CARVYKTI – Patient-Reported Outcomes

SUMMARY

• CARTITUDE-4 is a phase 3, open-label, multicenter study evaluating CARVYKTI vs standard care regimens, DARZALEX FASPRO (daratumumab and hyaluronidase), pomalidomide, and dexamethasone (DPd) or pomalidomide, bortezomib, and dexamethasone (PVd), in patients with relapsed/refractory multiple myeloma (RRMM) who received 1-3 prior lines of therapy (LOTs), including a proteasome inhibitor (PI) and an immunomodulatory drug, and are refractory to lenalidomide.^1-3
  • Einsele et al (2026)³ reported patient-reported outcomes (PROs) and treatment-free survival results from the CARTITUDE-4 study at a median follow-up of 33.6 months in both the CARVYKTI and standard care arms.
  • Bar et al (2025)⁴ reported PROs and time to next antimyeloma therapy at a median follow-up of 33.6 months in both the CARVYKTI and standard care arms.
  • Mina et al (2025)⁵ reported the PROs with the primary analysis at a median follow-up 15.9 months in both the CARVYKTI and standard care arms.
• CARTITUDE-1 was a phase 1b/2, open-label study evaluating the efficacy and safety of CARVYKTI in patients with RRMM after ≥3 prior LOTs including a PI, an immunomodulatory drug, and an anti-cluster of differentiation (CD) 38 monoclonal antibody (mAb).^6-9 
  • Cohen et al (2023)¹⁰ reported the expectations and experience of 36 patients treated with CARVYKTI through qualitative interviews.  
  • Martin et al (2022)¹¹ reported the health-related quality of life (HRQoL) secondary outcomes evaluated using PROs at a median follow-up of 16.9 months.

PRODUCT LABELING

• CARVYKTI (ciltacabtagene autoleucel) [Prescribing Information]. Horsham, PA: Janssen Biotech, Inc.; https://www.jnjlabels.com/package-insert/product-monograph/prescribing-information/CARVYKTI-pi.pdf.
• DARZALEX FASPRO (daratumumab and hyaluronidase-fihj) [Prescribing Information]. Horsham, PA: Janssen Biotech, Inc.; https://www.jnjlabels.com/package-insert/product-monograph/prescribing-information/DARZALEX+Faspro-pi.pdf.

CLINICAL DATA - CARTITUDE-4 PatIENT-REPORTED OUTCOMES

CARTITUDE-4 (NCT04181827) is a phase 3, randomized, open-label study evaluating the efficacy and safety of CARVYKTI vs standard care (physician's choice of DPd or PVd) in adult patients with lenalidomide-refractory multiple myeloma (MM) after 1-3 prior LOTs.^1-3

Einsele et al (2026)³ reported PROs and treatment-free survival results from the CARTITUDE-4 study at a median follow-up of 33.6 months (interquartile range [IQR], 20.3-35.0) in both the CARVYKTI and standard care arms.

Results

MySIm-Q Assessment

Time to Symptom Worsening

• At data cutoff, symptom worsening events based on the Multiple Myeloma Symptom and Impact Questionnaire (MySIm-Q) total symptom scale were reported in 30 patients in the CARVYKTI arm and 49 patients in the standard care arm.
• For the MySIm-Q time to symptom worsening analysis, the primary reasons for censoring are presented in Table: Reasons for Censoring in Time-To-Event Analyses.
• The median time to symptom worsening was not reached (NR; 95% confidence interval [CI], not estimable [NE]) in the CARVYKTI arm, and 34.3 months (95% CI, 32.2-NE) in the standard care arm (hazard ratio [HR], 0.38; 95% CI, 0.24-0.61; P<0.0001).
• Estimated 30-month MySIm-Q event-free rates were 76.8% (95% CI, 67.6-83.8) in patients receiving CARVYKTI and 63.1% (95% CI, 52.7-71.8) in those receiving standard care therapy.

Bar et al (2025)⁴ reported PROs and time to next antimyeloma therapy at a median follow-up of 33.6 months (range, 0.1-45.0) in both the CARVYKTI and standard care arms.

