HemophiliaNCT07099313
Association of Prophylactic Treatment With Treatment Burden and Psychosocial Variables in Patients With Hemophilia
An observational study of Hemophilia, run by Investigación en Hemofilia y Fisioterapia at 1 site in Spain.
Trial summary
Introduction: Hemophilia is a congenital coagulopathy characterised by recurrent haemarthrosis, leading to chronic arthropathy and functional impairment. Prophylactic treatment with extended half-life (EHL) or short half-life (SHL) clotting factor concentrates is the most effective strategy for preventing these… Read more about this study
- Who is it for?
- Ages 18 years and older, men only
- Conditions the study is for
- Hemophilia
- How many people
- 114 participants
- Where
- 1 siteSpain
- Study dates
- 31 Jul 2025to 15 Oct 2025
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 years and older
- Men only
- No healthy volunteers
- Adult, Older adult
Study population: Patients with hemophilia A or B, undergoing prophylactic treatment with clotting factor concentrates.
Who may be able to join
- Patients over 18 years of age.
- Patients with a medical diagnosis of hemophilia A or B.
- Patients who, regardless of disease phenotype (mild, moderate or severe), are receiving prophylactic treatment with recombinant or plasma clotting factor concentrates, either short half-life or extended half-life.
- Patients must have maintained the same treatment regimen (SHL or EHL) continuously for at least six months prior to participation.
Who cannot join
- Patients who have developed inhibitors or antibodies to FVIII or FIX concentrates.
- People with a concomitant diagnosis of other serious or disabling chronic diseases (neurological, oncological, severe psychiatric or rheumatological) that may interfere with the perceived burden of treatment, sleep quality, functionality or perception of self-efficacy.
- Patients with cognitive, linguistic or sensory impairments that prevent them from correctly understanding and completing the questionnaires.
- Patients who are participating in clinical trials or intensive monitoring programmes that may alter their perception of the treatment and generate biases in the evaluation.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Spain1 site, 1 recruiting
- Recruiting
Universidad de Oviedo
Oviedo, Principality of Asturias, 33006
Rubén Cuesta-Barriuso, PhD, 0034 985 103 386, cuestaruben@uniovi.es
About this study
Official title: Association of Prophylactic Treatment With Treatment Burden, Self-efficacy, Adherence, Sleep Quality, and Locus of Control in Patients With Hemophilia; an Ambispective Cohort Study
Introduction: Hemophilia is a congenital coagulopathy characterised by recurrent haemarthrosis, leading to chronic arthropathy and functional impairment. Prophylactic treatment with extended half-life (EHL) or short half-life (SHL) clotting factor concentrates is the most effective strategy for preventing these episodes. EHL products have demonstrated haemostatic efficacy, with a lower frequency of infusions, potentially reducing the treatment burden, although their psychosocial impact has not yet been sufficiently explored.
Objectives: To evaluate the association between perceived treatment burden and psychosocial variables such as self-efficacy, adherence, sleep quality and health locus of control, depending on the type of treatment received (EHL or SHL).
Read more
Methods. Multicentre, ambispective cohort study. A total of 114 patients with haemophilia A or B undergoing EHL or SHL prophylactic treatment will be included. The primary variable will be treatment burden (Treatment Burden Questionnaire). Secondary variables will be perceived self-efficacy (General Self-Efficacy Scale), adherence (Torres scale), sleep quality (Pittsburgh Sleep Quality Index), treatment adherence (Torres Questionnaire) and health locus of control (Multidimensional Health Locus of Control). Potential confounding variables will include sociodemographic data (age, educational level, living arrangements) and clinical data (number of weekly infusions, type of hospital).
Expected results: Patients treated with extended-half-life products are expected to report lower treatment burden, higher self-efficacy and better sleep quality, regardless of sociodemographic or clinical factors.
- Conditions
- Hemophilia
- Keywords
- Hemophilia, Clotting factor concentrates, Treatment burden, Perceived self-efficacy, Adherence, Locus of control
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Observational group | Patients diagnosed with hemophilia A or B, undergoing prophylactic treatment with short half-life (SHL) and extended half-life (EHL) clotting factor concentrates. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Assessment of treatment burden at the time of the study.
