Hemophilia ArthropathyNCT04715100
Pain and Joint Status With Psychosocial Factors in Patients With Hemophilia
An observational study of Hemophilia Arthropathy, run by Investigación en Hemofilia y Fisioterapia at 1 site in Spain.
Trial summary
Background. The recurrence of hemarthrosis in patients with congenital coagulopathies favors the development of a progressive, degenerative intra-articular lesion (hemophilic arthropathy) that mainly affects the knees, ankles and elbows. Read more about this study
- Who is it for?
- Ages 18 to 65 years, men onlyHealthy volunteers can take part
- Conditions the study is for
- Hemophilia Arthropathy
- What is being tested?
- Observational group
- How many people
- 60 participants
- Where
- 1 siteSpain
- Study dates
- 5 Apr 2022to 1 Nov 2022
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- Men only
- Healthy volunteers accepted
- Adult, Older adult
Study population: Patients with hemophilia attending in hemophilia Associations into Spanish Federation of Hemophilia (Fedhemo).
Who may be able to join
- Patients diagnosed with hemophilia A and B
- Patients of legal age
- Patients on prophylactic or on demand treatment with clotting factor concentrates.
Who cannot join
- Patients with neurological or cognitive disorders that impede the understanding of the questionnaires
- Dependent patients who require help from a third person to get around
- Patients who have developed a hemarthros in the 4 weeks prior to the study.
- Patients who have not signed the informed consent document.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Relationship of Pain and Joint Status With the Perception of Psychosocial Factors in Patients With Hemophilic Arthropathy: A Multicenter Observational Study
Background. The recurrence of hemarthrosis in patients with congenital coagulopathies favors the development of a progressive, degenerative intra-articular lesion (hemophilic arthropathy) that mainly affects the knees, ankles and elbows. Disability and functional limitations are characteristic sequelae of these patients from an early age.
Objective. To assess the joint status, the perception of quality of life, the performance of roles and the performance of daily life activities, in patients with hemophilic arthropathy.
Read more
Study design. Observational, multicenter study. Method. 70 patients with hemophilia A and B will be included in this study. Patients will be recruited from 4 centers in different regions of Spain. The dependent variables will be: pain (measured with the Visual Analogue Scale), joint status (Hemophilia Joint Health Score), perception of quality of life (SF-36 questionnaire), list of roles, performance of daily life activities (Barthel scale), performing the instrumental activities of daily life (Lawton and Brody scale). The evolution of the variables will be analyzed using parametric (t-student or ANOVA) or non-parametric (Wilcoxon or Friedman) tests. A correlation analysis will be carried out between the dependent and independent variables, as well as a linear regression analysis between the observed determining variables.
Expected results. The aim is to observe the relationship of pain and joint status in patients with hemophilic arthropathy, with their perception of quality of life, the development of the roles they play and the performance of basic and instrumental activities of daily living.
Read the detailed description
Main objective: To analyze the relationship of pain and joint status with psychosocial and functional variables in adult patients with hemophilic arthropathy.
Secondary objectives: i) To detect the relationship between joint damage in psychosocial disorders in patients with hemophilia; ii) Evaluate the performance and perception of roles with the clinical situation of patients with hemophilic arthropathy; iii) Identify the factors that influence the performance of basic and instrumental activities of daily living in adult patients with hemophilia; iv) Observe changes in the perception of quality of life, performance of roles and activities of daily life based on the clinical and sociodemographic characteristics of patients with hemophilia.
Patients who meet the selection criteria will be summoned to a meeting where the principal investigator will present, orally and in writing, the characteristics of the investigator, the intended objectives and the risks of the intervention. At said meeting, the following documentation will be provided: patient information document and informed consent document for patients.
After obtaining the informed consent of the patients, the evaluations will be carried out in the dependencies of the locations included in the study. Six dependent variables will be measured: pain, joint condition, perception of quality of life, list of roles, performance of basic activities of daily life, and performance of instrumental activities of daily life.
