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Development and validation of the Child Hemophilia Treatment Experience Measure: A new observer-reported outcome
Meryl Brod1, Donald M Bushnell2, Anne Kirstine Busk3
1Health Outcomes Research, The Brod Group, Mill Valley, California, USA.
Insights
The Child Hemophilia Treatment Experience Measure (Child Hemo-TEM) is a new tool to assess treatment burden in children with hemophilia. This observer-reported outcome measure is brief, valid, and reliable.
Area of Science:
- Pediatric Hematology
- Patient-Reported Outcomes
- Health Services Research
Background:
- The Child Hemophilia Treatment Experience Measure (Child Hemo-TEM) was developed to capture the treatment burden experienced by children with hemophilia (CwH).
- Existing measures may not adequately capture the unique aspects of treatment burden for pediatric patients.
- A specific, validated tool is needed to understand the impact of hemophilia treatment on children's lives.
Purpose of the Study:
- To describe the development and validation of a novel, hemophilia-specific measure for children.
- To create a reliable and valid tool to assess treatment burden in children with hemophilia.
- To generate an observer-reported outcome measure suitable for pediatric use.
Main Methods:
- Interviews with clinical experts, children with hemophilia (ages 8 to <12), and their caregivers.
- Analysis using adapted grounded theory principles, followed by preliminary measure development and debriefing.
- Psychometric validation using a cross-sectional web survey (N=187) with item reduction and factor analysis.
Main Results:
- Key concepts identified included adherence, ease of use, emotional/physical impacts, treatment concerns, and daily life interference.
- Cognitive debriefing revealed challenges for children completing the measure, leading to an observer-reported outcome version.
- A 7-item, internally consistent (α=.855) factor was generated, confirming convergent and known groups validity hypotheses.
Conclusions:
- The Child Hemo-TEM is a brief, well-designed measure for assessing hemophilia treatment burden in children.
- Evidence supports the validity and reliability of the Child Hemo-TEM as an observer-reported outcome.
- This measure can aid in understanding and addressing the treatment burden experienced by children with hemophilia.
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.
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