HEAD-US SCORING SYSTEM: Assessment of the Real-world Impact of Ultrasound

Study Overview
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Brief Summary
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Ultrasound represents a promising technique for the assessment of joint health in persons with haemophilia (PWH) by non-imaging specialists. The Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) programme has been developed with the aim of integrating joint ultrasound in the routine assessment of PWH through the use of a simplified scoring system. The inter-operator reliability of the technique among European haemophilia treaters has been validated and described elsewhere. Further work is needed to assess the real-world impact of ultrasound on disease management and treatment decision-making.

Detailed Description
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Patients are seen routinely in clinic by specialist haemophilia physiotherapists who assess the patients using the Haemophilia Joint Health Score tool (HJHS). Trial participants will be seen during their routine clinic visits and will be seen by a clinician and the physiotherapist who will carry out the routine assessment along with the HEAD-US scan. Patients will be seen a maximum of 4 times throughout the year from registration of the first patient, according to their visit schedule, but will only be scanned at the first visit. Results will be analysed to determine if the treatment decision-making is influenced by the results of the scan

Official Title
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HEAD-US SCORING SYSTEM: Assessment of the Real-world Impact of Ultrasound on Disease Management and Treatment Decision-making in Moderate and Severe Haemophilia

Conditions
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Moderate and Severe Haemophilia

Intervention / Treatment
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* Other: HEAD-US scoring system


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Moderate (FVIII/FIX:C 1-5%) and severe (FVIII/FIX: C <1%) patients with haemophilia A or B aged ≥ 6 years On demand or prophylaxis treatment regimen Exclusion Criteria: Joints with a previous history of surgery Joints with damage as a result of causes other than haemophilia-related bleeding

Ages Eligible for Study
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Minimum Age: 6 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Diagnostic
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Single<br>All patients will undergo ultrasound scan using the HEAD-US Scoring System by a non-imaging specialist | Other: HEAD-US scoring system<br>* HEAD-US scoring system - Ultrasound scan performed by a non-imaging specialist to assess for joint bleed or damage<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Any change in treatment | Any change in treatment including, management of a bleed and/or change in routine treatment protocol (including changes to prophylaxis regimen, or decision to start/stop prophylaxis regimen) as a result of the ultrasound findings | 12 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| % joints with normal HJHS and abnormal HEAD-US score | % joints with normal HJHS and abnormal HEAD-US score (for knees, ankles amd elbows separately | 12 months | 
| % joints with abnormal HJHS and normal HEAD-US score | % joints with abnormal HJHS and normal HEAD-US score | 12 months |