Are we using the relevant questionnaires to assess health related quality of life in multiple myeloma patients in the era of new generation targeted and novel immunotherapies?

Therapeutic advances have significantly improved the survival of multiple myeloma (MM) patients. Clinicians and researchers should ensure that patients with this incurable cancer are not only living longer, but also living better. Several validated questionnaires are available and frequently used to assess health related quality of life (HRQOL). However, they were developed >20 years ago before the era of new generation treatments, which have unique toxicity profiles. Introduction of new treatment combinations and maintenance therapies also means that these questionnaires may not be able to fully capture all HRQOL aspects. To better understand which of these aspects are truly important, we asked MM patients and healthcare professionals (HCP) from a single centre to indicate the level of relevance of each item in 9 HRQOL questionnaires. Our results revealed that patients ranked items related to social functioning, receiving information about MM and involvement in their management as most relevant. In contrast, HCPs placed highest relevance to items corresponding to symptoms from MM or treatment toxicities. Subgroup analysis of patients revealed additional topics that were deemed relevant: those ≤70 y.o. were more concerned about disease progression whereas those >70 y.o. prioritised items related to physical function; patients with renal impairment and bone disease found having energy and pain more relevant than in the general MM cohort. These findings can contribute to guiding the content development of new HRQOL tools relevant to both MM patients as a whole, as well as specific subgroups receiving newer treatments.

治疗领域的进步已显著改善了多发性骨髓瘤（multiple myeloma, MM）患者的生存结局。临床医师与科研人员应确保这类不可治愈的恶性肿瘤患者不仅能够获得更长的生存期，同时也能拥有更高的生活质量。目前已有多款经过验证的问卷被广泛用于评估患者的健康相关生存质量（health related quality of life, HRQOL），但此类问卷均开发于20余年前，早于新一代治疗药物问世的时代——这类新型治疗具有独特的毒性谱。随着新型联合治疗方案与维持疗法的临床应用，现有HRQOL问卷或已无法全面覆盖所有相关维度。为明确哪些HRQOL维度才是真正具有临床意义的核心要素，本研究招募了单中心的MM患者与医疗保健专业人员（healthcare professionals, HCP），请他们对9款HRQOL问卷中的每个条目进行相关性评分。研究结果显示，患者群体将社会功能、获取MM相关信息以及参与自身治疗管理相关的条目评为最具相关性的内容；与之相对，HCP则最为看重与MM症状或治疗毒性相关的条目。进一步的患者亚组分析还揭示了额外的关注差异：年龄≤70岁的患者更担忧疾病进展，而年龄＞70岁的患者则优先关注躯体功能相关条目；合并肾功能损害与骨骼疾病的患者，相较于整体MM队列，更看重精力与疼痛相关的条目。本研究结果可为面向全体MM患者以及接受新型治疗方案的特定亚群的新型HRQOL评估工具的内容开发提供科学指导依据。