All joint state evaluations will be performed by the same physiotherapist, blinded to the objectives of the study. The evaluation of the questionnaires will be analyzed by another evaluator, blinded to the objectives of the study.
- Conditions
- Hemophilia Arthropathy
- Being tested
- Observational group
- Keywords
- Hemophilic arthropathy, Educational intervention, Joint pain, Quality of life, Anxiety, Stress
- Medical subject headings
- Arthralgia, Anxiety Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Observational groupOther
The study dependent variables will be: pain (vas scale), joint status (Hemophilia Joint Health Score), perception of quality of life (SF-36 questionnaire), list of roles (Oakley questionnaire), performance of basic activities of daily life (Barthel index) and the performance of instrumental activities of daily living (Lawton and Brody scale).
| Study group | Role | What they receive |
|---|---|---|
| Experimental group | The inclusion criteria to participate in the present study are patients: with a diagnosis of hemophilia A and B; adults; and on a prophylactic or on-demand treatment regimen with FVIII / FIX concentrates.
For their part, patients with: neurological or cognitive alterations that impede understanding of the questionnaires will be excluded from the study; Dependent patients who require help from a third person to get around; patients who have developed a hemarthros in the 4 weeks prior to the study; and those who have not signed the informed consent document.
For inclusion in the study, patients will continue to be administered the dose of FVIII / FIX concentrates, following the guidelines of the medical criteria established by their reference hematologist. Throughout this study, the medical criteria for drug treatment, the dosage, and the replacement treatment periods will not be changed. Receives: Other: Observational group |
What is being measured
The primary outcome is the main question the study is designed to answer.
Assess the perception of joint pain with numerical scale of pain
Measurement with numerical scale of pain. The patient must assign pain to a numerical value between two extreme points (0 to 10). Although the subject is asked to use numerical values to indicate the level of pain, the use of keywords, as well as previous instructions, are necessary if we expect the patient to conceptualize their pain in numerical terms. The scale is discrete, not continuous, but to perform statistical analyzes, equal intervals can be assumed between categories (score 0-10; higher scores mean a worse joint pain).
Measured: Screening visit
Assess the joint status with Hemophilia Joint Health Score
Measurement the joint status with the instrument: Haemophilia Joint Health Score (score 0-120; higher scores mean a worse joint status).
Measured: Screening visit
Secondary outcomes (4)
Assess the perception of quality of life with the questtionaire Short Form 36 Health Survey
Measurement with Short Form 36 Health Survey [SF-36]. This health questionnaire is one of the most widely used, validated and translated instruments in the field of measuring the quality of life related to health. The SF-36 health questionnaire is a generic instrument consisting of 36 questions, which provides a profile of the health status and is applicable to both patients and the healthy population. The questionnaire covers 8 dimensions: physical functioning, physical role, corporal pain, general health, vitality, social functioning, emotional role and mental health of the patient. (score 0-100; higher scores mean a better outcome).
Measured: Screening visit
Assess the list of roles with Oakley scale
It will be measured with Oakley et al (1985). The list is divided into 2 parts: the first assesses the roles that have served the most to organize the individual's daily life and the second identifies the degree to which the individual values each role. The list presents 10 roles: student, worker, caregiver, homeowner, friend, family member, religious participant, hobbyist, and participant in organizations. The list aims to identify the roles that organize the individual's daily life, for this reason the definition of each role refers to the frequency of performance. (score 0-60; higher scores mean a better outcome).
Measured: Screening visit
Assess the carrying out activities of daily living
It will be measured with the Barthel index. This scale measures the capacity of the person to carry out ten basic activities of daily life, obtaining a quantitative estimate of the degree of dependence of the subject. This instrument assesses each of the corresponding activities and, depending on your ability to perform it, scores with 0, 5 or 10 points (up to 15 for certain activities). (score 0-100; higher scores mean a better outcome).
Measured: Screening visit
Assess the instrumental activities of daily living
It will be evaluated with the Lawton and Brody scale. This questionnaire measures the degree of independence that the patient maintains. It is made up of eight items with various response alternatives that together evaluate the ability to carry them out autonomously. The answer options score 1 (the person performs them independently) or 0 (dependent). (score 0-8; higher scores mean a better outcome).
