HOLISTIC: Multidisciplinary Evaluation of Patients With Hemophilia
Study Details
Study Description
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.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
|
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.
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Patients Patients with haemophilia recruited for multidisciplinary assessment of the main physical, functional and psychosocial variables. |
|
| Control group Children without hemophilia |
Outcome Measures
Primary Outcome Measures
- 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 Outcome Measures
- Assessment of Clinical Patient Variables [Screening visit]
Hemophilia type measuring (A or B)
- Measure of Weight [Screening visit (pretreatment assessment)]
Measure of weight
Eligibility Criteria
Criteria
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.
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Universidad de Murcia | Murcia | Spain | 30100 |
Sponsors and Collaborators
- Universidad de Murcia
Investigators
- Principal Investigator: Rubén Cuesta-Barriuso, PhD, Catholic University San Antonio of Murcia
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- Hemo-HOLISTIC
- HOLISTIC
Study Results
Participant Flow
| Recruitment Details | |
|---|---|
| Pre-assignment Detail |
| Arm/Group Title | Patients | Control Group |
|---|---|---|
| Arm/Group Description | Patients with haemophilia recruited for multidisciplinary assessment of the main physical, functional and psychosocial variables. | Children without hemophilia |
| Period Title: Overall Study | ||
| STARTED | 53 | 51 |
| COMPLETED | 53 | 51 |
| NOT COMPLETED | 0 | 0 |
Baseline Characteristics
| Arm/Group Title | Patients | Control Group | Total |
|---|---|---|---|
| Arm/Group Description | Patients with haemophilia recruited for multidisciplinary assessment of the main physical, functional and psychosocial variables. | Children without hemophilia | Total of all reporting groups |
| Overall Participants | 53 | 51 | 104 |
| Age (years) [Mean (Standard Deviation) ] | |||
| Mean (Standard Deviation) [years] |
10.08
(1.36)
|
9.78
(1.22)
|
10.04
(1.31)
|
| Sex: Female, Male (Count of Participants) | |||
| Female |
0
0%
|
0
0%
|
0
0%
|
| Male |
53
100%
|
51
100%
|
104
100%
|
| Region of Enrollment (participants) [Number] | |||
| Spain |
53
100%
|
51
100%
|
104
100%
|
Outcome Measures
| Title | Assess the Joint Damage |
|---|---|
| Description | Measurement with Haemophilia Joint Health Score 2.1 (HJHS) |
| Time Frame | Screening visit |
Outcome Measure Data
| Analysis Population Description |
|---|
| 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/Group Title | Patients | Control Group |
|---|---|---|
| Arm/Group Description | Patients with haemophilia recruited for multidisciplinary assessment of the main physical, functional and psychosocial variables. | Children without hemophilia |
| Measure Participants | 53 | 51 |
| Mean (Standard Deviation) [points] |
0.36
(0.84)
|
0
(0)
|
| Title | Assess the Perception of Quality of Life |
|---|---|
| Description | Measurement through the Child health profile (Childhood Health and Illness Perception; CHIP-CE). |
| Time Frame | Screening visit |
Outcome Measure Data
| Analysis Population Description |
|---|
| The score ranges from 0 (poor QoL) to 100 points (good perception of QoL). |
| Arm/Group Title | Patients | Control Group |
|---|---|---|
| Arm/Group Description | Patients with haemophilia recruited for multidisciplinary assessment of the main physical, functional and psychosocial variables. | Children without hemophilia |
| Measure Participants | 53 | 51 |
| Mean (Standard Deviation) [points] |
38.32
(4.72)
|
37.55
(5.91)
|
| Title | Assessment of Clinical Patient Variables |
|---|---|
| Description | Hemophilia type measuring (A or B) |
| Time Frame | Screening visit |
Outcome Measure Data
| Analysis Population Description |
|---|
| [Not Specified] |
| Arm/Group Title | Patients | Control Group |
|---|---|---|
| Arm/Group Description | Patients with haemophilia recruited for multidisciplinary assessment of the main physical, functional and psychosocial variables. | Children without hemophilia |
| Measure Participants | 53 | 51 |
| Number [Percentage of Participants with Hemophil] |
92.5
174.5%
|
0
0%
|
| Title | Measure of Weight |
|---|---|
| Description | Measure of weight |
| Time Frame | Screening visit (pretreatment assessment) |
Outcome Measure Data
| Analysis Population Description |
|---|
| [Not Specified] |
| Arm/Group Title | Patients | Control Group |
|---|---|---|
| Arm/Group Description | Patients with haemophilia recruited for multidisciplinary assessment of the main physical, functional and psychosocial variables. | Children without hemophilia |
| Measure Participants | 53 | 51 |
| Mean (Standard Deviation) [Kg] |
41.59
(11.82)
|
39.36
(9.64)
|
Adverse Events
| Time Frame | Serious and Other (Not Including Serious) Adverse Events were not collected/assessed | |||
|---|---|---|---|---|
| Adverse Event Reporting Description | ||||
| Arm/Group Title | Patients | Control Group | ||
| Arm/Group Description | Patients with haemophilia recruited for multidisciplinary assessment of the main physical, functional and psychosocial variables. | Children without hemophilia | ||
All Cause Mortality |
||||
| Patients | Control Group | |||
| Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
| Total | / (NaN) | / (NaN) | ||
Serious Adverse Events |
||||
| Patients | Control Group | |||
| Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
| Total | 0/0 (NaN) | 0/0 (NaN) | ||
Other (Not Including Serious) Adverse Events |
||||
| Patients | Control Group | |||
| Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
| Total | 0/0 (NaN) | 0/0 (NaN) | ||
Limitations/Caveats
More Information
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
Results Point of Contact
| Name/Title | Dr. Rubén Cuesta-Barriuso |
|---|---|
| Organization | Real Fundación Victoria Eugenia |
| Phone | +34913146508 |
| ruben.cuestab@gmail.com |
- Hemo-HOLISTIC
- HOLISTIC