Usage of Patient-Reported Outcomes in Blood Cancer Clinical Trials

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In recent years, there has been a growing interest in the use of patient-reported outcomes (PROs) in clinical trials. When it comes to evaluating subjective symptoms, patients themselves are often in the best position to determine how they feel. For this reason, PROs are considered to be the criterion standard for the assessment of subjective symptoms.¹

With the growing interest in PROs, a recent study published in JAMA Network Open reviewed 90 therapeutic randomized clinical trials (RCTs) of hematological malignant neoplasms to evaluate how often these outcomes were used as endpoints and whether they were being reported in primary trial publications.

“[…] there are sparse data about the use of PROs in RCTs for hematological malignant neoplasms,” the study authors wrote. “Given the significant morbidity associated with many blood cancers as well as the adoption of prolonged, non–fixed duration treatment in multiple hematological malignant neoplasms, PROs are critical to evaluate the effectiveness of drugs in this patient population.”

For the review, the authors utilized primary publications of therapeutic Phase III trials for adults with hematological malignant neoplasms. These were obtained from a search of 8 journals known for publishing high-impact RCTs—NEJM, Lancet, Lancet Hematology, Lancet Oncology, Journal of Clinical Oncology, Blood, JAMA, and JAMA Oncology—between January 1, 2018, and December 13, 2022. Data regarding PROs were extracted from manuscripts and the master protocols.

Following review, 90 RCTs were eligible for analysis. According to the results of the study, PROs were an endpoint in 66 (73%) trials, with one trial (1%) as a primary endpoint, in 50 (56%) as a secondary endpoint, and in 15 (17%) as an exploratory endpoint. PRO data were reported in 26 of 66 primary publications (39%): outcomes were unchanged in 18 and improved in eight, with none reporting worse PROs with experimental treatment.

“In this systematic review, almost 3 of every 4 therapeutic RCTs for blood cancers collected PRO data; however, only 1 RCT included PROs as a primary end point,” the authors wrote of the results. “Moreover, most did not report resulting PRO data in the primary publication and when reported, PROs were either better or unchanged, raising concern for publication bias. This analysis suggests a critical gap in dissemination of data on the lived experiences of patients enrolled in RCTs for hematological malignant neoplasms.”

One of the more notable trends in the results the authors noticed was a significant difference between blood cancer disease groups regarding the collection and reporting of PRO data. RCTs involving plasma cell disorders or multiple myeloma were significantly more likely to both collect and report PRO data. However, for RCTs of lymphoma, leukemia, and myelodysplastic syndromes, just over half of trials included PROs as an endpoint, and only a minority reported PRO results in their respective publications. The authors suggested a possible explanation for this contrast is the increased adoption of triplet and/or quadruplet regimens as standard-of-care for multiple myeloma treatment.

While there was a noticeable contrast in the reporting of PROs by blood cancer disease groups, the authors did not identify a significant variation in the collection or reporting of PROs based on the line of treatment.

“Our analysis suggests a critical gap in dissemination of data on the lived experiences of patients with hematological malignant neoplasms during phase III trials, which may be addressed with more inclusive PRO design and more stringent reporting requirements,” the authors concluded.

Reference

1. Patel K, Ivanov A, Jocelyn T, Hantel A, Garcia JS, Abel GA. Patient-Reported Outcomes in Phase 3 Clinical Trials for Blood Cancers: A Systematic Review. JAMA Netw Open. 2024;7(6):e2414425. doi:10.1001/jamanetworkopen.2024.14425