TY - JOUR
T1 - Moderate aplastic anemia in children
T2 - Preliminary outcomes for treatment versus observation from a single-institutional experience
AU - Brock, Katharine
AU - Goldenberg, Neil
AU - Graham, Douglas K.
AU - Liang, Xiayuan
AU - Hays, Taru
N1 - Copyright:
Copyright 2013 Elsevier B.V., All rights reserved.
PY - 2013/3
Y1 - 2013/3
N2 - 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.
AB - 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.
KW - children
KW - hypoplastic anemia
KW - immunosuppressive therapy
KW - moderate aplastic anemia
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U2 - 10.1097/MPH.0b013e3182755f36
DO - 10.1097/MPH.0b013e3182755f36
M3 - Article
C2 - 23128338
AN - SCOPUS:84874456265
SN - 1077-4114
VL - 35
SP - 148
EP - 152
JO - Journal of Pediatric Hematology/Oncology
JF - Journal of Pediatric Hematology/Oncology
IS - 2
ER -