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Comparison of Characteristics and Outcomes of Trial Participants and Nonparticipants: Example of Blood and Marrow Transplant Clinical Trials Network 0201 Trial.

ABSTRACT: Controversy surrounds the question of whether clinical trial participants have better outcomes than comparable patients who are not treated on a trial. We explored this question using a recent large, randomized, multicenter study comparing peripheral blood (PB) with bone marrow transplantation from unrelated donors, conducted by the Blood and Marrow Transplant Clinical Trials Network (BMT CTN). We compared characteristics and outcomes of study participants (n = 494) and nonparticipants (n = 1384) who appeared eligible and received similar treatment without enrolling on the BMT CTN trial at participating centers during the study time period. Data were obtained from the Center for International Blood and Marrow Transplant Research. Outcomes were compared between the 2 groups using Cox proportional hazards regression models. No significant differences in age, sex, disease distribution, race/ethnicity, HLA matching, comorbidities, and interval from diagnosis to hematopoietic cell transplantation were seen between the participants and nonparticipants. Nonparticipants were more likely to have lower performance status, lower risk disease, and older donors, and to receive myeloablative conditioning and antithymocyte globulin. Nonparticipants were also more likely to receive PB grafts, the intervention tested in the trial (66% versus 50%, P < .001). Overall survival, transplantation-related mortality, and incidences of acute or chronic graft-versus-host disease were comparable between the 2 groups though relapse was higher (hazard ratio, 1.22; 95% confidence interval, 1.02 to 1.46; P = .028) in nonparticipants. Despite differences in certain baseline characteristics, survival was comparable between study participants and nonparticipants. The results of the BMT CTN trial appear generalizable to the population of trial-eligible patients.

SUBMITTER: Khera N

PROVIDER: S-EPMC4568172 | biostudies-literature | 2015 Oct

REPOSITORIES: biostudies-literature

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Comparison of Characteristics and Outcomes of Trial Participants and Nonparticipants: Example of Blood and Marrow Transplant Clinical Trials Network 0201 Trial.

Khera Nandita N Majhail Navneet S NS Brazauskas Ruta R Wang Zhiwei Z He Naya N Aljurf Mahmoud D MD Akpek Görgün G Atsuta Yoshiko Y Beattie Sara S Bredeson Christopher N CN Burns Linda J LJ Dalal Jignesh D JD Freytes César O CO Gupta Vikas V Inamoto Yoshihiro Y Lazarus Hillard M HM LeMaistre Charles F CF Steinberg Amir A Szwajcer David D Wingard John R JR Wirk Baldeep B Wood William A WA Joffe Steven S Hahn Theresa E TE Loberiza Fausto R FR Anasetti Claudio C Horowitz Mary M MM Lee Stephanie J SJ

Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation 20150611 10

Controversy surrounds the question of whether clinical trial participants have better outcomes than comparable patients who are not treated on a trial. We explored this question using a recent large, randomized, multicenter study comparing peripheral blood (PB) with bone marrow transplantation from unrelated donors, conducted by the Blood and Marrow Transplant Clinical Trials Network (BMT CTN). We compared characteristics and outcomes of study participants (n = 494) and nonparticipants (n = 1384 ...[more]

