Collection Of Patient-reported Outcomes Research Articles

Toward facilitating the collection and utilization of patient-reported outcomes in the Military Health System: Lessons learned from a pragmatic clinical trial on physical therapy management for low back pain.

Toward facilitating the collection and utilization of patient-reported outcomes in the Military Health System: Lessons learned from a pragmatic clinical trial on physical therapy management for low back pain.

The Oswestry Disability Index in Elective Single level Lumbar Fusion: Is 3 month follow up enough?

BackgroundProspective, longitudinal collection of patient reported outcomes (PRO) has become an essential metric in orthopedics. Despite the utility of PROs, data collection presents a significant challenge to the healthcare system. There is a need to better understand if serial data collection over a 1 to 2 year postoperative period is truly warranted. The purpose of this study was to determine if continued PRO collection after 3 months postop is needed in patients that underwent lumbar fusion. MethodsThis retrospective study utilized 239 patients that underwent an elective single level lumbar fusion between April 1, 2020 and February 1, 2023. Changes in the Oswestry Disability Index (ODI) scores over a one year period were assessed for all patients. Patients were placed into one of three study groups, those that improved by 10 points, those that worsened by 10 points, and those that did not change (score change less than 10 points in either direction). Movement between study groups, the minimal clinical important difference (MCID), and patient satisfaction were analyzed for each patient. ResultsImprovement between preoperative and the 3 month postoperative evaluations was noted for most patients. There was limited change in scores after 3 months. Patients in the improved group continued to improve through 1 year post-operative. Patients in the worsened group continued to decline by 1 year postoperative. Patients in the no change group demonstrated the highest potential to change groups. ConclusionsPatients with substantial improvement or worsening at 3 months have a low likelihood of substantial clinical change thereafter. This subset of patients may not need further evaluation with PROs, but those in the worsened group may benefit from other interventions to potentially alter their course.

Use of Patient-Reported Outcomes in Risk Prediction Model Development to Support Cancer Care Delivery: A Scoping Review.

The integration of patient-reported outcomes (PROs) into electronic health records (EHRs) has enabled systematic collection of symptom data to manage post-treatment symptoms. The use and integration of PRO data into routine care are associated with overall treatment success, adherence, and satisfaction. Clinical trials have demonstrated the prognostic value of PROs including physical function and global health status in predicting survival. It is unknown to what extent routinely collected PRO data are used in the development of risk prediction models (RPMs) in oncology care. The objective of the scoping review is to assess how PROs are used to train risk RPMs to predict patient outcomes in oncology care. Using the scoping review methodology outlined in the Joanna Briggs Institute Manual for Evidence Synthesis, we searched four databases (MEDLINE, CINAHL, Embase, and Web of Science) to locate peer-reviewed oncology articles that used PROs as predictors to train models. Study characteristics including settings, clinical outcomes, and model training, testing, validation, and performance data were extracted for analyses. Of the 1,254 studies identified, 18 met inclusion criteria. Most studies performed retrospective analyses of prospectively collected PRO data to build prediction models. Post-treatment survival was the most common outcome predicted. Discriminative performance of models trained using PROs was better than models trained without PROs. Most studies did not report model calibration. Systematic collection of PROs in routine practice provides an opportunity to use patient-reported data to develop RPMs. Model performance improves when PROs are used in combination with other comprehensive data sources.

MyMS: An Interface for Patient-Reported Outcomes for Finnish Individuals With Multiple Sclerosis.

