Patient Reported Outcomes Measurement Information System T-scores Research Articles

Longitudinal Patient-Reported Outcomes (PROs) in a Prospective Registry of CAR T-Cell Therapy Recipients

Background Chimeric antigen receptor T-cell therapies (CAR-T) significantly improve survival and health-related quality of life (HRQoL) in clinical trials of relapsed hematologic malignancies. However, the impact of CAR-T on real-world longitudinal patient-reported outcomes (PROs), particularly in the immediate post-CAR-T period during which patients are hospitalized and at risk for acute CAR-T-related toxicities, has not been studied. Further, few CAR-T studies have utilized Patient-Reported Outcomes Measurement Information System (PROMIS) survey instruments, which are recommended to enable comparison of PROs across various CAR-T products and disease types. To characterize real-world HRQoL and symptom burden in patients receiving CAR-T, we integrated PROMIS PRO instruments into a prospective cellular therapy registry. Methods The University of Chicago Cellular Therapy Biobank prospectively administers the PROMIS v1.2 Global Health and PROMIS-29 Profile v2.1 instruments to CAR-T recipients. PRO instruments are administered at the following timepoints, anchored to the day (D) of CAR-T receipt: baseline (at time of apheresis), D+1, D+15, D+28, D+90, D+180, and then every 3 months until the 2-year timepoint. The Biobank was queried for all patients who completed a baseline and at least one post-CAR-T survey. PROMIS T-scores were analyzed by one-way repeated measures ANOVA up to the 12-month timepoint. PROMIS T-scores were longitudinally visualized as mean change from baseline. Minimally important differences (MID) that were clinically significant were defined as T-score changes of at least 2. Statistical analyses were performed in STATA, version 17.0. Results There were 68 patients available for PRO analysis. Median age was 65 years (range, 24-80) and 63% were male. Most patients had large B-cell lymphomas (n = 27, 40%) or multiple myeloma (n = 27, 40%). Median prior lines of therapy were 4 (range, 1-12), with only 3 patients receiving 1 prior therapy. The majority of patients received CD19-targeted products (n = 35, 51%), followed by BCMA-targeted products (n = 27, 40%). Patients lived a median of 36 miles from the CAR-T center (range, 2-544), with 65% living over 30 miles away. Survey completion rates at timepoints were as follows: 73% at D+15, 82% at D+28, 84% at D+90, 76% at D+180, and 95% at 12 months. For PROMIS Global Health, there was a significant longitudinal change in Global Physical Health (p = .002), with a clinically meaningful improvement by D+28 which was no longer observed by D+90 ( Figure 1), but no significant change in Global Mental Health (p = .09). For PROMIS-29 Profile, there were significant longitudinal changes in physical function (p = .0001), fatigue (p = .001), and social role functioning (p = .002), but not anxiety (p = .08), depression (p = .45), sleep (p = .92), or pain (p = .27). There were clinically meaningful improvements in both physical and social role functioning in the first 28 days after CAR-T which were no longer observed by D+90 ( Figure 1). Fatigue significantly worsened in the first 28 days after CAR-T to a clinically meaningful degree, but subsequently improved to better than baseline after D+90 ( Figure 2). With a median follow-up of 9 months (range, 1-28), 40% of patients had post-CAR-T disease progression. Relapses occurred a median of 4.9 months after CAR-T, with 31% of relapses (n = 8) occurring before D+90. There were 10 deaths (15%) at a median of 6.7 months after CAR-T. Conclusions In this real-world prospective PRO analysis, there were clinically meaningful improvements in several HRQoL domains in the first month after CAR-T, namely physical and social functioning, despite significantly worsening fatigue during the same time period. However, these HRQoL improvements diminished after D+28, concurrent with the transition of patients from the CAR-T center to home. These results suggest that intensive support by the inpatient and outpatient care teams may improve physical HRQoL during the immediate post-CAR-T period. In addition, given the low rate of relapses before D+90 in this cohort, worsening patient-reported functioning after D+28 may be driven by changes in care support after discharge to home. Interventions to optimize the post-CAR-T transition to home are warranted, as well as studies utilizing PROMIS PROs to identify patients with deteriorating HRQoL who may benefit from augmented support and/or closer surveillance for relapsed disease.

