Incorporating patient-reported outcome data into a predictive calculator for allogeneic hematopoietic cell transplantation recipients.

BACKGROUND: The Center for International Blood and Marrow Transplant Research (CIBMTR) provides a 1-year overall survival calculator to estimate outcomes for individual patients before they undergo allogeneic hematopoietic cell transplantation (HCT) to inform risk. The calculator considers pre-HCT clinical and demographic characteristics, but not patient-reported outcomes (PROs). Because pre-HCT PRO scores have been associated with post-HCT outcomes, the authors hypothesized that adding PRO scores to the calculator would enhance its predictive power. METHODS: Clinical data were obtained from the CIBMTR and the Blood and Marrow Transplant Clinical Trials Network. The PRO measures used were the 36-Item Short Form Survey (SF-36) and the Functional Assessment of Cancer Therapy-Bone Marrow Transplantation. One thousand thirty-three adult patients were included. RESULTS: When adjusted for clinical characteristics, the SF-36 physical component score was significantly predictive of 1-year survival (hazard ratio [HR], 0.88; 95% confidence interval [CI], 0.81-0.95; p = .0015), whereas the mental component score was not (HR, 1.02; 95% CI, 0.95-1.10; p = 0.6396). The baseline single general health question on the SF-36 was also significantly associated with mortality (HR, 1.91 for those reporting fair/poor health vs. good, very good, or excellent health; 95% CI, 1.33-2.76; p = .0005). The addition of PRO scores to the calculator did not result in a significant change in the model's predictive ability. Self-reported pre-HCT scores were strongly predictive of self-reported health status (odds ratio, 3.35; 95% CI, 1.66-6.75; p = .0007) and quality of life (odds ratio, 3.24; 95% CI, 1.93-5.41; p < .0001) after HCT. CONCLUSIONS: The authors confirmed the significant, independent association of pre-HCT PRO scores with overall survival, although adding PRO scores to the survival calculator did not improve its performance. They also demonstrated that a single general health question was as accurate as the full measure for predicting survival, an important finding that may reduce respondent burden and promote its inclusion in routine clinical practice. Validation of these findings should be performed.

Identifying barriers and facilitators to care for infants with bronchopulmonary dysplasia after NICU discharge: a prospective study of parents and clinical stakeholders.

OBJECTIVE: Understand barriers and facilitators to follow-up care for infants with bronchopulmonary dysplasia (BPD). METHODS: Qualitative study of parents and clinical stakeholders caring for infants with BPD. The interview guide was developed by a mother of a former 23-week preterm infant, neonatologist, pulmonologist, nurse, and qualitative researcher. Purposive sampling obtained a heterogenous sociodemographic and professional cohort. Subjects discussed their experience with BPD, barriers to care, caregiver quality of life and health education. Interviews were audio-recorded, transcribed and coded. Thematic analysis was used. RESULTS: Eighteen parents and 20 stakeholders completed interviews. Family-level themes included pragmatic barriers like transportation being multi-faceted; and caregiving demands straining mental health. System-level themes included caregiver education needing to balance immediate caregiving activities with future health outcomes; and integrating primary care, specialty, and community supports. CONCLUSIONS: Individual and system barriers impact follow-up for infants with BPD. This conceptual framework can be used to measure and improve care.

Decision regret among couples experiencing infertility: a mixed methods longitudinal cohort study.

BACKGROUND: Decisions for how to resolve infertility are complex and may lead to regret. We examined whether couples and individuals who sought a consultation from a reproductive specialist for infertility later expressed decisional regret about their family-building choices and whether regret was associated with parental role, family-building paths, or outcomes. METHODS: This longitudinal mixed methods study included women and their partners who completed a questionnaire prior to their initial consultation with a reproductive specialist and 6 years later. The six-year questionnaire included the Ottawa Decision Regret Scale referencing "the decisions you made about how to add a child to your family." A score of 25+ indicates moderate-to-severe regret. Additional items invited reflections on family-building decisions, treatments, and costs. A systematic content analysis assessed qualitative themes. RESULTS: Forty-five couples and 34 individuals participated in the six-year questionnaire (76% retention rate), Half (n = 61) of participants expressed no regret, which was similar by role (median 0 for women and supporting partners, F = .08; p = .77). One in 5 women and 1 in 7 partners expressed moderate-to-severe regret. Women who did not pursue any treatment had significantly higher regret (median 15; F = 5.6, p < 0.01) compared to those who pursued IVF (median 0) or other treatments (median 0). Women who did not add a child to their family had significantly higher regret (median 35; F = 10.1, p < 0.001) than those who added a child through treatment (median 0), through fostering/adoption (median 0), or naturally (median 5). Among partners, regret scores were not associated with family-building paths or outcomes. More than one-quarter of participants wished they had spent less money trying to add a child to their family. Qualitative themes included gratitude for parenthood despite the burdensome process of family-building as well as dissatisfaction or regret about the process. Results should be confirmed in other settings to increase generalizability. CONCLUSION: This longitudinal study provides new insight into the burden of infertility. For women seeking parenthood, any of the multiple paths to parenthood may prevent future decision regret. Greater psychosocial, financial, and decision support is needed to help patients and their partners navigate family-building with minimal regret.

