Patient-reported Outcome Studies Research Articles

Patient-reported outcomes in studies of diabetes technology: What matters.

In recent years, diabetes technologies have revolutionized the care of people with type 1 diabetes (T1D). Emerging evidence suggests that people with type 2 diabetes (T2D) can experience similar benefits from these advances in technology. While glycaemic outcomes are often a primary focus, the lived experience of the person with diabetes is equally important. In this review, we describe the impact of diabetes technologies on patient-reported outcome measures (PROMs). We highlight that most of the published studies investigated PROMs as secondary outcomes. Continuous glucose monitoring systems may have an important role in improving PROMs in individuals with T1D, which may be driven by the prevention or proactive management of hypoglycaemia. In people with T2D, continuous glucose monitoring may also have an important role in improving PROMs, particularly in those treated with insulin therapy. The impact of insulin pumps on PROMs seems positive in T1D, while there is limited evidence in T2D. Studies of hybrid closed-loop therapies suggest increased treatment satisfaction, improved quality of life and decreased diabetes-related distress in T1D, but it is unclear whether these benefits are because of a 'class-effect' or individual systems. We conclude that PROMs deserve a more central role in trials and clinical practice, and we discuss directions for future research.

Patient reported outcomes for anterior and postpectoral prosthesis implantation in single-port laparoscopic breast.

e12601 Background: Breast reconstruction surgery is on the rise and can significantly improve patients' quality of life, and single-port lumpectomy breast reconstruction surgery is becoming more widely used because of its minimally invasive nature, clear field of view, and aesthetics. However, patient-reported outcome studies of anterior versus posterior pectoralis muscle in this procedure are still scarce, and more evidence is needed to provide guidance to clinicians. Methods: Patients who underwent single-port laparoscopic breast reconstruction surgery at Xijing Hospital between March 2021 to December 2023 were included and divided into two groups according to the position of the prosthesis in the pectoral muscle. Patient safety was assessed for surgical safety and tumor safety. Patient Reported Outcome (PRO) was assessed using the BREAST-Q questionnaire. Results: General situation: This study was included in 58 patients with a single -hole laparoscopic breast reconstruction, and 48 patients were included in the post -pectoral muscle group. Tumor safety: 100% recurrence-free survival, 100% disease-free survival, and 100% overall survival in the follow-up patients; there was no significant difference between the anterior and posterior pectoral muscle groups in the single-port cavity. Surgical safety: there was no significant difference between the pre-pectoral and post-pectoral groups in terms of operative time, intraoperative bleeding, nipple areola complex ischemia, periosteal contracture. there were significant differences in postoperative infection, postoperative pain, and flap ischemia between the two groups, with the anterior pectoralis muscle group having a lower rate of postoperative infection, less pain, and a lower rate of flap ischemia. Patient-reported outcomes: The breast satisfaction of the breast reconstruction of the thoracic muscles under the laparoscopy, the higher social psychological health score, and the lower score in chest pain. Conclusions: In our research, the two groups of patients with single -hole laparoscopic breast reconstruction did not have a bad ending, and the single -hole laparoscopic pectoral muscle reconstruction had lower postoperative complications, and had better patient reporting ending Therefore, without considering the economic situation, we recommend the reconstruction of a single -hole laparoscopic pectoral muscles.

A Real-World Evidence Review of Post-Marketing Safety and Compliance with Pegcetacoplan in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) in the US

