Patient Self-report Research Articles

Carcinogenic effects of prolonged daily low-emission phototherapy in psoriasis.

BackgroundLow‐dose UV treatment has been shown to be effective in mild psoriasis. However, the prolonged use of this treatment modality may raise concerns about its safety. These concerns are mainly focused on potential carcinogenic risks and overuse of this treatment modality.ObjectivesThis study was set out to evaluate possible carcinogenic risks of prolonged low‐dose phototherapy.MethodsThree groups of psoriasis patients were evaluated: patients with local treatment only (n = 15); low‐dose UV treatment at home for at least 18 months (n = 39); and patients with conventional NB‐UVB (n = 8). Patients underwent visual inspection for signs of photoageing, and p53, CPDs and γH2AX were measured in skin biopsies. Patients undergoing low‐dose phototherapy answered a survey about their recent patterns of use in a survey.ResultsIn the skin biopsies, low‐dose UV treatment caused a lower amount of CPDs (p = .016) and p53 (p = .015) than NB‐UVB. γH2AX did not show a significant difference. Self‐report in patients undergoing low‐dose phototherapy showed only one case of overuse (2.7%). Visual skin inspection showed no difference in signs of photoageing in the three groups.ConclusionProlonged treatment with low‐dose UV for 18 months appears at least as safe as a course of conventional NB‐UVB.

Heterogeneous digital biomarker integration out-performs patient self-reports in predicting Parkinson\u2019s disease

Parkinson’s disease (PD) is one of the first diseases where digital biomarkers demonstrated excellent performance in differentiating disease from healthy individuals. However, no study has systematically compared and leveraged multiple types of digital biomarkers to predict PD. Particularly, machine learning works on the fine-motor skills of PD are limited. Here, we developed deep learning methods that achieved an AUC (Area Under the receiver operator characteristic Curve) of 0.933 in identifying PD patients on 6418 individuals using 75048 tapping accelerometer and position records. Performance of tapping is superior to gait/rest and voice-based models obtained from the same benchmark population. Assembling the three models achieved a higher AUC of 0.944. Notably, the models not only correlated strongly to, but also performed better than patient self-reported symptom scores in diagnosing PD. This study demonstrates the complementary predictive power of tapping, gait/rest and voice data and establishes integrative deep learning-based models for identifying PD.

Do Patients and Therapists Talk About the Same Thing? An Analysis of the Correspondence between the Self-Reports of Patients and Therapists in Transdiagnostic Treatments for Emotional Disorders

Do Patients and Therapists Talk About the Same Thing? An Analysis of the Correspondence between the Self-Reports of Patients and Therapists in Transdiagnostic Treatments for Emotional Disorders

Dual-Energy Computed Tomography for the Diagnosis of Urethral Foreign Body: A Case Study

Foreign body insertion in the urethra and bladder is not uncommon and has been reported in many studies to date. However, since foreign bodies are often accidentally introduced into the urethra and bladder during masturbation, they take a variety of shapes and sizes. Furthermore, patient self-reports are typically unreliable as many patients feel ashamed; thus, appropriate preoperative diagnosis is critical. Diagnosis of foreign body insertion in the urethra and bladder is performed using imaging modalities such as abdominal X-ray and computed tomography (CT). However, single-energy CT (SECT) is not sufficient in detecting foreign bodies in some cases. In the present study, we report a successful preoperative identification of urethral foreign body in a patient using dual-energy CT (DECT).

EXOGEN Mitigates Risk of Fifth Metatarsal Fracture Nonunion: Results of a Novel Real-World Clinical Study.