When people experience infertility, there are many decisions that can be challenging, such as whether to seek fertility treatments, to pursue fostering/adoption, and how to manage costs. With each decision, there is an opportunity for regret. The goal of this study was to look at whether people who were experiencing infertility and made an appointment with a doctor who specializes in infertility felt any regret about their decisions 6 years later. We also looked at whether different roles (that is, women seeking pregnancy or their supporting partners), different family-building paths (that is, medical treatments or not), or different outcomes (that is, adding a child to their family or not) were associated with different levels of regret. Results showed that half of the 120 people in the study did not have any regret 6 years after meeting with a specialty doctor. However, some patients did have regret, including 20% of women and 14% of partners who expressed moderate-to-severe regret. Women who did not add a child to their family in the six years during the study reported higher regret compared to women who did add a child to their family. There were no such differences among partners. About 25% of participants wished they had tried more, fewer, or different treatments. More than 25% wished they spent less money to try to add a child to their family. For people who want to add a child to their family, there are multiple ways to become a parent, any of which may be linked to lower decision regret. Decision regret is experienced differently between women seeking to add a child to their family and their partners. Would-be parents need more emotional, financial, and decision making support to help them navigate family-building with minimal regret.

Identifying Barriers and Facilitators to Care for Infants with Bronchopulmonary Dysplasia After NICU Discharge: A Prospective Study of Parents and Clinical Stakeholders.

Objective: Understand barriers and facilitators to follow-up care for infants with bronchopulmonary dysplasia (BPD). Methods: Qualitative study of parents and clinical stakeholders caring for infants with BPD. The interview guide was developed by a mother of a former 23-week preterm infant, neonatologist, pulmonologist, nurse, and qualitative researcher. Purposive sampling obtained a heterogenous sociodemographic and professional cohort. Subjects discussed their experience with BPD, barriers to care, caregiver quality of life and health education. Interviews were audio-recorded, transcribed and coded. Thematic analysis was used. Results: Eighteen parents and 20 stakeholders completed interviews. Family-level themes included pragmatic barriers like transportation being multi-faceted; and caregiving demands straining mental health. System-level themes included caregiver education needing to balance process needs with future trajectories; and integration of primary care, specialty care, and community supports. Conclusions: Individual and system barriers impact follow-up for infants with BPD. This conceptual framework can be used to measure and improve care.

Prevalence of Sleep Aid Medication Use in Patients Receiving a Hematopoietic Cell Transplant on an Inpatient Unit.

BACKGROUND AND OBJECTIVES: There is a lack of research regarding the use of sleep aids after hematopoietic stem cell transplantation (HCT). We describe the prevalence of sleep aid administration in the HCT unit and identify associations with patient or clinical characteristics. PATIENTS AND METHODS: In this retrospective analysis of sequential inpatient HCTs from July 1 to December 31, 2016 we describe whether and when patients were prescribed sleep aid medications. Chi-square tests determined significant differences between patient characteristics, sleep aid prescription, and time of prescription. RESULTS: Of the 225 patients identified, 193 (86%) were prescribed sleep aids. Significantly more women received prescriptions for sleep aids (90.4%) than men (81%; P = .047). One hundred patients (44%) received prescriptions exclusively while in the hospital. CONCLUSION: Findings show a high prevalence of sleep medication use in patients undergoing inpatient HCT, primarily during hospitalization. Future efforts toward standardized recommendations to optimize peri-transplant sleep would help clinicians and patients.

