Group Develops Measure They Say Could Help Tailor Hemophilia Treatment

A group of researchers has developed a multidimensional measure they say reliably captures the burden of hemophilia treatment in adults and adolescents with the disorder, publishing their findings in Journal of Patient-Reported Outcomes.

The measure, coined the Hemophilia Treatment Experience Measure (Hemo-TEM), will allow for a more complete picture of treatment burden and how it affects things like health-related quality of life (HRQOL) and adherence, explained the researchers.

The group leveraged concept elicitation (CE) interviews with 5 clinical experts and 30 adults with hemophilia to create the measure, performed cognitive debriefing (CD) interviews with 34 adolescent and adult patients to refine the measure, and analyzed data from 3 clinical trials and 4 analysis sets for psychometric validation.

“Findings from the CE, CD, 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 patients with hemophilia,” commented the group. “Although other measures exist which may capture some aspects of treatment burden, the Hemo-TEM is the first measure designed specifically to only assess the broad spectrum of treatment burdens distinct from disease burden or treatment satisfaction.”

The measure takes into account 26 items spanning 5 domains—injection difficulties, physical impact, treatment bother, interference with daily life, and emotional impact—that patients flagged as being important to them and interviewees identified as resulting in better outcomes. These 5 domains were also included in the literature analyzed. All scores included in the measure were deemed reliable, exceeding the threshold of 0.70, and construct validity was acceptable, with all but 1 domain meeting all hypothesized associations.

The researchers noted that clinicians and patients shared similar sentiments in their interviews, pointing to venous access issues, treatment frequency, and time for preparing and administering treatment as key drivers of treatment burden. Through these insights captured in the measure, treatment could be tailored to patient preferences, reducing the burden of treatment, explained the researchers, who added that treatment efficacy and adherence should be captured alongside the measure.

“The concept of adherence was not included in the final Hemo-TEM, as it did not perform psychometrically and conceptually. One could view adherence as a more distal consequence rather than proximal impact of treatment burden and thus not suitable for a measure of the proximal impacts designed to capture treatment benefit in a clinical trial setting,” wrote the researchers. “Further, it has been suggested that quantitative methods of assessing adherence, such as counting of factor concentrate vials returned, may be a more accurate methodology for the assessment of adherence.”

The group noted that more research is required to validate their findings and added that the study was limited to the United States, potentially limiting its generalizability.

Reference

Brod M, Bushnell D, Neergaard J, Waldman L, Busk A. Understanding treatment burden in hemophilia: development and validation of the Hemophilia Treatment Experience Measure (Hemo-TEM). J Patient-Rep Outcomes. Published online February 23, 2023. doi:10.1186/s41687-023-00550-6