Parent perspectives on decisions to participate in a phase I hepatocyte transplant trial

Authors

  • Alexandra Dreyzin,

    1. University of Pittsburgh School of Medicine, Pittsburgh, PA, USA
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  • Amber E. Barnato,

    Corresponding author
    1. Section of Decision Sciences, Division of General Internal Medicine, Department of Medicine, University of Pittsburgh Medical Center, Pittsburgh, PA, USA
    • Amber Barnato, Section of Decision Sciences, Division of General Internal Medicine, Department of Medicine, University of Pittsburgh Medical Center 200 Meyran Avenue, Suite 200, Pittsburgh, PA, USA

      Tel.: 412 692 4875

      Fax: 412 246 6954

      E-mail: barnatoae@upmc.edu

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  • Kyle A. Soltys,

    1. Thomas E. Starzl Transplant Institute, Children's Hospital of Pittsburgh of UPMC, Pittsburgh, PA, USA
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  • Coreen Farris,

    1. RAND Corporation, Pittsburgh, PA, USA
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  • Rachel Sada,

    1. Department of Surgery, McGowan Institute for Regenerative Medicine, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA
    2. Children's Hospital of Pittsburgh of UPMC, Pittsburgh, PA, USA
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  • Kimberly Haberman,

    1. Thomas E. Starzl Transplant Institute, Children's Hospital of Pittsburgh of UPMC, Pittsburgh, PA, USA
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  • Ira J. Fox

    1. Department of Surgery, McGowan Institute for Regenerative Medicine, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA
    2. Children's Hospital of Pittsburgh of UPMC, Pittsburgh, PA, USA
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Abstract

We examined factors that affect decision-making for families presented with a phase I clinical trial of hepatocyte transplant as a potential alternative to liver transplant for their children among two groups: (i) families who were actually offered enrollment in the hepatocyte trial and; (ii) families whose children had liver transplants before the trial was available. We conducted semi-structured interviews about actual and hypothetical decision-making regarding trial participation and used grounded theory analysis to identify common themes. The most common motivator for participation was decline in the child's health. The most common deterrent was lack of data from prior hepatocyte transplants, particularly when compared with data available about liver transplant. Interviewees’ point of comparison for evaluating relative benefits and risks of hepatocyte transplant oscillated between the alternative of doing nothing while waiting for a liver (the relevant alternative) vs. the alternative of getting a liver. These results suggest that families’ reluctance to participate may result from misconceptions about severity of the child's disease, underestimating risks of liver transplant, or confusion about the role of hepatocyte transplant in the treatment pathway. Clarification of available treatment alternatives and associated risks as part of informed consent may improve the quality of decision-making regarding trial enrollment.

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