Category:Midfoot/ForefootIntroduction/Purpose:Fifth metatarsal fractures are the most commonly fractured metatarsal in adults and are associated with high rates of nonunion. This study compared the 9-month incidence of nonunion in fifth metatarsal fracture patients using EXOGEN (Bioventus LLC), low-intensity pulsed ultrasound, versus a control cohort of patients receiving standard of care.Methods:Adults (21-80 years) prescribed EXOGEN for any acute or delayed fifth metatarsal fracture and initiated treatment within 91 days of the fracture were enrolled via prescription record abstraction and direct-to-patient outreach. Open, closed, surgically repaired and conservatively managed fractures were eligible. Jones' fractures consisted of 31.2% of the overall EXOGEN population. EXOGEN treatment compliance was assessed using patient self-report and the device's software. A control cohort of patients with the same eligible fractures were extracted from the MarketScanTM national insurance database (IBM Watson Health). Propensity score (PS) modeling accounting for demographics, comorbidities, concomitant medication, and type of fracture/fracture treatment was used to address potential selection bias via three methods: subclassification, 1:1 matching on individual covariates, and PS weighting. Effectiveness was compared for 1,943 EXOGEN and 10,259 controls. Study endpoint was based on presence or absence of an ICD-10-CM nonunion diagnosis code in the medical billing records for both groups.Results:Matched MarketScan controls had a clinically and statistically significant increase in risk of nonunion compared to patients who used EXOGEN for at least 90 total once-daily treatments (McNemar's OR = 1.67; p=0.048). Results improved with more treatments; the nonunion risk was 2.44 times (p=0.0239) and 5.00 times (p=0.008) for the control patients than EXOGEN patients with at least 120 and 140 total treatments, respectively. The nonunion rate was reduced by over half in Jones' fracture patients treated with at least 90 total once-daily EXOGEN treatments (n=269; 3.3%) as compared to those not reaching this minimum threshold (n=337, 6.8%); such results approached statistical significance (p=0.057). Analyses of the treatment effect by nonunion risk factors showed EXOGEN attenuated the effect of many of these factors on nonunion rates. Early treatment was found beneficial; treatment delay was associated with increased nonunion risk of ~20% for every 15 days of latency 45 days post- fracture.Conclusion:In this large external comparator study, EXOGEN was found to be effective in mitigating the risk of nonunion in fifth metatarsal fracture patients when used for a minimum of 90 total treatments, even in the presence of many nonunion risk factors. Treatment should be initiated as soon as possible following fracture (at least by 45 days post-fracture) to further reduce the risk of nonunion.

Assessment of dietary intake by self-report in adult patients with type 1 diabetes treated with a personal insulin pump

Abstract Introduction Appropriate nutrition is an element affecting the metabolic control of patients with diabetes. There are only a few studies assessing the implementation of dietary recommendations in adult patients with type 1 diabetes; none of them assessed the implementation of nutritional standards. Our study aimed to assess the implementation of dietary recommendations and their relation to metabolic control in adults with T1DM treated with personal insulin pumps. Materials and Methods The study included 48 adult patients who were divided into two subgroups and compared, based on HbA1c above and below 6.5%. Each patient's nutrient, vitamin, and mineral intake was assessed on self-reported 3-day 24-hour surveys of food consumption. Records were introduced into the dietetic software DietaPro, (source: http://www.dietapro.eu/) which revealed nutrient content. We evaluated the percentage of patients with nutrient consumption below recommended values based on current recommendations. Results The studied population was characterized by insufficient consumption of most nutrients and vitamins: sodium, potassium, calcium, magnesium, iron, zinc, copper, iodine, manganese, vitamin A, vitamin D, vitamin E, thiamine, riboflavin, niacin, vitamin B6, folate, vitamin B12, vitamin C. Patients’ diet did supply correct amounts of phosphorus, and too much fatty acid and cholesterol. There were no statistically significant differences in most of the nutrient intakes across the two groups. Nevertheless, we observed a significant difference in the polyunsaturated fatty acids, sodium, niacin, and calcium intakes. Conclusions The studied patients consumed too much saturated fatty acid and dietary cholesterol. The consumption amounts of most nutrients and vitamins were associated with the risk of deficiency. The obtained results indicate the need for further dietary education for patients with T1DM.

Post-Discharge Transitional Care Program and Patient Compliance With Follow-Up Activities.

We explore whether the frequency of post-hospital discharge transitional care calls affects patients’ adherence to their discharge plans. We reviewed 1,000 call records of a post-discharge transitional care program run by a large U.S. tertiary care hospital in 2018–2019 and generated binary outcomes capturing patient self-reports of (1) scheduling or completing follow-up appointments, procedures, or prescription fills, (2) missing a scheduled event, and (3) reporting a new adverse clinical event. Our predictor variables captured callers’ success in completing the first and each subsequent call with discharged patients. We ran linear probability models (LPM) for each binary outcome after controlling for sociodemographic and clinical characteristics. Results indicate successful completion of the first two calls was associated with the increased probability of scheduling or completing follow-up appointment (15.5% points, p < 0.01) and follow-up procedure (13.5% points, p < 0.01), and reporting new adverse event (5.9% points, p < 0.05). Our findings suggest that making the first phone call to patients, while crucial, may not be sufficient for effective care transition; making two to three phone calls seems to be more optimal, while further calls may have limited value.

Reliability of patient self-report of cognition, awareness, and consciousness during seizures.