Introduction: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired hematologic condition characterized by complement-mediated, life-threatening hemolysis and thrombosis which are associated with significant impacts on health-related quality of life (HRQoL). Pegcetacoplan is the first targeted complement component 3 (C3) therapy approved for adults with PNH (US) and adults with PNH plus anemia despite receiving a complement component 5 inhibitor (C5i) for ≥3 months (EU). In clinical trials including patients with PNH who were C5i-experienced or -naive, pegcetacoplan significantly improved levels of hemoglobin (Hb) and lactate dehydrogenase (LDH) and reduced transfusion burden. Pegcetacoplan had a favorable safety profile in parent studies and an ongoing long-term, rollover, open-label extension study (NCT03531255). Compliance with pegcetacoplan treatment was high across all clinical studies. In the US real-world OPERA and Adelphi PNH Disease Specific Programme™ (DSP) patient-reported outcomes (PRO) studies, improvements in levels of Hb and LDH, along with self-reported fatigue and cognitive outcomes, were observed. Pegcetacoplan treatment also led to reductions in the frequency and severity of PNH symptoms (including shortness of breath and fatigue) and improvements in social, physical, and emotional HRQoL domains in the US real-world Voice of the Patient PRO study. Further, both patient and physician satisfaction with pegcetacoplan treatment was high in the Adelphi PNH DSP study. However, to date, compliance and safety with pegcetacoplan treatment in the real-world setting have not been described. The objective of this real-world evidence review study was to evaluate compliance and safety with pegcetacoplan treatment in patients with PNH in the US post-marketing setting. Methods: Post-marketing data from the US, including the number of patients who received pegcetacoplan and reported safety events, were collected and analyzed from the pegcetacoplan Risk Evaluation and Mitigation Strategies (REMS) program which controls the distribution of pegcetacoplan and the pharmacovigilance safety database through May 13, 2023. Treatment compliance was calculated as the proportion of days a patient had the drug in possession, divided by the total number of days of follow-up, using central pharmacy prescription refill data. Thrombosis (a leading cause of death in patients with PNH prior to the availability of complement inhibitors) and meningococcal infections (a potential risk with complement inhibitors) were safety events of interest. To reduce the risk of serious meningococcal infections, clinical trial protocols and pegcetacoplan labeling required vaccination against Streptococcus pneumoniae; N meningitidis types A, C, W, Y, and B; and Haemophilus influenzae type B before starting treatment with pegcetacoplan. If pegcetacoplan treatment must be initiated before vaccination, the US label mandates prophylactic antibiotic use. In this study, the rates of thrombosis and meningococcal infection per 100 patient-years were estimated based on the total number of thrombosis and meningococcal infection events reported in the REMS program. Results: As of May 13, 2023, the cumulative pegcetacoplan exposure was 273.10 patient-years in the US post-marketing setting. Based on prescription refill data, compliance with pegcetacoplan treatment was ~98%. There were 2 confirmed thrombotic events reported and the incidence of thrombotic events was 0.73 events/100 patient-years in 273.10 patient-years. Similar to clinical trials, no meningococcal infections were reported in the US post-marketing setting. Conclusions: The high compliance rate and favorable safety profile of pegcetacoplan in the US post-marketing setting were consistent with clinical trial observations. These data, coupled with the effectiveness on hematologic parameters and improvements in symptoms and HRQoL reported in pegcetacoplan real-world PRO studies, support a positive benefit:risk profile for pegcetacoplan in the real-world setting.

Patient-reported outcomes among virally suppressed people living with HIV after switching to Co-formulated bictegravir, emtricitabine and tenofovir alafenamide.

While some evidence has suggested the benefits of co-formulated bictegravir, emtricitabine and tenofovir alafenamide (B/F/TAF) in improving the quality of life of people living with HIV (PLWH), patient-reported outcome studies that focus on Asian population remain scarce. We aimed to determine the changes in HIV-related symptom burden in virally-suppressed PLWH switching to B/F/TAF in a real-world setting. PLWH on stable antiretroviral therapy (ART) for ≥6 months with plasma HIV RNA <200 copies/mL who decided to switch to B/F/TAF were eligible for the study. Participants' experience with 20 symptoms were assessed using HIV Symptom Index at baseline and weeks 24 and 48. Responses were dichotomized in two ways: 1) present vs. not present; and 2) bothersome vs. not bothersome, and compared across time points. Six hundred and thirty participants (prior regimen, 94.4% integrase inhibitor-based) who completed week 48 visit were included in the analysis. Forty-eight weeks after switching to B/F/TAF, six symptoms were significantly less prevalent, and seven symptoms were significantly less bothersome. Improvement was more pronounced in participants whose prior regimen was elvitegravir-based versus dolutegravir-based. Logistic regression results showed that prior dolutegravir-based ART and pre-existing diabetes independently predicted improvement in diarrhea/loose bowels and muscle aches/joint pain, respectively. Despite the overall improvement, some symptoms persisted in a substantial proportion of participants. Virally-suppressed PLWH might benefit from a regimen switch to B/F/TAF to reduce the prevalence and level of bother of HIV-related symptoms. Nevertheless, additional multidisciplinary interventions are warranted to further alleviate the symptom burden of PLWH.

