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Christopher Hillis,1 Kathryn E Flynn,2 Erinn Hoag Goldman,3 Tracy S Moreira-Lucas,4 Josie Visentini,4 Stephanie Dorman,4 Rachel Ballinger,5 Hilary F Byrnes,6 Andrea De Palma,7 Valentin Barbier,8 Lisa Machado,9 Ehab Atallah2 1Department of Oncology, McMaster University, Hamilton, Canada; 2Department of Medicine, Medical College of Wisconsin, Milwaukee, WI, USA; 3US Medical Affairs, Pfizer Inc, New York, NY, USA; 4Medical Affairs, Pfizer Canada Inc., Kirkland, Quebec, Canada; 5Patient Centred Outcomes (PCO), ICON Clinical Research Inc., Reading, UK; 6Patient Centred Outcomes (PCO), ICON Clinical Research Inc., Blue Bell, PA, USA; 7Patient Centred Outcomes (PCO), ICON Clinical Research Inc., Milan, Italy; 8Patient Centred Outcomes (PCO), ICON Clinical Research Inc., Lyon, France; 9The Canadian CML Network, Toronto, Ontario, CanadaCorrespondence: Andrea De Palma, Tel +39 06 45 20 8037, Email Andrea.DePalma@iconplc.comPurpose: With treatment, chronic myeloid leukemia (CML) has a favorable prognosis, however, individuals with CML experience impairment to their quality of life (QoL). The aim of this study was to examine the perspectives and experiences of individuals with CML and to understand their challenges communicating with their CML physician.Patients and Methods: An online survey in adults with CML (n=100) in the US and Canada assessed QoL, patient-provider relationships, treatment satisfaction, and understanding of CML and treatment goals via the MD Anderson Symptom Inventory, the Cancer Therapy Satisfaction Questionnaire and de novo survey questions. Participants were recruited via an external patient recruiter and CML Patient Groups.Results: Many participants reported hardships due to CML and its treatment. The main impacts were on the ability to work (21%), engage in personal activities (e.g., hobbies, 28%), and to enjoy sexual relations (median=2.00, IQR=8.50). A substantial proportion (21– 39%) wished to discuss additional topics with their providers (e.g., management of CML and/or its impacts). While participants reported satisfaction with therapy overall (median=85.71, IQR=17.86), they indicated low to moderate treatment satisfaction with specific components, including concerns regarding side effects (median=43.75, IQR=43.75). Participants generally had a good understanding of CML (97%) and its treatment goals (92%).Conclusion: These findings advance our understanding of issues that need improvement to support QoL for individuals living with CML. Future work is needed to improve patient-provider relationships, address treatment-related side effects, and provide clinical information that is easier for patients to understand.Keywords: chronic myeloid leukemia, quality of life, patient-health care provider relationship, patient experience, survey, North America

Purpose: To evaluate the reliability and construct validity of measures from the Patient-Reported Outcomes Measurement Information System® (PROMIS®) for patients with heart failure before and after heart transplantation. Methods: We assessed reliability of the PROMIS short forms using Cronbach’s alpha and the average marginal reliability. To assess the construct validity of PROMIS computerized adaptive tests and short-form measures, we calculated Pearson product moment correlations between PROMIS measures of physical function, fatigue, depression, and social function and existing PRO measures of similar domains (i.e., convergent validity) as well as different domains (i.e., discriminate validity) in patients with heart failure awaiting heart transplant. We evaluated the responsiveness of these measures to change after heart transplant using effect sizes. Results: Forty-eight patients were included in the analyses. Across the many domains examined, correlations between conceptually similar domains were larger than correlations between different domains of health, demonstrating construct validity. Health status improved substantially after heart transplant (standardized effect sizes, 0.63–1.24), demonstrating the responsiveness of the PROMIS measures. Scores from the computerized adaptive tests and the short forms were similar. Conclusions: This study provides evidence for the reliability and construct validity (including responsiveness to change) of four PROMIS domains in patients with heart failure before and after heart transplant. PROMIS measures are a reasonable choice in this context and will facilitate comparisons across studies and health conditions.

Objective: Cancer and treatments for cancer affect specific aspects of sexual functioning and intimacy; however, limited qualitative work has been done in diverse cancer populations. As part of an effort to improve measurement of self-reported sexual functioning, we explored the scope and importance of sexual functioning and intimacy to patients across cancer sites and along the continuum of care.Methods: We conducted 16 diagnosis- and sex-specific focus groups with patients recruited from the Duke University tumor registry and oncology/hematology clinics (N=109). A trained note taker produced field notes summarizing the discussions. An independent auditor verified field notes against written transcripts. The content of the discussions was analyzed for major themes by two independent coders.Results: Across all cancers, the most commonly discussed cancer- or treatment-related effects on sexual functioning and intimacy were fatigue, treatment-related hair loss, weight gain and organ loss or scarring. Additional barriers were unique to particular diagnoses, such as shortness of breath in lung cancer, gastrointestinal problems in colorectal cancers and incontinence in prostate cancer. Sexual functioning and intimacy were considered important to quality of life. While most effects of cancer were considered negative, many participants identified improvements to intimacy after cancer.Conclusion: Overall evaluations of satisfaction with sex life did not always correspond to specific aspects of functioning (e.g. erectile dysfunction), presenting a challenge to researchers aiming to measure sexual functioning as an outcome. Health-care providers should not assume that level of sexual impairment determines sexual satisfaction and should explore cancer patients' sexual concerns directly. [ABSTRACT FROM AUTHOR]

Objective: Cancer and its treatments disturb sleep-wake functioning; however, there is little information available on the characteristics and consequences of sleep problems associated with cancer. As part of an effort to improve measurement of sleep-wake functioning, we explored the scope of difficulties with sleep in a diverse group of patients diagnosed with cancer.Methods: We conducted 10 focus groups with patients recruited from the Duke University tumor registry and oncology/hematology clinics. Separate groups were held with patients scheduled to begin or currently undergoing treatment for breast, prostate, lung, colorectal, hematological, and other cancer types and with patients who were in posttreatment follow-up. The content of the focus group discussions was transcribed and analyzed for major themes by independent coders.Results: Participants not only reported causes of sleep disturbance common in other populations, such as pain and restless legs, but they also reported causes that may be unique to cancer populations, including abnormal dreams, anxiety about cancer diagnosis and recurrence, night sweats, and problems with sleep positioning. Many participants felt that sleep problems reduced their productivity, concentration, social interactions, and overall quality of life. Many also shared beliefs about the increased importance of sleep when fighting cancer.Conclusions: The findings underscore the need for interventions that minimize the negative impact of cancer and its treatments on sleep. This study will inform efforts now underway to develop a patient-reported measure of sleep-wake functioning that reflects the breadth of concepts considered important by patients with cancer. [ABSTRACT FROM AUTHOR]

Context: Findings from previous studies of the effects of exercise training on patient-reported health status have been inconsistent.Objective: To test the effects of exercise training on health status among patients with heart failure.Design, Setting, and Patients: Multicenter, randomized controlled trial among 2331 medically stable outpatients with heart failure with left ventricular ejection fraction of 35% or less. Patients were randomized from April 2003 through February 2007.Interventions: Usual care plus aerobic exercise training (n = 1172), consisting of 36 supervised sessions followed by home-based training, vs usual care alone (n = 1159). Randomization was stratified by heart failure etiology, which was a covariate in all models.Main Outcome Measures: Kansas City Cardiomyopathy Questionnaire (KCCQ) overall summary scale and key subscales at baseline, every 3 months for 12 months, and annually thereafter for up to 4 years. The KCCQ is scored from 0 to 100 with higher scores corresponding to better health status. Treatment group effects were estimated using linear mixed models according to the intention-to-treat principle.Results: Median follow-up was 2.5 years. At 3 months, usual care plus exercise training led to greater improvement in the KCCQ overall summary score (mean, 5.21; 95% confidence interval, 4.42 to 6.00) compared with usual care alone (3.28; 95% confidence interval, 2.48 to 4.09). The additional 1.93-point increase (95% confidence interval, 0.84 to 3.01) in the exercise training group was statistically significant (P < .001). After 3 months, there were no further significant changes in KCCQ score for either group (P = .85 for the difference between slopes), resulting in a sustained, greater improvement overall for the exercise group (P < .001). Results were similar on the KCCQ subscales, and no subgroup interactions were detected.Conclusions: Exercise training conferred modest but statistically significant improvements in self-reported health status compared with usual care without training. Improvements occurred early and persisted over time.Trial Registration: clinicaltrials.gov Identifier: NCT00047437. [ABSTRACT FROM AUTHOR]

For this report, the authors described the initial activities of the Cancer Patient-Reported Outcomes Measurement Information System (PROMIS)-Sexual Function domain group, which is part of the National Institutes of Health Roadmap Initiative to develop brief questionnaires or individually tailored assessments of quality-of-life domains. Presented are a literature review of sexual function measures used in cancer populations and descriptions of the domains found in those measures. By using a consensus-driven approach, an electronic bibliographic search was conducted for articles that were published from 1991 to 2007, and 486 articles were identified for in-depth review. In total, 257 articles reported the administration of a psychometrically evaluated sexual function measure to individuals who were diagnosed with cancer. Apart from the University of California-Los Angeles Prostate Cancer Index, the International Index of Erectile Function, and the Female Sexual Function Index, the 31 identified measures have not been tested widely in cancer populations. Most measures were multidimensional and included domains related to the sexual response cycle and to general sexual satisfaction. The current review supports the need for a flexible, psychometrically robust measure of sexual function for use in oncology settings and strongly justifies the development of the PROMIS-Sexual Function instrument. When the PROMIS-Sexual Function instrument is available publicly, cancer clinicians and researchers will have another measure with which to assess patient-reported sexual function outcomes in addition to the few legacy measures that were identified through this review. [ABSTRACT FROM AUTHOR]