Feasibility of Acupuncture and Exploration of Metabolomic Alterations for Psychoneurological Symptoms Among Breast Cancer Survivors.

Approximately 24-68% of breast cancer survivors report co-occurring psychoneurological symptoms of pain, fatigue, sleep disturbance, depression, and anxiety during and after cancer treatment. This study aimed to assess the feasibility and acceptability of acupuncture for the treatment of multiple psychoneurological symptoms among breast cancer survivors and explore metabolomic changes before and after acupuncture. We conducted a single-arm, prospective pilot study of breast cancer survivors with at least two moderate to severe psychoneurological symptoms (>3 on a 0-10 scale). Acupuncture was administered twice weekly for 5weeks, for 30minutes per session. Along with Patient-Reported Outcomes Measurement Information System (PROMIS) questionnaires, a fasting serum comprehensive hydrophilic metabolites panel was analyzed at baseline and after acupuncture. Eight participants (mean age 52.5 ± 10.9years; 62.5% Black) were enrolled. Feasibility was supported, with 67% recruitment, 87.5% retention, and 98% acceptability. Post intervention, PROMIS T-scores were reduced for all psychoneurological symptoms. Significant differences in serum metabolites before and after acupuncture were F-1,6/2,6-DP, glutathione disulfide, phosphorylcholine, 6-methylnicotinamide, glutathione, and putrescine (variable importance of projection values larger than 1.5 and p values <0.05). Pathway analysis indicated that glutathione metabolism (p = 0.002, q = 0.071), and arginine and proline metabolisms (p = 0.009, q = 0.166) were potentially involved in mechanisms of acupuncture. Acupuncture to reduce multiple psychoneurological symptoms among breast cancer survivors was feasible and acceptable. Study findings also shed light on the metabolic pathways involved in the acupuncture response and will be tested in future studies.

Can We Utilize PROMIS Neuropathic Pain Quality for Assessment of Neuropathic Heel Pain?

Category: Hindfoot; Other Introduction/Purpose: Heel pain is one of the most common presentations encountered by foot and ankle specialists. While most etiologies of heel pain such as plantar fasciitis are mechanical stress driven nociceptive pain, a combined neuropathic mechanism may contribute to worse outcomes and challenges in pain management for a subset of these patients. Diagnosis and management of neuropathic pain in foot and ankle patients remain challenging. Since neuropathic pain may play an important part in indicating surgery or counseling heel pain patients, we investigated the plausibility of utilizing the Patient-Reported Outcomes Measurement Information System (PROMIS) Neuropathic Pain Quality (PQN) as an initial screening tool to diagnose and monitor the clinical effects of treatment. Secondary outcomes were prevalence of neuropathic pain and associated risk factors. Methods: New patients presenting to a tertiary care academic institution with heel pain were prospectively recruited. PROMIS PQN t-scores were obtained from all patients to detect and determine the severity of NP. Patients were grouped into no neuropathic pain (NP), mild neuropathic pain (NP), and severe NP based on the initial PROMIS PQN t-scores. Pain Interference (PI), Physical Function (PF), and Self-Efficacy (SE) scores were assessed at baseline, 30, and 90-day follow-up timepoints. Other factors such as age, smoking, body mass index (BMI), low back/neck pain, anxiety, depression, and medications were analyzed for association with outcomes. Linear mixed modeling was utilized to assess the main effects of time and NP on PROMIS t-scores, and meaningful clinically important difference (MCID) was compared. Results: A total of 48 patients with mean age of 52.4+-12.5 years old were recruited at the initial visit. The cohort consisted of 35 (73%) female patients, 7 (15%) diabetics, 6 (13%) current smokers, 18 (38%) with low back/neck pain, 6 (13%) with anxiety, 15 (31%) with depression, and 9 (19%) had taken neuromodulator, such as gabapentin. When using the PROMIS PQN as the assessment tool, 33 patients (69%) had NP at baseline; 23 (48%) with mild, and 10 (21%) with severe NP. BMI was the only significant independent factor associated with NP (p = 0.011). Higher baseline NP was significantly associated with higher PQN (p&lt;0.001) and PI (p=0.005), and lower SE (p=0.04) across timepoints. Patients who were detected to have NP based on the PQN t-score had lower PF at baseline with significantly less improvement in PF (3 vs 9.9, p=0.035), and did not meet MCID (Figure 1). Conclusion: Prevalence of neuropathic pain in patients presenting with heel pain was high. Neuropathic pain at baseline was significantly associated with worse pain and self-efficacy over time, and less clinically meaningful improvements in function. The PROMIS PQN may serve as a valuable tool to identify and guide clinical treatment decision pathways for patients with neuropathic heel pain.

