KEY TAKEAWAYS
- The study aimed to investigate the patient-reported symptoms of CLL/SLL and MCL and assess their impact on patients through conceptually-selected EORTC Item Library items.
- Researchers noticed a significant potential in utilizing the EORTC Item Library to assess CLL/SLL- and MCL-related symptoms, with promising implications for clinical trial settings.
No dedicated measures exist for assessing patient-reported symptoms in chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) or mantle cell lymphoma (MCL). Jessica T Markowitz and her team aimed to uncover and understand CLL/SLL- and MCL-related symptoms, exploring their impact on patients’ lives. Through a qualitative and quantitative assessment of conceptually-selected EORTC Item Library items, the study seeks to identify promising measures for these rare blood and lymphatic cancers.
Researchers performed an inclusive analysis, commencing with a comprehensive literature review, clinician consultations, and patient interviews to identify and code concepts vital to patients. Cognitive debriefing of the selected library items was undertaken with patients. The study organized CLL/SLL and MCL-related symptoms and impacts into a structured conceptual model, subsequently mapped to item sets from the EORTC Item Library. The quantitative component involved exploratory macro-level Rasch measurement theory (RMT) analysis, offering supportive quantitative insights on the identified item sets.
About 41 participants (21-MCL; 20-CLL/SLL), along with 5 clinicians, engaged in the qualitative study. Subsequently, 57 unique patients (30-MCL; 27-CLL/SLL) completed the EORTC items. The conceptual models derived from the qualitative analysis delineated symptoms and functional impacts of CLL/SLL and MCL. Symptom domains encompassed swollen lymph nodes, B symptoms, abdominal issues, pain, fatigue, subjective cognitive impairment, anemia-related symptoms, bleeding, infection, and miscellaneous issues (appetite loss, temperature fluctuation, rash, weight gain, sleep problems, cough). Impact domains included physical function, role function, and other functions (psychological, social). Cognitive debriefing demonstrated that separate item sets for CLL/SLL and MCL-related symptoms were well understood and aligned with patients’ experiences. All selected items were encompassed in the conceptual models. In the exploratory RMT analysis, the item sets demonstrated sufficient coverage of the continuum of CLL/SLL- and MCL-related symptom severity.
The study concluded that the EORTC Item Library demonstrates qualitative and early quantitative evidence supporting its use in assessing CLL/SLL- and MCL-related symptoms and impacts. These items are promising candidates for measurement of patient-reported disease symptoms in these populations. However, a larger sample size will be essential to establish the necessary psychometric properties for clinical trial use. Patients suffering from rare blood, bone marrow, and lymph node cancers experience chronic and debilitating symptoms, yet dedicated instruments for assessing their experiences are currently lacking. The researchers selected relevant and clinically meaningful symptoms from the EORTC Item Library that assess fatigue, B symptoms, and CLL/SLL- and MCL-specific symptoms. Through patient and clinician interviews, along with quantitative analyses, the study revealed no major concerns with using these item sets to assess CLL/SLL and MCL symptoms. Interviews with patients indicated a clear understanding and alignment with their experiences. All selected items were included in the conceptual models, suggesting their potential use in assessing patient-reported symptom endpoints in clinical trial settings for these diseases.
The study is sponsored by Eli Lilly & Company
Source: https://pubmed.ncbi.nlm.nih.gov/38252198/
Markowitz JT, Mazerolle F, Lovell T, et.al (2024). Mixed-methods research to support the use of new lymphoma-specific patient-reported symptom measures derived from the EORTC item library. J Patient Rep Outcomes. 2024 Jan 22;8(1):8. doi: 10.1186/s41687-024-00683-2. PMID: 38252198; PMCID: PMC10803695.