Patient-generated data are a cornerstone of individualized multiple sclerosis (MS) treatment. MyMS, an interface for patient-reported outcomes (PROs) was developed by the Finnish MS Register to enable systematic collection of PROs. MyMS collects data on demographics, lifestyle factors, disease-related factors, and validated questionnaires, including the Quality of Life Questionnaire (15D), the Multiple Sclerosis Impact Scale (MSIS-29), and the Fatigue Severity Scale (FSS). At the end of 2020, the patient-reported Expanded Disability Status Scale (PREDSS), the EuroQOL-5 Dimension (EQ-5D), the Fatigue Scale for Motor and Cognitive Functions (FSMC), and the Multiple Sclerosis Neuropsychological Questionnaire (MSNQ) were added. As of January 1, 2023, 1201 individuals with MS (79% female) have added data to MyMS. Of the validated PRO measures (PROMs), the 15D, MSIS-29, and FSS are the most used. The mean PREDSS score is 3.0 and median disease duration is 6.4 years. According to the existing PROMs, patients report mildly compromised quality of life and problems with fatigue and cognition. The patient interface of the Finnish MS Register consists of data from 17 of 21 counties with well-being services. The interface is used by 10% of Finnish individuals with MS. The addition of the PREDSS, EQ-5D, FSMC, and MSNQ to the interface has increased health care professional and patient interest in the use of PROMs. We suggest that PROs should be integrated into electronic health records to improve shared decision-making and diminish documentation burden.

Patient-centered clinical decision support challenges and opportunities identified from workflow execution models.

To use workflow execution models to highlight new considerations for patient-centered clinical decision support policies (PC CDS), processes, procedures, technology, and expertise required to support new workflows. To generate and refine models, we used (1) targeted literature reviews; (2) key informant interviews with 6 external PC CDS experts; (3) model refinement based on authors' experience; and (4) validation of the models by a 26-member steering committee. We identified 7 major issues that provide significant challenges and opportunities for healthcare systems, researchers, administrators, and health IT and app developers. Overcoming these challenges presents opportunities for new or modified policies, processes, procedures, technology, and expertise to: (1) Ensure patient-generated health data (PGHD), including patient-reported outcomes (PROs), are documented, reviewed, and managed by appropriately trained clinicians, between visits and after regular working hours. (2) Educate patients to use connected medical devices and handle technical issues. (3) Facilitate collection and incorporation of PGHD, PROs, patient preferences, and social determinants of health into existing electronic health records. (4) Troubleshoot erroneous data received from devices. (5) Develop dashboards to display longitudinal patient-reported data. (6) Provide reimbursement to support new models of care. (7) Support patient engagement with remote devices. Several new policies, processes, technologies, and expertise are required to ensure safe and effective implementation and use of PC CDS. As we gain more experience implementing and working with PC CDS, we should be able to begin realizing the long-term positive impact on patient health that the patient-centered movement in healthcare promises.

Patient engagement in hematopoietic stem cell transplantation and cell therapy: a survey by the EBMT patient engagement task force & transplantation complications working party.

The EBMT (European Blood and Marrow Transplantation Society) aims to connect patients, the scientific community, and other stakeholders to improve hematopoietic stem cell transplantation and cellular therapy outcomes. We performed a cross-sectional online survey to understand the perceptions regarding Patient Reported Outcomes (PROs) and Patient Active Involvement in Research (PAIR) in over 800 stakeholders (n = 813). Patients (n = 278) and health care professionals (HCPs) (n = 351) were compared. We observed high openness for EBMT PRO collection (n = 680, 84.5% across stakeholders' groups; patients n = 256, 93.1% versus HCPs n = 273, 78.4% [p < 0.001]) and PAIR (n = 702, 87.3% across stakeholder groups; patients n = 256, 92.4% versus HCPs n = 296, 85.8% [p = 0.009]), with a significantly higher proportion of patients expressing interest compared to HCPs. Priority domains for PROs data-collection identified were the assessment of symptom experience, psychosocial and cognitive functioning. The most important issues for patients specifically were the data-collection of PROs reflecting cognitive function, the option of reporting data at home, the importance of identifying actionable targets to improve their recovery, and receiving feedback on their input when participating in research projects. Our multistakeholder approach suggests an added value to embracing patient engagement in the development of meaningful research and service design within the transplantation and cellular therapy community.

International trends in shoulder replacement: a meta-analysis from 11 public joint registers.