ObjectiveClinicians rely on patient self‐report of impairment during seizures for decisions including driving eligibility. However, the reliability of patient reports on cognitive and behavioral functions during seizures remains unknown.MethodsWe administered a daily questionnaire to epilepsy patients undergoing continuous video‐EEG monitoring, asking about responsiveness, speech, memory, awareness, and consciousness during seizures in the preceding 24 hours. We also administered a questionnaire upon admission about responsiveness, speech, and awareness during seizures. Subjective questionnaire answers were compared with objective behavioral ratings on video review. Criteria for agreement were Cohen’s kappa >0.60 and proportions of positive and negative agreement both >0.75.ResultsWe analyzed 86 epileptic seizures in 39 patients. Memory report on the daily questionnaire met criteria for agreement with video review (κ = 0.674 for early, 0.743 for late recall). Subjective report of awareness also met agreement criteria with video ratings of memory (κ = 0.673 early, 0.774 late). Concordance for speech was relatively good (κ = 0.679) but did not meet agreement criteria, nor did responsiveness or consciousness. On the admission questionnaire, agreement criteria were met for subjective report of awareness versus video ratings of memory (κ = 0.814 early, 0.806 late), but not for other comparisons.InterpretationPatient self‐report of memory or awareness showed the best concordance with objective memory impairment during seizures. Self‐report of impairment in other categories was less reliable. These findings suggest that patient reports about impaired memory during seizures may be most reliable, and otherwise determining functional impairments should be based on objective observations.

İnhaler Tedavilerine Uyum: Hasta ve Hasta Yakınlarının Beyanı: Kesitsel Çalışma

Objective: It is obvious that adherence to inhaler treatments is insufficient in patients with obstructive lung diseases. In this study, we aimed to evaluate the consistency of the information provided by patients with obstructive pulmonary disease and their households on medication adherence. Material and Methods: A cross-sectional study was conducted between January and March 2020 in a tertiary chest diseases hospital, where patients with asthma and chronic obstructive pulmonary disease (COPD) were regularly followed up. The study included patients who had been followed up with a diagnosis of asthma or COPD for at least 1 year, were using maintenance inhaler therapy, and attended the follow-up visit with a member of the same household. Patients and their household members were independently questioned about patients' medication adherence. According to the information provided by the patients and their households, medication adherence of the patients and the consistency of the information were evaluated. Results: Of the 66 patients with obstructive lung disease, 59 (89.39%) were adherent and 7 (10.60%) were non-adherent according to their own statements. Seventeen (28.81%) of 59 patients who were adherent according to their own statements were non-adherent to treatment with respect to household members. The mean age, gender distribution, smoking status, diagnosis of asthma or COPD, and education level did not differ significantly between self-reported adherent and non-adherent patients or between adherent and non-adherent patients with respect to household members. Conclusion: Adding information from household members to the use of patient self-reports may increase the clinician's judgment in monitoring adherence.

Sleep position, patient comfort, and technical performance with two established procedures for home sleep testing

PurposeIn patients with a high pre-test probability of suffering from obstructive sleep apnea (OSA), (cardio)-respiratory polygraphy (RP; level 3) is commonly used for home sleep testing (HST); however, testing based on peripheral arterial tonometry (PAT) is increasingly recognized as an alternative method. The aim of the study was to compare sleep position, patients’ comfort, and technical failure rates of HST with RP and PAT in patients with suspected OSA.MethodsSleep position, patients’ comfort, and technical failure rates of RP and PAT were compared in 56 patients receiving two nights of HST with either RP or PAT in a randomized fashion.ResultsTime in supine position with PAT was significantly lower (173.7±88 min) compared to RP (181.7±103.7 min; p < 0.001), although the absolute mean difference was not clinically significant. Patients reported to sleep better, feeling less disturbed when falling asleep, losing less sensors, and fewer nightly awakenings with PAT, but experienced more pain at the side of the finger probe. Forty-five out of 56 patients (80%) rated PAT as being the superior sleep test and 49 out of 56 (88%) would prefer PAT for further investigations (p<0.001). PAT testing was associated with less technical failures.ConclusionThe results demonstrate that HST with PAT leads to less time in supine sleep positioning, which may be clinically relevant in selected patients. Moreover, PAT is associated with less technical failures and is perceived with less discomfort during testing and a reduced number of nocturnal awakenings in patient self-reports.

Scoping review to evaluate existing measurement parameters and clinical outcomes of transtibial prosthetic alignment and socket fit.