Measured: Screening visit
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Jan 2021
- Study start5 Apr 2022
- Primary completion30 Jun 2022
- Study completion1 Nov 2022 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Hemophilia ArthropathyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 60 participants (actual)
Sponsor and investigators
- Lead sponsor
- Investigación en Hemofilia y Fisioterapia
- Sponsor type
- Research network
- Responsible party
- Sponsor
- Investigators
- Rubén Cuesta-Barriuso, PhD, Principal investigator, Universidad de Murcia
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04715100
- Sponsor's protocol ID
- He-Perception
- Status last verified
- Mar 2022
- First submitted
- 14 Jan 2021
- Last updated
- 6 Oct 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Seoane-Martin ME, Cuesta-Barriuso R, Rodriguez-Martinez MC. Performance of instrumental activities of daily living in patients with haemophilic arthropathy. A cross-sectional cohort study. Haemophilia. 2024 Nov;30(6):1406-1413. doi: 10.1111/hae.15114. Epub 2024 Oct 24.
PubMed 39447049
Study at a glance
From the registry record, last updated by the sponsor on 6 Oct 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Relationship of Pain and Joint Status With the Perception of Psychosocial Factors in Patients With Hemophilic Arthropathy: A Multicenter Observational Study
- ClinicalTrials.gov ID
- NCT04715100
- Acronym / sponsor ID
- He-Perception
- Conditions
- Hemophilia Arthropathy
- Keywords
- Hemophilic arthropathy, Educational intervention, Joint pain, Quality of life, Anxiety, Stress
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 60 participants (actual)
- Lead sponsor
- Investigación en Hemofilia y FisioterapiaResearch network
- Study start
- 5 Apr 2022 (actual)
- Primary completion
- 30 Jun 2022 (actual)
- Study completion
- 1 Nov 2022 (estimated)
- First posted
- 20 Jan 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Background. The recurrence of hemarthrosis in patients with congenital coagulopathies favors the development of a progressive, degenerative intra-articular lesion (hemophilic arthropathy) that mainly affects the knees, ankles and elbows. Disability and functional limitations are characteristic sequelae of these patients from an early age.
Objective. To assess the joint status, the perception of quality of life, the performance of roles and the performance of daily life activities, in patients with hemophilic arthropathy.
Study design. Observational, multicenter study. Method. 70 patients with hemophilia A and B will be included in this study. Patients will be recruited from 4 centers in different regions of Spain. The dependent variables will be: pain (measured with the Visual Analogue Scale), joint status (Hemophilia Joint Health Score), perception of quality of life (SF-36 questionnaire), list of roles, performance of daily life activities (Barthel scale), performing the instrumental activities of daily life (Lawton and Brody scale). The evolution of the variables will be analyzed using parametric (t-student or ANOVA) or non-parametric (Wilcoxon or Friedman) tests. A correlation analysis will be carried out between the dependent and independent variables, as well as a linear regression analysis between the observed determining variables.
Expected results. The aim is to observe the relationship of pain and joint status in patients with hemophilic arthropathy, with their perception of quality of life, the development of the roles they play and the performance of basic and instrumental activities of daily living.
Detailed description
Main objective: To analyze the relationship of pain and joint status with psychosocial and functional variables in adult patients with hemophilic arthropathy.
Secondary objectives: i) To detect the relationship between joint damage in psychosocial disorders in patients with hemophilia; ii) Evaluate the performance and perception of roles with the clinical situation of patients with hemophilic arthropathy; iii) Identify the factors that influence the performance of basic and instrumental activities of daily living in adult patients with hemophilia; iv) Observe changes in the perception of quality of life, performance of roles and activities of daily life based on the clinical and sociodemographic characteristics of patients with hemophilia.
Patients who meet the selection criteria will be summoned to a meeting where the principal investigator will present, orally and in writing, the characteristics of the investigator, the intended objectives and the risks of the intervention. At said meeting, the following documentation will be provided: patient information document and informed consent document for patients.
