Lisocabtagene maraleucel as a second-line treatment for R/R LBCL: PROs from the PILOT study

Results from the phase II PILOT trial (NCT03483103), which were previously covered by the Lymphoma Hub, demonstrated the effectiveness of lisocabtagene maraleucel (liso-cel) as a second-line therapy for patients with relapsed/refractory (R/R) large B-cell lymphoma (LBCL) not intended for hematopoietic stem cell transplantation.¹ Briefly, liso-cel was associated with an overall response rate and complete response rate of 80% and 54%, respectively.¹ Results from this trial, in addition to results from the phase III TRANSFORM trial (NCT03575351), led to the approval of liso-cel for patients with R/R LBCL.

Here, we summarize a health-related quality of life (HRQoL) and patient-reported outcomes (PROs) analysis from the PILOT trial, published by Gordon et al.¹ in Haematologica.

Study design and patient population¹

• The study design and patient characteristics were previously reported by the Lymphoma Hub.
• HRQoL was a secondary endpoint, and was assessed using three PRO instruments:
  • European Organization for Research and Treatment of Cancer Quality of Life Questionnaire-30 items (EORTC QLQ-C30)
  • Functional Assessment of Cancer Therapy-Lymphoma “Additional Concerns” Subscale (FACT-LymS)
  • EuroQoL 5 Dimension 5 Level (EQ-5D-5L).
• PROs were assessed at screening (baseline), before treatment (≤7 days before lymphodepletion), preinfusion on the day of liso-cel infusion (Day 1), after treatment on Days 29, 60, 90, 180, 270, 365, 545, and 730 (end of study), and at disease progression.
  • Final PROs were recorded from patients who discontinued the study early.

Key findings¹

• Of 61 patients included in the study, 56, 49, 55, and 54 were evaluable for the EORTC QLQ-C30, FACT-LymS, EQ-5D-5L health utility index, and EQ-5D-5L visual analog scale measurements, respectively.

HRQoL at baseline

• When compared with reference age and matched general populations, baseline mean HRQoL scores were slightly worse across most domains, with clinically meaningful worse scores in the EORTC QLQ-C30 fatigue, social functioning, and appetite loss domains.

Within-group changes in HRQoL scores

• Following transient worsening at Day 1 or Day 29, improvements in least squares mean HRQoL scores were generally observed for the primary domains of interest.
• Overall least squares mean changes from baseline through Day 545 are shown in Table 1.

Table 1. Overall least squares mean changes from baseline to Day 545*

Within-patient changes in HRQoL scores

• Most patients showed an improvement or no change in EORTC QLQ-C30 primary domains of interest and FACT-LymS scores from baseline throughout treatment (Figure 1).

Figure 1. Within-patient analysis of changes from baseline for EORTC QLQ-C30 domains of A GH/QoL, B physical functioning, C role functioning, D cognitive functioning, E fatigue, F pain, and for G FACT-LymS* 

EORTC QLQ-C30, European Organization for Research and Treatment of Cancer Quality of Life Questionnaire-30 items; FACT-LymS, Functional Assessment of Cancer Therapy-Lymphoma “Additional Concerns” Subscale; GH, global health; QoL, quality of life.
*Adapted from Gordon, et al.¹