Real-World Data Showing Trends and Outcomes by Race and Ethnicity in Allogeneic Hematopoietic Cell Transplantation: A Report from the Center for International Blood and Marrow Transplant Research.

The use of HLA-mismatched donors could enable more patients with ethnically diverse backgrounds to receive allogeneic hematopoietic cell transplantation (HCT) in the United States. However, real-world trends and outcomes following mismatched donor HCT for diverse patients remain largely undefined. We conducted this study to determine whether the use of mismatched donor platforms have increased the access to allogeneic HCT for ethnically diverse patients, particularly through the application of novel graft-versus-host disease (GVHD) prophylaxis regimens, and whether outcomes for diverse patients are comparable to those of non-Hispanic White patients. This observational cross-sectional study used real-world data from the Center for International Blood and Marrow Transplant Research (CIBMTR) registry. All patients receiving their first allogeneic HCT in the United States between 2009 and 2020 were included, with a focus on transplantations performed in 2020. Data from patients undergoing allogeneic HCT using bone marrow, peripheral blood, or cord blood from HLA-matched or mismatched related and unrelated donors were analyzed. Specifically, relative proportion of allogeneic HCT was generated as percentage of total for donor type and for patient age, disease indication, GVHD prophylaxis, and race and ethnicity. Causes of death were summarized using frequencies, and the Kaplan-Meier estimator was used for estimating overall survival. Compared to matched related donor and matched unrelated donor HCT, more ethnically diverse patients received mismatched unrelated donor, haploidentical donor, and cord blood HCT. Although matched unrelated donor remains the most common donor type, the use of haploidentical donors has increased significantly over the last 5 years. Paralleling this increase in haploidentical HCT is the increased use of post-transplantation cyclophosphamide (PTCy) as GVHD prophylaxis. Relative to previous transplantation eras, the most contemporary era is associated with the highest survival rates following allogeneic HCT irrespective of patient race and ethnicity. Nonetheless, disease relapse remains the primary cause of death for both adult and pediatric allogeneic HCT recipients by donor type and across all patient racial/ethnic groups. Ethnically diverse patients are undergoing allogeneic HCT at higher rates, largely through the use of alternative donor platforms incorporating PTCy. Maintaining access to potential life-saving allogeneic HCT using alternative donors and novel GVHD prophylaxis strategies and improving HCT outcomes, particularly disease relapse, remain urgent clinical needs.

Multi-Stakeholder Qualitative Interviews to Inform Measurement of Patient Reported Outcomes After CAR-T.

Toxicities after chimeric antigen receptor T cell (CAR-T) therapy are well known, yet the patient experience during and after CAR-T therapy has not been well described outside of the trial setting. We explored the patient experience after CAR-T therapy to inform the patient-reported outcomes (PRO) measurement approach for the Center for International Blood and Marrow Transplant Research (CIBMTR). We recruited (1) adult patients diagnosed with a hematologic malignancy 14 days to 6 months after receiving a commercial CAR T cell product who had agreed to be contacted by the CIBMTR, (2) caregivers of those patients, and (3) clinical experts in CAR-T therapy. Telephone interviews were conducted following a semistructured guide that included open-ended questions about symptoms and functioning. We conducted a systematic content analysis of each transcript using prespecified codes representing common domains of health, as well as open coding for emergent themes. Forty patients at 29 centers, 15 of their caregivers, and 15 experts from 9 centers participated, representing diversity with respect to age, sex, race/ethnicity, and years in practice (experts). Patients, caregivers, and experts shared largely consistent impressions of the patient experience after CAR-T therapy. Commonly described themes included anxiety, cognitive dysfunction, depression, fatigue, pain, impaired physical function, gastrointestinal symptoms, sexual dysfunction, sleep difficulties, need for support, financial impact, hospitalization, communication with healthcare providers, and the COVID-19 pandemic. Limitations in patients' ability to participate in social roles and activities was the most prevalent theme, found in nearly all interviews. In the setting of CAR-T therapy, a multidimensional approach to PRO measurement is needed that includes physical, mental, and social health, as well as the financial impact of this novel treatment. High-quality existing PRO tools are available to measure these concepts. Results will inform the CIBMTR measurement of PROs after CAR-T therapy and may be applicable to other CAR-T studies that aim to represent patient experiences.

Health-related quality of life in reduced-intensity hematopoietic cell transplantation based on donor availability in patients aged 50-75 with advanced myelodysplastic syndrome: BMT CTN 1102.

