Brod Meryl, Bushnell Donald M, Busk Anne Kirstine, Neergaard Jesper Skov
Health Outcomes Research, The Brod Group, Mill Valley, California, USA.
Patient Centered Research, Evidera | PPD, Bethesda, Maryland, USA.
Haemophilia. 2025 Jan;31(1):48-62. doi: 10.1111/hae.15102. Epub 2024 Oct 10.
The Child Hemophilia Treatment Experience Measure (Child Hemo-TEM) was developed to capture the treatment burden experience of children with haemophilia (CwH).
Describe the development of this novel haemophilia-specific measure.
Interviews were conducted with clinical experts, CwH and CwH's caregivers. Interviews were analysed according to adapted grounded theory principles. Based on the analysis, a preliminary measure was developed and debriefed. Psychometric analyses were performed according to an a priori analysis plan using data collected in a cross-sectional web survey and a final measure was generated.
Interviews with four clinical experts, 25 CwH ages 8 to <12 years, and 25 caregivers of CwH <12 years were conducted. Concepts endorsed by ≥10% of CwH and caregivers were: adherence, ease of use, emotional impacts, physical impacts, treatment concerns, and interference with daily life. Cognitive debriefing assessments were conducted to ensure participant understanding and item relevance. Caregivers found the measure to be understandable, comprehensive, and relevant. However, several issues with CwH completing the measure were identified and it was decided to only develop an observer-reported outcome version. Data for psychometric validation was collected in a web survey (N = 187). Item reduction dropped 12 items. Factor analysis generated a single, 7-item, internally consistent (α = .855) factor, which consisted of items covering all relevant a priori concepts. The majority of a priori convergent and all known groups validity hypotheses were confirmed.
The study findings provide evidence that the Child Hemo-TEM is a brief, well-designed, and valid and reliable measure of haemophilia treatment burden.
儿童血友病治疗体验量表（Child Hemo-TEM）旨在反映血友病患儿（CwH）的治疗负担体验。
描述这一新型血友病特异性量表的开发过程。
对临床专家、血友病患儿及其照料者进行访谈。根据适应性扎根理论原则对访谈进行分析。基于分析结果，制定了初步量表并进行汇报。根据预先制定的分析计划，利用横断面网络调查收集的数据进行心理测量分析，最终生成量表。
对4名临床专家、25名8至12岁的血友病患儿以及25名12岁以下血友病患儿的照料者进行了访谈。血友病患儿及其照料者中≥10%认可的概念包括：依从性、易用性、情绪影响、身体影响、治疗担忧以及对日常生活的干扰。进行了认知汇报评估，以确保参与者理解并确保项目相关性。照料者认为该量表易于理解、全面且相关。然而，发现血友病患儿在完成该量表时存在一些问题，因此决定仅开发观察者报告结局版本。通过网络调查（N = 187）收集心理测量学验证数据。项目缩减减少了12个项目。因子分析生成了一个由7个项目组成的单一、内部一致（α = 0.855）的因子，这些项目涵盖了所有相关的先验概念。大多数先验收敛效度和所有已知组效度假设得到证实。
研究结果表明，儿童血友病治疗体验量表（Child Hemo-TEM）是一种简短、设计良好且有效可靠的血友病治疗负担测量工具。
