An observational, patient-reported outcome study of sleep quality and depression among individuals with overactive bladder syndrome.

INTRODUCTION: Overactive bladder (OAB) can adversely affect health-related quality-of-life (HRQoL) and adherence to treatments; however, the extent of their association is unknown. This study sought to characterize Sleep Disturbance, Depression, Fatigue, and patient-reported medication adherence among adults with OAB in the United States. MATERIALS AND METHODS: In this descriptive, observational study, patients completed patient-reported outcome (PRO) measures of urinary symptoms, anxiety, depression, fatigue, sleep quality, and medication adherence. PRO scores were compared across age, sex, body mass index, and sleep and antidepressant medication-taking subgroups. Exploratory analyses compared PRO scores between groups and estimated the effect size of differences. RESULTS: Of 1013 patients contacted, 159 completed the assessments (female: 67.3%; ≥65 years of age: 53.5%; most severe OAB symptom: nocturia). Scale scores for Sleep Disturbance, Fatigue, and Depression were consistent with US population norms. No correlations of moderate or greater magnitude were observed between the severity of lower urinary tract symptoms and Sleep Disturbance, Fatigue, or Depression. When comparing individuals receiving antidepressants with those who were not, almost all outcomes including urinary symptoms, anxiety, and depression were significantly worse. Patients taking antidepressants also had poorer adherence to their OAB medications. CONCLUSION: In this cohort of individuals with OAB, Sleep Disturbance, Fatigue, and Depression scores were in line with general population reference values; however, among the subgroups analyzed, patients on antidepressants had worse HRQoL and more substantial impacts on medication adherence, highlighting the importance of the assessment and management of depression in this population.

Enhanced clinical decisions for management of benign prostatic hyperplasia using patient-reported outcomes: protocol for a prospective observational study.

BACKGROUND: Lower urinary tract symptoms (LUTS) due to benign prostatic hyperplasia (BPH) significantly impact quality of life among older men. Despite the prevalent use of the American Urological Association Symptom Index (AUA-SI) for BPH, this measure overlooks key symptoms such as pain and incontinence, underscoring the need for more comprehensive patient-reported outcome (PRO) tools. This study aims to integrate enhanced PROs into routine clinical practice to better capture the spectrum of LUTS, thereby improving clinical outcomes and patient care. METHODS: This prospective observational study will recruit men with LUTS secondary to BPH aged ≥ 50 years from urology clinics. Participants will be stratified into medical and surgical management groups, with PRO assessments scheduled at regular intervals to monitor LUTS and other health outcomes. The study will employ the LURN Symptom Index (SI)-29 alongside the traditional AUA-SI and other non-urologic PROs to evaluate a broad range of symptoms. Data on comorbidities, symptom severity, and treatment efficacy will be collected through a combination of electronic health records and PROs. Analyses will focus on the predictive power of these tools in relation to symptom trajectories and treatment responses. Aims are to: (1) integrate routine clinical tests with PRO assessment to enhance screening, diagnosis, and management of patients with BPH; (2) examine psychometric properties of the LURN SIs, including test-retest reliability and establishment of clinically meaningful differences; and (3) create care-coordination recommendations to facilitate management of persistent symptoms and common comorbidities measured by PROs. DISCUSSION: By employing comprehensive PRO measures, this study expects to refine symptom assessment and enhance treatment monitoring, potentially leading to improved personalized care strategies. The integration of these tools into clinical settings could revolutionize the management of LUTS/BPH by providing more nuanced insights into patient experiences and outcomes. The findings could have significant implications for clinical practices, potentially leading to updates in clinical guidelines and better health management strategies for men with LUTS/BPH. TRIAL REGISTRATION: This study is registered in ClinicalTrials.gov (NCT05898932).

Measuring PROMIS® Physical Activity and Sleep Problems in Early Childhood.

OBJECTIVE: Physical activity (PA) and sleep are leading health indicators for individuals of all ages. Monitoring young children's PA and sleep using psychometrically sound instruments could help facilitate timely interventions to promote healthy development. This article describes the development of the PROMIS® Early Childhood (EC) Parent Report Physical Activity (PA) and Sleep Problems (SP) measures for children aged 1-5 years. METHODS: Item pools were generated by interviewing parents, input from content experts, and literature review. Data from a U.S. general population sample were used to determine factor structures of item pools via factor analytic approaches, estimate item parameters via item response theory (IRT) models, and establish norms. Pearson correlations were used to evaluate across-domain associations. Analysis of variance was used and known-groups' validity of PA and SP by comparing their scores to PROMIS EC Parent Report Global Health: child's physical, emotional, and mental conditions. RESULTS: Initial item pools consisted of 19 and 26 items for PA and SP, respectively. Factor analyses' results supported unidimensionality of 5 and 16 items measuring PA and SP, respectively, which were then calibrated using IRT. Norms were established by centering to a probability-based U.S. general population. Computerized adaptive testing algorithms were established. Some analyses supported initial measure validity. CONCLUSIONS: The PROMIS EC PA calibrated scale and SP item banks are user-friendly and brief, yet produce precise scores. Both measures enable psychometrically sound assessment of PA behavior and sleep problems. Future studies to comprehensively evaluate the validity of these two measures are warranted.