OBJECTIVE: To determine if patients vary in perceptions of safety if interpersonal continuity were to be disrupted. If so, which characteristics are associated with feeling unsafe? BACKGROUND: The extent to which patients' preference for continuity with a personal physician is due to perceptions of safety is unclear. DESIGN: Observational study (Wisconsin Longitudinal Study Graduate and Sibling Survey). SETTING AND PARTICIPANTS: A total of 6827 respondents (most aged 63-66 years) who completed the 2003-06 survey round. MAIN VARIABLES STUDIED: Age, gender, marital status, education, health insurance type, illnesses, medications, length of relationship with provider and place, personality type, decision-making preference and trust in physician deliberation. MAIN OUTCOME MEASURES: Safety perception when visiting another doctor or clinic if own doctor were not available. RESULTS: Twelve percent of respondents felt unsafe. After adjustment, as compared to those who felt safe, those who felt unsafe were more likely to be women (Odds ratio=1.65, 95% confidence interval=1.35-2.01), have more chronic conditions (1.27, 1.08-1.50) and have a longer relationship with a usual provider: 5-9 years (1.53, 1.11-2.10) 10-14 years (1.41, 1.02-1.95) and 15 or more years (1.62, 1.20-2.17) compared to 0-4 years. Those who preferred active participation in decision making and had trust in their physician were less likely to feel safe (1.63, 1.10-2.41). CONCLUSIONS: Certain older adults perceive being unsafe if not seeing their usual physician. Further research should investigate reasons for perceptions of safety if continuity were disrupted and any implications for care. [ABSTRACT FROM AUTHOR]

Using the Wisconsin Longitudinal Study Graduate Survey (N = 5,830), a population-based cohort of older adults (most aged 63-66 years), we explored relationships between five factors of personality and four preference types that account for multiple components of the health care decision-making process (information exchange, deliberation, and selection of treatment choice). After adjustment for personal, health, social, and economic factors, we found that increased conscientiousness and openness to experience and decreased agreeableness and neuroticism corresponded to preferring the most active decision-making style compared with the least active. A better understanding of how personality traits relate to patient decision-making styles may help clinicians tailor treatment discussions to the needs and preferences of individual patients. [ABSTRACT FROM AUTHOR]

The Center for International Blood and Marrow Transplant Research (CIBMTR) prepares an annual set of summary slides to summarize the trends in transplantation and cellular therapies. For the first time in the 2023 summary slides, the CIBMTR incorporated data for patients receiving chimeric antigen receptor T cell (CAR-T) infusions. In addition, data on patient-reported outcomes (PROs) are included. This report aims to update the annual trends in US hematopoietic cell transplantation (HCT) activity and incorporate data on the use of CAR-T therapies. A second aim is to present and describe the development, implementation, and current status of PRO data collection. In August 2020, the CIBMTR launched the Protocol for Collection of Patient-Reported Outcomes Data (CIBMTR PRO Protocol). The CIBMTR PRO Protocol operates under a centralized infrastructure to reduce the burden to centers. Specifically, PRO data are collected from a prospective convenience sample of adult HCT and CAR-T recipients who received treatment at contributing centers and consented for research. Data are merged and stored with the clinical data and used under the governance of the CIBMTR Research Database Protocol. Participants answer a series of surveys developed by the Patient Reported Outcomes Measurement Information System (PROMIS) focusing on physical, social and emotional, and other measures assessing financial well-being, occupational functioning, and social determinants of health. To complement traditionally measured clinical outcomes, the surveys are administered at the same time points at which clinical data are routinely collected. As of September 2023, PRO data have been collected from 993 patients across 25 different centers. With the goal of incorporating these important patient perspectives into standard clinical care, the CIBMTR has added the PRO data to Data Back to Centers (DBtC). Through expanding the data types represented in the registry, the CIBMTR aims to support holistic research accounting for the patients' perspective in improving patient outcomes. CIBMTR PRO data aim to provide a foundation for future large-scale, population-level evaluations to identify areas for improvement, emerging disparities in access and health outcomes (eg, by age, race, and ethnicity), and new therapies that may impact current treatment guidelines. Continuing to collect and grow the PRO data is critical for understanding these changes and identifying methods for improving patients' quality of life., (Copyright © 2024 The American Society for Transplantation and Cellular Therapy. Published by Elsevier Inc. All rights reserved.)

Background: The smartphone Preemie Prep for Parents (P3) program was developed to address the gap in prenatal education of preterm birth in high-risk pregnancies. Despite a higher incidence of preterm birth, Black women are less likely to receive prenatal education., Methods: Pregnant women with medical conditions that predisposed them to preterm birth were randomized to receive the P3 program or links to American College of Obstetricians and Gynecologists webpages (control). The P3 group received periodic text messages, starting as early as 18 weeks gestational age, each with a link to a short, animated educational video. Participants completed the Parent Prematurity Knowledge Questionnaire, PROMIS Anxiety scale, and a feedback survey. This is a subgroup analysis of the Black, non-Hispanic participants in the P3 trial., Results: Of the 26 Black non-Hispanic women enrolled, the P3 group (n=14) had higher knowledge scores than the control group (n=12), 67.5% correct vs. 43.6% (difference 24.0; 95% CI, 7.4 to 40.6), without experiencing an increase in anxiety. More P3 participants reported discussing preterm birth with their partner (100%) than control participants (57%; difference 43; 95% CI, 6 to 80)., Conclusions: The P3 program appears to be an effective method of providing preterm birth education to Black pregnant women.

Background: While nearly 50% of adult women report at least one episode of urinary incontinence (UI), most never receive treatment., Objective: To better integrate primary and specialty UI care, we conducted (i) an environmental scan to assess the availability of key pathway resources in primary care, (ii) interviews with primary care providers to understand barriers to care, and (iii) a pilot UI care pathway intervention., Methods: Environmental scan: Clinic managers from all primary care clinics within a Midwestern healthcare system were invited to participate in an interview covering the availability of clinic resources. Provider interviews: Primary care providers were invited to participate in an interview covering current practices and perceived barriers to UI care. Pilot UI care pathway: Patients who screened positive for UI were provided resources for first-line behavioral management. Pilot patients completed questionnaires at baseline, 8 weeks, and 6 months., Results: While many clinics had point-of-care urinalysis (17/21, 81%), most did not have a working bladder ultrasound (14/21, 67%) or on-site pelvic floor physical therapy (18/21, 86%). Providers (n = 5) described barriers to completing almost every step of diagnosis and treatment for UI. The most persistent barrier was lack of time. Patients (n = 15) reported several self-treatment strategies including avoiding bladder irritants (7/15, 47%) and performing Kegel exercises (4/15, 27%). Five patients (33%) requested follow-up care. At 6 months, patients reported small improvements in UI symptoms., Conclusion: Promising results from a novel UI care pathway pilot indicate that streamlining UI care may assist primary care providers in the first-line treatment of UI., (© The Author(s) 2024. Published by Oxford University Press. All rights reserved. For commercial re-use, please contact reprints@oup.com for reprints and translation rights for reprints. All other permissions can be obtained through our RightsLink service via the Permissions link on the article page on our site—for further information please contact journals.permissions@oup.com.)

Background: Central venous access devices (CVAD) are associated with central line associated bloodstream infection (CLABSI) and venous thromboembolism (VTE). We identified trends in non-intensive care unit (ICU) CVAD utilization, described complication rates, and compared resources between low and high CVAD sites., Methods: We combined data from the Pediatric Health Information System (PHIS) database and surveys from included hospitals. We analyzed 10-year trends in CVAD encounters for non-ICU children between 01/2012-12/2021 and described variation and complication rates between 01/2017-12/2021. Using Fisher's exact test, we compared resources between low and high CVAD users., Results: CVAD use decreased from 6.3% to 3.8% of hospitalizations over 10 years. From 2017-2021, 67,830 encounters with CVAD were identified. Median age was 7 (IQR 2-13) years; 46% were female. Significant variation in CVAD utilization exists (range 1.4-16.9%). Rates of CLABSI and VTE were 4.0% and 3.4%, respectively. Survey responses from 33/41 (80%) hospitals showed 91% had vascular access teams, 30% used vascular access selection guides, and 70% used midline/long peripheral catheters. Low CVAD users were more likely to have a team guiding device selection (100% vs 43%, p = 0.026)., Conclusions: CVAD utilization decreased over time. Significant variation in CVAD use remains and may be associated with hospital resources., Impact: Central venous access device (CVAD) use outside of the ICU is trending down; however, significant variation exists between institutions. Children with CVADs hospitalized on the acute care units had a CLABSI rate of 4% and VTE rate of 3.4%. 91% of surveyed institutions have a vascular access team; however, the services provided vary between institutions. Even though 70% of the surveyed institutions have the ability to place midline/long peripheral catheters, the majority use these catheters less than a few times per month. Institutions with low CVAD use are more likely to have a vascular access team that guides device selection., (© 2024. The Author(s), under exclusive licence to the International Pediatric Research Foundation, Inc.)

