Medical Decision Making in Multiple System Atrophy

Официальное название

Medical Decision Making in Multiple System Atrophy: Developing Personalized Best Medical Care with Integrated Telemedicine and Mobile Palliative Care for Individuals with Multiple System Atrophy

Обзор

The goal of this clinical study is to evaluate the effects of a personalized symptomatic treatment plan integrated with monthly telemedicine and mobile palliative care interventions on a population of individuals diagnosed with Multiple System Atrophy (MSA) and their informal caregivers. The aim is to improve the quality of life of MSA patients and their caregivers, as well as provide them with better support during the disease progression. After a baseline visit, all 46 patients will receive a personalized therapeutic plan (including medical treatment, physiotherapy, logotherapy and occupational therapy excercises and psychological support) and contact with social workers and a palliative care team. They willl then be re-evaluated at 6-,12-, 18- month visits. Semi-structured online interviews at baseline and 12 month visit will collect patients' individual healthcare preferences, which will be taken into account in the preparation of the individual therapeutic plan. Twenty-three patients will be randomized to receive monthly telemedicine visits. Assessment of patients´satisfaction with the therapeutic plan, with the palliative interventions (when they occurred) and the telemedicine visits will be carried over the 18 month period. Forty-six informal caregivers will be invited to participate with semi-structured online interviews and assessment of their QoL and caregivers' burden.

Подробное описание

This is a monocentric, 18-months, randomized, rater-blinded study to evaluate the influence of a multidisciplinary, personalized symptomatic treatment plan with integrated mobile palliative care and telemedicine on the baseline to 18-months change in the QoL of individuals with MSA compared to a sex-, age- and disease-duration matched historical European MSA cohort, whose data is stored at the Medical University of Innsbruck.

We plan to recruit 46 individuals with MSA fulfilling all the inclusionand none of the exclusion criteria. Upon collection of written informed consent, the recruited individuals will be instructed to complete a falls protocol referred to the month preceding the baseline visit, as well as a blood pressure (BP), bladder diary for up to 72 hours prior to the baseline visit. They will subsequently undergo a baseline examination including a comprehensive clinical, psychological and neuro-rehabilitation assessment, as well as an online semi-structured interview aimed at pinpointing the individual therapeutic needs and healthcare preferences. In case additional examinations are needed, these will be carried out on the same day of the baseline visit or, if not possible for individual or appointment reasons, at the earliest possible time point within the given timeframe as outlined in the visit schedule. Upon completion of the baseline visit and examinations, the individualized therapeutic plan, including mobile palliative care offer (for wheelchair-bound individuals) and guidance for self-practiced physio-, speech and occupational exercises will be prepared based on a standardized operational protocol drafted by the study team on the basis of published consensus recommendations, scientific evidence and principles of good clinical practice and adapted to the individual healthcare preferences and therapeutic needs of the recruited individuals.

Six, 12 and 18 months after the baseline visit, in-person visits will be scheduled, including a comprehensive clinical, psychological and neuro-rehabilitation re-assessment of the individual therapeutic needs. Therapeutic adaptations will be made following the standardized operational protocol and adapted to the individual healthcare preferences. In the event that the enrolled subjects are for legitimate reasons unable to travel to hospital, they will be offered the opportunity to complete the scheduled on-site assessments of month 6, 12, and 18 using the CHES online platform.

At month 12 the online semi-structured interview will be repeated to assess for eventual changes in the individual healthcare preferences due to the disease progression.

At month 1, 7 and 13, follow-up phone calls will be scheduled to verify the compliance with the individualized treatment plan and identify barriers to its application.

At month 1, 7, 13 and 18, the study participants will be invited to complete online satisfaction surveys with the overall individualized treatment plan.

Twenty-three patients will be block-wise randomized to receive additional monthly and on-demand neurological, psychological, physio-, occupational and speech therapy (based on individual needs) telemedicine visits through the CHES teleconsultation facility of the Tirol Kliniken.

Upon completion of the telemedicine visit (or on-demand mobile palliative interventions, for wheelchair-bound individuals, whenever needed and wished), brief satisfaction surveys will be sent to the study participants through the CHES platform.

Informal caregivers of the individuals with MSA recruited in the present study will be invited to participate upon written informed consent in an 18 months observational study, with baseline and 12 months semi-structured online interviews and baseline, 6-, 12- and 18 months assessment of their QoL and caregivers' burden.

Вмешательства

• Другое Telemedicine visits
  Monthly telemedicine neurological, psychological and neurorehabilitation (physio-, occupational and speech therapy) consultations
• Другое Multidisciplinary, personalized symptomatic treatment
  Multidisciplinary, personalized symptomatic treatment plus mobile palliative care interventions (if wished and needed)

Первичные конечные точки

• Change in the EQ-5D-5L score [Срок оценки: Baseline to 18-months]

Вторичные конечные точки (12)

