Symptom Burden and Its Impact on Daily Living with Idiopathic Multicentric Castleman’s Disease: Development and Psychometric Validation of a Bespoke International Patient and Caregiver Survey


Franklin M1, Jones E2, Shupo F3, Mason N2, Wayi-Wayi G4, Zibelnik N4, Repasky M5, Mukherjee S6, Brazier JE1
1University of Sheffield, Sheffield, UK, 2Lumanity, Sheffield, UK, 3EUSA Pharma, Hornchurch, UK, 4EUSA Pharma, Hemel Hempstead, UK, 5The Castleman Disease Collaborative Network, Paso Robles, CA, USA, 6Cleveland Clinic, Cleveland, OH, USA

Presentation Documents

OBJECTIVES: Idiopathic multicentric Castleman’s disease (iMCD) is a rare lymphoproliferative disorder driven by proinflammatory hypercytokinemia. Patient perspective of the burden and associated symptoms of iMCD on their daily life have not been previously studied. We developed a bespoke international survey to elicit the burden of iMCD and explored its psychometric properties in relation to construct validity.

METHODS: We generated hypotheses based on expert opinion (one clinician, one patient, and one caregiver) via interviews to understand whether specific questions and response options within the survey could be: grouped together as the same; related to one another; if items are related, the expected strength of the relationship and, for the clinical expert only, the potential direction of relationship. We used Spearman’s rank absolute correlation strength and associated p-value, and Cohen’s d standardized effect size methods.

RESULTS: Overall, 57 patients with iMCD completed the survey between April–November 2021. Patients had a mean number of 7 symptoms (range: 0 to 22 symptoms). Three a priori hypothesis sets were generated: 1. How specific symptoms relate to each other; 2. Number of symptoms and their relationship with aspects of daily life; and 3. Receiving treatment or not, and the overall relationship with aspects of daily life. Although the results did not enable support or refutation of all a priori hypotheses, we found a strong significant correlation between having a higher number of symptoms and greater adverse impact on daily life.

CONCLUSIONS: This exploratory analysis provides confidence in the internal construct validity of the survey. It supports use of this disease burden questionnaire as a tool for assessing disease severity, aiding treatment decision, and evaluating responses in daily practice and clinical research.

Conference/Value in Health Info

2022-11, ISPOR Europe 2022, Vienna, Austria

Value in Health, Volume 25, Issue 12S (December 2022)




Patient-Centered Research

Topic Subcategory

Instrument Development, Validation, & Translation, Patient Engagement, Patient-reported Outcomes & Quality of Life Outcomes


No Additional Disease & Conditions/Specialized Treatment Areas

Your browser is out-of-date

ISPOR recommends that you update your browser for more security, speed and the best experience on Update my browser now