Identifying Existing Guidelines, Frameworks, Checklists, and Recommendations for Implementing Patient-Reported Outcome Measures: Protocol for a Scoping Review.

BACKGROUND: Implementing patient-reported outcome measures (PROMs) to measure and evaluate health outcomes is increasing worldwide. Along with this emerging trend, it is important to identify which guidelines, frameworks, checklists, and recommendations exist, and if and how they have been used in implementing PROMs, especially in clinical quality registries (CQRs). OBJECTIVE: This review aims to identify existing publications, as well as publications that discuss the application of actual guidelines, frameworks, checklists, and recommendations on PROMs' implementation for various purposes such as clinical trials, clinical practice, and CQRs. In addition, the identified publications will be used to guide the development of a new guideline for PROMs' implementation in CQRs, which is the aim of the broader project. METHODS: A literature search of the databases MEDLINE, Embase, CINAHL, PsycINFO, and Cochrane Central Register of Controlled Trials will be conducted since the inception of the databases, in addition to using Google Scholar and gray literature to identify literature for the scoping review. Predefined inclusion and exclusion criteria will be used for all phases of screening. Existing publications of guidelines, frameworks, checklists, recommendations, and publications discussing the application of those methodologies for implementing PROMs in clinical trials, clinical practice, and CQRs will be included in the final review. Data relating to bibliographic information, aim, the purpose of PROMs use (clinical trial, practice, or registries), name of guideline, framework, checklist and recommendations, the rationale for development, and their purpose and implications will be extracted. Additionally, for publications of actual methodologies, aspects or domains of PROMs' implementation will be extracted. A narrative synthesis of included publications will be conducted. RESULTS: The electronic database searches were completed in March 2024. Title and abstract screening, full-text screening, and data extraction will be completed in May 2024. The review is expected to be completed by the end of August 2024. CONCLUSIONS: The findings of this scoping review will provide evidence on any existing methodologies and tools for PROMs' implementation in clinical trials, clinical practice, and CQRs. It is anticipated that the publications will help us guide the development of a new guideline for PROMs' implementation in CQRs. TRIAL REGISTRATION: PROSPERO CRD42022366085; https://tinyurl.com/bdesk98x. INTERNATIONAL REGISTERED REPORT IDENTIFIER (IRRID): DERR1-10.2196/52572.

Patient-Reported Outcome Measures in High-Risk Medical Device Registries: A Scoping Review.

Little is known about the methods and outcomes of patient-reported outcome measure (PROM) use among high-risk medical device registries. The objective of this scoping review was to assess the utility and predictive ability of PROMs in high-risk medical device registries. We searched Ovid Medline, Embase, APA PsychINFO, Cochrane Library, and Scopus databases for published literature. After searching, 4323 titles and abstracts were screened, and 262 full texts were assessed for their eligibility. Seventy-six papers from across orthopedic (n = 64), cardiac (n = 10), penile (n = 1), and hernia mesh (n = 1) device registries were identified. Studies predominantly used PROMs as an outcome measure when comparing cohorts or surgical approaches (n = 45) or to compare time points (n = 13) including pre- and postintervention. Fifteen papers considered the predictive ability of PROMs. Of these, 8 treated PROMs as an outcome, 5 treated PROMs as a risk factor through regression analysis, and 2 papers treated PROMs as both a risk factor and as an outcome. One paper described PROMs to study implant survival. To advance methods of PROM integration into clinical decision-making for medical devices, an understanding of their use in high-risk device registries is needed. This scoping review found that there is a paucity of studies using PROMs to predict long-term patient and clinical outcomes in high-risk medical device registries. Determination as to why PROMs are rarely used for predictive purposes in long-term data collection is needed if PROM data are to be considered suitable as real-world evidence for high-risk device regulatory purposes, as well as to support clinical decision-making.

Comparing Patient-Reported Outcome Measures for Pain in Women With Pelvic Floor Disorders: A Scoping Review.

