Development of a conceptual framework for an electronic patient-reported outcome (ePRO) system measuring symptoms and impacts of CAR-T cellular therapies in patients with haematological malignancies: A rapid review

Chimeric antigen receptor T-cell (CAR-T) therapy is associated with potentially severe toxicities that pose significant burden. Patient-reported outcomes (PROs) offer valuable insights into symptoms, functioning and other complex constructs of interest. This rapid review aimed to: 1) identify symptom and impact concepts important to CAR-T patients; 2) construct a conceptual framework for an electronic patient-reported outcome (ePRO) system; and 3) identify timepoints to capture PRO data for CAR-T cell therapies. We searched MEDLINE (OVID) and Web of Science (Clarivate) for English articles published from 2017 to Mar 2, 2023. No restrictions on study design were applied. 178 symptoms/constructs were extracted from 44 articles reporting PRO collection in adults with hematologic malignancies receiving CAR-T cell therapy. Six healthcare professionals and 11 patients and caregiver partners verified construct relevance to clinical management and lived experience respectively. 109 constructs were sorted according to the four domains of conceptual framework: Symptom Burden, Impact of Disease and Treatment, Tolerability, and Health-Related Quality of Life. The identification of concepts beyond symptom burden underscores the importance of PRO measurement for long-term monitoring, to align outcomes with patient concerns. The framework will facilitate PRO measure selection for systematic gathering of PROs from individuals with haematological malignancy receiving CAR-T cell therapies.