BACKGROUND: Center for International Blood and Marrow Transplant Research (CIBMTR) prepares an annual set of summary slides to detail the trends in transplantation and cellular therapies. For the first time in the 2023 summary slides, CIBMTR incorporates data for patients receiving chimeric antigen receptor T-cell (CAR-T) infusions. In addition, the data on patient-reported outcomes (PROs) is also included.
OBJECTIVE: This report aims to update the annual trends in US HCT activity and incorporate data on the use of CAR-T therapies. Here we also aim to present and describe the development, implementation, and current status of the PRO data collection.
METHODS: In August 2020, CIBMTR launched the Protocol for Collection of Patient Reported Outcomes Data (CIBMTR PRO Protocol). The CIBMTR PRO Protocol operates under a centralized infrastructure to reduce burden to centers. Specifically, PRO data is collected from a prospective convenience sample of adult HCT and CAR-T patients who received treatment at contributing centers and consented for research. Data are merged and stored with the clinical data and used under the governance of the CIBMTR Research Database Protocol. Participants answer a series of surveys developed by the Patient Reported Outcomes Measurement Information System© (PROMIS) focusing on physical, social and emotional, and others measures assessing financial well-being, occupational functioning, and social determinants of health. To complement traditionally measured clinical outcomes, the surveys are administered at the same timepoints that clinical data is routinely collected.
RESULTS: As of September 2023, PRO data from 993 patients across 25 different centers has been collected. With the goal of incorporating these important patient perspectives into standard clinical care, CIBMTR has added the PRO data to Data Back to Centers (DBtC). Through expanding the data types represented in the registry, CIBMTR aims to support holistic research accounting for the patient perspective in improving patient outcomes.
CONCLUSIONS: PRO data at CIBMTR aims to provide the foundation for future large scale, population-level evaluations to determine areas for improvement, emerging disparities in access and health outcomes (eg, by age, race, and ethnicity), and new therapies that may impact current treatment guidelines. Continuing to collect and grow the PRO data is critical for understanding these changes and identifying methods for improving patient quality of life.

Burn reconstruction outcomes are an area of growing investigation. Although there is evidence of measured physical improvements in scar characteristics after laser treatment, there is little information on patient reported outcomes. The purpose of this study is to compare patient reported outcomes between burn survivors with and without laser treatment. The study included participants in the Burn Model Systems National Database at a single center; participants that received outpatient laser treatment for burn scars were compared to a matched group of burn survivors that did not receive laser. The following outcomes were examined: Satisfaction With Life Scale, Mental and Physical Component Summary of the Veterans Rand Survey, and the PROMIS Pain Intensity Scale. Regression analyses examined the associations between laser treatment and each outcome at 12 and 24 months. The study population included 287 adult burn survivors (65 laser group, 222 control group). The significant differences found between the two groups included: burn size (laser: 14.9, 13.5 SD, control: 8.9, 11.1 SD; p<0.001), insurance type (p=0.036), inhalation injury (laser: 17.2%, control: 2.7%; p<0.001), and ventilator requirement (laser: 27.7%, control: 13.5%; p=0.013). Laser treatment was not associated with any of the outcomes at both follow-up time points. Further research is needed to develop patient reported outcome measures that are more sensitive to the clinical changes experienced by burn survivors receiving laser treatment.