Recruiting Adolescent and Young Adult Cancer Survivors for Patient-Reported Outcome Research: Experiences and Sample Characteristics of the SURVAYA Study.

Background: Participation of Adolescents and Young Adults with cancer (AYAs: 18–39 years at time of diagnosis) in patient-reported outcome studies is warranted given the limited knowledge of (long-term) physical and psychosocial health outcomes. We examined the representativeness of AYAs participating in the study, to observe the impact of various invitation methods on response rates and reasons for non-participation. Methods: A population-based, cross-sectional cohort study was performed among long-term (5–20 years) AYA cancer survivors. All participants were invited using various methods to fill in a questionnaire on their health outcomes, including enclosing a paper version of the questionnaire, and sending a reminder. Those who did not respond received a postcard in which they were asked to provide a reason for non-participation. Results: In total, 4.010 AYAs (response 36%) participated. Females, AYAs with a higher socio-economic status (SES), diagnosed more than 10 years ago, diagnosed with a central nervous system tumor, sarcoma, a lymphoid malignancy, stage III, or treated with systemic chemotherapy were more likely to participate. Including a paper questionnaire increased the response rate by 5% and sending a reminder by 13%. AYAs who did not participate were either not interested (47%) or did want to be reminded of their cancer (31%). Conclusions: Study participation was significantly lower among specific subgroups of AYA cancer survivors. Higher response rates were achieved when a paper questionnaire was included, and reminders were sent. To increase representativeness of future AYA study samples, recruitment strategies could focus on integrating patient-reported outcomes in clinical practice and involving AYA patients to promote participation in research.

Returning Athletes to Sports Following Anterior Cruciate Ligament Tears.

The purpose of this review is to discuss treatment options, rehabilitation protocols, return-to-play criteria, and expected outcomes after non-operative and operative treatment of anterior cruciate ligament (ACL) tears among an athletic population. Non-operative treatment may be a viable option for some athletes with an ACL tears but can be difficult to predict "copers," and those that resume to sports return at lower performance level and/or less intense activities. Most studies assessing function after ACL reconstruction demonstrate favorable outcomes using patient-reported outcome studies. However, return-to-play and graft re-rupture rates vary substantially based on patient characteristics and level and type of athletic activity. Grafts used to reconstruct ACL produce similar objective outcomes and favorable patient-reported outcomes but have variable re-rupture rates depending on study and differ largely on morbidity associated with graft harvest. Various treatment methods including non-operative and operative techniques have been demonstrated to be efficacious in returning athletes to athletic activity depending on patient age and level of activity. Adherence to fundamental rehabilitation principles and accepted return-to-play guidelines can optimize outcomes and limit re-injury to the injured or contralateral limb.

Characterization of Symptoms and Symptom Clusters for Type 2 Diabetes Using a Large Nationwide Electronic Health Record Database.

A variety of symptoms may be associated with type 2 diabetes and its complications. Symptoms in chronic diseases may be described in terms of prevalence, severity, and trajectory and often co-occur in groups, known as symptom clusters, which may be representative of a common etiology. The purpose of this study was to characterize type 2 diabetes-related symptoms using a large nationwide electronic health record (EHR) database. We acquired the Cerner Health Facts, a nationwide EHR database. The type 2 diabetes cohort (n = 1,136,301 patients) was identified using a rule-based phenotype method. A multistep procedure was then used to identify type 2 diabetes-related symptoms based on International Classification of Diseases, 9th and 10th revisions, diagnosis codes. Type 2 diabetes-related symptoms and co-occurring symptom clusters, including their temporal patterns, were characterized based the longitudinal EHR data. Patients had a mean age of 61.4 years, 51.2% were female, and 70.0% were White. Among 1,136,301 patients, there were 8,008,276 occurrences of 59 symptoms. The most frequently reported symptoms included pain, heartburn, shortness of breath, fatigue, and swelling, which occurred in 21-60% of the patients. We also observed over-represented type 2 diabetes symptoms, including difficulty speaking, feeling confused, trouble remembering, weakness, and drowsiness/sleepiness. Some of these are rare and difficult to detect by traditional patient-reported outcomes studies. To the best of our knowledge, this is the first study to use a nationwide EHR database to characterize type 2 diabetes-related symptoms and their temporal patterns. Fifty-nine symptoms, including both over-represented and rare diabetes-related symptoms, were identified.

