Cleft-related Patient Reported Outcome Measure (PROM) results were formatted into graphical displays for children scoring below the 25th percentile on one or more scales. Reports were piloted in a multidisciplinary clinic where providers reviewed them, and their impact was qualitatively recorded. Graphical PROM reports informed discussions, led to treatment plan changes, and raised awareness of unmet psychosocial needs. Because of the success of this quality improvement pilot, visual PROM reports will become a regular part of our multidisciplinary cleft care. More broadly, graphical PROM data display facilitates better understanding of the patient\'s perspective and leads to more informed visits.

Physical activity can worsen migraine, leading to reduced activity levels in adults with chronic migraine. This study investigated the change in average steps per day, as a surrogate marker of physical activity, in adults with chronic migraine successfully treated with monoclonal antibodies against calcitonin gene-related peptide or its receptor. Data were obtained from adults with chronic migraine, who were classified as responders to preventive treatment with monoclonal antibodies. The primary endpoint was the difference in a mean number of steps per day between the 3 months prior to treatment initiation and the first 3 months after treatment initiation. The secondary endpoint was the correlation between the change in steps per day and the change in monthly migraine days. Twenty-two (20 females) participants with a median age of 48.5 years were enrolled. The median number of steps per day increased from 4421 at baseline to 5241 after treatment (P = 0.039). We found a positive correlation between the increase in steps per day and the treatment response (P = 0.013). In conclusion, an increase in physical activity, based on steps per day, positively correlated with treatment response to monoclonal antibodies. Automatically registered daily step count data might be used to monitor physical activity as a response to preventive treatment in adults with chronic migraine.

BACKGROUND: Patient-reported outcome measures (PROMs) and patient-reported experiences measures (PREMs) are crucial for understanding the impact of GD on quality of life and patient\'s perceptions on care, but also to guide decision-making processes. Nevertheless, no specific PREMs in GD have been published, neither PROMs for Spanish GD patients have been developed.
METHODS: Two project coordinators selected key-points to be included in a PROMs/PREMs questionnaire, and the scientific committee and a group of expert patients contributed to the initial draft. Then, 9 meetings with experts were held to discuss controversial points. After, a questionnaire with 103 items regarding symptomatology, aspects of daily life and care experience was developed. Finally, it was conducted a Delphi survey among a multidisciplinary group of experts in GD.
RESULTS: Consensus was reached on 85 out of the 103 items. Recommendations on PROMs and PREMs regarding symptomatology, aspects of daily life and care experience were obtained. Consensus was reached on the importance of considering fatigue, concentration problems, and communication issues in GD patients using 5-step analog scales. Panelists recommended asking GD patients about the impact on social functioning and work/school performance. Finally, consensus was reached on considering care experiences, such as treatment satisfaction, treatment interruptions or transitions and healthcare professionals involved in patient\'s management to perceive patient\'s perceptions.
CONCLUSIONS: This expert consensus may help developing GD-specific PROMs/PREMs for improving GD management. Properly developed and validated PROMs/PREMs may help decision-making, establishing patient-tailored therapeutic and follow-up goals.

Background and Objective: Pregnancy in mothers with multiple sclerosis (MS) commonly results in significant changes in disease activity and changes in clinical care, including the discontinuation of disease modifying therapy (DMT). This study aimed at understanding the clinical and patient-reported outcomes (PROs) before, during and 1-year after delivery. Materials and Methods: A total of 30 pregnant mothers with MS were recruited as part of the study. Clinical (relapse activity and disability changes), PRO information and MRI outcomes were collected on four separate visits: one baseline visit-0-30 days post-delivery; and 3 follow-up visits at week 24, week 36 and week 52 from the baseline. PRO was assessed using a validated questionnaire called the Fatigue Scale for Motor and Cognitive Function (FSMC). The MRI scans were analyzed, and the count of new T2 lesions and/or contrast-enhancing lesions was determined. Results: The average time between delivery and the start of DMT was 142.5 days. Relapse activity before the pregnancy was numerically linked with the activity during the pregnancy, where up to 57.1% of the activity during pregnancy occurred in pwMS with previously active disease before conception (statistically trending with p = 0.073). The relapse activity after the pregnancy occurred twice as often in pwMS whose MS was clinically active before conception. All five pwMS who experienced a relapse prior to the pregnancy experienced worsening in their physical PRO domain. Conclusions: Pre-pregnancy activity is crucial in the screening of mothers with MS at risk for post-partum relapses, worsening of clinical disability and/or PRO measures. A post-partum MS period may benefit from the routine PRO utilization and screening for its worsening. The inflammatory activity during pregnancy was not associated with short-term disease progression.

