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1.
Clin Trials ; 17(6): 684-695, 2020 12.
Article in English | MEDLINE | ID: mdl-32820647

ABSTRACT

BACKGROUND/AIMS: The Pediatric Heart Network Marfan Trial was a randomized trial comparing atenolol versus losartan on aortic root dilation in 608 children and young adults with Marfan syndrome. Barriers to enrollment included a limited pool of eligible participants, restrictive entry criteria, and a diverse age range that required pediatric and adult expertise. Retention was complicated by a 3-year commitment to a complex study and medication regimen. The Network partnered with the Marfan Foundation, bridging the community with the research. The aims of this study are to report protocol and medication adherence and associated predictive factors, and to describe recruitment and retention strategies. METHODS: Recruitment, retention, and adherence to protocol activities related to the primary outcome were measured. Retention was measured by percentage of enrolled participants with 3-year outcome data. Protocol adherence was calculated by completion rates of study visits, ambulatory electrocardiography (Holter monitoring), and quarterly calls. Medication adherence was assessed by the number of tablets or the amount of liquid in bottles returned. Centers were ranked according to adherence (high, medium, and low tertiles). Recruitment, retention, and adherence questionnaires were completed by sites. Descriptive statistics summarized recruitment, retention, and adherence, as well as questionnaire results. Regression modeling assessed predictors of adherence. RESULTS: Completion rates for visits, Holter monitors, and quarterly calls were 99%, 94%, and 96%, respectively. Primary outcome data at 3 years were obtained for 88% of participants. The mean percentage of medication taken was estimated at 89%. Site and age were associated with all measures of adherence. Young adult and African American participants had lower levels of adherence. Higher adherence sites employed more strategies; had more staffing resources, less key staff turnover, and more collaboration with referring providers; utilized the Foundation's resources; and used a greater number of strategies to recruit, retain, and promote protocol and medication adherence. CONCLUSION: Overall adherence was excellent for this trial conducted within a National Institutes of Health-funded clinical trial network. Strategies specifically targeted to young adults and African Americans may have been beneficial. Many strategies employed by higher adherence sites are ones that any site could easily use, such as greeting families at non-study hospital visits, asking for family feedback, providing calendars for tracking schedules, and recommending apps for medication reminders. Additional key learnings include adherence differences by age, race, and site, the value of collaborative learning, and the importance of partnerships with patient advocacy groups. These lessons could shape recruitment, retention, and adherence to improve the quality of future complex trials involving rare conditions.


Subject(s)
Marfan Syndrome/drug therapy , Patient Compliance/statistics & numerical data , Patient Selection , Randomized Controlled Trials as Topic/methods , Adolescent , Black or African American , Anti-Arrhythmia Agents/therapeutic use , Atenolol/therapeutic use , Child , Child, Preschool , Clinical Protocols , Female , Humans , Infant , Losartan/therapeutic use , Male , Medication Adherence/statistics & numerical data , Surveys and Questionnaires , Young Adult
2.
J Pediatr ; 204: 250-255.e1, 2019 01.
Article in English | MEDLINE | ID: mdl-30270167

ABSTRACT

OBJECTIVE: To assess health-related quality of life (HRQOL) in a large multicenter cohort of children and young adults with Marfan syndrome participating in the Pediatric Heart Network Marfan Trial. STUDY DESIGN: The Pediatric Quality of Life Inventory (PedsQL) 4.0 Generic Core Scales were administered to 321 subjects with Marfan syndrome (5-25 years). PedsQL scores were compared with healthy population norms. The impact of treatment arm (atenolol vs losartan), severity of clinical features, and number of patient-reported symptoms on HRQOL was assessed by general linear models. RESULTS: Mean PedsQL scores in children (5-18 years) with Marfan syndrome were lower than healthy population norms for physical (P ≤ .003) and psychosocial (P < .001) domains; mean psychosocial scores for adults (19-25 years) were greater than healthy norms (P < .001). HRQOL across multiple domains correlated inversely with frequency of patient-reported symptoms (r = 0.30-0.38, P < .0001). Those <18 years of age with neurodevelopmental disorders (mainly learning disability, attention-deficit/hyperactivity disorder) had lower mean PedsQL scores (5.5-7.4 lower, P < .04). A multivariable model found age, sex, patient-reported symptoms, and neurodevelopmental disorder to be independent predictors of HRQOL. There were no differences in HRQOL scores by treatment arm, aortic root z score, number of skeletal features, or presence of ectopia lentis. CONCLUSIONS: Children and adolescents with Marfan syndrome were at high risk for impaired HRQOL. Patient-reported symptoms and neurodevelopmental disorder, but not treatment arm or severity of Marfan syndrome-related physical findings, were associated with lower HRQOL.


