Patient-reported Outcomes Responses Research Articles

Patients with High Baseline Neutrophil-to-Lymphocyte Ratio Exhibit Better Response to Filgotinib as Treatment for Rheumatoid Arthritis.

High baseline neutrophil-to-lymphocyte ratio (NLR) in rheumatoid arthritis (RA) has been associated with positive responses to biologic tumor necrosis factor inhibition and negative responses to conventional synthetic disease-modifying antirheumatic drug (csDMARD) triple therapy. Datasets from three randomized clinical trials in patients with RA were used to test the hypothesis that baseline NLR is associated with improved clinical response to filgotinib in methotrexate (MTX)-naïve or MTX-experienced RA populations. Patients from FINCH 1 (inadequate response to MTX, MTX-IR; NCT02889796), FINCH 2 (inadequate response to biologic DMARDs; NCT02873936), and FINCH 3 (MTX-naïve; NCT02886728) were classified as baseline NLR-High or baseline NLR-Low based on a previously published cut point of 2.7. In total, 3365 patients were included across the three studies. Differences in clinical outcomes and patient-reported outcomes (PROs) were determined using linear-regression models. Control-arm patients (placebo + MTX/placebo + csDMARD) classified as NLR-High exhibited worse continuous clinical and PRO responses at week 12 across clinical trials compared to NLR-Low patients. In contrast, NLR-High patients who received FIL 200mg + MTX/csDMARD exhibited consistently better responses after 12weeks compared to NLR-Low patients across clinical trials, clinical endpoints, and PROs. These trends were most prominent among the MTX-IR population. The 2.7 baseline NLR cut point could be used to enrich for patients most likely to benefit from the addition of filgotinib to background MTX/csDMARD. Use of baseline NLR as part of therapeutic decision-making would not require additional diagnostics and could contribute to improved outcomes for patients with RA. Clinicaltrials.gov: NCT02889796; NCT02873936; NCT02886728.

Efficacy and Safety of Tofacitinib in Ankylosing Spondylitis by Baseline C-Reactive Protein Level: Post Hoc Analysis of Phase II and Phase III Clinical Trials.

This post hoc analysis assessed the effect of baseline C-reactive protein (CRP) on the efficacy and safety of tofacitinib (TOF) use in ankylosing spondylitis (AS), as well as patient-reported outcomes (PROs). Phase II (ClinicalTrials.gov: NCT01786668) and phase III (ClinicalTrials.gov: NCT03502616) data from patients with active AS were used. Endpoints (weeks 12, 16, and 48), including 20% and 40% improvement in Assessment of SpondyloArthritis international Society (ASAS), AS Disease Activity Score with CRP low disease activity, 50% improvement in Bath AS Disease Activity Index (BASDAI50), and PROs (pain and fatigue), were stratified by baseline CRP (mg/L) as follows: < 5 (normal), ≥ 5 (elevated), < 10, and ≥ 10. Safety outcomes were evaluated between < 5 and ≥ 5 mg/L subgroups. Overall, 372 patients were included (69.6% ≥ 5mg/L; 50.8% ≥ 10 mg/L). At baseline in the < 5mg/L group, more placebo-treated than TOF-treated patients received concomitant nonsteroidal antiinflammatory drugs (NSAIDs) or sulfasalazine (SSZ). Week 12 efficacy and PRO responses were generally higher for TOF vs placebo, regardless of baseline CRP. The treatment effect (placebo-adjusted response) at week 12 was generally numerically higher in ≥ 5 mg/L and ≥ 10 mg/L vs < 5 mg/L and < 10 mg/L groups. Incidence rates for treatment-emergent adverse events (TEAEs) and "all infections" were numerically higher for TOF vs placebo in patients in the < 5 mg/L group, but similar for TOF vs placebo in patients in the ≥ 5 mg/L group. Regardless of baseline CRP, TOF was more efficacious vs placebo at week 12. The placebo-adjusted efficacy and PRO responses were generally numerically higher in patients with CRP ≥ 5 mg/L and ≥ 10 mg/L vs < 5 mg/L and < 10 mg/L. The higher concomitant NSAID/SSZ exposure may have improved efficacy responses in the baseline < 5 mg/L placebo group, and ultimately affected the TOF treatment effect. Safety was consistent with previous studies of TOF use in AS, with numerically higher incidence rates for TEAEs and "all infections" for TOF vs placebo in patients with CRP < 5 mg/L.

Association between enthesitis/dactylitis resolution and patient-reported outcomes in guselkumab-treated patients with psoriatic arthritis