International variation exists in the types of shoulder replacement used for treatment of specific diseases. Implant choice continues to evolve without high-quality evidence. Our aim was to evaluate trends in incidence rates of shoulder replacement and assess any recent changes in practice between countries by using registry data. Patient characteristics, indication and year of surgery, type of replacement, and collection methods of patient-reported outcomes (PROMs) was extracted from 11 public joint registries. Meta-analyses examined use of reverse total shoulder replacement (RTSR) for osteoarthritis, cuff tear arthropathy, and acute fracture; use of anatomical total shoulder replacement (TSR) for osteoarthritis; and use of humeral hemiarthroplasty for fracture. The annual growth rate of shoulder replacements performed is 6-15% (2011-2019). The use of RTSR has almost doubled (93%). RTSR is now universally performed for cuff tear arthropathy (97.3%, 95% confidence interval [CI] 96.0-98.1). Its use for avascular necrosis, trauma, and inflammatory arthropathy is increasing. The use of RTSR was similar (43.1%, CI 30.0-57.2) versus TSR (44.7%, CI 31.1-59.1) for osteoarthritis. The types of PROMs used, collection time points, and response rates lack standardization. COVID-19 had a varying inter-registry impact on incidence rates. The incidence of shoulder replacements has grown. Use of RTSR has increased for all disease indications despite limited high-quality evidence driving this change in indications outside of cuff arthropathy. Consequently, less variation is observed in international practice. Existing differences now relate to use of newer implant types and methodology of PROMs collection, which prevents international comparison and outcome analysis.

Patient-Reported Outcomes in Phase 3 Clinical Trials for Blood Cancers: A Systematic Review

Published research suggests that patient-reported outcomes (PROs) are neither commonly collected nor reported in randomized clinical trials (RCTs) for solid tumors. Little is known about these practices in RCTs for hematological malignant neoplasms. To evaluate the prevalence of PROs as prespecified end points in RCTs of hematological malignant neoplasms, and to assess reporting of PROs in associated trial publications. All issues of 8 journals known for publishing high-impact RCTs (NEJM, Lancet, Lancet Hematology, Lancet Oncology, Journal of Clinical Oncology, Blood, JAMA, and JAMA Oncology) between January 1, 2018, and December 13, 2022, were searched for primary publications of therapeutic phase 3 trials for adults with hematological malignant neoplasms. Studies that evaluated pretransplant conditioning regimens, graft-vs-host disease treatment, or radiotherapy as experimental treatment were excluded. Data regarding trial characteristics and PROs were extracted from manuscripts and trial protocols. Univariable analyses assessed associations between trial characteristics and PRO collection or reporting. Ninety RCTs were eligible for analysis. PROs were an end point in 66 (73%) trials: in 1 trial (1%) as a primary end point, in 50 (56%) as a secondary end point, and in 15 (17%) as an exploratory end point. PRO data were reported in 26 of 66 primary publications (39%): outcomes were unchanged in 18 and improved in 8, with none reporting worse PROs with experimental treatment. Trials sponsored by for-profit entities were more likely to include PROs as an end point (49 of 55 [89%] vs 17 of 35 [49%]; P < .001) but were not significantly more likely to report PRO data (20 of 49 [41%] vs 6 of 17 [35%]; P = .69). Compared with trials involving lymphoma (18 of 29 [62%]) or leukemia or myelodysplastic syndrome (18 of 28 [64%]), those involving plasma cell disorders or multiple myeloma (27 of 30 [90%]) or myeloproliferative neoplasms (3 of 3 [100%]) were more likely to include PROs as an end point (P = .03). Similarly, compared with trials involving lymphoma (3 of 18 [17%]) or leukemia or myelodysplastic syndrome (5 of 18 [28%]), those involving plasma cell disorders or multiple myeloma (16 of 27 [59%]) or myeloproliferative neoplasms (2 of 3 [67%]) were more likely to report PROs in the primary publication (P = .01). In this systematic review, almost 3 of every 4 therapeutic RCTs for blood cancers collected PRO data; however, only 1 RCT included PROs as a primary end point. Moreover, most did not report resulting PRO data in the primary publication and when reported, PROs were either better or unchanged, raising concern for publication bias. This analysis suggests a critical gap in dissemination of data on the lived experiences of patients enrolled in RCTs for hematological malignant neoplasms.