Detection of changes of functioning over time after asthma exacerbation in children with the use of PROMIS domains

Objectives Patient reported outcome measures, such as the Patient Reported Outcomes Measurement Information System (PROMIS) may be utilized to understand experiences of patients. The purpose of this study was to determine the ability of PROMIS domains to detect changes in pain, physical functioning, and asthma impact over time for children experiencing asthma exacerbation. Methods Our prospective cohort study included children presenting to the emergency department (ED) for asthma exacerbation. Children completed PROMIS surveys in the ED, 7–10 days, and 1–3 months post-discharge. We used linear mixed models adjusted for age, gender, acute care utilization, and child global health to determine changes in PROMIS T-scores. We used self-reported child health response (Much better now versus a little better now or worse) at discharge as an anchor to determine if change in PROMIS scores corresponded with changes in health. A change was statistically significant if the 95% CI did not include 0. Results Our study included 63 children who presented to the ED for acute asthma exacerbation. We identified that children improved significantly in all domains over time. There was improvement over time following discharge from ED for all pain and physical functioning domains, and asthma impact. Using the clinical anchor, those with considerable improvement in asthma symptoms had improved T scores from 4–17. Conclusions PROMIS domains of pain, physical functioning, depression, fatigue, peer relationships, and asthma impact are responsive to changes in health states over time. These domains may be used to measure clinically significant change in children experiencing asthma exacerbation.

Prediction of Hypoglycemia By Quality of Life Measures: A Retrospective Analysis of Electronically Patient-Reported Data

We aimed to examine the utility of electronically patient-reported data (e-PRD) in exploring the risk of diabetes-related hypoglycemia and to evaluate hypoglycemia prediction by the quality of life (QoL) measures. A retrospective analysis of e-PRD for patients with diabetes mellitus who completed the American Diabetes Association's Low Blood Sugar Questionnaire (Hypo-Q) and the Patient-Reported Outcomes Measurement Information System (PROMIS) for QoL assessment. Associations between Hypo-Q answers and PROMIS scores were described using Spearman correlations and 95% confidence intervals, or medians and quartiles. Records of 538 subjects were reviewed; 55% were female, 95% were non-Hispanic, with a mean age (± SD) of 53 ± 15 years. Patients with type 1 diabetes had a longer disease duration and more hypoglycemic episodes (P<.001) with higher PROMIS Physical and Mental T-scores (P<.001, both), when compared to patients with type 2 diabetes. The latter had a higher number of co-existing conditions. Having >5 episodes of either moderate or severe hypoglycemia in a year were reported by 18% and 5% of all patients, respectively. Mean PROMIS Physical and Mental health T-scores were 46 ± 10 and 47 ± 10, respectively. Patients with fewer moderate and severe hypoglycemic episodes had better Physical (P = .047 and P<.001) and Mental (P = .015 and P<.001) PROMIS T-scores with incremental decreases in the odds of hypoglycemia with each point increase in PROMIS T-scores. e-PRD of QoL measures and Hypo-Q were effective in exposing the risks for hypoglycemia and reproducing published findings with significant associations between QoL measures and hypoglycemia risks while providing new insights.

