Effect Of Pulmonary Rehabilitation in Patients With Alpha-1 Antitrypsin Deficiency
- Conditions
- Alpha-1 Antitrypsin Deficiency (AATD)
- Registration Number
- NCT07242079
- Lead Sponsor
- University of Parma
- Brief Summary
This single-center, longitudinal, observational, prospective study aims to assess the applicability, adherence, and clinical impact of the Active Cycle of Breathing Technique (ACBT) with augmented reality support in patients with alpha-1 antitrypsin deficiency (AATD).
A total of 50 adult AATD patients will be recruit from the Lung Function Unit of the University Hospital of Parma, meeting specific inclusion criteria. Participants will perform ACBT twice daily and walk at least 5000 steps per day. Clinical and functional outcomes including dyspnea perception, lung function, and quality of life, will be assessed before and after a six-week ACBT program.
The study explores whether augmented reality enhance adherence and efficacy compared to conventional pulmonary rehabilitation (PR) methods. The expected outcome is improved adherence to PR.
- Detailed Description
Alpha-1 antitrypsin deficiency (AATD) is a genetic disorder caused by mutations in the SERPINA1 gene, located on chromosome 14. The condition is characterized by low circulating levels of alpha-1 antitrypsin (AAT), a glycoprotein primarily produced by hepatocytes, which plays a crucial role in inhibiting neutrophil elastase (NE). Without adequate AAT, unregulated NE activity damages the lung parenchyma, leading to progressive emphysema. AATD presents with variable phenotypic expression, ranging from asymptomatic individuals to those with severe pulmonary and hepatic complications.
Treatment includes intravenous AAT replacement therapy, vaccination and guideline-recommended pulmonary rehabilitation (PR) based on the patient's clinical condition.
PR consists of a structured program combining breathing exercises, physical training, education, and psychological support to optimize respiratory function and improve patients' quality of life. One of the most effective techniques within PR is the Active Cycle of Breathing Technique (ACBT), which enhances airway clearance and lung function. ACBT is a simple, standardized, and home-based technique that patients can perform with or without digital tools. ACBT has already been shown to improve forced vital capacity, peak expiratory flow, arterial oxygenation and exercise capacity.
A tendency towards poor adherence to PR has been reported in the literature. To facilitate the performance of PR procedures, technological evolution in recent decades has brought new complementary techniques such as active video games, virtual reality and augmented reality.
ThIs study aims to assess satisfaction, adherence, and usability of pulmonary rehabilitation techniques in AATD patients, both with and without technological support, through relevant questionnaires (VAS, RAI, USE, TAM). Additionally, it aims to describe changes in the perception of dyspnea (mMRC), quality of life (EuroQol 5), impact of the pathology on daily life, and some functional parameters (FEV1, FVC, R5-R20, LCI, metres walked assessed by the six-minute walking test) at baseline and at the end of the rehabilitation program.
Data will be collected in a dedicated electronic Clinical Records Form (CRF). The database will be saved on a password-protected company Personal Computer (PC) which will be updated at each visit and used exclusively for scientific research purposes. At the time of enrollment, each patient will receive an alphanumeric code so that any information collected during the study, and in particular sensitive data, is treated in an anonymous manner. Data reporting patients' identifications will only be used to file patients and collect informed consent.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 50
- Male or female adults aged ≥18 years;
- Signed informed consent;
- All AATD patients, regardless of nephelometric alpha-1 antitrypsin dose and clinical phenotype, who require PR according to guidelines
- Subjects unable to perform the lung function tests and rehabilitation program required by the protocol
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Patient satisfaction with PR techniques using Technology Acceptance Model (TAM) questionnaire. six weeks Satisfaction will be assessed using the Technology Acceptance Model (TAM) questionnaire, with scores ranging from 1 to 7, where lower scores indicate more satisfaction.
Patient adherence to the PR techniques using the Rehabilitation Adherence Index (RAI) questionnaire. six weeks Adherence will be assessed using the Rehabilitation Adherence Index (RAI) questionnaire, with scores ranging from 1 to 7, where higher scores indicate better adherence.
Usability of pulmonary rehabilitation techniques using the Usefulness, Satisfaction and Ease of Use (USE) questionnaire six weeks Usability will be measured using the Usefulness, Satisfaction and Ease of Use (USE) questionnaire, with scores ranging from 1 to 7, where higher scores indicate better usability.
- Secondary Outcome Measures
Name Time Method To describe the change in perception of dyspnea at baseline and at the end of the PR using Modified Medical Research Council (mMRC) questionnaire. six weeks The Modified Medical Research Council (mMRC) questionnaire assesses the perception of dyspnea with scores ranging from 0 to 4, with higher scores indicating greater severity.
To describe the change in quality of life at baseline and at the end of the PR using EuroQol-5D (EQ-5D) questionnaire. six weeks The EQ-5D questionnaire assesses health-related quality of life across five dimensions: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. Each dimension has five levels of severity: no problems, slight problems, moderate problems, severe problems, and unable to perform/extreme problems. Additionally, the EQ-5D includes a visual analogue scale (VAS) where respondents rate their overall health on a scale from 0 to 100, with 0 being the worst imaginable health and 100 being the best.
To assess the impact of Chronic Obstructive Pulmonary Disease (COPD) on AATD- related COPD patients using COPD Assessment Test (CAT) six weeks The COPD Assessment Test (CAT) is assess the impact of Chronic Obstructive Pulmonary Disease (COPD) on a person's daily life. It ranges from 0 to 5, where higher scores indicate a greater impact of COPD on daily life
To assess the impact of asthma on AATD patients with asthma using the Asthma Control Test (ACT) six weeks The Asthma Control Test (ACT) is a questionnaire used to assess asthma control, with scores ranging from 5 to 25. Higher scores indicate better asthma control, meaning the asthma is well-managed and less likely to interfere with daily activities.
To describe the change in functional respiratory parameters at baseline and at the end of the PR program using spirometry. six weeks Spirometry will be performed according to ATS/ERS standards to obtain Forced Expiratory Volume in 1 second (FEV₁, % predicted), Forced Vital Capacity (FVC, % predicted), and FEV₁/FVC, %.
Trial Locations
- Locations (1)
University of Parma
🇮🇹Parma, Italy, Italy
University of Parma🇮🇹Parma, Italy, Italy