Health Outcomes Solutions, Winter Park, Florida.
Department of Dermatology, University of California, San Francisco.
JAMA Dermatol. 2014 Feb;150(2):169-76. doi: 10.1001/jamadermatol.2013.5870.
IMPORTANCE Little is known about patients' experiences of advanced basal cell carcinoma (aBCC) and basal cell carcinoma nevus syndrome (BCCNS), a rare genetic disorder that greatly increases the number of BCCs. OBJECTIVE To develop a questionnaire to measure patient-reported outcomes (PROs) in these populations. DESIGN, SETTING, AND PARTICIPANTS Concept elicitation interviews were conducted with patients with aBCC and BCCNS from 5 US clinical sites and the BCCNS Life Support Network and 4 physicians. The PRO questionnaires were drafted based on results from a literature review and findings from these interviews. Questionnaires were finalized after cognitive debriefing interviews were conducted with patients. Concept elicitation interviews were conducted with 30 patients (14 with aBCC, 16 with BCCNS) and 4 physicians (2 dermatologists, 1 Mohs surgeon, and 1 oncologist) in the United States. A subset of 10 of these patients (5 with aBCC, 5 with BCCNS) took part in cognitive debriefing interviews. MAIN OUTCOMES AND MEASURES Development of 2 questionnaires to allow clinicians to assess the emotional, social, and physical impacts of the disease on patients with aBCC and BCCNS. RESULTS Most concept elicitation interview patients were male (63%) and white (93%); their mean age was 57 years. There were impacts on emotional, social, and physical functioning in both conditions. Patients were unable to do many activities and avoided other activities. Seventy-nine percent of patients with aBCC and all patients with BCCNS reported scarring. Physician interviews revealed similar findings. During cognitive debriefing interviews, the questionnaires were found to be relevant, clear, and comprehensive. CONCLUSIONS AND RELEVANCE Advanced BCC and BCCNS affect patients in unique and substantial ways. These PRO questionnaires were developed with patient and clinician input and measure the key areas that have an impact on patients with these conditions.
人们对患有晚期基底细胞癌（aBCC）和基底细胞癌痣综合征（BCCNS）患者的体验知之甚少，BCCNS 是一种罕见的遗传疾病，会大大增加基底细胞癌的数量。目的：开发一种问卷来衡量这些人群的患者报告结局（PROs）。设计、地点和参与者：从美国的 5 个临床地点和 BCCNS 生活支持网络以及 4 名医生那里对患有 aBCC 和 BCCNS 的患者进行了概念启发访谈。基于文献综述的结果和这些访谈的结果，起草了 PRO 问卷。在对患者进行认知性访谈后，确定了问卷的最终版本。概念启发访谈在美国进行，共访谈了 30 名患者（14 名患有 aBCC，16 名患有 BCCNS）和 4 名医生（2 名皮肤科医生、1 名 Mohs 外科医生和 1 名肿瘤学家）。其中有 10 名患者（5 名患有 aBCC，5 名患有 BCCNS）参加了认知性访谈。主要结果和措施：开发了 2 种问卷，以便医生评估 aBCC 和 BCCNS 患者疾病对其情绪、社交和身体的影响。结果：大多数概念启发访谈患者为男性（63%）和白人（93%）；他们的平均年龄为 57 岁。两种情况下均对情绪、社交和身体功能产生了影响。患者无法进行许多活动并回避其他活动。79%的 aBCC 患者和所有 BCCNS 患者都报告了疤痕。医生访谈也揭示了类似的发现。在认知性访谈中，这些问卷被认为是相关的、清晰的和全面的。结论和相关性：晚期 BCC 和 BCCNS 以独特且实质性的方式影响患者。这些 PRO 问卷是在患者和临床医生的投入下开发的，可衡量对患有这些疾病的患者有影响的关键领域。