Abstract P2-12-08: Triaging breast care center patients to supportive care services based on the Athena Breast Health Network online intake form and the patient-reported outcomes measurement information system (PROMIS)

Abstract Background: The Athena Breast Health Network is a multi-site effort aimed at integrating cutting-edge research and clinical care for breast cancer screening and treatment. At the University of California, San Francisco (UCSF) Breast Care Center (BCC), an Athena-based electronic Health Questionnaire System (eHQS) is distributed to all new patients. This questionnaire assesses self-reported personal and familial health history, comorbidities, and Patient-Reported Outcome Measurement and Information System (PROMIS) quality of life domains. Based on these responses, patients are proactively triaged to genetic counseling, psycho-oncology, onco-fertility, smoking cessation, peer support, nutritional counseling, behavioral sleep, and social work. These referrals are based on pre-defined thresholds, such as Ashkenazi Jewish Ancestry (referral to Genetic Counseling), or PROMIS Depression and Anxiety scores greater than or equal to 9 and 11, respectively. This analysis aims to assess the eHQS as a means to refer patients to supportive care services and assess if high-need patients (those with the highest symptom burden as assessed by quality of life PROMIS scores) are referred to services more frequently. Patients and Methods: Since Athena’s launch in 2013, over 6,000 patients have completed the online intake questionnaire at the UCSF BCC, 4,984 of whom have consented to research. 3,627 patients completed all relevant data to this study and were used in our analysis. A review of patient referrals was conducted to assess the number of referrals as a result of eHQS completion. PROMIS T-Scores in 8 domains (depression, anxiety, fatigue, sleep-related impairment and disturbance, cognitive function, applied cognition and physical function) were calculated with the Health Measures system and compared across patients stratified by age and cancer type and against the US general population. Results: When compared to the US population as a whole, PROMIS scores indicate that UCSF BCC patients have impaired quality of life in every assessed PROMIS domain except for depression. As age increases, PROMIS scores across multiple domains (depression, anxiety, sleep disturbance, cognitive function) increase, signifying lower symptom burden. Importantly, high symptom burden in the anxiety PROMIS domain was suggestive of lower quality of life scores in 6 out of the other 7 assessed PROMIS domains. 39% (1408 patients) of all analyzed BCC patients received at least one referral to a supportive care service. 721 of 1450 (50%) patients with an Invasive Breast Cancer (IBC) diagnosis and 246 of 571 (43%) patients with a DCIS diagnosis triggered at least one referral. 31% of all patients received a referral to genetic counseling, 16.3% to psycho-oncology, 11% to behavioral sleep, 8% to nutritional counseling, 7.6% to social work, 2.4% to smoking cessation, and 1.6% to onco-fertility. When compared to patients with DCIS or without a cancer diagnosis, patients with IBC are most likely to receive a referral to genetic counseling and psychosocial services. Referrals to supportive care services are less frequent as age increases. Conclusion: The Athena online intake form at the UCSF BCC effectively triages patients with high levels of anxiety (PROMIS &amp;gt; 8) and depression (PROMIS &amp;gt;10) to supportive care services. Preliminary results regarding the correlation between anxiety and overall poor quality of life suggest that referrals to psycho-oncology may be most efficacious in decreasing symptom burden. Using these analyses, we aim to refine our use of the eHQS to tailor the most relevant and effective referrals to the highest-need patients. Citation Format: Madeline Matthys, André Dempsey, Amrita Basu, Emily Wong, Nickolas Dreher, Irene Acerbi, Suzanne Ackerson, Laura Esserman, Athena Breast Health Network Investigators and Advocate Partners, Michelle Melisko, Laura van 't Veer. Triaging breast care center patients to supportive care services based on the Athena Breast Health Network online intake form and the patient-reported outcomes measurement information system (PROMIS) [abstract]. In: Proceedings of the 2019 San Antonio Breast Cancer Symposium; 2019 Dec 10-14; San Antonio, TX. Philadelphia (PA): AACR; Cancer Res 2020;80(4 Suppl):Abstract nr P2-12-08.

Utilizing the patient-reported outcomes measurement information system (PROMIS) to assess quality of life among breast cancer patients at an academic center.