Women with pelvic floor disorders (PFDs) typically undergo surgery involving transvaginal mesh implants; however, transvaginal mesh surgery is associated with many adverse events including post-surgical pain. Assessment of pain as a symptom is necessary using patient-reported outcome measures (PROMs). This scoping review aimed to describe and compare existing PROMs previously used to measure pain in women with PFDs. A scoping search of Ovid MEDLINE, EMBASE, CINAHL Plus, Ovid PsycInfo, and grey literature was conducted. Studies published in English describing the development, implementation, and application of PFD-associated pain PROMs among adult women before and after pelvic floor surgery were included. From each article, a descriptive summary organised by study first author, publication year, country, setting, aim, study characteristics, and results were extracted. From 2,604 articles identified, 54 studies were included, describing 44 PROMs. Most studies described at least 2 to 3 instruments. The Pelvic Floor Distress Inventory-20 was most commonly described by 12 studies followed by the Patient Global Impression of Improvement scale. Of the 44 PROMs, 27 were condition-specific, of which 9 included items on pain; 17 generic PROMs, 4 of which contained items on pain; and 6 pain-specific PROMs. PROMs identified in our review measured pain not related to PFDs or pelvic floor surgery and quantified the pain experienced. These PROMs did not measure all areas of pain including region, sensation, impact, and triggers. The findings of this review will assist with developing a new pain-specific PROM in this population. PERSPECTIVE: Women with pelvic floor disorders often undergo surgery involving transvaginal mesh, from which they experience debilitating pain. Pain is a major issue impacting women's lives. Patient-reported outcome measures can be used to assess the pain; however, it is unclear whether existing instruments are relevant.

Informing a national rare disease registry strategy in Australia: a mixed methods study.

BACKGROUND: Rare disease registries (RDRs) facilitate monitoring of rare diseases by pooling small datasets to increase clinical and epidemiological knowledge of rare diseases and promote patient centred best practice. The aim of this study was to understand the current state of RDRs in Australia, data captured, impact on patient outcomes, funding models, and barriers and enablers regarding their establishment and maintenance. METHODS: An exploratory sequential mixed methods study design was adopted. First, a list of Australian RDRs, primary contacts and data custodians was generated through online and consumer group (Rare Voices Australia (RVA)) contacts. A cross-sectional, anonymous online survey was distributed to registry custodians, managers, or principal investigators of 74 identified Australian RDRs, 88 RVA Partners, 17 pharmaceutical organizations and 12 RVA Scientific and Medical Advisory Committee members. Next, managers and coordinators of RDRs and databases who participated in the survey were invited to participate in semi-structured interviews. Quantitative and qualitative data were analysed using basic descriptive statistics and content analysis, respectively. RESULTS: Forty RDRs responded to the survey; nine were national, five were based in Australia and New Zealand, and the remaining were global. Of the 40 survey respondents, eight were interviewed. Most of the RDRs captured similar information regarding patient characteristics, comorbidities and clinical features, diagnosis, family history, genetic testing, procedures or treatment types, response to treatments and complications of treatments. Better treatment outcomes, changes in process of care and changes in quality of care were the most frequently reported benefits of the RDRs. The main challenges proved to be cost/funding of data collection, data completeness, and patient consent. When asked, the participants identified opportunities and challenges regarding potential options to streamline RDRs in Australia in the future. CONCLUSION: Findings from this study highlighted significant dataset heterogeneity based on the individual disease, and current lack of interoperability and coordination between different existing RDRs in Australia. Nevertheless, a nationally coordinated approach to RDRs should be investigated given the particular benefits RDRs offer, such as access to research and the monitoring of new disease-modifying treatments.

Current state of rare disease registries and databases in Australia: a scoping review.