MSK Engage: A comprehensive program for collection of patient reported outcomes at scale.

e13666 Background: Collecting patient-reported outcomes (PROs) yields high value data for clinical care and research and is associated with improved survival in oncology. However, collection of PROs at scale requires a multifaceted program that in most institutions is not available. This study describes a comprehensive PRO program to support research and standard clinical care at scale. Methods: Memorial Sloan Kettering Cancer Center is a high-volume cancer center in New York. Beginning in 2016, we developed a comprehensive PRO program, MSK Engage, with the following capabilities: (i) A governance process to oversee the creation of questionnaires for both research and clinical care. This process includes alignment with the IRB and standardization of questions presented to patients. (ii) Technology to determine which patients should get an assessment (i.e., instance of a questionnaire) at what point in time. (iii) An interactive module in our patient portal that would present the assessment to the patient. (iv) Various methods to present the PRO data to providers to support clinical decision making. These methods included (a) alerts based on threshold logic that would send secure messages to the clinical team, (b) integration of the PRO data into the EHR to be viewed with other clinical data, and (c) the creation of specialized workflows. The results of assessments are also stored in an analytics database to support operations, research, and the creation of smart reports that compare a patient’s responses with other similar patients. Results: We analyzed the use of the program from 2016 to 2023 (Table). 189 questionnaires have been implemented, generating 3,098,574 completed assessments from 314,685 distinct patients. Completion rates varied. The median completion time for all questionnaires was 2 minutes. Examples of programs enabled by patient-reported outcomes include post-operative symptom monitoring, family history questionnaires to support clinical genetics decision making and operations, intake and follow up questionnaires that support clinical documentation and quality of life questionnaires to support goal concordant care. Conclusions: Adoption of PROs at scale for both research and standard of care in oncology is feasible and innovative programs of care can be supported while maintaining a centralized and acceptable patient experience. This model can be applied across institutions seeking to develop or scale PRO programs to assure patient data are meaningfully incorporated into decision-making. [Table: see text]

Randomized, placebo-controlled trial of atorvastatin to prevent hearing loss in patients with head and neck squamous cell carcinoma receiving cisplatin based chemoradiation (CRT).

TPS12153 Background: Cisplatin chemotherapy is associated with several adverse effects, including a permanent, high-frequency, sensorineural hearing loss in about 50% of patients. Preclinical studies suggest that statin drugs can mitigate cisplatin ototoxicity (Fernandez et al., Hearing Research 2020). In our prior combined retrospective/prospective dataset of patients with head and neck squamous cell carcinoma (HNSCC) treated with cisplatin-based CRT, the incidence of CTCAE grade ≥ 2 hearing loss was 29.4% in statin non-users, versus 9.7% in atorvastatin users (Fernandez et al., J Clin Invest 2021). An adjusted odds ratio analysis indicated that atorvastatin users are significantly less likely to develop hearing loss versus statin non-users (odds ratio 0.47), providing the rationale for a randomized, controlled, prospective study. Methods: This multi-center, randomized, placebo-controlled study is enrolling patients with previously-untreated, locally advanced head and neck squamous cell carcinoma (HNSCC) for which cisplatin-based chemoradiation is indicated, either in the definitive or adjuvant setting. Patients who will be treated with cisplatin (at 75-100 mg/m2 bolus every three weeks or 40 mg/m2 weekly) are eligible. Patients currently taking a statin or those without measurable hearing in at least one ear are excluded. A baseline audiogram is performed, and patients are randomized to atorvastatin (40 mg/day) or placebo, which is started 2-7 days before CRT and taken daily until 3 months after completion of CRT. Post-treatment audiometry is repeated at 3 months and 2 years after CRT, along with collection of patient-reported outcomes on hearing, tinnitus, and peripheral neuropathy. Patients are monitored for liver and muscle toxicity, with dose de-escalation or cessation of atorvastatin as needed. The primary endpoint is the incidence of CTCAE grade ≥2 hearing loss at 3 months after CRT. Secondary endpoints include other treatment related grade 3-4 adverse events, overall survival, and disease-free survival. Up to 214 subjects will be enrolled at 4 U.S. sites (NIH Clinical Center, Emory University, University of Maryland, University of Rochester). Funding: NIH/NIDCD, U01 DC020452 and intramural project ZIA DC000079. Clinical trial information: NCT04915183 .

