Evolution of a research team: the patient partner perspective.

INTRODUCTION: Despite a movement toward the inclusion of patient partners or advisors as part of the research team in all funded studies, few publications have discussed patient engagement from the patient partners' perspective. METHODS: Qualitative interviews were conducted by independent qualitative researchers to collect and summarize the experiences and perspectives of the 16 Patient Partners (PPs) on the study team for PeRson EmPowered Asthma RElief (PREPARE), a large, pragmatic study of 1200 African American/Black (AA/B) and Hispanic/Latinx (H/L) adults with asthma. This study was funded by the Patient-Centered Outcomes Research Institute. RESULTS: This paper, authored by the PPs themselves, summarizes qualitative interview findings. The journey of the PREPARE PPs began with a desire to learn more about asthma and advocate for other individuals with asthma. Many challenges, including intimidation and lack of trust, were overcome as the research team prioritized building a comfortable environment in which PPs' lived experiences, opinions, and cultural beliefs are valued, and in which PP voices are centered and respected. Over time, the PPs gained confidence in expressing ideas and feedback, and in taking ownership of their role as valued members of the research team. The PP experience has had tremendous personal and professional impact on the PPs themselves, while also modeling a change in the way researchers and PPs relate to and partner with each other. CONCLUSION: The partnership between PPs and researchers in the PREPARE study has elevated the PP role from external advisors to integral and empowered members of a collective research team, and the partnership developed and evolved over time.

Patients and caregivers who have lived with a condition have an important and unique perspective researchers should consider. To this end, patients and caregivers have expanded their involvement in the design and conduct of clinical research, joining research teams and being engaged in the research process from start to finish. Researchers have reported on the positive impact that these "patient partners" or "patient advisors" have on research. But few papers report on the impact on the patient partners/advisors from being on a research team.Here we report the lived experiences of the 16 Patient Partners who served on the research team for a large study of 1200 African American/Black (AA/B) and Hispanic/Latinx (H/L) adults with asthma. These Patient Partners offered their input over 7 years, spanning from study design, study conduct, understanding results, to sharing results.To understand the Patient Partners' experiences, we conducted interviews asking about their journey on the research team. Motivations and reasons they became Patient Partners Initial experiences with the research team If and how over time, they became comfortable and developed confidence If and how the research team made them feel valued and respected The personal and professional impact of being a Patient Partner Their advice to researchers and fellow patients considering including or joining as Patient Partners or advisors By reporting the perspectives of these 16 Patient Partners, we hope to support continued movement toward broader and better inclusion of patients and caregivers on research teams.

Reliever-Triggered Inhaled Glucocorticoid in Black and Latinx Adults with Asthma.

BACKGROUND: Black and Latinx patients bear a disproportionate burden of asthma. Efforts to reduce the disproportionate morbidity have been mostly unsuccessful, and guideline recommendations have not been based on studies in these populations. METHODS: In this pragmatic, open-label trial, we randomly assigned Black and Latinx adults with moderate-to-severe asthma to use a patient-activated, reliever-triggered inhaled glucocorticoid strategy (beclomethasone dipropionate, 80 µg) plus usual care (intervention) or to continue usual care. Participants had one instructional visit followed by 15 monthly questionnaires. The primary end point was the annualized rate of severe asthma exacerbations. Secondary end points included monthly asthma control as measured with the Asthma Control Test (ACT; range, 5 [poor] to 25 [complete control]), quality of life as measured with the Asthma Symptom Utility Index (ASUI; range, 0 to 1, with lower scores indicating greater impairment), and participant-reported missed days of work, school, or usual activities. Safety was also assessed. RESULTS: Of 1201 adults (603 Black and 598 Latinx), 600 were assigned to the intervention group and 601 to the usual-care group. The annualized rate of severe asthma exacerbations was 0.69 (95% confidence interval [CI], 0.61 to 0.78) in the intervention group and 0.82 (95% CI, 0.73 to 0.92) in the usual-care group (hazard ratio, 0.85; 95% CI, 0.72 to 0.999; P = 0.048). ACT scores increased by 3.4 points (95% CI, 3.1 to 3.6) in the intervention group and by 2.5 points (95% CI, 2.3 to 2.8) in the usual-care group (difference, 0.9; 95% CI, 0.5 to 1.2); ASUI scores increased by 0.12 points (95% CI, 0.11 to 0.13) and 0.08 points (95% CI, 0.07 to 0.09), respectively (difference, 0.04; 95% CI, 0.02 to 0.05). The annualized rate of missed days was 13.4 in the intervention group and 16.8 in the usual-care group (rate ratio, 0.80; 95% CI, 0.67 to 0.95). Serious adverse events occurred in 12.2% of the participants, with an even distribution between the groups. CONCLUSIONS: Among Black and Latinx adults with moderate-to-severe asthma, provision of an inhaled glucocorticoid and one-time instruction on its use, added to usual care, led to a lower rate of severe asthma exacerbations. (Funded by the Patient-Centered Outcomes Research Institute and others; PREPARE ClinicalTrials.gov number, NCT02995733.).

