Understanding treatment burden in hemophilia: development and validation of the Hemophilia Treatment Experience Measure (Hemo-TEM)
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)...
| Main Authors: | , , , , |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
SpringerOpen
2023-02-01
|
| Series: | Journal of Patient-Reported Outcomes |
| Subjects: | |
| Online Access: | https://doi.org/10.1186/s41687-023-00550-6 |
| _version_ | 1797864546599174144 |
|---|---|
| author | Meryl Brod Donald M. Bushnell Jesper Skov Neergaard Laura Tesler Waldman Anne Kirstine Busk |
| author_facet | Meryl Brod Donald M. Bushnell Jesper Skov Neergaard Laura Tesler Waldman Anne Kirstine Busk |
| author_sort | Meryl Brod |
| collection | DOAJ |
| description | 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. |
| first_indexed | 2024-04-09T22:53:40Z |
| format | Article |
| id | doaj.art-2ff47f0b4b2047fe8f4f7be1ab2f040e |
| institution | Directory Open Access Journal |
| issn | 2509-8020 |
| language | English |
| last_indexed | 2024-04-09T22:53:40Z |
| publishDate | 2023-02-01 |
| publisher | SpringerOpen |
| record_format | Article |
| series | Journal of Patient-Reported Outcomes |
| spelling | doaj.art-2ff47f0b4b2047fe8f4f7be1ab2f040e2023-03-22T11:22:30ZengSpringerOpenJournal of Patient-Reported Outcomes2509-80202023-02-017112310.1186/s41687-023-00550-6Understanding treatment burden in hemophilia: development and validation of the Hemophilia Treatment Experience Measure (Hemo-TEM)Meryl Brod0Donald M. Bushnell1Jesper Skov Neergaard2Laura Tesler Waldman3Anne Kirstine Busk4The Brod GroupEvidera | PPDNovo Nordisk A/SThe Brod GroupNovo Nordisk A/SAbstract 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.https://doi.org/10.1186/s41687-023-00550-6HemophiliaPatient-reported outcome measuresConcept elicitationInterviewsQualitative researchPsychometrics |
| spellingShingle | Meryl Brod Donald M. Bushnell Jesper Skov Neergaard Laura Tesler Waldman Anne Kirstine Busk Understanding treatment burden in hemophilia: development and validation of the Hemophilia Treatment Experience Measure (Hemo-TEM) Journal of Patient-Reported Outcomes Hemophilia Patient-reported outcome measures Concept elicitation Interviews Qualitative research Psychometrics |
| title | Understanding treatment burden in hemophilia: development and validation of the Hemophilia Treatment Experience Measure (Hemo-TEM) |
| title_full | Understanding treatment burden in hemophilia: development and validation of the Hemophilia Treatment Experience Measure (Hemo-TEM) |
| title_fullStr | Understanding treatment burden in hemophilia: development and validation of the Hemophilia Treatment Experience Measure (Hemo-TEM) |
| title_full_unstemmed | Understanding treatment burden in hemophilia: development and validation of the Hemophilia Treatment Experience Measure (Hemo-TEM) |
| title_short | Understanding treatment burden in hemophilia: development and validation of the Hemophilia Treatment Experience Measure (Hemo-TEM) |
| title_sort | understanding treatment burden in hemophilia development and validation of the hemophilia treatment experience measure hemo tem |
| topic | Hemophilia Patient-reported outcome measures Concept elicitation Interviews Qualitative research Psychometrics |
| url | https://doi.org/10.1186/s41687-023-00550-6 |
| work_keys_str_mv | AT merylbrod understandingtreatmentburdeninhemophiliadevelopmentandvalidationofthehemophiliatreatmentexperiencemeasurehemotem AT donaldmbushnell understandingtreatmentburdeninhemophiliadevelopmentandvalidationofthehemophiliatreatmentexperiencemeasurehemotem AT jesperskovneergaard understandingtreatmentburdeninhemophiliadevelopmentandvalidationofthehemophiliatreatmentexperiencemeasurehemotem AT laurateslerwaldman understandingtreatmentburdeninhemophiliadevelopmentandvalidationofthehemophiliatreatmentexperiencemeasurehemotem AT annekirstinebusk understandingtreatmentburdeninhemophiliadevelopmentandvalidationofthehemophiliatreatmentexperiencemeasurehemotem |