Adoption of decentralized trial elements in cancer clinical trials supporting FDA approvals during COVID-19.

e13802 Background: The COVID-19 pandemic significantly disrupted the conduct of cancer clinical trials. Facilitated by emergency FDA guidance, many sponsors integrated decentralized clinical trial (DCT) elements to prioritize patient safety and maintain access. The FDA’s Oncology Center of Excellence, in collaboration with the Clinical Trials Transformation Initiative (CTTI), assessed the adoption of DCT elements in registrational cancer clinical trials conducted during the pandemic that resulted in an FDA approval. Methods: CTTI sent trial sponsors a recruitment email with a link to an online survey and the name of the trial that resulted in an FDA approval. Sponsors were asked to select the DCT elements used in that trial prior to and in response to the pandemic, challenges faced when implementing these approaches, and the likelihood of using the approaches in the future. Data were collected from December 6, 2022 to January 17, 2023. Descriptive statistics were used to summarize sponsor responses. Results: Out of 52 eligible studies, 19 surveys were completed from 13 unique sponsors (37%; 12 pharmaceutical companies and 1 chose not to disclose). Sixty-three percent of sponsors identified as a large pharmaceutical company with a market cap of over $10 billion. The majority of cancer clinical trials (89%) occurred both in the United States and abroad, with almost all sponsors (95%) incorporating at least one DCT element during the pandemic. The most widely adopted DCT elements were remote site monitoring (89%), telemedicine (68%), remote laboratory assessments (63%), remote distribution of investigational product (58%) and local imaging assessments (47%). The main challenges in implementing DCT elements included site institutional policies (83%), site challenges with technology adoption (61%) and site regulatory restraints (56%). Many sponsors intend to incorporate some degree of decentralization in future cancer clinical trials, with remote site monitoring (95%), remote collection of patient-reported outcomes (68%), telemedicine (68%), and electronic informed consent (58%) being deemed "highly likely" or "likely" for future adoption. Conclusions: The COVID-19 pandemic accelerated the use of DCT elements in cancer clinical trials. This study represents the first systematic evaluation of the use of DCT elements in cancer clinical trials intended for regulatory submission and provides proof of concept that cancer trials with DCT elements can generate data suitable for FDA review and marketing approval. The appropriate use of DCT elements holds promise to expand clinical trial access and reduce patient burden. Further characterization and mitigation of clinical site-level challenges is warranted to encourage continued adoption of DCT elements in cancer clinical trials.

Implementing paper-based patient-reported outcome collection within outpatient integrative health and medicine.

To investigate the feasibility of pre- and post-encounter patient-reported outcome (PRO) measure collection within an outpatient integrative health and medicine (IHM) clinic and to characterize factors associated with successful completion. We conducted a retrospective review of 27,464 outpatient IHM encounters including 9,520 chiropractic; 8,237 acupuncture; 5,847 massage; 2,345 IHM consultation; and 1,515 osteopathic manipulation treatment encounters at four clinics offering IHM over 18 months. Patients were asked to complete paper questionnaires rating pain, anxiety, and stress from 0-10 immediately pre- and post-encounter. Generalized linear mixed effect regression models were used to examine the relationship between demographic, clinical, and operational covariates and completing (1) pre-encounter and (2) paired (i.e., pre and post) PROs. Patients (N = 5587, mean age 49 years, 74% white, 77% female) generally presented for musculoskeletal conditions (81.7%), with a chief complaint of pain (55.1%). 21,852 (79.6%) encounters were among patients who completed pre-encounter PROs; 11,709/21,852 (53.6%) completed subsequent post-encounter PROs. Odds of PRO completion were more impacted by provider, operational, and clinical-level factors than patient factors. Covariates associated with increased odds of pre-encounter PRO completion included being female, having additional IHM encounters, and having a pain or anxiety complaint. Covariates associated with increased odds of paired PRO completion included being aged 31-40 vs. 51-60 years and having additional IHM encounters. Implementing a paper-based PRO collection system in outpatient IHM is feasible; however, collecting post-encounter PROs was challenging. Future endeavors should leverage the electronic health record and patient portals to optimize PRO collection and engage patients and clinical providers.

