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

NCT07099313 | Recruiting

• 1 other identifier: He-ProF
• Study Type: observational
• Target: 114
• Locations: 1 country
• Sites: 1

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

Keywords

Hemophilia; Clotting factor concentrates; Treatment burden; Perceived self-efficacy; Adherence; Locus of control

Outcome Measures

Primary Outcomes (1)

• 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).

  Screening visit.

Secondary Outcomes (6)

• Assessment of perceived self-efficacy in health at the time of the study.

  Screening visit.

• Assessment of adherence to treatment at the time of the study.

  Screening visit.

• Assessment of sleep quality at the time of the study.

  Screening visit.

• Assessment of health locus of control at the time of the study.

  Screening visit.

• Assessment of the sociodemographic variable age at the time of the study.

  Screening visit.

Other Outcomes (3)

• Assessment of the sociodemographic variable educational level at the time of the study.

  Screening visit.

• Assessment of the sociodemographic variable 'living arrangements at the time of the study'.

  Screening visit.

• Assessment of the clinical variable cohabitation at the time of the study.

  Screening visit.

Study Arms (1)

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.

Eligibility Criteria

• Age: 18 Years+
• Sex: male
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patients with hemophilia A or B, undergoing prophylactic treatment with clotting factor concentrates.

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Investigación en Hemofilia y Fisioterapia: lead

Study Sites (1)

Universidad de Oviedo

Oviedo, Principality of Asturias, 33006, Spain

RECRUITING

MeSH Terms

Conditions

Hemophilia A

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Rubén Cuesta-Barriuso, PhD

  Universidad de Oviedo

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Rubén Cuesta-Barriuso, PhD

CONTACT

0034 985103386; cuestaruben@uniovi.es

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: CROSS SECTIONAL
• Sponsor Type: NETWORK
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: July 25, 2025
• First Posted: August 1, 2025
• Study Start: July 31, 2025
• Primary Completion: September 13, 2025
• Study Completion: October 15, 2025
• Last Updated: August 5, 2025

Record last verified: 2025-07

Data Sharing

• IPD Sharing: Will not share

Locations

ES (1)