Urinary Incontinence Medications: Patient-Initiated Concerns in Primary Care.

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.

Current trends and outcomes in cellular therapy activity in the United States, including prospective Patient Reported Outcomes data collection within the CIBMTR registry.

BACKGROUND: Center for International Blood and Marrow Transplant Research (CIBMTR) prepares an annual set of summary slides to detail the trends in transplantation and cellular therapies. For the first time in the 2023 summary slides, CIBMTR incorporates data for patients receiving chimeric antigen receptor T-cell (CAR-T) infusions. In addition, the data on patient-reported outcomes (PROs) is also included. OBJECTIVES: This report aims to update the annual trends in US HCT activity and incorporate data on the use of CAR-T therapies. Here we also aim to present and describe the development, implementation, and current status of the PRO data collection. STUDY DESIGN: In August 2020, 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 burden to centers. Specifically, PRO data is collected from a prospective convenience sample of adult HCT and CAR-T patients 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 others 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 timepoints that clinical data is routinely collected. RESULTS: As of September 2023, PRO data from 993 patients across 25 different centers has been collected. With the goal of incorporating these important patient perspectives into standard clinical care, CIBMTR has added the PRO data to Data Back to Centers (DBtC). Through expanding the data types represented in the registry, CIBMTR aims to support holistic research accounting for the patient perspective in improving patient outcomes. CONCLUSION: PRO data at CIBMTR aims to provide the foundation for future large scale, population-level evaluations to determine 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 patient quality of life.

Use of central venous access devices outside of the pediatric intensive care units.

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.

Adherence to adjuvant endocrine therapy for breast cancer: a qualitative exploration of attribution of symptoms among post-menopausal women.

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.

Development and initial validation of the AL-PROfile patient-reported outcome measure in light chain (AL) amyloidosis.

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.

Effectiveness of educational interventions for the prevention of lead poisoning in children: a systematic review.

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.

Incorporating patient-reported outcome data into a predictive calculator for allogeneic hematopoietic cell transplantation recipients.

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.

Estimated Savings After Stopping Tyrosine Kinase Inhibitor Treatment Among Patients With Chronic Myeloid Leukemia.

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.

Video Education in Early Pregnancy and Parent Knowledge of Neonatal Resuscitation Options: A Secondary Analysis of a Randomized Clinical Trial.

This secondary analysis of a randomized clinical trial investigates the proportion of correct answers on neonatal resuscitation options among parents after seeing a video on these options.

Decision regret among couples experiencing infertility: a mixed methods longitudinal cohort study.

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.

When people experience infertility, there are many decisions that can be challenging, such as whether to seek fertility treatments, to pursue fostering/adoption, and how to manage costs. With each decision, there is an opportunity for regret. The goal of this study was to look at whether people who were experiencing infertility and made an appointment with a doctor who specializes in infertility felt any regret about their decisions 6 years later. We also looked at whether different roles (that is, women seeking pregnancy or their supporting partners), different family-building paths (that is, medical treatments or not), or different outcomes (that is, adding a child to their family or not) were associated with different levels of regret. Results showed that half of the 120 people in the study did not have any regret 6 years after meeting with a specialty doctor. However, some patients did have regret, including 20% of women and 14% of partners who expressed moderate-to-severe regret. Women who did not add a child to their family in the six years during the study reported higher regret compared to women who did add a child to their family. There were no such differences among partners. About 25% of participants wished they had tried more, fewer, or different treatments. More than 25% wished they spent less money to try to add a child to their family. For people who want to add a child to their family, there are multiple ways to become a parent, any of which may be linked to lower decision regret. Decision regret is experienced differently between women seeking to add a child to their family and their partners. Would-be parents need more emotional, financial, and decision making support to help them navigate family-building with minimal regret.

Referral and Prescription Patterns for Female Patients With Urinary Incontinence.

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.

Smartphone-Based Video Antenatal Preterm Birth Education: The Preemie Prep for Parents Randomized Clinical Trial.

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.

Differences in patient-reported outcomes (PROs) by disease severity in light chain (AL) amyloidosis.

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.

GAP (gestational diabetes and pharmacotherapy) - study protocol for a randomized controlled, two-arm, single-site trial.

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.

Psychometric evaluation of the Swedish version of the PROMIS Sexual Function and Satisfaction Measures in clinical and nonclinical young adult populations.

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.

Breast sensorisexual function: a novel patient-reported outcome measure of sexual sensory functions of the breast.

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.

A cross-sectional study of patient-reported outcomes and symptom burden using PROMIS and PRO-CTCAE measures in light chain amyloidosis.

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.

A Survey of Patient Experience in CML: American and Canadian Perspectives.

Purpose: 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.

Comparison of CAT and short forms for PROMIS pain and physical health domains in children with sickle cell disease.

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.