatments³

SOC Treatments, n (%)	N=248
Autologous stem cell transplant	6 (2.4)
Glucocorticoids	222 (89.5)
PI	134 (54.0)
Immunomodulatory drugs	118 (47.6)
Alkylating agents	107 (43.1)
Anti-CD38 mAbs	24 (9.7)
Anthracyclines	18 (7.3)
Topoisomerase inhibitor	16 (6.5)
Other antineoplastic agents	15 (6.0)
HDAC inhibitors	12 (4.8)
Anti-SLAMF7 mAbs	9 (3.6)
BCMA-targeted ADC	7 (2.8)
BCL-2 inhibitor	6 (2.4)
Mitotic inhibitor	2 (0.8)
SINE	2 (0.8)
Abbreviations: ADC, antibody-drug conjugate; BCL-2, B-cell lymphoma 2; BCMA, B-cell maturation antigen; CD, cluster of differentiation; HDAC, histone deacetylase; mAb, monoclonal antibody; PI, proteasome inhibitor; SINE, selective inhibitor of nuclear export; SLAM, signaling lymphocytic activation molecule; SOC, standard of care.

Efficacy - Final Analysis

The median duration of follow-up was 26.4 months (95% CI, 25.0-28.1).⁴

Index SOC Treatment

Efficacy outcomes after the index treatment with SOC regimen at a median follow-up of 26.4 months (95% CI, 25.0-28.1) are summarized in Table: Efficacy Outcomes of Patients on SOC Therapy (Final Analysis).

Efficacy Outcomes of Patients on SOC Therapy (Final Analysis)⁴

Parameter	Total (N=248)
ORR, % (95% CI)	31.9 (26.1-38.0)
Median PFS, months (95% CI)	4.6 (3.9-5.6)
12-month PFS rate, % (95% CI)	21 (15.3-27.3)
24-month PFS rate, % (95% CI)	10.5 (6.1-16.3)
Median OS, months (95% CI)	13.8 (10.8-17.0)
12-month OS rate, % (95% CI)	53.4 (46.7-59.6)
24-month OS rate, % (95% CI)	33.7 (27.3-40.2)
Median duration of treatment, months (range)	4.0 (0.1-33.6)
Median DOR, months (95% CI)	7.4 (4.9-11.1)
Abbreviations: CI, confidence interval; DOR, duration of response; ORR, overall response rate; OS, overall survival; PFS, progression-free survival.

Subsequent LOT

Overall, 152 patients (61.3%) received a subsequent LOT. A total of 78 patients had 1 subsequent LOT and 74 patients had ≥2 subsequent LOTs (1 LOT, 31%; 2 LOTs, 13%; 3 LOTs, 10%; >3 LOTs, 7%).⁴
The median progression-free survival on subsequent LOT (PFS2) by investigator assessment was 10.8 months (95% CI, 8.4-13.0). Only PFS events that occurred after starting the subsequent LOT was considered, and patients who withdrew before subsequent LOT 1 were censored.⁴
A total of 79 different regimens were used in subsequent LOT 1. The most common subsequent LOT regimens are detailed in Table: Treatment Regimens Used in Index and Subsequent LOT. The median duration of treatment was 2.8 months (range, <1-29.7).⁴
- The usage of B cell maturation antigen (BCMA)-targeted therapy increased from 2.8% in index LOT to 24.2% in the subsequent LOT.

Treatment Regimens Used in Index and Subsequent LOT⁴

LOT	No. of Regimens	No. of Patients	Most Common Regimens,^a,b%
Index LOT	91	248	Kd (14.1), CPd (14.1), Pd (11.7)
Subsequent LOT 1	79	152	blmf (6.5), CPd (4.0)
Subsequent LOT 2	47	74	blmf (5.6), KCd (1.6), Kd (1.6)
Subsequent LOT 3	30	42	blmf (3.2), IPd (2), DKd (0.8)
Subsequent LOT 4+	34	17	blmf (1.2)
Abbreviations: blmf, belantamab mafodotin; CPd, cyclophosphamide, pomalidomide, and dexamethasone; DKd, DARZALEX, carfilzomib, and dexamethasone; IPd, isatuximab, pomalidomide, and dexamethasone; KCd, carfilzomib, cyclophosphamide, and dexamethasone; Kd, carfilzomib and dexamethasone; Pd, pomalidomide and dexamethasone. ^aPatients can be counted in more than one regimen. ^bPercentage calculated out of N=248; for subsequent LOT, the percentage does not account for patients who died or were lost to follow-up.

