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Patient-reported outcomes and patient-reported outcome measures in interstitial lung disease: where to go from here?

ABSTRACT: Patient-reported outcome measures (PROMs), tools to assess patient self-report of health status, are now increasingly used in research, care and policymaking. While there are two well-developed disease-specific PROMs for interstitial lung diseases (ILD) and idiopathic pulmonary fibrosis (IPF), many unmet and urgent needs remain. In December 2019, 64 international ILD experts convened in Erice, Italy to deliberate on many topics, including PROMs in ILD. This review summarises the history of PROMs in ILD, shortcomings of the existing tools, challenges of development, validation and implementation of their use in clinical trials, and the discussion held during the meeting. Development of disease-specific PROMs for ILD including IPF with robust methodology and validation in concordance with guidance from regulatory authorities have increased user confidence in PROMs. Minimal clinically important difference for bidirectional changes may need to be developed. Cross-cultural validation and linguistic adaptations are necessary in addition to robust psychometric properties for effective PROM use in multinational clinical trials. PROM burden of use should be reduced through appropriate use of digital technologies and computerised adaptive testing. Active patient engagement in all stages from development, testing, choosing and implementation of PROMs can help improve probability of success and further growth.

SUBMITTER: Kalluri M

PROVIDER: S-EPMC9488962 | biostudies-literature | 2021 Jun

REPOSITORIES: biostudies-literature

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Patient-reported outcomes and patient-reported outcome measures in interstitial lung disease: where to go from here?

Kalluri Meena M Luppi Fabrizio F Vancheri Ada A Vancheri Carlo C Balestro Elisabetta E Varone Francesco F Mogulkoc Nesrin N Cacopardo Giulia G Bargagli Elena E Renzoni Elizabeth E Torrisi Sebastiano S Calvello Mariarosaria M Libra Alessandro A Pavone Mauro M Bonella Francesco F Cottin Vincent V Valenzuela Claudia C Wijsenbeek Marlies M Bendstrup Elisabeth E

European respiratory review : an official journal of the European Respiratory Society 20210525 160

Patient-reported outcome measures (PROMs), tools to assess patient self-report of health status, are now increasingly used in research, care and policymaking. While there are two well-developed disease-specific PROMs for interstitial lung diseases (ILD) and idiopathic pulmonary fibrosis (IPF), many unmet and urgent needs remain. In December 2019, 64 international ILD experts convened in Erice, Italy to deliberate on many topics, including PROMs in ILD. This review summarises the history of PROMs ...[more]

PMID: 34039675

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Project description:PurposeTo analyze time to completion of preoperative legacy patient-reported outcomes (PROs) and more recent computer adaptive Patient-Reported Outcomes Measurement Information System (PROMIS) questionnaires in patients with symptomatic femoroacetabular impingement syndrome undergoing primary hip arthroscopy.MethodsA retrospective analysis was conducted on patients undergoing hip arthroscopy by a single fellowship-trained hip arthroscopist. Inclusion criteria were patients undergoing primary arthroscopic hip surgery and completion of at least 1 legacy PRO or PROMIS questionnaire at the preoperative time point. Exclusion criteria were history of contralateral or ipsilateral hip surgery, non-English-speaking patients, patients who completed PROs by phone or by paper form, and patients who did not complete preoperative PROs. Legacy PROs included modified Harris Hip Score (mHHS), Hip Outcome Score (HOS), International Hip Outcome Tool (iHOT-12), and Hip Pain Visual Analog Scale (VAS-Pain). PROMIS PROs included Physical Function (PROMIS-PF), Pain Interference (PROMIS-PI), and Depression (PROMIS-D). Only preoperative PROs were included in the analysis. Completion time was calculated using the questionnaire start and stop time reported by the survey collecting software. The median and interquartile range of each PRO were reported for analysis of central tendency and statistical dispersion, respectively.ResultsA total of 1,901 patients and 269 patients were included in the legacy and PROMIS groups, respectively. The median time required for completion of each PRO in (minutes: seconds) format was as follows: mHHS (1:29), HOS (3:58), iHOT (2:11), VAS-Pain (0:32), PROMIS-PF (0:46), PROMIS-PI (0:37), and PROMIS-D (0:43). The interquartile range of the middle 50% of respondents was as follows; mHHS (0:58), HOS (2:46), iHOT (1:22), VAS-Pain (0:28), PROMIS-PI (0:19), PROMIS-D (0:29), and PROMIS-PF (0:20).ConclusionsThis study supports that preoperative PROMIS forms require less time to complete than preoperative legacy PROs and are not significantly influenced by age, race, or workers compensation status.Level of evidenceLevel IV, case series.

