Hauber A Brett, Gonzalez Juan Marcos, Coombs John, Sirulnik Andres, Palacios David, Scherzer Norman
RTI Health Solutions, Research Triangle Park, NC, USA;
Patient Prefer Adherence. 2011;5:307-14. doi: 10.2147/PPA.S20445. Epub 2011 Jun 24.
To quantify gastrointestinal stromal tumor (GIST) patients' preferences for reducing treatment toxicities and the likely effect of toxicities on patients' stated adherence.
English-speaking members of the Life Raft Group, a GIST patient advocacy and research organization, aged 18 years and older, completed a web-enabled survey including a series of treatment-choice questions, each presenting a pair of hypothetical GIST medication toxicity profiles. Each profile was defined by common or concerning toxicities verified via pretest interviews including: severity of edema, diarrhea, nausea, fatigue, rash, hand-foot syndrome, and heart failure; and risk of serious infection. Each subject answered 13 choice-format questions based on a predetermined experimental design with known statistical properties. Subjects were asked to rate the likelihood that they would miss or skip doses of medications with different toxicity profiles. Random-parameters logit was used to estimate a relative preference weight for each level of toxicity.
173 subjects completed the survey. Over the ranges of toxicity levels included in the study, heart failure was the most important toxicity. Edema was the least important. For all toxicities, reducing severity from severe to moderate was more important to subjects than reducing severity from moderate to mild. Reducing heart failure from moderate to mild and diarrhea from severe to moderate had the largest effects on subjects' evaluation of adherence.
All toxicities included in the study are important to patients. Treating or reducing severe toxicities is much more important to patients than treating or reducing moderate toxicities. Focused reductions of certain toxicities may improve treatment adherence.
量化胃肠道间质瘤(GIST)患者对降低治疗毒性的偏好以及毒性对患者所述依从性的可能影响。
生命筏组织(一个GIST患者倡导和研究组织)中年龄在18岁及以上的说英语的成员完成了一项基于网络的调查,其中包括一系列治疗选择问题,每个问题都呈现一对假设的GIST药物毒性概况。每个概况由通过预测试访谈验证的常见或令人担忧的毒性定义,包括:水肿、腹泻、恶心、疲劳、皮疹、手足综合征和心力衰竭的严重程度;以及严重感染的风险。每个受试者根据具有已知统计特性的预定实验设计回答13个选择格式的问题。受试者被要求对他们错过或跳过不同毒性概况药物剂量的可能性进行评分。使用随机参数logit来估计每个毒性水平的相对偏好权重。
173名受试者完成了调查。在研究中包括的毒性水平范围内,心力衰竭是最重要的毒性。水肿是最不重要的。对于所有毒性,将严重程度从重度降低到中度对受试者来说比将严重程度从中度降低到轻度更重要。将心力衰竭从中度降低到轻度以及将腹泻从重度降低到中度对受试者的依从性评估影响最大。
研究中包括的所有毒性对患者都很重要。对患者来说,治疗或降低严重毒性比治疗或降低中度毒性重要得多。针对性地降低某些毒性可能会提高治疗依从性。