BACKGROUND: Rare diseases (RDs) affect approximately 8% of all people or > 400 million people globally. The Australian Government's National Strategic Action Plan for Rare Diseases has identified the need for a national, coordinated, and systematic approach to the collection and use of RD data, including registries. Rare disease registries (RDRs) are established for epidemiological, quality improvement and research purposes, and they are critical infrastructure for clinical trials. The aim of this scoping review was to review literature on the current state of RDRs in Australia; to describe how they are funded; what data they collect; and their impact on patient outcomes. METHODS: We conducted a literature search on MEDLINE, EMBASE, CINAHL and PsychINFO databases, in addition to Google Scholar and grey literature. Dissertations, government reports, randomised control trials, conference proceedings, conference posters and meeting abstracts were also included. Articles were excluded if they did not discuss RDs or if they were written in a language other than English. Studies were assessed on demographic and clinical patient characteristics, procedure or treatment type and health-related quality of life captured by RDRs or databases that have been established to date. RESULTS: Seventy-four RDRs were identified; 19 were global registries in which Australians participated, 24 were Australian-only registries, 10 were Australia and New Zealand based, and five were Australian jurisdiction-based registries. Sixteen "umbrella" registries collected data on several different conditions, which included some RDs, and thirteen RDRs stored rare cancer-specific information. Most RDRs and databases captured similar types of information related to patient characteristics, comorbidities and other clinical features, procedure or treatment type and health-related quality of life measures. We found considerable heterogeneity among existing RDRs in Australia, especially with regards to data collection, scope and quality of registries, suggesting a national coordinated approach to RDRs is required. CONCLUSION: This scoping review highlights the current state of Australian RDRs, identifying several important gaps and opportunities for improvement through national coordination and increased investment.

Response rates in clinical quality registries and databases that collect patient reported outcome measures: a scoping review.

BACKGROUND: Patient Reported Outcome Measures (PROMs) are being increasingly introduced in clinical registries, providing a personal perspective on the expectations and impact of treatment. The aim of this study was to describe response rates (RR) to PROMs in clinical registries and databases and to examine the trends over time, and how they change with the registry type, region and disease or condition captured. METHODS: We conducted a scoping literature review of MEDLINE and EMBASE databases, in addition to Google Scholar and grey literature. All English studies on clinical registries capturing PROMs at one or more time points were included. Follow up time points were defined as follows: baseline (if available), < 1 year, 1 to < 2 years, 2 to < 5 years, 5 to < 10 years and 10 + years. Registries were grouped according to regions of the world and health conditions. Subgroup analyses were conducted to identify trends in RRs over time. These included calculating average RRs, standard deviation and change in RRs according to total follow up time. RESULTS: The search strategy yielded 1,767 publications. Combined with 20 reports and four websites, a total of 141 sources were used in the data extraction and analysis process. Following the data extraction, 121 registries capturing PROMs were identified. The overall average RR at baseline started at 71% and decreased to 56% at 10 + year at follow up. The highest average baseline RR of 99% was observed in Asian registries and in registries capturing data on chronic conditions (85%). Overall, the average RR declined as follow up time increased. CONCLUSION: A large variation and downward trend in PROMs RRs was observed in most of the registries identified in our review. Formal recommendations are required for consistent collection, follow up and reporting of PROMs data in a registry setting to improve patient care and clinical practice. Further research studies are needed to determine acceptable RRs for PROMs captured in clinical registries.

Telehealth use in Australian cystic fibrosis centers: Clinician experiences.

BACKGROUND: Telehealth has been rapidly adopted by cystic fibrosis (CF) centers and ongoing use in routine CF care is endorsed by CF consumers. However, data describing CF clinician perceptions regarding telehealth are scarce. We aimed to describe clinician experiences and attitudes towards telehealth in CF care among health professionals across Australia. METHODS: CF multidisciplinary health professionals from all CF clinics in Australia were sent an anonymous electronic survey. RESULTS: Eighty-five responses were received representing 15 of 23 (65%) centers. Most clinicians reported using telehealth for routine clinic visits, and a range of other clinical encounters (69.9%). Telehealth was widely perceived as acceptable (91.8%), and clinicians were comfortable/very comfortable (81.2%) integrating telehealth into future CF care. Despite this, 64.1% of respondents considered telehealth clinics to be much worse than face-to-face clinics and 57.5% reported quality of care was somewhat/much worse using telehealth. Home spirometry was available in 73.7% of centers, however, only 26.7% of clinics could provide spirometers for >75% eligible patients. Growth and microbiology assessments were often missed in telehealth clinics and 75.7% reported a technical issue had prevented a telehealth consultation from occurring. CONCLUSIONS: Telehealth for CF in Australia is considered feasible and acceptable by CF clinicians, although use of telehealth varies widely between centers. Concerns exist around the impact of telehealth on health outcomes, especially given core assessments are frequently omitted. Guidelines may help ensure the benefits of telehealth are realized for people with CF without compromising the standard of care.