e23171 Background: NCI PROMIS assesses physical, mental and social health across multiple health conditions. Despite interest in using PROMIS in oncology trials and clinical care, reference values in cancer are not well established. Methods: Electronic intake questionnaires assessing self-reported history and 8 PROMIS domains (depression, anxiety, fatigue, sleep-related impairment (SRI), sleep disturbance, cognitive function, applied cognition and physical function) are sent to all new patients (pts). 2886 pts with self-reported breast cancer completed the questionnaire/consented to research. PROMIS T-Scores were calculated using the Health Measures system. Results: For all domains except depression, cognitive function and applied cognition, pts had clinically meaningful ( &gt; 3 point diff.) worse mean PROMIS scores than US means (50). Compared to stage 0-III, stage IV pts had worse scores for all assessed domains (p &lt; 0.05). Among Stage IV pts &lt; 49, QoL was significantly worse in 7/8 domains (see table). Across all age groups, there were significant differences in mean PROMIS scores for Stage IV pts in anxiety, depression, cognitive function and applied cognition (p &lt; 0.05). Conclusions: PROMIS scores among pts indicate impaired QoL in multiple domains. These scores provide insight into the physical, mental and social health of our pts and can serve as robust estimates for breast cancer trials and clinical care. [Table: see text]

Electronically self-assessed functional capacity and exercise testing: A comparison of the Duke Activity Status Index and Patient-Reported Outcomes Measurement Information System tools

Electronic screening tools, such as Patient-Reported Outcomes Measurement Information System (PROMIS) Physical Function Short-Form 12a (PF-SF12a), may aid in the assessment of functional capacity. However, PROMIS PF-SF12a has not been validated against exercise capacity, or compared with established questionnaires, including the Duke Activity Status Index (DASI). We compared the DASI and PROMIS PF-SF12a to the maximum metabolic equivalents (METs) achieved during exercise stress testing. DASI and PROMIS PF-SF12a were electronically administered to 100 adult patients (median age 56years, 61% male) immediately before exercise stress testing. DASI-predicted METs and PROMIS T score were calculated. Correlations with exercise METs with and without age adjustment were examined. Linear regression lines were derived and adjusted r2 statistic was calculated. We compared models with the Davidson-Mackinnon J test. The median (interquartile range) DASI-predicted METs, PROMIS Tscore, and exercise METs were 8.97 (7.61-9.89), 47.90 (43.33-52.40), and 10.10 (10.10-12.80), respectively. In unadjusted correlation analyses, PROMIS accounted for 26% of the variance in exercise METs compared with 38% with DASI. With age adjustment, the r2values increased to 0.36 (PROMIS) and 0.46 (DASI). In both unadjusted and age-adjusted analyses, inclusion of DASI improved prediction of exercise METs beyond PROMIS T score (P<.0001). In contrast, PROMIS T score did not improve exercise MET prediction compared with DASI alone (P>.10). Among patients undergoing clinically indicated exercise stress testing, DASI outperformed PROMIS PF-SF12a as a predictor of exercise METs.

Obesity and symptoms and quality of life indicators of individuals with disabilities

BackgroundHealth risks of obesity are well known, but effects of obesity on health-related quality of life (HRQOL) have not been well-studied in people with physical disabilities. Objective/hypothesisWe examined the association between obesity and HRQOL in people with disabilities relative to the general US population. We hypothesized (a) overall, individuals with disabilities will report worse HRQOL than the general US population and (b) obese individuals with disabilities will report worse HRQOL than non-obese individuals. MethodsIndividuals with muscular dystrophy, multiple sclerosis, post-polio syndrome, and spinal cord injury (N = 1849) completed Patient Reported Outcomes Measurement Information System (PROMIS) measures of fatigue, pain interference, physical and social function, depression, sleep disturbance, and sleep-related impairment. Participants were classified as obese or non-obese based on self-reported weight and height (BMI) and/or waist circumference (WC). PROMIS T-scores were compared to norms and between obesity groups. ResultsMean BMI was 26.4 kg/m2 with 23.4% classified as obese. Mean WC was 37.5 inches (males) and 34.0 inches (females); 26.4% reported abdominal obesity. Based on BMI and/or WC, 33.3% (n = 616) were classified obese. Compared to PROMIS norms, obese individuals reported worse HRQOL on all domains (p < 0.0001). Compared to non-obese individuals, obese individuals reported worse functioning on all domains except depression (p < 0.01). Obese individuals with MS and MD reported worse outcomes than non-obese counterparts. ConclusionsObesity in people with physical disabilities is associated with poorer HRQOL. More research is needed to inform clinicians in identifying obese patients and helping them achieve healthy weight, reduce symptom burden, and improve QOL.