Quality of Patient-Reported Outcome Studies Utilizing the BREAST-Q: A Systematic Review.

Patient-reported outcome (PRO) studies are essential in the assessment of surgical procedures in plastic surgery. One accepted and validated questionnaire is the BREAST-Q. The aim of this study was to assess the quality of PRO studies in plastic surgery utilizing the BREAST-Q questionnaire. This study involved 2 steps: (1) a systematic review of 23 key criteria assessing the quality of survey research in studies utilizing the BREAST-Q that were published between 2015 and 2018; (2) a review of current guidance for survey research in journals related to plastic surgery and breast surgery which were included in the systematic review. Seventy-nine studies were included in the systematic review. Many key criteria were poorly reported: 51.9% of the studies did not provide a defined response rate and almost 90% did not provide a method for analysis of nonresponse error; 67.1% lacked a description of the sample's representativeness of the population of interest, and 82.3% did not present a sample size calculation. The methods used to analyze data were not described in 11.4% of the papers; in 27.8% the data analysis presented could not allow replication of the results. Of the 16 journals in the fields of plastic surgery and breast surgery for which the "instructions to authors" were reviewed, 15 (93.7%) did not provide any guidance for survey reporting. The majority of key criteria are underreported by authors publishing their survey research in peer-reviewed journals in the fields of plastic and breast surgery. There is an urgent need to construct well-developed reporting guidelines for survey research in plastic surgery, and particularly in breast surgery.

Patient Reported Outcomes after Local Flap Coverage for Lower Extremity Trauma

Background: &#x0D; Complex lower extremity trauma can result in devastating outcomes including amputation and poor quality of life. Limb salvage can be achieved with the use of local flaps like muscle flaps such as the gastrocnemius or soleus flaps, which detach muscles in order to use them for local coverage. Fasciocutaneous flaps such as reverse sural and propeller flaps are other examples of local flaps which utilize overlying soft tissue for local rearrangement. Patient-reported outcome (PRO) studies have not been reported for local flaps. Additionally, the potential for functional deficits as a result of muscle flaps in comparison to fasciocutaneous flaps has not been investigated. &#x0D; &#x0D; Methods: &#x0D; Local flap coverage for lower extremity traumas between 2014-2019 were reviewed. 248 local flaps were performed for lower extremity salvage following trauma. PROs were recorded utilizing both the Lower Extremity Functional scale (LEFS) and the 36-Item Short-Form Health Survey (SF-36). &#x0D; &#x0D; Results: &#x0D; Surveys were completed by 37 patients (response rate 18.3%, mean follow-up time 3.16 years, average age 49.72 years old). The average LEFS score was 42.09 ± 14.18, and the average physical functioning score was 43.03 ± 21.54. LEFS score and SF-36 physical functioning scores were significantly lower in patients who underwent muscle flaps compared to fasciocutaneous flaps (p=0.021 and p=0.022 respectively). &#x0D; &#x0D; Conclusions and Impact: &#x0D; Patients undergoing local flaps for lower extremity reconstruction have low quality of life scores. Patients who underwent fasciocutaneous flaps had significantly higher quality of life scores compared to muscle flap patients suggesting that fasciocutaneous flaps may cause less functional deficit in salvage operations.

Patient-Reported Outcomes in Sinonasal and Skull Base Malignancy: An Assessment of Study Quality and Clinical Relevance.

Sinonasal and skull base malignancies can cause significant adverse effects on functional status and survival. The goal of this study was to systematically review the published literature of patient-reported outcomes pertaining to treatment of sinonasal and skull base malignancy. A systematic literature search of Medline was conducted with PubMed to identify studies that assessed patient-reported outcomes in patients with sinonasal or skull base malignancy. Patient-reported outcomes studies with at least 10 patients published in English from January 2000 to April 2017 were included. Criteria from International Society for Quality of Life guidelines and criteria unique to sinonasal and skull base malignancies were used to calculate a composite score for each article. Studies with the top 33% of scores were categorized as high quality articles. Twenty-two articles met inclusion/exclusion criteria. Three studies (14%) reported a priori hypothesis. Eleven (50%) assessed specific quality of life domains and 10 studies (45%) performed statistical analysis on these domains. Specific symptoms were assessed in up to 32% of studies. Eight studies were characterized as high quality; these studies had higher sample sizes and more often assessed patient-reported outcomes prior to treatment compared to low quality studies. The goal of the current study was to evaluate the quality of the current patient-reported outcomes literature on sinonasal and skull base malignancies. Areas of improvement for future studies include analysis of individual domains and disease-specific symptoms, reporting a priori hypotheses, and collecting preoperative and longitudinal patient-reported outcomes data.