Evaluation and acceptability of patient-reported outcome measures in women following pelvic organ prolapse procedures.

BACKGROUND: The Australasian Pelvic Floor Procedure Registry (APFPR) captures clinical and surgical data in women undergoing pelvic floor procedures. The inclusion of patient reported outcome measures (PROMs) in the APFPR is a critical activity providing the additional patient perspective of their condition prior to surgery as well as monitoring beyond the usual post-surgical follow-up time. This study aimed to evaluate the acceptability of seven PROMs for women with pelvic organ prolapse (POP) and to determine the most suitable instrument for the APFPR. METHODS: Semi-structured qualitative interviews were conducted with women with POP (n = 15) and their treating clinicians (n = 11) in Victoria, Australia. Interview topics covered appropriateness, content, and acceptability of seven POP-specific instruments identified through the literature to determine their suitability and acceptability for inclusion in the APFPR. We analysed the interview data using conventional content analysis. RESULTS: All study participants agreed that PROMs were needed for the APFPR. Both women and clinicians suggested that some of the instruments were ambiguous, too long and confusing. The Australian Pelvic Floor Questionnaire was accepted widely amongst women and clinicians and recommended for inclusion in the APFPR. All participants agreed it would be appropriate to capture PROMs before surgery, and then followed up post-surgically. Email, phone call or postal mail-out were the preferred options for PROMs data collection. CONCLUSION: Most women and clinicians supported incorporating PROMs in the APFPR. Study participants believed that capturing PROMs would have potential use in individual care and improve outcomes of women with POP.

Hereditary Endocrine Tumor Registries.

Context: Endocrine neoplasia syndromes are phenotypically complex, and there is a misconception that they are universally rare. Genetic alterations are increasingly recognized; however, true prevalence is unknown. The purpose of a clinical registry is to monitor the quality of health care delivered to a specified group of patients through the collection, analysis, and reporting of relevant health-related information. This leads to improved clinical practice, decision-making, patient satisfaction, and outcome. Objective: This review aims to identify, compare, and contrast active registries worldwide that capture data relevant to hereditary endocrine tumors (HETs). Methods: Clinical registries were identified using a systematic approach from publications (Ovid MEDLINE, EMBASE) peer consultation, clinical trials, and web searches. Inclusion criteria were hereditary endocrine tumors, clinical registries, and English language. Exclusion criteria were institutional audits, absence of clinical data, or inactivity. Details surrounding general characteristics, funding, data fields, collection periods, and entry methods were collated. Results: Fifteen registries specific for HET were shortlisted with 136 affiliated peer-reviewed manuscripts. Conclusion: There are few clinical registries specific to HET. Most of these are European, and the data collected are highly variable. Further research into their effectiveness is warranted. We note the absence of an Australian registry for all HET, which would provide potential health and economic gains. This review presents a unique opportunity to harmonize registry data for HET locally and further afield.

Establishment and initial implementation of the Australasian Pelvic Floor Procedure Registry.

INTRODUCTION AND HYPOTHESIS: Stress urinary incontinence (SUI) and pelvic organ prolapse (POP) are common pelvic floor disorders (PFDs). Owing to significant adverse events associated with mesh-related pelvic floor procedures (PFPs) in a proportion of the surgically treated population, and deficits in collection and reporting of these events, the Australian Government identified an urgent need for a tracking mechanism to improve safety and quality of care. The Australasian Pelvic Floor Procedure Registry (APFPR) was recently established following the 2018 Senate Committee Inquiry with the aim of tracking outcomes of PFP involving the use of devices and/or prostheses, with the objective of improving the health outcomes of women who undergo these procedures. This paper will describe the APFPR's aims, development, implementation and possible challenges on the way to its establishment. METHODS: The APFPR has been developed and implemented in accordance with the national operating principles of clinical quality registries (CQRs). The minimum datasets (MDS) for the registry's database have been developed using a modified Delphi process, and data are primarily being collected from participating surgeons. Patient recruitment is based on an opt-out approach or a waiver of consent. Patient-reported outcome measures (PROMs) providing additional health and outcome information will be obtained from participating women to support safety monitoring of mesh-related adverse events. RESULTS: Currently in the Australasian Pelvic Floor Procedure Registry (APFPR) there are 32 sites from various jurisdictions across Australia, that have obtained relevant ethics and governance approvals to start patient recruitment and data collection as of January 2023. Additionally, there are two sites that are awaiting governance review and five sites that are having documentation compiled for submission. Seventeen sites have commenced patient registration and have entered data into the database. Thus far, we have 308 patients registered in the APFPR database. The registry also published its first status report and a consumer-friendly public report in 2022. CONCLUSIONS: The registry will act as a systematic tracking mechanism by collecting outcomes on PFP, especially those involving devices and/or prostheses to improve safety and quality of care.