Importance: Guideline-recommended medications for overactive bladder and urge urinary incontinence (OAB/UUI) are effective but have high costs and side effects. Little is known about patient concerns regarding these medications when prescribed by their primary care providers (PCPs)., Objective: The aim of the study was to describe PCP-patient interactions when prescribing medications for OAB/UUI, specifically clinical concerns, cost and authorization issues, and mode of communication for these interactions., Study Design: Using electronic health records, we identified a retrospective cohort of women aged 18-89 years who were prescribed a medication for OAB/UUI during a primary care office visit from 2017 to 2018. We examined the electronic health record from initial prescription through 15 subsequent months for documentation of prior authorization requests and patient concerns about cost, side effects, or ineffectiveness. The association of patient demographics, comorbidity, and medication class with these concerns was examined with logistic regression models., Results: Overall, 46.2% of patients (n = 123) had 1 or more OAB/UUI medication concerns, and 52 reported outside an office visit. Only higher comorbidity was associated with reduced concern of any type. Although the overall percent age of patients reporting concerns was similar by medication type, the patterns of concern type varied. Compared with those taking short-acting antimuscarinics, patients taking long-acting antimuscarinics other than oxybutynin were less likely to have side effect concerns (adjusted odds ratio 0.35, 95% CI 0.16-0.78) and more likely to have cost concerns (adjusted odds ratio 5.10, 95% CI 1.53-17.03)., Conclusions: Patient concerns regarding OAB/UUI medications were common in primary care practices and frequently reported outside of office visits. However, the patterns of concerns (cost vs side effects) varied between medication classes., Competing Interests: The authors have declared they have no conflicts of interest., (Copyright © 2024 American Urogynecologic Society. All rights reserved.)

Objective: To evaluate the psychometric properties of the AL-PROfile, a patient-reported outcome measure combining the Patient-Reported Outcomes Measurement Information System (PROMIS)-29, two items from PROMIS Cognitive Function, and select Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE) items., Methods: Content validity was assessed through cognitive debriefing interviews of 20 patients who completed the AL-PROfile (Study 1). Study 2 involved 297 participants who completed the AL-PROfile and Medical Outcomes Study 36-item Short-Form Health Survey (SF-36). Reliability (internal consistency and test-retest reliability) and validity (convergent and discriminant validity, known groups validity by stage/organ involvement) were calculated., Results: Study 1 participants found the AL-PROfile straightforward confirming the relevance of the included content. Some felt that certain questions were not related to their amyloidosis experience. Study 2 demonstrated acceptable internal consistency for all domains/items except PROMIS Cognitive Function and acceptable test-retest reliability for all except PROMIS Cognitive Function and PRO-CTCAE nausea. Large correlations were seen for the same domain across measures while correlations for divergent domains within a measure and different domains across different measures were small. The PRO-CTCAE items showed small to medium correlations with each other and with PROMIS and SF-36 domains. Stage was associated with physical function, fatigue, social roles, swelling, and shortness of breath scores., Conclusion: The AL-PROfile has acceptable reliability and validity for use in systemic light chain amyloidosis patients., (© 2024 The Authors. European Journal of Haematology published by John Wiley & Sons Ltd.)

Background: The Center for International Blood and Marrow Transplant Research (CIBMTR) provides a 1-year overall survival calculator to estimate outcomes for individual patients before they undergo allogeneic hematopoietic cell transplantation (HCT) to inform risk. The calculator considers pre-HCT clinical and demographic characteristics, but not patient-reported outcomes (PROs). Because pre-HCT PRO scores have been associated with post-HCT outcomes, the authors hypothesized that adding PRO scores to the calculator would enhance its predictive power., Methods: Clinical data were obtained from the CIBMTR and the Blood and Marrow Transplant Clinical Trials Network. The PRO measures used were the 36-Item Short Form Survey (SF-36) and the Functional Assessment of Cancer Therapy-Bone Marrow Transplantation. One thousand thirty-three adult patients were included., Results: When adjusted for clinical characteristics, the SF-36 physical component score was significantly predictive of 1-year survival (hazard ratio [HR], 0.88; 95% confidence interval [CI], 0.81-0.95; p = .0015), whereas the mental component score was not (HR, 1.02; 95% CI, 0.95-1.10; p = 0.6396). The baseline single general health question on the SF-36 was also significantly associated with mortality (HR, 1.91 for those reporting fair/poor health vs. good, very good, or excellent health; 95% CI, 1.33-2.76; p = .0005). The addition of PRO scores to the calculator did not result in a significant change in the model's predictive ability. Self-reported pre-HCT scores were strongly predictive of self-reported health status (odds ratio, 3.35; 95% CI, 1.66-6.75; p = .0007) and quality of life (odds ratio, 3.24; 95% CI, 1.93-5.41; p < .0001) after HCT., Conclusions: The authors confirmed the significant, independent association of pre-HCT PRO scores with overall survival, although adding PRO scores to the survival calculator did not improve its performance. They also demonstrated that a single general health question was as accurate as the full measure for predicting survival, an important finding that may reduce respondent burden and promote its inclusion in routine clinical practice. Validation of these findings should be performed., (© 2024 American Cancer Society.)

Importance: Although behavioral modifications, medications, and other interventions can improve urinary incontinence (UI), many women never receive them., Objectives: To better characterize UI treatment patterns in primary care, we examined prescriptions and referrals to pelvic floor physical therapy (PFPT) and specialist physicians within a large Midwestern academic health system., Study Design: Electronic health records were queried to identify a cohort of adult female patients receiving a new UI diagnosis during outpatient primary care visits from 2016 to 2020. Urinary incontinence referrals and referral completion were examined for the overall cohort, and medication prescriptions were examined for women with urgency or mixed UI. Logistic regression was used to assess the association of prescriptions and/or referrals with patient demographics, comorbidities, and UI diagnosis dates., Results: In the year after primary care UI diagnosis, 37.2% of patients in the overall cohort (n = 4,382) received guideline-concordant care. This included 20.6% of women who were referred for further management: 17.7% to urology/urogynecology and 3.2% to PFPT. Most women who were referred attended an initial appointment. Among those with urgency (n = 2,398) or mixed UI (n = 552), 17.1% were prescribed medication. Women with stress (odds ratio [OR], 3.10; 95% CI, 2.53-3.79) and mixed UI (OR, 6.17; 95% CI, 4.03-9.66) were more likely to be referred for further management, and women diagnosed during the COVID-19 pandemic were less likely to be referred for further care (OR, 0.39; 95% CI, 0.29, 0.48)., Conclusion: Only slightly above 1 in 3 women with a new diagnosis of UI in primary care received guideline-based medications or referrals within 1 year, suggesting missed opportunities for timely care., Competing Interests: The authors have declared they have no conflicts of interest., (Copyright © 2023 American Urogynecologic Society. All rights reserved.)

Purpose: Oral adjuvant endocrine therapy (AET) is an effective treatment for hormone receptor positive breast cancer to decrease recurrence and mortality, but adherence is poor. This study explored post-menopausal women's experiences with AET, with a particular focus on adherence to AET as well as distress and symptoms experienced prior to and during AET treatment., Methods: Participants were recruited from a hospital registry, stratified by adherence to/discontinuation of AET. Telephone interviews followed a semi-structured interview guide and were recorded and transcribed verbatim. Transcripts were systematically coded using team-based coding, with analysis of themes using a grounded theory approach., Results: Thirty-three participants were interviewed; ages ranged from 57 to 86 years. Participants included 10 discontinued patients and 23 patients who completed their AET course or were adherent to AET at the time of interviewing. Both adherent and discontinued patients reported symptoms throughout their AET treatment course, and both attributed symptoms to factors other than AET (e.g., older age and pre-existing comorbidities). However, discontinued patients were more likely to attribute symptoms to AET and to describe difficulty managing their symptoms, with some directly citing symptoms as the reason for discontinuing AET therapy. Conversely, adherent patients were more likely to describe the necessity of taking AET, despite symptoms., Conclusions: AET adherence was associated with beliefs about AET, symptom attribution, and symptom management. Routine symptom monitoring during AET and addressing both symptoms and patients' understanding of their symptoms may promote adherence to AET., (© 2024. The Author(s), under exclusive licence to Springer-Verlag GmbH Germany, part of Springer Nature.)

Introduction: Childhood exposure to lead has severe health consequences including long-term physical, behavioral, and learning problems. Lead poisoning often occurs in the home and persists as a form of environmental injustice, disparately impacting certain children based on factors such as socioeconomic status, immigration status, and race. Because abatement is costly, many prevention programs rely on educational interventions. We conducted a systematic review to assess the effectiveness of educational interventions on reducing blood lead levels (BLL) in children., Content: Following PRISMA-P guidelines, a librarian-guided search strategy incorporated database-specific subject headings and keywords related to lead poisoning and education, and encompassed four databases: Ovid MEDLINE, Scopus, Web of Science Core Collection and CINAHL. Two reviewers screened the results for those that met inclusion criteria (original research, study population of children under 18 years, inclusion of an educational intervention, outcome of BLL)., Summary and Outlook: We screened the titles of 2,062 non-duplicate studies, the abstracts of 78 studies, and full texts of 23 articles, resulting in 17 articles that met eligibility criteria. Thirteen studies used multi-pronged interventions, which precluded comprehensive assessment of the effectiveness of the educational component. Interventions that had success in lowering BLL included some notable elements: longevity of intervention, consideration of culture and ethnicity; use of a community or home-based approach; and provision of supplies or assistance with cleaning. Of the four of studies that used solely educational interventions, three were successful in reducing BLL. Among the 12 studies that used a control group, six found their interventions to be successful in reducing BLL. This review found that educational interventions, either alone or as part of a multi-pronged approach, do not consistently reduce BLL in children. However, educational interventions may decrease severity of lead poisoning in children when more robust interventions are not feasible., (© 2024 Walter de Gruyter GmbH, Berlin/Boston.)

