ReviewJournal of patient-reported outcomes2023
Understanding treatment burden in hemophilia: development and validation of the Hemophilia Treatment Experience Measure (Hemo-TEM).
Review in Journal of patient-reported outcomes, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 25 papers, 1 of them a synthesis that pooled it.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
25 citing papers in PubMed, 1 synthesis or guideline pooled it.
- Raising the patient's voice in influenza research and care: a systematic review of patient-reported outcomes.BMC infectious diseases · 2026Pooled it
- Denecimig (Mim8) prophylaxis over 52 weeks in adolescents and adults with hemophilia A: FRONTIER2 extension study.Research and practice in thrombosis and haemostasis · 2026Trial
- Expression-linked promoter selection (ELiPS) engineers short, strong ubiquitous promoters for gene therapy applications.bioRxiv : the preprint server for biology · 2026Article
- rAAV8 encapsidated HMR-001/z enables high efficiency hepatic transduction and restores hemostasis in hemophilic mice.Gene therapy · 2026Article
- Estimating the Impact of Hemophilia A Treatment Administration on Patient Health Utility: Combining a Discrete Choice Experiment with Time Trade-Off Method.Advances in therapy · 2026Article
- Building a gene editing lexicon: a model for rare and inherited disorders.Gene therapy · 2026Article
- Integrating Clinical, Functional, and Patient-Reported Outcomes in Haemophilia Care: A Delphi-Based Consensus on a New Monitoring Tool.Journal of clinical medicine · 2026Article
- Immune Response Associated Hepatotoxicity in Hemophilia Gene Therapy: Mechanisms, Management, and Challenges.Journal of hematology · 2026Review
- Real-World Effectiveness and Safety of Damoctocog Alfa Pegol in Severe and Nonsevere Patients With Hemophilia A From the Prospective, Multinational, Ongoing HEM-POWR Study.European journal of haematology · 2026Observational
- A Delphi Consensus by Hematologists on the Burden of Treatment on Patients with Severe Hemophilia in Mexico.Journal of blood medicine · 2026Article
- Burden of Treatment and Disease on People with Hemophilia: Global Real-World Survey.Patient preference and adherence · 2026Article
- Managing Haemophilic Arthropathy in People With Haemophilia in Japan: An Informal Consensus.Haemophilia : the official journal of the World Federation of Hemophilia · 2025Article
- Treatment and Disease Burden in a Cohort of People With Haemophilia Without Inhibitors in the United States.Haemophilia : the official journal of the World Federation of Hemophilia · 2025Article
- Real-World Unmet Needs of Patients With Haemophilia A and Haemophilia B With or Without Inhibitors: End-of-Study Results From the explorer6 Non-Interventional Study.Haemophilia : the official journal of the World Federation of Hemophilia · 2025Observational
- Concizumab, a Non-Replacement Therapy for Persons with Hemophilia with Inhibitors.Journal of clinical medicine · 2025Review
- Concizumab prophylaxis in people with hemophilia A or B without inhibitors: patient-reported outcome results from the phase 3 explorer8 study.Research and practice in thrombosis and haemostasis · 2025Article
- Burden of Treatment in Children and Adolescents With Type 1 Diabetes Evaluated by Focus Groups.Pediatric diabetes · 2025Article
- Development and validation of the Child Hemophilia Treatment Experience Measure: A new observer-reported outcome measure.Haemophilia : the official journal of the World Federation of Hemophilia · 2025Article
- A time trade-off study in the UK, Canada and the US to estimate utilities associated with the treatment of haemophilia.Health and quality of life outcomes · 2024Article
- A novel gene editing lexicon strategy for the haemophilia community: Research plan for development and preliminary results.Haemophilia : the official journal of the World Federation of Hemophilia · 2024Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
5 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundTo 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.
resultsConcept 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.
conclusionsFindings 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.
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