Measurement Properties in People with Hemophilia
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Measurement Properties (Reliability and Validity) of a Set of Assessments evaluating Physical Function in adult Participants with Hemophilia.
- Who it may be relevant to
- Registry conditions: Hemophilia a, Hemophilia B, Musculoskeletal Complication, Measurement Error. Basic parameters: from 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Switzerland
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
Measurement Properties (Reliability and Validity) of a Set of Assessments Evaluating Physical Function in People with Hemophilia
Overview
The aim of this project is to determine the measurement properties (namely reliability and validity) of a set of clinical assessments, measuring different aspects of physical function.
Detailed description
The medical treatment of hemophilia has advanced greatly in recent years. There are now medications that reduce the physical symptoms of hemophilia. Nevertheless, those affected by hemophilia suffer from restricted mobility, reduced strength, reduced balance and pain as a result of the repeated bleeding. In order to make well-founded decisions regarding possible supportive therapeutic or medical measures, it is important to be able to estimate the extent of such limitations and whether these will change or remain stable.
The study is needed to validate the measurement properties of measurement instruments used to measure the effects of physical training interventions in participants with hemophilia. Clinical questions can only be solved if measuring instruments with sufficient measuring properties are used. These measurement properties must be evaluated in a relevant population. Otherwise, health professionals cannot assess whether measures or treatment are not effective or whether measured changes are not large enough to detect an actual change. Therefore, research is required to ensure that adequately studied measurement instruments are available for clinical assessment of those affected by adult participants with hemophilia.
Some of the tests (such as the Unipedal Stance Test or Four Square Step Test) have already been studied in children with hemophilia or in participants with other diseases. Since hemophilia is a disease with specific effects, the measuring instruments must also be researched in this participant group. There is still a research gap that the investigators would like to close with this study. For the study, the investigators selected tests that are already being used on adult participants with hemophilia and have had good experiences in practice. The tests are safe and can be carried out with little strain.
Interventions
- Other Measurement Properties (Reliability and Validity) of a Set of Assessments evaluating Physical Function in adult Participants with Hemophilia
This study evaluates the reliability (Test-Retest) of six health related instruments that assess physical function in people with hemophilia: Short Physical Performance Battery, Unipedal Stance Test, Four Square Step Test, Timed up and Go, 1 kg Arm Lift Test, 30 Second Chair Stand Test. This study is needed to determine the psychometric properties of these six assessments. Validity of these test will be determined with the Short Form 36, the Hemophilia Activities List and the Hemophilia Joint He
Primary outcome measures
- Short Physical Performance Battery (SPPB) [Time frame: Baseline (Day 1) followed after a minimum break of 48 hours by Follow up (Day 2).]
- Four Square Step Test (FSST) [Time frame: Baseline (Day 1) followed after a minimum break of 48 hours by Follow up (Day 2).]
- Timed up and Go (TUG) [Time frame: Baseline (Day 1) followed after a minimum break of 48 hours by Follow up (Day 2).]
- Unipedal Stance Test (UST) [Time frame: Baseline (Day 1) followed after a minimum break of 48 hours by Follow up (Day 2).]
- 30-Second Stair Stand Test [Time frame: Baseline (Day 1) followed after a minimum break of 48 hours by Follow up (Day 2).]
- 1 kg Arm Lift Test [Time frame: Baseline (Day 1) followed after a minimum break of 48 hours by Follow up (Day 2).]
Secondary outcome measures (3)
- Short Form 36 [Time frame: Baseline (Day 1)]
- Hemophilia Activities List [Time frame: Baseline (Day 1)]
- Hemophilia Joint Health Score Version 2.1 [Time frame: Baseline (Day 1)]
Eligibility criteria
Inclusion criteria
- Diagnosed Hemophilia Types A or B
- Adult people ≥ 18 years
- Patients on prophylactic factor or non-factor replacement treatment, or on demand factor replacement treatment.
- Able to walk for 20 meters independently, with or without walking aid
- Signed informed consent to participate in the study
Exclusion criteria
- Severe health conditions like severe cardiovascular, pulmonary, neurological or musculoskeletal diseases.
- Unable to perform the assessments due to disability or language problems.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Other
Study locations
Switzerland · 1 center
- Ruud Knols — Zurich
Identifiers
NCT: NCT06565481 · 2024-01354