Adherence to adding inhaled corticosteroids to rescue therapy in a pragmatic trial with adults with asthma: A pilot study.

BACKGROUND: Underuse of guideline-recommended inhaled corticosteroids (ICS) controller therapy is a risk factor for greater asthma burden. ICS concomitantly used with rescue inhalers (Patient-Activated Reliever-Triggered ICS ['PARTICS']) reduced asthma exacerbations in efficacy trials, but whether PARTICS is effective in pragmatic trials is unknown. OBJECTIVE: We conducted this pilot to determine the feasibility of executing a large-scale pragmatic PARTICS trial and to improve study protocols. METHODS: Four sites recruited 33 Hispanic or black adults with persistent asthma, randomized them approximately 3:1 to intervention or usual care, and followed them for 12 weeks. All participants received asthma guideline-based educational videos; intervention participants received video-based instructions on implementing PARTICS plus usual medications. The study involved 1 randomization visit and monthly questionnaires. Timely questionnaire responses (±2 weeks) were monitored. Participants underwent qualitative phone interviews to assess self-reported adherence to PARTICS and understand barriers to completing study procedures. RESULTS: Timely questionnaire response rates were 61%, 64%, and 70% at 4, 8, and 12 weeks, respectively. Self-reported adherence to PARTICS was 76% (95% confidence interval [CI], 58%-94% [n = 21]), 88% (95%CI, 72%-100% [n = 16]), and 62% (95%CI, 36%-88% [n = 13]) at weeks 1, 6, and 12, respectively. Barriers to completing study procedures included difficulties with questionnaire access, remembering to use ICS and rescue inhalers together, and obtaining refills. Only 22% of participants recognized their short-acting bronchodilator as "reliever" or "rescue." CONCLUSION: Recruitment was feasible within the allocated period. Adherence to PARTICS was incomplete, questionnaire completion was suboptimal, and common rescue inhaler nomenclature usage was limited. We have modified the full study protocol to attempt to improve adherence to PARTICS and minimize barriers to study procedures. CLINICAL TRIALS REGISTRATION: pilot study for 'PeRson EmPowered Asthma Relief' (PREPARE, NCT02995733).

The asthma Symptom Free Days Questionnaire: how reliable are patient responses?

Objective: Symptom free days are a widely used patient-reported outcome (PRO) in asthma clinical trials. We assessed the internal consistency of one instrument for this PRO, the Symptom Free Days Questionnaire (SFDQ), in a population of Black adults with asthma enrolled in the Blacks and Exacerbations on Long-acting beta agonists and Tiotropium (BELT) trial. Methods: We assessed responses to the SFDQ collected at baseline, 6 and 12 months as part of the BELT trial. The internal consistency of responses, specifically number of patient-reported days with symptoms in 14 days were compared to the number of patient-reported days with no symptoms in the same 14 days. Lin concordance correlation coefficients (Lin ccc) were calculated over time to assess "learning" and by age, sex, geographic location, and annual family income. Results: The internal consistency of the responses of the 1070 enrolled patients was consistently low over the 12 months of the study; varying from 43.8% at baseline to 52.1% at 12 months. This corresponded to Lin cccs of 0.33-0.32 over the study period. Internal consistency and the Lin ccc did not vary by age group, sex, geographic location or percent poverty. Concordance was slightly but not significantly higher at all time points in those with family annual income of ≥$50,000 compared to those with lower annual incomes. Conclusions: The SFDQ did not work well in the BELT population of Black adults with asthma. Further validation is required before the SFDQ is used in other large clinical trials with any population.

