Moderate Aplastic Anemia in Children: Preliminary Outcomes for Treatment Versus Observation From a Single-Institutional Experience

Departments of §Pediatrics, Center for Cancer and Blood Disorders, Section of Hematology/Oncology/Bone Marrow Transplant *Pediatrics ∥Pathology, University of Colorado/Children's Hospital Colorado, Aurora, CO †Department of Pediatrics and Medicine, Johns Hopkins School of Medicine, Division of Hematology, Baltimore, MD ‡All Children's Research Institute, All Children's Hospital, Johns Hopkins Medicine (ACH-JHM), St Petersburg, FL.
Journal of Pediatric Hematology/Oncology (Impact Factor: 0.9). 11/2012; 35(2). DOI: 10.1097/MPH.0b013e3182755f36
Source: PubMed


Because of the variety of definitions used to describe moderate aplastic anemia (MAA), we review our institutional experience period with patients who met a proposed set of criteria for this disorder. On an exploratory basis, we sought to evaluate the influence of treatment with immunosuppressive therapy (IST) versus observation on long-term outcomes.

Materials and methods:
Records from 1999 to 2010 were screened for patients who met the criteria for MAA: (1) bone marrow cellularity of 20% to 50%; (2) cytopenias in at least 1 cell line (absolute neutrophil count<1000/µL, hemoglobin<9 g/dL, platelet count<100,000/µL); (3) mean corpuscular volume ≥90; (4) persistence >6 months; and (5) negative Fanconi studies. Data were collected for patient/disease characteristics, treatments, and outcomes.

Eight patients met the criteria for MAA. Three of 8 patients received IST. Of 3 patients who received IST, complete response was observed in 2 and transfusion independence in 1, as compared with 2 of 5 and 3 of 5 in the group who were observed without IST. Median duration of follow-up was 48 months.

As several patients spontaneously resolved, and none developed severe aplastic anemia, acute myelogenous leukemia, or myelodysplastic syndrome, the criteria used here may identify a group of children with favorable prognosis who can be managed supportively.

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