For myelodysplastic syndrome (MDS), allogeneic hematopoietic cell transplantation (alloHCT) is the only available curative therapy. The Blood and Marrow Transplant Clinical Trials Network study 1102 (BMT CTN 1102, NCT02016781) was a multicenter, biologic assignment trial based on matched donor availability in adults aged 50-75 with higher risk de novo MDS who were candidates for reduced-intensity conditioning (RIC) alloHCT. The primary analysis showed that those who received alloHCT had a survival benefit, but whether this is at the cost of worse quality of life (QOL) has not been described in detail. English- and Spanish-speaking trial participants completed the Functional Assessment of Cancer Therapy-General (FACT-G), the SF-36, and the EQ-5D, at enrollment, every 6 months until 24 months, and 36 months. We compared patient-reported outcome (PRO) scores between study arms using an inverse probability weighted-independent estimating equation (IPW-IEE) model. Between January 2014 and November 2018, 384 subjects (median age 66.7 years, range: 50.1-75.3) enrolled at 34 centers. PRO completion rates were generally high at 65%-78%. The PRO trajectories for both arms were similar, with most decreasing or stable from baseline to 6 months and improving thereafter. Baseline PRO scores were the most consistent independent predictors of subsequent QOL outcomes and survival, even after controlling for clinical and patient-level factors. For older adults with MDS, the survival advantage associated with donor availability and alloHCT did not come at the cost of worse QOL. These results should reassure older patients and clinicians who prefer a curative approach to treating MDS.

Navigating the perils and pitfalls throughout the consent process in hematopoietic cell transplantation.

Hematopoietic cell transplantation (HCT) is a complex treatment used in malignancies and some non-malignant diseases. The informed consent process for HCT can also be complex due to patient- and process-related barriers. The informed consent process needs to be a dynamic and ongoing process, not simply a checklist. As a result of the realities of HCT, we highlight some potential pitfalls to the informed consent process including uncertainty, sociocultural and communication barriers, and decisional regret. The purpose of this comprehensive review is to highlight unique situations which can result in failure of the informed consent process. We also offer potential solutions to these pitfalls, primarily making the informed consent more patient focused through dynamic and continuous processes to mitigate decisional regret.

Patient-reported cognitive function among hematopoietic stem cell transplant and cellular therapy patients: a scoping review.

PURPOSE: Cognitive dysfunction is a known complication following cellular therapies (CT), which can be assessed through performance based and patient-reported measures. We performed a systematic scoping review to assess self-reported cognitive function measures used among adult CT patients and describe long-term results, including associations with clinical outcomes. METHODS: Library databases were searched from inception to February 2020 according to PRISMA guidelines. Additional studies were identified through reference lists and trial protocols. Two members of the research team screened titles and abstracts and resolved discrepancies. Articles that met eligibility criteria continued to full-text review, with 25% double screening. Articles were removed if they (1) were not original research, peer-reviewed articles; (2) were the wrong disease, age, or treatment-specific patient population; (3) did not use patient-reported outcomes; (4) did not separately report cognitive function outcomes. RESULTS: Of the1952 articles, 56 were included. Twenty-one patient-reported measures of cognitive function were used; most frequently the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC-QLQ-C30), which includes a two-item cognitive function subscale (57%; n = 32). Thirteen studies collected performance-based and self-reported measures and of those (n = 6) who assessed associations found moderate correlations (range r = .13-.58). Longitudinal patterns showed declines in cognitive function soon after treatment (< 1 month) returning to baseline at 1 year. Cognitive function was often associated with other quality of life measures, chiefly depression (n = 5). CONCLUSIONS: EORTC-QLQ-C30 is the most commonly used to measure, though there remain numerous measures used, including several measures with little previous validation and investigator developed items.

Associations between Relationship Status, Anxiety, Sexual Function, and Sexual Satisfaction in Lesbian, Gay, and Bisexual People.

Objectives: The purpose of this study was to describe associations between relationship status, anxiety, and sexual function and satisfaction in lesbian, gay, and bisexual (LGB) adults. Methods: Linear regression determined associations between sexual function, relationship characteristics and anxiety in 979 LGB participants. Results: Relationship status was not related to sexual interest in gay men. Partnered relationship status was associated with higher sexual satisfaction for gay men, lesbians, and bisexual women. Anxiety symptoms were negatively associated with sexual interest, satisfaction, and orgasm pleasure for gay men but not associated with sexual outcomes in lesbian women. Conclusion: Using a nationally representative sample of LGB adults, differences were found in factors related to sexual function and satisfaction by gender and sexual orientation.

