Hester Beke, Tresckow Julia Von, Voigtländer Minna, Beckmann Helen, Rusch Judith, Blome Christine
Institute for Health Services Research in Dermatology and Nursing (IVDP), University Medical Center Hamburg-Eppendorf (UKE), Martinistraße 52, 20246, Hamburg, Germany.
Clinic for Hematology and Stem Cell Transplantation, West German Cancer Centre (WTZ), University Hospital Essen, Essen, Germany.
J Patient Rep Outcomes. 2025 Apr 29;9(1):45. doi: 10.1186/s41687-025-00882-5.
Chronic lymphocytic leukaemia (CLL) is the most common form of leukaemia in adults in western countries. Asymptomatic patients are under clinical observation; when indication for treatment according to guidelines is met, treatment is initiated. When choosing from the numerous new treatment options, individual patient needs should be considered. To date, no instrument exists to capture these needs.
The ePROM was developed based on the Patient Benefit Index (PBI) methodology which captures the importance of treatment goals as well as the achievement of these goals. The development considered the COSMIN guidelines and included semi-structured interviews with 28 patients with CLL and free-text questionnaires (n = 15). Data were analysed via qualitative content analysis according to Kuckartz. The PBI-CLL was finalised through an expert consensus and cognitive debriefing interviews with 14 patients.
The content elicitation showed that the individual treatment burden and treatment goals in CLL vary considerably between patients, underlining the heterogeneity of this patient group. Patients reported disease burden in their physical constitution as well as mental burden. Many patients' main goal was to live normally and with the lowest impact possible through the CLL and its therapy. The PBI-CLL developed based on these data consists of three modules: therapy outcomes, process quality and relative treatment preferences. The cognitive debriefing interviews showed that patients find the instrument relevant, comprehensive, and comprehensible.
The PBI-CLL is the first instrument to assess patients' needs and benefits in CLL. The heterogeneity we found in patient needs and preferences underlines the importance of a modular instrument which measures treatment goals and benefits in a standardized way. The PBI-CLL shall support both patient-centred therapeutic decision making and treatment evaluation in clinical practice, as well as patient-centred benefit assessment in clinical and health care research. It should therefore be tested for its psychometric properties in a subsequent validation study.
慢性淋巴细胞白血病（CLL）是西方国家成年人中最常见的白血病形式。无症状患者处于临床观察中；当符合指南的治疗指征时，即开始治疗。在众多新的治疗选择中进行选择时，应考虑个体患者的需求。迄今为止，尚无工具来获取这些需求。
电子患者报告结局量表（ePROM）是基于患者获益指数（PBI）方法开发的，该方法可获取治疗目标的重要性以及这些目标的实现情况。开发过程遵循国际健康结局测量工具的标准制定和评价（COSMIN）指南，包括对28例CLL患者进行半结构化访谈以及自由文本问卷调查（n = 15）。根据库卡茨的方法通过定性内容分析对数据进行分析。PBI-CLL通过专家共识和对14例患者的认知反馈访谈得以最终确定。
内容引出显示，CLL患者的个体治疗负担和治疗目标差异很大，突出了该患者群体的异质性。患者报告了身体状况方面的疾病负担以及心理负担。许多患者的主要目标是正常生活，且CLL及其治疗对生活的影响尽可能小。基于这些数据开发的PBI-CLL由三个模块组成：治疗结果、过程质量和相对治疗偏好。认知反馈访谈表明，患者认为该工具相关、全面且易于理解。
PBI-CLL是首个评估CLL患者需求和获益的工具。我们在患者需求和偏好中发现的异质性突出了模块化工具的重要性，该工具以标准化方式衡量治疗目标和获益。PBI-CLL应支持临床实践中以患者为中心的治疗决策和治疗评估，以及临床和卫生保健研究中以患者为中心的获益评估。因此，应在后续的验证研究中对其心理测量特性进行测试。