Anticholinergic vs Long-Acting ß-Agonist in Combination With Inhaled Corticosteroids in Black Adults With Asthma: The BELT Randomized Clinical Trial.

IMPORTANCE: The efficacy and safety of long-acting ß-agonists (LABAs) have been questioned. Black populations may be disproportionately affected by LABA risks. OBJECTIVE: To compare the effectiveness and safety of tiotropium vs LABAs, when used with inhaled corticosteroids (ICS) in black adults with asthma and to determine whether allelic variation at the Arg16Gly locus of the ß2-adrenergic receptor (ADRB2) geneis associated with treatment response. DESIGN, SETTING, AND PARTICIPANTS: A multisite (n = 20), open-label, parallel-group, pragmatic randomized clinical trial conducted from March 2011 through July 2013, enrolling black adults with moderate to severe asthma in the United States. INTERVENTIONS: Patients eligible for, or receiving, step 3 or step 4 combination therapy per National Heart, Lung, and Blood Institute guidelines, received ICS plus either once-daily tiotropium (n = 532) or twice-daily LABAs (n = 538,) and were followed up for up to 18 months. Patients underwent genotyping, attended study visits at baseline, 1, 6, 12, and 18 months, and completed monthly questionnaires. MAIN OUTCOMES AND MEASURES: The primary outcome was time to asthma exacerbation, defined as a worsening asthma event requiring oral or parenteral corticosteroids. Secondary outcomes included patient-reported outcomes (Asthma Quality of Life Questionnaire, Asthma Control Questionnaire [ACQ], Asthma Symptom Utility Index, and Asthma Symptom-Free Days questionnaire), spirometry (FEV1), rescue medication use, asthma deteriorations, and adverse events. RESULTS: There was no difference between LABA + ICS vs tiotropium + ICS in time to first exacerbation (mean No. of exacerbations/person-year, 0.42 vs 0.37 (rate ratio, 0.90 [95% CI, 0.73 to 1.11], log-rank P = .31). There was no difference in change in FEV1 at 12 months (0.003 L for LABA + ICS vs -0.018 L for tiotropium + ICS; between-group difference, 0.020 [95% CI, -0.021 to 0.061], P = .33) and at 18 months (-0.053 L vs -0.078 L; between-group difference, 0.025 [95% CI, -0.045 to 0.095], P = .49). There were no differences in ACQ score at 18 months (change in score from baseline, -0.68 for LABA + ICS vs -0.72 for tiotropium + ICS; between-group difference, 0.04 [95% CI, -0.18 to 0.27], P = .70). There were no differences in other patient-reported outcomes. Arg16Gly ADRB2 alleles were not associated with differences in the effects of tiotropium + ICS vs LABA + ICS (hazard ratio for time to first exacerbation, 0.84 [95% CI, 0.47 to 1.51] for Arg/Arg vs 0.85 [95% CI, 0.63 to 1.15] for Arg/Gly or Gly/Gly, P = .97). CONCLUSIONS/RELEVANCE: Among black adults with asthma treated with ICS, adding a LABA did not improve time to asthma exacerbation compared with adding tiotropium. These findings were not affected by polymorphisms at the Arg16Gly locus of ADRB2. These findings do not support the superiority of LABA + ICS compared with tiotropium + ICS for black patients with asthma. TRIAL REGISTRATION: ClinicalTrials.gov identifier: NCT01290874.

Preventing the voltage drop: keeping practice-based research network (PBRN) practices engaged in studies.

