Long-term outcomes among survivors of childhood osteosarcoma: A report from the Childhood Cancer Survivor Study (CCSS).

PURPOSE: Treatment strategies for osteosarcoma evolving between 1970 and 1999 improved 5-year survival and continue as standard of care today. This report evaluates the impact of these evolving therapies on long-term health outcomes. METHODS: Five-year survivors of childhood osteosarcoma in CCSS treated from 1970 to 1999 were evaluated for late (>5 years from diagnosis) mortality, chronic health conditions (CHCs), and health status using piecewise-exponential and logistical models. Comparisons were made between survivors and siblings without cancer, and among survivors examining historical and current standard chemotherapies (e.g., methotrexate/doxorubicin/cisplatin [MAP] vs. others), specific chemotherapy agents and surgical approaches (amputation vs. limb salvage [LS]). Models were evaluated adjusting for attained age, sex, race, ethnicity, and age at diagnosis. RESULTS: A total of 1257 survivors of osteosarcoma were followed on average for 24.4 years. Twenty-year all-cause late mortality was 13.3% (95% confidence interval [CI]: 11.7%-14.9%) overall and 11.7% (95% CI: 6.9%-16.5%) for the subset treated with MAP plus LS. Survivors were at higher risk of CHCs (rate ratio [RR] 3.7, 95% CI: 3.2-4.3) than the sibling cohort, most notably having more serious cardiac, musculoskeletal, and hearing CHCs. Within the survivor cohort, the risk of severe CHCs was twice as high with MAP versus no chemotherapy (RR 2.1, 95% CI: 1.3-3.4). Compared with primary amputation, serious musculoskeletal CHCs were higher after LS (RR 6.6, 95% CI: 3.6-13.4), without discernable differences in health status. CONCLUSION: Contemporary osteosarcoma therapy with MAP plus LS, while improving 5-year disease-free survival, continues to be associated with a high burden of late mortality, CHCs, and health status limitations.

Factors associated with worsening sexual function during adjuvant endocrine therapy in a prospective clinic-based cohort of women with early-stage breast cancer.

PURPOSE: Sexual function problems are common but under-reported among women receiving adjuvant endocrine therapy for breast cancer. Worsening scores on patient-reported outcomes (PROs) may identify those at risk for sexual function problems during treatment. We performed a secondary analysis of prospectively collected PROs in women receiving adjuvant endocrine therapy to identify factors associated with worsening sexual function. METHODS: Women with stage 0-III breast cancer initiating adjuvant endocrine therapy participating in a prospective cohort completed PROs at baseline, 3, 6, 12, 24, 36, 48, and 60 months. Sexual function was evaluated by the MOS-SP measure. Other measures included PROMIS pain interference, fatigue, depression, anxiety, physical function, and sleep disturbance and the Endocrine Symptom Subscale of the FACT-ES. We evaluated associations between score worsening of at least the minimal important difference (MID) in PROMIS T-scores (4 points) and FACT-ES scores (5 points) with score worsening of at least the MID in MOS-SP scores (8 points) using logistic regression. RESULTS: Among 300 participants, 45.7% experienced ≥ 8-point worsening of MOS-SP score at any time point compared to baseline. Worsening endocrine symptoms (OR 1.34, 95% CI 1.22-1.49, p < 0.001), worsening physical function (OR 1.09, 95% CI 1.00-1.18, p = 0.06), and prior mastectomy (OR 1.45, 95% CI 0.94-2.23, p = 0.09) were associated with MOS-SP score worsening by at least the MID. CONCLUSION: Worsening endocrine symptoms and physical function identified on PROs are associated with worsening sexual function during adjuvant endocrine therapy. Routine assessment of these domains with PROs may identify women at risk for sexual function problems. TRIAL REGISTRATION NUMBER: NCT01937052; Date of Registration: 09/09/2013.

Colorectal cancer survivorship care plans: Variations in documentation and posttreatment surveillance recommendations.