Efficacy - Primary and Updated Analyses

The median duration of follow-up for the primary analysis was 11.0 months (range, 0.119.2) and that of the updated analysis was 16.1 months (range, 0.1-25.9).²^,³
Efficacy outcomes are summarized in Table: Efficacy Outcomes of Patients on SOC Therapy (Primary and Updated Analyses).

Efficacy Outcomes of Patients on SOC Therapy (Primary and Updated Analyses)²^,³

Parameter	Total (N=248)
Parameter	11.0 Month Follow-up	16.1-Month Follow-up
ORR, % (95% CI)	29.8 (24.2-36.0)	31.5 (25.7-37.6)
Triple class refractory at baseline^a	25.1 (19.0-32.1)	-
Not triple class refractory at baseline^b	43.1 (30.8-56.0)	-
Best response rate, %
sCR	0	0
CR	0.4	0.4
VGPR	12.1	12.9
PR	17.3	18.1
Minimal response	5.2	-
Stable disease	31.0	-
PD	18.5	-
NE^c	15.3	-
Median PFS, months (95% CI)	4.6 (3.9-5.6)	4.6 (3.9-5.6)
Patients not achieving VGPR	3.9 (3.4-4.6)	3.9 (3.4-4.6)
Patients achieving VGPR or better	NR (8.54-NE)	15.2 (13.3-NE)
Triple class refractory at baseline	3.9 (3.4-4.6)	-
Not triple class refractory at baseline	8.2 (5.7-12.0)	-
12-month PFS rate, % (95% CI)	19.9 (13.6-27.0)	-
Median PFS from the initiation of SOC through subsequent LOT (PFS2), months (95% CI)	-	9.9 (7.6-12.0)
Median OS, months (95% CI)	12.4 (10.28-NE)	13.8 (10.8-18.5)
Patients not achieving VGPR	10.9 (8.4-14.2)	10.9 (8.4-14.2)
Patients achieving VGPR or better	NE (NE-NE)	NE (17.9-NE)
Triple class refractory at baseline	11.1 (8.8-14.2)	-
Not triple class refractory at baseline	NE (12.4-NE)	-
12-month OS rate, % (95% CI)	51.8 (44.1-58.8)	-
Median DOR, months (95% CI)	7.4 (4.7-12.5)	7.7 (5.1-13.1)
Patients not achieving VGPR	4.5 (3.5-7.3)	-
Patients achieving VGPR or better	NE (7.7-NE)	-
Triple class refractory at baseline	4.5 (3.7-NE)	-
Not triple class refractory at baseline	9.1 (7.3-NE)	-
Median time to first response, months (range)	1.9 (0.7-9.5)	-
Median time to best response, months (range)	2.4 (0.7-12.2)	-
Abbreviations: CI, confidence interval; CR, complete response; DOR, duration of response; NE, not estimable; NR, not reached; ORR; overall response rate; OS, overall survival; PD, progressive disease; PFS, progression-free survival; SOC, standard of care; VGPR, very good partial response. ^aN=183. ^bN=65. ^cFourteen patients were NE due to death within 2 months after initiating SOC therapy; 12 patients were NE due to discontinuation or switching of SOC therapy.