Importance: Patients with chronic myeloid leukemia (CML) who have a sustained deep molecular response using tyrosine kinase inhibitors (TKIs) can safely attempt to stop their use. As these medications are very costly, this change in treatment protocols may result in large savings., Objective: To estimate future savings from attempting to stop TKI use among patients with CML who have deep molecular response., Design, Setting, and Participants: A microsimulation model was developed for this decision analytical modeling study to estimate costs for US adults moving from using a TKI, to attempting discontinuation and then reinitiating TKI therapy, if clinically appropriate. Estimates were calculated for US patients who currently have CML and simulated newly diagnosed cohorts of patients over the next 30 years., Exposure: Attempting to stop using a TKI., Main Outcomes and Measures: Estimated savings after attempted discontinuation of TKI use., Results: A simulated population of individuals with CML in 2018 and future populations were created using estimates from the SEER*Explorer website. The median age at diagnosis was 66 years for men and 65 years for women. Between 2022 and 2052, the savings associated with eligible patients attempting discontinuation of TKI therapy was estimated at more than $30 billion among those currently diagnosed and over $15 billion among those who will develop CML in the future, for a total savings of over $54 billion by 2052 for drug treatment and polymerase chain reaction testing. The estimate is conservative as it does not account for complications and other health care-associated costs for patients continuing TKI therapy., Conclusions and Relevance: The findings of this decision analytical modeling study of patients with CML suggest that attempting discontinuation of TKI therapy could save over $54 billion during the next 30 years. Further education for patients and physicians is needed to safely increase the number of patients who can successfully attain treatment-free remission.

Background: Decisions for how to resolve infertility are complex and may lead to regret. We examined whether couples and individuals who sought a consultation from a reproductive specialist for infertility later expressed decisional regret about their family-building choices and whether regret was associated with parental role, family-building paths, or outcomes., Methods: This longitudinal mixed methods study included women and their partners who completed a questionnaire prior to their initial consultation with a reproductive specialist and 6 years later. The six-year questionnaire included the Ottawa Decision Regret Scale referencing "the decisions you made about how to add a child to your family." A score of 25+ indicates moderate-to-severe regret. Additional items invited reflections on family-building decisions, treatments, and costs. A systematic content analysis assessed qualitative themes., Results: Forty-five couples and 34 individuals participated in the six-year questionnaire (76% retention rate), Half (n = 61) of participants expressed no regret, which was similar by role (median 0 for women and supporting partners, F = .08; p = .77). One in 5 women and 1 in 7 partners expressed moderate-to-severe regret. Women who did not pursue any treatment had significantly higher regret (median 15; F = 5.6, p < 0.01) compared to those who pursued IVF (median 0) or other treatments (median 0). Women who did not add a child to their family had significantly higher regret (median 35; F = 10.1, p < 0.001) than those who added a child through treatment (median 0), through fostering/adoption (median 0), or naturally (median 5). Among partners, regret scores were not associated with family-building paths or outcomes. More than one-quarter of participants wished they had spent less money trying to add a child to their family. Qualitative themes included gratitude for parenthood despite the burdensome process of family-building as well as dissatisfaction or regret about the process. Results should be confirmed in other settings to increase generalizability., Conclusion: This longitudinal study provides new insight into the burden of infertility. For women seeking parenthood, any of the multiple paths to parenthood may prevent future decision regret. Greater psychosocial, financial, and decision support is needed to help patients and their partners navigate family-building with minimal regret., (© 2023. The Author(s).)

Objective: To assess the impact of organ involvement on patient-reported outcomes (PROs) in light chain (AL) amyloidosis., Methods: PROs were evaluated using the KCCQ-12, PROMIS-29 + 2, and SF-36 in individuals with AL amyloidosis. The 2004 Mayo system was used to stage disease and cardiac, neurologic, and renal involvement was considered. Global physical and mental health (MH) scores, physical function (PF), fatigue, social function (SF), pain, sleep, and MH domains were evaluated. Effect sizes between scores were measured using Cohen's d., Results: Of 297 respondents, the median age at diagnosis was 60 years with 58% cardiac, 58% renal, and 30% neurologic involvement. Fatigue, PF, SF, and global physical health with PROMIS and SF-36 discriminated the most by stage. Significant discrimination in PROMIS and/or SF-36 was seen in PF, fatigue, and global physical health with cardiac involvement. For neurologic involvement, PF, fatigue, SF, pain, sleep, global physical, and MH with PROMIS and role physical, vitality, pain, general health, and physical component summary with SF-36 were discriminatory. For renal amyloid, pain by SF-36 and PROMIS, and SF-36 MH and role emotional subscales were significant., Conclusions: Fatigue, PF, SF, and global physical health can discriminate stage, cardiac and neurologic, but not renal, AL amyloidosis involvement., (© 2023 John Wiley & Sons A/S. Published by John Wiley & Sons Ltd.)

Background: Gestational diabetes (GDM) complicates 10% of pregnancies in the US. First-line treatment is medical nutrition therapy (MNT) and exercise. Second line is pharmacotherapy. The definition of what constitutes an unsuccessful trial of MNT and exercise has not been established. Tight glycemic control has been demonstrated to reduce GDM-related neonatal and maternal clinical complications. However, it could also increase rates of small-for-gestational age and carry negative effects on patient-reported outcomes such as anxiety and stress. We will study the effect of earlier and stricter pharmacotherapy in GDM on clinical and patient-reported outcomes., Methods: GDM and pharmacotherapy (GAP) study is a two-arm parallel, pragmatic randomized controlled trial, where 416 participants with GDM are randomized 1:1 to: 1) Intervention group - insulin initiation at 20% elevated glucose values on a weekly glucose log following MNT and exercise trial and insulin titration to keep elevated glucose values <20%; or 2) Active control group - insulin initiation at 40% elevated glucose values on a weekly log following MNT and exercise and insulin titration to keep elevated glucose values <40%. The primary outcome is a composite neonatal outcome of large-for-gestational-age, macrosomia, birth trauma, preterm birth, hypoglycemia, and hyperbilirubinemia. Secondary outcomes include preeclampsia, cesarean birth, small-for-gestational-age, maternal hypoglycemia, and patient-reported outcomes of anxiety, depression, perceived stress, and diabetes self-efficacy., Conclusions: The GAP study will investigate the optimal glycemic threshold for pharmacotherapy addition to MNT and exercise in GDM. The GAP study will promote standardization in GDM management and will have direct relevance for clinical practice., Competing Interests: Declaration of Competing Interest The authors declare that they have no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper., (Copyright © 2023 Elsevier Inc. All rights reserved.)

Importance: Preterm birth is a leading cause of infant mortality and child morbidity. Preterm birth is not always unexpected, yet standard prenatal care does not offer anticipatory education to parents at risk of delivering preterm, which leaves parents unprepared to make health care choices during the pregnancy that can improve survival and decrease morbidity in case of preterm birth., Objective: To evaluate the effect of the Preemie Prep for Parents (P3) program on maternal knowledge of preterm birth, preparation for decision-making, and anxiety., Design, Setting, and Participants: Recruitment for this randomized clinical trial conducted at a US academic medical center took place from February 3, 2020, to April 12, 2021. A total of 120 pregnant persons with a risk factor for preterm birth were enrolled between 16 and 21 weeks' gestational age and followed up through pregnancy completion., Intervention: Starting at 18 weeks' gestational age, P3 program participants received links delivered via text message to 51 gestational age-specific short animated videos. Control participants received links to patient education webpages from the American College of Obstetricians and Gynecologists., Main Outcomes and Measures: At 25 weeks' gestation, scores on the Parent Prematurity Knowledge Questionnaire (scored as percent correct), Preparation for Decision Making Scale (scored 0-100), and Patient-Reported Outcomes Measurement Information System (PROMIS) Anxiety computerized adaptive test. Analysis was based on an intention to treat., Results: A total of 120 pregnant participants (mean [SD] age, 32.5 [4.9] years) were included in the study; 60 participants were randomized to each group. Participants in the P3 group scored higher than those in the control group on knowledge of long-term outcomes at 25 weeks (88.5% vs 73.2%; estimated difference, 15.3 percentage points; 95% CI, 8.3-22.5 percentage points; P < .001). Participants in the P3 group reported being significantly more prepared than did participants in the control group for neonatal resuscitation decision-making at 25 weeks (Preparation for Decision Making Scale score, 76.0 vs 52.3; difference, 23.7; 95% CI, 14.1-33.2). There was no difference between the P3 group and the control group in anxiety at 25 weeks (mean [SE] PROMIS Anxiety scores, 53.8 [1.1] vs 54.0 [1.1]; difference, -0.1; 95% CI, -3.2 to 2.9)., Conclusions and Relevance: In this randomized clinical trial, pregnant persons randomly assigned to the P3 program had more knowledge of core competencies and were more prepared to make decisions that affect maternal and infant health, without experiencing worse anxiety. Mobile antenatal preterm birth education may provide a unique benefit to parents with preterm birth risk factors., Trial Registration: ClinicalTrials.gov Identifier: NCT04093492.