ObjectivesTo evaluate the association between enthesitis resolution (ER) and dactylitis resolution (DR) and meaningful improvements in patient-reported outcomes (PROs) among biologic-naïve patients with PsA receiving guselkumab in the DISCOVER-2 study.MethodsEnthesitis and dactylitis, characteristic lesions of PsA, were evaluated by independent assessors using the Leeds Enthesitis Index (range, 0–6) and Dactylitis Severity Score (range, 0–60). Proportions of patients with ER or DR (score = 0) among those with score > 0 at baseline were determined at weeks 24, 52, and 100. PROs included: fatigue (Functional Assessment of Chronic Illness Therapy-Fatigue [FACIT-Fatigue]), pain (0–100 visual analog scale), physical function (Health Assessment Questionnaire-Disability Index [HAQ-DI]), and health-related quality of life (36-item Short-Form Health Survey physical/mental component summary [SF-36 PCS/MCS]). Meaningful responses were defined as: improvements of ≥ 4 for FACIT-Fatigue, ≥ 0.35 for HAQ-DI, and ≥ 5 for SF-36 PCS/MCS and absolute scores of ≤ 15 for minimal pain and ≤ 0.5 for normalized HAQ-DI. Associations between ER/DR status and PRO response status were tested using a Chi-square test.ResultsGuselkumab-treated patients with ER were more likely than those without ER to achieve minimal pain (p < 0.001), normalized HAQ-DI (p < 0.001), and PCS response (p < 0.05) at weeks 24, 52, and 100. Patients with DR were more likely than those without DR to achieve FACIT-Fatigue response at week 24 and week 52 (both p ≤ 0.01) and minimal pain at week 24 and normalized HAQ-DI at week 52 (both p ≤ 0.03).ConclusionIn biologic-naïve patients with active PsA treated with guselkumab, achieving ER or DR was associated with durable improvements in selected PROs, including those of high importance to patients.Trial registrationClinicalTrials.gov (https://clinicaltrials.gov) NCT03158285; Registered: May 16, 2017.Key Points• At week 100, 65% and 76% of guselkumab-treated patients achieved enthesitis and dactylitis resolution (ER/DR).• Achieving ER was associated with achieving DR and vice versa through the end of study.• Achieving ER or DR was associated with durable and meaningful improvements in selected patient-reported outcomes.

The Effect of JAK Inhibitors on Patient-Reported Outcomes in Psoriatic Arthritis.

Psoriatic arthritis (PsA) is a chronic inflammatory disease that affects the joints and skin of patients with psoriasis. In this review we aimed to summarise the available evidence regarding the effect of Janus kinase inhibitors (JAKi) on patient-reported outcomes (PROs) when used for the management of PsA. We utilised a narrative review approach as we searched the available literature for articles to be included in our study. JAKi have been found to be effective in inducing better PRO responses compared to placebo. These findings have been consistent across various patient populations, including those with active PsA, those with an inadequate response to conventional therapies, and those with comorbidities. The evidence supporting the benefits of JAKi on PROs in PsA is compelling, demonstrating consistent improvements in pain, physical function, fatigue, and quality of life. Numerous studies have demonstrated the the efficacy of JAKi in improving PROs in patients with PsA.

Treatment of Severely Shortened or Comminuted Clavicular Fractures in Older Adolescent Athletes

Background: Recent evidence suggests that for completely displaced midshaft clavicular fractures, surgery offers no clear benefit over nonoperative treatment in a general adolescent population from 10 to 18 years of age. However, the comparative outcomes of comminuted and/or severely shortened clavicular fractures specifically in older adolescent athletes have not been explored in a focused, methodologically rigorous fashion. Hypothesis: The study hypothesis was that outcomes would be superior in older adolescent athletes who underwent operative treatment compared with nonoperative treatment for comminuted and/or severely shortened clavicular fractures. Study Design: Cohort study; Level of evidence, 2. Methods: A level 2, multicenter, prospective cohort study investigating the outcomes of midshaft fractures in adolescents between 2013 and 2017 was filtered to analyze the subcohorts of athletes 14 to 18 years of age with either fracture comminution or fracture shortening of ≥25 mm or both. Patient characteristics, injury mechanisms, fracture characteristics, and treatments were compared. Complications, rates, timing of return to sports (RTS), and patient-reported outcomes (PROs) were analyzed. Results: The 2 treatment groups, which included 136 older adolescent athletes (69 nonoperative, 67 operative), showed similar distributions of primary sport type, competition level, comminution, shortening, and 2-year PRO response rate (n = 99; 73%). The operative group demonstrated 3 mm–greater mean superior displacement, which was therefore statistically controlled for as a confounder in the comparative PRO analysis. No 2-year differences in nonunion, delayed union, symptomatic malunion, refracture, clinically significant complications, or rates of RTS were detected between treatment groups. The difference in timing of RTS (operative, 10.3 weeks; nonoperative, 13.5 weeks) was statistically significant. After controlling for the minor difference in superior displacement, regression analysis and matched comparison cohorts demonstrated no differences between the nonoperative and operative groups in mean or dichotomized PRO scores. Conclusion: In this prospective, multicenter cohort study investigating older adolescent athletes with comminuted and/or severely shortened clavicular fractures, contrary to the study hypothesis, there were no differences in complications, RTS, or PROs between nonoperatively and operatively treated patients at 2 years. Comparably excellent outcomes of severe clavicular fractures in adolescent athletes can be achieved with nonoperative treatment.

Providing meaningful interpretation of performance outcome measures by co-calibration with patient-reported outcomes through the Rasch model: illustration with multiple sclerosis measures

ABSTRACT Performance outcome (PerfO) measures are based on tasks performed by patients in a controlled environment, making their meaningful interpretation challenging to establish. Co-calibrating PerfO and patient-reported outcome (PRO) measures of the same target concept allow for interpretation of the PerfO with the item content of the PRO. The Rasch model applied to the discretized PerfO measure together with the PRO items allows expressing parameters related to the PerfO measure in the PRO metric for it to be linked to the PRO responses. We applied this approach to two PerfO measures used in multiple sclerosis (MS) for walking and manual ability: the Timed 25-Foot Walk (T25FW) and the 9-Hole Peg Test (9HPT). To determine meaningful interpretation of these two PerfO measures, they were co-calibrated with two PRO measures of closely related concepts, the MS walking scale − 12 items (MSWS-12) and the ABILHAND, using the data of 2,043 subjects from five global clinical trials in MS. The probabilistic relationships between the PerfO measures and the PRO metrics were used to express the response pattern to the PRO items as a function of the unit of the PerfOs. This example illustrates the promises of the co-calibration approach for the interpretation of PerfO measures but also highlights the challenges associated with it, mostly related to the quality of the PRO metric in terms of coverage of the targeted concept. Co-calibration with PRO measures could also be an adequate solution for interpretation of digital sensor measures whose meaningfulness is also often questioned.