BACKGROUND: Chronic pruritus is a clinically heterogeneous symptom that manifests itself with varying duration, intensity, or quality. To date, there is no validated German-language instrument that systematically assesses the relevant parameters. With the support of the Pruritus Research Working Group (Arbeitsgemeinschaft Pruritusforschung, AGP), a questionnaire for the assessment of chronic pruritus (AGP questionnaire) was developed in 2008. The subsequently revised instrument, now called the German Pruritus Questionnaire, records pruritus-specific parameters such as localization, course, intensity and quality, anamnestic data on the general state of health, sociodemographic data, quality of life, and coping methods. It is to be validated in the study presented here.
METHODS: The questionnaire was used in 366 patients with chronic pruritus of different etiologies from Germany (University Hospitals Heidelberg, Münster, Mainz, Erlangen, Giessen, private practice Bad Bentheim, TU Munich, Wiesbaden Kidney Center), Austria (Graz University Hospital) and Switzerland (Aarau Cantonal Hospital).
RESULTS: The reliability for repeated completion (retest reliability) with regard to localization, first occurrence, and concomitant diseases showed high values for Cohen\'s kappa (> 0.8). The data on the retest reliability of the pruritus characteristics showed lower values (< 0.7). With regard to the measurability of practically relevant changes (change sensitivity), medium to strong effect sizes were found (0.09-0.19). A statistically significant differentiation of the pruritus etiologies based on the recorded parameters was not possible.
CONCLUSIONS: The German Pruritus Questionnaire allows a comprehensive and structured recording of patient- and clinician-reported, relevant dimensions of chronic pruritus of different etiologies. Further adaptation and development are planned.

UNASSIGNED: Posterior malleolar involvement can drastically affect patient outcomes. Literature has supported the use of preoperative Computed Tomography (CT) to assess posterior malleolar fracture morphology. The purpose of this study is to determine whether preoperative CT is associated with significant improvement in surgical time, postoperative complications, reoperation rates in trimalleolar ankle fractures. Surgeons were also asked to complete survey regarding use of CT scans to gauge utility preoperatively.
UNASSIGNED: Adult patients with trimalleolar ankle fractures who underwent operative fixation between 2018-2020 were retrospectively reviewed. Primary outcomes included surgical time, postoperative complications, and reoperations. Secondary outcome was presence of posterior malleolar fixation. 15 surgeons who performed ankle ORIF were surveyed to gain information regarding why or why not preoperative CT scan was obtained.
UNASSIGNED: 288 patients with trimalleolar ankle fractures were included, 94 had preoperative CT scans (32.6%). No significant differences found in patient age, gender, BMI, smoking status between the groups that did and did not have preoperative CT scan. No significant differences were observed in AO/OTA classification between groups. Average surgical time was significantly higher in group that received a preoperative CT (114 without CT vs. 145 with CT, p<0.05). Complications (10.3% no CT vs 7.4% with CT, p=0.55) and reoperations (6.7% without CT vs. 7.4% with CT, p=0.16) not significantly different between groups. No significant difference was observed in rate of posterior malleolus fixation between groups (43.8% without CT vs 39.4% with CT; p=0.52). Of surveyed surgeons, 87% reported they don\'t routinely obtain preoperative CT scan for trimalleolar ankle fractures. Most common reasons for preoperative scans were deciding on approach/positioning, assessing for impaction, determining the size of the posterior malleolus.
UNASSIGNED: Although preoperative CT scans are obtained in one third of patients with operative trimalleolar ankle fractures, we did not find an improvement in surgical time, complications, and reoperation. Level of Evidence: III.

