Novel initiatives of the National Institutes of Health to support congenital heart disease research

Burns, Kristin M.; Pearson, Gail D.; Kaltman, Jonathan R.

doi:10.1097/mop.0b013e328364b8a7

Cited by 4 publications

(3 citation statements)

References 22 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…To address these concerns, the Paediatric Heart Network (PHN) was established in 2001 by the National Heart, Lung and Blood Institute, using an investigator-led model of core clinical centres and auxiliary sites across the USA and Canada [ 4 , 17 ]. Policies and procedures are developed by a Central Steering Committee, with representation from all participating centres, and implemented by a Data Coordinating Centre, with scientific and safety oversight provided by Protocol Review Committee and DMC, respectively.…”

Section: Discussionmentioning

confidence: 99%

“…The landmark Single Ventricle Reconstruction (SVR) trial randomized 555 infants with single-ventricle lesions (84% of those eligible) to the Norwood procedure, with either a modified Blalock–Taussig shunt or a right ventricle to pulmonary artery shunt across 15 sites in just over 3 years [ 9 ]. This trial demonstrated not only the power of collaboration to increase sample size in a rare condition but also that surgeons and parents were willing to randomize children to different treatments, even in high-risk complex neonatal cardiac surgery, when there is clinical equipoise [ 17 ]. To date, it has produced more than 20 publications including a publicly available data set and remains the only Phase III trial in children’s heart surgery to demonstrate a difference in 1-year transplant-free survival [ 9 ]; perhaps, more importantly, this difference was lost by 3 years [ 20 ], and further follow-up is keenly awaited.…”

Section: Discussionmentioning

confidence: 99%

See 1 more Smart Citation

Randomized controlled trials in children’s heart surgery in the 21st century: a systematic review

Drury

Patel

Oswald

et al. 2017

European Journal of Cardio-Thoracic Surgery

View full text Add to dashboard Cite

OBJECTIVESRandomized controlled trials are the gold standard for evaluating health care interventions, yet are uncommon in children’s heart surgery. We conducted a systematic review of clinical trials in paediatric cardiac surgery to evaluate the scope and quality of the current international literature.METHODSWe searched MEDLINE, CENTRAL and LILACS, and manually screened retrieved references and systematic reviews to identify all randomized controlled trials reporting the effect of any intervention on the conduct or outcomes of heart surgery in children published in any language since January 2000; secondary publications and those reporting inseparable adult data were excluded. Two reviewers independently screened studies for eligibility and extracted data; the Cochrane Risk of Bias tool was used to assess for potential biases.RESULTSWe identified 333 trials from 34 countries randomizing 23 902 children. Most were early phase (313, 94.0%), recruiting few patients (median 45, interquartile range 28–82), and only 11 (3.3%) directly evaluated a surgical intervention. One hundred and nine (32.7%) trials calculated a sample size, 52 (15.6%) reported a CONSORT diagram, 51 (15.3%) were publicly registered and 25 (7.5%) had a Data Monitoring Committee. The overall risk of bias was low in 22 (6.6%), high in 69 (20.7%) and unclear in 242 (72.7%).CONCLUSIONSThe recent literature in children’s heart surgery contains few late-phase clinical trials. Most trials did not conform to the accepted standards of reporting, and the overall risk of bias was low in few studies. There is a need for high-quality, multicentre clinical trials to provide a robust evidence base for contemporary paediatric cardiac surgical practice.

show abstract

Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

Randomized controlled trials in children’s heart surgery in the 21st century: a systematic review

Drury

Patel

Oswald

et al. 2017

European Journal of Cardio-Thoracic Surgery

View full text Add to dashboard Cite

show abstract

“…Companion diagnostics for specific cardiovascular diseases will advance our ability to realize the promise of personalized medicine [60]. To this end, the pediatric cardiology research community is in an excellent position to leverage consortia that combine genomics with patient care [61]. The Pediatric Cardiac Genomics Consortium (PCGC) is linked to the Pediatric Heart Network (PHN) and therefore is in a position to apply combined genetic information and predictive biomarkers to new clinical trials.…”

Section: Discussionmentioning

confidence: 99%

Advances in the treatment of aortic valve disease

Hinton¹

2014

Current Opinion in Pediatrics

View full text Add to dashboard Cite

Purpose of the review Aortic valve disease (AVD) is a growing public health problem, and the pathogenesis underlying AVD is complex. The lack of durable bioprostheses and pharmacologic therapies remain central needs in care. The purpose of this review is to highlight recent clinical studies that impact the care of children with AVD and to explore ongoing translational research efforts. Recent findings Clinical studies have evaluated the durability of bioprosthetics and surgical strategies, tested statins during early disease, and identified new predictive biomarkers. Large animal models have demonstrated the effectiveness of a novel bioprosthetic scaffold. Mouse models of latent AVD have advanced our ability to elucidate natural history and perform preclinical studies that test new treatments in the context of early disease. Summary Current priorities for AVD patients include identifying new pharmacologic treatments and developing durable bioprostheses. Multidisciplinary efforts are needed that bridge pediatric and adult programs, bring together different types of expertise and leverage network and consortium resources. As our understanding of the underlying complex genetics is better defined, companion diagnostics may transform future clinical trials and ultimately improve the care of patients with AVD by promoting personalized medicine and early intervention.

show abstract

Identification and Evaluation of Controlled Trials in Pediatric Cardiology: Crowdsourced Scoping Review and Creation of Accessible Searchable Database

Ashkanase

Nama

Sandarage

et al. 2020

Canadian Journal of Cardiology

View full text Add to dashboard Cite

Novel initiatives of the National Institutes of Health to support congenital heart disease research

Cited by 4 publications

References 22 publications

Randomized controlled trials in children’s heart surgery in the 21st century: a systematic review

Randomized controlled trials in children’s heart surgery in the 21st century: a systematic review

Advances in the treatment of aortic valve disease

Identification and Evaluation of Controlled Trials in Pediatric Cardiology: Crowdsourced Scoping Review and Creation of Accessible Searchable Database

Contact Info

Product

Resources

About