Association of Prophylactic Treatment With Treatment Burden and Psychosocial Variables in Patients With Hemophilia

NCT ID: NCT07099313

Last Updated: 2025-08-05

Basic Information

• Recruitment Status: RECRUITING
• Total Enrollment: 114 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2025-07-31
• Study Completion Date: 2025-10-15

Brief 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 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.

Conditions

Hemophilia

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: CROSS_SECTIONAL

Study Groups

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.

No interventions assigned to this group

Eligibility Criteria

Inclusion Criteria

• 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.

Exclusion Criteria

• 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.

• Minimum Eligible Age: 18 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: No

Sponsors

Investigación en Hemofilia y Fisioterapia

NETWORK

Sponsor Role: lead

Responsible Party

Rubén Cuesta-Barriuso

Principal Investigator

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Rubén Cuesta-Barriuso, PhD

Role: PRINCIPAL_INVESTIGATOR

Universidad de Oviedo

Locations

Universidad de Oviedo

Oviedo, Principality of Asturias, Spain

Site Status: RECRUITING

Countries

Spain

Central Contacts

Rubén Cuesta-Barriuso, PhD

Role: CONTACT

cuestaruben@uniovi.es

0034 985103386

Facility Contacts

Rubén Cuesta-Barriuso, PhD

Role: primary

cuestaruben@uniovi.es

0034 985 103 386

Other Identifiers

He-ProF

Identifier Type: -

Identifier Source: org_study_id