Safety

Overall, at a median follow-up of 26.4 months, an AE was reported in 86.7% of patients during the index LOT. The most common AEs reported were cytopenias.⁴
- At a median follow-up of 16.1 months, treatment-emergent adverse events (TEAEs) had been reported in 85.9% of patients (n=213), of whom 56.5% (n=140) reported grade 3/4 TEAEs. The most common grade 3/4 TEAEs were hematologic; thrombocytopenia (18.5%), neutropenia (16.1%), and anemia (10.1%).³
- Non-hematologic TEAEs at a median follow-up of 11.01 months are summarized in
  Table: Non-hematologic TEAEs at a Median Follow-Up of 11.01 Months.
A second primary malignancy was reported in 13 patients (5.2%; during index LOT, n=5; after index LOT, n=8).⁴
Throughout the entire study period, 158 patients (63.7%) died, predominantly due to PD, regardless of the stage of treatment (during or after index LOT or after starting subsequent LOT).⁴

Non-hematologic TEAEs at a Median Follow-Up of 11.01 Months²

TEAE	Total (N=248)
TEAE	Any grade, n (%)^a	Grade 3/4, n (%)^a
Non-hematologic TEAEs^b
Infections and infestations	71 (28.6)	16 (6.5)
Nervous system disorders	49 (19.8)	8 (3.2)
General disorders and administration site conditions
Pyrexia	31 (12.5)	6 (2.4)
Fatigue	30 (12.1)	2 (0.8)
Asthenia	23 (9.3)	2 (1.2)
Peripheral edema	19 (7.7)	1 (0.4)
Gastrointestinal disorders
Diarrhea	38 (15.3)	2 (0.8)
Nausea	23 (9.3)	3 (1.2)
Constipation	14 (5.6)	0
Vomiting	14 (5.6)	2 (0.8)
Metabolism and nutrition disorders	31 (12.5)	9 (3.6)
Musculoskeletal and connective tissue disorders
Back pain	20 (8.1)	4 (1.6)
Arthralgia	15 (6.0)	3 (1.2)
Respiratory, thoracic, and mediastinal disorders
Dyspnea	28 (11.3)	6 (2.4)
Investigations	25 (10.1)	6 (2.4)
Psychiatric disorders	22 (8.9)	3 (1.2)
Renal and urinary disorders	22 (8.9)	13 (5.2)
Injury, poisoning, and procedural complications	21 (8.5)	6 (2.4)
Skin and subcutaneous tissue disorders	20 (8.1)	1 (0.4)
Cardiac disorders	18 (7.3)	9 (3.6)
Vascular disorders	18 (7.3)	7 (2.8)
Abbreviations: TEAE, treatment-emergent adverse event. ^aPercentages are calculated with the all-treated analysis set as denominator. ^bReported in ≥5% of patients.

LocoMMotion Subgroup Analysis

An analysis of efficacy outcomes stratified by treatment history, patient characteristics and disease characteristics was conducted in 248 patients participating in the LocoMMotion study at a median follow-up of 16 months.⁶

Results

Patient Characteristics

Overall, 248 patients were included. The duration of follow-up was 16 months, spanning a median of 4 cycles (range, 1-25) of SOC therapy.⁶

Efficacy

The ORR ranged from 11.8% to 43.9% across all subgroups.⁶
The characteristics associated with worse efficacy outcomes included triple class refractory, penta-drug exposed, penta-drug refractory, ECOG PS ≥1, poor renal function (creatinine clearance ≤60 mL/min), ISS stage II-III, presence of extramedullary plasmacytomas, high LDH (>245 U/L), bone marrow plasma cells ≥60%, and high-risk cytogenetics.⁶
Efficacy outcomes stratified by treatment history, patient characteristics, and disease characteristics are summarized in Table: Efficacy Outcomes by Patient Treatment History, Table: Efficacy Outcomes by Patient Characteristics, and Table: Efficacy Outcomes by Disease Characteristics, respectively.⁶

