Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 2025 Jan;31(1):48-62.
doi: 10.1111/hae.15102. Epub 2024 Oct 10.

Development and validation of the Child Hemophilia Treatment Experience Measure: A new observer-reported outcome measure

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

Development and validation of the Child Hemophilia Treatment Experience Measure: A new observer-reported outcome measure

Meryl Brod et al. Haemophilia. 2025 Jan.

Abstract

Introduction: The Child Hemophilia Treatment Experience Measure (Child Hemo-TEM) was developed to capture the treatment burden experience of children with haemophilia (CwH).

Aim: Describe the development of this novel haemophilia-specific measure.

Methods: 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.

Results: 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.

Conclusions: The study findings provide evidence that the Child Hemo-TEM is a brief, well-designed, and valid and reliable measure of haemophilia treatment burden.

Keywords: child; factor analysis; haemophilia; observer‐reported outcome measure; psychometrics; statistical; surveys and questionnaires.

PubMed Disclaimer

Conflict of interest statement

MB is a consultant to the pharmaceutical industry, including Novo Nordisk. DMB is an employee of Evidera and has received funding for research carried out in this work.

AKB is an employee and shareholder of Novo Nordisk A/S. JSN is an employee of Novo Nordisk A/S.

Medical writing support was funded by Novo Nordisk A/S.

Figures

FIGURE 1
FIGURE 1
Theoretical model of haemophilia treatment impacts for children with haemophilia. HTC, haemophilia treatment centre.
FIGURE 2
FIGURE 2
Conceptual framework of the child haemophilia treatment experience measure (child hemo‐TEM).
See this image and copyright information in PMC

References

    1. Barr RD, Saleh M, Furlong W, et al. Health status and health‐related quality of life associated with hemophilia. Am J Hematol. 2002;71(3):152‐160. doi:10.1002/ajh.10191 - DOI - PubMed
    1. Riley RR, Witkop M, Hellman E, Akins S. Assessment and management of pain in haemophilia patients. Haemophilia. 2011;17(6):839‐845. doi:10.1111/j.1365-2516.2011.02567.x - DOI - PubMed
    1. Arcieri R, Molinari AC, Farace S, et al. Present and future challenges in the treatment of haemophilia: the patient's perspective. Blood Transfus. 2013;11(4):s82‐s85. doi:10.2450/2013.013s - DOI - PMC - PubMed
    1. Mannucci PM, Franchini M. Present and future challanges in the treatment of haemophilia: a clinician's perspective. Blood Transfus. 2013;11(4):s77‐s81. doi:10.2450/2013.012s - DOI - PMC - PubMed
    1. Specchia ML, Capizzi S, Kheiraoui F, et al. The use of Advate in hemophilia A treatment: impacts on organization and management, finance and quality of life. Ital J Public Health. 2012;9(4 supp2):S75‐S96.

Publication types

Grants and funding