Blade Joan, Calleja Miguel Ángel, Lahuerta Juan José, Poveda José Luis, de Paz Héctor David, Lizán Luis
Hematology Department, Hospital Clinic, Institut d'Investigacions Biomédiques August Pi I Sunyer (IDIBAPS), Barcelona, Spain.
Pharmacy Department, Hospital Virgen Macarena, Sevilla, Spain.
BMJ Open. 2018 Feb 22;8(2):e018850. doi: 10.1136/bmjopen-2017-018850.
To define a standard set of outcomes and the most appropriate instruments to measure them for managing newly diagnosed patients with multiple myeloma (MM).
A literature review and five discussion groups facilitated the design of two-round Delphi questionnaire. Delphi panellists (haematologists, hospital pharmacists and patients) were identified by the scientific committee, the Spanish Program of Haematology Treatments Foundation, the Spanish Society of Hospital Pharmacies and the Spanish Community of Patients with MM. Panellist's perception about outcomes' suitability and feasibility of use was assessed on a seven-point Likert scale. Consensus was reached when at least 75% of the respondents reached agreement or disagreement. A scientific committee led the project.
Fifty-one and 45 panellists participated in the first and second Delphi rounds, respectively. Consensus was reached to use overall survival, progression-free survival, minimal residual disease and treatment response to assess survival and disease control. Panellists agreed to measure health-related quality of life, pain, performance status, fatigue, psychosocial status, symptoms, self-perception on body image, sexuality and preferences/satisfaction. However, panellist did not reach consensus about the feasibility of assessing in routine practice psychosocial status, symptoms, self-perception on body image and sexuality. Consensus was reached to collect patient-reported outcomes through the European Organisation for the Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire (QLQ) Core questionnaire 30 (C30), three items from EORTC-QLQ-Multiple Myeloma (MY20) and EORTC-QLQ-Breast Cancer (BR23), pain Visual Analogue Scale, Morisky-Green and ad hoc questions about patients' preferences/satisfaction.
A consensual standard set of outcomes for managing newly diagnosed patients with MM has been defined. The feasibility of its implementation in routine practice will be assessed in a future pilot study.
定义一套标准的结局指标以及用于管理新诊断的多发性骨髓瘤（MM）患者时测量这些指标的最合适工具。
通过文献综述和五个讨论小组推动两轮德尔菲问卷的设计。德尔菲专家小组成员（血液科医生、医院药剂师和患者）由科学委员会、西班牙血液学治疗计划基金会、西班牙医院药剂师协会和西班牙MM患者团体确定。专家小组成员对结局指标的适用性和使用可行性按照七点李克特量表进行评估。当至少75%的受访者达成一致同意或不同意时即达成共识。一个科学委员会领导该项目。
分别有51名和45名专家小组成员参与了第一轮和第二轮德尔菲调查。就使用总生存期、无进展生存期、微小残留病和治疗反应来评估生存和疾病控制达成了共识。专家小组成员同意测量与健康相关的生活质量、疼痛、体能状态、疲劳、心理社会状态、症状、身体形象自我认知、性功能以及偏好/满意度。然而，专家小组未就常规实践中评估心理社会状态、症状、身体形象自我认知和性功能的可行性达成共识。就通过欧洲癌症研究与治疗组织（EORTC）生活质量问卷（QLQ）核心问卷30（C30）、EORTC-QLQ-多发性骨髓瘤（MY20）中的三个条目、EORTC-QLQ-乳腺癌（BR23）、疼痛视觉模拟量表、Morisky-Green问卷以及关于患者偏好/满意度的特设问题来收集患者报告结局达成了共识。
已定义了一套用于管理新诊断MM患者的经共识的标准结局指标集。其在常规实践中实施的可行性将在未来的一项试点研究中进行评估。