Patient Reported Outcomes Measurement Information System and Quality of Life in Neurological Disorders Measurement System to Evaluate Quality of Life for Children and Adolescents with Neurofibromatosis Type 1 Associated Plexiform Neurofibroma.

OBJECTIVE: To assess the health-related quality of life of children with neurofibromatosis type 1-related plexiform neurofibromas (pNF) using a battery of patient-reported outcome measures selected based on a conceptual framework derived from input by patients, parents, and clinicians regarding the most important pNF symptoms and concerns. STUDY DESIGN: There were 140 children with pNF ages 8-17 years who completed the Patient-Reported Outcomes Measurement Information System (including domains anxiety, depressive symptom, psychosocial stress experiences, fatigue, pain interference, meaning and purpose, positive affect, peer relationships, physical function-mobility) and Quality of Life in Neurological Disorders measurement system (stigma) via an online platform. T-scores for each measure were compared with US population norms. RESULTS: Children with pNF reported significantly worse scores than the population norms on 8 of 10 domains. Children with at least 1 family member having a diagnosis of neurofibromatosis type 1 and those having pain reported significantly worse symptoms and functioning on all domains. Boys reported significantly worse pain interference, stigma, meaning and purpose, mobility function, and upper extremity function than girls. CONCLUSIONS: Children with pNF experience significantly worse health-related quality of life on all but 1 domain, highlighting the importance of monitoring children's quality of life over time in clinical research and practice. Future research should evaluate the replicability of these findings and evaluate the validity of the Patient-Reported Outcomes Measurement Information System and Quality of Life in Neurological Disorders measurement system in relation to clinical characteristics among children with pNF.

Further content validation of the 18-item NCCN/FACT Ovarian Symptom Index and its Disease Related Symptom-Physical (DRS-P) subscale for use in advanced ovarian cancer clinical trials.

BACKGROUND: This study evaluated pre-defined aspects of content validity of the 18-item NCCN FACT-Ovarian Symptom Index (NFOSI-18) and its Disease-Related Symptoms-Physical (DRS-P) subscale, as clinical trial outcome tools for patients with advanced ovarian cancer. METHODS: Twenty-one women (mean age 59.5 years) diagnosed with advanced ovarian cancer completed the NFOSI-18 and participated in a cognitive interview to explore: (1) whether 'pain' and 'cramps' are considered redundant; (2) whether 'fatigue' and 'lack of energy' are overlapping concepts; (3) whether patients consider severity when responding to the item "I am bothered by constipation;" and (4) factors considered when responding to the item "I am sleeping well." Interviews were audio-recorded, transcribed, and analyzed qualitatively. RESULTS: Pain was associated with discomfort, hurt, and life interference; 'cramps' was associated with pain, muscle tightening, and menstrual or digestive issues. Most (81%) considered the items "I have pain" and "I have cramps in my stomach area" to be more different than similar. Participants associated 'fatigue' with intense tiredness and 'lack of energy' with motivation and capability to complete daily activities. Item comparisons revealed a majority (65%) considered the items to be more different than similar. When responding to "I am bothered by constipation," patients indicated constipation severity was related to bother. Finally, patients considered disease, treatment, and other factors when responding to "I am sleeping well." CONCLUSIONS: Findings support content validity of the NFOSI-18 and its DRS-P as originally constructed. We propose an alternative scoring option that excludes the item "I am sleeping well" from the DRS-P when used as a symptom-focused index for clinical research in a regulatory context.

Lifespan Development: Symptoms Experienced by Individuals with Neurofibromatosis Type 1 Associated Plexiform Neurofibromas from Childhood into Adulthood.