Efficacy Outcomes by Patients Treatment History⁶

Treatment History	N^a	Median OS, months (95% CI)	Median PFS, months (95% CI)	ORR, % (95% CI)	Median DOR^b, months (95% CI)
Overall	248	13.8 (10.8-18.5)	4.6 (3.9-5.6)	31.5 (25.7-37.6)	7.7 (5.1-13.1)
Number of LOTs
≤3	65	11.1 (7.4-15.1)	4.3 (3.1-6.0)	36.9 (25.3-49.8)	4.9 (3.4-NE)
≥4	183	15.9 (10.9-20.2)	4.9 (4.0-5.7)	29.5 (23.0-36.7)	9.0 (5.6-NE)
Triple-class refractory
Yes	182	11.1 (9.0-15.9)	4.1 (3.4-4.6)	26.9 (20.6-34.0)	5.1 (4.0-9.0)
No	66	NE (13.0-NE)	8.2 (5.7-12.9)	43.9 (31.7-56.7)	13.1 (7.7-NE)
Penta-drug exposed
Yes	112	11.6 (8.2-17.9)	3.9 (3.0-5.1)	27.7 (19.6-36.9)	4.9 (3.7-NE)
No	136	15.1 (11.1-NE)	5.6 (4.3-7.2)	34.6 (26.6-43.2)	11.1 (5.6-NE)
Penta-drug refractory
Yes	43	8.2 (5.7-13.0)	3.4 (2.1-4.5)	23.3 (11.8-38.6)	4.0 (1.2-NE)
No	205	15.5 (12.0-NE)	5.4 (4.2-6.0)	33.2 (26.8-40.1)	9.0 (5.1-17.8)
Abbreviations: CI, confidence interval; DOR, duration of response; LOTs, lines of therapy; NE, not estimable; ORR, overall response rate; OS, overall survival; PFS, progression-free survival. ^aN values correspond to ORR data. ^bIn responders (partial response or better).

Efficacy Outcomes by Patient Characteristics⁶

Patient Characteristics	N^a	Median OS, months (95% CI)	Median PFS, months (95% CI)	ORR, % (95% CI)	Median DOR^b, months (95% CI)
Overall	248	13.8 (10.8-18.5)	4.6 (3.9-5.6)	31.5 (25.7-37.6)	7.7 (5.1-13.1)
Age, years
<65	88	13.3 (8.9-NE)	4.4 (3.3-6.0)	34.1 (24.3-45.0)	11.1 (3.5-NE)
≥65	160	14.2 (10.3-18.5)	4.9 (3.7-5.7)	30.0 (23.0-37.7)	7.4 (5.0-13.1)
Baseline ECOG PS
0	65	NE (18.5-NE)	5.7 (3.7-6.7)	30.8 (19.9-43.4)	8.1 (4.5-NE)
≥1	183	11.1 (7.6-13.8)	4.4 (3.6-5.6)	31.7 (25.0-39.0)	7.7 (4.9-17.8)
Baseline renal function (creatinine clearance), mL/min
≤60	92	11.1 (7.6-NE)	4.4 (3.5-5.7)	31.5 (22.2-42.0)	5.6 (4.5-9.0)
>60	139	16.0 (11.8-NE)	5.1 (3.7-6.0)	33.1 (25.4-41.6)	13.1 (5.1-NE)
Abbreviations: CI, confidence interval; DOR, duration of response; ECOG PS, Eastern Cooperative Oncology Group performance status; NE, not estimable; ORR, overall response rate; OS, overall survival; PFS, progressionfree survival. ^aN values correspond to ORR data. ^bIn responders (partial response or better).

