Patients With Blood Cancers Rate Use of Common ePRO Measures Used in Trials

Non-Hodgkin lymphoma (NHL) is a heterogeneous group of blood cancers, with diffuse large B-cell lymphoma (DLBCL) and mantle cell lymphoma (MCL) among its subtypes. Although treatment advances have improved survival, many patients continue to experience poor health-related quality of life, and the qualitative literature on how NHL affects patients' daily lives remains limited.

The need for more information in this area is growing: an estimated 80,350 new cases of NHL were diagnosed and 19,390 deaths were attributable to the disease in the United States.¹

A study funded Sanofi and conducted by researchers from Evidera Health Economics and Sanofi aimed to characterize the symptoms and impacts of diffuse large B-cell lymphoma (DLBCL) and mantle cell lymphoma (MCL) from patients' own perspectives and to evaluate 4 patient-reported outcome (PRO) measures as potential clinical trial endpoints: the EORTC QLQ-C30, EORTC QLQ-NHL-HG29, EORTC QLQ-NHL-LG20, and FACT-Lym.²

“These findings help researchers and doctors understand the importance of patient experiences with non-Hodgkin lymphoma and support the use of appropriate health questionnaires in future studies,” the authors wrote.

Between June and September 2023, researchers conducted 60-minute qualitative interviews with 30 adults in the United States with clinician-confirmed DLBCL (n = 20) or MCL (n = 10) who were not receiving active treatment. The average age of participants was 57.8 years, and the sample was nearly evenly split between men and women. Interviews combined open-ended “concept elicitation”questions about symptoms and life impacts with a “cognitive debriefing” exercise, in which participants read aloud and reacted to items from the 4 PRO questionnaires. The transcripts were coded using a structured dictionary, with inter-coder agreement of 81.7%, exceeding the study's 80% threshold. A preliminary conceptual model, built from an earlier literature review of 15 qualitative studies, was updated with findings from these interviews.

The participants reported 58 distinct signs and symptoms in total. The most common were fatigue (93% overall), tiredness (87%), body aches or pain (80%), night sweats (80%), and headache or migraine (70%). Fatigue, tiredness, body aches, night sweats, lethargy, headache, appetite loss, altered taste, and weakness were both frequently reported and rated highly bothersome, with exhaustion and weakness receiving the highest average disturbance ratings (8 out of 10). Notably, exhaustion was reported predominantly by patients with MCL patients while weakness was more common among patients with DLBCL. Some symptoms, such as body aches and fatigue, arose spontaneously during interviews, suggesting strong salience, whereas others, like lethargy and weight loss, typically emerged only after direct probing.

On the impact side, participants described 64 distinct effects of NHL on their lives. The most frequently reported were limitations on outdoor activities (93%) and indoor activities (90%), decreased physical performance (90%), worry about the future (83%), and sadness or depression (77%). Emotional and psychological impacts, which included distress, fear of recurrence, and emotional exhaustion, were common but tended to surface only when participants were specifically probed, suggesting these experiences may be harder for patients to volunteer spontaneously. Patients with MCL uniquely emphasized difficulties with self-care and the need for support from family and friends as highly disturbing, while patients with DLBCL more often cited fear of recurrence, uncertainty about outcomes, and hair loss as top concerns. These findings fed into an expanded conceptual model that added 12 new symptoms for DLBCL, 11 for MCL, and 26 new quality-of-life impacts.

In the cognitive debriefing portion, participants generally responded favorably to all 4 PRO instruments. About 79% held a positive overall opinion of the EORTC QLQ-C30, and all who responded (100%) felt it adequately captured their NHL experience. Similarly strong endorsement was found for the EORTC QLQ-NHL-HG29 (75% positive, 100% comprehensive) and FACT-Lym (71% positive, 92% comprehensive). The EORTC QLQ-NHL-LG20 received more mixed feedback: 67% positive, with some participants noting certain items felt irrelevant to them, although all respondents ultimately agreed it covered their experience comprehensively. Across measures, participants generally found instructions, recall periods, and response options clear and easy to use.

The authors note several limitations. The sample was drawn only from the United States, skewed younger than the typical NHL diagnosis age of 68, and included fewer Asian participants and more women than population norms, which may limit generalizability. Detailed treatment histories were not systematically collected, making it difficult to fully separate disease-related symptoms from possible lingering treatment effects. The MCL subgroup was too small for formal saturation analysis, though the authors report the data collected were nonetheless comprehensive.

The study concludes that fatigue, pain, and sleep-related symptoms alongside restricted activity and emotional distress represent the core burden of NHL from patients' own perspectives, and that all 4 PRO instruments tested are viewed favorably by patients and appear relevant and comprehensive for capturing these experiences. The authors state that the resulting conceptual model and PRO evaluation offer a practical framework to help researchers select or refine meaningful endpoints for future NHL clinical trials.

“While these findings support the potential utility of these instruments in NHL clinical trials,” they concluded, “additional validation will be necessary to confirm their fit for purpose application and appropriate implementation in clinical trials.”²

References

1. Siegel RL, Kratzer TB, Giaquinto AN, Sung H, Jemal A. Cancer statistics, 2025. CA Cancer J Clin. 2025;75:10–45. https://doi.org/10.3322/caac.21871

2. Clark A, Van Tomme S, Hetherington L, Dias Barbosa C, Cordero P. Patient perspectives on non-Hodgkin lymphoma: A qualitative study to guide selection of clinical trial end points. Curr Oncol. 2026;33(7):427. doi:10.3390/curroncol33070427.