Implementation of the International Consortium of Health Outcomes Measurement CHD standard set in patients undergoing pulmonary valve replacement.

Despite the burden of CHD, a high cost and utilization condition, an implementation of long-term outcome measures is lacking. The objective of this study is to pilot the implementation of the International Consortium of Health Outcomes Measurement CHD standard set in patients undergoing pulmonary valve replacement, a procedure performed in mostly well patients with diverse CHD. Patients ≥ 8 years old undergoing catheterization-based pulmonary valve replacement were approached via various approaches for patient-reported outcomes, with a follow-up assessment at 3 months post-procedure. Implementation strategy analysis was performed via a hybrid type 2 design. Of the 74 patients undergoing pulmonary valve replacement, 32 completed initial patient-reported outcomes with variable response rates by strategy (email and in-person explanation 100%, email only 54%, and email followed by text/call 64%). Ages ranged 8-67 years (mean 30). Pre-procedurally, 34% had symptomatic arrhythmias, which improved post-procedure. For those in school, 43% missed ≥ 6 days per year, and over half had work absenteeism. Financial concerns were reported in 34%. Patients reported high satisfaction with life (50% [n = 16]) and health-related quality of life (90% [n = 26]). Depression symptoms were reported in 84% (n = 27) and anxiety in 62.5% (n = 18), with tendency towards improvement post-procedurally. Pilot implementation of the International Consortium of Health Outcomes Measurement CHD standard set in pulmonary valve replacement patients reveals a significant burden of disease not previously reported. Barriers to the implementation include a sustainable, automated system for patient-reported outcome collection and infrastructure to assess in real time. This provides an example of implementing cardiac outcomes set in clinical practice.

Follow-up for human papillomavirus-related oropharynx cancer concentrated on unequal symptom change (FOCUS): Prospective patient-reported outcome collection.

Post-treatment surveillance recommendations for oropharyngeal cancer do not vary with p16 status despite the differences in outcomes. The optimal algorithm personalizing follow-up for these patients remains undefined. Here, we evaluate the feasibility and utility of incorporating electronic patient-reported outcomes (ePROs) and circulating tumor DNA (ctDNA) into routine surveillance for patients treated for p16+ oropharynx cancer. A prospective registry was developed in which ePROs and ctDNA were incorporated into routine surveillance among patients with oropharynx cancer. ePROs were emailed monthly for 1 year and blood HPV ctDNA testing was performed every 3-6 months. The primary objective was to assess patient compliance with ePRO-based surveillance with adequate compliance defined as ≥85% of patients completing monthly ePROs. Sensitivity, specificity, and positive/negative predictive values to detect recurrence were calculated for ePROs, HPV ctDNA, or the combination. Of 122 patients who initially expressed interest, 76 completed the electronic consent process and 44/76 (58%) were compliant with monthly surveys over 1 year; thus adequate compliance was not achieved. Technical difficulties associated with ePRO receipt through email largely limited participation. Provider feedback was significantly associated with heightened ePRO compliance. One hundred and six patients had ctDNA testing with a mean number of three tests per patient. Sensitivity to detect recurrence was 75% for the combination of ePROs and ctDNA. Despite lower than anticipated compliance with ePROs, our findings show promise for incorporation of HPV ctDNA into surveillance paradigms for HPV-related oropharynx cancer with suggestions of methods to optimize ePRO formats for personalized surveillance.