Background: We conducted a cross-sectional study to characterize health-related quality of life and symptom burden in individuals living with light chain (AL) amyloidosis., Methods: Members of the Amyloidosis Support Groups, Inc. with AL amyloidosis who consented to this IRB-approved survey provided information on their amyloidosis diagnosis, treatment, symptoms, and functioning. HRQL was measured using PROMIS and PRO-CTCAE questionnaires., Results: Among 297 participants who responded, the median age at diagnosis was 60 years (23-82) with 52% female and 90% white race. There were 69% AL (lambda) and 39% reported 3 or more organs involved with amyloidosis (58% cardiac, 58% renal, 30% neurological AL). Time from diagnosis was less than 2 years in 64 (22%), 2-5 years in 105 (36%), > 5 years in 126 (43%), and unknown in 2 (< 1%) individuals. Therapy included prior chemotherapy in 88% and stem cell transplant in 52%. Fifty percent of the cohort was on active treatment. Multiple domains were impaired in AL amyloidosis compared to the general population, including physical function, fatigue, and social roles. While highest among those within 2 years of diagnosis, high symptom burden was also seen in long-term survivors. A trend to decreased severity and number of impaired symptoms was seen with longer treatment-free interval but many symptoms remained persistent., Conclusions: Significant and persistent symptom burden is seen in AL amyloidosis. Patient-reported outcomes should be routinely measured and used to provide best supportive care to all AL amyloidosis patients, including long-term survivors and those not on active therapy., (© 2023. The Author(s), under exclusive licence to Springer Nature Switzerland AG.)

Background: A validated measure assessing sexual sensory functions of the breast is needed to optimize sexual and other health outcomes after breast procedures., Aim: To describe the development of a patient-reported outcome measure (PROM) to assess breast sensorisexual function (BSF)., Methods: We applied the PROMIS standards (Patient Reported Outcomes Measurement Information System) for measure development and evaluation of validity. An initial conceptual model of BSF was developed with patients and experts. A literature review yielded a pool of 117 candidate items that underwent cognitive testing and iteration. Forty-eight items were administered to an ethnically diverse, national panel-based sample of sexually active women with breast cancer (n = 350) or without (n = 300). Psychometric analyses were performed., Outcomes: The main outcome was BSF, a measure that assesses affective (satisfaction, pleasure, importance, pain, discomfort) and functional (touch, pressure, thermoreception, nipple erection) sensorisexual domains., Results: A bifactor model fit to 6 domains-excluding 2 domains with only 2 items each and 2 pain-related domains-revealed a single general factor representing BSF that may be adequately measured by the average of the items. This factor, with higher values denoting better function and with the standard deviation set to 1, was highest among women without breast cancer (mean, 0.24), intermediate among women with breast cancer but not bilateral mastectomy and reconstruction (-0.01), and lowest among those with bilateral mastectomy and reconstruction (-0.56). Between women with and without breast cancer, the BSF general factor accounted for 40%, 49%, and 100% of the difference in arousal, ability to orgasm, and sexual satisfaction, respectively. Items in each of 8 domains demonstrated unidimensionality (ie, they measured 1 underlying BSF trait) and high Cronbach's alphas for the entire sample (0.77-0.93) and the cancer group (0.71-0.95). Correlations with sexual function, health, and quality of life were positive for the BSF general factor and mostly negative for the pain domains., Clinical Implications: The BSF PROM can be used to assess the impact of breast surgery or other procedures on the sexual sensory functions of the breast in women with and without breast cancer., Strengths and Limitations: The BSF PROM was developed by using evidence-based standards, and it applies to sexually active women with and without breast cancer. Generalizability to sexually inactive women and other women warrants further study., Conclusion: The BSF PROM is a measure of women's breast sensorisexual function with evidence of validity among women affected and unaffected by breast cancer., (© The Author(s) 2023. Published by Oxford University Press on behalf of The International Society of Sexual Medicine. All rights reserved. For permissions, please e-mail: journals.permissions@oup.com.)

Purpose: Cognitive dysfunction is a known complication following cellular therapies (CT), which can be assessed through performance based and patient-reported measures. We performed a systematic scoping review to assess self-reported cognitive function measures used among adult CT patients and describe long-term results, including associations with clinical outcomes., Methods: Library databases were searched from inception to February 2020 according to PRISMA guidelines. Additional studies were identified through reference lists and trial protocols. Two members of the research team screened titles and abstracts and resolved discrepancies. Articles that met eligibility criteria continued to full-text review, with 25% double screening. Articles were removed if they (1) were not original research, peer-reviewed articles; (2) were the wrong disease, age, or treatment-specific patient population; (3) did not use patient-reported outcomes; (4) did not separately report cognitive function outcomes., Results: Of the1952 articles, 56 were included. Twenty-one patient-reported measures of cognitive function were used; most frequently the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC-QLQ-C30), which includes a two-item cognitive function subscale (57%; n = 32). Thirteen studies collected performance-based and self-reported measures and of those (n = 6) who assessed associations found moderate correlations (range r = .13-.58). Longitudinal patterns showed declines in cognitive function soon after treatment (< 1 month) returning to baseline at 1 year. Cognitive function was often associated with other quality of life measures, chiefly depression (n = 5)., Conclusions: EORTC-QLQ-C30 is the most commonly used to measure, though there remain numerous measures used, including several measures with little previous validation and investigator developed items., (© 2022. The Author(s), under exclusive licence to Springer Nature Switzerland AG.)

Toxicities after chimeric antigen receptor T cell (CAR-T) therapy are well known, yet the patient experience during and after CAR-T therapy has not been well described outside of the trial setting. We explored the patient experience after CAR-T therapy to inform the patient-reported outcomes (PRO) measurement approach for the Center for International Blood and Marrow Transplant Research (CIBMTR). We recruited (1) adult patients diagnosed with a hematologic malignancy 14 days to 6 months after receiving a commercial CAR T cell product who had agreed to be contacted by the CIBMTR, (2) caregivers of those patients, and (3) clinical experts in CAR-T therapy. Telephone interviews were conducted following a semistructured guide that included open-ended questions about symptoms and functioning. We conducted a systematic content analysis of each transcript using prespecified codes representing common domains of health, as well as open coding for emergent themes. Forty patients at 29 centers, 15 of their caregivers, and 15 experts from 9 centers participated, representing diversity with respect to age, sex, race/ethnicity, and years in practice (experts). Patients, caregivers, and experts shared largely consistent impressions of the patient experience after CAR-T therapy. Commonly described themes included anxiety, cognitive dysfunction, depression, fatigue, pain, impaired physical function, gastrointestinal symptoms, sexual dysfunction, sleep difficulties, need for support, financial impact, hospitalization, communication with healthcare providers, and the COVID-19 pandemic. Limitations in patients' ability to participate in social roles and activities was the most prevalent theme, found in nearly all interviews. In the setting of CAR-T therapy, a multidimensional approach to PRO measurement is needed that includes physical, mental, and social health, as well as the financial impact of this novel treatment. High-quality existing PRO tools are available to measure these concepts. Results will inform the CIBMTR measurement of PROs after CAR-T therapy and may be applicable to other CAR-T studies that aim to represent patient experiences., (Copyright © 2023 The American Society for Transplantation and Cellular Therapy. Published by Elsevier Inc. All rights reserved.)

Background: Parents of premature infants engage in shared decision-making regarding the care of their infant. The process of prenatal counseling typically involves a verbal conversation with a neonatal provider during hospitalization. Support people may not be available, and the pregnant person's memory is impaired by medications, pain, and stress. The American Academy of Pediatrics, American College of Obstetricians and Gynecologists, and the Eunice Kennedy Shriver National Institute of Child Health and Human Development have called for improvements to this process, including the development of educational aids., Objective: This study aimed to investigate whether a multimedia tablet would be more effective than a paper handout in supplementing verbal clinician counseling during preterm birth hospitalization., Study Design: This was a randomized controlled trial including English-speaking pregnant people aged ≥18 years and hospitalized at 22 to 33 weeks' gestation for preterm birth. Exclusion criteria were known fetal or chromosomal anomaly and delivery before study completion. Pregnant people received either a multimedia tablet or a paper handout before verbal clinician counseling. Preintervention assessment included demographics and State-Trait Anxiety Inventory, and postintervention assessment included the Parent Knowledge of Premature Birth Questionnaire and State-Trait Anxiety Inventory. Continuous variables were analyzed by t-test and categorical variables by Fisher exact test., Results: A total of 122 pregnant people referred for counseling were screened; 76 were randomized, and 59 completed the study. Demographics were similar between groups, except that pregnant people in the handout group were older (mean 32 vs 29 years; P=.03). The multimedia tablet group (n=32) was less likely to report reviewing all the educational material than the paper handout group (n=27) (41% vs 72%; P=.037). Both groups correctly answered a similar number of knowledge items (P=.088). Postintervention state anxiety decreased in both groups (P<.0001), with no difference between groups. Computerized tracking showed that the multimedia group spent a median of 37 minutes reviewing the tablet., Conclusion: Contrary to our hypothesis, a paper handout and multimedia tablet were equally effective in the labor unit for supplementing verbal preterm birth counseling, and both decreased parental anxiety., (Copyright © 2023 Elsevier Inc. All rights reserved.)