Efficacy Outcomes by Disease Characteristics⁶

Disease Characteristics	N^a	Median OS, months (95% CI)	Median PFS, months (95% CI)	ORR, % (95% CI)	Median DOR^b, months (95% CI)
Overall	248	13.8 (10.8-18.5)	4.6 (3.9-5.6)	31.5 (25.7-37.6)	7.7 (5.1-13.1)
Baseline ISS^c
I	46	NE (20.2-NE)	6.0 (4.9-9.4)	37.0 (23.2-52.5)	NE (4.7-NE)
II	38	8.4 (5.3-10.9)	3.9 (1.9-5.7)	21.1 (9.6-37.3)	5.1 (1.2-NE)
III	40	16.4 (5.4-NE)	5.1 (3.5-6.4)	35.0 (20.6-51.7)	9.5 (2.6-NE)
Presence of extramedullary plasmacytomas
Yes	26	6.3 (4.9-8.2)	2.3 (1.4-3.5)	23.1 (9.0-43.6)	11.1 (3.5-NE)
No	222	15.5 (12.0-20.2)	5.1 (4.3-6.0)	32.4 (26.3-39.0)	7.7 (4.9-13.1)
LDH, U/L
≤245	114	19.4 (13.3-NE)	5.6 (4.6-6.3)	36.0 (27.2-45.5)	5.6 (4.5-NE)
>245	79	8.1 (5.5-12.0)	3.4 (2.3-4.4)	26.6 (17.3-37.7)	9.5 (3.0-NE)
Bone marrow plasma cells, %
≤30	48	18.5 (12.4-NE)	4.9 (3.6-6.4)	29.2 (17.0-44.1)	NE (3.7-NE)
>30 to <60	11	10.9 (4.1-NE)	5.4 (0.8-6.3)	27.3 (6.0-61.0)	4.5 (4.5-NE)
≥60	17	7.6 (2.6-11.8)	4.4 (1.0-8.5)	11.8 (1.5-36.4)	NE (5.0-NE)
Cytogenetic risk^d
High	11	6.2 (1.9-9.0)	4.9 (1.0-6.4)	18.2 (2.3-51.8)	4.3 (4.0-NE)
Standard	29	18.5 (9.2-NE)	5.6 (2.0-9.9)	31.0 (15.3-50.8)	NE (4.7-NE)
Abbreviations: CI, confidence interval; del, deletion; DOR, duration of response; ISS, International Staging System; LDH, lactate dehydrogenase; NE, not estimable; ORR, overall response rate; OS, overall survival; PFS, progression-free survival; t, translocation. ^aN values correspond to ORR data. ^bIn responders (partial response or better). ^cISS staging is calculated at study entry based on laboratory parameters for albumin and β2-microglobulin. ^dMutations of t(4;14), t(14;16), or del17p13 were considered high-risk; cytogenetic assessments were performed at local laboratory.

Health-Related Quality of Life in patients in the locommotion study

An analysis of HRQoL was conducted in 248 patients in the LocoMMotion study.⁷

Study Design/Methods

HRQoL was assessed using the following PRO questionnaires⁷:
- European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 item (EORTC QLQ-C30)
- Four single items (ie, feeling restless or agitated, thinking about illness, worried about dying, worried about future health) from the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire myeloma module (EORTC QLQ-C30 MY20).
- EuroQol Five Dimension Five Level Questionnaire (EQ-5D-5L).
HRQoL was assessed at the following timepoints⁷:
- Baseline (defined as the closest value to day 1 of SOC treatment; screening visit or cycle 1 day 1 visit)
- Day 1 of each treatment cycle
- End of treatment visit
- During follow-up (every 4 weeks)

Results

Mean questionnaire response rate was approximately 78% during SOC treatment and approximately 71% during follow-up.⁷
In the overall population, the LS mean changes from baseline during SOC treatment showed minimal improvement in HRQoL, primarily worsening during subsequent treatment (see Table: LS Mean Changes in Scores From Baseline During SOC and Subsequent LOT).⁷
Patients who achieved ≥VGPR during SOC treatment had greater improvement in PRO scores, including LS mean change for pain score (see Table: LS Mean Changes in Scores from Baseline by Response).⁷
A meaningful improvement in PRO scores (defined by a literature-based minimally important difference of 10 points in mean score) was not achieved in most patients, especially for pain symptoms.⁷

