Haemophilia
Conditions
Keywords
Haemophilia, Arthropathy, Range of motion, Strength, Proprioception, Gait, Quality of Life.
Brief summary
Multidisciplinary assessment of the physical, functional and psychosocial alterations in patients with hemophilia around the country. The aims of this study are: * Descriptive study of joint involvement in hemophilia patients with and without a history of hemarthrosis. * Descriptive study of the alterations of periarticular muscle strength as a result of acute processes or the development of chronic articular sequelae. * Descriptive study of biomechanical changes in lower limb joints and their impact on gait in patients with hemophilia. * Descriptive study of the joint space, regarding hemarthrosis and synovitis, intraarticular by ultrasonography. * Descriptive study of the relationship between skeletal muscle pathology in patients with hemophilia and their perceptions of it and their quality of life.
Detailed description
Observational study that will allow us: Analyze and evaluate the skeletal muscle of patients with hemophilia involvement. Describe the periarticular muscular deficit, depending on the degree of injury, age, presence of inhibitors and the development of physical activity. Identify the relationship between the functional deficit by clinical assessment performed with the use of validated scales measure force production with patients. Observe the presence of joint bleeds in children with hemophilia and synovial hypertrophy developing therein. Detect biomechanical abnormalities of the lower limbs and their impact on gait in patients with hemophilia. Assess the factors that influence the perception of illness and quality of life of patients with hemophilia.
Interventions
None listed
Sponsors
Universidad de Murcia
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
MALE
Age
8 Years to 60 Years
Healthy volunteers
No
Inclusion criteria
* Patients with hemophilia A and B. * Patients of all ages (pediatric, adolescents, youth and adults). * With or without joint involvement clinically diagnosed, and with or without a previous history of hemarthrosis joint loading. * Patients with or without inhibitors.
Exclusion criteria
* Patients without prior walking capacity. * Patients diagnosed with other congenital coagulopathy (von Willebrand disease, etc..). * Patients with neurological or cognitive impairments that prevent understanding the questionnaires and physical tests.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Assess the Joint Damage | Screening visit | Measurement with Haemophilia Joint Health Score 2.1 (HJHS) |
| Assess the Perception of Quality of Life | Screening visit | Measurement through the Child health profile (Childhood Health and Illness Perception; CHIP-CE). |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Assessment of Clinical Patient Variables | Screening visit | Hemophilia type measuring (A or B) |
| Measure of Weight | Screening visit (pretreatment assessment) | Measure of weight |
Countries
Spain
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Patients Patients with haemophilia recruited for multidisciplinary assessment of the main physical, functional and psychosocial variables. | 53 |
| Control Group Children without hemophilia | 51 |
| Total | 104 |
Baseline characteristics
| Characteristic | Patients | Control Group | Total |
|---|---|---|---|
| Age, Continuous | 10.08 years STANDARD_DEVIATION 1.36 | 9.78 years STANDARD_DEVIATION 1.22 | 10.04 years STANDARD_DEVIATION 1.31 |
| Region of Enrollment Spain | 53 participants | 51 participants | 104 participants |
| Sex: Female, Male Female | 0 Participants | 0 Participants | 0 Participants |
| Sex: Female, Male Male | 53 Participants | 51 Participants | 104 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — |
| other Total, other adverse events | 0 / 0 | 0 / 0 |
| serious Total, serious adverse events | 0 / 0 | 0 / 0 |
Outcome results
Primary
Assess the Joint Damage
Measurement with Haemophilia Joint Health Score 2.1 (HJHS)
Time frame: Screening visit
Population: Haemophilia Joint Health Score assesses joint health in patients with hemophilia. It consists of eight dimensions: swelling, muscular atrophy, crepitation and range of motion, joint pain, strength, motion and axial alignment. The score range is from 0 to 24 points (a score of 0 indicates no joint damage. The higher the score, the higher).
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Patients | Assess the Joint Damage | 0.36 points | Standard Deviation 0.84 |
| Control Group | Assess the Joint Damage | 0 points | Standard Deviation 0 |
Primary
Assess the Perception of Quality of Life
Measurement through the Child health profile (Childhood Health and Illness Perception; CHIP-CE).
Time frame: Screening visit
Population: The score ranges from 0 (poor QoL) to 100 points (good perception of QoL).
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Patients | Assess the Perception of Quality of Life | 38.32 points | Standard Deviation 4.72 |
| Control Group | Assess the Perception of Quality of Life | 37.55 points | Standard Deviation 5.91 |
Secondary
Assessment of Clinical Patient Variables
Hemophilia type measuring (A or B)
Time frame: Screening visit
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Patients | Assessment of Clinical Patient Variables | 92.5 Percentage of Participants with Hemophil |
| Control Group | Assessment of Clinical Patient Variables | 0 Percentage of Participants with Hemophil |
Secondary
Measure of Weight
Measure of weight
Time frame: Screening visit (pretreatment assessment)
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Patients | Measure of Weight | 41.59 Kg | Standard Deviation 11.82 |
| Control Group | Measure of Weight | 39.36 Kg | Standard Deviation 9.64 |