Is the digital divide between older adults and other age groups closing? Impact of telemedicine and adherence with remote symptom monitoring in older adults compared to middle age and AYA patients with cancer.

e13689 Background: The wide adoption of telehealth during the pandemic brought the concern of widening technology disparity especially between AYAs 15-39y, mid-age 40-64y, older adults 65y+. Also, patient-reported outcomes (PRO)-based remote monitoring of patients (pts) with cancer between clinic visits improves quality of life and overall survival but is also often app-based. Since telehealth’s impact on other tech-related health behaviors such as adherence w remote symptom monitoring is not fully understood, we investigated PRO completion patterns in routine cancer care, pre- and mid pandemic. Methods: We queried a prospectively maintained registry of all remotely delivered PROs through the electronic health record (EHR) at our institution across two consecutive time periods: ‘prepandemic’ 9/1/17-3/31/20; ‘midpandemic’ 4/1/20-1/31/23. We captured pt demographics, cancer characteristics, and PRO adherence outcomes; we summarized descriptive statistics and compared (t-test, ANOVA) between time and age cohorts, PRO adherence and independent variables. Results: In total, 182,850 unique patients receiving routine cancer care were sent 1+ remote PROs outside of a clinic visit. Patient adherence w PROs overall increased, from 34% prepandemic (12548 of 37377 pts responding) to 62% mid-pandemic (89,634 of 145,473 patients responding; p &lt;0.00001). We then analyzed completion patterns of one global health PRO used widely across different oncologic settings to understand patient-level response behaviors. Of 4996 pts sent PROs prepandemic (1/1/18-3/31/20), 2517 (50%) responded; of 13543 pts sent PROs midpandemic (4/1/20-12/31/22), 10171 (75%) responded, an increase of 25%, p&lt;0.001). Prepandemic, 77% of pts responded only after a reminder from the clinical team, while midpandemic, only 2% needed these reminders, with 98% responding without a nudge (p&lt;0.001). Prepandemic, only 42% of seniors responded vs 54% mid-age &amp; 64% AYA (p&lt;0.01); midpandemic response rates increased across age cohorts (seniors increasing to 72%; mid-age to 77%; AYA to 77%) to where now, the difference in completion rates across age cohorts was no longer significant (p0.42). Furthermore, virtual visits accounted for the improvements seen in PRO adherence across age cohorts – with response rate among those with virtual visits being 99.9% in seniors, 99.7% in mid-age, and 100% in AYA during year 2022 while PRO response rates midpandemic in pts with in-person visits decreased across all age cohorts to AYA 29%, mid-age 33%, senior 32%. Conclusions: Telehealth was associated with significantly increased adherence with remote symptom monitoring with older adults accounting for the largest increases in this technology’s uptake to where the previously observed divide in EHR-based PRO adherence between older adults and mid-age &amp; AYA patients was closed.

Transarticular Versus Retroarticular Drilling of Stable Osteochondritis Dissecans of the Knee: A Prospective Multicenter Randomized Controlled Trial by the ROCK Group

Background: When stable osteochondritis dissecans (OCD) lesions of the femoral condyle in a skeletally immature patient fail to heal with nonoperative methods, the standard of care treatment is condylar OCD drilling. Two primary OCD drilling techniques have been described, but no prospective studies have compared their relative effectiveness. Purpose/Hypothesis: The purpose of this study was to compare the healing and function after transarticular drilling (TAD) with that after retroarticular drilling (RAD). It was hypothesized that there would be no difference in rate or time to healing, rate or time to return to sports, patient-reported outcomes (PROs), or secondary OCD-related surgery. Study Design: Randomized controlled clinical trial; Level of evidence, 1. Methods: Skeletally immature patients with magnetic resonance imaging–confirmed stable OCD lesions of the medial femoral condyle who did not demonstrate substantial healing after a minimum of 3 months of nonoperative treatment were prospectively enrolled by 1 of 17 surgeon-investigators at 1 of 14 centers. Patients were randomized to the TAD or RAD group. Tourniquet time, fluoroscopy time, and complications were compared between the treatment groups. Postoperatively, serial radiographs were obtained every 6 weeks to assess healing, and PROs were obtained at 6 months, 12 months, and 24 months. Results: A total of 91 patients were included, consisting of 51 patients in the TAD and 40 patients in the RAD group, who were similar in age, sex distribution, and 2-year PRO response rate. Tourniquet time and fluoroscopy time were significantly shorter with TAD (mean, 38.1 minutes and 0.85 minutes, respectively) than RAD (mean, 48.2 minutes and 1.34 minutes respectively) (P = .02; P = .004). In the RAD group, chondral injury from K-wire passage into the intra-articular space was reported in 9 of 40 (22%) patients, but no associated postoperative clinical sequelae were identified in these patients. No significant differences between groups were detected in follow-up Pediatric–International Knee Documentation Committee, Lysholm, Marx Activity Scale, or Knee injury and Osteoarthritis Outcome Score Quality of Life scores. Healing parameters were superior at 6 months and 12 months in the TAD group, compared with the RAD group, and secondary OCD surgery occurred in 4% of patients who underwent TAD and 10% of patients who underwent RAD (P = .40). Patients in the TAD group returned to sports earlier than those in the RAD group (P = .049). Conclusion: TAD showed shorter operative time and fluoroscopy time and superior healing parameters at 6 and 12 months, but no differences were seen in 24-month healing parameters or PROs at all follow-up time points, when compared with RAD. Registration: NCT01754298 (ClinicalTrials.gov identifier).

