Study into patients’ views of disease’s impact opens in Europe
PROFA uses mobile app, questionnaires to delve into life quality
An observational study in Europe is using a mobile health app to better understand how Friedreich’s ataxia (FA) affects patients’ health-related quality of life, as well as their economic and psychosocial well-being, in real time.
Called PROFA, the study is taking place across six centers in Germany, Austria, and France, and currently enrolling eligible patients, ages 12 and older.
“The in-depth and multidisciplinary real-time data assessment will provide a better understanding of the FA impact on patients’ everyday life, firming the basis for the design of improved care and rehabilitation programmes and future clinical and healthcare research trials,” the researchers wrote.
Findings also aim to “improve the current treatment, care and living situation of patients with FA and their families,” they added.
Friedreich’s ataxia study recruiting about 200 children and adults
A report detailing the study’s protocol, “Patient-reported, health economic and psychosocial outcomes in patients with Friedreich ataxia (PROFA): protocol of an observational study using momentary data assessments via mobile health app,” was published in the journal BMJ Open.
FA is a progressive disease of the nerves and muscles, causing loss of control of movement (ataxia) as well as impairments in vision, hearing, and speech. The condition has a major negative impact on health-related quality of life (HRQoL).
However, a comprehensive analysis of FA’s impact on daily life, HRQoL, and economic resources is lacking. That’s the goal of the PROFA study (NCT05943002), which is recruiting about 200 children and adults diagnosed with FA and with access to a smartphone or a similar digital device.
Additional criteria include a maximum score of 30 points on the Scale for the Assessment and Rating of Ataxia (SARA), an eight-item scale whose scores range from zero (no ataxia) to 40 (severe ataxia).
Participants will be interviewed in person at the study’s start, with follow-up assessments conducted at their homes over six months using a study-specific mobile health app, called Atom5, reported to provide continuous data capture and remote patient monitoring.
Besides data collection via the app, the study will assess HRQoL via the generic EQ-5D-5L questionnaire and its pediatric version EQ-5D-Y-5L, as well as with the ataxia-specific and patient-rated PROM-Ataxia Short Form.
EQ-5D-5L asks respondents to rate their mobility, self care, usual activities, and levels of pain or discomfort, and anxiety or depression. The PROM-Ataxia Short Form evaluates ataxia-related symptoms, including physical and mental health, and daily living activities.
Goal is a ‘comprehensive and better understanding of disease burden’
Clinical parameters will be evaluated in person using the SARA scale and the Inventory of Non-Ataxia Signs. Daily living activities are assessed with the FA Rating Scale and the Cerebellar Cognitive Affective/Schmahmann Syndrome Scale. A version of SARA available through the app will be used monthly.
Speech and communication will be evaluated using several tests, along with psychological well-being and coping strategies.
A new scale, called Communication in Ataxia, will help in assessing patient-rated problems in communication. Use of health services will be analyzed with a modified version of the German Questionnaire for Health-Related Resource Use.
The study, expected to conclude in October 2024, is divided in two parts. Its validation part will analyze the data from the Atom5 app and the self-reported measures in FA patients, whereas in the evaluation part, analysis will focus on the factors influencing daily life.
“The PROFA Study will provide a comprehensive and better understanding of the disease burden of everyday life, determinants of psychosocial health and HRQoL of patients with FA, as well as a detailed description of specific health events, healthcare service utilisation and costs,” the researchers wrote.