Background: Pain and physical health domains included in Patient-Reported Outcomes Measurement Information System® (PROMIS®) can be administered as short forms (SF) or as computer adaptive tests (CAT). CAT is ideal in many settings but cannot be administered without specialized technology. We compared SF and CAT to identify items for customized SFs to improve the SF performance for children with sickle cell disease (SCD)., Methods: Eligible children 8-17 years old were administered CATs for 5 domains of physical health and 2 domains of pain, followed by any items on the corresponding SF that were not included in the CAT assessments. We describe the range of scores on the CAT and SFs, including the percentage of participants with floor or ceiling effects using the SF. The agreement and correlation between CAT and SF scores were assessed using Bland-Altman plots. Items frequently offered on CAT that had variable responses and were not already present on SF are recommended as additional items for customized SFs., Results: Among 90 children with SCD, there were strong correlations between CAT and SF scores (Concordance Correlation Coefficient > 0.8) however, the SFs for fatigue, mobility, strength impact, pain behavior, and pain interference had substantial floor/ceiling effects. Fatigue, mobility, physical stress experience, and pain behavior domains had items that were frequently offered on CAT, variable responses, and were not present on the SF., Conclusions: Adding items to the SFs for the fatigue, mobility, physical stress experience, and pain behavior domains may improve these domains' SFs performance for children with SCD., (© 2023. The Author(s).)

For myelodysplastic syndrome (MDS), allogeneic hematopoietic cell transplantation (alloHCT) is the only available curative therapy. The Blood and Marrow Transplant Clinical Trials Network study 1102 (BMT CTN 1102, NCT02016781) was a multicenter, biologic assignment trial based on matched donor availability in adults aged 50-75 with higher risk de novo MDS who were candidates for reduced-intensity conditioning (RIC) alloHCT. The primary analysis showed that those who received alloHCT had a survival benefit, but whether this is at the cost of worse quality of life (QOL) has not been described in detail. English- and Spanish-speaking trial participants completed the Functional Assessment of Cancer Therapy-General (FACT-G), the SF-36, and the EQ-5D, at enrollment, every 6 months until 24 months, and 36 months. We compared patient-reported outcome (PRO) scores between study arms using an inverse probability weighted-independent estimating equation (IPW-IEE) model. Between January 2014 and November 2018, 384 subjects (median age 66.7 years, range: 50.1-75.3) enrolled at 34 centers. PRO completion rates were generally high at 65%-78%. The PRO trajectories for both arms were similar, with most decreasing or stable from baseline to 6 months and improving thereafter. Baseline PRO scores were the most consistent independent predictors of subsequent QOL outcomes and survival, even after controlling for clinical and patient-level factors. For older adults with MDS, the survival advantage associated with donor availability and alloHCT did not come at the cost of worse QOL. These results should reassure older patients and clinicians who prefer a curative approach to treating MDS., (© 2022 Wiley Periodicals LLC.)

Background: The Patient-Reported Outcomes Measurement Information System (PROMIS®) Sexual Function and Satisfaction (SexFS) version 2.0 measurement tool was developed to assess sexual functioning and satisfaction in the general population regardless of health condition and sexual orientation., Aim: The study aimed to evaluate the psychometric properties of the Swedish version of the PROMIS SexFS measure in clinical and nonclinical populations of young adults (aged <40 years)., Methods: The SexFS was answered by a clinical population of young adult women ( n  = 180) and men ( n  = 110) with breast cancer and testicular cancer, respectively, and a nonclinical population of young adult women ( n  = 511) and men ( n  = 324) from the general population. Psychometric properties were evaluated by examining data quality (score distribution, floor and ceiling effects, proportion of missing data), construct validity (corrected item, total correlation, scaling success), and reliability (Cronbach α)., Outcomes: The following domains of the SexFS 2.0 were investigated: Vaginal Lubrication, Vaginal Discomfort, Vulvar Discomfort- Clitoral, Vulvar Discomfort- Labial, Erectile Function, Interest in Sexual Activity, Satisfaction With Sex Life, Orgasm- Ability, and Orgasm- Pleasure., Results: The Swedish version of the SexFS 2.0 generated data of acceptable quality. Some noteworthy floor or ceiling effects were identified across domains and respondent groups. Corrected item totals were used to express the coherence between an item and the other items in the domain. The correlation coefficients were above 0.40 for all items, except for 1 of the items within the Vaginal Discomfort domain and for the items in the Erectile Function domain in the nonclinical group of men. High proportions of scaling success were noted across domains (96%-100%). Reliability was satisfactory (α = 0.74-0.92) for all domains, expect for Erectile Function of the nonclinical group (α = 0.53), due to low variability in item responses, which was improved somewhat (α = 0.65) when combined with the clinical group., Clinical Implications: A flexible tool to measure self-reported sexual function and satisfaction in young men and women is available for researchers and clinicians in Sweden., Strengths and Limitations: The nationwide population-based sample of patients with cancer, identified from national quality registers, minimized selection bias. However, men in the general population had a lower response rate (34%) compared to the other groups, which introduced a risk of bias in estimates. The psychometric evaluation was limited to young adults (aged 19-40 years)., Conclusion: The results provide evidence for the validity and reliability of the Swedish version of the SexFS measure for the assessment of sexual functioning and satisfaction in young adults from both clinical and nonclinical populations., (© The Author(s) 2023. Published by Oxford University Press on behalf of The International Society of Sexual Medicine.)

Early autologous hematopoietic cell transplantation (AHCT) with post-transplant maintenance therapy is standard of care in multiple myeloma (MM). While short-term quality of life (QOL) deterioration after AHCT is known, the long-term trajectories and symptom burden after transplantation are largely unknown. Toward this goal, a secondary analysis of QOL data of the BMT CTN 0702, a randomized controlled trial comparing outcomes of three treatment interventions after a single AHCT (N = 758), was conducted. FACT-BMT scores up to 4 years post-AHCT were analyzed. Symptom burden was studied using responses to 17 individual symptoms dichotomized as 'none/mild' for scores 0-2 and 'moderate/severe' for scores of 3 or 4. Patients with no moderate/severe symptom ratings were considered to have low symptom burden at 1-year. Mean age at enrollment was 55.5 years with 17% African Americans. Median follow-up was 6 years (range, 0.4-8.5 years). FACT-BMT scores improved between enrollment and 1-year and remained stable thereafter. Low symptom burden was reported by 27% of patients at baseline, 38% at 1-year, and 32% at 4 years post-AHCT. Predictors of low symptom burden at 1-year included low symptom burden at baseline: OR 2.7 (1.8-4.1), p < 0.0001; older age: OR 2.1 (1.3-3.2), p = 0.0007; and was related to being employed: OR 2.1 (1.4-3.2), p = 0.0004). We conclude that MM survivors who achieve disease control after AHCT have excellent recovery of FACT-BMT and subscale scores to population norms by 1-year post-transplant, though many patients continue to report moderate to severe severity in some symptoms at 1-year and beyond., (© 2022 Wiley Periodicals LLC.)

Purpose: Multiple myeloma (MM) is the second most common hematologic malignancy in the USA, with higher rates observed in older adults and African Americans (AA). Survivors experience fatigue, bone pain, reduced functioning, and obesity, highlighting the value of developing lifestyle interventions for this diverse group. This study explores lifestyle behaviors and supportive care needs to inform future programs tailored to the MM community., Methods: MM survivors, ≥ 100 days post autologous stem cell transplant (ASCT) with a BMI ≥ 20 kg/m 2 , were recruited from two university hospitals. Diet, physical activity, and quality of life (QOL) were measured using validated measures. Qualitative interviews gathered information on survivorship needs and interests related to supportive interventions. Quantitative data was analyzed using descriptive statistics; qualitative data were analyzed using deductive strategies., Results: Seveny-two MM survivors participated (65% white, 35% black). Participants were 62.5 ± 15.8 years of age. Fifty percent were classified as obese and 65% were insufficiently active. Participants reported diets high in added sugars and saturated fats. QOL measures indicated clinically significant challenges in physical and sexual function. Most (87%) were interested in a lifestyle program. Predominant themes regarding survivors' desires for a lifestyle program included social support, guided exercise, meal preparation support, and disease management information., Conclusion: This study demonstrates the need for and interest in lifestyle change support among a racially diverse sample of MM survivors. Interventions that are group-based, target knowledge gaps, social connections, accountability, and provide structured framework with professional instruction will best address the needs of this survivor population., (© 2022. The Author(s), under exclusive licence to Springer-Verlag GmbH Germany, part of Springer Nature.)

