Patient Participation In Studies Research Articles

Understanding patient participation behaviour in studies of COPD support programmes such as pulmonary rehabilitation and self-management: a qualitative synthesis with application of theory.

Background:In chronic obstructive pulmonary disease (COPD), the problem of poor patient participation in studies of self-management (SM) and pulmonary rehabilitation (PR) programmes (together referred to as COPD support programmes) is established. Understanding this problem beyond the previously reported socio-demographics and clinical factors is critical.Aims:The aim of this study was to explore factors that explain patient participation in studies of COPD support programmes.Methods:Thematic ‘framework’ synthesis was conducted on literature published from 1984 to 1 February 2015. Emergent themes and subthemes were mapped onto the adapted ‘attitude–social influence–external barriers’ and the ‘self-regulation’ models to produce analytical themes.Results:Ten out of 12 studies were included: PR (n=9) and SM (n=1). Three descriptive themes with 38 subthemes were mapped onto the models' constructs, and it generated four analytical themes: ‘attitude’, ‘social influences’ and ‘illness’ and ‘intervention representations’. The following factors influenced (1) attendance—helping oneself through health improvements, perceived control of worsening condition, perceived benefits and positive past experience of the programme, as well as perceived positive influence of professionals; (2) non-attendance—perceived negative effects and negative past experience of the programme, perceived physical/practical concerns related to attendance, perceived severity of condition/symptoms and perceived negative influence of professionals/friends; (3) dropout—no health improvements perceived after attending a few sessions of the programme, perceived severity of the condition and perceived physical/practical concerns related to attendance.Conclusions:Psychosocial factors including perceived practical/physical concerns related to attendance influenced patients’ participation in COPD support programmes. Addressing the negative beliefs/perceptions via behaviour change interventions may help improve participation in COPD support programmes and, ultimately, patient outcomes.

Successful Patient Recruitment in CT Imaging Clinical Trials: What Factors Influence Patient Participation?

Analyze factors that influence participation in research studies that use coronary computed tomography (CT) imaging. A 12-point survey using a questionnaire was conducted on 80 subjects, of whom 40 agreed to participate in a cardiovascular CT imaging research study (enrolling subjects) and 40 declined participation (non-enrolling subjects). Potential factors that motivated the acceptance or refusal of enrollment were evaluated using a 5-point Likert scale. The following aspects were addressed: (1) additional health information, (2) free imaging, (3) altruistic benefit to society, (4) monetary compensation, (5) radiation exposure, (6) role asan experimental subject, (7) possible loss of confidentiality, (8) contrast or investigational drug use, (9) premedication use, (10) blood draw or intravenous placement, (11) time commitment, and (12) personal medical opinion. Response distributions were obtained for each question and compared between enrolling and non-enrolling groups. Enrolling subjects gave significantly higher ratings than non-enrolling subjects for the following factors: additional health information (P < .001), free imaging (P < .001), and the altruistic benefit to society (P < .001). For non-enrolling subjects, concern for possible drug use or contrast injection (P < .001), concern for possible premedication (P < .001), and personal availability or time commitment (P<.001) were all given significantly higher ratings. Concern for radiation exposure (P = .002) and personal medical opinion (P<.001) received significantly high ratings among both groups but did not differ between groups. Several influential concerns and benefits were identified from potential research subjects. Knowledge of what influences patient participation in studies involving CT imaging may allow researchers to effectively address concerns and highlight the potential benefits related to participation.

Reporting participation rates in studies of non-pharmacological interventions for patients with chronic obstructive pulmonary disease: a systematic review

BackgroundPulmonary rehabilitation (PR) and self-management (SM) support programmes are effective in the management of patients with chronic obstructive pulmonary disease (COPD), but these interventions are not widely implemented in routine care. One reason may be poor patient participation and retention. We conducted a systematic review to determine a true estimate of participation and dropout rates in research studies of these interventions.MethodsStudies were identified from eight electronic databases including MEDLINE, UK Clinical Trial Register, Cochrane library, and reference lists of identified studies. Controlled clinical trial studies of structured SM, PR and health education (HE) programmes for COPD were included. Data extraction included ‘participant flow’ data using the Consolidated Standards of Reporting Trials (CONSORT) statement and its extension to pragmatic trials. Patient ‘participation rates’ (study participation rate (SPR), study dropout rate (SDR) and intervention dropout rate (IDR)) were calculated using prior participation definitions consistent with CONSORT. Random effects logistic regression analysis was conducted to examine effects of four key study characteristics (group vs. individual treatment, year of publication, study quality and exercise vs. non-exercise) on participation rates.ResultsFifty-six quantitative studies (51 randomised controlled trials, three quasi-experimental and two before-after studies) evaluated PR (n = 31), SM (n = 21) and HE (n = 4). Reports of participant flow were generally incomplete; ‘numbers of potential participants identified’ were only available for 16%, and ‘numbers assessed for eligibility’ for only 39% of studies. Although ‘numbers eligible’ were better reported (77%), we were unable to calculate SPR for 23% of studies. Overall we found ‘participation rates’ for studies (n = 43) were higher than previous reports; only 19% of studies had less than 50% SPR and just over one-third (34%) had a SPR of 100%; SDR and IDR were less than or equal to 30% for around 93% of studies. There was no evidence of effects of study characteristics on participation rates.ConclusionUnlike previous reports, we found high participation and low dropout rates in studies of PR or SM support for COPD. Previous studies adopted different participation definitions; some reported proportions without stating definitions clearly, obscuring whether proportions referred to the study or the intervention. Clear, uniform definitions of patient participation in studies are needed to better inform the wider implementation of effective interventions.