• Changes in the MSA-QoL score and subscores [Срок оценки: Baseline to 6-, 12- and 18-months]
• Change in motor and non-motor scales [Срок оценки: Baseline to 6-, 12- and 18-months]
• Time to clinical milestones [Срок оценки: over the 18-months study period]
• Changes in the individual healthcare preferences assessed by means of the Autonomy Preference Index (API) [Срок оценки: Baseline to 12- months]
• In-person and telemedicine medical, psychological and neurorehabilitation interventional needs of individuals with MSA [Срок оценки: over the 18-months study period]
• Change in the Short Assessment of Patient Satisfaction (SAPS) referred to the overall individualized treatment plan [Срок оценки: Month 1 to 7, 13 and 18]
• Individual satisfaction with the single telemedicine and mobile palliative interventions [Срок оценки: over the 18-months study period]
• Healthcare professionals satisfaction with the single telemedicine and mobile palliative interventions [Срок оценки: over the 18-months study period]
• Number of medical complications [Срок оценки: over the 18-months study period]
• Single-intervention and cumulative healthcare costs [Срок оценки: over the 18-months study period]
• Changes in the EQ-5D-5L score of informal caregivers of individuals with MSA recruited in the present study [Срок оценки: Baseline to 6-, 12- and 18-months]
• Change in the Carers quality-of-life questionnaire for parkinsonism (PQoL Carers) score and other caregiver-burden indicators in informal caregivers of individuals with MSA recruited in the present study [Срок оценки: Baseline to 6-, 12- and 18-months]

Критерии участия

Критерии включения

For individuals with MSA:

• Age ≥30 years at the time of consent;
• Diagnosed with clinically probable or clinically established MSA according to the current MDS MSA criteria (1);
• Life-expectancy of at least 24 months as assessed by the investigator at the time of consent;
• Understands and agrees to comply with the study procedures and provides written informed consent (Note: a legal representative may NOT provide consent on behalf of the subject);
• Signed and dated informed consent document;
• Fluency in German;
• If not able to walk or stand without assistance/support at the time of consent, the participant lives in Tyrol or in another Austrian Region with available mobile palliative care.

For informal caregivers:

• Informal caregiver (i.e. person not receiving payment for his/her caregiving) of an individual with MSA recruited in the present study;
• Life-expectancy of at least 24 months as assessed by the investigator at the time of consent;
• Age≥ 18 years at the time of consent;
• Understands and agrees to provide information as outlined in the study protocol and to engage in semi-structured online interviews;
• Provides signed and dated written informed consent;
• Full legal capacity;
• Fluency in German.

Критерии исключения

For individuals with MSA

• Participation in an interventional clinical study at screening and throughout the study that would interfere with the MeDeMSA Care personalized treatment plan or would not permit telemedicine and mobile palliative care strategies;
• Charlson comorbidity index >4 at the time of consent;
• Other major underlying medical conditions that may confound interpretation of study results as assessed by the investigator.

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

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Рандомизированное

Модель

Параллельные группы

Маскирование

Открытое

Основная цель

Поддерживающая терапия

Центры проведения

Австрия · 1 центр

• Innsbruck Medical University — Innsbruck

Публикации

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• Wenning GK, Krismer F. Multiple system atrophy. Handb Clin Neurol. 2013;117:229-41. doi: 10.1016/B978-0-444-53491-0.00019-5. PMID 24095129
• Kwakkenbos L, Fransen J, Vonk MC, Becker ES, Jeurissen M, van den Hoogen FH, van den Ende CH. A comparison of the measurement properties and estimation of minimal important differences of the EQ-5D and SF-6D utility measures in patients with systemic sclerosis. Clin Exp Rheumatol. 2013 Mar-Apr;31(2 Suppl 76):50-6. Epub 2013 Jul 22. PMID 23910610
• Fanciulli A, Wenning GK. Multiple-system atrophy. N Engl J Med. 2015 Jan 15;372(3):249-63. doi: 10.1056/NEJMra1311488. No abstract available. PMID 25587949
• Cortelli P, Calandra-Buonaura G, Benarroch EE, Giannini G, Iranzo A, Low PA, Martinelli P, Provini F, Quinn N, Tolosa E, Wenning GK, Abbruzzese G, Bower P, Alfonsi E, Ghorayeb I, Ozawa T, Pacchetti C, Pozzi NG, Vicini C, Antonini A, Bhatia KP, Bonavita J, Kaufmann H, Pellecchia MT, Pizzorni N, Schindler A, Tison F, Vignatelli L, Meissner WG. Stridor in multiple system atrophy: Consensus statement PMID 31570638
• Calandra-Buonaura G, Alfonsi E, Vignatelli L, Benarroch EE, Giannini G, Iranzo A, Low PA, Martinelli P, Provini F, Quinn N, Tolosa E, Wenning GK, Abbruzzese G, Bower P, Antonini A, Bhatia KP, Bonavita J, Pellecchia MT, Pizzorni N, Tison F, Ghorayeb I, Meissner WG, Ozawa T, Pacchetti C, Pozzi NG, Vicini C, Schindler A, Cortelli P, Kaufmann H. Dysphagia in multiple system atrophy consensus statement PMID 33839029
• Brignole M, Moya A, de Lange FJ, Deharo JC, Elliott PM, Fanciulli A, Fedorowski A, Furlan R, Kenny RA, Martin A, Probst V, Reed MJ, Rice CP, Sutton R, Ungar A, van Dijk JG; ESC Scientific Document Group. 2018 ESC Guidelines for the diagnosis and management of syncope. Eur Heart J. 2018 Jun 1;39(21):1883-1948. doi: 10.1093/eurheartj/ehy037. No abstract available. PMID 29562304
• Gibbons CH, Schmidt P, Biaggioni I, Frazier-Mills C, Freeman R, Isaacson S, Karabin B, Kuritzky L, Lew M, Low P, Mehdirad A, Raj SR, Vernino S, Kaufmann H. The recommendations of a consensus panel for the screening, diagnosis, and treatment of neurogenic orthostatic hypotension and associated supine hypertension. J Neurol. 2017 Aug;264(8):1567-1582. doi: 10.1007/s00415-016-8375-x. Epub 2017 Jan 3. PMID 28050656

Идентификаторы

NCT: NCT06072105 · 1225/2023

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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