Development of a conceptual framework for a new patient-reported outcome measure for pain in women following mesh surgery for pelvic floor disorders: a qualitative study.

INTRODUCTION AND HYPOTHESIS: The Australasian Pelvic Floor Procedure Registry (APFPR) collects both clinical and health-related quality of life (HRQoL) data on women undergoing surgery using a prosthesis such as mesh for pelvic organ prolapse (POP) and stress urinary incontinence (SUI). The registry lacks a suitable instrument to assess pain in women following mesh surgery for SUI and POP. This qualitative study describes the views on pain following mesh surgery in women and clinicians through the development of a conceptual framework, which may inform the development of a new instrument for the APFPR. METHODS: We conducted semi-structured interviews with women following mesh surgery for POP and SUI (n=17) and clinicians (n=6) in Victoria, Australia. We sought to reveal aspects of any sort of pain after a pelvic floor procedure. Interviews covered sensation, region, continuity of pain, triggers, and the mode and method of administration for a new pain-specific patient-reported outcome measure. Data were analysed using thematic analysis. RESULTS: We identified the important components of pain felt by women with POP and SUI after surgery using mesh. From the seven themes outlined, a conceptual framework was developed compiling related components of pain into six specific domains. CONCLUSIONS: This study identifies the important components of pain felt by women following mesh surgery. It is hoped that the development of a pain-specific PROM, as supported by clinicians, will assist in the timely and appropriate diagnosis and management of POP and SUI.

The inequity of targeted cystic fibrosis reproductive carrier screening tests in Australia.

OBJECTIVE: European and Australian guidelines for cystic fibrosis (CF) reproductive carrier screening recommend testing a small number of high frequency CF causing variants, rather than comprehensive CFTR sequencing. The study objective was to determine variant detection rates of commercially available targeted reproductive carrier screening tests in Australia. METHODS: Next-generation DNA sequencing of the CFTR gene was performed on 2552 individuals from a whole population sample to identify CF causing variants. The variant detection rates of two commercially available Australian reproductive carrier screening tests, which target 50 or 175 CF causing variants, in this population were calculated. The ethnicity of individuals was determined using principal component analysis. RESULTS: Variant detection rates of the tests for 50 and 175 CF causing variants were 88.2% and 90.8%, respectively. No CF causing variants in individuals of East Asian ethnicity (n = 3) were detected by either test, while >86.6% (n = 69) of CF causing variants in Europeans would be identified by either test. CONCLUSIONS: Reproductive carrier screening tests for a targeted set of high frequency CF variants are unable to detect approximately 10% of CF variants in a multiethnic Australian population, and individuals of East Asian ethnicity are disproportionally affected by this test limitation.

Clinical outcomes of adults and children with cystic fibrosis during the COVID-19 pandemic.

