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

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

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.

Study Overview

• Status: Unknown
• Conditions: Moderate and Severe Haemophilia
• Intervention / Treatment: Other: HEAD-US scoring system

Detailed Description

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

• Study Type: Interventional
• Enrollment (Anticipated): 200
• Phase: Not Applicable

Contacts and Locations

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 6 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Diagnostic
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

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

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: Guy's and St Thomas' NHS Foundation Trust
• Collaborators: Pfizer
• Investigators: Principal Investigator: Gerard Dolan, MD, Guys and St Thomas' NHS Foundation Trust

Study record dates

Study Major Dates

• Study Start: December 1, 2015
• Primary Completion (Anticipated): November 1, 2017
• Study Completion (Anticipated): December 1, 2017

Study Registration Dates

• First Submitted: July 31, 2017
• First Submitted That Met QC Criteria: July 31, 2017
• First Posted (Actual): August 2, 2017

Study Record Updates

• Last Update Posted (Actual): August 2, 2017
• Last Update Submitted That Met QC Criteria: July 31, 2017
• Last Verified: July 1, 2017

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Hematologic Diseases; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Blood Coagulation Disorders; Hemophilia A
• Other Study ID Numbers: 63143

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No
• IPD Plan Description: Data will be analysed and reported as results not individual data

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No