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

Brock, Katharine MD; Goldenberg, Neil MD, PhD; Graham, Douglas K. MD, PhD; Liang, Xiayuan MD; Hays, Taru MD

Journal of Pediatric Hematology/Oncology: March 2013 - Volume 35 - Issue 2 - p 148–152
doi: 10.1097/MPH.0b013e3182755f36
Original Articles

Introduction: 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.

Results: 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.

Discussion: 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.

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

The authors declare no conflict of interest.

Reprints: Taru Hays, MD, Department of Pediatrics, Center for Cancer and Blood Disorders, Section of Hematology/Oncology/Bone Marrow Transplant, University of Colorado/Children’s Hospital Colorado, 13123 E. 16th Ave, B-115 Aurora, CO 80045 (e-mail: taru.hays@childrenscolorado.org).

Received March 11, 2012

Accepted September 20, 2012

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