Objective: To examine patient barriers to intermittent pneumatic compression (IPC) device compliance during antepartum hospital admissions., Methods: This was a prospective survey study of a diverse group of pregnant patients who were admitted to the antepartum floor in a quaternary care hospital from June 2019 through March 2021. Patients receiving pharmacotherapy for venous thromboembolism prophylactic or therapeutic indications were excluded. The primary outcome was patient reported barriers to use of IPC during antepartum hospital admission. Responses were summarized using descriptive statistics., Results: A total of 40 patients were surveyed. All participants correctly identified the purpose of IPC. When asked regarding IPC importance, 7.5% reported not important, 40% reported somewhat important but not a priority, 5% important only on the first day they were given, and 47.5% reported important whenever the patient was in bed. The most bothersome characteristics of IPC use identified by the respondents on a Likert scale from 1 to 5 were sweating, made the legs too hot, restricted movement, and inconvenience and time spent putting on and taking off the IPC. Making the device wireless/cordless (77.5%), using a lighter weight or cooler material for the cuffs (55%), and better availability of nurses to assist with putting on, taking off, and troubleshooting the IPC (17.5%) were identified by patients as ways to increase compliance with IPC., Conclusions: This survey identified several modifiable barriers to the use of IPC on the antepartum floor.

Purpose: Bladder diaries are a key source of information about lower urinary tract symptoms (LUTS); however, many patients do not complete them as instructed. Questionnaire-based patient-reported outcome measures (PROMs) are another option for reporting LUTS but may have recall bias. We assessed the strength of the associations between PROMs and a 3-day bladder diary., Materials and Methods: Symptomatic adults from 6 tertiary care sites completed a 3-day paper bladder diary and 3-, 7-, and 30-day electronic PROMs. We assessed the linear associations between mapped pairs of diary variables and responses to PROM items using biserial and polyserial correlation coefficients with 95% confidence intervals., Results: Of 290 enrolled participants, 175 (60%) completed the bladder diary as instructed and at least one corresponding PROM. Linear associations were strongest between the diary and 3-day recall of daytime frequency (r = 0.75) and nighttime frequency (r = 0.69), followed by voids with urgency sensations (r = 0.62), and an item reporting any incontinence (r = 0.56). Linear associations between bladder diary and specific incontinence variables (e.g., stress, urgency) were low to negligible (ranging from r = 0.16-0.39). Linear associations were consistent across the 3-, 7-, and 30-day recall periods., Conclusions: Missing and unusable bladder diary data were common, highlighting the patient burden associated with this method of data collection. A questionnaire-based PROM is a reasonable alternative to a diary for reporting voiding frequency and may offer an easier option for reporting some symptoms., (© 2022 Wiley Periodicals LLC.)

For patients with optimally treated chronic myeloid leukemia (CML), discontinuation of tyrosine kinase inhibitor (TKI) therapy can lead to treatment-free remission. In previous trials, TKI discontinuation has been associated with increased musculoskeletal pain in some patients ("withdrawal syndrome"), based on physician-reported adverse events (AE). Patient-reported pain has not been described. The Life After Stopping TKI study was a 14-site prospective, non-randomized clinical trial of TKI discontinuation. We defined increased pain after discontinuation as: (i) a physician-reported pain AE, (ii) a 2-level increase in self-reported musculoskeletal pain (4-level single item), or (iii) initiation of a medication for pain. We plotted the trajectory of patient-reported pain over time using a piecewise mixed-effects ordinal logistic model. Within 3 months of discontinuation, 35 of 172 patients (20.3%) had a physician-reported pain AE, 22 of 172 (12.8%) had an increase in self-reported pain, and 18 of 154 (11.7%) initiated a pain medication. Agreement among these measures was limited; overall, 60 of 172 patients (34.9%) had increased pain. Three patients (1.7%) restarted a TKI because of pain. The modelpredicted trajectory showed an increase in pain in the first 3 months followed by a decrease, returning to baseline levels by 6 months and further decreasing after that. This trajectory was similar among patients who did and did not restart TKI, suggesting that resuming a TKI for withdrawal syndrome may be necessary for some, but other approaches to manage pain should be tried so that patients can remain in treatment-free remission when possible.

Objectives: To identify pediatric patient-reported outcomes (PROs) that are associated with chronic conditions and to evaluate the effects of chronic disease activity on PROs., Study Design: Participants (8-24 years old) and their parents were enrolled into 14 studies that evaluated Patient-Reported Outcome Measurement Information System PROs across 10 chronic conditions-asthma, atopic dermatitis, cancer, cancer survivors, chronic kidney disease, Crohn's disease, juvenile idiopathic arthritis, lupus, sickle cell disease, and type 1 diabetes mellitus. PRO scores were contrasted with the US general population of children using nationally representative percentiles. PRO-specific coefficients of variation were computed to illustrate the degree of variation in scores within vs between conditions. Condition-specific measures of disease severity and Cohen d effect sizes were used to examine PRO scores by disease activity., Results: Participants included 2975 child respondents and 2392 parent respondents who provided data for 3409 unique children: 52% were 5-12 years old, 52% female, 25% African American/Black, and 14% Hispanic. Across all 10 chronic conditions, children reported more anxiety, fatigue, pain, and mobility restrictions than the general pediatric population. Variation in PRO scores within chronic disease cohorts was equivalent to variation within the general population, exceeding between-cohort variation by factors of 1.9 (mobility) to 5.7 (anxiety). Disease activity was consistently associated with poorer self-reported health, and these effects were weakest for peer relationships., Conclusions: Chronic conditions are associated with symptoms and functional status in children and adolescents across 10 different disorders. These findings highlight the need to complement conventional clinical evaluations with those obtained directly from patients themselves using PROs., (Copyright © 2022 Elsevier Inc. All rights reserved.)

Purpose: The impact of nonurological factors on male lower urinary tract symptoms (LUTS) remains unclear. We investigated cross-sectional and longitudinal associations among anxiety, depression, physical function, sleep quality and urinary symptom subdomains., Materials and Methods: Data from 518 men in the LURN (Symptoms of Lower Urinary Tract Dysfunction Research Network) study were analyzed to identify associations between Patient-Reported Outcomes Measurement Information System® (PROMIS®) depression, anxiety, sleep disturbance and physical function measures and LUTS subdomains, as derived from the American Urological Association Symptom Index and LUTS Tool. Multivariable linear regression was used to assess the relationships between PROMIS measures and LUTS subdomains at baseline and at 3- and 12-month followup., Results: Baseline depression and anxiety were associated with urinary incontinence (p <0.001), voiding symptoms (p <0.001) and quality of life (p=0.002), whereas baseline sleep disturbance was associated with voiding and storage symptoms and quality of life (p <0.001 for all). Urinary symptom severity improved in all subdomains at 3 and 12 months. Similar associations between PROMIS measures and LUTS subdomains were observed at all time points, but baseline depression, anxiety, sleep disturbance and physical function measures were not associated with longitudinal trajectories of LUTS., Conclusions: Urinary symptom subdomains are independently associated with modifiable clinical variables including sleep quality and depression at all time points, but these variables do not predict the degree of improvement in LUTS following urological evaluation and treatment over the medium term. Bidirectional assessment and randomized experiments may improve our understanding of these relationships.

Background: Adjuvant endocrine therapy (AET) for breast cancer reduces mortality, but one-third to one-half of patients discontinue it early or are nonadherent., Objective: We developed a pilot single-site study of patients with evidence of early nonadherence to AET to assess the feasibility of a novel, clinical pharmacist-led intervention targeting symptom and medication management., Methods: Patients with prescription fill records showing nonadherence were enrolled in a single-arm feasibility study. Automated reminders were sent by e-mail or text with a link to symptom monitoring assessments weekly for 1 month and monthly until 6 months. Clinical oncology pharmacists used guideline-based symptom management and other medication management tools to support adherence and ameliorate symptoms reported on the assessments. Patient-reported outcome assessments included physical, mental, and social health domains and self-efficacy to manage symptoms and medications. Feasibility outcomes included completion of symptom reports and pharmacist recommendations., Results: Of 19 participants who were nonadherent who enrolled and completed initial assessments, 18 completed all final study procedures, with 14 completing all assessments and no patient missing more than 3 assessments. All 18 participants reported at least one of 3 symptom types, and the majority reported attempting pharmacist recommendations. Patient-reported measures of physical, mental, and social health and self-efficacy improved, and 44% of the patients became adherent., Conclusion: An intervention using pharmacists in an oncology practice to systematically monitor and manage symptoms shows promise to reduce symptoms, enhance support and self-efficacy, and improve adherence to AET., (Copyright © 2022 American Pharmacists Association®. Published by Elsevier Inc. All rights reserved.)