Fit and alignment are observable objectives of the prosthesis rendering process for individuals with lower limb amputation. Nevertheless, there is a dearth of validated measures to directly assess the quality of this clinical procedure. The objectives of this scoping review are to evaluate existing measurement parameters and clinical outcomes used in investigations of transtibial socket fit or prosthetic alignment and to identify gaps in the literature regarding tools for evaluation of prosthetic fitting. Scoping literature review. A comprehensive search was conducted in the following databases: MEDLINE (through PubMed), Embase (through Elsevier), Scopus (through Elsevier), and Engineering Village (through Elsevier), resulting in 6107 studies to be screened. Sixty-three studies were included in the review. When measuring fit, studies most frequently reported on patient-reported comfort (n = 22) and socket size compared with the residual limb volume (n = 9). Alignment was most frequently measured by the prosthetists' judgment and/or use of an alignment jig (n = 34). The measurement parameters used to determine alignment or fit varied greatly among the included studies. This review demonstrated that most measures of socket fit rely on a patient's self-report and may vary with biopsychosocial factors unrelated to the socket fitting process. Meanwhile, alignment is determined mostly by the prosthetist's judgment, paired with objective measurements, such as alignment jigs and gait analysis. Efforts to standardize and validate measures of these parameters of prosthetic fitting are vital to improving clinical practice and reporting outcomes.

Benefits, Challenges, and Opportunities of Integrating Patient-Reported Outcome Measures in Geriatric Oncology to Advance Patient Screening for Functional Fitness for Treatment.

To discuss potential benefits, challenges, and opportunities of integrating patient-reported outcome measures in geriatric oncology, with the goal being to advance patient screening for 'functional fitness for treatment.' This is narrative review of the relevant literature using PubMed. Patient screening in geriatric cancer care can be driven by patient self-reports. Most of the research on patient-reported outcome measures in cancer care to date has involved, in different proportions and to different extents, older patients with cancer (≥70 years of age). However, reports specifically targeting implementation of patient-reported outcome measures in geriatric cancer care are surprisingly fewer. The International Society of Geriatric Oncology Priorities Initiative calls for more research into the use of patient-reported outcome measures for older adults with cancer. More feasibility studies will be required to evaluate patient-reported outcome measures as fit-for-purpose, reporting frequency, patient burden, integration and display of patient-reported information, and workflow impact. Developing, adapting, and validating patient-reported outcome measures for use in geriatric oncology will be key to ensure adequacy of measurement. Groundwork involving consultation of all potential end users of patient-reported information is paramount because there is no one size fits all. When the ground is ready for implementation adequate preparation, training, resources, and ongoing support will be needed. Working within the multidisciplinary team, nurses can promote patient screening that is led by the patient. Nursing roles can be developed, supported, expanded, or repurposed to involve greater use of patient-reported outcome measures for several key patient outcomes in geriatric cancer care.

Prescriber Attitudes, Experiences, and Proclivities Toward Digital Medicine and How They Influence Adoption of Digital Medicine Platforms.

BackgroundPsychiatric prescribers (prescribers) typically assess medication adherence by patient or caregiver self-report. Despite likely clinical benefit of a new digital medicine technology, the role of specific prescriber attitudes, behaviors, and experiences in the likelihood of adoption is unclear.ObjectiveTo identify prescriber characteristics that may affect adoption of the ingestible event marker (IEM) platform.DesignA survey of prescribers treating seriously mentally ill patients was conducted. Factor analysis was performed on 11 items representing prescriber characteristics believed to be related to endorsement of the IEM platform. Four factors were extracted. Regression analysis was used to test the strength of the relationships between the factors and likelihood of adoption of the IEM platform.ResultsA total of 131 prescribers completed the survey. Most (84%) agreed that visits allow enough time to monitor adherence. Factor analysis revealed four underlying dimensions: 1) perspectives on the value of adherence; 2) concerns about measuring adherence; 3) views toward digital health technologies; and 4) views on payer role/reimbursement. Factors 1 and 3 were related to gender, the belief that computerization benefits prescribers, the presence of office support staff, and the belief that new digital medicine (DM) technology will be cost prohibitive. Willingness to adopt the IEM platform was related to gender (p < 0.05) and perspectives on the value of adherence (p < 0.05), with those scoring higher on that measure also being more likely to adopt.ConclusionPsychiatric prescribers are concerned about medication adherence, perceive current monitoring tools to be problematic, and are open to using digital technologies to improve accuracy of adherence assessment. Relationships among prescriber characteristics, beliefs, and experiences should be considered when developing educational materials, particularly when the goal is to encourage adoption and use of the IEM platform.