This secondary data analysis qualitatively identified salient concerns reported by individuals with Neurofibromatosis Type 1 (NF1)-associated plexiform neurofibromas (pNFs) at different stages of development. Past literature has focused on overall symptomatology, but has not examined nuances in how these symptoms are experienced across developmental phases. Therefore, we aimed to identify commonalities and differences in symptom experiences across age groups to better assist individuals to adjust to symptoms across the lifespan. Thirty-one children, adolescents, and adults (age ≥ 5 years old) and 15 parents participated in semi-structured interviews. Analyses focused on the following symptom categories: pain, social functioning, physical function impact, and stigma. Aspects of pain endorsed by all age groups included localized brief pain on contact with pNF and abnormal sensations; however, only adolescents and adults reported chronic pain and change in pain over time. Social functioning themes of limited activity participation, role limitations, and relationship impact were endorsed by all age groups, but differences emerged across age groups in the types of activity and role limitations, the type of relationship impact, and family planning concerns. All age groups described difficulty with mobility, but only parents reported problems with coordination and physical developmental milestones. While all age groups reported external stigma, internalized stigma was predominately endorsed by adults. While individuals in all age groups described pNF concerns related to pain, social function, physical function, and stigma, specific aspects of these symptoms differed across the developmental continuum. These findings can help assist individuals with pNF better transition to the next developmental phases.

Using a qualitative approach to conceptualize concerns of patients with neurofibromatosis type 1 associated plexiform neurofibromas (pNF) across the lifespan.

Neurofibromatosis Type 1 (NF1) plexiform neurofibromas (pNFs) are associated with a variety of symptoms and concerns that affect patients' quality of life (QOL), highlighting the value of incorporating the patients' perspective when evaluating treatment outcomes. To better conceptualize the experience of patients with pNFs, this qualitative study sought to identify the most important outcomes to assess from the perspective of patients, families, and clinicians. Clinicians, patients age 5 years old and above, and parents of patients aged 5-17 years participated in semi-structured interviews to elicit the pNF symptoms/concerns considered most important to assess. The data were analyzed using an iterative coding procedure and the frequency with which symptoms/concerns emerged was tabulated. Eight clinicians, 31 patients, and 17 parents of patients participated in semi-structured interviews. The most frequently reported concerns raised by patients across all age groups included pain, appearance/disfigurement, social activity/role participation, stigma, and anxiety. For parents, physical functioning was the primary concern, followed by pain, social activity/role participation, appearance/disfigurement, and social relationships. The resulting conceptual framework included five domains to represent the most important identified symptoms/concerns: pain, social functioning, physical function impact, stigma, and emotional distress. This conceptual framework describing the symptoms/concerns of patients with pNF can help investigators create a measurement system to improve assessment of aspects of QOL only patients can report on. It may also provide the ability to identify symptoms/concerns that might warrant referrals to various clinical disciplines. © 2016 Wiley Periodicals, Inc.

Considerations for conducting qualitative research with pediatric patients for the purpose of PRO development.

PURPOSE: To provide an overview of methodological considerations when conducting qualitative research with pediatric patients for the purpose of patient-reported outcome measure development METHODS: A literature review of qualitative methods in pediatric measure development was completed. Eight clinicians providing care to pediatric patients were interviewed for their expert input. Thematic analysis of the literature and clinician interviews was used to identify themes for consideration. RESULTS: Findings from the literature and expert interviews emphasized the way in which cognitive, linguistic, and social developmental factors affect pediatric patients' understanding of their condition and ability to communicate about their experiences in an interview. There was consensus among the experts that traditional semi-structured interviews with children younger than eight lack characteristics necessary to yield meaningful information about condition and symptom report because they may fail to capture children's understanding and awareness of their condition and may limit their ability to express themselves comfortably. Our findings include recommended strategies to optimize data collected in qualitative interviews with pediatric patients, including modifications to the interview process to establish rapport, construction of interview questions to ensure they are developmentally appropriate, and the use of supplementary techniques to facilitate communication. CONCLUSION: When employing qualitative methods in pediatric measure development, interview guides, methods, and length require careful tailoring to ensure the child's perspectives are captured. This may be best achieved through research performed with narrow age bands that employs flexibility in methods to allow children a comfortable way in which to communicate about their experiences.

Elevated plasma albumin and apolipoprotein A-I oxidation under suboptimal specimen storage conditions.

