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Review
. 2023 Feb 23;7(1):17.
doi: 10.1186/s41687-023-00550-6.

Understanding treatment burden in hemophilia: development and validation of the Hemophilia Treatment Experience Measure (Hemo-TEM)

Meryl Brod  1 , Donald M Bushnell  2 , Jesper Skov Neergaard  3 , Laura Tesler Waldman  4 , Anne Kirstine Busk  3
Affiliations
Review

Understanding treatment burden in hemophilia: development and validation of the Hemophilia Treatment Experience Measure (Hemo-TEM)

Meryl Brod et al. J Patient Rep Outcomes. .

Abstract

Background: To capture the broad range of treatment burden issues experienced by adolescent and adult people with hemophilia (PWH), the Hemophilia Treatment Experience Measure (Hemo-TEM) was developed. We describe the development of this new hemophilia-specific patient-reported outcome (PRO) measure including concept elicitation, cognitive debriefing, and psychometric validation.

Results: Concept elicitation interviews were conducted with 5 clinical experts and 30 adult PWH in the United States (US). The qualitative analysis of these interviews and a review of the literature informed the PRO measure development. The project team reviewed concept endorsement rates and generated a 27-item preliminary version of the Hemo-TEM. Cognitive debriefing interviews were conducted to ensure participant understanding and item relevance in samples of (adolescent (n = 20) and adult (n = 14)) PWH in the US. The refined, validation-ready version of the Hemo-TEM included 30 items. Lastly, data from 3 clinical trials comprised the 4 analysis sets used for the psychometric validation with a sample size of N = 88. Item reduction dropped 4 items resulting in a final 26-item measure. Factor analysis generated 5 domains in the Hemo-TEM [injection difficulties (3 items), physical impact (6 items), treatment bother (7 items), interference with daily life (4 items), and emotional impact (6 items)] and a total score. All scores were reliable [internally consistent (0.84-0.88)]. For convergent validity, with the exception of one domain, all hypothesized associations were met. Preliminary sensitivity to change effect sizes were between - 0.30 and - 0.70. Meaningful change thresholds ranged from 6 points (physical impact and emotional impact) to 10 points (treatment bother) with 8 points for the Hemo-TEM total score.

Conclusions: Findings from the concept elicitation, cognitive debriefing, and psychometric validation phases provide evidence that the Hemo-TEM is a well-designed, valid, and reliable measure of the burden of hemophilia treatment, including treatment impact on adolescent and adult PWH.

Keywords: Adolescent; Adult; Concept elicitation; Hemophilia; Interviews; Patient-reported outcome measures; Psychometrics; Qualitative research.

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Conflict of interest statement

MB and DBM are consultants to the pharmaceutical industry, including Novo Nordisk. LTW was a consultant to the pharmaceutical industry during the time this research was conducted. AKB is an employee and shareholder of Novo Nordisk A/S and JSN is an employee of Novo Nordisk A/S.

Figures

Fig. 1
Fig. 1
Theoretical Model
Fig. 2
Fig. 2
Hemo-TEM Conceptual Framework
See this image and copyright information in PMC

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