Background: Sexual dysfunction is a common symptom of multiple sclerosis (MS). Clinically meaningful and psychometrically sound measures of sexual function validated in people with MS are necessary to identify people with MS who experience problems with sexual function., Aim: To evaluate the Patient-Reported Outcomes Measurement Information System (PROMIS) Sexual Function and Satisfaction (SexFS) v2.0 measure in people with MS and to extend the PROMIS SexFS Brief and Full Profiles to include additional aspects of sexual function relevant to people living with MS., Methods: A convenience sample of MS clinicians and sexually active individuals with MS ranked relevance of 26 items that listed specific factors that interfere with sexual function. Rankings were used to select items to include in the modified SexFS for Multiple Sclerosis (SexFS-MS) profiles. Sex-FS Brief and Full profiles along with the top 22 ranked interfering factor items underwent cognitive interviews (CI) to assess whether the items were understandable and meaningful., Outcomes: The SexFS as originally published functioned well in people with MS after minor modifications., Results: Twelve MS clinicians and 26 people with MS ranked items. The 10 highest ranked questions about factors that interfere with sexual function most relevant to people with MS were added to the SexFS-MS Brief profiles and 18 to the Full profiles. Ten men and 12 women with MS participated in CIs and found most items to be clear and meaningful. However, important changes were made to the profile instructions, some response sets, and to some items to improve clarity and function. New items to assess numbness and reasons why sexually active people choose at times not to engage in sexual activity were added., Clinical Implications: Brief and Full profiles are freely available and are recommended for research and clinical practice that include people with MS., Strengths & Limitations: This study is the first to provide validity evidence for the PROMIS SexFS in people living with MS. Though the PROMIS SexFS was tested in people who identify as lesbian, gay, or bisexual, only individuals who identified as heterosexual participated in this study. Results may not represent views of people with MS who identify as other sexual orientations who may have different concerns and priorities related to sexual function., Conclusion: This study extended the PROMIS SexFS Brief and Full profiles to create the SexFS-MS by adding items that measure most relevant issues related to sexual function in individuals living with MS. Amtmann D, Bamer AM, Salem R, et al. Extension and Evaluation of the PROMIS Sexual Function and Satisfaction Measures for Use in Adults Living With Multiple Sclerosis. J Sex Med 2022;19:719-728., (Copyright © 2022 International Society for Sexual Medicine. Published by Elsevier Inc. All rights reserved.)

Lead is an environmental hazard; even small elevations in blood lead level can cause serious negative health effects in children, including irreversible impacts such as learning difficulties, attention disorders, and behavioral issues. Previous research has shown that some groups are at higher risk for lead poisoning including racial/ethnic minorities, those with low economic status, and immigrants, especially refugees. This systematic review explores recent literature studying disparities in lead poisoning in refugee children. Search terms were chosen with the assistance of a medical librarian, and two independent reviewers assessed articles using a PICOS criteria (Population, Intervention, Comparison, Outcome, Study Design) following the Preferred Reporting Items for Systematic review and Meta-Analysis Protocols (PRIMSA-P) guidelines, a set of evidence-based minimum standards for reporting in systematic reviews. 321 article titles were reviewed, 61 abstracts screened, and 17 methods sections reviewed, ultimately including 13 studies. The studies found a high prevalence of elevated blood lead level among refugee populations when compared to reference populations. Both pre-migration and post-migration factors were identified as contributors to the disparity, and associations were identified between elevated blood lead levels and factors such as country of origin, age, and other health variables. Health providers and resettlement workers should be aware of these disparities and related factors. Testing, care, education and consistent follow-up should be provided., (© 2022 Walter de Gruyter GmbH, Berlin/Boston.)

Background: Light chain (AL) amyloidosis is a plasma cell neoplasm associated with high early mortality and severe morbidity that can cause severe disability. We explored the impact of AL amyloidosis on symptoms and well-being from the perspectives of patients and health care providers who regularly care for AL patients. We intended to develop a conceptual understanding of patient-reported outcomes in AL amyloidosis to identify the context of use and concept of interest for a clinical outcome assessments tool in this disease., Method: Twenty patients and ten professionals were interviewed. Patient interviews captured the spectrum of amyloidosis experience including time from diagnosis, type of organ involvement, and presence and type of treatment received. Interviews with professionals included physicians, advanced practice providers, registered nurse, and a patient advocate; these interviews covered similar topics., Results: The impact of AL amyloidosis on patients' life was multidimensional, with highly subjective perceptions of normality and meaning. Four major themes from patients and experts included diagnosis of AL amyloidosis, living with AL amyloidosis, symptom burden, and social roles. Barriers to patient-reported outcomes data collection in patients were additionally explored from experts. The themes provide a comprehensive understanding of the important experiences of symptom burden and its impact on daily life from AL amyloidosis patients' and from the perspectives of professionals who care for patients with AL amyloidosis., Conclusion: These findings further the conceptual understanding and identification of a preliminary model of concept of interest for development of a clinical outcome assessments tool for AL amyloidosis., (© 2021. The Author(s), under exclusive licence to Springer Nature Switzerland AG.)

The importance of patient-reported outcomes (PROs) in cellular therapies, including hematopoietic cell transplantation (HCT) is highlighted in this study. Longitudinal collection of PROs in a registry is recommended for several reasons, yet to date, PROs are not routinely collected from HCT patients to augment clinical registry data. The aim of this study was to determine the feasibility of electronic PRO data collection by a national clinical outcomes registry, by assessing differences between who does and does not report PROs. We conducted a cross-sectional pilot collection of PROs from HCT recipients after treatment using computer-adapted tests from the Patient-Reported Outcome Measurement Information System (PROMIS). We implemented centralized data collection through the Center for International Blood and Marrow Transplant Research (CIBMTR) among patients who underwent HCT for myelodysplastic syndromes (MDS), were at least 6 months post-HCT, and spoke English or Spanish. The main objective was identifying patient, disease, and transplant-related differences associated with completion of electronic PROs. Patients were excluded from analysis if they were determined to be ineligible (deceased, did not speak English or Spanish, refused to be contacted by the CIBMTR). A total of 163 patients were contacted and potentially eligible to participate; of these, 92 (56%) enrolled and 89 (55%) completed the PRO assessment. The most frequent reason for incomplete surveys was inability to contact patients (n = 88), followed by declining to participate in the study (n = 37). There were no sociodemographic or age differences between those who completed the PRO survey (n = 89) and eligible nonresponders (n = 155). Patient scores were within 3 points of the US average of 50 for all symptoms and functioning except physical functioning. Responders and nonresponders did not exhibit meaningfully different sociodemographic characteristics. Difficulty contacting patients posed the greatest barrier and also provided the greatest opportunity for improvement. Once enrolled, survey completion was high. These results support standardizing centralized PRO data collection through the CIBMTR registry., (Copyright © 2021 The American Society for Transplantation and Cellular Therapy. Published by Elsevier Inc. All rights reserved.)

Treatment-free remission (TFR) is a goal for patients with chronic myeloid leukemia (CML). Functional outcomes after discontinuing tyrosine kinase inhibitor (TKI) treatment have not been described. Patient-Reported Outcomes Measurement Information System (PROMIS) measures of social, physical, cognitive, and sexual function were assessed over 36 months in 172 adult patients with chronic phase CML from 14 sites at baseline (on TKI) and after discontinuation. Linear mixed-effects models described the average trajectories for each patient-reported outcome measure after discontinuation and in those who restarted TKI. Of 112 patients in TFR at 12 months, 103 (92.0%) had a 3-point or greater improvement in social function, 80 (71.4%) in social isolation, 11 (9.8%) in satisfaction with sex life, 4 (3.6%) in physical function, and no patients had a 3-point or greater improvement in cognitive function or interest in sexual activity. Patients' scores worsened after restarting TKI. This novel information on functional outcomes in TFR can help guide patient and clinician decision making., (© The Author(s) 2021. Published by Oxford University Press. All rights reserved. For permissions, please email: journals.permissions@oup.com.)

Background: Cognitive interviewing is a technique that can be used to improve and refine questionnaire items. We describe the basic methodology of cognitive interviewing and illustrate its utility through our experience using cognitive interviews to refine a questionnaire assessing parental understanding of concepts related to preterm birth., Methods: Cognitive interviews were conducted using current best practices. Results were analyzed by the multidisciplinary research team and questionnaire items that were revealed to be problematic were revised., Results: Revisions to the questionnaire items were made to improve clarity and to elicit responses that truly reflected the participants understanding of the concept., Conclusion: Cognitive interviewing is a useful methodology for improving validity of questionnaire items, we recommend researchers developing new questionnaire items design and complete cognitive interviews to improve their items and increase confidence in study conclusions.

Purpose of Review: Tyrosine kinase inhibitors (TKIs) allow many patients with chronic myeloid leukemia (CML) to live normal life spans but have the potential to impact patients' health-related quality of life (HRQOL). Patient-reported outcome (PRO) measures can provide valuable information to inform treatment decision-making. Here, we review pivotal studies that used PRO measures to evaluate HRQOL of patients with CML in the first-line and treatment-free remission (TFR), and identify areas for future research., Recent Findings: PRO measures commonly studied in patients with CML include the SF-36, FACT-Leu, EORTC QLQ-CML24, and MDASI CML. Cohort or cross-sectional studies provide the most data on PRO measures in patients with CML, with less information available from randomized controlled trials (RCTs). Patients with CML taking TKIs have worse HRQOL compared to matched controls, with a few studies seeing a larger effect in younger patients (< 60 years old). No single TKI consistently has better HRQOL compared to other agents. Fatigue is a predominant symptom associated with impaired HRQOL across many studies. Studies evaluating TFR show stable or improved HRQOL after TKI discontinuation. There are areas of HRQOL detrimental to patients with other types of cancer (e.g., cognition, sexuality) that warrant further evaluation in patients with CML. Understanding the HRQOL of patients with CML is increasingly important as patients live near-normal life expectancies. PRO measures have the potential to inform treatment decisions in this patient population. Future research opportunities include using PRO measures in RCTs and expanding the HRQOL topics studied in patients with CML., (© 2021. The Author(s), under exclusive licence to Springer Science+Business Media, LLC, part of Springer Nature.)