S112 Estimating Participation Rates of COPD Patients in Pulmonary Rehabilitation and Self-Management Programmes: The Importance of Defining Participation

Introduction and ObjectivesDelivery of pulmonary rehabilitation (PR) and self-management (SM) support programmes to patients living with chronic obstructive pulmonary disease (COPD) is emphasised in national guidelines, but these interventions are...

Safety of Recombinant Activated Factor VII (rFVIIa) in Patients with Congenital Hemophilia with Inhibitors: Overall Dose Exposure and Intervals Following >240 Mcg/Kg Doses Across Trial, Registry and Diary Studies,

Abstract 3316▪▪This icon denotes a clinically relevant abstract Objective:Recombinant FVIIa (rFVIIa) is indicated for the treatment of bleeding episodes in hemophilia patients with inhibitors at a dose of 90 mcg/kg every 2–3 hours, or as a single 270 mcg/kg rFVIIa dose (outside US). As patient response and bleeds are variable, treatment is often individualized by the center and by the patient/caregiver administering rFVIIa. With home treatment, it is difficult to ascertain how often patients use higher doses (>90 mcg/kg), and whether these doses are associated with increased risk of thromboembolic events (TEs). The aim of this analysis was to understand the safety of higher rFVIIa doses and the intervals to subsequent doses. Methods:Data for rFVIIa on-demand (OD) bleed treatment in inhibitor patients were obtained from 6 sources: 2 prospective randomized studies; 1 prospective observational US diary study (DOSE); the US-based Hemostasis and Thrombosis Research Society (HTRS) Registry; the international ONE Registry; and the HemoRec registry (Czech data only). Data on prophylactic (PPX) rFVIIa use was obtained from 3 sources: 1 prospective randomized trial; 1 retrospective chart review study (PRO-PACT); and the HemoRec registry (Czech data). Data on intervals between high initial/subsequent doses and any further bypassing agent dose in OD treatment were analyzed from 3 sources that reported time of administration (DOSE, ONE and HemoRec). All studies had data safety monitoring in the form of explicit surveillance, positive affirmations of lack of adverse events, or chart reviews. Overlap in patient participation in studies could not be ruled out; however, all data are believed to be non-overlapping due to study timelines. Demographics, rFVIIa dosing, and frequency of TEs were analyzed and reported using descriptive statistics. Results:A total of 481 inhibitor patients reported using 61,734 rFVIIa doses either for OD treatment or PPX. Most patients had hemophilia A (87%) and were Caucasian (79%). Age ranged from <1 to 64 years (40% >18y). All data sources included the use of rFVIIa doses higher than current US-recommendations. Overall, 52.2% of doses (45.9% in adults) were >120 mcg/kg, the highest dose referenced from previous clinical trials in the US package insert.All PatientsAdults (Age >18)# Patients481192# Bleeds3,9471,706# Prophylaxis Days43,1357,282# rFVIIa Doses (#, %)>240 mcg/kg9,27415.0%1,37112.4%>160 to 240 mcg/kg13,72322.2%2,05918.7%>120 to 160 mcg/kg9,24715.0%1,62514.7%80–120 mcg/kg25,53741.4%5,38048.8%<80 mcg/kg3,9536.4%5865.3%Total61,734100.0%11,021100.0%For the152 patients from DOSE, ONE and HemoRec included in the dosing interval analyses, age ranged from <1 to 64 y (45% >18y). Patients received 3,042 rFVIIa doses for 1,017 bleeds. These included 361 initial rFVIIa doses (36%) >240 mcg/kg, including 136 followed by a second dose of bypassing agent (130 with rFVIIa; 15 with pd-aPCC). There were 670 doses >240 mcg/kg used throughout treatments (22%), including 327 followed by a subsequent dose (311 with rFVIIa; 23 with pd-aPCC). Most subsequent doses (79%) occurred after >8 hrs.After Initial Dose >240 mcg/kgAfter Any Dose >240 mcg/kgn%n%Doses >240 mcg/kg136327Interval to next dose<3 hrs118.1%298.9%3 up to 4 hrs1410.3%185.5%4 up to 6 hrs32.2%61.8%6 up to 8 hrs64.4%175.2%8 up to 12 hrs1410.3%247.3%12 up to 18 hrs2518.4%4513.8%18 up to 24 hrs96.6%144.3%24 hrs or greater5439.7%17453.2%No TEs were reported across the 8 sources. Conclusions:A review of 61,734 cumulative rFVIIa doses in studies with explicit safety monitoring demonstrates the use and safety of rFVIIa doses >90 mcg/kg in inhibitor patients. When doses >240 mcg/kg were followed by other bypassing agent doses, the dosing interval was >8 hrs after 79% of doses and >24 hrs after 53% of doses. Unlike MedWatch and passive surveillance where underreporting is common, we expect that the explicit surveillance in these studies would adequately identify TE events. Thus, the lack of TEs supports the overall low TE occurrence (0.28%) reported in the original registration trials in inhibitor patients using ≤90 mcg/kg. These data reinforce that the arterial thrombosis risk identified in meta-analyses outside of licensed indications (critical bleeds) are unique to those populations and do not apply to inhibitor patients. Furthermore, higher doses may provide an opportunity to improve bleed treatment with less frequent injections and decreased follow-up infusions. Disclosures:Shapiro:Novo Nordisk inc.: Consultancy, Research Funding, Speakers Bureau. Off Label Use: rFVIIa use in indicated population of congential hemophilia with inhibitors at doses that differ from those in the prescribing information. Neufeld:Novo Nordisk Inc.: Consultancy, Research Funding. Blanchette:Novo Nordisk: Consultancy. Chambost:Novo Nordisk: Consultancy. Salaj:Novo Nordisk: Consultancy. Gut:Novo Nordisk Inc.: Employment. Cooper:Novo Nordisk Inc.: Employment.