S-cysteinylated albumin and methionine-oxidized apolipoprotein A-I (apoA-I) have been posed as candidate markers of diseases associated with oxidative stress. Here, a dilute-and-shoot form of LC-electrospray ionization-MS requiring half a microliter of blood plasma was employed to simultaneously quantify the relative abundance of these oxidized proteoforms in samples stored at -80 °C, -20 °C, and room temperature and exposed to multiple freeze-thaw cycles and other adverse conditions in order to assess the possibility that protein oxidation may occur as a result of poor sample storage or handling. Samples from a healthy donor and a participant with poorly controlled type 2 diabetes started at the same low level of protein oxidation and behaved similarly; significant increases in albumin oxidation via S-cysteinylation were found to occur within hours at room temperature and days at -20 °C. Methionine oxidation of apoA-I took place on a longer time scale, setting in after albumin oxidation reached a plateau. Freeze-thaw cycles had a minimal effect on protein oxidation. In matched collections, protein oxidation in serum was the same as that in plasma. Albumin and apoA-I oxidation were not affected by sample headspace or the degree to which vials were sealed. ApoA-I, however, was unexpectedly found to oxidize faster in samples with lower surface-area-to-volume ratios. An initial survey of samples from patients with inflammatory conditions normally associated with elevated oxidative stress-including acute myocardial infarction and prostate cancer-demonstrated a lack of detectable apoA-I oxidation. Albumin S-cysteinylation in these samples was consistent with known but relatively brief exposures to temperatures above -30 °C (the freezing point of blood plasma). Given their properties and ease of analysis, these oxidized proteoforms, once fully validated, may represent the first markers of blood plasma specimen integrity based on direct measurement of oxidative molecular damage that can occur under suboptimal storage conditions.

Content validity of the NCCN-FACT ovarian symptom index-18 (NFOSI-18).

OBJECTIVE: This study examined the content validity of the NCCN-FACT Ovarian Symptom Index-18 (NFOSI-18), an advanced ovarian cancer symptom index comprised of symptoms perceived as most important by clinical experts and women with advanced ovarian cancer. METHODS: Eighteen women with advanced ovarian cancer completed the NFOSI-18 and participated in cognitive interviews to assess: (a) the understandability of the NFOSI-18; and (b) the things patients have in mind when responding to the item, "I am bothered by side effects of treatment;" and (c) the interpretation patients place on items relating to fatigue and lack of energy. Interviews were recorded and transcribed for qualitative analysis. RESULTS: All but 2 (89%) participants indicated that each item was clear and understandable and the same proportion (89%) stated they were "very confident" or "confident" about providing accurate answers to all but one item. When responding to the item, "I am bothered by side effects of treatment," fatigue, nausea, and neuropathy constituted the most frequently mentioned concerns. Among the participants who were asked, eight participants responded that "fatigue" and "lack of energy" were the same concept and nine responded they were different. Participants associated "fatigue" with tiredness and associated "lack of energy" with the inability to perform daily tasks and activities. CONCLUSIONS: The findings support the content validity of the NFOSI-18. Item revisions, deletions or additions do not appear warranted. Future research can address the reliability and validity of the NFOSI-18 in clinical research.

Development of a symptom index for patients with primary brain tumors.

OBJECTIVES: This study's primary goals included identifying the highest priority symptoms of patients with advanced brain tumors on treatment, comparing patient priority ratings with those of oncology experts, and constructing a brief symptom index using combined input to assess these symptoms and concerns. METHODS: Fifty patients with advanced primary brain tumors and 10 physician experts were recruited from the National Comprehensive Cancer Network institutions and community support agencies. By using a 40-item symptom checklist, patients first selected up to 10 of the most important symptoms/concerns to monitor when assessing the value of drug treatment for brain tumors, then nominated up to 5 of the very most important concerns, and finally generated additional symptoms/concerns. By using the same checklist as patients, physicians rated each symptom/concern as disease- or treatment-related. RESULTS: By using the combined input, a 24-item National Comprehensive Cancer Network/Functional Assessment of Cancer Therapy-Brain Symptom Index (NFBrSI-24) was developed. The NFBrSI-24 showed good internal consistency (α = 0.84), significantly differentiated patients with different levels of functional status (F2,47 = 8.21; P < .001), and demonstrated good convergent validity with the Functional Assessment of Cancer Therapy-General functional, physical, social, emotional, and brain tumor-specific concerns (ρ = 0.59, 0.57, 0.40, 0.35, and 0.50, respectively; Ps < 0.05). CONCLUSIONS: The NFBrSI-24, an index of the symptoms in advanced brain tumors perceived as most important by both patients and clinicians, improves upon existing measures of brain tumor symptoms through better satisfaction of regulatory requirements for measure development. The findings suggest good reliability and validity, indicating that the NFBrSI-24 is a promising brief assessment of high-priority advanced brain tumor symptoms for research and clinical settings.