INTRODUCTION: Practice-based research continues to evolve and has become a major methodology for many pragmatic studies. While early practice-based network projects were usually short term, current studies often introduce or compare practice innovations that require long-term evaluation. That change requires that practice sites remain engaged in research work for up to 5 years, a time that can allow for a significant "voltage drop," or decline in active participation. METHODS: Over the past 15 years we have developed and adapted several strategies to facilitate and encourage the continued active engagement of practices in practice-based research network studies of up to 5 years' duration. The concepts, details, evaluation, and results (when available) of the strategies are described. RESULTS: Eight strategies that enhance practice sites' attention to enrollment, data collection and continued use of the implemented practice change are described. CONCLUSION: The loss of momentum, or "voltage drop," that happens in longer-term practice-based research network studies can be addressed using multiple strategies.

Communication is the key to success in pragmatic clinical trials in Practice-based Research Networks (PBRNs).

Effective communication is the foundation of feasibility and fidelity in practice-based pragmatic research studies. Doing a study with practices spread over several states requires long-distance communication strategies, including E-mails, faxes, telephone calls, conference calls, and texting. Compared with face-to-face communication, distance communication strategies are less familiar to most study coordinators and research teams. Developing and ensuring comfort with distance communications requires additional time and use of different talents and expertise than those required for face-to-face communication. It is necessary to make sure that messages are appropriate for the medium, clearly crafted, and presented in a manner that facilitates practices receiving and understanding the information. This discussion is based on extensive experience of 2 groups who have worked collaboratively on several large, federally funded, pragmatic trials in a practice-based research network. The goal of this article is to summarize lessons learned to facilitate the work of other research teams.

TRIPPD: a practice-based network effectiveness study of postpartum depression screening and management.

PURPOSE: Postpartum depression is common but inadequately recognized and undertreated. Continuing depressive symptoms are associated with adverse outcomes for the woman, her infant, and family. We wanted to determine the effect of a practice-based training program for screening, diagnosis, and management of depression in postpartum mothers. METHODS: In this practice-based effectiveness study, 28 practices were randomized to usual care (n = 14) or intervention (n = 14), and 2,343 women were enrolled between 5 and 12 weeks' postpartum. The intervention sites received education and tools for postpartum depression screening, diagnosis, initiation of therapy, and follow-up within their practices. Usual-care practices received a 30-minute presentation about postpartum depression. Screening information for the usual care was obtained from baseline surveys sent directly to the central site but was not available for patient care. Outcomes were based on patient-reported outcomes (level of depressive symptoms) from surveys at 6 and 12 months, plus medical record review (diagnosis and therapy initiation). RESULTS: Among the 2,343 women enrolled, 1,897 (80.1%) provided outcome information, and were included in the analysis. Overall, 654 (34.5% of 1,897) women had elevated screening scores indicative of depression, with comparable rates in the intervention and usual-care groups. Among the 654 women with elevated postpartum depression screening scores, those in the intervention practices were more likely to receive a diagnosis (P = .0006) and therapy for postpartum depression (P = .002). They also had lower depressive symptom levels at 6 (P = .07) and 12 months' (P=.001) postpartum. CONCLUSIONS: Primary care-based screening, diagnosis, and management improved mother's depression outcomes at 12 months. This practical approach could be implemented widely with modest resources.

Financial management and job social skills training components in a summer business institute: a controlled evaluation in high achieving predominantly ethnic minority youth.

Ninety-two adolescents, predominantly ethnic minority high school students, participated in a structured Summer Business Institute (SBI). Participating youth were randomly assigned to receive either job social skills or financial management skills training components. Students who additionally received the job social skills training component were more likely to recommend their employment agency to others than were youth who received the financial management component, rated their overall on-the-job work experience more favorably, and demonstrated higher scores in areas that were relevant to the skills that were taught in the job social skills workshops. The financial management component also appeared to be relatively effective, as youth who received this intervention improved their knowledge of financial management issues more than youth who received job social skills, and rated their workshops as more helpful in financial management, as well as insurance management. Future directions are discussed in light of these results.