Study Identifies Core Instruments for Monitoring Physical Function in Hemophilia

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In a recent study, investigators identified 11 components from the Activities & Participation category of the WHO International Classification of Functioning (WHO-ICF) as potential markers for monitoring physical function in people with hemophilia.

Among these components, instruments were pinpointed for 5 activities: maintaining a standing position, walking long distances, walking up and down stairs, hopping, and squatting. Both children and adults with hemophilia highlighted walking long distances and hopping as particularly challenging tasks.

Recommendations for core outcome sets in hemophilia highlighted the necessity of incorporating measures related to performance-based physical health and sustainability of physical function, according to the study. Despite this recognition, the medical community has yet to reach a consensus on the valuable outcomes for both clinicians and patients.

“Rapid medical advances in hemophilia care demand performance measures that are sensitive and meaningful to both clinician and patient,” investigators wrote.

Therefore, the primary goal of this research was to identify practical instruments for monitoring musculoskeletal health in individuals with hemophilia, focusing specifically on performance-based physical function in real clinical settings.

The investigation encompassed a comprehensive approach, incorporating components from the ICF. A consensus-based decision analysis method was utilized, involving 3 key steps:

• Identification of Challenging Activities: Researchers identified activities posing the most difficulty for individuals with hemophilia. This step laid the foundation for subsequent evaluations.
• Scoping Review: A scoping review was conducted to identify quantitative performance-based measures related to the activities identified in the previous step. This comprehensive review formed the basis for evaluating potential instruments.
• Expert Opinion: To gauge the acceptability of the identified tests, a DELPHI approach was employed. International physiotherapists participated in a two-round DELPHI survey, providing expert insights crucial for finalizing the selected instruments.

Despite the wide array of identified components, international physiotherapists were able to establish practical and clinically useful performance-based tests for only 5 of the original 11 Activities & Participation ICF components. These tests were identified for children and adults and included one-legged balance, tandem stance, 6-minute walk test, timed up and down stairs, and 30-second sit-to-stand. Additionally, specific tests were chosen for children (single leg hop) and adults (timed up and go) to monitor their physical function.

In the realm of managing hemophilia, standardized and validated assessments of observed and self-reported outcomes have been deemed essential by clinicians worldwide, investigators noted. While existing tools like FISH, HAL, and pedHAL are valuable, they have limitations including ceiling and floor effects and an inability to discern differences in task execution and participation capacity.

To address these limitations, this study systematically reviewed performance-based instruments applicable to all ages and stages of arthropathy, ensuring continuous scale assessment and minimizing ceiling effects.

As supplementation to these findings, the study provided a specific set of core instruments to measure objective performance-based function and physical function maintenance. It was stated that its structured consensus-based approach, rooted in the WHO-ICF framework, coupled with empirical evidence, Delphi processes, patient involvement, and multinational clinician input, showcased methodological strength.

However, investigators acknowledged the study’s limitations. First, it lacked representation from diverse healthcare professionals and under-resourced regions, focusing mainly on physiotherapists in affluent, English-literate countries. The absence of translations for the Delphi survey further restricted inclusivity.

Additionally, certain critical aspects, like upper limb and lower limb complex tasks, were not addressed, requiring further research. The sensitivity and specificity of identified instruments regarding physical function sustainability and health require validation before recommendation. Furthermore, 6 WHO-ICF components lacked corresponding instruments, emphasizing the need for continued research in these areas.

“This study is the first step in defining a core set of performance-based instruments to monitor physical health and sustainability of physical function outcomes in people with hemophilia,” investigators concluded. “Establishing the psychometric properties of the identified instruments and whether they are meaningful to people with hemophilia is essential and is currently being investigated.”

Reference

Bladen, M, Harbidge, H, Drechsler, W, et al. Identifying performance-based outcome measures of physical function in people with haemophilia (IPOP). Haemophilia. 2023; 1-10. https://doi.org/10.1111/hae.14886