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.

Symptom clusters and their impact on quality of life in multiple myeloma survivors: Secondary analysis of BMT CTN 0702 trial.

Autologous haematopoietic cell transplantation (autoHCT) and continuous post-transplant maintenance therapy are the standard of care in transplant-eligible multiple myeloma (MM) patients. We sought to describe symptom burden and identify symptom clusters occurring in MM patients after autoHCT using data from the BMT CTN 0702 randomized controlled trial comparing the outcomes of three treatment interventions after an autoHCT in 758 MM patients. We analysed individual transplant-related symptoms assessed via the FACT-BMT questionnaire at enrolment and annually for 4-year post-autoHCT. We also described the effect the individual symptoms and symptom clusters have on quality of life (QoL). We identified three stable symptom clusters: malaise symptom cluster (lack of energy, feeling ill, having pain, experiencing nausea, loss of appetite), physical symptom cluster (having skin problems, tremors, worsening eyesight, change in taste, shortness of breath, frequent colds) and emotional symptom cluster (feeling sad, being nervous, experiencing sleep problems). Malaise and emotional symptom clusters have a greater impact on QoL than the physical symptoms cluster. Identifying these symptoms warrant additional support in terms of psychosocial support, in addition to treatment of the physical symptoms themselves.

A Patient-Prioritized Research Agenda for Clinical Trials in Kidney Stone Disease.

PURPOSE: To ensure that research on kidney stones provides meaningful impact for the kidney stone community, patients and caregivers should be engaged as stakeholders in clinical trial design, starting at study inception. This project aimed to elicit, refine, and prioritize research ideas from kidney stone stakeholders to develop a patient-centered research agenda for clinical trials. MATERIALS AND METHODS: The Kidney Stone Engagement Core, a group of patients, caregivers, advocates, clinicians, and researchers, executed an iterative process of surveys and focus groups to elicit and refine research themes, which were then translated into research questions. A separate group of patients, caregivers, and clinicians prioritized these questions through parallel modified Delphi and crowd-sourced digital platforms. A research agenda was developed by the Kidney Stone Engagement Core based on the highest rated questions during a hybrid virtual/in-person capstone session. RESULTS: A total of 70 individuals (57 patients and caregivers, 13 researchers and clinicians) participated in the elicitation, 20 individuals (15 patients and caregivers, 5 researchers and clinicians) participated in refinement, and an additional 80 individuals (81 patients and caregivers, 9 researchers and clinicians) participated in prioritization. Key novel themes emerged from elicitation and refinement: ureteral stents, genetic evaluation, shared surgical decision-making, key subgroups, cumulative disease burden, genetic evaluation, and psychosocial support. Stakeholders generated 6 proposed trials from these themes focused on surveillance, surgical intervention, and medical prevention. CONCLUSIONS: Patients and caregivers valued comparative effectiveness kidney stone research that focused on individualized care, shared decision-making, and improvement of patient-reported experiences. This process provided actionable recommendations for future patient-centered clinical trials within kidney stone disease.

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.

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.

Patient Perspectives in the Diagnosis and Treatment of Gallstone Disease: A Qualitative Analysis.

INTRODUCTION: Gallstone disease is one of the most common surgical diagnoses in the United States. Notably absent from the literature is the patient's perspective on priorities in management. Understanding patient values will assist surgeons and systems in achieving high-quality, patient-focused care for biliary disease. METHODS: Patients who underwent elective or urgent cholecystectomy were invited to participate in a semistructured interview to assess their experience. Interviews were performed over the phone or in person and recordings were transcribed. Each transcription was analyzed independently by two authors using the MAXQDA software, and a mixed deductive-inductive approach was used to develop themes. Anonymized quotes were used to illustrate themes and subthemes regarding the patient's experiences and priorities surrounding gallstone disease. RESULTS: A total of 29 interviews were completed. Most participants were female, but represented a diverse racial and educational group. The most common diagnosis was acute cholecystitis (48%), and 76% of patients underwent an emergency operation. Patients indicated that their main priority regarding treatment was prompt pain control with definitive management so they could return to their previous quality of life. Most patients wanted face-to-face communication with the surgical team both pre and postoperatively. Patients wished they had more information about postoperative care and expectations in the preoperative setting. CONCLUSIONS: Patients' priorities in their care for gallstone disease are centered around definitive management and quality of life. Improvements in communication were identified regarding meeting the surgeon, and postoperative communication. These results can inform surgeons how to prioritize patient perspectives in an acute care surgical system that was not designed with patient input.

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.

Developing a urinary incontinence primary care pathway: a mixed methods study.

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.

Although the majority of women will experience urine leakage at some point during their lives, most will never receive treatment. To better understand this discrepancy, we embarked on a multimodal investigation into the barriers to care and trialed a new treatment pathway in the primary care setting within a large academic medical system in the Midwest. Speaking with the clinic managers from 21 primary care clinics, we determined that many clinics lacked the tools to perform the steps outlined in the professional society guidelines for urinary incontinence diagnosis. Additionally, there was limited access to pelvic floor physical therapy, a proven treatment strategy. Interviews with five primary care providers revealed barriers, most notably lack of time during clinic visits, to almost every step of diagnosis and treatment. Finally, we trialed a care pathway for primary care providers to make it easier to provide patients with self-management education or to refer them to specialist care. Fifteen patients participated in a pilot study, about half reported trying self-management, and about 1/3 requested follow-up care. Streamlining urinary incontinence care at the primary care level may alleviate some of the barriers to patients receiving care.

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.

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.

Health-related quality of life in reduced-intensity hematopoietic cell transplantation based on donor availability in patients aged 50-75 with advanced myelodysplastic syndrome: BMT CTN 1102.

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.

Trajectories of quality of life recovery and symptom burden after autologous hematopoietic cell transplantation in multiple myeloma.

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.

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.

Patient-reported cognitive function among hematopoietic stem cell transplant and cellular therapy patients: a scoping review.

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.

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.

Impact of Sleep Disturbance, Physical Function, Depression and Anxiety on Male Lower Urinary Tract Symptoms: Results from the Symptoms of Lower Urinary Tract Dysfunction Research Network (LURN).

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.

Self-Reported Health Outcomes of Children and Youth with 10 Chronic Diseases.

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.

Patient- and physician-reported pain after tyrosine kinase inhibitor discontinuation among patients with chronic myeloid leukemia.

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.

Extension and Evaluation of the PROMIS Sexual Function and Satisfaction Measures for Use in Adults Living With Multiple Sclerosis.

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.

Comparing clinical bladder diaries and recalled patient reports for measuring lower urinary tract symptoms in the symptoms of Lower Urinary Tract Dysfunction Research Network (LURN).

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.