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The Pediatric Heart Network: A Primer for the Conduct of Multicenter Studies in Children with Congenital and Acquired Heart Disease

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Abstract

Most contemporary diagnostic and treatment strategies for pediatric patients with cardiovascular disease are not supported by evidence from clinical trials but instead are based on expert opinion, single-institution observational studies, or extrapolated from adult cardiovascular medicine. In response to this concern, the National Heart, Lung, and Blood Institute established the Pediatric Heart Disease Clinical Research Network (PHN) in 2001. The purposes of this article are to describe the initiation, structure, and function of the PHN; to review the ongoing studies; and to address current and future challenges. To date, four randomized clinical trials and two observational studies have been launched. Design and conduct of complex, multicenter studies in children with congenital and acquired heart disease must address numerous challenges, including identification of an appropriate clinically relevant primary endpoint, lack of preliminary data on which to base sample size calculations, and recruitment of an adequate number of subjects. The infrastructure is now well developed and capable of implementing complex, multicenter protocols efficiently and recruiting subjects effectively. The PHN is uniquely positioned to contribute to providing evidence-based medicine for and improving the outcomes of pediatric patients with cardiovascular disease.

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Acknowledgments

This work was supported by grants U01 HL068270 (Sleeper), U01 HL068269 (Anderson), U01 HL068292 (Minich), U01 HL068290 (Gersony), U01 HL068288 (McCrindle), U01 HL068285 (Newburger), U01 HL068281 (Saul), and U01 HL068279 (Vetter) from the National Heart, Lung, and Blood Institute, NIH/DHHS.

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Authors and Affiliations

  1. University of Texas Southwestern Medical Center, Dallas, TX, USA

    L. Mahony & G.D. Pearson (for the Pediatric Heart Network Investigators)

  2. New England Research Institutes, Watertown, MA, USA

    L.A. Sleeper

  3. Duke University Medical Center, Durham, NC, USA

    P.A.W. Anderson

  4. Columbia University Medical Center, New York, NY, USA

    W.M. Gersony

  5. The Hospital for Sick Children, Toronto, Ontario, Canada

    B.W. McCrindle

  6. Primary Children’s Medical Center, Salt Lake City, UT, USA

    L.L. Minich

  7. Children’s Hospital Boston, Boston, MA, USA

    J.W. Newburger

  8. Medical University of South Carolina, Charleston, SC, USA

    J.P. Saul

  9. Children’s Hospital of Philadelphia, Philadelphia, PA, USA

    V.L. Vetter

  10. CMC-Cardiology, 1935 Motor Street, Dallas, TX, 75235-7701, USA

    L. Mahony

Authors
  1. L. Mahony
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  2. L.A. Sleeper
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  3. P.A.W. Anderson
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  4. W.M. Gersony
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  5. B.W. McCrindle
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  6. L.L. Minich
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  7. J.W. Newburger
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  8. J.P. Saul
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  9. V.L. Vetter
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  10. G.D. Pearson
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Corresponding author

Correspondence to L. Mahony.

Appendix

Appendix

  • National Heart, Lung, and Blood Institute: Gail Pearson, Tracey Hoke, Carl Hunt, Mario Stylianou, Judith Massicot-Fisher, Marsha Mathis, Victoria Pemberton

  • Data coordinating center: New England Research Institutes—Lynn Sleeper, Steven Colan, Paul Mitchell, Gloria Klein, Dianne Gallagher, Patty Connell, Lisa Wruck

  • Network chair: Lynn Mahony, University of Texas Southwestern Medical Center

  • Clinical site investigators: Children’s Hospital Boston—Jane Newburger (PI), Roger Breitbart, Jami Levine, Renee Margossian, Peter Laussen, Pedro del Nido, John E. Mayer, Jodi Elder, Ellen McGrath, Annette L. Baker, Seda Tierney; Children’s Hospital of New York—Welton Gersony (PI), Daphne Hsu, Seema Mital, Beth Printz, Ashwin Prakash, Ralph Mosca, Karen Altmann, William Hellenbrand, Charles Kleinman, Rosalind Korsin, Darlene Servedio; Children’s Hospital of Philadelphia—Victoria Vetter (PI), Bernard J. Clark, Geoffrey Bird, Maryanne Chrisant, Meryl Cohen, Troy Dominguez, Stanford Ewing, J. William Gaynor, Beth Kaufman, Brad Marino, Stephen Paridon, Chitra Ravishankar, Jack Rychik, Sarah Tabbutt, Gil Wernovsky, Lisa Zaoutis, Meg Hawkins, Jamie Koh, La Verne Murphy; Duke University—Page A.W. Anderson (PI), Jennifer S. Li, James F. Cnota, Wesley Covitz, Kari L. Crawford, David W. Hannon, Michael H. Hines, Rob Holeman, James Jaggers, Jill Kaplan, Theodore C, Koutlas, Karen F. Lurito, Charlie J. Sang, Jr., R. Dennis Steed, Sharon N. Welsh, Mingfen Xu; Medical University of South Carolina—J. Philip Saul (PI), Andrew M. Atz, Andrew D. Blaufox, Girish Shirali, Scott M Bradley, Jon Lucas, Denise Mulvihill, Anthony Hlavacek, Eric Graham, Amy Jones, Jennifer Young; Primary Children’s Medical Center, Salt Lake City, UT—LuAnn Minich (PI), John Hawkins, Richard Williams, Lloyd Tani, Linda Lambert, Michael Puchalski, David Bradley, Robert Shaddy, Deborah Frank, Karen Osborne; Toronto Hospital for Sick Children—Brian McCrindle (PI), Andrew Redington, Elizabeth Stephenson, Christopher Caldarone, Jennifer Russell, Anne Dipchand, Elizabeth Radojewski, Nancy Slater, Timothy Bradley, Fraser Golding, Bill Williams, Annette Flynn, Joel Kirsh, Tilman Humpl

Core Laboratories

  • Angiography: Duke University—John F. Rhodes

  • Cardiac MRI: Children’s Hospital Boston—Tal Geva; Cardiac MRI infant single ventricle: Children’s Hospital of Philadelphia—Mark Fogel

  • Echocardiography: Children’s Hospital Boston—Steven Colan (director), Marcy Schwartz, Renee Margossian; Medical College of Wisconsin—Peter C. Frommelt

  • Genetics: Columbia University—Wendy Chung (director), Liyong Deng, Patricia Lonzano; Children’s Hospital of Philadelphia—Catherine A. Stolle

  • Serology: Children’s Hospital of Philadelphia—Steven Douglas (director), Donald E. Campbell, Joseph M. McMann

  • PRC: Michael Artman, chair; Judith Massicot-Fisher, executive secretary; Erle Austin, Daniel Bernstein, Timothy Feltes, Julie Johnson, Jeffrey Krischer, G. Paul Matherne, Anne Murphy, ad hoc, Anne Rowley, ad hoc

  • DSMB: John Kugler, chair; Tracey R. Hoke, executive secretary; Kathryn Davis, David J. Driscoll, Mark Galantowicz, Sally A. Hunsberger, Thomas J. Knight, Catherine L. Webb, Lawrence Wissow

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Mahony, L., Sleeper, L., Anderson, P. et al. The Pediatric Heart Network: A Primer for the Conduct of Multicenter Studies in Children with Congenital and Acquired Heart Disease. Pediatr Cardiol 27, 191–198 (2006). https://doi.org/10.1007/s00246-005-1151-9

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  • DOI: https://doi.org/10.1007/s00246-005-1151-9

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