BACKGROUND: The onset of the COVID-19 pandemic was associated with restricted community movement and limited access to healthcare facilities, resulting in changed clinical service delivery to people with cystic fibrosis (CF). This study aimed to determine clinical outcomes of Australian adults and children with CF in the 12-months following the onset of the COVID-19 pandemic. METHODS: This longitudinal cohort study used national registry data. Primary outcomes were 12-month change in percent predicted forced expiratory volume in one second (FEV1 %pred), body mass index (BMI) in adults and BMI z-scores in children. A piecewise linear mixed-effects model was used to determine trends in outcomes before and after pandemic onset. RESULTS: Data were available for 3662 individuals (median age 19.6 years, range 0-82). When trends in outcomes before and after pandemic onset were compared; FEV1 %pred went from a mean annual decline of -0.13% (95%CI -0.36 to 0.11) to a mean improvement of 1.76% (95%CI 1.46-2.05). Annual trend in BMI improved from 0.03 kg/m2 (95%CI -0.02-0.08) to 0.30 kg/m2 (95%CI 0.25-0.45) and BMI z-scores improved from 0.05 (95%CI 0.03-0.07) to 0.12 (95%CI 0.09-0.14). Number of hospitalisations decreased from a total of 2656 to 1957 (p < 0.01). Virtual consultations increased from 8% to 47% and average number of consultations per patient increased from median (IQR) of 4(2-5) to 5(3-6) (p < 0.01). CONCLUSION: In the 12-months following the onset of the COVID-19 pandemic, there was an improvement in the clinical outcomes of people with CF when compared to the pre-pandemic period.

Beyond borders: cystic fibrosis survival between Australia, Canada, France and New Zealand.

BACKGROUND: Life expectancy for people with cystic fibrosis (CF) varies considerably both within and between countries. The objective of this study was to compare survival among countries with single-payer healthcare systems while accounting for markers of disease severity. METHODS: This cohort study used data from established national CF registries in Australia, Canada, France and New Zealand from 2015 to 2019. Median age of survival for each of the four countries was estimated using the Kaplan-Meier method. A Cox proportional hazards model was used to compare risk of death between Canada, France and Australia after adjusting for prognostic factors. Due to low number of deaths, New Zealand was not included in final adjusted models. RESULTS: Between 2015 and 2019, a total of 14 842 people (3537 Australia, 4434 Canada, 6411 France and 460 New Zealand) were included. The median age of survival was highest in France 65.9 years (95% CI: 59.8 to 76.0) versus 53.3 years (95% CI: 48.9 to 59.8) for Australia, 55.4 years (95% CI: 51.3 to 59.2) for Canada and 54.8 years (95% CI: 40.7 to not available) for New Zealand. After adjusting for individual-level factors, the risk of death was significantly higher in Canada (HR 1.85, 95% CI: 1.48 to 2.32; p<0.001) and Australia (HR 2.08, 95% CI: 1.64 to 2.64; p<0.001) versus France. INTERPRETATION: We observed significantly higher survival in France compared with countries with single-payer healthcare systems. The median age of survival in France exceeded 60 years of age despite having the highest proportion of underweight patients which may be due to differences in availability of transplant.

Survival of people with cystic fibrosis in Australia.

Survival statistics, estimated using data from national cystic fibrosis (CF) registries, inform the CF community and monitor disease progression. This study aimed to estimate survival among people with CF in Australia and to identify factors associated with survival. This population-based cohort study used prospectively collected data from 23 Australian CF centres participating in the Australian CF Data Registry (ACFDR) from 2005-2020. Period survival analysis was used to calculate median age of survival estimates for each 5-year window from 2005-2009 until 2016-2020. The overall median survival was estimated using the Kaplan-Meier method. Between 2005-2020 the ACFDR followed 4,601 people with CF, noting 516 (11.2%) deaths including 195 following lung transplantation. Out of the total sample, more than half (52.5%) were male and 395 (8.6%) had undergone lung transplantation. Two thirds of people with CF (66.1%) were diagnosed before six weeks of age or by newborn/prenatal screening. The overall median age of survival was estimated as 54.0 years (95% CI: 51.0-57.04). Estimated median survival increased from 48.9 years (95% CI: 44.7-53.5) for people with CF born in 2005-2009, to 56.3 years (95% CI: 51.2-60.4) for those born in 2016-2020. Factors independently associated with reduced survival include receiving a lung transplant, having low FEV1pp and BMI. Median survival estimates are increasing in CF in Australia. This likely reflects multiple factors, including newborn screening, improvement in diagnosis, refinements in CF management and centre-based multidisciplinary care.

Factors associated with clinical progression to severe COVID-19 in people with cystic fibrosis: A global observational study.