Twenty-first century house calls: a survey of ambulatory care providers to inform organisational telehealth strategy.

OBJECTIVES: While patient interest in telehealth increases, clinicians' perspectives may influence longer-term adoption. We sought to identify facilitators and barriers to continued clinician incorporation of telehealth into practice. METHODS: A cross-sectional 24-item web-based survey was emailed to 491 providers with ≥50 video visits (VVs) within an academic health system between 1 March 2020 and 31 December 2020. We quantitatively summarised the characteristics and perceptions of respondents by using descriptive and test statistics. We used systematic content analysis to qualitatively code open-ended responses, double coding at least 25%. RESULTS: 247 providers (50.3%) responded to the survey. Seventy-nine per cent were confident in their ability to deliver excellent clinical care through VV. In comparison, 48% were confident in their ability to troubleshoot technical issues. Most clinicians (87%) expressed various concerns about VV. Providers across specialties generally agreed that VV reduced infection risk (71%) and transportation barriers (71%). Three overarching themes in the qualitative data included infrastructure and training, usefulness and expectation setting for patients and providers. DISCUSSION: As healthcare systems plan for future delivery directions, they must address the tension between patients' and providers' expectations of care within the digital space. Telehealth creates new friction, one where the healthcare system must fit into the patient's life rather than the usual dynamic of the patient fitting into the healthcare system. CONCLUSION: Telehealth infrastructure and patient and clinician technological acumen continue to evolve. Clinicians in this survey offered valuable insights into the directions healthcare organisations can take to right-size this healthcare delivery modality.

Real-world implementation evaluation of an electronic health record-integrated consumer informatics tool that collects patient-generated contextual data.

OBJECTIVE: Use the RE-AIM framework to examine the implementation of a patient contextual data (PCD) Tool designed to share patients' needs, values, and preferences with care teams ahead of clinical encounters. MATERIALS & METHODS: Observational study that follows initial PCD Tool scaling across primary care at a Midwestern academic health network. Program invitations, enrollment, patient submissions, and clinician views were tracked over a 1-year study period. Logistic regression modeled the likelihood of using the PCD Tool, accounting for patient covariates. RESULTS: Of 58,874 patients who could be contacted by email, 9,183 (15.6%) became PCD Tool users. Overall, 76% of primary care providers had patients who used the PCD Tool. Older age, female gender, non-minority race, patient portal activation, and Medicare coverage were significantly associated with increased likelihood of use. Number of office visits, medical issues, and behavioral health conditions also associated with use. Primary care staff viewed 18.7% of available PCD Tool summaries, 1.1% to 57.6% per clinic. DISCUSSION: The intervention mainly reached non-minority patients and patients who used more health services. Given the requirement for an email address on file, some patients may have been underrepresented. Overall, patient reach and adoption and clinician adoption, implementation, and maintenance of this Tool were modest but stable, consistent with a non-directive approach to fostering adoption by introducing the Tool in the absence of clear expectations for use. CONCLUSION: Healthcare organizations must implement effective methods to increase the reach, adoption, implementation, and maintenance of PCD tools across all patient populations. Assisting people, particularly racial minorities, with PCD Tool registration and actively supporting clinician use are critical steps in implementing technology that facilitates care.

Patient-reported outcomes and neurotoxicity markers in patients treated with bispecific LV20.19 CAR T cell therapy.

Background: With the rising number of chimeric antigen receptor (CAR) T cell treated patients, it is increasingly important to understand the treatment's impact on patient-reported outcomes (PROs) and, ideally, identify biomarkers of central nervous system (CNS) adverse effects. Methods: The purpose of this exploratory study was to assess short-term PROs and serum kynurenine metabolites for associated neurotoxicity among patients treated in an anti-CD20, anti-CD19 (LV20.19) CAR T cell phase I clinical trial (NCT03019055). Fifteen CAR T treated patients from the parent trial provided serum samples and self-report surveys 15 days before and 14, 28, and 90 days after treatment. Results: Blood kynurenine concentrations increased over time in patients with evidence of neurotoxicity (p = 0.004) and were increased in self-reported depression (r = 0.52, p = 0.002). Depression improved after CAR T infusion (p = 0.035). Elevated 3-hydroxyanthranilic acid (3HAA) concentrations prior to cell infusion were also predictive of neurotoxicity onset (p = 0.031), suggesting it is a biomarker of neurotoxicity following CAR T cell therapy. Conclusions: Elevated levels of kynurenine pathway metabolites among CAR T cell recipients are associated with depressed mood and neurotoxicity. Findings from this exploratory study are preliminary and warrant validation in a larger cohort.

