An Electronic Patient-Reported Outcome Mobile App for Data Collection in Type A Hemophilia: Design and Usability Study.

BACKGROUND: There is currently limited evidence on the level and intensity of physical activity in individuals with hemophilia A. Mobile technologies can offer a rigorous and reliable alternative to support data collection processes but they are often associated with poor user retention. The lack of longitudinal continuity in their use can be partly attributed to the insufficient consideration of stakeholder inputs in the development process of mobile apps. Several user-centered models have been proposed to guarantee that a thorough knowledge of the end user needs is considered in the development process of mobile apps. OBJECTIVE: The aim of this study is to design and validate an electronic patient-reported outcome mobile app that requires sustained active input by individuals during POWER, an observational study that aims at evaluating the relationship between physical activity levels and bleeding in patients with hemophilia A. METHODS: We adopted a user-centered design and engaged several stakeholders in the development and usability testing of this mobile app. During the concept generation and ideation phase, we organized a need-assessment focus group (FG) with patient representatives to elicit specific design requirements for the end users. We then conducted 2 exploratory FGs to seek additional inputs for the app's improvement and 2 confirmatory FGs to validate the app and test its usability in the field through the mobile health app usability questionnaire. RESULTS: The findings from the thematic analysis of the need-assessment FG revealed that there was a demand for sense making, for simplification of app functionalities, for maximizing integration, and for minimizing the feeling of external control. Participants involved in the later stages of the design refinement contributed to improving the design further by upgrading the app's layout and making the experience with the app more efficient through functions such as chatbots and visual feedback on the number of hours a wearable device had been worn, to ensure that the observed data were actually registered. The end users rated the app highly during the quantitative assessment, with an average mobile health app usability questionnaire score of 5.32 (SD 0.66; range 4.44-6.23) and 6.20 (SD 0.43; range 5.72-6.88) out of 7 in the 2 iterative usability testing cycles. CONCLUSIONS: The results of the usability test indicated a high, growing satisfaction with the electronic patient-reported outcome app. The adoption of a thorough user-centered design process using several types of FGs helped maximize the likelihood of sustained retention of the app's users and made it fit for data collection of relevant outcomes in the observational POWER study. The continuous use of the app and the actual level of engagement will be evaluated during the ongoing trial. TRIAL REGISTRATION: ClinicalTrials.gov NCT04165135; https://clinicaltrials.gov/ct2/show/NCT04165135.

Raising awareness on physician-patient communication in IPF: an Italian multicenter study exploring the pulmonologist's perspective.

BACKGROUND: Idiopathic pulmonary fibrosis (IPF) remains a debilitating, poor prognosis disease requiring a patient-centered approach. OBJECTIVES: To explore the pulmonologist's perspective on physician-patient communication. METHODS: A faculty of psychologists and pulmonologists organized a training course consisting of two workshops 12 months apart. Self-assessment questionnaires (pre- and post-course), role play (RP) simulations (during both workshops) and clinical consultation observations followed by semi-structured interviews (during the 12 months) were employed to evaluate the pulmonologists' knowledge of patient-centered medicine and communication/relational skills (questionnaires), their communication style (RP) and possible communication/relational difficulties (semi-structured interviews). RESULTS: Twenty-three pulmonologists attended the first workshop and 14 the second one; 10 attended both. The questionnaires revealed the interest in patient-centered medicine and communication but also the need for deeper knowledge and improved skills. From the RP sessions performed during the first workshop, a disease-oriented approach emerged; notably, after the training, some improvements suggested a more patient-centered approach, e.g., a more frequent exploration of the patient agenda. Finally, the semi-structured interviews allowed to identify the low patients' cultural level and the poor general knowledge of IPF among the barriers hampering an effective communication with the clinician, who, however, is responsible for overcoming these obstacles. CONCLUSIONS: Despite the overall disease-prone approach to IPF patients, there was room for improvement through adequate training, which, in practice, may ameliorate communication and drive towards patient-centeredness. Exploring the pulmonologists' needs may help tailoring training interventions. Raising awareness on these topics is crucial to ensure IPF patients optimal care.

PerFECT 2.0: A Web-Based Platform Designed to Facilitate and Support the Diagnosis of Patients with Idiopathic Pulmonary Fibrosis in Italy.