BACKGROUND: Survivorship care plans (SCP) should outline pertinent information about cancer treatment and follow-up. METHODS: We descriptively analyzed the content of 74 colorectal cancer SCPs completed as part of a randomized, controlled trial of SCPs at an academic and community cancer center. Surveillance recommendations were compared with American Cancer Society, American Society of Clinical Oncology and National Comprehensive Cancer Network guidelines. RESULTS: SCP information provided in >80% of the plans included participant age, cancer diagnosis, details, and side-effects of treatment (surgery, chemotherapy, radiation) and health promotion recommendations. SCP content documented less frequently included predisposing conditions, genetic counseling/testing information and staging. Posttreatment surveillance recommendations were documented in >90% SCPs. For stage 2-3 cancer, rates of guideline concordant recommendations were 100% for colonoscopy surveillance (Year 1 only), 87% for imaging surveillance, 65% for carcinoembryonic antigen surveillance, and 33% for follow-up visits. Excluding colonoscopy, >15 unique recommendations were listed for each modality across stages and sites, with more variation at the academic site. CONCLUSIONS: SCPs consistently recorded information about cancer diagnosis and treatment but omitted critical information about cancer-specific details denoting risk. Surveillance recommendations varied considerably between cancer centers. Future work to improve the consistency of surveillance recommendations documented in SCPs may be needed.

Late effects in a high-risk population of breast cancer survivors.

PURPOSE: To better understand the impact of cancer and treatment on outcomes and guide program development, we evaluated breast cancer survivors at risk for long-term medical and psychosocial issues who participated in survivorship care visits (SVs) at Johns Hopkins Hospital. METHODS: We conducted a prospective survey study of women with stage I-III breast cancer who participated in SVs from 2010-2016. The same 56-item questionnaire administered at SV and follow-up included an assessment of symptoms, social factors, demographics, anxiety, depression, and comorbidities. We added the Godin Exercise questionnaire to the follow-up. RESULTS: In 2018, 74 participants were identified as disease-free and mailed a follow-up survey; 52 (70.3%) completed the survey. At a median follow-up time of 3.1 years after diagnosis, participants were less likely to be employed (54% vs. 67%) than at the SV. About two-thirds were sedentary, and this was associated with high body mass index (p = 0.02). Sufficiently active participants (≥ 150 min per week of moderate-intensity activity) were less likely to report pain (p = 0.02) or fatigue (p = 0.001). Although 19% had moderate/severe anxiety or depression at follow-up, participants who reported employment satisfaction were less likely to be depressed (p = 0.02). CONCLUSIONS: Awareness of issues faced by survivors is critical for enhancing care and developing models to identify patients who might benefit most from targeted long-term interventions. IMPLICATIONS FOR CANCER SURVIVORS: Interventions to address physical activity, persistent symptoms, and mental health are critical for breast cancer survivors.

Knowledge translation concerns for the CONSORT-PRO extension reporting guidance: a review of reviews.

This review of reviews aimed to appraise the use of the CONSORT-PRO Extension as an evaluation tool for assessing the reporting of patient-reported outcome (PROs) in publications, and to describe the reporting of PRO research across reviews. We also outlined how variation in such evaluations impacts knowledge translation and may lead to potential misuse of the CONSORT-PRO Extension. We systematically searched Medline, Pubmed and CINAHL from 2013 to 2025 March 2021 for reviews of the completeness of reporting of PRO endpoints according to CONSORT-PRO criteria. Two reviewers extracted details of each review, the percentage of included studies that addressed each CONSORT-PRO item, and key recommendations from each review. Fourteen reviews met inclusion criteria, and only six of these used the full CONSORT-PRO checklist with minimal justified modifications. The remaining eight studies made significant or unjustified adjustments to the CONSORT-PRO Extension. Review studies also varied in how they scored multi-component CONSORT-PRO items. CONSORT-PRO items were often unreported in trial reports, and certain CONSORT-PRO items were reported less often than others. The reporting of statistical approaches to dealing with missing PRO data were poor in RCTs included in all 14 review articles. Studies reviewing PRO publications often omitted recommended CONSORT-PRO items from their evaluations, which may cause confusion among readers regarding how best to report their PRO research according to the CONSORT-PRO extension. Many trials published since CONSORT-PRO's release did not report recommended CONSORT-PRO items, which may lead to misinterpretation and consequently to research waste.

A PRO-cision medicine intervention to personalize cancer care using patient-reported outcomes: intervention development and feasibility-testing.