Abstract P5-08-01: Pilot study of a patient-reported outcome (PRO) measurement strategy to determine impact of screening for minimal residual disease (MRD) in high-risk breast cancer survivors

Abstract Background: Patients treated for early stage breast cancer (BC) have a 30% lifetime risk of developing metastatic disease. Numerous studies have demonstrated that dormant bone marrow disseminated tumor cells (DTCs) are independently associated with risk of recurrence and death, yet interventions targeting these cells are lacking. The PENN-SURMOUNT (Surveillance Markers of Utility for Recurrence after (Neo)adjuvant Therapy) Screening Study was launched in 2016 to screen high risk BC survivors for DTCs using bone marrow aspirate (BMA) and identify eligible DTC positive patients for clinical trials. Given the novelty of this approach, we concurrently developed and pilot tested a PRO measurement strategy to study how the screening method of BMA and disclosure of DTC results impacts early-stage BC patients. Methods: PENN-SURMOUNT is a single center prospective, longitudinal cohort study examining BM and blood biomarkers of MRD among patients within 5 years of BC diagnosis who have high risk criteria (positive axillary nodes, triple negative biology, ER+ with Oncotype Dx ≥ 25 and/or high risk Mammaprint, or pathologic residual disease after neoadjuvant chemotherapy). From May 2019 – August 2021, we recruited patients on SURMOUNT to complete PRO surveys at baseline (T0), after BMA (T1), and after disclosure of DTC results (T2). Surveys were administered in paper form initially, then electronic form starting Feb 2021. PRO survey instruments were selected through literature review, followed by consensus among multidisciplinary clinical and research experts and patient advocates. PRO measures assess recurrence distress (Quality of Life in Adult Cancer Survivors, QLACS), illness intrusiveness (Illness Intrusiveness Ratings Scale, IIRS), and decision making (Decision Regret Scale). Additional survey items assess tolerability of the BMA and patients’ risk perception and cognitive understanding after DTC results disclosure. Descriptive statistics summarize PRO survey compliance and responses at T0, T1, and T2 in the total population and the population who reported longitudinal data for T2. Results: 61 of 66 eligible patients on the SURMOUNT trial enrolled in the PRO pilot study and completed a baseline survey, of which 47 (77%) tested negative for DTCs. Mean completion rates were 0.92 at T0, 0.85 at T1, and 0.56 at T2. After electronic survey implementation, completion rates increased to 0.94 (T0), 0.97 (T1) and 0.81 (T2). At T0, 36 (59%) patients reported a high risk perception of developing BC recurrence at 5 years and 42 (69%) during their lifetime. Mean T0 recurrence distress using the QLACS subscale was 14.6 (SD 6.3) out of possible score 4-28, compared to an expected mean of 11.42 (SD 5.48) in a general survivorship population. Mean T0 illness intrusiveness was 27.3 (SD 13.9) out of possible score 13-91. At T1, approximately 85% of patients agreed that they correctly understood the purpose of the bone marrow procedure and what the procedure would entail. 44 (72%) of patients reported a maximum pain score &amp;lt;= 4 in the week post-procedure and 42 (69%) reported the BMA was same or better than expected tolerability. Exploratory subset analysis of patients with complete longitudinal data at T2 (n = 34) showed average scores of 13.4 (SD 6.0), 30.1 (SD 14.0), and 2.8 (SD 6.2) for recurrence distress, illness intrusive, and decision regret scores (scale 0-100), respectively. At T2, 26 (76%) of patients reported no decision regret for undergoing testing for DTCs; 27 (79%) reported feeling less anxious after DTC results disclosure. Conclusions: Participants of PENN-SURMOUNT perceived risk of recurrence as high. The BMA procedure was well-tolerated and better than expected among the majority of this cohort, and most did not regret having undergone BMA after DTC status disclosure. Longitudinal completion rates were low, limiting assessment of PROs at later time points, a major focus of future work in this setting. Citation Format: Tara Kaufmann, Patrick Chang, Shoshana Rosenberg, Elizabeth Frank, Brian Hobbs, Lauren J. Bayne, Isoris Nivar, Brooke L. Goodspeed, Killian M. Rohn, Emily M. Kugler, Kevin Fox, Susan Domchek, Angela Bradbury, Payal Shah, Hayley Knollman, Rachel C. Jankowitz, Igor Makhlin, Amy S. Clark, Lewis A. Chodosh, Angela DeMichele, Katherine Goodfellow. Pilot study of a patient-reported outcome (PRO) measurement strategy to determine impact of screening for minimal residual disease (MRD) in high-risk breast cancer survivors [abstract]. In: Proceedings of the 2022 San Antonio Breast Cancer Symposium; 2022 Dec 6-10; San Antonio, TX. Philadelphia (PA): AACR; Cancer Res 2023;83(5 Suppl):Abstract nr P5-08-01.

One-Third of European Patients With Axial Spondyloarthritis Reach Pain Remission With Routine Care Tumor Necrosis Factor Inhibitor Treatment.