BACKGROUND: Patient reported outcomes (PROs) are being used frequently in clinical practice. PROs often serve several purposes, such as increasing patient involvement, assessing health status, and monitoring and improving the quality-of-care at an aggregated level. However, the lack of representative PRO-data may have implications for all these purposes. This study aims to assess the association of non-administration of (not sending an electronic invite to PRO) and non-response to (not responding to PRO) electronically administered PROs with social inequality in a primary healthcare cancer rehabilitation setting. Furthermore, it examines whether the workflows surrounding PRO have an impact on non-administration and non-response.
METHODS: This is a cross sectional study using routinely collected data from electronic health records and registers including cancer survivors (CSs) over 18 years booked for an initial consultation in a primary healthcare cancer rehabilitation setting using PROs for systematic health status assessment. During the study period two different PRO platforms were used, each associated with different workflows. Non-administration and non-response rates were calculated for sociodemographic characteristics for each PRO platform. Crude and adjusted odds ratios were calculated using univariate and multivariate logistic regression.
RESULTS: In total, 1868 (platform 1) and 1446 (platform 2) CSCSs were booked for an initial consultation. Of these, 233 (12.5%) (platform 1) and 283 (19.6%) (platform 2) were not sent a PRO (non-administration). Among those who received a PRO, 157 (9.6%) on platform 1 and 140 (12.0%) on platform 2 did not respond (non-response). Non-administration of and non-response to PROs were significantly associated with lower socioeconomic status. Moreover, the workflows surrounding PROs seem to have an impact on non-inclusion in and non-response to PROs.
CONCLUSIONS: Non-administration of and non-response to PROs in clinical practice is associated with determinants of social inequality. Clinical workflows and the PRO platforms used may potentially worsen this inequality. It is important to consider these implications when using PROs at both the individual and aggregated levels. A key aspect of implementing PROs in clinical practice is the ongoing focus on representativeness, including a focus on monitoring PRO administration and response.

The Setting International Standards in Analyzing Patient-Reported Outcomes and Quality of Life Endpoints Data (SISAQOL) initiative was established in 2016 to assess the quality and standardization of patient-reported outcomes (PRO) data analysis in randomized controlled trials (RCTs) on advanced breast cancer. The initiative identified deficiencies in PRO data reporting, including nonstandardized methods for handling missing data. This study evaluated the reporting of health-related quality of life (HRQOL) in Japanese cancer RCTs to provide insights into the state of PRO reporting in Japan. The study reviewed PubMed articles published from 2010 to 2018. Eligible studies included Japanese cancer RCTs with ≥50 adult patients (≥50% were Japanese) with solid tumors receiving anticancer treatments. The evaluation criteria included clarity of the HRQOL hypotheses, multiplicity testing, primary analysis methods, and reporting of clinically meaningful differences. Twenty-seven HRQOL trials were identified. Only 15% provided a clear HRQOL hypothesis, and 63% examined multiple HRQOL domains without adjusting for multiplicity. Model-based methods were the most common statistical methods for the primary HRQOL analysis. Only 22% of the trials explicitly reported clinically meaningful differences in HRQOL. Baseline assessments were reported in most trials, but only 26% reported comparisons between the treatment groups. HRQOL analysis was based on the intention-to-treat population in 19% of the trials, and 74% reported compliance at follow-up; however, 41% did not specify how missing values were handled. Although the rates of reporting clinical hypotheses and clinically meaningful differences were relatively low, the current state of HRQOL evaluation in the Japanese cancer RCT appears comparable to that of previous studies.

BACKGROUND: The Multiple Sclerosis Walking Scale-12 (MSWS-12) has typically been delivered through paper-and-pencil or computer-based administration.
OBJECTIVE: This study examined the validity of inferences from scores derived via a telephone administration of the MSWS-12 applied as part of screening of participants with walking dysfunction into a clinical trial of exercise training in MS.
METHODS: The MSWS-12 was administered on two occasions separated by approximately 2 weeks through the telephone and then in-person (i.e., computer-based administration). Participants further completed the Patient Determined Disease Steps (PDDS) scale, timed 25-foot walk (T25FW), six-minute walk (6MW), Modified Fatigue Impact Scale (MFIS), and Multiple Sclerosis Impact Scale-29 (MSIS-29), and underwent a neurological exam for generating an expanded disability status scale (EDSS) score. The primary set of data (Full Sample) for analyses included all persons who passed the telephone screening for inclusion with MSWS-12 scores between 25 and 75 (N = 374). The secondary set of data (Truncated Sample) included only persons with MSWS-12 scores between 25 and 75 for both the telephone and computer administrations of the MSWS-12 (N = 248).
RESULTS: The results in the Full Sample indicated a difference in overall and item levels scores between the telephone and computer data collections, and the computer version had higher internal consistency and stronger unidimensionality. Nevertheless, MSWS-12 scores from both modes of administration had comparable correlations with the T25FW, 6MW, EDSS, PDDS, MFIS, and MSIS-29, but the correlation between the two MSWS-12 administrations did not approach unity. There was a systematic difference in scores between telephone and computer administrations across levels of walking dysfunction based on a Bland-Altman plot, and the difference was predicted by MFIS physical, 6MW, and EDSS scores. The comparison of results between the Full and Truncated Samples suggested that the primary analysis might have been influenced by the larger range of scores on the computer than telephone administrations of the MSWS-12.
CONCLUSIONS: The telephone administration of the MSWS-12 provides an efficient and cost-effective measure of walking dysfunction in persons with MS.