This study examined the impact of chimeric antigen receptor (CAR) T cell therapy­a therapy that gets immune cells to fight cancer by changing them in the lab to find and destroy cancer cells­on blood markers associated with depression, anxiety, pain, fatigue, and poor sleep. Fifteen CAR T cell patients provided blood samples and completed surveys before and three timepoints after treatment. We found that the amount of kynurenine, a normal blood constituent, and related molecules was higher in patients who experienced significant CAR T cell side effects on the brain and in patients reporting more depression. These results identify the excessive elevation of blood constituents related to the mood that may also be associated with depression and brain dysfunction following CAR T. These blood constituents could potentially be used as markers and targeted with interventions to prevent brain dysfunction.

Updated Trends in Hematopoietic Cell Transplantation in the United States with an Additional Focus on Adolescent and Young Adult Transplantation Activity and Outcomes.

Hematopoietic cell transplantation (HCT) has been successfully used to treat many malignant and nonmalignant conditions. As supportive care, donor selection, and treatment modalities evolve, documenting HCT trends and outcomes is critical. This report from the Center for International Blood and Marrow Transplant Research (CIBMTR) provides an update on current transplantation activity and survival rates in the United States. Additional data on the use and outcomes of HCT in the adolescent and young adult (AYA) population are included. AYA patients more frequently receive peripheral blood stem cell grafts than pediatric patients, which may reflect differences in practice in pediatric versus adult treatment centers. The proportions of donor types also differ those in adult and pediatric populations. Outcomes for patients in the AYA age range are similar to those of pediatric patients for acute myelogenous leukemia but worse for acute lymphoblastic leukemia. Outcomes for both leukemias are better in AYA patients compared with older adults. Comparing the time periods 2000 to 2009 and 2010 to 2019 revealed significant improvement in overall survival across the age spectrum, but the greatest improvement in the AYA age group.

Effective questionnaire design: How to use cognitive interviews to refine questionnaire items.

BACKGROUND: Cognitive interviewing is a technique that can be used to improve and refine questionnaire items. We describe the basic methodology of cognitive interviewing and illustrate its utility through our experience using cognitive interviews to refine a questionnaire assessing parental understanding of concepts related to preterm birth. METHODS: Cognitive interviews were conducted using current best practices. Results were analyzed by the multidisciplinary research team and questionnaire items that were revealed to be problematic were revised. RESULTS: Revisions to the questionnaire items were made to improve clarity and to elicit responses that truly reflected the participants understanding of the concept. CONCLUSION: Cognitive interviewing is a useful methodology for improving validity of questionnaire items, we recommend researchers developing new questionnaire items design and complete cognitive interviews to improve their items and increase confidence in study conclusions.

Development of a conceptual model of patient-reported outcomes in light chain amyloidosis: a qualitative study.

BACKGROUND: Light chain (AL) amyloidosis is a plasma cell neoplasm associated with high early mortality and severe morbidity that can cause severe disability. We explored the impact of AL amyloidosis on symptoms and well-being from the perspectives of patients and health care providers who regularly care for AL patients. We intended to develop a conceptual understanding of patient-reported outcomes in AL amyloidosis to identify the context of use and concept of interest for a clinical outcome assessments tool in this disease. METHOD: Twenty patients and ten professionals were interviewed. Patient interviews captured the spectrum of amyloidosis experience including time from diagnosis, type of organ involvement, and presence and type of treatment received. Interviews with professionals included physicians, advanced practice providers, registered nurse, and a patient advocate; these interviews covered similar topics. RESULTS: The impact of AL amyloidosis on patients' life was multidimensional, with highly subjective perceptions of normality and meaning. Four major themes from patients and experts included diagnosis of AL amyloidosis, living with AL amyloidosis, symptom burden, and social roles. Barriers to patient-reported outcomes data collection in patients were additionally explored from experts. The themes provide a comprehensive understanding of the important experiences of symptom burden and its impact on daily life from AL amyloidosis patients' and from the perspectives of professionals who care for patients with AL amyloidosis. CONCLUSION: These findings further the conceptual understanding and identification of a preliminary model of concept of interest for development of a clinical outcome assessments tool for AL amyloidosis.