PURPOSE: PRO-cision medicine refers to personalizing care using patient-reported outcomes (PROs). We developed and feasibility-tested a PRO-cision Medicine remote PRO monitoring intervention designed to identify symptoms and reduce the frequency of routine in-person visits. METHODS: We conducted focus groups and one-on-one interviews with metastatic breast (n = 15) and prostate (n = 15) cancer patients and clinicians (n = 10) to elicit their perspectives on a PRO-cision Medicine intervention's design, value, and concerns. We then feasibility-tested the intervention in 24 patients with metastatic breast cancer over 6-months. We obtained feedback via end-of-study surveys (patients) and interviews (clinicians). RESULTS: Focus group and interview participants reported that remote PRO symptom reporting could alert clinicians to issues and avoid unneeded/unwanted visits. However, some patients did not perceive avoiding visits as beneficial. Clinicians were concerned about workflow. In the feasibility-test, 24/236 screened patients (10%) enrolled. Many patients were already being seen less frequently than monthly (n = 97) or clinicians did not feel comfortable seeing them less frequently than monthly (n = 31). Over the 6-month study, there were 75 total alerts from 392 PRO symptom assessments (average 0.19 alert/assessment). Patients had an average of 4 in-person visits (vs. expected 6.5 without the intervention). Patients (n = 19/24) reported high support on the end-of-study survey, with more than 80% agreeing with positive statements about the intervention. Clinician end-of-study interviews (n = 11/14) suggested that PRO symptom monitoring be added to clinic visits, rather than replacing them, and noted the increasing role of telemedicine. CONCLUSIONS: Future research should explore combining remote PRO symptom monitoring with telemedicine and in-person visits.

The PROTEUS-Trials Consortium: Optimizing the use of patient-reported outcomes in clinical trials.

BACKGROUND: The assessment of patient-reported outcomes in clinical trials has enormous potential to promote patient-centred care, but for this potential to be realized, the patient-reported outcomes must be captured effectively and communicated clearly. Over the past decade, methodologic tools have been developed to inform the design, analysis, reporting, and interpretation of patient-reported outcome data from clinical trials. We formed the PROTEUS-Trials Consortium (Patient-Reported Outcomes Tools: Engaging Users and Stakeholders) to disseminate and implement these methodologic tools. METHODS: PROTEUS-Trials are engaging with patient, clinician, research, and regulatory stakeholders from 27 organizations in the United States, Canada, Australia, the United Kingdom, and Europe to develop both organization-specific and cross-cutting strategies for implementing and disseminating the methodologic tools. Guided by the Knowledge-to-Action framework, we conducted consortium-wide webinars and meetings, as well as individual calls with participating organizations, to develop a workplan, which we are currently executing. RESULTS: Six methodologic tools serve as the foundation for PROTEUS-Trials dissemination and implementation efforts: the Standard Protocol Items: Recommendations for Interventional Trials-patient-reported outcome extension for writing protocols with patient-reported outcomes, the International Society for Quality of Life Research Minimum Standards for selecting a patient-reported outcome measure, Setting International Standards in Analysing Patient-Reported Outcomes and Quality of Life Endpoints Data Consortium recommendations for patient-reported outcome data analysis, the Consolidated Standards for Reporting of Trials-patient-reported outcome extension for reporting clinical trials with patient-reported outcomes, recommendations for the graphic display of patient-reported outcome data, and a Clinician's Checklist for reading and using an article about patient-reported outcomes. The PROTEUS-Trials website (www.TheProteusConsortium.org) serves as a central repository for the methodologic tools and associated resources. To date, we have developed (1) a roadmap to visually display where each of the six methodologic tools applies along the clinical trial trajectory, (2) web tutorials that provide guidance on the methodologic tools at different levels of detail, (3) checklists to provide brief summaries of each tool's recommendations, (4) a handbook to provide a self-guided approach to learning about the tools and recommendations, and (5) publications that address key topics related to patient-reported outcomes in clinical trials. We are also conducting organization-specific activities, including meetings, presentations, workshops, and webinars to publicize the existence of the methodologic tools and the PROTEUS-Trials resources. Work to develop communications strategies to ensure that PROTEUS-Trials reach key audiences with relevant information about patient-reported outcomes in clinical trials and PROTEUS-Trials is ongoing. DISCUSSION: The PROTEUS-Trials Consortium aims to help researchers generate patient-reported outcome data from clinical trials to (1) enable investigators, regulators, and policy-makers to take the patient perspective into account when conducting research and making decisions; (2) help patients understand treatment options and make treatment decisions; and (3) inform clinicians' discussions with patients regarding treatment options. In these ways, the PROTEUS Consortium promotes patient-centred research and care.

Effect of Electronic Symptom Monitoring on Patient-Reported Outcomes Among Patients With Metastatic Cancer: A Randomized Clinical Trial.