Treatment burden will be assessed using the Spanish version of the Treatment Burden Questionnaire (TBQ). This self-administered multidimensional instrument consists of 15 items that measure the perceived burden of treatment in dimensions such as time spent, emotional impact, physical effort, medical coordination, and interference with daily life. Each item is scored on a Likert scale from 0 (no burden) to 10 (extreme burden). A total score is obtained, with higher values indicating greater perceived burden. This version has demonstrated good internal reliability in the Spanish chronic population (Cronbach's α = 0.89).
Measured: Screening visit.
Secondary outcomes (6)
Assessment of perceived self-efficacy in health at the time of the study.
Perceived self-efficacy in health will be measured using the Spanish version of the General Self-Efficacy Scale (GSE). This questionnaire consists of 10 items with a 4-point Likert scale (1 = strongly disagree to 4 = strongly agree). It assesses the individual's general belief in their ability to cope with difficulties and manage their health. The total score ranges from 10 to 40, with higher values indicating greater self-efficacy. The scale has shown good internal consistency (Cronbach's α = 0.87) in the Spanish population.
Measured: Screening visit.
Assessment of adherence to treatment at the time of the study.
Adherence to treatment will be assessed using the Torres scale. This structured self-report, specific for patients with hemophilia, includes 25 items, grouped into 5 dimensions: awareness of the disease, knowledge of sequelae, treatment difficulties, doctor-patient relationship, and treatment of bleeding episodes. This scale has proven to be valid and reliable for assessing adherence in these patients, with good internal consistency across all dimensions. The total score ranges from 0 to 100, with higher values indicating poorer adherence.
Measured: Screening visit.
Assessment of sleep quality at the time of the study.
Sleep quality will be assessed using the Pittsburgh Sleep Quality Index (PSQI) in its validated Spanish version. This self-administered questionnaire assesses sleep quality over the last month through 19 items grouped into seven components (latency, duration, efficiency, disturbances, medication use, daytime dysfunction, and subjective sleep quality). Each component is scored from 0 to 3, generating an overall score from 0 to 21, where a score >5 indicates poor sleep quality. Its test-retest reliability and internal consistency have been adequate (Cronbach's α = 0.81).
Measured: Screening visit.
Assessment of health locus of control at the time of the study.
Health locus of control will be measured using the Spanish version of the Multidimensional Health Locus of Control Form C (MHLC-C). This scale consists of 18 items that assess the extent to which people attribute control of their health to internal, medical or random factors. It consists of three subscales of 6 items each, scored on a scale from 1 (total disagreement) to 6 (total agreement). Each subscale generates an independent score. Validation in Spanish has shown good internal reliability (Cronbach's α > 0.70 in all three dimensions).
Measured: Screening visit.
Assessment of the sociodemographic variable age at the time of the study.
The sociodemographic variable age will be assessed by calculating the age (in months) from the date of birth to the date of assessment.
Measured: Screening visit.
Assessment of the clinical variable number of weekly infusions of clotting factor concentrates at the time of the study.
The clinical variable number of weekly infusions of clotting factor concentrates will be assessed by calculating the age (in months) from the date of birth to the date of assessment.
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 posted1 Aug 2025
- Study start31 Jul 2025 (estimated)
- Primary completion13 Sep 2025 (estimated)
- Study completion15 Oct 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for HemophiliaStudies 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
- Cross-sectional
- Enrolment
- 114 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Investigación en Hemofilia y Fisioterapia
- Sponsor type
- Research network
- Responsible party
- Principal investigator, Rubén Cuesta-Barriuso, Investigación en Hemofilia y Fisioterapia
- Investigators
- Rubén Cuesta-Barriuso, PhD, Principal investigator, Universidad de Oviedo
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07099313
- Sponsor's protocol ID
- He-ProF
- Status last verified
- Jul 2025
- First submitted
- 25 Jul 2025
- Last updated
- 5 Aug 2025
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Aug 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Association of Prophylactic Treatment With Treatment Burden, Self-efficacy, Adherence, Sleep Quality, and Locus of Control in Patients With Hemophilia; an Ambispective Cohort Study
- ClinicalTrials.gov ID
- NCT07099313
- Acronym / sponsor ID
- He-ProF
- Conditions
- Hemophilia
- Keywords
- Hemophilia, Clotting factor concentrates, Treatment burden, Perceived self-efficacy, Adherence, Locus of control
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 114 participants (estimated)
- Lead sponsor
- Investigación en Hemofilia y FisioterapiaResearch network
- Study start
- 31 Jul 2025 (estimated)
- Primary completion
- 13 Sep 2025 (estimated)
- Study completion
- 15 Oct 2025 (estimated)
- First posted
- 1 Aug 2025 (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.