After obtaining the informed consent of the patients, the evaluations will be carried out in the dependencies of the locations included in the study. Six dependent variables will be measured: pain, joint condition, perception of quality of life, list of roles, performance of basic activities of daily life, and performance of instrumental activities of daily life.
All joint state evaluations will be performed by the same physiotherapist, blinded to the objectives of the study. The evaluation of the questionnaires will be analyzed by another evaluator, blinded to the objectives of the study.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Experimental group | – | Other: Observational groupThe inclusion criteria to participate in the present study are patients: with a diagnosis of hemophilia A and B; adults; and on a prophylactic or on-demand treatment regimen with FVIII / FIX concentrates. For their part, patients with: neurological or cognitive alterations that impede understanding of the questionnaires will be excluded from the study; Dependent patients who require help from a third person to get around; patients who have developed a hemarthros in the 4 weeks prior to the study; and those who have not signed the informed consent document. For inclusion in the study, patients will continue to be administered the dose of FVIII / FIX concentrates, following the guidelines of the medical criteria established by their reference hematologist. Throughout this study, the medical criteria for drug treatment, the dosage, and the replacement treatment periods will not be changed. |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- Male only
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
- Study population
- Patients with hemophilia attending in hemophilia Associations into Spanish Federation of Hemophilia (Fedhemo).
Key inclusion criteria (3 listed)
- Patients diagnosed with hemophilia A and B
- Patients of legal age
- Patients on prophylactic or on demand treatment with clotting factor concentrates.
Key exclusion criteria (4 listed)
- Patients with neurological or cognitive disorders that impede the understanding of the questionnaires
- Dependent patients who require help from a third person to get around
- Patients who have developed a hemarthros in the 4 weeks prior to the study.
- Patients who have not signed the informed consent document.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Observational groupThe study dependent variables will be: pain (vas scale), joint status (Hemophilia Joint Health Score), perception of quality of life (SF-36 questionnaire), list of roles (Oakley questionnaire), performance of basic activities of daily life (Barthel index) and the performance of instrumental activities of daily living (Lawton and Brody scale). | Other | Experimental group |
Primary outcome
Assess the perception of joint pain with numerical scale of pain
Time frame: Screening visit
Measurement with numerical scale of pain. The patient must assign pain to a numerical value between two extreme points (0 to 10). Although the subject is asked to use numerical values to indicate the level of pain, the use of keywords, as well as previous instructions, are necessary if we expect the patient to conceptualize their pain in numerical terms. The scale is discrete, not continuous, but to perform statistical analyzes, equal intervals can be assumed between categories (score 0-10; higher scores mean a worse joint pain).
Assess the joint status with Hemophilia Joint Health Score
Time frame: Screening visit
Measurement the joint status with the instrument: Haemophilia Joint Health Score (score 0-120; higher scores mean a worse joint status).
4 secondary outcomes
Assess the perception of quality of life with the questtionaire Short Form 36 Health Survey
Time frame: Screening visit
Assess the list of roles with Oakley scale
Time frame: Screening visit
Assess the carrying out activities of daily living
Time frame: Screening visit
Assess the instrumental activities of daily living
Time frame: Screening visit
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Oct 2022.
- Derived from this study: PubMed 39447049Seoane-Martin ME, Cuesta-Barriuso R, Rodriguez-Martinez MC. Performance of instrumental activities of daily living in patients with haemophilic arthropathy. A cross-sectional cohort study. Haemophilia. 2024 Nov;30(6):1406-1413. doi: 10.1111/hae.15114. Epub 2024 Oct 24.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Rubén Cuesta-Barriuso, PhD principal investigator
Universidad de Murcia
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hemophilia Arthropathy, run by Investigación en Hemofilia y Fisioterapia at 1 site in Spain.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 6 Oct 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Spain. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Assess the perception of joint pain with numerical scale of pain (time frame: Screening visit). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04715100, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Oct 2022.