Quality of Life (QOL) Instruments Used in Clinicaltrials.gov-Indexed Trials of Gene-Modified Cell Therapy (GMCT) in Patients With Multiple Myeloma (MM)

OBJECTIVES: GMCT is emerging as an important treatment class that can prolong survival of patients with MM. The quality of the additional life-years is critical for patients, and QoL and related utilities are a key part of the health technology assessment approval process. This work examined the use of QoL instruments across trials indexed in clinicaltrials.gov evaluating GMCT in MM.

METHODS: Clinicaltrials.gov was searched in April 2023 for trials evaluating any type of gene therapy. Filters were applied to limit results to interventional studies and exclude suspended, withdrawn, and terminated studies. The results were screened to identify phase 2 and above trials reporting on GMCT for the treatment of MM.

RESULTS: Of 5,889 trials retrieved, 83 met the inclusion criteria of which 12 reported on QoL. Trials reporting QoL as an outcome were either international collaborations (n=9) or were conducted in the United States (n=2) or China (n=1). All trials were conducted in adults. Trial phases were 1/2, 2, or 3 (n=4 each) and most trials (n=7) had a single group assignment design. In total 7 different GMCT were assessed across studies. Outcomes included change from baseline in QoL measured by the following instruments: European Organization for Research and Treatment of Cancer Quality-of-life Questionnaire Core 30 (EORTC-QLQ-C30) (n=10); European Quality of Life Multiple Myeloma Module (EORTC-QLQ-MY20) (n=7), European Quality of Life-5 Dimensions-5 Levels (EQ-5D-5L) (n=9), Patient Global Impression of Symptom Severity (PGIS) Scale Score (n=3); Patient-reported Outcomes Version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE) (n=3); multiple myeloma symptom and impact questionnaire (MySIm-Q) (n=3).

CONCLUSIONS: Despite the importance of QoL outcomes, most trials evaluating GMCT in MM did not include QoL measurements and very few used MM-specific tools. Future trials should incorporate QoL tools to measure this important aspect of the impact of MM treatment on patients’ lives.