Adding Centralized Electronic Patient-Reported Outcome Data Collection to an Established International Clinical Outcomes Registry.

The importance of patient-reported outcomes (PROs) in cellular therapies, including hematopoietic cell transplantation (HCT) is highlighted in this study. Longitudinal collection of PROs in a registry is recommended for several reasons, yet to date, PROs are not routinely collected from HCT patients to augment clinical registry data. The aim of this study was to determine the feasibility of electronic PRO data collection by a national clinical outcomes registry, by assessing differences between who does and does not report PROs. We conducted a cross-sectional pilot collection of PROs from HCT recipients after treatment using computer-adapted tests from the Patient-Reported Outcome Measurement Information System (PROMIS). We implemented centralized data collection through the Center for International Blood and Marrow Transplant Research (CIBMTR) among patients who underwent HCT for myelodysplastic syndromes (MDS), were at least 6 months post-HCT, and spoke English or Spanish. The main objective was identifying patient, disease, and transplant-related differences associated with completion of electronic PROs. Patients were excluded from analysis if they were determined to be ineligible (deceased, did not speak English or Spanish, refused to be contacted by the CIBMTR). A total of 163 patients were contacted and potentially eligible to participate; of these, 92 (56%) enrolled and 89 (55%) completed the PRO assessment. The most frequent reason for incomplete surveys was inability to contact patients (n = 88), followed by declining to participate in the study (n = 37). There were no sociodemographic or age differences between those who completed the PRO survey (n = 89) and eligible nonresponders (n = 155). Patient scores were within 3 points of the US average of 50 for all symptoms and functioning except physical functioning. Responders and nonresponders did not exhibit meaningfully different sociodemographic characteristics. Difficulty contacting patients posed the greatest barrier and also provided the greatest opportunity for improvement. Once enrolled, survey completion was high. These results support standardizing centralized PRO data collection through the CIBMTR registry.

Impact of Pre-visit Contextual Data Collection on Patient-Physician Communication and Patient Activation: a Randomized Trial.

BACKGROUND: Patient contextual data (PCD) are often missing from electronic health records, limiting the opportunity to incorporate preferences and life circumstances into care. Engaging patients through tools that collect and summarize such data may improve communication and patient activation. However, differential tool adoption by race might widen health care disparities. OBJECTIVE: Determine if a digital tool designed to collect and present PCD improves communication and patient activation; secondarily, evaluate if use impacts outcomes by race. DESIGN, SETTING, AND PARTICIPANTS: A pragmatic, two-armed, non-blinded, randomized controlled trial conducted during 2019 in a primary care setting. INTERVENTION: The PCD tool (PatientWisdom) invited patients to identify preferences, values, goals, and barriers to care. Patients were randomized to a standard pre-visit email or facilitated enrollment with dedicated outreach to encourage use of the tool. MAIN OUTCOMES AND MEASURES: Outcomes of interest were post-visit patient communication and patient activation measured by the Communication Assessment Tool (CAT) and Patient Activation Measure (PAM), respectively. Outcomes were evaluated using treatment-on-the-treated (TOT) and intention-to-treat (ITT) principles. KEY RESULTS: A total of 301 patients were enrolled. Facilitated enrollment resulted in a five-fold increase in uptake of the PCD tool. TOT analysis indicated that the PCD tool was associated with notable increases in specific CAT items rated as excellent: "treated me with respect" (+ 13 percentage points; p = 0.04), "showed interest in my ideas" (+ 14 percentage points; p = 0.03), "showed care and concern" (+ 16 percentage points; p = 0.02), and "spent about the right amount of time with me" (+ 11 percentage points; p = 0.05). There were no significant pre/post-visit differences in PAM scores between arms (- 4.41 percentage points; p = 0.58). ITT results were similar. We saw no evidence of the treatment effect varying by race in ITT or TOT analyses. CONCLUSIONS AND RELEVANCE: The inclusion of PCD enhanced essential aspects of patient-provider communication but did not affect patient activation. Outcomes did not differ by race. TRIAL REGISTRATION: Clincaltrials.gov identifier: NCT03766841.