Results

PRO Compliance

• PRO compliance rates are presented in Figure: PRO Compliance Rates - Interim Analysis.
  • Primary reasons for noncompliance included mistakes, forgetfulness, site errors/oversights, unknown causes, unavailability of site staff, administrative failure, use of paper, tablet issues, and technical failure.⁴

MySIm-Q Total Symptom and Impact Scales

• From the MySIm-Q time to sustained worsening analysis, 86% of patients in the CARVYKTI arm and 77% of patients in the standard care arm were censored.⁴
  • The primary reasons for censoring were progressive disease (PD)/receipt of subsequent antimyeloma therapy (CARVYKTI, 32%; standard care, 64%) and study cutoff (CARVYKTI, 57%; standard care, 22%).
• The median time to symptom worsening was NR in the CARVYKTI arm and 34.3 months in the standard care arm.⁴
  • The event-free rate at 30 months was 77% in the CARVYKTI arm and 63% in the standard care arm.
• The median time to impact worsening was 39.2 months (95% CI, 38.7- NE) in the CARVYKTI arm and 35.9 months (95% CI, 32.2-NE) in the standard care arm (HR, 0.42; 95% CI, 0.26-0.70; P=0.0007).⁴
  • The event-free rate at 30 months was 83% in the CARVYKTI arm and 69% in the standard care arm.
• The least-squares (LS) mean change from baseline in the MySIm-Q total symptom and impact scores at month 30 are presented in Figure: LS Mean Change From Baseline in the MySIm-Q Total Symptom and Impact Scores at Month 30 - Interim Analysis.⁴

EORTC QLQ-C30 GHS/Quality of Life

• At month 30, a higher proportion of patients in the CARVYKTI arm compared to the standard care arm achieved clinically meaningful improvements in global health status (GHS), physical functioning, and fatigue and pain symptoms. Details are presented in Figure: CARTITUDE-4: Clinically Meaningful Improvement and LS Mean Change from Baseline on EORTC QLQ-C30 Scales at Month 30 - Interim Analysis.⁴

Time to Next Antimyeloma Therapy and Treatment-free Survival

• The median time to the next antimyeloma therapy was NR (95% CI, 38.4 months-NE) for CARVYKTI and 13.4 months (95% CI, 12.0-17.1) for standard care (HR [95% CI], 0.34 [0.26-0.46]; P<0.0001).⁴
• The median treatment-free survival was NR (95% CI, 36.6 months-NE) for CARVYKTI and 1.0 month (95% CI, 0.8-1.2) for standard care.⁴

Mina et al (2025)⁵ reported the PROs from the primary analysis at a median follow-up 15.9 months (IQR 12.4-17.8) in both the CARVYKTI and standard care arms.

Results

PRO Assessment

• At baseline PRO assessment, 208 patients in the CARVYKTI arm and 209 patients in the standard care arm were on study without disease progression and were not receiving subsequent therapy.⁵
  • PRO scores at baseline (cycle 1) are presented in Table: PRO Scores at Baseline.⁵
  • The number of patients who returned the baseline questionnaire assessment is summarized below.
    • European Organization for Research and Treatment of Cancer quality of life questionnaire core 30 (EORTC QLQ-C30): CARVYKTI, n=191 (92%); standard care, n=190 (91%).
    • EuroQuol 5-dimensional health-related quality of life (EQ-5D-5L): CARVYKTI, n=191 (92%); standard care, n=182 (87%).
    • MySIm-Q: CARVYKTI, n=191 (92%); standard care, n=183 (88%).
  • At month 12, compliance for all 3 questionnaires was 74% (107 returned of 145 expected) in the CARVYKTI arm and 81% (74 returned of 91 expected) in the standard care arm.
  • For most assessments conducted between months 3 and 9, compliance exceeded 75% in both arms and was consistent across all PRO questionnaires.
    • The primary reason for noncompliance was listed as “Other”, which included responses “mistake,” “forgot,” “site error/oversight,” “administrative failure,” “tablet issues,” “completed on paper,” and “unknown.”
    • The second most common reason for noncompliance was technical failure.

