Franklin Matthew, Shupo Francis, Wayi-Wayi Grace, Zibelnik Natasa, Jones Emily, Mason Nicola, Brazier John, Mukherjee Sudipto
Sheffield Centre for Health and Related Research (SCHARR), School of Medicine and Population Health, University of Sheffield, Sheffield, UK.
EUSA Pharma (UK), Hemel Hempstead, UK.
Oncol Ther. 2024 Sep;12(3):491-508. doi: 10.1007/s40487-024-00293-4. Epub 2024 Jul 16.
Idiopathic multicentric Castleman disease (iMCD) is a rare, chronic, debilitating lymphoproliferative disorder where the mainstay of treatment is symptom management. Our recent international patient survey showed that patients with iMCD have a high symptom burden that has a significant negative patient-reported impact on several aspects of daily life. As part of our ongoing work towards the development of an iMCD symptom burden scale, assessing the survey's psychometric properties is a critical step in understanding its adequacy, relevance, and usefulness. As iMCD is a rare disease, there are challenges to conducting such psychometric analyses which we describe.
As part of the exploratory psychometric analysis, three a priori hypothesis sets (HS) were generated by interviewing an iMCD-experienced clinician, a patient, and a caregiver to explore the iMCD patient survey's internal construct validity, given no gold standard iMCD measure exists for external construct validation. HS-1 hypothesized that a convergent or discriminant relationship exists with the patients' self-assessment of symptom effect on daily life between two potentially related or unrelated symptoms, respectively. HS-2 hypothesized that having a greater number of symptoms has a positive convergent relationship with the patients' assessment of symptoms' effect on daily life. Finally, HS-3 hypothesized that patients receiving treatment versus no treatment was associated with patients reporting less effect of symptom burden on their daily life. Spearman's rank absolute correlation strength (ACS) was used for HS-1 and HS-2 (convergent relationship, ACS ≥ 0.3 and p value < 0.05; divergent relationship, ACS < 0.3), and Cohen's d to quantify standardized absolute effect sizes (AES) for HS-3 (AES ≥ 0.5 and p value < 0.05).
Our analyses partially supported HS-1. None of the three positive convergent relationships were supported. Of the six discriminant relationships, only dizziness with impaired cognitive function and tiredness with dizziness were supported. HS-2 analyses showed there was convergent validity between the number of symptoms and their effect on aspects of daily life. HS-3 analyses did not provide evidence to support the hypothesis.
These internal psychometric construct analyses provide initial support for the bespoke iMCD patient survey and will guide additional work towards the development of the first iMCD-specific symptom burden scale.
特发性多中心Castleman病（iMCD）是一种罕见的慢性衰弱性淋巴增殖性疾病，治疗的主要手段是症状管理。我们最近的一项国际患者调查显示，iMCD患者的症状负担很重，这对患者报告的日常生活的多个方面产生了重大负面影响。作为我们正在进行的iMCD症状负担量表开发工作的一部分，评估该调查的心理测量特性是了解其充分性、相关性和实用性的关键步骤。由于iMCD是一种罕见疾病，进行此类心理测量分析存在挑战，我们对此进行了描述。
作为探索性心理测量分析的一部分，通过采访一位有iMCD经验的临床医生、一位患者和一位护理人员，生成了三个先验假设集（HS），以探讨iMCD患者调查的内部结构效度，因为不存在用于外部结构验证的iMCD金标准测量方法。HS-1假设分别在两种潜在相关或不相关症状之间，与患者对症状对日常生活影响的自我评估存在收敛或区分关系。HS-2假设症状数量越多，与患者对症状对日常生活影响的评估呈正收敛关系。最后，HS-3假设接受治疗与未接受治疗的患者相比，报告症状负担对其日常生活影响较小。HS-1和HS-2使用Spearman等级绝对相关强度（ACS）（收敛关系，ACS≥0.3且p值<0.05；区分关系，ACS<0.3），HS-3使用Cohen's d来量化标准化绝对效应大小（AES）（AES≥0.5且p值<0.05）。
我们的分析部分支持了HS-1。三个正收敛关系均未得到支持。在六个区分关系中，仅认知功能受损伴头晕和头晕伴疲劳得到支持。HS-2分析表明，症状数量与其对日常生活各方面的影响之间存在收敛效度。HS-3分析没有提供支持该假设的证据。
这些内部心理测量结构分析为定制的iMCD患者调查提供了初步支持，并将指导开发首个iMCD特异性症状负担量表的后续工作。