To investigate the distribution of patient-reported outcomes (PROs) in patients with axial spondyloarthritis (axSpA) initiating a tumor necrosis factor inhibitor (TNFi), to assess the proportion reaching PRO "remission" across registries and treatment series, and to compare patients registered to fulfill the modified New York (mNY) criteria for ankylosing spondylitis (AS) vs patients with nonradiographic axSpA (nr-axSpA). Fifteen European registries contributed PRO scores for pain, fatigue, patient global assessment (PtGA), Bath Ankylosing Spondylitis (AS) Disease Activity Index (BASDAI), Bath AS Functional Index (BASFI), and Health Assessment Questionnaire (HAQ) from 19,498 patients with axSpA. Changes in PROs and PRO remission rates (definitions: ≤ 20 mm for pain, fatigue, PtGA, BASDAI, and BASFI; ≤ 0.5 for HAQ) were calculated at 6, 12, and 24 months of treatment. Heterogeneity in baseline characteristics and outcomes between registries were observed. In pooled data, 6 months after the start of a first TNFi, pain score was reduced by approximately 60% (median at baseline/6/12/24 months: 65/25/20/20 mm) in patients on treatment. Similar patterns were observed for fatigue (68/32/30/25 mm), PtGA (66/29/21/20 mm), BASDAI (58/26/21/19 mm), BASFI (46/20/16/16 mm), and HAQ (0.8/0.4/0.2/0.2). Patients with AS (n = 3281) had a slightly better response than patients with nr-axSpA (n = 993). The Lund Efficacy Index (LUNDEX)-adjusted remission rates at 6 months for pain/fatigue/PtGA/BASDAI/BASFI/HAQ were 39%/30%/38%/34%/35%/48% for the AS cohort and 30%/21%/26%/24%/33%/47% for the nr-axSpA cohort. Better PRO responses were seen with a first TNFi compared to a second and third TNFi. Patients with axSpA starting a TNFi achieved high PRO remission rates, most pronounced in those fulfilling the mNY criteria and for the first TNFi.

Patient-Reported-Outcomes and Safety of Autologous Stem Cell Transplantation in Multiple Sclerosis: A Single Center Experience with the "Mexican Method" in 1300 Persons

BACKGROUND Multiple sclerosis (MS) is a central nervous system autoimmune, inflammatory, demyelinating, and neurodegenerative disease that significantly lowers the quality of life for those affected. Autologous hematopoietic cell transplantation (aHSCT) has been demonstrated to be superior to new immunomodulatory drugs and is currently a proven therapeutic strategy, the patient reported outcomes (PROs) assessing the expanded-disability status scale (EDSS) improving in around 80%. We have previously reported that autotransplants for several hematologic malignancies can be done in the outpatient setting using refrigerated non frozen peripheral blood stem cells. We now report data from persons with MS receiving autotransplants in an outpatient setting using refrigerated blood cells followed for up to 24 months after aHSCT. MATERIALS AND METHODS Since June 2015, 1,300 consecutive patients with MS have been autografted in the Centro de Hematología y Medicina Interna de Puebla, México using non-frozen peripheral blood stem cells. All the aHSCT were started as outpatients, employing the "Mexican method": Cyclophosphamide 200 mg/kg and rituximab 1,000 mg; protocol is registered in ClinicalTrials.gov identifier NCT02674217. Eligibility criteria included all the following: Karnofsky performance score >70%, EDSS ≤8 in the 2 weeks pretransplant, CNS magnetic resonance image (MRI) ≤3 mo pretransplant, normal heart, liver, lung, and kidney function, ≥6 mo since exposure to immune suppressive drugs. The primary outcome measure was the PRO (EDSS changes) and secondary outcomes were overall survival (OS) and progression-free survival (PFS). Disability progression was defined as patients with EDSS ≤5.5 who experienced a PRO-EDSS increase of ≥1.0, whereas for patients with EDSS >5.5, progression was a PRO-EDSS increase of ≥0.5. RESULTS A total of 1300 persons were prospectively included (871 females and 429 males); median age was 47 years. 637 patients presented with relapsing remitting MS (RRMS), 390 with secondary progressive (SPMS) and 273 with primary progressive (PPMS). All procedures were started on an outpatient basis; 45 persons (3.5%) needed to be admitted to the hospital during the procedure as a result of neutropenic fever (20), pneumothorax (4), nausea and/or vomiting (4) or other complications; there were two fatalities (0.1%). One to three apheresis (median 1) were needed to obtain at least 1x106/Kg viable CD34 cells. Patients recovered above 0.5 x109/L absolute granulocytes on day 8 (median, range 2-13), whereas platelet recovery above 20 x109/L on day 4 (median, range 0-10). Patients remained in our premises for 28 days and PROs were surveyed by phone / zoom every 3 months after aHSCT. PROs could be recorded in 382/1300 persons (29%): PRO-response rate (no disability progression) at 12 months was 80% for all the patients,( 81.4% for RRMS, 76.2% for PPMS and 80% for SPMS). At 24 months the response rate was 85.8% for RRMS, 77.3% for PPMS and. 70% for SPMS. Changes in the EDSS score were statistically better at 12 (p<0.0001), 18 (p=0.0017) and 24 months (p=0.0003). CONCLUSIONS aHSCT for persons with MS employing the "Mexican method" is very safe and related with a positive PRO response rate (around 80% for all patients) at 12 and 24 months; results are better than those informed employing immunosuppressive drugs. Figure 1View largeDownload PPTFigure 1View largeDownload PPT Close modal

Integration of patient-reported outcomes into survivorship care.