Enhanced Clinical Decisions for Management of Benign Prostatic Hyperplasia using Patient-Reported Outcomes: Protocol for a Prospective Observational Study.

Background: Lower urinary tract symptoms (LUTS) due to benign prostatic hyperplasia (BPH) significantly impact quality of life among older men. Despite the prevalent use of the American Urological Association Symptom Index (AUA-SI) for BPH, this measure overlooks key symptoms such as pain and incontinence, underscoring the need for more comprehensive patient-reported outcome (PRO) tools. This study aims to integrate enhanced PROs into routine clinical practice to better capture the spectrum of LUTS, thereby improving clinical outcomes and patient care. Methods: This prospective observational study will recruit men with LUTS secondary to BPH aged ≥ 50 years from urology clinics. Participants will be stratified into medical and surgical management groups, with PRO assessments scheduled at regular intervals to monitor LUTS and other health outcomes. The study will employ the LURN Symptom Index (SI)-29 alongside the traditional AUA-SI and other non-urologic PROs to evaluate a broad range of symptoms. Data on comorbidities, symptom severity, and treatment efficacy will be collected through a combination of electronic health records and PROs. Analyses will focus on the predictive power of these tools in relation to symptom trajectories and treatment responses. Aims are to: (1) integrate routine clinical tests with PRO assessment to enhance screening, diagnosis, and management of patients with BPH; (2) examine psychometric properties of the LURN SIs, including test-retest reliability and establishment of clinically meaningful differences; and (3) create care-coordination recommendations to facilitate management of persistent symptoms and common comorbidities measured by PROs. Discussion: By employing comprehensive PRO measures, this study expects to refine symptom assessment and enhance treatment monitoring, potentially leading to improved personalized care strategies. The integration of these tools into clinical settings could revolutionize the management of LUTS/BPH by providing more nuanced insights into patient experiences and outcomes. The findings could have significant implications for clinical practices, potentially leading to updates in clinical guidelines and better health management strategies for men with LUTS/BPH. Trial registration: This study is registered in ClinicalTrials.gov (NCT05898932).

Development and initial validation of the NCCN/FACT symptom index for advanced kidney cancer.

OBJECTIVES: There is a need for a brief symptom index for advanced kidney cancer that includes perspectives of both patients and clinicians and is consistent with the Food and Drug Administration's guidance for patient-reported outcome measures. This study developed and examined the preliminary reliability and validity of the new National Comprehensive Cancer Network/Functional Assessment of Cancer Therapy (FACT)-Kidney Symptom Index 19. METHODS: Fifty patients with advanced kidney cancer provided open-ended and survey responses ranking their most important symptoms. Responses were reconciled with published clinician reports of the most important symptoms. Ten experienced oncologists rated symptoms as disease- or treatment-related. Patients completed quality-of-life and performance status measures. RESULTS: A 19-item index was produced from symptoms that were rated as most important by patients or clinicians. It includes three subscales: disease-related symptoms (DRS), treatment side effects (TSE), and general function and well-being (FWB). Internal consistency was good for the full instrument (α = 0.83), the DRS subscale (α = 0.76), and the FWB subscale (α = 0.78) but lower for the TSE subscale (α = 0.59). Convergent validity was demonstrated through correlations with the FACT-General. Patients with differing performance status were distinguished by the total score (F2,47 = 17.37; P < .0001), the DRS subscale (F2,47 = 14.22; P < .0001), and the FWB subscale (F2,47 = 13.40; P < .0001) but not the TSE subscale (F2,47 =1.48; P = 0.2380). CONCLUSIONS: The National Comprehensive Cancer Network/FACT-Kidney Symptom Index 19 combines symptoms deemed most important by patients and clinicians. Preliminary evidence suggests that the total score and DRS and FWB subscales are reliable and valid as summary indexes. The TSE subscale may be least relevant given the advent of newer therapies.

Measuring priority symptoms in advanced bladder cancer: development and initial validation of a brief symptom index.