Project description:BackgroundThe aim of this scoping review is to identify and summarize patient-reported outcome measures (PROMs) that are being used to track long-term patient-reported outcomes (PROs) after injury and can potentially be included in trauma registries.MethodsOnline databases were used to identify studies published between 2013 and 2019, from which we selected 747 articles that involved survivors of acute physical traumatic injury aged 18 years or older at time of injury and used PROMs to evaluate recovery between 6 months and 10 years postinjury. Data were extracted and summarized using descriptive statistics and a narrative synthesis of the results.ResultsMost studies were observational, with relatively small sample sizes, and predominantly on traumatic brain injury or orthopedic patients. The number of PROs assessed per study varied from one to 12, for a total of 2052 PROs extracted, yielding 74 unique constructs (physical health, 25 [34%]; mental health, 27 [37%]; social health, 12 [16%]; cognitive health, 7 [10%]; and quality of life, 3 [4%]). These 74 constructs were assessed using 355 different PROMs. Mental health was the most frequently examined outcome domain followed by physical health. Health-related quality of life, which appeared in more than half of the studies (n = 401), was the most common PRO evaluated, followed by depressive symptoms. Physical health was the domain with the highest number of PROMs used (n = 157), and lower-extremity functionality was the PRO that contributed most PROMs (n = 33).ConclusionWe identified a wide variety of PROMs available to track long-term PROs after injury in five different health domains: physical, mental, social, cognitive, and quality of life. However, efforts to fully understand the health outcomes of trauma patients remain inconsistent and insufficient. Defining PROs that should be prioritized and standardizing the PROMs to measure them will facilitate the incorporation of long-term outcomes in national registries to improve research and quality of care.Level of evidenceSystematic Reviews & Meta-analyses, Level IV.

Project description:BackgroundLate-onset Pompe Disease (LOPD) is a rare, heterogeneous disease manifested by a range of symptoms varying in severity. Research establishing the frequency of these symptoms and their impact on patients' daily lives is limited. The objective of this study was to develop a conceptual model that captures the most relevant symptoms and functional limitations experienced by patients with LOPD, to inform the development of new patient-reported outcome (PRO) tools.MethodsA preliminary conceptual model was constructed following a literature review and revised through interviews with expert clinicians to identify important and relevant concepts regarding symptoms and impacts of LOPD. This preliminary model informed the development of a qualitative patient interview guide, which was used to gather the patient perspective on symptoms and impacts relating to LOPD or its treatment (including symptom/impact frequency and levels of disturbance). Patient interviews aided further refinement of the conceptual model. The findings from the patient interviews were triangulated with the literature review and clinician interviews to identify the most relevant and significant effects of LOPD from the patient perspective.ResultsMuscle weakness, fatigue, pain, and breathing difficulties (especially while lying down) were the most common and highly disturbing symptoms experienced by patients. Limitations associated with mobility (e.g., difficulty rising from a sitting position, getting up after bending) and activities of daily living, (e.g., reduced ability to participate in social/family activities or work/study) were the most frequently reported impacts with the highest levels of disturbance on the patient's daily life. These identified symptoms and impacts were included in the new conceptual model of disease.ConclusionsThis qualitative patient interview study, also informed by a literature review and clinician interviews, identified the most frequent and relevant symptoms and the functional impact of LOPD on patients. The study interviews also captured the patient-preferred language to describe symptoms and impacts of LOPD. The results from this study can be used to develop future PRO instruments that are tailored to the specific symptoms and impacts experienced by patients with LOPD.

Project description:BackgroundObtaining patients' views of their health and outcomes of interventions utilising patient-reported outcome measures (PROMs) is a well-established method, but there is still uncertainty about the impact of PROMs on services and patient care. Studies are now needed of alternative ways of implementing PROMs. This paper describes a case study of the introduction of a new PROM to assess musculoskeletal (MSK) problems, known as the Musculoskeletal Health Questionnaire (MSK-HQ).MethodsFollowing an invitation from the Arthritis Research UK (ARUK), 11 groups and organisations agreed to become 'partners' in piloting the MSK-HQ. Twenty-nine interviews and a focus group were carried out with key informants from the partners. Interviews were supplemented with some documentary evidence of partners' meetings. Data were coded and analysed with NVivo software V.10. Analysis was carried out via a framework method.ResultsParticipants reported positive evidence that the MSK-HQ is feasible and practical for use in patient care with content that helped health professionals identify and address patients' main presenting problems. Although mediated and reported through health professionals' judgments, the questionnaire was also seen as very relevant and acceptable to a wide spectrum of patients.There was also broad support for the view that whilst the MSK-HQ is relevant to individual patient care, it could also, when aggregated, reflect the experiences of patients as a group and be used as evidence for third parties concerned with the provision and commissioning of services.The main difficulties revealed by the case study were in the form of logistics and sustainability. It was recognised that electronic systems would be more effective for administration and data processing but they were not feasible to develop and implement within reasonable timelines and available budgets. A sustainable approach to using the PROM required significant long-term commitment of budget, a coherent system, and active support from diverse organisations.ConclusionsThe current study supports the view that a bottom-up approach is a promising method to generate PROM-related insights that are relevant to patients and health professionals. The partnership approach to developing and using PROMs may have wider relevance and potential as a model of implementation.

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Patient-reported outcomes and patient-reported outcome measures in interstitial lung disease: where to go from here?

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Patient-reported outcomes and patient-reported outcome measures in interstitial lung disease: where to go from here?

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