BACKGROUND: Measurement-based care in behavioral health uses patient-reported outcome measures (PROMs) to screen for mental health symptoms and substance use and to assess symptom change over time. While PROMs are increasingly being integrated into electronic health record systems and administered electronically, paper-based PROMs continue to be used. It is unclear if it is feasible to administer a PROM on paper when the PROM was initially developed for electronic administration.
OBJECTIVE: This study aimed to examine the feasibility of patient self-administration of a 2-part substance use screener-the Tobacco, Alcohol, Prescription medications, and other Substances (TAPS)-on paper. This screener was originally developed for electronic administration. It begins with a limited number of questions and branches to either skip or reflex to additional questions based on an individual\'s responses. In this study, the TAPS was adapted for paper use due to barriers to electronic administration within an urgent care behavioral health clinic at an urban health safety net hospital.
METHODS: From August 2021 to March 2022, research staff collected deidentified paper TAPS responses and tracked TAPS completion rates and adherence to questionnaire instructions. A retrospective chart review was subsequently conducted to obtain demographic information for the patients who presented to the clinic between August 2021 and March 2022. Since the initial information collected from TAPS responses was deidentified, demographic information was not linked to the individual TAPS screeners that were tracked by research staff.
RESULTS: A total of 507 new patients were seen in the clinic with a mean age of 38.7 (SD 16.6) years. In all, 258 (50.9%) patients were male. They were predominantly Black (n=212, 41.8%), White (n=152, 30%), and non-Hispanic or non-Latino (n=403, 79.5%). Most of the patients were publicly insured (n=411, 81.1%). Among these 507 patients, 313 (61.7%) completed the TAPS screener. Of these 313 patients, 76 (24.3%) adhered to the instructions and 237 (75.7%) did not follow the instructions correctly. Of the 237 respondents who did not follow the instructions correctly, 166 (70%) answered more questions and 71 (30%) answered fewer questions than required in TAPS part 2. Among the 237 patients who did not adhere to questionnaire instructions, 44 (18.6%) responded in a way that contradicted their response in part 1 of the screener and ultimately affected their overall TAPS score.
CONCLUSIONS: It was challenging for patients to adhere to questionnaire instructions when completing a substance use screener on paper that was originally developed for electronic use. When selecting PROMs for measurement-based care, it is important to consider the structure of the questionnaire and how the PROM will be administered to determine if additional support for PROM self-administration needs to be implemented.

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

BACKGROUND: Early discontinuation of endocrine therapy (ET) is higher among patients with early breast cancer (EBC) compared to patients with metastatic hormone receptor-positive (HR+) breast cancer (MBC). In our clinical experience the reasons for this may include a significant burden of ET side effects impacting quality of life (QOL) in patients with EBC.  We hypothesized that QOL is lower in patients with HR + EBC compared to patients with HR + MBC on ET.
METHODS: We conducted a cross-sectional observational study to assess QOL utilizing FACT-ES & EORTC QLQ C30 tools among patients with EBC and MBC receiving ET across 5 Irish hospitals.
RESULTS: A total of 417 patients were enrolled-EBC (79% n = 331) and MBC 21% (n = 86). Using the FACT-ES, we found no difference in overall QOL by stage (139.2 vs 141, P  = .33). Patients with HR + MBC had a lower symptom burden from ET compared to HR + EBC (61.4 vs 54, P < .01). In adjusted multivariate linear regression models, there was no difference in QOL for patients with EBC and MBC receiving ET.
CONCLUSIONS: There was no significant difference in overall QOL for patients with EBC and MBC. However, patients with EBC experienced more endocrine symptoms. In adjusted multivariate linear regression models, the stage did not predict QOL. Our results suggest that endocrine symptoms are significant contributors to impaired QOL for patients with EBC but the role of other determinants of QOL (eg, stage) is less clear. Future work could include the development of stage-specific QOL tools and utilization of electronic patient-reported outcomes (ePROs) to identify and manage emergent toxicities.

Incorporating person-centered outcomes into clinical trials for neurodegenerative diseases has been challenging due to a deficiency in quantitative measures. Meanwhile, the integration of personally meaningful treatment targets in clinical practice remains qualitative, failing to truly inform evaluations, therapeutic interventions and longitudinal monitoring and support. We discuss the current advances and future directions in capturing individualized brain health outcomes and present an approach to integrate person-centered outcome in a scalable manner. Our approach stems from the evidence-based electronic Person-Specific Outcome Measure (ePSOM) program which prompts an individual to define personally meaningful treatment priorities and report level of confidence in managing items that matter to the individual the most (e.g., \"Do I feel confident in my ability to contribute to a conversation?\"). Deployed either as a single version (person only) or a dyad version (person and care partner), our proposed tool could be used as an endpoint in clinical trials, offering proof of meaningful intervention benefits and in clinical practice, by establishing an anchor for the therapeutic objectives sought by the individual.