Measuring Duchenne muscular dystrophy impact: development of a proxy-reported measure derived from PROMIS item banks

BackgroundPerson-reported outcomes measurement development for rare diseases has lagged behind that of more common diseases. In studies of caregivers of patients with rare diseases, one relies on proxy report to characterize this disability. It is important to measure the child’s disability accurately and comprehensively because it affects caregiver burden. We aimed to create a condition-specific caregiver proxy-report measure for Duchenne Muscular Dystrophy (DMD) in order to understand the impact of DMD on the caregiver. Drawing on relevant item banks from the Patient-Reported Outcome Measurement Information System (PROMIS), we sought to confirm their reliability and validity in the target sample of DMD caregivers.MethodsThis web-based study recruited DMD caregivers via Rare Patient Voice, patient-advocacy groups, and word of mouth. Recruitment was stratified by age of the caregiver’s child with DMD, which broadly represents stages of DMD progression: 2–7, 8–12, 13–17, and > 18. Telephone interviews with DMD parent-caregivers pretested possible measures for content validity. The web-based study utilized an algorithm to categorize respondents’ ambulatory status for tailored administration of PROMIS Parent-Proxy items as well as some new items developed based on caregiver interviews. Item response theory analyses were implemented.ResultsThe study sample included 521 DMD caregivers representing equally the four age strata. The proxy-report measure included the following domains: fatigue impact, strength impact, cognitive function, upper extremity function, positive affect, negative affect, sleep-device symptoms, and mobility. The first five domains had strong psychometric characteristics (unidimensionality; acceptable model fit; strong standardized factor loadings; high marginal reliability). Negative Affect, covering anger, anxiety, depressive symptoms, and psychological stress, fit a bifactor model with good model fit, high marginal reliability, and strong factor loadings. The Sleep-device symptoms domain was not unidimensional, and the mobility domain did not have a simple structure due to residual correlations among items at opposite end of the mobility-disability continuum. These two domain scores were retained as clinimetric indices (i.e., uncalibrated scales), to achieve the overall goal of having a content-valid DMD-specific measure across all stages of disease severity.ConclusionsThe present study derived a DMD-specific proxy-report measure from PROMIS item banks and supplemental items that could potentially be utilized in caregiver research across all stages of the care recipient’s DMD. Future research will focus on assessing the responsiveness and validity of the measure over time and its comparison to DMD patient self-report.

A pilot study of participatory and rapid implementation approaches to increase depression screening in primary care

BackgroundMost individuals with depression go unidentified and untreated. In 2016 the US Preventive Services Task Force released guidelines recommending universal screening in primary care to identify patients with depression and to link them to treatment. Feasible, acceptable, and effective strategies to implement these guidelines are needed.MethodsThis three-phased study employed rapid participatory methods to design and test strategies to increase depression screening at Penn Medicine, a large health system with 90 primary care practices. First, researchers solicited ideas and barriers from stakeholders to increase screening using an innovation tournament—a crowdsourcing method that invites stakeholders to submit ideas to address a workplace challenge. Second, a panel of stakeholders and scientists deliberated over and ranked the tournament ideas. An instant runoff election was held to select the winning idea. Third, the research team piloted the winning idea in a primary care practice using rapid prototyping, an approach that quickly refines and iterates strategy designs.ResultsThe innovation tournament yielded 31 ideas and 32 barriers from diverse stakeholders (12 primary care physicians, 10 medical assistants, 4 nurse practitioners, 2 practice managers, and 4 patient support assistants). A panel of 6 stakeholders and scientists deliberated on the ideas and voted for patient self-report (i.e., through tablet computers, text message, or an online patient portal) as the winning idea. The research team rapid prototyped tablets in one primary care practice with one physician over 5 five-hour shifts to examine the feasibility, acceptability, and effectiveness of the strategy. Most patients, the physician, and medical assistants found the tablets acceptable and feasible. However, patient support assistants struggled to incorporate them in their workflow and expressed concerns about scaling up the process. Depression screening rates were higher using tablets compared to usual care; follow-up was comparable between tablets and usual care.ConclusionsRapid participatory methods engaged and amplified the voices of diverse stakeholders in primary care. These methods helped design an acceptable and feasible implementation strategy that showed promise for increasing depression screening in a primary care setting. The next step is to evaluate the strategy in a randomized controlled trial across primary care practices.

Long-term impact of adult WHO grade II or III gliomas on health-related quality of life: A systematic review