Prospective Study of Saline versus Silicone Gel Implants for Subpectoral Breast Augmentation.

Silicone gel implants are regarded as esthetically superior to saline implants, offering a more natural consistency. They are also considered less susceptible to rippling. However, objective measurements and patient-reported outcome studies are lacking. Similarly, minimal data are available quantitating animation deformity. A 3-year prospective study was undertaken among 223 women undergoing primary subpectoral breast augmentation using either saline (n = 145) or silicone gel (n = 78) implants. Photographs obtained included frontal views with the patient flexing the pectoral muscles. Images were matched, and vertical differences in nipple position were measured. Breast implants were evaluated using high-resolution ultrasound to detect any ripples or folds at least 3 months after surgery. Outcome surveys were administered. Statistical analysis included the χ2 test, point-biserial correlations, and a power analysis. Respondents reported visible rippling in 18% of women and palpable rippling in 32% of patients, with no significant difference between women treated with saline and silicone gel implants. Ripples were detected on ultrasound scans in 24% of women with saline implants and in 27% of women with silicone gel implants (difference not significant). Ripples were more common in women with lower body mass indices. Fifty percent of patients demonstrated nipple displacement <1 cm on animation. Nipple displacement occurred either up or down with equal frequency and a mean overall nipple displacement of zero. Saline and silicone breast implants produce similar degrees of rippling, as determined on outcome surveys and ultrasound examination. Animation deformities tend to be minor and well-tolerated.

P159 Current patient reported outcomes (PROs) poorly reflect changes in lung function in patients with systemic sclerosis

Abstract Background In a recent clinical trail the reduction of FVC was not accompanied by a benefit with respect to health-related quality of life and patient-reported outcomes (PROs). The aim of this study is to assess how the change in Pulmonary Function Test (PFTs) parameters correlates with the patient reported outcomes (PROs) in an observational cohort of patients with systemic sclerosis (SSc). Methods We conducted a retrospective study of 330 clinic episodes from 121 unselected patients diagnosed with systemic sclerosis according to EULAR/ACR 2013 criteria, in annual follow-up (for a total of 165 patients/year) with PFTs, Health Assessment Questionnaire Disability Index (HAQ-DI), Scleroderma Health Assessment Questionnaire (sHAQ), Modified Borg Dyspnea Scale (Borg) and Cochin Hand Function Score (CHFS). We assessed the correlation between the HAQ and the Visual Analogical Scale 1-7 at baseline (VAS1 pain, VAS2 disease severity, VAS3 arthritis activity, VAS4 intestinal problems, VAS5 dyspnea, VAS6 Raynaud’s phenomenon, VAS7 digital ulcers). We evaluated the correlation of PFTs with PROs at every time period and the correlation between the change of PFTs parameters (δFVC, δDLCO) with the change of the PROs over a year of follow-up. Following analysis of distribution, Spearman or Pearson Test were used to determine correlation coefficients, as appropriate (Prism 7). Results The median disease duration was 5 years (IQR 3-10). The median of 12 months δFVC% and δDLCO% were 0 (IQR -5.81 to 3.28) and -2.439 (IQR -8.76 to 5.98), respectively. The analysis evidenced a strong positive correlation between VAS1-7 and HAQ. We observed also significant correlation between FVC%, DLCO% and HAQ-DI (r= -0.355 and -0.266, respectively; p &amp;lt; 0.0001 for both), Borg (r= -0.403 and -0.379, respectively; p &amp;lt; 0.0001) and CHFS (r = -0.355 and -0.256, respectively; p &amp;lt; 0.0001). Nevertheless, in longitudinal setting there was no significant correlation between δPROs and changes lung function, as continuous variables, neither there was any significant PROs difference in patients that did or did not lose more than 10% of FVC and DLCO over a year of follow-up. Conclusion This analysis of a monocentric non-selected population evidenced that the current commonly used PROs in SSc while showing a good correlation with lung function are poorly sensitive to change or to reflect changes in lung function over 12 months. In this sense, prudent interpretation of the lack of correlation between FVC and patient-reported outcomes in studies of phase 3 is warranted. Disclosures F. Danzo: None. K. Gjeloshi: None. G. Abignano: None. A. Dean: None. F. Masini: None. G. Cuomo: None. F. del Galdo: None.