PMID: 26071866

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Project description:ImportanceAssessing generalizability of clinical trials is important to ensure appropriate application of interventions, but most assessments provide minimal granularity on comparisons of clinical characteristics.ObjectiveTo assess the extent of underlying clinical differences between clinical trial participants and nonparticipants by using a combination of electronic health record and trial enrollment data.Design, setting, and participantsThis cross-sectional study used data obtained from a single academic medical center between September 1996 and January 2019 to identify 1645 clinical trial participants from a diverse set of 202 available trials conducted at the center. Using an aggregated resampling procedure, nonparticipants were matched to participants 1:1 based on trial conditions, number of recent visits to a health care professional, and calendar time.ExposuresClinical trial enrollment vs no enrollment.Main outcomes and measuresThe primary outcome was standardized differences in clinical characteristics between participants and nonparticipants in clinical trials stratified into the 4 most common disease domains.ResultsThis cross-sectional study included 1645 participants from 202 trials (929 [56.5%] male; mean [SD] age, 54.65 [21.38] years) and an aggregated set of 1645 nonparticipants (855 [52.0%] male; mean [SD] age, 57.24 [21.91] years). The most common disease domains for the selected trials were neoplastic disease (86 trials; 737 participants), disorders of the digestive system (31 trials; 321 participants), inflammatory disorders (28 trials; 276 participants), and disorders of the cardiovascular system (27 trials; 319 participants); trials could qualify for multiple disease domains. Among 31 conditions, the percentage of conditions for which the prevalence was lower among participants than among nonparticipants per standardized differences was 64.5% (20 conditions) for neoplastic disease trials, 61.3% (19) for digestive system trials, 58.1% (18) for inflammatory disorder trials, and 38.7% (12) for cardiovascular system trials. Among 17 medications, the percentage of medications for which use was less among participants than among nonparticipants per standardized differences was 64.7% (11) for neoplastic disease trials, 58.8% (10) for digestive system trials, 88.2% (15) for inflammatory disorder trials, and 52.9% (9) for cardiovascular system trials.Conclusions and relevanceUsing a combination of electronic health record and trial enrollment data, this study found that clinical trial participants had fewer comorbidities and less use of medication than nonparticipants across a variety of disease domains. Combining trial enrollment data with electronic health record data may be useful for better understanding of the generalizability of trial results.

Project description:INTRODUCTION:The Supplemental Nutrition Assistance Program provides financial assistance for food and beverage purchases to approximately 1 in 7 Americans, with benefits distributed once monthly. Most Supplemental Nutrition Assistance Program benefits are spent early in the month, leading to decreased caloric intake later in the month. The effects of this early benefit depletion on the types of foods and beverages purchased over the course of the month is unclear. METHODS:Using individually tracked sales data from 950 participants enrolled in 2 supermarket-based RCTs in Maine (October 2015-April 2016 and October 2016-June 2017), purchases of selected food categories by Supplemental Nutrition Assistance Program participants (n=248) versus nonparticipants (n=702) in the first 2 weeks compared with the last 2 weeks of the Supplemental Nutrition Assistance Program benefit month were examined. Analyses were completed in 2019. RESULTS:For Supplemental Nutrition Assistance Program participants, adjusted mean food spending decreased 37% from the first 2 weeks to the last 2 weeks of the Supplemental Nutrition Assistance Program benefit month (p<0.0001) compared with a 3% decrease (p=0.02) for nonparticipants. The decline in spending by Supplemental Nutrition Assistance Program participants occurred in all examined categories: vegetables (-25%), fruits (-27%), sugar-sweetened beverages (-30%), red meat (-37%), convenience foods (-40%), and poultry (-48%). Difference-in-difference estimators comparing Supplemental Nutrition Assistance Program participants with nonparticipants were statistically significant (p<0.05) for all examined categories. CONCLUSIONS:In the second half of the Supplemental Nutrition Assistance Program benefit month, individuals reduced purchases of all examined categories. More research is needed to understand the impact of these fluctuations in spending patterns on the dietary quality of Supplemental Nutrition Assistance Program participants.

