Horodniceanu Erica G, Bal Vasudha, Dhatt Harman, Carter John A, Huang Vicky, Lasch Kathryn
Pharmerit International, 4350 East West Highway, Suite 1110, Bethesda, MD, 20814, USA.
Novartis Pharmaceuticals Corporation, One Health Plaza, East Hanover, NJ, 07936, USA.
Health Qual Life Outcomes. 2017 Jun 23;15(1):129. doi: 10.1186/s12955-017-0702-0.
Compliance, palatability, gastrointestinal (GI) symptom, and treatment satisfaction patient- and observer-reported outcome (PRO, ObsRO) measures were developed/modified for patients with transfusion-dependent anemias or myelodysplastic syndrome (MDS) requiring iron chelation therapy (ICT).
This qualitative cross-sectional observational study used grounded theory data collection and analysis methods and followed PRO/ObsRO development industry guidance. Patients and caregivers of patients with transfusion-dependent anemias or MDS were individually interviewed face-to-face to cognitively debrief the Compliance, Palatability, GI Symptom Diary, and Modified Satisfaction with Iron Chelation Therapy (SICT) instruments presented electronically. Interviews were conducted in sets. Interviews began open-endedly to spontaneously elicit ICT experiences. Item modifications were debriefed during the later interviews. Interviews were audio recorded, transcribed, and coded. Data was analyzed using ATLAS.ti qualitative research software.
Twenty-one interviews were completed (Set 1: 5 patients, 6 caregivers; Set 2: 6 patients, 4 caregivers) in 6 US cities. Mean age was 43 years for patients and 9 years for children of caregivers. Conditions requiring ICT use across groups included transfusion-dependent anemias (85.7%) and MDS (14.3%). Concepts spontaneously reported were consistent with instruments debriefed. Interview analysis resulted in PRO and ObsRO versions of each instrument: Compliance (2 items), Palatability (4 items), GI Symptom Diary (6 items), and Modified SICT (PRO = 13, ObsRO = 17 items).
Qualitative research data from cognitive interviews supports the content validity and relevance of the instruments developed/modified. Quantitative validation of these PRO and ObsRO measures is needed testing for validity, reliability, and responsiveness for future research use with new formulations of oral ICT.
针对需要铁螯合疗法（ICT）的输血依赖性贫血或骨髓增生异常综合征（MDS）患者，开发/修改了依从性、适口性、胃肠道（GI）症状以及患者和观察者报告结局（PRO，ObsRO）等指标。
本定性横断面观察性研究采用扎根理论数据收集和分析方法，并遵循PRO/ObsRO开发行业指南。对输血依赖性贫血或MDS患者及其护理人员进行面对面的单独访谈，以对以电子方式呈现的依从性、适口性、胃肠道症状日记和铁螯合疗法改良满意度（SICT）工具进行认知总结。访谈分批次进行。访谈开始时采用开放式提问，以自发引出ICT体验。在后续访谈中对条目修改进行总结。访谈进行录音、转录和编码。使用ATLAS.ti定性研究软件对数据进行分析。
在美国6个城市完成了21次访谈（第1组：5名患者，6名护理人员；第2组：6名患者，4名护理人员）。患者的平均年龄为43岁，护理人员的儿童平均年龄为9岁。各群体中需要使用ICT的病症包括输血依赖性贫血（85.7%）和MDS（14.3%）。自发报告的概念与总结的工具一致。访谈分析得出了每个工具的PRO和ObsRO版本：依从性（2项）、适口性（4项）、胃肠道症状日记（6项）和改良SICT（PRO = 13项，ObsRO = 17项）。
认知访谈的定性研究数据支持所开发/修改工具的内容效度和相关性。需要对这些PRO和ObsRO指标进行定量验证，以测试其有效性、可靠性和反应性，供未来使用新型口服ICT进行研究时参考。