The Relationship between Animation Deformity and Patient-Reported Outcomes: Application of the BREAST-Q to a Quantitative Stratification of Animation Severity.

Animation deformity can occur following subpectoral breast reconstruction and is an oft-touted rationale for prepectoral reconstruction. Despite increasing recognition, there is a paucity of patient-reported outcome studies in women with animation deformity. Women presenting after subpectoral implant-based breast reconstruction were evaluated for animation deformity. Video analysis and quantitative deformity assessment were performed in conjunction with BREAST-Q surveys. BREAST-Q data were compared to our quantitative animation grading scale to assess the relationship between animation severity and patient-reported outcomes. One hundred forty-one subpectoral breast reconstructions met inclusion criteria. Average scores were 67.8 ± 17.9 of 100 for satisfaction with breasts and 78.3 ± 14.1 of 100 for physical well-being. Animation deformity severity did not correlate with satisfaction with breasts (p = 0.44). Physical well-being, particularly pain-related questions, increased with increasing animation (p = 0.01); specifically, patients reported significantly less pulling, nagging, and aching in the breast (p = 0.01, p = 0.001, and p = 0.004, respectively). Patients with the least and most severe animation deformity had significantly higher numbers of revision procedures (0.89 and 1.03 procedures, respectively) compared with patients with intermediate deformity (0.49 procedures; p = 0.01 and p = 0.009, respectively). Although pectoralis release creates a more mobile-and more animating-reconstruction, this same release may lead to less pain because muscle is no longer contracting against a fixed space. This may lead to two distinct origins of subpectoral revision: (1) patients in pain (but low animation) and (2) patients with visibly distorted animation (but low pain). Risk, II.

Chinese multicentre prospective registry of breast cancer patient-reported outcome-reconstruction and oncoplastic cohort (PRO-ROC): a study protocol

IntroductionAvailable patient-reported outcome (PRO) studies are mainly from single institution or of small sample size, and the variations across hospitals and regions were not fully analysed. A multicentre, prospective, patient-reported...

Genetic services for Hereditary Cancer: a systematic review of Patient Reported Outcomes studies

Abstract Background Genetic services for Hereditary Breast and Ovarian Cancer (HBOC) have become part of clinical and public health practice. Nevertheless, the evaluation of such services, including genetic testing and counseling, is challenging as they rarely affect health status measures (e.g. mortality and morbidity). A possible way is using Patient Reported Outcomes (PROs) i.e. subjective reports coming from patients, directly attributable to genetic services. We performed a systematic review to explore the use of PROs in HBOC genetic services. Methods We searched Pubmed, Scopus, and ISI Web of Knowledge for observational studies using PROs to assess standard genetic counseling (i.e. in person and individual) for HBOC. Results We identified ten surveys from various countries (USA n = 5; Europa n = 6), published between 2000 and 2018 and mainly conducted in Teaching hospitals and Cancer Research and Treatment Institutes (n = 9). The majority assessed pre-test counseling (n = 6) with diagnostic or predictive purpose. The most frequently measured outcomes were patient satisfaction (n = 9), adherence to recommended interventions (n = 3), information sharing with relatives (n = 3); disease risk perception (n = 2), and psychosocial distress (n = 2). Six studies adopted standardized PROs collection tools: the most common were the Genetic Counseling Satisfaction Scale and the Hospital Anxiety and Depression Scale. Questionnaires were mainly administered by post, soon after genetic counseling or up to seven years later. Overall, patients seem satisfied with genetic counseling. Nevertheless, more attention to the psycho-social aspects of genetic testing is needed. Conclusions PROs are very promising for the assessment of HBOC genetic services. Their routine use could provide important elements to improve the quality and the patient-centeredness of genetic services. Emerging information and communication technologies will help this process by making it easier collecting patient data. Key messages Precision medicine, where medical decisions are tailored to an individual’s characteristics, including the patient’s genetic profile, is becoming a paradigm for chronic diseases, particularly cancer. PROs are expected to be increasingly used as a measure of performance in order to drive the changes in how clinical genetic services, and healthcare in general, are organized, delivered and founded.