BACKGROUND: This international study aimed to characterise the impact of acute SARS-CoV-2 infection in people with cystic fibrosis and investigate factors associated with severe outcomes. Methods Data from 22 countries prior to 13th December 2020 and the introduction of vaccines were included. It was de-identified and included patient demographics, clinical characteristics, treatments, outcomes and sequalae following SARS-CoV-2 infection. Multivariable logistic regression was used to investigate factors associated with clinical progression to severe COVID-19, using the primary outcome of hospitalisation with supplemental oxygen. RESULTS: SARS-CoV-2 was reported in 1555 people with CF, 1452 were included in the analysis. One third were aged <18 years, and 9.4% were solid-organ transplant recipients. 74.5% were symptomatic and 22% were admitted to hospital. In the non-transplanted cohort, 39.5% of patients with ppFEV1<40% were hospitalised with oxygen verses 3.2% with ppFEV >70%: a 17-fold increase in odds. Worse outcomes were independently associated with older age, non-white race, underweight body mass index, and CF-related diabetes. Prescription of highly effective CFTR modulator therapies was associated with a significantly reduced odds of being hospitalised with oxygen (AOR 0.43 95%CI 0.31-0.60 p<0.001). Transplanted patients were hospitalised with supplemental oxygen therapy (21.9%) more often than non-transplanted (8.8%) and was independently associated with the primary outcome (Adjusted OR 2.45 95%CI 1.27-4.71 p=0.007). CONCLUSIONS: This is the first study to show that there is a protective effect from the use of CFTR modulator therapy and that people with CF from an ethnic minority are at more risk of severe infection with SARS-CoV-2.

Preliminary development of recommendations for the inclusion of patient-reported outcome measures in clinical quality registries.

BACKGROUND: Clinical quality registries (CQRs) monitor compliance against optimal practice and provide feedback to the clinical community and wider stakeholder groups. Despite a number of CQRs having incorporated the patient perspective to support the evaluation of healthcare delivery, no recommendations for inclusion of patient-reported outcome measures (PROMs) in CQRs exist. The aim of this study was to develop a core set of recommendations for PROMs inclusion of in CQRs. METHOD: An online two-round Delphi survey was performed among CQR data custodians, quality of life researchers, biostatisticians and clinicians largely recruited in Australia. A list of statements for the recommendations was identified from a literature and survey of the Australian registries conducted in 2019. The statements were grouped into the following domains: rationale, setting, ethics, instrument, administration, data management, statistical methods, and feedback and reporting. Eighteen experts were invited to participate, 11 agreed to undertake the first online survey (round 1). Of these, nine experts completed the online survey for round 2. RESULTS: From 117 statements presented to the Delphi panel in round 1, a total of 72 recommendations (55 from round 1 and 17 from round 2) with median importance (MI) ≥ 7 and disagreement index (DI) < 1 were proposed for inclusion into the final draft set and were reviewed by the project team. Recommendations were refined for clarity and to read as stand-alone statements. Ten overlapped conceptually and, therefore, were merged to reduce repetition. The final 62 recommendations were sent for review to the panel members for their feedback, which was incorporated into the final set. CONCLUSION: This is the first study to develop preliminary recommendations for PROMs inclusion in CQRs. Recommendations for PROMs implementation are critically important for registries to assure meaningful PROMs data capture, use, interpretation, and reporting to improve health outcomes and healthcare value.

Patient-reported outcome measures for pain in women with pelvic floor disorders: a systematic review.