BackgroundGlioma diagnosis can be devastating and result in a range of symptoms. Relatively little is known about the long-term health-related quality of life (HRQOL) challenges faced by these patients. Establishing the impact of diagnosis on HRQOL could help positively tailor clinical decision making regarding patient support and treatment. The aim of this review is to identify the long-term HRQOL issues reported at least 2 years following diagnosis of WHO grade II/III glioma.MethodSystematic literature searches were carried out using Medline, EMBASE, CINAHL, PsycINFO, and Web of Science Core Collection. Searches were designed to identify patient self-reports on HRQOL aspects defined as physical, mental, or social issues. Quality assessment was conducted using the Mixed Methods Appraisal Tool (MMAT). Narrative synthesis was used to collate findings.ResultsThe search returned 8923 articles. Two hundred seventy-eight titles remained after title and abstract screening, with 21 full-text articles included in the final analysis. The majority of studies used quantitative methods, with 3 articles reporting mixed methodology. Negative emotional/psychological/cognitive changes were the most commonly reported. Physical complaints included fatigue, seizures, and restricted daily activity. Social challenges included strained social relationships and financial problems. Patient coping strategies were suggested to influence patient’s survival quality.ConclusionThe consequences of a glioma diagnosis and treatment can have substantial implications for patients’ long-term HRQOL and daily functioning. Findings from this review lay the groundwork for efforts to improve patient HRQOL in long-term survivorship.

Integrating Touchscreen-Based Geriatric Assessment and Frailty Screening for Adults with Acute Myelogenous Leukemia to Drive Personalized Treatment Decisions

Background: Acute Myeloid Leukemia (AML) is a disease of older adults, with a median age of 68 years at diagnosis. The NCCN guidelines recommend comprehensive geriatric assessments (GA) be included in clinical practice to guide treatment decisions. Utility of GA in older AML patients in a real-world environment is not yet established. We tested the feasibility of using a modified GA (mGA), administered by patient self-report on a touchscreen computer, real-time use and utility by clinicians and the correlation of mGA results on treatment decision-making.Methods: 77 newly diagnosed patients were recruited from three sites to complete a tablet-based mGA screening at a treatment decision visit. The mGA includes four domains: age, activities of daily living (ADLs), instrumental ADLs, and comorbidities. Survey results along with history of falls was used to create the Frailty Index (FI). Providers were asked what they thought the fit/frailty status of the patient was before viewing the results on a dashboard. After viewing the survey results, the clinician discussed the treatment plan with the patient. Patients received intensive or non-intensive therapy. Additional information was captured on clinical trial enrollment. Baseline and 3 month surveys recorded presence and severity of 8 symptoms using the Edmonton Symptom Assessment Scale (ESAS) with 0=no symptom and 10=worst possible symptom and quality of life using the Functional Assessment of Cancer Treatment: Leukemia (FACT-LEU).Results: Participants had a median age of 71 years ( range:61-88y); 50% were female, and 87% white. Frailty Index results for 76 patients were 28 (36.4%) fit, 25 (32.5%) intermediate, and 23 (29.9%) frail. (One patient did not complete the mGA). 52 of 77 (69%) enrolled patients were alive at 3 months; 21(27%) died and 4 (5%) were lost to follow-up. Providers were asked the fit/frailty status prior to seeing the results of the mGA. Of 75 provider responses, results were 27 (36.0%) fit, 29 (38.7%) intermediate, and 19 (25.3%) frail. There was 63% (n=47) provider concordance with the mGA result. There was more agreement with fit (n=22, 81.5%) and frail status (n=11, 57.9%) and less with intermediate (n=14, 48.3%). Of the 25 of 75 (33.%) provider reports that indicated that the mGA result influenced the treatment decision, 6 patients (5 fit, 1 intermediate) received intensive treatment, 15 received non intensive treatment (1 fit, 6 intermediate, 8 frail) and 4 enrolled in a clinical trial (1 fit, 2 intermediate, and 1 frail). Significant symptom improvement at 3 months was seen for drowsiness, lack of appetite, shortness of breath, and anxiety. FACT Leu results did not change over 3 months. Providers reported an average of 4.45 minutes to review the dashboard. Patients were able to complete the surveys unassisted in an average time of 16.24 minutes.Discussion: There was nearly 40% discordance between the provider and mGA, with the most discordance on the intermediate fit status. However, results of the mGA influenced treatment decision making in one third of provider/patient interactions. Further analysis of the mGA domains is warranted to see if additional insights can be gained. With time, some symptoms improve and others don't. This points to the opportunity to direct resources towards symptom assessment during treatment. Feasibility was demonstrated in this study as providers received the aggregated results in real time and reviewed them in less than 5 minutes. In addition, patients were able to complete the survey unassisted without disturbing clinic operations. [Display omitted] DisclosuresNo relevant conflicts of interest to declare.