LS Mean Changes in Scores From Baseline During SOC and Subsequent LOT⁷

Scale	LS mean change from baseline (95% CI)^a
Scale	During SOC treatment (n=173)	During subsequent treatment (n=99)
EORTC QLQ-C30
Physical functioning^b	0.7 (-2.0, 3.4)	-11.2 (-15.5, -6.8)
Global health status^b	2.9 (0.0, 5.9)^d	-4.6 (-8.3, -0.9)^e
Pain score^c	-2.4 (-6.3, 1.5)	1.0 (-3.5, 5.4)
Fatigue symptoms^c	-2.4 (-5.5, 0.7)	6.5 (2.5, 10.4)
EORTC QLQ-MY20
Restless or agitated^c	-5.1 (-8.8, -1.4)^d	3.5 (-1.1, 8.1)^e
Thinking about illness^b	9.0 (4.9, 13.0)^f	3.7 (-1.3, 8.8)^e
Worried about dying^b	4.8 (1.1, 8.4)^d	-4.2 (-10.1, 1.8)^e
Worried about health^b	10.3 (6.5, 14.2)^f	0.9 (-3.7, 5.6)
EQ-5D-5L
Visual analog scale^b	2.2 (-0.1, 4.6)^f	-4.0 (-7.5, -0.5)^g
Utility score^b	0.00 (-0.03, 0.02)^h	-0.04 (-0.07, 0.01)ⁱ
Abbreviations: EORTC QLQ-C30, European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 item; EORTC QLQ-C30 MY20, European Organization for Research and Treatment of Cancer Quality of Life Questionnaire myeloma module; EQ-5D-5L, EuroQol Five Dimension Five Level Questionnaire; LOT, lines of therapy; LS, least square; SOC, standard of care. ^aBolded scores indicate worsening. ^bHigher score indicates better outcome. ^cHigher score indicates worse outcome. ^dn=171. ^en=98. ^fn=172. ^gn=97. ^hn=170. ⁱn=95.

LS Mean Changes in Scores from Baseline by Response^a,⁷

Scale		LS mean change from baseline (95% CI)^a
Scale		≥VGPR (n=25)	PR (n=42)	NR (n=71)	PD (n=26)
EORTC QLQ-C30
Physical functioning^b		1.9 (-3.0, 6.7)	3.9 (-0.4, 8.3)	-2.8 (-8.3, 2.6)	-8.9 (-17.6, -0.3)
Global health status^b		6.4 (1.2, 11.7)	5.6 (0.5, 10.7)^d	-3.5 (-8.6, 1.7)^e	-7.2 (-14.8, 0.4)
Pain^c		-14.1 (-21.5, -6.7)	-2.2 (-8.7, 4.4)	2.3 (-3.6, 8.2)	8.5 (-3.0, 20.1)
Fatigue^c		-5.8 (-11.2, -0.5)	-0.7 (-5.8, 4.5)	-2.6 (-7.7, 2.5)	-1.8 (-9.2, 5.6)
EORTC QLQ-MY20
Restless or agitated^c		2.2 (-3.5, 8.0)	-5.4 (-11.8, 1.1)	-5.0 (-10.7, 0.6)^f	-6.6 (-19.8, 6.7)
Thinking about illness^b		10.1 (4.0, 16.2)	11.5 (4.8, 18.3)	7.1 (0.5, 13.6)^e	-0.5 (-12.9, 11.9)
Worried about dying^b		2.1 (-3.6, 7.8)	10.5 (4.1, 16.9)	-0.3 (-6.3, 5.7)^f	0.1 (-7.2, 7.5)
Worried about health^b		8.0 (0.8, 15.2)	13.3 (7.2, 19.4)	4.9 (-1.0, 10.9)^e	-0.1 (-12.2, 11.9)
EQ-5D-5L
	Visual analog scale^b	5.6 (1.2, 10.0)^g	5.7 (1.9, 9.5)	-1.3 (-5.1, 2.5)	-9.9 (-16.7, -3.0)
	Utility score^b	0.03 (-0.02, 0.08)^g	0.01 (-0.02, 0.04)	-0.01 (-0.05, 0.02)^f	-0.09 (-0.14, -0.03)
Abbreviations: EORTC QLQ-C30, European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 item; EORTC QLQ-C30 MY20, European Organization for Research and Treatment of Cancer Quality of Life Questionnaire myeloma module; EQ-5D-5L, EuroQol Five Dimension Five Level Questionnaire; LS, least square; NR, nonresponder; PD, progressive disease; PR, partial response; VGPR, very good partial response. ^aBolded scores indicate worsening. ^bHigher score indicates better outcome. ^cHigher score indicates worse outcome. ^dn=41. ^en=70. ^fn=69. ^fn=24. Note: Non-evaluable patients were excluded from analysis.

Literature Search

A literature search of MEDLINE^®, Embase^®, BIOSIS Previews^®, and Derwent Drug File (and/or other resources, including internal/external databases) was conducted on 02 July 2024.

References

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