Project description:We report a comparison of time to recovery, side effects, and change in blood counts from baseline to after donation from unrelated donors who participated in the Blood and Marrow Transplant Clinical Trials Network phase III randomized, multicenter trial (0201) in which donor-recipient pairs were randomized to either peripheral blood stem cell (PBSC) or bone marrow (BM) donation. Of the entire cohort, 262 donated PBSC and 264 donated BM; 372 (71%) donors were from domestic and 154 (29%) were from international centers (145 German and 9 Canadian). PBSC donors recovered in less time, with a median time to recovery of 1 week compared with 2.3 weeks for BM donors. The number of donors reporting full recovery was significantly greater for donors of PBSC than of BM at 1, 2, and 3 weeks and 3 months after donation. Multivariate analysis showed that PBSC donors were more likely to recover at any time after donation compared with BM donors (hazard ratio, 2.08; 95% confidence interval [CI], 1.73 to 2.50; P < .001). Other characteristics that significantly increased the likelihood of complete recovery were being an international donor and donation in more recent years. Donors of BM were more likely to report grades 2 to 4 skeletal pain, body symptoms, and fatigue at 1 week after donation. In logistic regression analysis of domestic donors only in which toxicities at peri-collection time points (day 5 filgrastim for PBSC donors and day 2 after collection of BM donors) could be analyzed, no variable was significantly associated with grades 2 to 4 skeletal pain, including product donated (BM versus PBSC; odds ratio, 1.13; 95% CI, .74 to 1.74; P = .556). Blood counts were affected by product donated, with greater mean change from baseline to after donation for white blood cells, neutrophils, mononuclear cells, and platelets in PBSC donors whereas BM donors experienced a greater mean change in hemoglobin. This analysis provided an enhanced understanding of donor events as product donated was independent of physician bias or donor preference.

Project description:ImportanceRacial/ethnic minority groups, women, and elderly people experience a disproportionate burden of disease in rheumatoid arthritis (RA), making it particularly important to examine drug therapies in these populations. Despite a national health agenda to improve representation of diverse populations in randomized clinical trials (RCTs), there have been few large-scale analyses examining RCT demographic characteristics within rheumatology and none focusing on RA.ObjectiveTo characterize the representation of racial/ethnic minority groups, women, and elderly people through a comprehensive systematic review of RA RCTs.Data sourcesA literature search of PubMed's MEDLINE database was conducted to identify RA RCTs in adults 19 years and older published in English between January 1, 2008, and January 1, 2018.Study selectionRandomized double-blind RCTs examining any systemic, disease-modifying therapy were included. Secondary analyses of previously published RCTs were excluded. Of 1195 identified records, 240 articles (20.1%) met final selection criteria. The analysis focused on RCTs with at least 1 US-based site.Data extraction and synthesisData were extracted and synthesized according to the PRISMA guidelines for systematic reviews. Studies were screened for eligibility criteria. Demographic data on the age, sex, and race/ethnicity of RCT participants were extracted. Data analysis was conducted from October 25, 2018, to March 15, 2019.Main outcomes and measuresRepresentation of race/ethnicity and sex, defined as the proportion of total participants that belonged to each racial/ethnic group or sex. Trends in proportions over time were examined and compared with US demographic data.ResultsA total of 240 RCTs with 77 071 participants were included. Of 126 RCTs with at least 1 US-based site (52.5%), the enrollment of minority racial/ethnic groups was significantly lower than their representation within the US Census population (16% vs 40%; P < .001), and the enrollment of men was significantly lower than the incidence of RA in men nationally (20.4% vs 28.6%; P < .001). There was no trend toward improved representation of racial/ethnic minority groups or men over time.Conclusions and relevanceGiven the disproportionate burden of RA among racial/ethnic minority groups, it is imperative that policy makers better incentivize the inclusion of racial/ethnic minority groups in RA RCTs.