271 Background: Patient reported outcomes (PRO) can be valuable clinical tools to embed the voice of patients into the clinical experience. The use of PROs in the context of cancer survivorship is relatively under-explored. Methods: Disease-specific modules of the MD Anderson Symptom Inventory (MDASI) PRO were integrated into the electronic health record (EHR), and distributed 3-7 days prior to survivorship clinic visits through the patients’ EHR portal. Patients with virtual visits were called 24 hours prior, instructed how to video link, and how to complete the MDASI PRO. For patients who completed the PRO electronically, results were reviewed during clinic visit. High symptom alerts (&gt; = 7) for four key symptoms were established and triggered an automatic message to care teams for review. Results: From Sept 1, 2020 – August 31, 2021, 2,196 PRO surveys were sent to patients with 1,268 submitting results (57.74%) (see Table). Within specific clinics, the submission rates varied greatly between in-person and virtual appointments (33% vs. 83%). Submission rates varied by clinics, with responses ranging from 28.57% - 73.59%. Clinics with higher overall rates had a higher proportion of virtual visits. Conclusions: Electronic distribution of PROs can be successfully integrated into long term survivorship clinic visits. Virtual visits had overall much higher PRO response rates, due to pre-visit workflows which encouraged the completion of PROs. We conclude that patient education and real time support is needed to facilitate patients’ completion of PRO surveys through the electronic portal.[Table: see text]

Telehealth closing racial/ethnic disparities? Increased adherence with remote symptom monitoring using patient-reported outcomes (PROs) among Black and Hispanic patients with telemedicine compared to in-person visits.

392 Background: Adherence with remote PROs in the real-world, non-clinical trial setting remains varied in pts with cancer. The rapid widespread telehealth into routine healthcare delivery raises concern for disparate impact on race/ethnic minorities. In this study, we sought to understand the impact of telehealth via electronic health record (EHR) on pt adherence with PRO-based remote symptom monitoring implemented through that same EHR portal. Methods: We queried a prospective institutional database of all PROs remotely delivered to our pts bw 1/1/18 - 12/31/21. Pts were divided into 2 time cohorts (pre-pandemic 1/1/18 to 3/31/20, mid-pandemic 4/1/20 to 12/31/21), their self-identified race/ethnicity, and age cohorts (AYA 15-39y, midage 40-64y, older adults 65y+). Then, one representative global symptom burden PRO measure used across different clinics was analyzed for completion in a representative month over 4 years, 2 pre- (Oct ’18, ‘19) and 2 mid-pandemic (Oct ’20, ‘21). We calculated descriptive statistics and compared (t-test, ANOVA) between time, race/ethnicity and age cohorts and independent variables. Results: Over 4 years, 93,875 unique patients received 1+ EHR-based remote PROs within their routine cancer care. Remote EHR-based PRO response rate almost doubled from pre-pandemic (35%, 12011 responders of 34742 pts) to mid-pandemic time (67%, 39812 of 59200 pts, p &lt; 0.00001). Analysis of one global symptom burden PRO widely used at our institution pre- and during pandemic showed the response rates rising (56.7% pre-pandemic vs 84.9% mid-pandemic, p 0.0004) with the sharp rise traced to 2nd and 3rd quarters of 2020 (response rate, 42.4% vs 85%, p &lt; 0.001), with the transition to telehealth. Within the representative 4m period, 2738 pts (1075 pts in 2 pre- &amp; 1663 in 2 mid-pandemic months) were sent that PRO – median age 60y, range 17-94y; 290 AYA 15-39y, 1444 midage 40-64y, 1004 older adults 65y+. PRO response rate increased (52% pre- to 81% mid-pandemic) w more responding without reminders from clinic team (13% pre-pandemic to 79% mid, p &lt; 0.001). Older adults saw the highest increases in PRO response rates up by 35% vs AYA up by 21% &amp; midage up 27%, p 0.012). Response rate without clinic reminders increased significantly (AYA, by 71%, midage by 78%, senior by 61%, p &lt; 0.00001). When further analyzing by visit type during pandemic, the increased PRO response rates are driven by telehealth where in-person PRO completion decreased by 19% (pre-pandemic 52%, during 33%) while pts who had an upcoming virtual visit had 94% PRO response rate (p &lt; 0.00001). Conclusions: Pt adherence w PRO-based remote symptom monitoring increased during the pandemic due to telehealth with the greatest gains seen in racial/ethinic minorities, inc. Black and Hispanic patients with cancer.

233. A ’low-resource' pilot to increase PRO response rates in an urban spine clinic

233. A ’low-resource' pilot to increase PRO response rates in an urban spine clinic

P79. Development of an evidence-based framework to increase patient-reported outcome response rates for orthopedic practices

