Chimeric Antigen Receptor T-cell Therapy in Hematologic Malignancies and Patient-reported Outcomes: A Scoping Review

Abstract

The inclusion of patient-reported outcome (PRO) measures in chimeric antigen receptor (CAR) T-cell therapy research is critical for understanding the impact of this novel approach from a unique patient standpoint. We performed a scoping review to map the available literature on the use of PRO measures in CAR T-cell therapy studies of patients with hematologic malignancies published between January 2015 and July 2022. Fourteen studies were identified, of which 7 (50%) were investigational early-phase trials, 6 (42.9%) were observational studies, and 1 (7.1%) was a pilot study. The EQ-5D and the PROMIS-29 were the 2 most frequently used PRO measures, being included in 6 (42.9%) and 5 (35.7%) studies, respectively. Despite differences in study designs, there seems to be evidence of improvements over time since CAR T-cell infusion in important domains such as physical functioning and fatigue, at least in patients who respond to therapy. Overall, the studies identified in our review have shown the added value of PRO assessment in CAR T-cell therapy research by providing novel information that complements the knowledge on safety and efficacy. However, there are several questions which remain to be answered in future research. For example, limited evidence exists regarding patient experience during important phases of the disease trajectory as only 4 (28.6%) and 5 (35.7%) studies provided information on PROs during the first 2 weeks from CAR T-cell infusion and after the first year, respectively. Time is ripe for a more systematic implementation of high-quality PRO assessment in future clinical trials and in real-life settings of patients treated with CAR T-cell therapy.

Protocol for a mixed-methods study to develop and feasibility test a digital system for the capture of patient-reported outcomes (PROs) in patients receiving chimeric antigen receptor T-cell (CAR-T) therapies (the PRO-CAR-T study)

“…10 11 While PRO data can help to ensure that patients' views about their treatment and side effects remain at the forefront of care, they are currently under-reported in clinical trials of CAR-T products and there is limited literature exploring the use of PROs for monitoring of CAR-T-related adverse events and quality of life in clinical settings, particularly in a UK context. 12 The field of precision cellular therapy is forecast to undergo rapid expansion in the coming decade and there is an urgent need for evidence-based tools, coproduced with patients, to better support the growing number of individuals with haematological malignancies who will receive these potentially curative treatments.…”

“…PROs may also be useful in clinical practice to inform decision-making around treatment and care, support communication between the patient and their healthcare team, and aid self-management 10 11. While PRO data can help to ensure that patients’ views about their treatment and side effects remain at the forefront of care, they are currently under-reported in clinical trials of CAR-T products and there is limited literature exploring the use of PROs for monitoring of CAR-T-related adverse events and quality of life in clinical settings, particularly in a UK context 12…”

“… 10 11 While PRO data can help to ensure that patients’ views about their treatment and side effects remain at the forefront of care, they are currently under-reported in clinical trials of CAR-T products and there is limited literature exploring the use of PROs for monitoring of CAR-T-related adverse events and quality of life in clinical settings, particularly in a UK context. 12 …”

Feasibility of collecting longitudinal patient-reported outcomes in individuals with relapsed or refractory large B-cell lymphoma who received chimeric antigen receptor T-cell (CART) therapy

“…They were unable to assess non-responders as these patients either succumbed to their disease or withdrew from the JULIET trial to pursue alternative therapies. A recent scoping review of 14 studies of PROs (HRQoL and patient-reported symptoms) in CART therapy indicates that after a decline in the early phase after infusion, there is a general improvement in PROs, at least in some domains in responding patients [15].…”

Patients’ experience with chimeric antigen receptor T-cell therapy for DLBCL in China: a qualitative study

“…Apart from together, CRS typically occurs within 2 weeks and ICANS usually occurs in a delayed form for 3~4 weeks after CAR T-cell infusion [ 9 ]. However, it was previously demonstrated that patients undergoing CAR T-cell therapy may experience long-term adverse reactions, such as memory disorder, disorientation, infection, or B-cell proliferation [ 16 ]. These long-term CAR T-cell therapy–related adverse effects seriously affect the patients’ quality of life.…”