Unicenter, proof-of-concept, prospective, randomised, controlled, open-label and blinded end-point adjudication trial to assess the effect on patient-reported outcomes measures (PROMs), patient-reported experience measures (PREMs) and clinical events of a mHealth-based comprehensive management program for patients with chronic heart failure (HF) due to transthyretin-associated amyloidosis (ATTR)-cardiomyopathy (CM) by means of remote daily telemonitoring of signs and symptoms and remote structured follow-up using videoconference.
Background and Rationale: Early diagnosis as well as optimal care of transthyretin-associated amyloidosis (ATTR)-cardiomyopathy (CM) is crucial to improve the outcome of these patients. In order to perform a personalised and patient-centred ATTR-CM, it is essential to integrate new advanced diagnostic and monitoring tools to the pre-existing ones. In this regard, electronic health (eHealth)-based solutions have been gradually being incorporated into the follow-up of chronic patients (e.g. heart failure \[HF\] patients, among others) over the last few years. Objectives: The main objective of this study is to improve the process of care, diagnosis and monitoring of the patient with ATTR-CM by means of a multidisciplinary and transversal model using new digital technologies and optimizing work processes (eHealth-based healthcare management). The primary objective of the study will be the changes in patient-reported outcomes (PROMs) such as self-care and quality of life (QoL) between patients followed and monitored using the mHealth-based digital tool (mHealth) and those in standard follow-up (usual care \[UC\]). Key secondary objectives will include changes in patient-reported experience measures (PREMs), the occurrence of events, among others between those with and without mHealth-base-ATTR management. Methodology: The eOSS-ATTR study will be a study with 2 phases: i) #Phase 1: a design, implantation and pre-clinical evaluation of the digital platform; and ii) #Phase 2: a subsequent clinical evaluation phase. The clinical phase (#Phase 2) will be a unicenter, proof-of-concept, prospective, randomised, controlled, open-label and blinded end-point adjudication trial to assess the effect on PROMs, PREMs and clinical events of a mHealth-based comprehensive management program for patients with HF due to ATTR-CM by means of remote daily telemonitoring of signs and symptoms and remote structured follow-up using videoconference. Regarding #Phase 1, it is proposed to evolve the MAST (Model for Assessment of Telemedicine) model, adding the consideration of the stakeholders involved in the use of this technology, as well as the different use cases and scenarios. All assessed items will be compared before and after the eHealth solution implementation. Concering #Pase 2, HF patients due to ATTR-CM with a HF decompensation in the last 12 months will be randomised (1:1) to structured follow-up based on face-to-face appointments (UC group) or the delivery of health care using mHealth (mHealth group). Randomisation will be stratified at each ATTR subtype (variant \[ATTRv\] or wild-type form \[ATTRwt\] and according to the presence or absence of frailty to ensure balanced assignment of frail patients to each group. The follow-up will be of 6 months in two groups and will be nurse-led and based on mHealth solution designed and modeled for the follow-up of patients with ATTR-CM in the mHealth group. This technological solution is proposed to improve the care monitoring and outcomes of ATTR-CM patients. Patients will be recruited for 12 months. Expected Results: eOSS-ATTR study is the first trial to integrate and assess mHealth solutions combining telemonitoring and structured teleintervention via videoconference in 'real-world' HF patients due to ATTR-CM to improve clinical outcomes and new clinical events. Its great value is that it assesses 'real-world' patients in terms of frailty, comorbidity, literacy level, skills with new technologies among others. Such strategies are of enormous value for the prognosis, QoL and experience of patients with HF due to ATTR-CM. Integration of solutions based on mHealth into day-to-day HF monitoring with user-friendly technology that provides the possibility of direct contact with their care team can contribute to a clear clear improvement in PROMs and PREMs as well as a reduction of new clinical events. It will allow us to optimise the care of HF due to ATTR-CM patients in this period in terms of self-care education as well as treatment optimisation and empowerment in disease management.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
HEALTH_SERVICES_RESEARCH
Masking
NONE
Enrollment
50
Follow-up and treatment of patients in the mHealth arm will be based on a comprehensive solution for the care and monitoring of chronic patients, modelled and tested in chronic HF patients. This is the PIRENe platform, previously described and evaluated in HF patients. It is a digital solution, external to the centre's information systems (external platform). However, it has some special features in its design in terms of remote monitoring, educational tools, among others, expressly conceived and designed for patients with ATTR-CM. This platform allows the provision of multichannel service and monitoring of patients through: 1. Patient monitoring of: Biometric data and symptom report. 2. Generation and management of warning alarms notified to the professionals assigned to each patient in case one of the following situations occurs. 3. Follow-up via videoconference.
Improvement of self-care using a validated scale (European Heart Failure Self-Care Behavior Scale).
changes in patient-reported outcomes (PROMs) such as self-care European Heart Failure Self-Care Behaviour Scale) between patients followed and monitored using the mHealth-based digital tool (mHealth) and those in standard follow-up (usual care \[UC\]).
Time frame: Six months after inclusion of the patient
Improvement of quality of life using a validated questionnaire (EUROQOL - 5D).
Comparison of both strategies at the end of follow-up.
Time frame: Six months after inclusion of the patient
Occurrence of new clinical events (readmission [all-cause, HF, CV and non-CV] rate and total number; worsening HF events; urgent HF visits; all-cause, CV and non-CV death).
Comparison of both strategies at the end of follow-up.
Time frame: Six months after inclusion of the patient
Incidence of implantation of devices (pacemakers, cardiac resynchronization therapy [CRT] or implantable cardioverter defibrillator [ICD])
Comparison of both strategies at the end of follow-up.
Time frame: Six months after inclusion of the patient
Change of NYHA functional class
Comparison of both strategies at the end of follow-up.
Time frame: Six months after inclusion of the patient
Change of 6-minutes walking test [6MWT] distance.
Comparison of both strategies at the end of follow-up.
Time frame: Six months after inclusion of the patient
Evolution of ATTR-CM stages
Comparison of both strategies at the end of follow-up.
Time frame: Six months after inclusion of the patient
Changes in prognostic biomarkers: atrial natriuretic peptide levels (brain natriuretic peptide [BNP] and/or N-terminal-pro-brain natriuretic peptide [NT-proBNP]) and troponin levels (troponin T [TnT] and/or troponin I [TnI]).
Comparison of both strategies at the end of follow-up.
Time frame: Six months after inclusion of the patient
Improvement of patient reported experience measures such as IEXPAC questionnaire (Instrumento de Evaluación de la eXperiencia del PAciente Crónico) or Evaluation Tool of the Chronic Patient Experience and patient satisfaction (Net Promoter Score [NPS]).
Comparison of both strategies at the end of follow-up.
Time frame: Six months after inclusion of the patient
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