Importance: Electronic systems that facilitate patient-reported outcome (PRO) surveys for patients with cancer may detect symptoms early and prompt clinicians to intervene. Objective: To evaluate whether electronic symptom monitoring during cancer treatment confers benefits on quality-of-life outcomes. Design, Setting, and Participants: Report of secondary outcomes from the PRO-TECT (Alliance AFT-39) cluster randomized trial in 52 US community oncology practices randomized to electronic symptom monitoring with PRO surveys or usual care. Between October 2017 and March 2020, 1191 adults being treated for metastatic cancer were enrolled, with last follow-up on May 17, 2021. Interventions: In the PRO group, participants (n = 593) were asked to complete weekly surveys via an internet-based or automated telephone system for up to 1 year. Severe or worsening symptoms triggered care team alerts. The control group (n = 598) received usual care. Main Outcomes and Measures: The 3 prespecified secondary outcomes were physical function, symptom control, and health-related quality of life (HRQOL) at 3 months, measured by the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire (QLQ-C30; range, 0-100 points; minimum clinically important difference [MCID], 2-7 for physical function; no MCID defined for symptom control or HRQOL). Results on the primary outcome, overall survival, are not yet available. Results: Among 52 practices, 1191 patients were included (mean age, 62.2 years; 694 [58.3%] women); 1066 (89.5%) completed 3-month follow-up. Compared with usual care, mean changes on the QLQ-C30 from baseline to 3 months were significantly improved in the PRO group for physical function (PRO, from 74.27 to 75.81 points; control, from 73.54 to 72.61 points; mean difference, 2.47 [95% CI, 0.41-4.53]; P = .02), symptom control (PRO, from 77.67 to 80.03 points; control, from 76.75 to 76.55 points; mean difference, 2.56 [95% CI, 0.95-4.17]; P = .002), and HRQOL (PRO, from 78.11 to 80.03 points; control, from 77.00 to 76.50 points; mean difference, 2.43 [95% CI, 0.90-3.96]; P = .002). Patients in the PRO group had significantly greater odds of experiencing clinically meaningful benefits vs usual care for physical function (7.7% more with improvements of ≥5 points and 6.1% fewer with worsening of ≥5 points; odds ratio [OR], 1.35 [95% CI, 1.08-1.70]; P = .009), symptom control (8.6% and 7.5%, respectively; OR, 1.50 [95% CI, 1.15-1.95]; P = .003), and HRQOL (8.5% and 4.9%, respectively; OR, 1.41 [95% CI, 1.10-1.81]; P = .006). Conclusions and Relevance: In this report of secondary outcomes from a randomized clinical trial of adults receiving cancer treatment, use of weekly electronic PRO surveys to monitor symptoms, compared with usual care, resulted in statistically significant improvements in physical function, symptom control, and HRQOL at 3 months, with mean improvements of approximately 2.5 points on a 0- to 100-point scale. These findings should be interpreted provisionally pending results of the primary outcome of overall survival. Trial Registration: ClinicalTrials.gov Identifier: NCT03249090.

What are survivorship care plans failing to tell men after prostate cancer treatment?

BACKGROUND: Survivorship care plans contain important information for patients and primary care physicians regarding appropriate care for cancer survivors after treatment. We describe the completeness of prostate cancer survivorship care plans and evaluate the concordance of follow-up recommendations with guidelines. METHODS: We analyzed 119 prostate cancer survivorship care plans from one academic and one community cancer center, abstracting demographics, cancer/treatment details, and follow-up recommendations. Follow-up recommendations were compared with the American Cancer Society (ACS), American Society of Clinical Oncology (ASCO), and National Comprehensive Cancer Network (NCCN) guidelines. RESULTS: Content in >90% of plans included cancer TNM stage; prostate-specific antigen (PSA) at diagnosis; radiation treatment details (98% of men received radiation); and PSA monitoring recommendations. Potential treatment-specific side effects were listed for 82% of men who had surgery, 86% who received androgen deprivation therapy (ADT), and 97% who underwent radiation. The presence of posttreatment symptoms was noted in 71% of plans. Regarding surveillance follow-up, all guidelines recommend an annual digital rectal exam (DRE). No plans specified DRE. However, all 71 plans at the community site recommended at least annual follow-up visits with urology, radiation oncology, and primary care. Only 2/48 plans at the academic site specified follow-up visits. All guidelines recommend PSA testing every 6-12 months for 5 years, then annually. For the first 5 years, 90% of plans were guideline-concordant, 8% suggested oversurveillance, and 2% were incomplete. In men receiving ADT, ACS and ASCO recommend bone density imaging and NCCN recommends testosterone levels. Of 77 men on ADT, 1% were recommended bone density imaging and 16% testosterone level testing. CONCLUSIONS: While care plan content is more complete for demographic and treatment summary information, both sites had gaps in reporting posttreatment symptoms and ADT-related testing recommendations. These findings highlight the need to improve the quality of information in care plans, which are important in communicating appropriate follow-up recommendations to patients and primary care physicians.

