Taarnhøj Gry Assam, Lindberg Henriette, Johansen Christoffer, Pappot Helle
Department of Oncology, University Hospital of Copenhagen, Rigshospitalet, Blegdamsvej 9, 2100, Copenhagen, Denmark.
Department of Oncology, University Hospital of Copenhagen, Herlev Hospital, Herlev, Denmark.
J Patient Rep Outcomes. 2019 Aug 22;3(1):56. doi: 10.1186/s41687-019-0141-2.
Selection of specific patient-reported outcomes (PROs) for cancer patients requires careful consideration to the purpose and population at aim. Here we report the process of choosing which items to include in a bladder cancer population in chemo- or immunotherapy based on the Patient-Reported Outcomes Version of the Common Terminology Criteria of Adverse Events (PRO-CTCAE).
Initial PRO-CTCAE symptoms were chosen through 1) medical record audit 2) patient interviews 3) summary of product characteristics from European Medicines Agency and Food and Drug Administration for the applied chemotherapies, and 4) toxicity reporting from Phase 2 and 3 trials for immunotherapies applied in patients with urothelial cancer. The selected questions were applied in a prospective cohort of 78 bladder cancer patients receiving chemo- or immunotherapy at Rigshospitalet and Herlev Hospital, Denmark. Symptoms tested in this population were selected for the final module if they appeared in ≥3 of the following groupings a) the most prevalent PRO-CTCAE symptoms grade ≥ 2 overall during treatment b) the PRO-CTCAE symptoms reported in conjunction with hospital admissions or mentioned in focus group interviews discussing which symptoms were prevalent in this patient group with specialized c) nurses or d) physicians. The authors also included symptoms in the final module if they were present in two of the above groups and defined as actionable by clinicians.
From the initial selection of PRO-CTCAE symptoms, a total of 45 PRO-CTCAE symptoms explored by 84 PRO-CTCAE questions were retrieved. Through the second selection process based on the described criteria, the study group agreed on 15 PRO-CTCAE symptoms explored by 30 PRO-CTCAE items to be appropriate and relevant for the bladder population during medical oncological treatment.
The selection of disease specific PROs in a bladder cancer population was feasible. The process revealed several steps of selection needed in order to reach a final module for clinical application.
为癌症患者选择特定的患者报告结局（PRO）需要仔细考虑其目的和目标人群。在此，我们报告了基于不良事件通用术语标准的患者报告结局版本（PRO-CTCAE），选择哪些项目纳入膀胱癌化疗或免疫治疗人群的过程。
通过以下方式选择初始PRO-CTCAE症状：1）病历审核；2）患者访谈；3）欧洲药品管理局和美国食品药品监督管理局针对所用化疗药物的产品特征总结；4）尿路上皮癌患者免疫治疗2期和3期试验的毒性报告。将所选问题应用于丹麦里格霍斯医院和赫勒夫医院接受化疗或免疫治疗的78例膀胱癌患者的前瞻性队列中。如果以下分组中至少有3组出现这些症状，则将在该人群中测试的症状选入最终模块：a）治疗期间总体上最常见的PRO-CTCAE症状，分级≥2级；b）与住院相关报告的PRO-CTCAE症状，或在讨论该患者群体中哪些症状普遍存在的焦点小组访谈中提及的症状，这些访谈由专业的c）护士或d）医生进行。如果症状出现在上述两组中且被临床医生定义为可采取行动的症状，作者也将其纳入最终模块。
从最初选择的PRO-CTCAE症状中，共检索到84个PRO-CTCAE问题所探究的45个PRO-CTCAE症状。通过基于上述标准的第二轮选择过程，研究小组一致认为，30个PRO-CTCAE项目所探究的15个PRO-CTCAE症状适用于膀胱癌人群的医学肿瘤治疗，且与之相关。
在膀胱癌人群中选择疾病特异性PRO是可行的。该过程揭示了为达到临床应用的最终模块所需的几个选择步骤。