BACKGROUND: Improved measurement of clinically meaningful symptoms is needed in advanced bladder cancer. OBJECTIVE: This study developed and examined the initial reliability and validity of a new measure of advanced bladder cancer-specific symptoms, the NCCN-FACT Bladder Symptom Index-18 (NFBISI-18), which assesses the symptoms perceived as most important by patients and oncology clinical experts. METHODS: A total of 31 individuals with advanced bladder cancer rated the importance of 28 symptoms. In addition, 10 oncology clinical experts rated symptoms as treatment- or disease-related. Patient-rated symptoms were reconciled with published clinicians' symptom priorities, producing the NFBISI-18. Participants completed measures of quality of life (QOL) and performance status to examine initial validity. RESULTS: An 18-item symptom index for advanced bladder cancer included 3 subscales: disease-related symptoms, treatment side effects, and general function/well-being. Lower scores indicate greater symptom burden. Preliminary reliability reveals good internal consistency for the full NFBISI-18 (alpha = 0.83). The NFBISI-18 was significantly associated with QOL criteria and performance status, in the expected direction. LIMITATIONS: Limitations include the cross-sectional design and the relatively low reliability of the disease-related symptoms subscale. CONCLUSION: The NFBISI-18 demonstrates preliminary evidence as a valid brief measure of the most important symptoms of advanced bladder cancer, as rated by both patients and oncology clinical experts. The NFBISI-18 should have greater acceptability to regulatory authorities than previously developed questionnaires.

Using IT to Improve Outcomes for Children Living With Cancer (SyMon-SAYS): Protocol for a Single-Institution Waitlist Randomized Controlled Trial.

BACKGROUND: Children and adolescents with cancer may experience multiple disease- and treatment-related symptoms that negatively affect health-related quality of life. Routine symptom surveillance thus constitutes an important component of supportive care in pediatric oncology. The Symptom Monitoring and Systematic Assessment and Reporting System in Young Survivors (SyMon-SAYS) system will administer, score, interpret, and display the results of symptom assessments captured weekly using patient-reported outcomes presented via the electronic health record (EHR) portal between clinic visits in oncology ambulatory settings, when patients are likely to be more symptomatic. This study is testing a digital system for routine symptom surveillance that includes EHR-based reports to clinicians and alerts for severe symptoms. OBJECTIVE: In this randomized trial, we are examining the effects of the SyMon-SAYS system on perceived barriers to symptom management, self-efficacy, and symptom severity. Better self-management and timely clinical intervention to address symptoms promote adherence to treatment plans, strengthen child and parent self-efficacy, improve interactions between children, parents, and their clinical providers, and optimize clinical outcomes. METHODS: The SyMon-SAYS system is integrated into the EHR to streamline the presentation of symptom scores and delivery of alerts for severe symptoms to clinicians using EHR (Epic) messaging functionalities. Children (aged 8 to 17 years) complete the weekly symptom assessment and review the symptom report by logging into the patient portal (Epic MyChart). This single-institution waitlist randomized controlled trial is recruiting 200 children (aged 8-17 years) with cancer and their parents, guardians, or caregivers. Participating dyads are randomly assigned to receive the intervention over 16 weeks (Group A: 16-week SyMon-SAYS intervention; Group B: 8-week usual care and then an 8-week SyMon-SAYS intervention). Analyses will (1) evaluate the efficacy of SyMon-SAYS at week 8 and the maintenance of those effects at week 16; (2) evaluate factors associated with those efficacy outcomes, including contextual factors, adherence to the SyMon-SAYS intervention, demographic characteristics, and clinical factors; and (3) evaluate predictors of adherence to the SyMon-SAYS intervention and preference of SyMon-SAYS versus usual care. RESULTS: Data collection is currently in progress. We hypothesize that at 8 weeks, those receiving the SyMon-SAYS intervention will report decreased parent-perceived barriers to managing their children's symptoms, increased parent and child self-efficacy, decreased child symptom burden, and ultimately better child health-related quality of life, compared to waitlist controls. Feasibility, acceptability, and engagement from the perspectives of the children with cancer, their parents, and their clinicians will be examined using mixed methods. CONCLUSIONS: We anticipate that this system will facilitate prompt identification of problematic symptoms. Additionally, we hypothesize that with the availability of graphical symptom reports over time, and timely provider responses, children or parents will become better informed and take an active role in managing their symptoms, which will further improve clinical outcomes. TRIAL REGISTRATION: ClinicalTrials.gov NCT04789720; https://clinicaltrials.gov/study/NCT04789720. INTERNATIONAL REGISTERED REPORT IDENTIFIER (IRRID): DERR1-10.2196/50993.

Ethical and Psychosocial Factors in the Decision-Making and Informed Consent Process for Upper Extremity Vascularized Composite Allotransplantation: A Mixed-Methods Study.