International guidance on the selection of patient-reported outcome measures in clinical trials: a review.

PURPOSE: Patient-reported outcomes (PROs) are increasingly used in clinical trials to provide patients' perspectives regarding symptoms, health-related quality of life, and satisfaction with treatments. A range of guidance documents exist for the selection of patient-reported outcome measures (PROMs) in clinical trials, and it is unclear to what extent these documents present consistent recommendations. METHODS: We conducted a targeted review of publications and regulatory guidance documents that advise on the selection of PROMs for use in clinical trials. A total of seven guidance documents from the US Food and Drug Administration, European Medicines Agency, and scientific consortia from professional societies were included in the final review. Guidance documents were analyzed using a content analysis approach comparing them with minimum standards recommended by the International Society for Quality of Life Research. RESULTS: Overall there was substantial agreement between guidance regarding the appropriate considerations for PROM selection within a clinical trial. Variations among the guidance primarily related to differences in their format and differences in the perspectives and mandates of their respective organizations. Whereas scientific consortia tended to produce checklist or rating-type guidance, regulatory groups tended to use more narrative-based approaches sometimes supplemented with lists of criteria. CONCLUSION: The consistency in recommendations suggests an emerging consensus in the field and supports use of any of the major guidance documents available to guide PROM selection for clinical trials without concern of conflicting recommendations. This work represents an important first step in the international PROTEUS Consortium's ongoing efforts to optimize the use of PROs in clinical trials.

Breast cancer survivorship care plans: what are they covering and how well do they align with national guidelines?

PURPOSE: Survivorship care plans (SCPs) provide key information about cancer treatment history and follow-up recommendations. We describe the completeness of breast cancer SCPs and evaluate guideline concordance of follow-up recommendations. METHODS: We analyzed 149 breast cancer SCPs from two sites, abstracting demographics, cancer/treatment details, surveillance plans, and health promotion advice. SCP recommendations and provided information were compared to American Cancer Society/American Society of Clinical Oncology and National Comprehensive Cancer Network guidelines. RESULTS: SCP information provided in > 90% of the plans included patient age; relevant providers; cancer stage; treatment details; and physical exam, mammogram, and health promotion recommendations. SCP components completed less frequently included post-treatment symptoms/side effects (67%). All SCPs at the community site were uniform but had the potential for oversurveillance if visits occurred every 3 months in years 1-2 or every 6 months in years 3-5 with multiple cancer providers. The academic site recommended three predominant patterns of follow-up: (1) primary care provider every 6-12 months; (2) cancer team every 3-6 months (year 1), every 6-12 months (years 4-5); and (3) alternating oncology providers every 3-6 months (years 1-2) then every 6 months. Compared to guidelines, these patterns recommend under- and oversurveillance at various times. Mammography recommendations showed guideline concordance (annual) for 84%, oversurveillance for 10%, and were incomplete for 6%. SCPs of only 12/79 (15%) women on aromatase inhibitors recommended guideline-concordant bone density testing. CONCLUSIONS: SCP content is more complete for demographic and treatment summary information but has follow-up recommendation gaps. Efforts to improve follow-up recommendations are needed.

Selecting Patient-Reported Outcome Measures to Contribute to Primary Care Performance Measurement: a Mixed Methods Approach.