<h3>BACKGROUND CONTEXT</h3> Collection of patient-reported outcomes (PROs) is increasingly viewed as the standard of care in various orthopedic surgeries given the intrinsic need for quality metrics and outcome collection requirements from government agencies and insurance carriers. Despite these incentives, low response rates to PRO collection tools remain a problem. Currently, most studies that address PRO response rates mainly focus on patients and few studies analyze strategies to improve response rates. To date, an evidence-based overview of successful PRO collection strategies incorporated into an overarching human-centered design framework is still absent. <h3>PURPOSE</h3> We aimed to systematically review existing literature, synthesize evidence, and develop an overarching framework of strategies to increase PRO collection rates. <h3>METHODS</h3> First, we systematically searched existing literature from 06-2011 to 06-2021 (using PubMed, EMBASE, Scopus, Business Source Complete) for studies that 1) included a primary or secondary outcome of PRO response rates evaluating one or more interventions, 2) included the PRO response rate achieved for each comparator group, 3) calculated response rates per PRO, 4) involved an orthopedic practice, 5) involved mainly adults. Included studies were assessed according to their focus on survey collection tool design or patient engagement; subsequently, a human-centered design approach was used to develop an evidence-based framework. In order to develop a comprehensive framework for strategies to increase PRO response rates, we developed a two-pronged framework: survey collection tool strategies and patient engagement strategies. <h3>RESULTS</h3> Among 15 included studies (conducted in 3 countries) there were 3 randomized controlled trials, 1 prospective study and 11 retrospective studies. PRO response rates achieved by interventions ranged from 46% to 100%; 3 studies included more than one intervention, and only 1 of these studies looked at each intervention separately. The remaining two were unable to identify which specific intervention was most effective. The most common intervention studied was the method of survey distribution modality, however, modalities' effectiveness was not uniform. Additional interventions studied were: dedicated research assistants, length of surveys, frequency of surveys, automated provider reminders and communication regarding standard of care. Having a dedicated research assistant, shorter surveys, and automated reminders for the provider showed an increase in response rate. Color-coding indicates which strategies have been studied and examples are included in italics. There are significant gaps in the literature, especially on the patient engagement side. The strategy studied most for patient engagement is the number of dedicated research assistants, which has higher associated costs due to dedicated labor hours. <h3>CONCLUSIONS</h3> Considering the two aspects of survey data collection: survey collection tool design and patient engagement, current literature has prioritized the former, leaving a considerable gap in the orthopedic community's understanding of strategies to improve PRO response rate. The framework presented strives to provide a systematic roadmap for orthopedic practices seeking to increase PRO response rates that do not drastically increase cost. <h3>FDA DEVICE/DRUG STATUS</h3> This abstract does not discuss or include any applicable devices or drugs.

Association between telehealth and adherence with patient-reported outcomes (PRO)-based remote symptom monitoring among adolescent/young adults (AYA), middle age, and older adults with cancer.

1513 Background: PRO-based remote symptom monitoring favorably impacts quality of life, healthcare utilization, and overall survival in patients (pts) with cancer. However remote PRO completion rates outside of a clinical trial remained widely varied. With the wide adoption of telehealth in cancer care during the pandemic, telehealth’s impact on health behaviors such adherence w remote PROs is not fully characterized. To that end, we investigated PRO completion patterns in routine cancer care, pre- and during the pandemic. Methods: We queried a prospectively maintained institutional database of all PROs remotely delivered to pts at our institution from 1/1/18 to 12/31/21. Pts were divided into 2 time cohorts (“pre-pandemic” 1/1/18 to 3/31/20, “during pandemic” 4/1/20 to 12/31/21) and 3 age cohorts (AYA 15-39y, midage 40-64y, older adults 65y+). We calculated descriptive statistics and compared (t-test, ANOVA) between time and age cohorts and independent variables. Results: Overall 93,875 unique patients over 4 years received 1+ remote PROs as a part of their routine cancer care. PRO response rate increased from 35% prepandemic (12011 of 34742 pts responding) to 67% during pandemic (p &lt;0.00001). To understand patient-level response patterns, we selected one representative global health PRO tool used widely across clinics in our institution and analyzed completion in a representative month over 4 years, 2 before (Oct ’18, ‘19) and 2 mid-pandemic (Oct ’20, ‘21). Overall 2738 pts (median age 60y, range 17-94y; 290 AYA 15-39y, 1444 midage 40-64y, 1004 older adults 65y+) were sent 3249 PROs during these 4m, 1378 PROs to 1075 pts in 2 pre-pandemic months &amp; 1871 to 1663 pts in 2 mid-pandemic months. Overall, PRO response rate increased from 52% pre-pandemic to 81% during, non-responders dropping from 48% to 19%, and response rate without any reminder from the team increasing from 13% pre-pandemic to 79% during. Across all 3 age cohorts, overall PRO response rates increased (AYA up 21%, midage up 27%, seniors up 35%, p 0.012), PRO non-response rate decreased (AYA by 21%, midage by 27%, seniors by 35%, p 0.01), and PRO response rate without reminders from clinic team increased significantly (AYA, by 71%, midage by 78%, senior by 61%, p &lt;0.00001). When further analyzing by visit type during pandemic, the improvements in overall PRO response rates are driven almost exclusively by telehealth where in-person PRO completion decreased by 19% (pre-pandemic 52%, during 33%) while pts who had an upcoming virtual visit had 94% PRO response rate (p &lt; 0.00001). Conclusions: Substantially higher adherence with PRO-based remote symptom monitoring was seen during the pandemic with virtual visits accounting substantially for this broad adherence and the highest increases seen in older adults, highlighting the implications of telehealth on cancer care.

Evaluating the implementation and use of patient-reported outcome measures in a mental health hospital in Denmark: a qualitative study.