Subject(s)
Antihypertensive Agents/therapeutic use , Atenolol/therapeutic use , Losartan/therapeutic use , Marfan Syndrome/psychology , Quality of Life , Adolescent , Adult , Child , Child, Preschool , Female , Health Status Indicators , Humans , Male , Marfan Syndrome/complications , Marfan Syndrome/drug therapy , Patient Reported Outcome Measures , Severity of Illness Index , Young Adult
3.
Cardiol Young ; 23(2): 248-57, 2013 Apr.
Article in English | MEDLINE | ID: mdl-22931751

ABSTRACT

OBJECTIVES: Identify trends of enrolment and key challenges when recruiting infants with complex cardiac diseases into a multi-centre, randomised, placebo-controlled drug trial and assess the impact of efforts to share successful strategies on enrolment of subjects. METHODS: Rates of screening, eligibility, consent, and randomisation were determined for three consecutive periods of time. Sites collectively addressed barriers to recruitment and shared successful strategies resulting in the Inventory of Best Recruiting Practices. Study teams detailed institutional practices of recruitment in post-trial surveys that were compared with strategies of enrolment initially proposed in the Inventory. RESULTS: The number of screened patients increased by 30% between the Initial Period and the Intermediate Period (p = 0.007), whereas eligibility decreased slightly by 7%. Of those eligible for entry into the study, the rate of consent increased by 42% (p = 0.025) and randomisation increased by 71% (p = 0.10). During the Final Period, after launch of a competing trial, fewer patients were screened (−14%, p = 0.06), consented (−19%, p = 0.12), and randomised (−34%, p = 0.012). Practices of recruitment in the post-trial survey closely mirrored those in the Inventory. CONCLUSIONS: Early identification and sharing of best strategies of recruitment among all recruiting sites can be effective in increasing recruitment of critically ill infants with congenital cardiac disease and possibly other populations. Strategies of recruitment should focus on those that build relationships with families and create partnerships with the medical providers who care for them. Competing studies pose challenges for enrolment in trials, but fostering trusting relationships with families can result in successful enrolment into multiple studies.


Subject(s)
Angiotensin-Converting Enzyme Inhibitors/therapeutic use , Heart Defects, Congenital/drug therapy , Multicenter Studies as Topic/methods , Patient Selection , Randomized Controlled Trials as Topic/methods , Canada , Humans , Infant , Infant, Newborn , United States
4.
J Thorac Cardiovasc Surg ; 142(3): 531-7, 2011 Sep.
Article in English | MEDLINE | ID: mdl-21397260

ABSTRACT

OBJECTIVE: The purpose of this analysis was to evaluate a novel strategy for reporting adverse events in the Pediatric Heart Network's randomized surgical trial of systemic-pulmonary artery shunt versus right ventricle-pulmonary artery conduit in infants with hypoplastic left heart syndrome. The strategy was developed to align the reporting process with the needs of a surgical trial while maintaining participant safety. METHODS: Adverse event reporting was analyzed for 2 groups of study subjects: those randomized to a trial arm during a period in which a standard adverse event reporting system was used (period 1) and those randomized after institution of a system that focused serious adverse event reporting on 6 sentinel events (period 2). The analysis encompassed the period from randomization (Norwood surgery) to hospital discharge from stage II surgery. Adverse event rates were compared using a Poisson regression model for the number of events per subject. RESULTS: From period 1 to period 2, the rate of serious adverse events requiring expedited reporting decreased as expected (0.42 vs 0.14/subject/month of follow-up; P < .001). Subjects with a serious (sentinel) adverse event in period 2 had a significantly higher rate of death and cardiac transplantation. CONCLUSIONS: The new adverse event reporting system successfully targeted subjects at highest risk, while decreasing the administrative burden associated with adverse event reports. This methodology may be of benefit in trials evaluating surgical or device-based interventions and in critically ill populations where many common clinical events would qualify as serious adverse events in the context of a drug trial.