INTRODUCTION AND HYPOTHESIS: Patient-reported outcome measures (PROMs) are helpful instruments when measuring and reporting changes in patient health status (Al Sayah et al. J Patient Rep Outcomes 5 (Suppl 2):99, 2021) such as the health-related quality of life (HrQoL) of women with pelvic organ prolapse (POP) and stress urinary incontinence (SUI). The Australasian Pelvic Floor Procedure Registry (APFPR) aims to increase capacity for women to report surgical outcomes through the collection of HrQoL data (Ruseckaite et al. Qual Life Res. 2021) but currently lacks a pain-specific PROM for women with pelvic floor disorders (PFDs), particularly POP and SUI. This review aims to systematically review the existing literature and identify instruments that measure pain in women with POP and SUI for inclusion within the APFPR, which reports on complications from these conditions. METHODS: We conducted a literature search on OVID MEDLINE, Embase, CINAHL, PsycINFO and EMCARE databases in addition to Google Scholar and grey literature to identify studies from inception to April 2021. Full-text studies were included if they used PROMs to measure pain in women with POP and SUI. Two authors independently screened articles, extracted data and assessed methodological quality. RESULTS: From 2001 studies, 23 publications describing 19 different PROMs were included for analysis. Eight of these instruments were specific to the pelvic floor; four were only specific to pain and used across multiple disorders; three were generic quality of life instruments and four were other non-validated instruments such as focus group interviews. These instruments were not specific to pain in women with POP or SUI, as they did not identify all relevant domains such as the sensation, region and duration of pain, or incidents where onset of pain occurs. CONCLUSIONS: The findings of this review suggest there are no current PROMs that are suitable pain-specific instruments for women with POP or SUI. This knowledge may inform and assist in the development of a new PROM to be implemented into the APFPR.

Evaluation of the acceptability of patient-reported outcome measures in women following pelvic floor procedures.

PURPOSE: Patient-reported outcome measures (PROMs) are valuable tools in evaluating the outcomes of surgical treatment health-related quality of life (HRQoL) of women with stress urinary incontinence (SUI) and may be incorporated into related clinical quality registries. The aim of this study was to assess the feasibility and acceptability of incorporating PROMs into the Australian Pelvic Floor Procedure Registry (APFPR). METHODS: Semi-structured qualitative interviews were conducted with women with SUI (N = 12) and their managing clinicians (N = 11) in Victoria, Australia. Interview topics covered content and face validity, appropriateness, and acceptability of three incontinence-specific, two pain, one anxiety and depression, one sexual function and one patient global impression of improvement instruments identified through the literature to determine their suitability and acceptability for the APFPR. We analysed interview data into topics using conventional content analysis. RESULTS: Study participants agreed that PROMs were needed for the APFPR. Both participant groups suggested that some of the instruments were ambiguous, therefore only three instruments (one incontinence-specific, sexual function and patient global impression of improvement) will be included in the APFPR. Both clinicians and women agreed it would be appropriate to answer PROMs at baseline and then at 6- and 12-month postsurgically. Email, phone call and mail-out of the instruments were the preferred options for administration. CONCLUSION: Most women and clinicians supported the feasibility of incorporating PROMs in the APFPR. Participants believed the PROMs would demonstrate useful aggregate HRQoL data and have potential for use in individual care.

Patient Reported Outcome Measures After Breast Augmentation - Using the BREAST-Q IS.

PURPOSE: Cosmetic breast augmentation procedures are commonly performed breast device surgeries. The Australian Breast Device Registry (ABDR) administers a five-question patient reported outcome measure (PROM), the BREAST-Q Implant Surveillance module (BREAST-Q IS), to patients 1, 2 and 5 years after breast device surgery. The measure includes an open-ended question to add any comments. This study aimed to use the responses to this open-ended question to assess participants' experiences of breast devices 1 and 2 years after breast augmentation. The secondary objective was to identify emerging and important issues relating to breast augmentation and devices. PATIENTS AND METHODS: This qualitative descriptive study was conducted using a randomly selected sample of 268 responses to the open-ended question in the BREAST-Q IS, from the ABDR database. These responses were from patients who underwent breast augmentation between 2015 and 2018. Comments were analyzed using conventional content analysis in NVivo 12. RESULTS: Four major themes were identified: satisfaction following breast augmentation, dissatisfaction following breast augmentation, complications and breast symptoms following breast augmentation and other comments. Two dominant themes were regarding satisfaction (n = 112) with overall surgical outcome, medical team, and post-operative appearance and complications and breast symptoms (n = 177) following breast augmentation. Emerging issues identified were rippling of breast implants and breast implant illness (BII). CONCLUSION: PROMs can be used to understand patients' perspectives on various aspects of their own surgical experiences. Participants provided responses regarding complications and breast symptoms experienced, and rippling of the breast implants and BII are emerging issues after breast augmentation.