Burden and management of severe asthma in Russia: results from international observational study

To assess clinical and demographic characteristics of severe asthma (SA) patients and their management in Russian Federation. This publication provides data for Russian part of population of the international observational study. In Phase I, retrospective analysis of medical records of patients with SA was performed with assessment of clinical and demographic data, medical history, comorbidities, treatment approaches and healthcare utilization. Phase II was a cross-sectional collection of patient-reported outcomes: level of asthma control assessed by ACT (Asthma Control Test) and health-related quality of life (HRQoL) measured using the EQ-5D-5L questionnaire. Phase I patients were enrolled into Phase II if they signed a written consent form. A total of 315 patients were included in Phase I of the study, 106 (33.6%) of them entered Phase II. Majority of study participants were either obese (n=103; 39.8%) or overweight (n=94; 36.3%). The most common comorbidities were cardiovascular diseases (n=217; 71.4%), followed by chronic respiratory diseases (n=198; 68.8%). There were 268 (85.1%) patients who had at least one exacerbation during last 12 months. Data for blood eosinophil count were available in 176 patients; 81.3% of them (n=143) had only one test in the last 12 months. The mean (SD) last available blood eosinophil count was 161.2 (181.2) cells/mm3. Serum Immunoglobulin E (IgE) value was known for 88 patients, and the mean (SD) last measured IgE value was 254.3 (249.7) ng/mL. Only 4.7% of Phase II participants had ACT scores indicative of controlled asthma (>20). As much as 74.5% had scores ≤15 suggesting uncontrolled disease. Most patients also had impaired HRQoL. Most SA patients had poor disease control with frequent exacerbations and high number of comorbidities. Blood eosinophils and IgE level measurements were not evaluated routinely which might be a barrier for appropriate phenotyping and treatment selection.

Gene Therapy in Hemophilia A: Achievements, Challenges, and Perspectives.

Strides in advancements of care of persons with hemophilia include development of long-acting factor replacement therapies, novel substitution and hemostatic rebalancing agents, and most recently approved gene therapy. Several decades of preclinical and clinical trials have led to development of adeno-associated viral (AAV) vector-mediated gene transfer for endogenous production of factor VIII (FVIII) in hemophilia A (HA). Only one gene therapy product for HA (valoctocogene roxaparvovec) has been approved by regulatory authorities. Results of valoctocogene roxaparvovec trial show significant improvement in bleeding rates and use of factor replacement therapy; however, sustainability and duration of response show variability with overall decline in FVIII expression over time. Further challenges include untoward adverse effects involving liver toxicity requiring immunosuppression and development of neutralizing antibodies to AAV vector rendering future doses ineffective. Real-life applicability of gene therapy for HA will require appropriate patient screening, infrastructure setup, long-term monitoring including data collection of patient-reported outcomes and innovative payment schemes. This review article highlights the success and development of HA gene therapy trials, challenges including adverse outcomes and variability of response, and perspectives on approach to gene therapy including shared decision-making and need for future strategies to overcome the several unmet needs.

Patient-centered perspectives on perioperative care

IntroductionThe collection and evaluation of patient-reported outcomes is essential to the development of patient and family centered care. Current patient surveying techniques are limited by delayed response times and restriction to specific health systems. The use of random-domain intercept technology (RDIT), by Real-Time Interactive World-Wide Intelligence (RIWI, Toronto, ON, Canada) mitigates current barriers by creating a dynamic real-time feedback environment and providing a mass sampling technique.MethodsRDIT was employed to survey a wide sample of respondents across the United States (US). Respondents who self-identified as having had a surgical procedure or cared for someone having a surgical procedure were included in the analysis.Results1,004 participants completed the survey and answered questions regarding demographics, perioperative details, sentiments on postoperative recovery, postoperative clinical endpoints, sentiments on healthcare professionals, and opinions on future surgical care.DiscussionThe results of this cross-sectional study identified areas with potential for improvement in the patient perioperative experience that could improve the patient experience. This novel use of RDIT provided a valuable tool for real-time feedback and mass sampling allowing the creation of a dynamic healthcare environment that fosters timely and targeted improvements to patient experiences and outcomes.