Subject(s)
Cardiac Surgical Procedures/adverse effects , Documentation/methods , Hypoplastic Left Heart Syndrome/surgery , Documentation/standards , Humans , Postoperative Complications/epidemiology , Regression Analysis
5.
Pediatr Cardiol ; 32(4): 479-86, 2011 Apr.
Article in English | MEDLINE | ID: mdl-21331516

ABSTRACT

A potential advantage of the right ventricle to pulmonary artery versus modified Blalock-Taussig shunt in patients undergoing the Norwood procedure is limitation of diastolic runoff from the systemic to pulmonary circulation. We evaluated mesenteric flow patterns and gastrointestinal outcomes following the Norwood procedure associated with either shunt type. Patients randomized to a right ventricle to pulmonary artery versus modified Blalock-Taussig shunt in the Pediatric Heart Network Single Ventricle Reconstruction Trial at centers participating in this ancillary study were eligible for inclusion; those with active necrotizing enterocolitis, sepsis, or end-organ dysfunction were excluded. Celiac artery flow characteristics and gastrointestinal outcomes were collected at discharge. Forty-four patients (five centers) were included. Median age at surgery was 5 days [interquartile range (IQR) = 4-8 days]. Median celiac artery resistive index (an indicator of resistance to perfusion) was higher in the modified Blalock-Taussig shunt group (n = 19) versus the right ventricle to pulmonary artery shunt group (n = 25) [1.00 (IQR = 0.84-1.14) vs. 0.82 (IQR = 0.74-1.00), p = 0.02]. There was no difference in interstage weight gain, necrotizing enterocolitis, or feeding intolerance episodes between the groups. The celiac artery resistive index was higher in patients with the modified Blalock-Taussig shunt versus the right ventricle to pulmonary artery shunt but was not associated with measured gastrointestinal outcomes.


Subject(s)
Blalock-Taussig Procedure/methods , Celiac Artery/physiopathology , Heart Ventricles/surgery , Hypoplastic Left Heart Syndrome/surgery , Norwood Procedures/methods , Pulmonary Artery/surgery , Splanchnic Circulation/physiology , Celiac Artery/diagnostic imaging , Celiac Artery/surgery , Echocardiography, Doppler, Color , Female , Follow-Up Studies , Heart Ventricles/diagnostic imaging , Humans , Hypoplastic Left Heart Syndrome/diagnostic imaging , Hypoplastic Left Heart Syndrome/physiopathology , Infant, Newborn , Male , Prospective Studies , Pulmonary Artery/diagnostic imaging , Pulmonary Artery/physiopathology , Treatment Outcome
6.
Pediatrics ; 124(5): e942-9, 2009 Nov.
Article in English | MEDLINE | ID: mdl-19841109

ABSTRACT

OBJECTIVE: We sought to compare perceptions of functional health status between children who had undergone a Fontan procedure and their parents. METHODS: Fontan procedure survivors 10 to 18 years of age were included in the study if the child completed the Child Health Questionnaire (CHQ) and the parent completed the parent form to assess the child's functional health status. Comparisons were made between raw domain scores for the parent- and child-completed CHQs. RESULTS: Between March 2003 and April 2004, 1078 Fontan survivors were screened. Of the 546 eligible and consented patients, 354 were 10-18 years of age and 328 parent/child pairs completed the CHQs. Parents reported significantly lower scores (worse functioning) for their children than the children reported for themselves in the domains of physical functioning (P < .01), impact on school or activities from emotional and behavioral problems (P < .01), impact on school or activities from physical health issues (P < .01), general behavior (P < .01), mental health (P < .01), self-esteem (P < .01), and general health perceptions (P < .01). No significant differences were noted for the domains of bodily pain, family cohesiveness, or family activities. For the physical functioning domain, factors contributing to lower scores for parent versus child reports included pulmonary artery anomalies and fenestration at the time of the Fontan operation. Lower parent-reported scores also were associated with more noncardiac health problems in the child. CONCLUSIONS: Parents' perceptions of the functional health status of their children after the Fontan procedure were worse than the children's perceptions.


Subject(s)
Activities of Daily Living , Attitude to Health , Fontan Procedure , Health Status , Parents/psychology , Adolescent , Child , Female , Fontan Procedure/psychology , Heart Defects, Congenital/surgery , Humans , Male , Quality of Life , Surveys and Questionnaires
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