Psychometric Validation of the Hemophilia Functional Ability Scoring Tool (Hemo-FAST)

Abstract

Background

The Hemophilia Functional Ability Scoring Tool (Hemo-FAST), consisting of a patient-reported outcome (PRO) part and a clinician-reported outcome (ClinRO) part, was developed as a rapid and effective tool to assess functional mobility in clinical practice. This study (NCT04731701) aimed to validate the psychometric properties of Hemo-FAST for assessment of joint health in people with haemophilia (PwH).

Methods

PwH A or B aged ≥18 years completed questionnaires including the PRO part of Hemo-FAST and the short-form 36 health survey (SF-36) during one study visit. Clinicians completed the Haemophilia Joint Health Score (HJHS) and the ClinRO part of Hemo-FAST at the same visit. Validation was performed using reliability, construct validity, and structure validity assessments.

Results

The study enrolled 180 PwH A or B from 14 centres across France. Estimated time to complete the PRO part was mean (standard deviation) 4.6 (5.4) minutes. PRO items showed good test–retest reliability (intraclass correlation coefficient value ≥0.70). Inter-rater values were >0.70 for 7/9 ClinRO items, indicating good reliability. All items (15 PRO; 9 ClinRO) had high internal consistency (Cronbach's coefficient alpha: 0.97). Hemo-FAST demonstrated convergent construct validity with HJHS and the SF-36 physical component and discriminant construct validity with the SF-36 mental health component. Hemo-FAST scores distinguished between subgroups of people with expected differences in joint health status, including by haemophilia severity (p < 0.0001).

Conclusion

This study successfully validated Hemo-FAST as a rapid and reliable tool for the functional assessment of joint health in adults with haemophilia, both in clinical practice and clinical research settings.

Keywords

haemophilia - clinical scoring tool - real-world evidence - joint health - assessment

Data Availability Statement

Sobi is committed to responsible and ethical sharing of study data, while protecting individual participant integrity and compliance with applicable legislation. Data access will be granted in response to qualified research requests. All requests are evaluated by a cross-functional panel of experts within Sobi and a decision on sharing will be based on the scientific merit and feasibility of the research proposal, maintenance of personal integrity, and commitment to publication of the results. To request access to study data, a data sharing request form (available on www.sobi.com) should be sent to [e-mail]. Further information on Sobi's data sharing policy and process for requesting access can be found at: https://www.sobi.com/en/policies. Hemo-FAST© is protected by international copyright, with all rights reserved by Sobi. Do not use without permission. For information on, or permission to use Hemo-FAST©, please contact Mapi Research Trust at: https://eprovide.mapi-trust.org/instruments/hemophilia-functional-ability-scoring-tool.

Authors' Contribution

Conceptualization of the study: C.N., V.B., C.G., A.H., and A.L. Study design: C.N., A.H., and A.L. Data acquisition: C.N., D.D., F.G.-V., N.D., S.Car., V.B., Y.R., M.-L.P.-J., B.F., B.T., S.Cas., A.L., and C.G. Data analysis: C.N, N.K., E.B., and A.L. Data interpretation: C.N., N.K., S.Car., V.B., B.F., E.B., A.L., and C.G. All authors meet the International Committee of Medical Journal Editors criteria for authorship.

Publication History

Received: 23 August 2024

Accepted: 10 December 2024

Article published online:
21 January 2025

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