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

Официальное название

Association of Prophylactic Treatment With Treatment Burden, Self-efficacy, Adherence, Sleep Quality, and Locus of Control in Patients With Hemophilia; an Ambispective Cohort Study

Обзор

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.

Первичные конечные точки

• Assessment of treatment burden at the time of the study. [Срок оценки: Screening visit.]

Вторичные конечные точки (6)

• Assessment of perceived self-efficacy in health at the time of the study. [Срок оценки: Screening visit.]
• Assessment of adherence to treatment at the time of the study. [Срок оценки: Screening visit.]
• Assessment of sleep quality at the time of the study. [Срок оценки: Screening visit.]
• Assessment of health locus of control at the time of the study. [Срок оценки: Screening visit.]
• Assessment of the sociodemographic variable age at the time of the study. [Срок оценки: Screening visit.]
• Assessment of the clinical variable number of weekly infusions of clotting factor concentrates at the time of the study. [Срок оценки: Screening visit.]

Критерии участия

Критерии включения

• 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.

Критерии исключения

• 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.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Модель наблюдения

Когортное

Центры проведения

Испания · 1 центр

• Universidad de Oviedo — Oviedo

Идентификаторы

NCT: NCT07099313 · He-ProF

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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