New models of primary care include patient-reported outcome measures (PROMs) to promote patient-centered care. PROMs provide information on patient functional status and well-being, can be used to enhance care quality, and are proposed for use in assessing performance. Our objective was to identify a short list of candidate PROMs for use in primary care practice and to serve as a basis for performance measures (PMs). We used qualitative and quantitative methods to identify relevant patient-reported outcome (PRO) domains for use in performance measurement (PRO-PM) and their associated PROMs. We collected data from key informant groups: patients (n = 13; one-on-one and group interviews; concept saturation analysis), clinical thought leaders (n = 9; group discussions; thematic analysis), primary care practices representatives (n = 37; six focus groups; thematic analysis), and primary care payer representatives (n = 10; 12-question survey; frequencies of responses). We merged the key informant group information with findings from environmental literature scans. We conducted a targeted evidence review of measurement properties for candidate PROMs. We used a scoping review and key informant groups to identify PROM evaluation criteria, which were linked to the National Quality Forum measure evaluation criteria. We developed a de novo schema to score candidate PROMs against our criteria. We identified four PRO domains and 10 candidate PROMs: 3 for depressive symptoms, 2 for physical function, 3 for self-efficacy, 2 for ability to participate. Five PROMs met ≥ 70% of the evidence criteria for three PRO domains: PHQ-9 or PROMIS Depression (depression), PF-10 or PROMIS-PF (physical functioning), and PROMIS Self-Efficacy for Managing Treatments and Medications (self-efficacy). The PROMIS Ability to Participate in Social Roles and Activities met 68% of our criteria and might be considered for inclusion. Existing evidence and key informant data identified 5 candidate PROMs to use in primary care. These instruments can be used to develop PRO-PMs.

Change in health-related quality of life in older women after diagnosis of a small breast cancer.

BACKGROUND: Screening mammography reduces breast cancer mortality at the cost of frequent false-positive results that lead to unnecessary medical procedures, and the treatment of indolent breast cancers that would never threaten life or health. Earlier diagnosis generally permits less disruptive treatment, but it is possible that even the diagnosis of a very small breast cancer could significantly adversely impact health-related quality of life (HRQOL) in older women. METHODS: The authors compared changes in HRQOL measured by either the Medical Outcomes Study 36-Item Short Form (SF-36) or the Veterans Rand 12-item Health Survey (VR-12) between 198 women diagnosed with in situ or invasive breast cancer measuring ≤1 cm and 36,814 matched controls using the National Cancer Institute's Surveillance, Epidemiology, and End Results cancer registry linked with the Medicare Health Outcomes Survey. RESULTS: The mean age of the cases and controls was 75 years. The SF-36/VR-12 physical component score 12 was found to decrease by 1.6 points between the baseline and follow-up surveys for the controls compared with 3.2 points for women diagnosed with small breast cancers (P = .016). A 2-point decline is recognized as the minimally significant difference for this measure. On multivariable analysis, diagnosis of a small breast cancer was found to be one of the strongest predictors of a significant decrease in both the physical and mental domains of HRQOL (P = .012 and P = .023, respectively). CONCLUSIONS: Receiving the diagnosis of even a very small breast cancer significantly impacts the physical and mental domains of HRQOL in older women. This finding can inform discussions regarding the relative benefits and costs of screening mammography in older women.

Survivorship care visits in a high-risk population of breast cancer survivors.

PURPOSE: Breast cancer survivors face numerous challenges after diagnosis and treatment. Several models have been developed to attempt to improve quality of care. Here, we describe characteristics and outcomes of patients who participated in survivorship visits (SV) at Johns Hopkins (JH). METHODS: We retrospectively reviewed charts of breast cancer patients who participated in an optional SV 1-3 months after completing locoregional therapy and initial systemic therapy. We report patient demographics, comorbidities, tumor characteristics, treatments, and responses to symptom questionnaires. We compared the characteristics of SV participants to stage I-III analytical cases in the 2010-2015 JH Cancer Registry (JHCR). RESULTS: We identified 87 women with stage I-III breast cancer who participated in SVs from 2010 to 2016. Compared to patients in the JHCR (n = 2942), SV participants were younger, more likely to be African American and more likely to have a higher TNM stage, hormone receptor-negative disease, and HER2-positive disease. They were more likely to have received chemotherapy and radiation therapy. They also have similar recurrence rates despite the SV cohort's shorter median follow-up time. Among SV participants, the prevalence of comorbidities including peripheral neuropathy, anemia, lymphedema, anxiety, deep vein thrombosis, and depression increased significantly from time of diagnosis to most recent follow-up. CONCLUSIONS: Compared to the JHCR cohort, SV participants had higher risk cancers and a high frequency of comorbidities potentially associated with breast cancer and therapy. These high-risk patients may benefit most from specific interventions targeting survivorship care, and their experiences may help improve care delivery models.

A PRO-cision Medicine Methods Toolkit to Address the Challenges of Personalizing Cancer Care Using Patient-Reported Outcomes: Introduction to the Supplement.