Mobility and Muscle Strength in Recipients of Hematopoietic Cell Transplantation for Sickle Cell Disease: A Preliminary Report from Sickle Transplant Evaluation of Longterm and Late Effects Registry (STELLaR)

IntroductionRecipients of Hematopoietic Cell Transplantation (HCT) are at risk for treatment related late effects including cardiovascular disease and diabetes. Physical fitness including mobility and muscle strength are important predictors of cardiovascular health and quality of life. Physical fitness has been described in children and adults who are long term survivors of HCT for malignant disease [1]. Patients with sickle cell disease (SCD) are likely to be subject to unique morbidity post-HCT as a consequence of the pathophysiology of SCD which is characterized by anemia, inflammation, pain, bone infarcts and neurological complications. A systematic assessment of physical fitness is required to understand some of the long-term outcomes HCT for SCD.MethodsWe enrolled patients with SCD ≥ 1-year post-HCT in Sickle Transplantation Evaluating Long Term And Late Effects Registry (STELLaR). We assessed physical fitness including mobility using the timed up and go (TUG) test, and muscle strength using the handgrip strength (HGS) test. TUG scores were compared with published normative data for children (mean 9.2s; SD = 0.8) and young adults (mean 10.5s, SD = 1.1) [2]. TUG scores ≥ mean+ 2SD were considered abnormal (children: ≥10.8s, young adults ≥12.7s). The HGS of each patient was compared to published normative data of children and adults [3,4]. Patients ≤12 years were abnormal if they had a HGS of two standard deviations below the mean. Patients ≥13 years were considered abnormal if they had a HGS lower than the lower limit of the 85% age-sex standard. Additionally, patients were also assessed for chronic graft versus host disease (cGvHD) by means of the Lee Chronic GvHD Symptom Scale [5]. Patients rated their extent of being bothered by cGvHD symptoms on a scale from 1 to 5, with 1 being not at all and 5 being extremely. These scores were then organized into 7 categories of cGVHD symptoms and normalized to a 0-100 scale. We report the results of an interim analysis of physical fitness in these HCT recipients.ResultsOut of 121 patients enrolled in the study, 37 HCT recipients were assessed for mobility using the TUG test, and for muscle strength using the HGS test. Children were defined as being below the age of 18 and young adults between 18 and 40 years of age. Patient demographic and clinical results for TUG and HGS are presented in Table 1. Young adult participants were more likely to have abnormal TUG and HGS results than pediatric study participants. Patient self-reporting of overall (summary) cGvHD symptoms was found to be statistically significant between children and young adults (p=0.037) with young adults iappearing to be slightly more bothered by GvHD symptoms. There was no correlation in length of time in years between a patient's transplant and whether or not there was abnormal physical fitness (TUG and HGS) for both the young adult and children cohorts.ConclusionsThese data suggest the existence of impaired physical fitness in the long term in SCD patients who are HCT recipients. They provide the rationale for systematic evaluation of physical fitness and other risk factors for cardiovascular disease post-HCT.

Long-Term Quality of Life after Hematopoietic Cell Transplantation for Sickle Cell Disease: A Preliminary Report from the Sickle Cell Transplant Evaluation of Long Term and Late Effects Registry (STELLAR)

▪Introduction: Sickle cell disease (SCD) is characterized by unpredictable, recurrent acute and chronic persistent pain, and progressive organ damage leading to substantial morbidity, impaired quality of life and increased healthcare utilization. Hematopoietic cell transplantation (HCT) is the only therapy for SCD with curative intent. HCT is associated with excellent overall and event free survival especially in young children undergoing HCT from HLA identical family donors. Given these outcomes and improved supportive care, HCT is increasingly being offered to adults with SCD. However, a paucity of data exists on health related quality of life (HrQoL) in patients who are long term survivors of HCT. Therefore, a clear need exists to study HrQoL in children and adults post HCT to determine the long term benefit of HCT for SCD. We report the results of an interim analysis of HrQoL in recipients of HCT for SCD.Methods: We enrolledchildren and adults with SCD ≥ 1-year post-HCT in the Sickle Cell Transplant Evaluation of Long Term and Late Effects Registry (STELLAR). We obtained patient self-report of health status and health practices using a web based platform. Individuals under age 18 years and/or proxies completed Patient Reported Outcome Measurement Information System (PROMIS) version 25. Individuals over age 18 completed PROMIS version 29. Surveys were completed at enrollment and annually thereafter. T-scores were calculated for each survey domain. HrQoL in children (PROMIS25) and adults (PROMIS29) was compared to age appropriate norms (mean 50 ± 10). Univariate analysis was performed by age, gender, marital status, time from transplant, history of preHCT stroke, frequency of pain, number of emergency department (ED) visits, length of stay, income, and education assessed at the time of PROMIS survey administration. Statistical analysis was conducted using SAS Version 9.4. Descriptive statistics for each variable were reported. For numeric covariates, the mean and standard deviation (SD) were calculated and presented with frequency and its percentage shown for category variables. For repeated measures, generalized estimating equation (GEE) model was performed to detect the association. The significance level was set at 0.05.Results: Twenty-seven individuals completed 30 PROMIS25 surveys and 15 individuals completed 19 PROMIS29 surveys from 2017-2021. The mean age at transplant was 11.4years (SD 3.1) and 23.8 years (SD 6.2) in adults, respectively. Sixty percent and 50%, respectively, were female. The mean time from transplant was 4.2 years (SD 3.9) and 9.1 years (SD 7.1), respectively. Physical function/mobility, anxiety, depression, fatigue, and pain T-scores for each PROMIS measure are reported below in Table 1. Univariate analysis of PROMIS25 T-scores showed a statistically significant difference in anxiety by age and peer relationships by age, gender, and time from transplant. Time from transplant also had a statistically significant association with pain interference. PROMIS29 T-scores also showed a statistically significant difference in anxiety by age. Gender had a statistically significant association with depression and fatigue. Number of ED visits had a significant association with physical function, depression, social participation, and pain interference (Table 2). Other covariates lacked a statistically significant association or had insufficient sample sizes for comparison.Conclusions: Long-term HrQoL after HCT for SCD is within population norms for adults and children, though children have better HRQoL compared to adults. Better scores in females suggests the need for study of gender as a mediator of HrQoL outcomes. The observed relationship between HrQoL domains and education, income, time from transplant, and number of ED visits, a surrogate for healthcare utilization, suggest the need for careful study of these and other factors that can influence HrQoL post HCT over the long-term. In addition, further research in patient reported outcomes has the potential to identify needed areas of intervention and surveillance for this population. [Display omitted] DisclosuresGuilcher: Project Sickle Cure Study: Other: Principal Investigator, Research Funding; BlueBirdBio: Research Funding.