Quality of life among symptomatic compared to PSA-detected prostate cancer survivors - results from a UK wide patient-reported outcomes study

BackgroundQuality of life among prostate cancer survivors varies by socio-demographic factors and treatment type received; however, less in known about differences in functional outcomes by method of presentation. We investigate differences in reported urinary, bowel, sexual and hormone-related problems between symptomatic and PSA-detected prostate cancer survivors.MethodsA UK wide cross-sectional postal survey of prostate cancer survivors conducted 18-42 months post-diagnosis. Questions were included on presentation method and treatment. Functional outcome was determined using the EPIC-26 questionnaire. Reported outcomes were compared for symptomatic and PSA-detected survivors using ANOVA and multivariable log-linear regression.ResultsThirty-five thousand eight hundred twenty-three men responded (response rate: 60.8%). Of these, 31.3% reported presenting via PSA test and 59.7% symptomatically. In multivariable analysis, symptomatic men reported more difficulty with urinary incontinence (Adjusted mean ratio (AMR): 0.96, 95% CI: 0.96-0.97), urinary irritation (AMR: 0.95, 95% CI: 0.95-0.96), bowel function (AMR: 0.97, 95% CI: 0.97-0.98), sexual function (AMR: 0.90, 95% CI: 0.88-0.92), and vitality/hormonal function (AMR: 0.96, 95% CI: 0.96-0.96) than PSA-detected men. Differences were consistent across respondents of differing age, stage, Gleason score and treatment type.ConclusionProstate cancer survivors presenting symptomatically report poorer functional outcomes than PSA-detected survivors. Differences were not explained by socio-demographic or clinical factors. Clinicians should be aware that men presenting with symptoms are more likely to report functional difficulties after prostate cancer treatment and may need additional aftercare if these difficulties persist. Method of presentation should be considered as a covariate in patient-reported outcome studies of prostate cancer.

Cancer survivors not participating in observational patient-reported outcome studies have a lower survival compared to participants: the population-based PROFILES registry

PurposeThe ‘Patient Reported Outcomes Following Initial treatment and Long-term Evaluation of Survivorship’ (PROFILES) registry collects patient-reported outcomes (PROs) from short- and long-term cancer survivors in the Netherlands, in a population-based setting. The aim of this analysis is to assess the generalizability of observational PRO research among cancer survivors by comparing socio-demographic and clinical characteristics, and survival of participants and non-participants in cancer survivors invited for questionnaire research through the PROFILES registry.MethodsBetween 2008 and 2015, cancer survivors with different cancer diagnoses (N = 14,011) were invited to participate in PROFILES registry studies, of whom 69% (N = 9684) participated. Socio-demographic and clinical characteristics and survival data, collected through the Netherlands Cancer Registry, were associated with participation versus non-participation in multivariable logistic regression analyses and cox proportional hazard regression models, respectively.ResultsParticipants had a significantly better survival compared to non-participants (HR = 1.47, P < .01). Participation was associated with male gender, being 60–70 years old, high socio-economic status, receiving any treatment, receiving radiotherapy, having no comorbidities, and a cancer diagnosis 2–3 years before invitation. Sensitivity analysis demonstrates that the health-related quality of life (HRQoL) might be up to 1.3 points lower (scale 0–100) using hot deck imputation compared to non-imputed participant data.ConclusionsCancer survivors not participating in observational PROs research significantly differ from participants, with respect to socio-demographic and clinical characteristics, and survival. Their HRQoL scores may be systematically lower compared to participants. Therefore, even in PRO studies with relatively high participation rates, observed outcomes may represent the healthier patient with better outcomes.