Patients are increasingly being asked to complete standardized, validated questionnaires with regard to their symptoms, functioning, and well-being [ie, patient-reported outcomes (PROs)] as part of routine care. These PROs can be used to inform patients' care and management, which we refer to as "PRO-cision Medicine." For PRO-cision Medicine to be most effective, clinicians and patients need to be able to understand what the PRO scores mean and how to act on the PRO results. The papers in this supplement to Medical Care describe various methods that have been used to address these issues. Specifically, the supplement includes 14 papers: 6 describe different methods for interpreting PROs and 8 describe how different PRO systems have addressed interpreting PRO scores and/or acting on PRO results. As such, this "Methods Toolkit" can inform clinicians and researchers aiming to implement routine PRO reporting into clinical practice by providing methodological fundamentals and real-world examples to promote personalized patient care.

Interpreting and Acting on PRO Results in Clinical Practice: Lessons Learned From the PatientViewpoint System and Beyond.

INTRODUCTION: We have used several methods to aid in the interpretation of patient-reported outcome (PRO) scores, and to suggest recommendations for acting on them, both within the PatientViewpoint system and in work done since. METHODS FOR INTERPRETING PRO SCORES: For identifying possibly concerning change scores, PatientViewpoint uses questionnaires' minimally important differences or score worsening >½ SD. For poor scores in absolute terms, PatientViewpoint primarily uses distributions based on normative data (eg, worst quartile, >2 SD from the mean). To advance methods for score interpretation, we explored using needs assessments to identify health-related quality-of-life scores associated with unmet needs and requiring follow-up. We also investigated the ability of PRO scores to predict patients' most bothersome issues. METHODS FOR ADDRESSING PRO RESULTS: To develop suggestions for addressing issues identified by PRO questionnaires, we conducted a targeted literature review, interviewed experts from different disciplines, developed draft recommendations based on the literature and interviews, and finalized the recommendations in a consensus meeting with all experts. DISCUSSION: The needs assessment method requires strongly correlated content in the needs assessment and health-related quality-of-life questionnaire. Additional research is needed to explore using the worst scores in absolute terms to identify patients' most bothersome issues. The approach described for developing suggestions for addressing PRO results focuses on local resources and is best-suited for a local context. KEY POINTS: A combination of pragmatic solutions and exploratory research can inform interpreting and acting on PRO scores.

In proportion: approaches for displaying patient-reported outcome research study results as percentages responding to treatment.

PURPOSE: Patient-reported outcome (PRO) data from clinical trials can promote valuable patient-clinician communication and aid the decision-making process regarding treatment options. Despite these benefits, both patients and doctors face challenges in interpreting PRO scores. The purpose of this study was to identify best practices for presenting PRO results expressed as proportions of patients with changes from baseline (improved/stable/worsened) for use in patient educational materials and decision aids. METHODS: We electronically surveyed adult cancer patients/survivors, oncology clinicians, and PRO researchers, and conducted one-on-one cognitive interviews with patients/survivors and clinicians. Participants saw clinical trial data comparing two treatments as proportions changed using three different formats: pie charts, bar graphs, icon arrays. Interpretation accuracy, clarity, and format preference were analyzed quantitatively and online survey comments and interviews, qualitatively. RESULTS: The internet sample included 629 patients, 139 clinicians, and 249 researchers; 10 patients and 5 clinicians completed interviews. Bar graphs were less accurately interpreted than pie charts (OR 0.39; p < .0001) and icon arrays (OR 0.47; p < .0001). Bar graphs and icon arrays were less likely to be rated clear than pie charts (OR 0.37 and OR 0.18; both p < .0001). Qualitative data informed interpretation of these findings. CONCLUSIONS: For communicating PROs as proportions changed in patient educational materials and decision aids, these results support the use of pie charts.

Making a picture worth a thousand numbers: recommendations for graphically displaying patient-reported outcomes data.