Introduction: Hemophilia is a congenital coagulopathy characterised by recurrent haemarthrosis, leading to chronic arthropathy and functional impairment. Prophylactic treatment with extended half-life (EHL) or short half-life (SHL) clotting factor concentrates is the most effective strategy for preventing these episodes. EHL products have demonstrated haemostatic efficacy, with a lower frequency of infusions, potentially reducing the treatment burden, although their psychosocial impact has not yet been sufficiently explored.
Objectives: To evaluate the association between perceived treatment burden and psychosocial variables such as self-efficacy, adherence, sleep quality and health locus of control, depending on the type of treatment received (EHL or SHL).
Methods. Multicentre, ambispective cohort study. A total of 114 patients with haemophilia A or B undergoing EHL or SHL prophylactic treatment will be included. The primary variable will be treatment burden (Treatment Burden Questionnaire). Secondary variables will be perceived self-efficacy (General Self-Efficacy Scale), adherence (Torres scale), sleep quality (Pittsburgh Sleep Quality Index), treatment adherence (Torres Questionnaire) and health locus of control (Multidimensional Health Locus of Control). Potential confounding variables will include sociodemographic data (age, educational level, living arrangements) and clinical data (number of weekly infusions, type of hospital).
Expected results: Patients treated with extended-half-life products are expected to report lower treatment burden, higher self-efficacy and better sleep quality, regardless of sociodemographic or clinical factors.
Study design
- Observational model
- Cohort
- Time perspective
- Cross-sectional
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Observational group | – | –Patients diagnosed with hemophilia A or B, undergoing prophylactic treatment with short half-life (SHL) and extended half-life (EHL) clotting factor concentrates. |
Who may qualify
- Ages
- 18 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Patients with hemophilia A or B, undergoing prophylactic treatment with clotting factor concentrates.
Key inclusion criteria (4 listed)
- Patients over 18 years of age.
- Patients with a medical diagnosis of hemophilia A or B.
- Patients who, regardless of disease phenotype (mild, moderate or severe), are receiving prophylactic treatment with recombinant or plasma clotting factor concentrates, either short half-life or extended half-life.
- Patients must have maintained the same treatment regimen (SHL or EHL) continuously for at least six months prior to participation.
Key exclusion criteria (4 listed)
- Patients who have developed inhibitors or antibodies to FVIII or FIX concentrates.
- People with a concomitant diagnosis of other serious or disabling chronic diseases (neurological, oncological, severe psychiatric or rheumatological) that may interfere with the perceived burden of treatment, sleep quality, functionality or perception of self-efficacy.
- Patients with cognitive, linguistic or sensory impairments that prevent them from correctly understanding and completing the questionnaires.
- Patients who are participating in clinical trials or intensive monitoring programmes that may alter their perception of the treatment and generate biases in the evaluation.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Assessment of treatment burden at the time of the study.
Time frame: Screening visit.
Treatment burden will be assessed using the Spanish version of the Treatment Burden Questionnaire (TBQ). This self-administered multidimensional instrument consists of 15 items that measure the perceived burden of treatment in dimensions such as time spent, emotional impact, physical effort, medical coordination, and interference with daily life. Each item is scored on a Likert scale from 0 (no burden) to 10 (extreme burden). A total score is obtained, with higher values indicating greater perceived burden. This version has demonstrated good internal reliability in the Spanish chronic population (Cronbach's α = 0.89).
6 secondary outcomes
Assessment of perceived self-efficacy in health at the time of the study.
Time frame: Screening visit.
Assessment of adherence to treatment at the time of the study.
Time frame: Screening visit.
Assessment of sleep quality at the time of the study.
Time frame: Screening visit.
Assessment of health locus of control at the time of the study.
Time frame: Screening visit.
Assessment of the sociodemographic variable age at the time of the study.
Time frame: Screening visit.
Assessment of the clinical variable number of weekly infusions of clotting factor concentrates at the time of the study.
Time frame: Screening visit.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Aug 2025.
- 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.
Rubén Cuesta-Barriuso, PhD principal investigator
Universidad de Oviedo
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, 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 4 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: looking for participants now, as of the sponsor's last update on 5 Aug 2025. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Rubén Cuesta-Barriuso, PhD (0034 985103386, cuestaruben@uniovi.es), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Assessment of treatment burden at the time of the study. (time frame: Screening visit.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 5 Aug 2025.