EORTC QLQ-C30 Assessment

• Improvements in scores were observed for GHS, functional scales, and symptom scales over time in the CARVYKTI arm compared to the standard care arm.^5,13  
  • The median time to improvement from baseline in the GHS score was 3.5 months
  • (IQR, 0.7-6.7) in the CARVYKTI arm and 2.0 months (IQR, 1.2-3.0) in the standard care arm.
  • At month 12, LS mean changes from baseline for GHS, functional scales, and symptom scales are presented in Table: LS Mean Changes from Baseline at Month 12.
  • Median time to improvement for GHS, physical functioning, fatigue, and pain is presented in Table: Time to improvement on EORTC QLQ-C30 Assessment.
• For clinically meaningful improvements, see Table: Proportion Of Patients With Clinically Meaningful Changes From Baseline (≥10-Point Difference) in EORTC QLQ-C30 at Month 12 - Primary Analysis.⁵

EQ-5D-5L VAS Assessment

• The median time to improvement in the visual analogue scale (VAS) score was 2.8 months (IQR, 0.4-7.1) in the CARVYKTI arm and 1.6 months (IQR, 1.1-2.9) in the standard care arm.⁵
• The mean change in the EQ-5D-5L VAS score worsened from baseline to month 3 and later improved over time in the CARVYKTI arm, with a 12-month LS mean increase of 8.0 points (95% CI, 5.2 to 10.7) from baseline; however, the standard care arm showed little to no changes, with a 12-month LS mean increase of 1.4 points (95% CI, -1.9 to 4.7).⁵
• For clinically meaningful improvements from baseline (≥7-point difference), see Table: Proportion of Patients With Clinically Meaningful Changes From Baseline in the VAS Score at Month 12 - Primary Analysis.⁵
• At baseline, fatigue, tiredness, or lack of energy was reported by 80% (152 of 191) of patients in the CARVYKTI arm and 76% (138 of 181) of patients in the standard care arm.⁵
  • At month 12, the rate of fatigue, tiredness, or lack of energy decreased to 68% (72 of 106) in the CARVYKTI arm but increased to 81% (58 of 72) of patients in the standard care arm.
  • The symptom interfered with daily activities in 52% (55 of 106) of patients in the CARVYKTI arm and 74% (53 of 72) of patients in the standard care arm.
  • No major changes occurred in reports of diarrhea, shortness of breath, headache, and dizziness. 

MySIm-Q Assessment

• Details related to censorship from MySIm-Q assessment are presented in Table: Censoring Details for MySIm-Q Assessment - Primary Analysis.⁵
• The median time to improvement on the total symptom scale and total impact scale is presented in Table: Time to Improvement on MySlm-Q Assessment.⁵
• The median time to sustained worsening of MM symptoms on the MySIm-Q was 23.7 months (95% CI, 22.1-NE) in the CARVYKTI arm and 18.9 months (95% CI, 16.8-NE) in the standard care arm (HR, 0.42; 95% CI, 0.26-0.68; nominal P=0.0003).⁵
• The proportion of patients with clinically meaningful changes from baseline (≥0.32-point difference) in symptom scores at month 12 is presented in Table: Proportion of Patients With Clinically Meaningful Changes From Baseline in Symptom Scores at Month 12 - Primary Analysis.⁵

CLINICAL DATA - cartitude-1 Patient-Reported OUTCOMEs

CARTITUDE-1 (NCT03548207) was a phase 1b/2, open-label study evaluating the efficacy and safety of CARVYKTI in patients with RRMM after ≥3 prior LOTs including a PI, an immunomodulatory drug, and an anti-CD38 mAb.^6-9

Cohen et al (2023)¹⁰ reported the expectations and experience of patients treated with CARVYKTI through qualitative interviews.

Results

After Treatment: Changes in Symptoms and HRQoL

• After CARVYKTI treatment, the percentage of patients reporting symptoms decreased.¹⁰
  • Decrease of most symptoms was noted in patients from day 100 to day 184 and is presented in Table: Symptoms Reported Before and After CARVYKTI Treatment.
• Improvements in HRQoL domains were observed in physical functioning, activities of daily life, and emotional/psychological functioning after CARVYKTI treatment. No significant improvement was observed in relationships and social functioning.¹⁰
  • Proportion of patients with improvements increased from day 100 to day 184 and is presented in Table: Patient-Reported Change in HRQoL Domains After CARVYKTI Treatment.
• Most patients found improvements in symptoms and HRQoL to be highly meaningful.¹⁰
  • Patients reported feeling more optimistic about the future and felt like they were able to make life plans and live more like a “normal” person.
  • Patients also valued a break from continuous treatment.
  • During the treatment-free period, patients experienced fewer side effects, greater independence, improved social functioning, and the opportunity to return to work.
• Negative experiences by patients were reported after CARVYKTI infusion due to extended hospital stays, fever, and pain from multiple bone marrow biopsies.¹⁰
• In interview 2, positive and negative sentiment descriptors were used by 7 patients and 8 patients, respectively, and 8 patients reported a mix of positive and negative experiences.¹⁰
• Among 22 patients who completed interviews 2 and 3, 10 patients reported no change in sentiment descriptors, 7 patients reported a negative change, and 5 patients reported a positive change.¹⁰
  • Negative changes were linked to chimeric antigen receptor T-cell therapy-related complications like onset of neurological side effects and gastrointestinal side effects and hospitalization due to fever.