PURPOSE: Patient-reported outcomes (PROs) can promote patient-centered care in multiple ways: (1) using an individual patient's PRO data to inform his/her management, (2) providing PRO results from comparative research studies in patient educational materials/decision aids, and (3) reporting PRO results from comparative research studies in peer-reviewed publications. Patients and clinicians endorse the value of PRO data; however, variations in how PRO measures are scored and scaled, and in how the data are reported, make interpretation challenging and limit their use in clinical practice. We conducted a modified Delphi process to develop stakeholder-engaged, evidence-based recommendations for PRO data display for the three above applications to promote understanding and use. METHODS: The Consensus Panel included cancer survivors/caregivers, oncologists, PRO researchers, and application-specific end-users (e.g., electronic health record vendors, decision aid developers, journal editors). We reviewed the data display issues and their evidence base during pre-meeting webinars. We then surveyed participants' initial perspectives, which informed discussions during an in-person meeting to develop consensus statements. These statements were ratified via a post-meeting survey. RESULTS: Issues addressed by consensus statements relevant to both individual and research data applications were directionality (whether higher scores are better/worse) and conveying score meaning (e.g., none/mild/moderate/severe). Issues specific to individual patient data presentation included representation (bar charts vs. line graphs) and highlighting possibly concerning scores (absolute and change). Issues specific to research study results presentation included handling normed data, conveying statistically significant differences, illustrating clinically important differences, and displaying proportions improved/stable/worsened. CONCLUSIONS: The recommendations aim to optimize accurate and meaningful interpretation of PRO data.

Collecting sexual orientation and gender identity information in the emergency department : the divide between patient and provider perspectives.

BACKGROUND: In the USA, The Joint Commission and Institute of Medicine have called for collection of patient sexual orientation (SO) and gender identity (GI) information in healthcare. In a recent study, we reported that ED clinicians believe patients will refuse to provide this information; however, very few patients say they would refuse to provide SO/GI. As part of this study, we interviewed patients and providers regarding the importance of collecting this information. While these interviews were briefly summarised in our prior report, the qualitative data warranted a more thorough analysis and exposition to explore provider and patient views as well as risks and benefits of collecting SO/GI. METHODS: A purposive sample of 79 participants was recruited for semi-structured interviews between August 2014 and January 2015. Participants included community members who had a previous ED encounter and ED providers from 3 community and 2 academic centres in a major US metropolitan area. Interviews were conducted one-on-one in person, audio-recorded and transcribed verbatim. Data were analysed using the constant comparative method. RESULTS: Fifty-three patients and 26 ED providers participated. Patients perceived collection of SO/GI to be important in most clinical circumstances because SO/GI is relevant to their identity and allows providers to treat the whole person. However, many providers felt SO/GI was not relevant in most clinical circumstances because similar care is provided to all patients regardless of SO/GI. Patients and providers agreed there are risks associated with collecting SO/GI in the ED. CONCLUSIONS: ED clinicians do not perceive routine collection of SO/GI to be medically relevant in most circumstances. However, patients feel routine SO/GI collection allows for recognition of individual identity and improved therapeutic relationships in the ED. These discordant perspectives may be hindering patient-centred care, especially for sexual and gender minority patients.

A phase II study evaluating the efficacy of zoledronic acid in prevention of aromatase inhibitor-associated musculoskeletal symptoms: the ZAP trial.

PURPOSE: Aromatase inhibitor-associated musculoskeletal symptoms (AIMSS) are common adverse events of AIs often leading to drug discontinuation. We initiated a prospective clinical trial to evaluate whether bisphosphonates are associated with reduced incidence of AIMSS. METHODS: In the single-arm trial, the Zoledronic Acid Prophylaxis (ZAP) trial, we compared the incidence of AIMSS against historical controls from the Exemestane and Letrozole Pharmacogenomics (ELPh) trial. Eligible women were postmenopausal with stage 0-III breast cancer planning to receive adjuvant AIs. AIMSS was assessed using the Health Assessment Questionnaire and Visual Analog Scale over 12 months in both trials. Participants in the ZAP trial received zoledronic acid prior to initiating letrozole and after 6 months; ELPh participants included in the analysis were taking letrozole but not bisphosphonates. We analyzed patient-reported outcomes (PROs) and bone density in the ZAP trial using mixed-effects linear regression models and paired t tests, respectively. RESULTS: From 2011 to 2013, 59 postmenopausal women enrolled in ZAP trial. All 59 (100%) women received baseline and 52 (88%) received 6-month zoledronic acid, and had similar characteristics to historical controls from the ELPh trial (n = 206). Cumulatively during the first year of AI, 37 and 67% of ZAP and ELPh participants reported AIMSS (p < 0.001), respectively. Within the ZAP trial, we did not observe significant changes in other PROs; however, we report improvements in bone mineral density. CONCLUSIONS: Compared to historical controls, zoledronic acid administered concomitantly with adjuvant AIs was associated with a reduced incidence of AIMSS. A randomized controlled trial is required to confirm these findings.