PB0653 - Validation of the Hemophilia Treatment Experience Measure (Hemo-TEM): a New Haemophilia-specific Patient-reported Outcome Measure

Background: The Hemo-TEM is a patient-reported outcome (PRO) measure developed based on U.S. Food and Drug Administration (FDA) guidance to assess the burden of treatment on people with haemophilia. The measure is currently being administered in ongoing concizumab phase 3 trials.

Aims: The objective of this study was to validate the psychometric properties of the Hemo-TEM for adolescents and adults.

Methods: Patients from 3 clinical trials (NN7170-4213, NN7415-4255, NN7415-4310) currently taking an injection for haemophilia (n=88) completed a validation battery (demographics and PRO measures needed for the cross-sectional validation analysis) at a screening visit, at baseline (retest), and at 24 weeks post-baseline (n=56, sensitivity to change). Psychometric testing, including of the measurement model, reliability, validity, sensitivity to change, and meaningful change, followed FDA guidelines for PRO measure validation.

Results: Item Reduction dropped 4 items resulting in a final 26-item measure (Figure 1). 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 to 0.88) and reproducible (0.80 to 0.92)]. A-priori hypothesized associations for validity of the Hemo-TEM domains were confirmed. Preliminary estimates of sensitivity to change were seen with effect sizes between -0.30 and -0.70. The 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. The measure took approximately 5 minutes to complete suggesting minimal administration burden.

Conclusion(s): The Hemo-TEM can be considered a well-designed, valid, and reliable measure of the burden of haemophilia treatment on patients. This measure should prove useful to assess impacts related to haemophilia as well as to clinicians in tailoring treatments to patient characteristics and situations.