Reporting of barriers and successes associated with the implementation and use of patient-reported outcomes (PROs) is limited as a means to ensure enhanced patient involvement, shared decision-making and improved treatment and care. We set out to evaluate the implementation and use of the PRO-Psychiatry initiative on patient-reported outcome measures in Danish mental health care. We aimed to described four specific areas: the quality of the clinical consultations before and after the implementation of PRO-Psychiatry as perceived by the patients (objective A), the motivation for participating in PRO-Psychiatry as perceived by patients and clinicians (objective B), the implementation process as perceived by patients, clinicians and managers (objective C) and suggestions for improvement (objective D). The PRO-Psychiatry initiative was evaluated through a participatory approach, including patients, clinicians and managers. A repeated cross-sectional interview-based survey explored the quality of the clinical consultation before and after the implementation of PRO-Psychiatry. A three-step semi-structured group interview, inspired by the modified mini-Delphi method, was used to establish consensus on the evaluation of the implementation and use of the initiative. The evaluation pointed at PRO-Psychiatry as a meaningful initiative, which motivated patients and supported clinicians. The patients emphasised the importance of PROs, but they also found that PROs were not used enough. Clinically relevant improvements were detected after the implementation of the initiative; more patients felt heard and experienced that clinicians took a greater interest in their problems. The clinicians valued the easily accessible real-time graphical display of the PRO responses in the electronic health record (EHR). Clinicians and managers agreed that clinical PRO practices, patient compliance and use of PROs in treatment and care should be supported during implementation. The evaluation was overall positive. Patients and clinicians were willing to participate, found the online reporting easy and valued the direct access to PRO responses in the EHR. An essential feature was the integration of well-defined and functional PRO practices into the existing clinical workflow. Using PROs in the clinical sessions in a way that was palpable to the patient was found to be a significant improvement need. At the individual level, PRO-Psychiatry can use patient outcome information to support dialogue, encourage shared decision-making and promote self-management during recovery. At the aggregated patient level, the PROs can be used for monitoring the patient-perceived quality of care and for research.

Maintenance of Patient-Reported Outcomes in Baricitinib-Treated Patients with Moderate-to-Severe Active Rheumatoid Arthritis: Post Hoc Analyses from Two Phase 3 Trials.

IntroductionBaricitinib has been shown to improve patient-reported outcomes (PROs) in patients with rheumatoid arthritis (RA) who are inadequate responders (IR) to conventional synthetic and biologic disease-modifying antirheumatic drugs (csDMARDs and bDMARDs, respectively). We assessed the ability of baricitinib 2-mg to maintain minimal clinically important differences (MCIDs) in PROs until week 24 among week 4 and 12 responders.MethodsData were from two phase 3 trials, RA-BUILD (NCT01721057; csDMARD-IR patients) and RA-BEACON (NCT01721044; bDMARD-IR patients). PROs included Pain Visual Analogue Scale, Health Assessment Questionnaire-Disability Index, Functional Assessment of Chronic Illness Therapy-Fatigue, Short-Form 36 Physical Component Score, and Patient’s Global Assessment of Disease Activity. Outcomes were evaluated by proportions of patients achieving MCID improvements, number needed to treat (NNT) at weeks 4, 12, and 24, proportions of patients maintaining MCID responses at week 24 among week 4 or 12 responders, and median time to achieve substantial response with baricitinib 2-mg versus placebo.ResultsA higher proportion of baricitinib-treated patients achieved MCID improvements, with NNTs ranging from 5 to 8 for baricitinib 2-mg versus placebo at week 24. Generally, early MCID responses in PROs at weeks 4 or 12 were better maintained through week 24 in RA patients treated with baricitinib 2-mg versus placebo. Patients treated with baricitinib 2-mg also achieved substantial PRO responses or normative values more quickly than placebo.ConclusionsThese results suggest baricitinib-treated patients with RA achieving MCID improvement in PROs at weeks 4 and 12 maintained those improvements over time and that substantial PRO responses were achieved quickly.

A Novel Methodology for Building Longitudinal, Patient-Centric Real World Datasets in Hemophilia A

A Novel Methodology for Building Longitudinal, Patient-Centric Real World Datasets in Hemophilia A

Performance of Patient-Reported Outcome Measures in a Large Pragmatic Trial of Home-Based Palliative Care (HomePal): Methodological and Practical Considerations for Embedded Patient-Centered Design.

Background: The research enterprise has embraced patient centeredness in embedded efficient pragmatic trials, but limited data exist on using patient-reported outcomes (PROs) collected as part of usual clinical care for research. Objectives: We sought to assess the performance of different assessment methods for obtaining PROs in a pragmatic cluster randomized trial (HomePal study) designed to compare two models of home-based palliative care (HBPC). Design: Descriptive analytics, comparative trends, and psychometric performance of PROs collected in the HomePal study; measures included Edmonton Symptom Assessment System (ESAS), PROMIS-10, and others administered at baseline, 1, and 6 months. Setting/Subjects: HomePal was conducted in the Southern California and Northwest Kaiser Permanente regions in the United States; subjects were patients receiving HBPC and their caregivers. Measurements: We specifically compared ESAS obtained by research staff with those obtained by clinical HBPC nurses at the time of HBPC enrollment. We also compared ESAS completed by patients versus if done or assisted by a caregiver (proxy). Results: We enrolled 3533 patients and had 2205 ESAS measurements that met the criteria for analysis at baseline and 1447 at the one-month follow-up assessment. Research staff-obtained ESAS at admission to HBPC was higher overall (indicating more symptoms) than the clinically collected measure whether symptoms were reported by patients (31.7 ± 15.4 vs. 26.0 ± 13.4) or by proxies (36.9 ± 15.6 vs. 26.5 ± 13.5). These differences persisted with follow-up ESAS measures. Conclusions: We identified significant variability in PRO responses between different surveyors and whether proxy interaction was needed suggesting complex issues around PRO measure performance for pragmatic embedded trials. ClinicalTrials.gov Identifier: NCT03694431.