INTRODUCTION: Timely and accurate diagnosis of idiopathic pulmonary fibrosis (IPF) is challenging, requiring specific tests including chest high-resolution computed tomography (HRCT), and limited by access to specialist centres with a multidisciplinary team (MDT). Here we describe PerFECT 2.0, an Italian web-based platform designed to create a network between tertiary centres with an MDT (hubs) and secondary centres (spokes), aiming to facilitate the diagnosis of IPF. METHODS: PerFECT 2.0 went live on 1 November 2016. Spoke centres submit anonymised documentation (HRCT images, pathological samples, clinical data) for a second opinion on the potential diagnosis of IPF from a hub centre. HRCT images are quickly uploaded, with patient-identifying information automatically removed. The hub centre views documentation online (no downloads allowed), makes any further information requests, then returns their second opinion as free text. An e-learning area contains educational material and simulated training clinical cases. Metrics were collected for 2017-2019; a user survey was conducted from 30 June-31 July 2020. RESULTS: Ten hub centres and 137 spoke centres have registered. The requests for a second opinion numbered 251 in 2017, 270 in 2018 and 265 in 2019 (overall mean 19.9 requests per month). The proportion of requests answered was 100.0% (251) in 2017, 100.0% (270) in 2018 and 97.7% (259) in 2019. The mean response time was 15.7 days. In the user survey, of nine hub responders and 19 spoke responders, 78% and 74%, respectively, reported that the platform is easy to use, and 100% and 89%, respectively, would recommend the platform to colleagues. CONCLUSION: The PerFECT 2.0 web-based platform has created a network that enables secondary centres to gain quick and easy access to a second opinion from a tertiary centre with an MDT through online evaluation of anonymised documentation, thereby facilitating and supporting the timely and accurate diagnosis of IPF.

Epidemiology of idiopathic pulmonary fibrosis: a population-based study in primary care.

Idiopathic pulmonary fibrosis (IPF) is a chronic disease with unknown etiology and poor prognosis. Little is known about the epidemiology of this disease; most of the studies are limited by small and restricted cohort studies. We aim to investigate the epidemiology of IPF in the Italian primary care setting using the Health Search Database (HSD) between January 2002 and June 2017. In an attempt to define cases of IPF we adopted iterative combinations of International Classification of Diseases Ninth Revision (ICD-9-CM) and other clinical investigations according to three different operational Algorithms. Incidence and prevalence rate, according to the three Algorithms defining IPF, were calculated and the association with candidate determinants [sex, age, gastro-esophageal reflux (GERD) and smoking status] was evaluated. We identified 1,104,307 eligible patients. The prevalence rate of IPF varies between 2.6 to 24.3 per 100.000 person-year, using algorithm 1 and from 0.8 to 7 using algorithm 3. The incidence rate of IPF varies between 1.25 and 3.77 per 100.000 person-years, using algorithm 1 and from 0.10 to 1.61 using algorithm 3. The mean adjusted incidence rate ratio of IPF, using algorithm 1, is 2.33 (95% CI 2.11-2.57) per 100.000 person-years. Over the study years, the trend of prevalence was statistically significantly increasing while the incidence rate started to increase in the last 3 years. The analyses on candidate determinants showed that patients aged 61 years or older, those suffering from GERD, and former smokers were statistically significantly at greater risk of incurring IPF. To our knowledge, this is one of the first European IPF epidemiological studies conducted in primary care. The increase of the incidence rates is likely due to a growing awareness for IPF among General Practitioners, while the increase of prevalence rates may be due to an increase of survival, a result of recent advances in the diagnosis, management and therapies for the disease.

Pirfenidone in real life: A retrospective observational multicentre study in Italian patients with idiopathic pulmonary fibrosis.

RATIONALE: Real-world data on pirfenidone treatment of patients with idiopathic pulmonary fibrosis (IPF) are limited. This study assessed the effectiveness of pirfenidone in a large real-life Italian IPF cohort. METHODS: IRENE was an observational, retrospective study of patients with IPF treated with pirfenidone in routine clinical practice (18 centres). At Month 6, a mandatory re-evaluation of forced vital capacity (FVC) decline (absolute change < 10%) was required to continue pirfenidone. The primary effectiveness outcomes were absolute change from baseline in FVC and the percentage of patients with ≥ 10% absolute decline in % predicted FVC at Month 12. Safety was described by adverse event (AE) occurrence. Prespecified subgroups included sex, age, presence/absence of emphysema, usual interstitial pneumonia (UIP) pattern on high-resolution computed tomography, and baseline lung function. RESULTS: The study included 379 patients (mean age, 67.6 years; 78.1% male). Mean change from baseline in FVC and the percentage of patients with ≥ 10% absolute decline in % predicted FVC at Month 12 were -81.8 mL (SD, 419.6 mL; P = 0.002) and 16.0% (95% CI, 12.2-20.9%), respectively. Disease progression was similar across prespecified subgroups, including patients with definite vs possible UIP. Overall, 211 AEs occurred in 149 patients (39.3%), with serious AEs in 31 patients (8.2%) and 9 discontinuations due to AEs. Skin and gastrointestinal AEs were most frequent. Fifteen patients (4.0%) died. CONCLUSIONS: The decline in FVC and the safety profile observed in this real-world IPF cohort were consistent with the findings of the Phase III pirfenidone trials.