OBJECTIVE: To determine the effectiveness in terms of quality of life perceived by adult patients with moderate/severe plaque psoriasis treated with interleukin 17 or 23 inhibitors and to identify associated factors.
METHODS: Cross-sectional observational study including adult patients diagnosed with moderate/severe plaque psoriasis treated with interleukin 17 or 23 inhibitors for at least 12 or 16 weeks in follow-up, respectively.
RESULTS: Forty-one patients were included: 65% male, median age 54 years (SD=13). The included patients were treated with ixekizumab 35%, guselkumab 25%, secukinumab 17.5%, brodalumab 15%, and risankizumab 7.5%. Psoariasis area severity index (PASI) reduction was 94.6% (RIC 76.8-100%), DLQI of 1 (RIC 0-2.75), DLQI≤1 60%. The most affected health dimensions were symptoms and perceptions (57.5%), activities of daily living (27.5%), and discomfort caused with treatment (17.5%). No association was found between DLQI score <1 and demographic, comorbidities, and treatment-related variables. The median PASI reduction in patients with DLQI<1 was superior to patients with DLQI>1 (100% vs 90.2%, p=.025).
CONCLUSIONS: Patients with moderate/severe plaque psoriasis treated with interleukin 17 or 23 inhibitors achieve adequate therapeutic targets achieving the target set according to clinical practice guideline recommendations (score ≤1 on the DLQI questionnaire and 90-100% reduction in the PASI index) and in accordance with the results of recent meta-analyses and real-life studies. A greater reduction of the PASI index is observed in the group reaching the quality of life target, there being the possibility of using patient-reported outcomes in the evaluation of treatment effectiveness.

UNASSIGNED: To evaluate the acceptability, retention, and efficacy of face-to-face intervention, incorporating education and Motivational Interviewing (MI) to support persons with relapsing-remitting multiple sclerosis (PwRRMS) and increase self-reported medication adherence.
UNASSIGNED: PwRRMS (N = 60) prescribed Disease Modifying Treatment (DMT), who were identified as non-adherent and consented to participate in an intervention, received verbal education and counseling from their treating physician, a tailored MI counseling and a booster session via telephone with a health psychologist, and a concluding MI counseling six months later. Each PwRRMS filled a battery of patient-reported outcomes (PROs) at baseline, six and 12 months later. The design was a quasi-experimental pre-test post-test across a year.
UNASSIGNED: Of the sixty identified persons who consented to enroll, 52 completed the intervention and 46 completed the follow-up. At six months following the baseline, adherence scores increased (median = 12.0) and were significantly different than at baseline (median=10.0, p = 0.030). Still, at 12 months follow-up there was no significant difference from baseline in reported adherence (median = 11.0, p = 0.106).
UNASSIGNED: This study demonstrated reasonable retention and initial efficacy of a combined psycho-education and MI protocol for PwRRMS to enhance medication adherence to DMT. To maintain the change, a more sustained intervention is required.
The study focused on persons with relapsing-remitting multiple sclerosis (PwRRMS) who do not adhere to their prescribed medication. Following the identification of non-adherent persons, PwRRMS were offered an intervention to increase their adherence. The study examined how many of those identified consented to enroll in the intervention, how many remained in the intervention, and whether the intervention was efficacious in terms of self-reported adherence. The intervention included verbal education and counseling from the treating physician, immediately followed by tailored counseling by a psychologist. There was a booster session via telephone with the psychologist, and a concluding counseling meeting six months later. Participants were followed for a year after the initial counseling. Two-thirds of PWMS identified as non-adherent consented to enroll (n = 60), 52 completed the intervention and 46 completed the follow-up. At six months following counseling, self-reported adherence scores significantly increased, but at 12 months follow-up there was no significant difference from baseline in reported adherence. To maintain the change, a more sustained intervention is required.