Natural history of moderate aplastic anemia in children

Authors

  • Scott C. Howard MD, MS,

    Corresponding author
    1. Department of Hematology-Oncology, St. Jude Children's Research Hospital, Memphis, Tennessee
    2. Department of Pediatrics, University of Tennessee Health Science Center, Memphis, Tennessee
    • Department of Hematology-Oncology and International Outreach Program, St. Jude Children's Research Hospital, 332 North Lauderdale, Memphis, TN 38105-2794.
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  • Paula E. Naidu MPH,

    1. Department of Hematology-Oncology, St. Jude Children's Research Hospital, Memphis, Tennessee
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  • X. Joan Hu PhD,

    1. Department of Biostatistics, St. Jude Children's Research Hospital, Memphis, Tennessee
    2. Department of Mathematical Sciences, University of Memphis, Memphis, Tennessee
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  • Michael R. Jeng MD,

    1. Stanford University School of Medicine, Stanford, California
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  • Carlos Rodriguez-Galindo MD,

    1. Department of Hematology-Oncology, St. Jude Children's Research Hospital, Memphis, Tennessee
    2. Department of Pediatrics, University of Tennessee Health Science Center, Memphis, Tennessee
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  • Martha D. Rieman FNP,

    1. Department of Hematology-Oncology, St. Jude Children's Research Hospital, Memphis, Tennessee
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  • Winfred C. Wang MD

    1. Department of Hematology-Oncology, St. Jude Children's Research Hospital, Memphis, Tennessee
    2. Department of Pediatrics, University of Tennessee Health Science Center, Memphis, Tennessee
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Abstract

Background

Moderate aplastic anemia (MAA) in children is a rare, idiopathic condition of bone marrow insufficiency that can resolve spontaneously, persist for months or years, or progress to severe aplastic anemia (SAA). We evaluated the rate of progression to SAA.

Methods

We reviewed the records of 136 children referred for evaluation of bone marrow failure from 1978 to 2002 at St. Jude Children's Research Hospital. MAA was defined by a hypocellular bone marrow (<50%) and 2 or 3 cytopenias (absolute neutrophil count <1,500/mm3, absolute reticulocyte count <40,000/mm3, platelet count <100,000/mm3) lasting at least 6 weeks.

Results

Twenty-four patients met the criteria for MAA. At a median follow-up of 66 months (range, 10–293), 16 patients (67%) progressed to SAA, 5 (21%) had persistent MAA, and 3 (12%) had complete resolution of MAA. No risk factors for progression could be identified.

Conclusions

When childhood MAA is treated with supportive care alone, 2/3 of patients progress to SAA. © 2004 Wiley-Liss, Inc.

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