Although upper extremity (UE) vascularized composite allotransplantation (VCA) aims to improve quality of life, relatively few have been performed worldwide to support evidence-based treatment and informed decision-making. Methods: We qualitatively examined factors contributing to anticipated and actual decision-making about UE VCA and perceptions of the elements of informed consent among people with UE amputations, and UE VCA candidates, participants, and recipients through in-depth interviews. Thematic analysis was used to analyze qualitative data. Results: Fifty individuals participated; most were male (78%) and had a mean age of 45 y and a unilateral amputation (84%). One-third (35%) were "a lot" or "completely" willing to pursue UE VCA. UE VCA decision-making themes included the utility of UE VCA, psychosocial impact of UE VCA and amputation on individuals' lives, altruism, and anticipated burden of UE VCA on lifestyle. Most respondents who underwent UE VCA evaluation (n = 8/10) perceived having no reasonable treatment alternatives. Generally, respondents (n = 50) recognized the potential for familial, societal, cultural, medical, and self-driven pressures to pursue UE VCA among individuals with amputations. Some (n = 9/50, 18%) reported personally feeling "a little," "somewhat," "a lot," or "completely" pressured to pursue UE VCA. Respondents recommended that individuals be informed about the option of UE VCA near the amputation date. Conclusions: Our study identified psychosocial and other factors affecting decision-making about UE VCA, which should be addressed to enhance informed consent. Study participants' perceptions and preferences about UE VCA suggest re-examination of assumptions guiding the UE VCA clinical evaluation process.

A Patient-Centered Website (Within Reach) to Foster Informed Decision-making About Upper Extremity Vascularized Composite Allotransplantation: Development and Usability Study.

BACKGROUND: Upper extremity (UE) vascularized composite allotransplantation (VCA; hand transplantation) is a reconstructive treatment option for patients with UE loss. Approximately 37 UE VCAs have been performed in the United States to date; thus, little is known about long-term psychosocial outcomes and whether the benefits outweigh the risks. To make an informed treatment decision, patients must understand the procedure, risks, and potential benefits of UE VCA. However, few educational resources are publicly available providing unbiased, comprehensive information about UE VCA. OBJECTIVE: This paper described the development of a neutral, and accessible, educational website supporting informed decision-making about UE VCA as a treatment option for individuals with UE amputations. METHODS: Website content development was informed by 9 focus groups conducted with individuals with UE amputations at 3 study sites. After initial website development, we conducted usability testing to identify ways to improve navigability, design, content, comprehension, and cultural sensitivity. Participants were administered the After-Scenario Questionnaire to assess user performance after completing navigational tasks, System Usability Scale to measure the perceived usability of the website, and Net Promoter Score to measure user satisfaction. Quantitative data were analyzed using descriptive statistics. Qualitative data were analyzed using rapid thematic analysis. RESULTS: A total of 44 individuals with UE amputations participated in focus groups (n=37, 84%) and usability testing (n=14, 32%). Most participants in the focus groups and usability testing were male (24/37, 65% and 11/14, 79%, respectively) and White (27/37, 73% and 9/14, 64%, respectively), had unilateral limb loss (22/37, 59% and 12/14, 86%, respectively), and had mean ages of 48 (SD 9.2) and 50 (SD 12.0) years, respectively. Focus group results are organized into accessibility, website design, website development, website tone and values, sitemap, terminology, images and videos, and tables and graphics. Usability testing revealed that participants had a positive impression of the website. The mean After-Scenario Questionnaire score of 1.3 to 2.3 across task scenarios indicated high satisfaction with website usability, the mean System Usability Scale score of 88.9 indicated user satisfaction with website usability, and the mean Net Promoter Score of 9.6 indicated that users were enthusiastic and would likely refer individuals to the website. CONCLUSIONS: The findings suggest that our educational website, Within Reach, provides neutral, patient-centered information and may be a useful resource about UE VCA for individuals with UE amputations, their families, and health care professionals. Health care professionals may inform UE VCA candidates about Within Reach to supplement current VCA education processes. Within Reach serves as a resource about treatment options for patients preparing for scheduled or recovering from traumatic UE amputations. Future research should assess whether Within Reach improves knowledge about UE VCA and enhances informed decision-making about UE VCA as a treatment option.

Patient preferences of patient selection criteria for upper extremity vascularized composite allotransplantation: A qualitative study.

