Diagnosis and treatment of pediatric acquired aplastic anemia (AAA): An initial survey of the North American Pediatric Aplastic Anemia Consortium (NAPAAC)

Authors

  • David A. Williams MD,

    Corresponding author
    1. Division of Hematology/Oncology, Boston Children's Hospital and Dana-Farber Cancer Institute, Harvard Medical School, Boston, Massachusetts
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    • On behalf of the North American Pediatric Aplastic Anemia Consortium.
  • Carolyn Bennett MD,

    1. Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia
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  • Alison Bertuch MD, PhD,

    1. Texas Children's Hospital and Baylor College of Medicine, Houston, Texas
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  • Monica Bessler MD, PhD,

    1. Comprehensive Bone Marrow Failure Center, Division of Hematology, Department of Pediatrics, The Children's Hospital of Philadelphia and Division of Hemato-Oncology, Department of Medicine, Perlman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania
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  • Thomas Coates MD,

    1. Children's Center for Cancer and Blood Diseases, Children's Hospital of Los Angeles, Los Angeles, California
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  • Seth Corey MD, MPH,

    1. Division of Hematology/Oncology, Lurie Children's Hospital of Chicago, Chicago, Illinois
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  • Yigal Dror MD,

    1. Genetics and Genome Biology Program, Research Institute and Marrow Failure and Myelodysplasia Program, Division of Hematology/Oncology, Department of Pediatrics, The Hospital for Sick Children an University of Toronto, Toronto, Canada
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  • James Huang MD,

    1. Division of Pediatric Hematology/Oncology, Department of Pediatrics, University of California San Francisco and Benioff Children's Hospital, San Francisco, California
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  • Jeffrey Lipton MD, PhD,

    1. Cohen Children's Medical Center of New York, New Hyde Park, New York
    2. Feinstein Institute for Medical Research, Manhasset, New York
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  • Timothy S. Olson MD, PhD,

    1. Comprehensive Bone Marrow Failure Center, Division of Hematology, Department of Pediatrics, The Children's Hospital of Philadelphia and Division of Hemato-Oncology, Department of Medicine, Perlman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania
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  • Ulrike M. Reiss MD,

    1. Department of Hematology, St. Jude Children's Research Hospital, Memphis, Tennessee
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  • Zora R. Rogers MD,

    1. The University of Texas Southwestern, Dallas, Texas
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  • Colin Sieff MD,

    1. Division of Hematology/Oncology, Boston Children's Hospital and Dana-Farber Cancer Institute, Harvard Medical School, Boston, Massachusetts
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  • Adrianna Vlachos MD,

    1. Cohen Children's Medical Center of New York, New Hyde Park, New York
    2. Feinstein Institute for Medical Research, Manhasset, New York
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  • Kelly Walkovich MD,

    1. Division of Hematology/Oncology, Department of Pediatrics and Communicable Diseases, University of Michigan C.S. Mott Children's Hospital, Ann Arbor, Michigan
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  • Winfred Wang MD,

    1. Department of Hematology, St. Jude Children's Research Hospital, Memphis, Tennessee
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  • Akiko Shimamura MD

    Corresponding author
    1. Fred Hutchison Cancer Research Center and Seattle Children's Hospital, Seattle, Washington
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  • Conflict of interest: Nothing to declare.

Abstract

Background

Randomized clinical trials in pediatric aplastic anemia (AA) are rare and data to guide standards of care are scarce.

Procedure

Eighteen pediatric institutions formed the North American Pediatric Aplastic Anemia Consortium to foster collaborative studies in AA. The initial goal of NAPAAC was to survey the diagnostic studies and therapies utilized in AA.

Results

Our survey indicates considerable variability among institutions in the diagnosis and treatment of AA. There were areas of general consensus, including the need for a bone marrow evaluation, cytogenetic and specific fluorescent in situ hybridization assays to establish diagnosis and exclude genetic etiologies with many institutions requiring results prior to initiation of immunosuppressive therapy (IST); uniform referral for hematopoietic stem cell transplantation as first line therapy if an HLA-identical sibling is identified; the use of first-line IST containing horse anti-thymocyte globulin and cyclosporine A (CSA) if an HLA-identical sibling donor is not identified; supportive care measures; and slow taper of CSA after response. Areas of controversy included the need for telomere length results prior to IST, the time after IST initiation defining a treatment failure; use of hematopoietic growth factors; the preferred rescue therapy after failure of IST; the use of specific hemoglobin and platelet levels as triggers for transfusion support; the use of prophylactic antibiotics; and follow-up monitoring after completion of treatment.

Conclusions

These initial survey results reflect heterogeneity in diagnosis and care amongst pediatric centers and emphasize the need to develop evidence-based diagnosis and treatment approaches in this rare disease. Pediatr Blood Cancer 2014;61:869–874. © 2013 Wiley Periodicals, Inc.

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