Extended‑ vs short‑half‑life clotting factor prophylaxis and its impact on treatment burden and psychosocial wellbeing in adults with hemophilia
Association of Prophylactic Treatment With Treatment Burden, Self-efficacy, Adherence, Sleep Quality, and Locus of Control in Patients With Hemophilia; an Ambispective Cohort Study
This project will see if adults with hemophilia who use extended‑half‑life versus short‑half‑life prophylaxis feel a lower treatment burden and have better self‑efficacy, adherence, sleep, and health‑control beliefs.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 114 (estimated) |
| Ages | 18 Years and up |
| Sex | Male |
| Sponsor | Investigación en Hemofilia y Fisioterapia Research network |
| Locations | 1 site (Oviedo, Principality of Asturias) |
| Trial ID | NCT07099313 on ClinicalTrials.gov |
What this trial studies
This is a multicentre ambispective cohort study enrolling 114 adults with hemophilia A or B who have been on a stable prophylactic regimen (either extended‑half‑life or short‑half‑life clotting factor concentrates) for at least six months. The primary outcome is perceived treatment burden measured with a validated treatment burden questionnaire, with secondary measures including self‑efficacy, adherence, sleep quality and health locus of control. Data will be collected from medical records and patient‑completed questionnaires to compare psychosocial profiles between the two treatment groups. Patients with inhibitors or major comorbidities that could confound psychosocial measures are excluded to focus the comparison on treatment type.
Who should consider this trial
Good fit: Adults (age 18+) with a medical diagnosis of hemophilia A or B who have received continuous prophylactic treatment with either short‑half‑life or extended‑half‑life clotting factor concentrates for at least six months and who can complete questionnaires are ideal candidates.
Not a fit: Patients with inhibitors to factor concentrates, major disabling comorbidities, or cognitive/language barriers that prevent reliable questionnaire completion are unlikely to benefit from participating.
Why it matters
Potential benefit: If successful, the findings could help clinicians choose prophylaxis schedules that reduce treatment burden and improve adherence and daily functioning for people with hemophilia.
How similar studies have performed: Extended‑half‑life products have shown equivalent haemostatic efficacy and reduced infusion frequency in prior work, but robust evidence on their psychosocial benefits is limited and inconsistent.
Eligibility criteria
Show full inclusion / exclusion 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.
Where this trial is running
Oviedo, Principality of Asturias
- Universidad de Oviedo — Oviedo, Principality of Asturias, Spain (Recruiting)
Study contacts
- Principal investigator: Rubén Cuesta-Barriuso, PhD — Universidad de Oviedo
- Study coordinator: Rubén Cuesta-Barriuso, PhD
- Email: cuestaruben@uniovi.es
- Phone: 0034 985103386
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.