Understanding providers' perspectives using patient-reported outcomes in a pain management setting: a pilot study.

Aim: This pilot study evaluated an electronic patient-reported outcomes collection system in pain management to understand providers' experiences using the data, and how it affects their patient interaction and guides their clinical decision-making. Materials & methods: Using stratified convenience sampling, nine semi-structured interviews were conducted with consented pain physicians. The transcribed, de-identified interviews were coded and analyzed. Results: Although most physicians utilized patient reported outcomes (PROs), one-third reported no significant change in their practice since implementation and 56% stated it does not influence their treatment recommendations. Conclusion: Despite the importance of measuring the impact of chronic pain on quality of life, there are significant limitations to the real-world use of PRO that may limit the patient's assessment and care.

End-of-Life Symptom Burden among Patients with Cancer Who Were Provided Medical Assistance in Dying (MAID): A Longitudinal Propensity-Score-Matched Cohort Study.

Cancer is the primary underlying condition for most Canadians who are provided Medical Assistance in Dying (MAID). However, it is unknown whether cancer patients who are provided MAID experience disproportionally higher symptom burden compared to those who are not provided MAID. Thus, we used a propensity-score-matched cohort design to evaluate longitudinal symptom trajectories over the last 12 months of patients' lives, comparing cancer patients in Alberta who were and were not provided MAID. We utilized routinely collected retrospective Patient-Reported Outcomes (PROs) data from the Edmonton Symptom Assessment System (ESAS-r) reported by Albertans with cancer who died between July 2017 and January 2019. The data were analyzed using mixed-effect models for repeated measures to compare differences in symptom trajectories between the cohorts over time. Both cohorts experienced increasing severity in all symptoms in the year prior to death (β from 0.086 to 0.231, p ≤ .001 to .002). Those in the MAID cohort reported significantly greater anxiety (β = -0.831, p = .044) and greater lack of appetite (β = -0.934, p = .039) compared to those in the non-MAID cohort. The majority (65.8%) of patients who received MAID submitted their request for MAID within one month of their death. Overall, the MAID patients did not experience disproportionally higher symptom burden. These results emphasize opportunities to address patient suffering for all patients with cancer through routine collection of PROs as well as targeted and early palliative approaches to care.

Longitudinal comparison of the self-administered ALSFRS-RSE and ALSFRS-R as functional outcome measures in ALS

Objective Test the feasibility, adherence rates and optimal frequency of digital, remote assessments using the ALSFRS-RSE via a customized smartphone-based app. Methods This fully remote, longitudinal study was conducted over a 24-week period, with virtual visits every 3 months and weekly digital assessments. 19 ALS participants completed digital assessments via smartphone, including a digital version of the ALSFRS-RSE and mood survey. Interclass correlation coefficients (ICC) and Bland-Altman plots were used to assess agreement between staff-administered and self-reported ALSFRS-R pairs. Longitudinal change was evaluated using ANCOVA models and linear mixed models, including impact of mood and time of day. Impact of frequency of administration of the ALSFRS-RSE on precision of the estimate slope was tested using a mixed effects model. Results In our ALS cohort, digital assessments were well-accepted and adherence was robust, with completion rates of 86%. There was excellent agreement between the digital self-entry and staff-administered scores computing multiple ICCs (ICC range = 0.925–0.961), with scores on the ALSFRS-RSE slightly higher (1.304 points). Digital assessments were associated with increased precision of the slope, resulting in higher standardized response mean estimates for higher frequencies, though benefit appeared to diminish at biweekly and weekly frequency. Effects of participant mood and time of day on total ALSFRS-RSE score were evaluated but were minimal and not statistically significant. Conclusion Remote collection of digital patient-reported outcomes of functional status such as the ALSFRS-RSE yield more accurate estimates of change over time and provide a broader understanding of the lived experience of people with ALS.