General and Disease-Specific Factors Associated with Health Related Quality of Life Among Patients With Peripheral Artery Disease to Inform Treatment Selection in the Era of Patient-Reported Outcomes

<h3>Introduction and Objective</h3> Patient-reported outcomes (PRO) have been championed for assessment of peripheral artery disease (PAD) severity and treatment outcomes. Limited evidence exists regarding factors associated with PRO that may affect their sensitivity to change with PAD interventions. We performed a prospective, cross-sectional study to evaluate how patient-level factors are associated with a validated PAD-specific health related quality of life (QOL) instrument, the VascuQol-6. <h3>Methods</h3> Patients were recruited from a diagnostic vascular unit based on referral for ankle brachial index (ABI) testing. All participants were assessed for PAD symptoms using the San Diego Claudication Questionnaire and tested for neuropathy using the Michigan Neuropathy Screening Instrument (MNSI). PAD-specific QOL was assessed using the VascuQol-6. Clinical data was collected from the medical record and patient-self report. Factors associated with QOL were evaluated using ANOVA, confidence intervals, and linear models. <h3>Results</h3> 70 patients were recruited and participated. Mean age was 67.8±11.7 years (Figure 1Figure 1Plot of age versus VascuQOI-6 summary score (P=0.011).Figure 1); 41% of participants were women and 87.1% were white, and 29% were current smokers. Comorbid conditions included diabetes (23.2%) and peripheral neuropathy (44.3%). Mean ABI was 0.77±0.29 and mean TBI was 0.48±0.23. PAD symptoms were classified as claudication in 48.6%, CLTI in 31.4%, and asymptomatic in 20%. Among symptomatic patients, 21 had multi-level symptoms, 13 had calf symptoms only, 4 had buttock symptoms only, and 3 had thigh symptoms (Figure 2Figure 2Distribution of Vascuqol-6 summary scores by anatomic symptom distribution (P=0.025).Figure 2). Symptoms were bilateral in 55 patients (78.6%). Patients with multi-level disease had lower QOL scores (13.8±4.0; 90% CI 12.3-15.3) versus those with no symptoms (17.6±4.8; 90% CI 16.1-19.2), calf symptoms (17.8±3.1; 90% CI 16.2-19.3) (P=0.024). In multivariable models QOL was associated with minimum ABI (P=0.020), symptom anatomic level (P=0.002) and age (P=0.001); a trend was also observed for symptom category (P=0.121). No associations between QOL and smoking status, diabetes, neuropathy, or bilateral versus unilateral symptoms were observed. <h3>Conclusions</h3> PAD-specific QOL assessments are associated with factors related and unrelated to PAD. Higher QOL scores among older participants was unanticipated and may impart ceiling effects that limit potential for improvement through PAD interventions in this subgroup, while patients with multi-level symptoms may have greater potential gains from PAD treatment. Future work following this cohort will explore changes in QOL over time and associations with treatment interventions, adverse events, and patient-level factors.