After Treatment: Meeting Expectations and Comparison With Previous Treatments

• Majority of patients (>90%) reported their expectations with CARVYKTI treatment had been met or exceeded.¹⁰
  • Proportion of patients reporting CARVYKTI treatment expectations and comparison with previous treatment at day 100 and day 184 is presented in Table: Attainment of CARVYKTI Expectations and Comparison With Previous Treatment.
• Three patients reported their expectations were not met; at day 100, 2 patients reported treatment side effects as the reason and at day 184, 1 patient expected to feel “normal” but did not experience the outcome.¹⁰
• Patients compared few aspects of CARVYKTI treatment; effectiveness and administration were comparably better than previous treatment, whereas hospitalization and side effects were worse compared to previous treatment.¹⁰

Martin et al (2022)¹¹ reported the HRQoL secondary outcomes evaluated using PROs at a median follow-up of 16.9 months (IQR, 15.7-17.5).  

Results

PRO Assessment

• Improvements in EORTC QLQ-C30 GHS were observed over time compared to the baseline.¹¹
• On day 7, EORTC QLQ-C30 mean values decreased for GHS, physical functioning, role functioning, social functioning, fatigue, and nausea or vomiting, consistent with the potential onset of CARVYKTI adverse events associated with cytokine release.^11,14
  • The EORTC QLQ-C30 scores and changes from baseline by visit are summarized in Table: Summary of EORTC QLQ-C30: Value and Change From Baseline by Visit.
  • At day 100, clinically meaningful improvements calculated using Patient Global Impression of Change (PGIC) based anchoring were observed for pain, fatigue, physical functioning, and GHS and are presented in Table: Percentage of Patients Who Had Clinically Meaningful Improvements From Baseline to Day 100.
    • On PGIC itself, by day 28, 56, 78, and 100, improvements were reported by 35 (67%) of 52 patients, 40 (74%) of 54 patients, 42 (86%) of 49 patients, and 47 (87%) of 54 patients, respectively.
    • On day 100, 2 patients reported a change of worsening as per PGIC, and clinically meaningful worsening was not calculated for them.
• Improvements in the European Organization for Research and Treatment of Cancer multiple myeloma quality of life questionnaire (EORTC QLQ-MY20) were observed for future perspective scale and are summarized in Table: Summary of EORTC QLQ-MY20: Value and Change From Baseline by Visit.^11,14 
  • Clinically meaningful improvements achieved by patients from baseline to day 100 and day 464 are presented in Table: Percentage of Patients Who Had Clinically Meaningful Improvements From Baseline to Day 100 and Day 464.
  • On day 100 and day 464, meaningful worsening was reported by 0-6 (0%-12%) of 49 patients and 0-4 (0%-15%) of 26 patients, respectively.¹¹
• Improvements in EQ-5D-5L were observed for utility score and VAS and are summarized in Table: Summary of EQ-5D-5L: Value and Change From Baseline by Visit.^11,14
• Out of 68 patients, improvements and worsening in HRQoL were noticed in 30-42 (44%-62%) and 33-48 (49%-71%) patients, respectively.¹¹
  • Time to improvements and worsening occurred in 1-3 months and 1 week to 1 month, respectively, after CARVYKTI infusion. Incidence of improvement or worsening is summarized in Table: Time to First Event of Improvement or Worsening.

Literature Search

A literature search of MEDLINE^®, Embase^®, BIOSIS Previews^®, and Derwent Drug File databases (and/or other resources, including internal/external databases) was conducted on 02 September 2026.