Using Crowdsourcing as a Platform to Evaluate Lay Perception of Prophylactic Mesh Placement

BackgroundProphylactic mesh placement (PMP) at the time of open abdominal surgery has gained momentum over the last decade. However, there remains an identifiable gap in the literature regarding patient-reported outcomes and qualitative metrics. In effort to gauge the population's understanding or familiarity with PMP, this study provides an educational framework and uses crowdsourcing as a novel means to assess perception among the general population. MethodsA cross-sectional survey study was conducted among the general public to elicit perspectives on PMP. An online crowdsourcing platform was used to capture responses to a questionnaire. Pearson's correlation coefficients, paired t-test, chi-square test, and Fisher's exact tests were performed. ResultsOf 433 respondents, 338 (78.1%) were included. Individuals who had previously undergone surgery and those who had prior hernia repair were more likely to choose PMP than surgically naïve patients (P = 0.06). ConclusionsThe majority of respondents support the use of PMP. This study contributes to the existing body of literature on PMP and serves as the first qualitative description to gauge the population's perception and understanding of this surgical technique. Within the evolving health care landscape, understanding quality-of-life measures have become increasingly important in defining successful surgical outcomes. Although the data-driven level-I evidence supports the clinical use of PMP, this study intends to establish a framework for future patient-reported outcome studies.

Patient-reported outcomes with medical management of benign prostatic hyperplasia: a prospective study in a tertiary care teaching hospital of rural West Bengal, India

Background: Effectiveness of pharmacotherapy in benign prostatic hyperplasia (BPH) is generally evaluated by clinical measures. Outcome of the treatment from the patient’s perspective however cannot be evaluated which may result communication gap between the patient and the physician. Patient-Reported Outcomes (PRO) studies can bridge this gap. This study was undertaken to evaluate outcomes and to assess the impact of medical management of BPH on general health-related quality of life.Methods: In this prospective, observational study, eligible patients with BPH attending a Urology clinic in a tertiary care rural hospital of West Bengal were enrolled and followed up on third and sixth months from baseline. Symptom assessment of BPH were assessed through International Prostate Symptom Score (I-PSS), BPH Impact Index (BII) and Health Related Quality of life questionnaires. Tools of descriptive statistics were used for analysis of data.Results: In the study population of 66 patients, 50% were treated with monotherapy (alpha blockers) and 50% were treated with combinations (alpha blocker and 5alpha reductase inhibitors). After 6 months of medical management, I-PSS was decreased from 18.86±5.53 to 11.76±3.94 (p &lt;0.001), BII score decreased from 9.65±2.59 to 5.89±2.24 (p &lt;0.001) and VAS score increased from 51.44±10.03 to 54.24±11.38 (p &lt;0.001).Conclusions: We found medical management definitely improved quality of life in BPH patients and significantly decreased symptoms. This study is a step in the direction of development of larger and longer term PRO studies in BPH management.

The Reporting of Patient-reported Outcomes in Studies of Patients with Rheumatoid Arthritis: A Systematic Review of 250 Articles.

Patient-reported outcomes (PRO) in rheumatoid arthritis (RA) provide important information regarding disease effect. The study objective was to assess the frequency of PRO use in recent RA studies and compare results with a previous systematic review (SR) in 2005-2007. An SR was performed in PubMed MEDLINE (January 2015). Publications were identified using these MEdical Subject Headings terms: "arthritis, rheumatoid" with a limitation to "humans," "all adults: 19+ years," "English," "published in the last 2 years," and "clinical trials." All studies were assessed, whatever their designs. All PRO reported in publications were classified according to general domains of health by 2 authors. Statistics were descriptive. Two hundred fifty articles were analyzed. Of them, 113 (45.2%) were randomized controlled trials; 138 different PRO were reported. The most frequent PRO, similar to the 2007 SR, were function (68.0%), pain (40.0%), patient's global assessment (49.2%), and health-related quality of life (18.4%). Fatigue (14.4%), morning stiffness (10.0%), psychological status (9.6%), productivity losses (6.4%), utility (5.2%), sleep disturbance (2.4%), and coping (2.0%) were rarely reported. Although frequent domains were reported using well-validated questionnaires, the others were reported using heterogeneous questionnaires. The PRO collected and reported in RA studies are remarkably consistent with those seen in 2005-2007, and reflect the existing RA Core Set measures. Other domains of health prioritized by patients including fatigue, psychological status, productivity losses, sleep disturbance, and coping remain rarely reported. Further, heterogeneity in outcome measures used presents challenges in interpreting true disease effect and response to therapy.