OBJECTIVE: Recently, efforts have been made to use and report person-reported outcomes (PROs) in randomised clinical trials (RCTs). Here, we aim to (1) assess the status of inclusion of PROs in registered RCTs over 5 years in people with type 1 or 2 diabetes, and (2) map the PRO measures (PROMs) onto predefined domains.
METHODS: The largest trial registries (Clinicatrials.gov, International Clinical Trial Platform and ISRCTN) were systematically searched for RCTs in people with type 1 and/or type 2 diabetes of all ages between 2018 and 2023. Coding of PROs comprised: (1) PRO measure(s) included yes or no; if yes: (2) PRO(s) as primary outcome yes or no; and (3) mapping PROMs onto predefined PRO domains and per type of intervention.
RESULTS: N = 1543 trials met our inclusion criteria, of which n = 673 (44%) included PROs, assessed by 545 different measures. Twenty per cent of drug trials (n = 112) and 71% of behavioural interventions (n = 405) included PROs. In 149 trials (9.6%), a PRO was the primary outcome. The psychological functioning domain was most often assessed across all trials (21.6%), specifically in behavioural (44.8%) and medical device interventions (29.7%). In drug trials, the physical functioning and functional health domain was most included (9%). Across all trials, the social and family functioning domain was least assessed (3%).
CONCLUSIONS: We noticed an increase in the inclusion of PROs in diabetes RCTs. However, PROs are rarely included as primary outcomes in the majority of studies, particularly in drug trials. The heterogeneity of PROMs used in RCTs underscores the need for standardisation of PROs.

Patient Reported Outcomes (PROs) are widely used in quality of life (QOL) studies, health outcomes research, and clinical trials. The importance of PRO has been advocated by health authorities. We propose this R shiny web application, PROpwr, that estimates power for two-arm clinical trials with PRO measures as endpoints using Item Response Theory (GRM: Graded Response Model) and simulations. PROpwr also supports the analysis of PRO data for convenience of estimating the effect size. There are seven function tabs in PROpwr: Frequentist Analysis, Bayesian Analysis, GRM power, T-test Power Given Sample Size, T-test Sample Size Given Power, Download, and References. PROpwr is user-friendly with point-and-click functions. PROpwr can assist researchers to analyze and calculate power and sample size for PRO endpoints in clinical trials without prior programming knowledge.

Desmoid tumors (DT) are rare, intermediate-grade sarcomas characterized by locally aggressive growths that commonly occur intra-abdominally, in the abdominal wall, or in the extremities. Desmoid tumors are 2-3-fold more common in females than males, with most patients aged <40 years at diagnosis. Clinical course of DT is highly variable but rarely fatal, with median overall survival >80% at 20 years. However, patient morbidity and DT symptom burden can be high. DT significantly reduce patient quality of life, imposing substantial physical, emotional, and social burdens. Pain, fatigue, and insomnia are common symptoms; disfigurement, mobility restrictions, and, rarely, the need for amputation may also result. Despite its limited impact on survival, patients with DT may have anxiety and depression levels commensurate with those associated with malignant sarcomas. Thus, DT impose an array of significant, long-term morbidities on a young patient population. In order to evaluate the impact of these morbidities, patient-reported outcome (PRO) tools are used, which assess outcomes of importance to patients that extend beyond traditional oncology endpoints. General or oncology-related PROs can be used; although currently, the only DT-specific, validated PRO measure is the GOunder/Desmoid Tumor Research Foundation DEsmoid Symptom/Impact Scale (GODDESS©), consisting of an 11-item DT Symptom Scale (DTSS) and a 17-item DT Impact Scale (DTIS). DTSS and DTIS were secondary endpoints in DeFi, a randomized phase 3 trial of nirogacestat; blinded, pooled data from DeFi were used to validate GODDESS reliability and responsiveness as a PRO measure in DT. Another DT-specific PRO measure, the Desmoid-Type Fibromatosis Quality of Life (DTF-QoL) questionnaire, has been developed but not validated. As novel DT therapies continue to be developed, incorporating DT-specific PRO measures into clinical trials will be key to capturing patient voice, improving outcomes of importance to this unique patient population, and assisting patients and providers in selecting optimal treatment.