Project description:BackgroundThe generalizability of randomized controlled trials (RCTs) with a low response can be limited by systematic differences between participants and nonparticipants. This participation bias, however, is rarely investigated because data on nonparticipants is usually not available. The purpose of this article is to compare all participants and nonparticipants of a RCT to improve oral health among home care recipients at baseline and during follow-up using claims data.MethodsSeven German statutory health and long-term care insurance funds invited 9656 home care recipients to participate in the RCT MundPflege. Claims data for all participants (n = 527, 5.5% response) and nonparticipants (n = 9129) were analyzed. Associations between trial participation and sex, age, care dependency, number of Elixhauser diseases, and dementia, as well as nursing, medical, and dental care utilization at baseline, were investigated using multivariable logistic regression. Associations between trial participation and the probability of (a) moving into a nursing home, (b) being hospitalized, and (c) death during 1 year of follow-up were examined via Cox proportional hazards regressions, controlling for baseline variables.ResultsAt baseline, trial participation was positively associated with male sex (odds ratio 1.29 [95% confidence interval 1.08-1.54]), high (vs. low 1.46 [1.15-1.86]) care dependency, receiving occasional in-kind benefits to relieve caring relatives (1.45 [1.15-1.84]), having a referral by a general practitioner to a medical specialist (1.62 [1.21-2.18]), and dental care utilization (2.02 [1.67-2.45]). It was negatively associated with being 75-84 (vs. < 60 0.67 [0.50-0.90]) and 85 + (0.50 [0.37-0.69]) years old. For morbidity, hospitalizations, and formal, respite, short-term, and day or night care, no associations were found. During follow-up, participants were less likely to move into a nursing home than nonparticipants (hazard ratio 0.50 [0.32-0.79]). For hospitalizations and mortality, no associations were found.ConclusionsFor half of the comparisons, differences between participants and nonparticipants were observed. The RCT's generalizability is limited, but to a smaller extent than one would expect because of the low response. Routine data provide a valuable source for investigating potential differences between trial participants and nonparticipants, which might be used by future RCTs to evaluate the generalizability of their findings.Trial registrationGerman Clinical Trials Register DRKS00013517 . Retrospectively registered on June 11, 2018.

Project description:BackgroundWeb-based behavioral programs efficiently disseminate health information to a broad population, and online tailoring may increase their effectiveness. While the number of Internet-based behavioral interventions has grown in the last several years, additional information is needed to understand the characteristics of subjects who enroll in these interventions, relative to those subjects who are invited to enroll.ObjectiveThe aim of the study was to compare the characteristics of participants who enrolled in an online dietary intervention trial (MENU) with those who were invited but chose not to participate, in order to better understand how these groups differ.MethodsThe MENU trial was conducted among five health plans participating in the HMO Cancer Research Network in collaboration with the University of Michigan Center for Health Communication Research. Approximately 6000 health plan members per site, between the ages of 21 and 65, and stratified by gender with oversampling of minority populations, were randomly selected for recruitment and were mailed an invitation letter containing website information and a US$2 bill with the promise of US$20 for completing follow-up surveys. Administrative and area-based data using geocoding along with baseline survey data were used to compare invitees (HMO members sent the introductory letter), responders (those who entered a study ID on the website), and enrollees (those who completed the enrollment process). Generalized estimating equation multivariate and logistic regression models were used to assess predictors of response and enrollment.ResultsOf 28,460 members invited to participate, 4270 (15.0%) accessed the website. Of the eligible responders, 2540 (8.9%) completed the consent form and baseline survey and were enrolled and randomized. The odds of responding were 10% lower for every decade of increased age (P < .001), while the likelihood of enrolling was 10% higher for every decade increase in age (P < .001). Women were more likely to respond and to enroll (P < .001). Those living in a census tract associated with higher education levels were more likely to respond and enroll, as well as those residing in tracts with higher income (P < .001). With a 22% (n = 566) enrollment rate for African Americans and 8% (n = 192) for Hispanics, the enrolled sample was more racially and ethnically diverse than the background sampling frame.ConclusionsRelative to members invited to participate in the Internet-based intervention, those who enrolled were more likely to be older and live in census tracts associated with higher socioeconomic status. While oversampling of minority health plan members generated an enrolled sample that was more racially and ethnically diverse than the overall health plan population, additional research is needed to better understand methods that will expand the penetration of Internet interventions into more socioeconomically diverse populations.Trial registrationClinicaltrials.gov NCT00169312; http://clinicaltrials.gov/ct2/show/NCT00169312 (Archived by WebCite at http://www.webcitation.org/5jB50xSfU).

Dataset Information

Comparison of Characteristics and Outcomes of Trial Participants and Nonparticipants: Example of Blood and Marrow Transplant Clinical Trials Network 0201 Trial.

Publications

Comparison of Characteristics and Outcomes of Trial Participants and Nonparticipants: Example of Blood and Marrow Transplant Clinical Trials Network 0201 Trial.

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