Background: Upper extremity vascularized composite allotransplantation is a life-enhancing reconstructive treatment option that aims to improve recipients' quality of life and maximize function. This study assessed upper extremity vascularized composite allotransplantation patient selection criteria perceptions among individuals with upper extremity limb loss. The perceptions of individuals with upper extremity limb loss on patient selection criteria may enable vascularized composite allotransplantation centers to improve criteria to avoid mismatched expectations about the posttransplant vascularized composite allotransplantation experience and outcomes. Realistic patient expectations may increase patient adherence, improve outcomes, and reduce vascularized composite allotransplantation graft loss. Methods: We conducted in-depth interviews with civilian and military service members with upper extremity limb loss and upper extremity vascularized composite allotransplantation candidates, participants, and recipients from three US institutions. Interviews assessed perceptions of patient selection criteria for suitability as a candidate for upper extremity vascularized composite allotransplantation. Thematic analysis was used to analyze qualitative data. Results: A total of 50 individuals participated (66% participation rate). Most participants were male (78%), White (72%), with a unilateral limb loss (84%), and a mean age of 45 years. Six themes emerged regarding upper extremity vascularized composite allotransplantation patient selection criteria, including support for candidates who: (1) are of younger age, (2) are in good physical health, (3) have mental stability, (4) are willing to "put in the work," (5) have specific amputation characteristics, and (6) have sufficient social support. Patients had preferences about selecting candidates with unilateral versus bilateral limb loss. Conclusions: Our findings suggest that numerous factors, including medical, social, and psychological characteristics, inform patients' perceptions of patient selection criteria for upper extremity vascularized composite allotransplantation. Patient perceptions of patient selection criteria should inform the development of validated screening measures that optimize patient outcomes.

Patient definitions of transplant success in upper extremity vascularized composite allotransplantation: A mixed-methods study.

Objective: Upper extremity vascularized composite allotransplantation is an innovative treatment option for people with upper extremity amputations. Limited patient-relevant long-term outcomes data about transplant success may impede patients' informed treatment decision-making. We assessed perceptions of what constitutes upper extremity vascularized composite allotransplantation success among individuals with upper extremity amputations. Methods: This multisite study entailed interviews and focus groups with individuals with upper extremity amputations and upper extremity vascularized composite allotransplantation candidates, participants, and recipients. We examined perceptions of transplant success and preferences for five upper extremity vascularized composite allotransplantation outcomes. Qualitative data were analyzed using thematic analysis; and quantitative data were analyzed using descriptive statistics. Results: In all, 50 individuals participated in interviews (61.7% participation rate), and 37 participated in nine focus groups (75.5% participation rate). Most were White (72%, 73%), had a mean age of 45 and 48 years, and had a unilateral amputation (84%, 59%), respectively. Participants conceptualized upper extremity vascularized composite allotransplantation success as transplant outcomes: (1) restoring function and sensation to enable new activities; (2) accepting the transplanted limb into one's identity and appearance; (3) not having transplant rejection; (4) attaining greater quality of life compared to prosthetics; and (5) ensuring benefits outweigh risks. Participants rated their most important upper extremity vascularized composite allotransplantation outcomes as follows: not having transplant rejection, not developing health complications, grasping objects, feeling touch and temperature, and accepting the upper extremity vascularized composite allotransplantation into your identity. Conclusion: Individuals with upper extremity amputations maintain several conceptions of vascularized composite allotransplantation success, spanning functional, psychosocial, clinical, and quality of life outcomes. Providers should address patients' conceptions of success to improve informed consent discussions and outcomes reporting for upper extremity vascularized composite allotransplantation.

Measuring health-related quality of life in leukemia: the Functional Assessment of Cancer Therapy--Leukemia (FACT-Leu) questionnaire.

OBJECTIVE: Develop and validate a health-related quality-of-life (measure for patients with acute and chronic leukemia. METHODS: The study consisted of two phases: scale construction and scale validation. For the item-generation phase, a summary of the literature combined with qualitative results from item-generation interviews with 29 acute or chronic leukemia patients and 16 health care providers yielded an initial item pool reflecting leukemia-specific concerns and symptoms. Items underwent iterations of review and reduction according to defined retention criteria to support content validity, as defined by priority concerns of patients. Seventeen final leukemia-specific items were combined with the Functional Assessment of Cancer Therapy-General to create the FACT-Leukemia (FACT-Leu) scale. For the validation phase, 79 individuals with acute or chronic leukemia completed questionnaires at three time points. RESULTS: All FACT-Leu subscale and aggregated scores showed high internal consistency (αs ranging from 0.75 to 0.96). Test-retest reliability was adequate for all subscales (intraclass correlation range 0.765-0.890). The FACT-Leu scale demonstrated good convergent validity, with significant correlations with quality-of-life criteria and performance status, in the expected direction. FACT-Leu subscale scores were significantly different among the three performance status change groups, suggesting good responsiveness to change. CONCLUSIONS: The FACT-Leu scale is a valid, reliable, and efficient measure of leukemia-specific health-related quality of life for acute and chronic disease.