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Pediatr Blood Cancer. 2013 Mar;60(3):455-60. doi: 10.1002/pbc.24247. Epub 2012 Jul 18.

Immune markers of disease severity and treatment response in pediatric acquired aplastic anemia.

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Departments of Pediatrics and Cell & Developmental Biology, Papé Family Pediatric Research Institute, Oregon Stem Cell Center, Oregon Health & Science University, Portland, Oregon 97239, USA.



To investigate the immune status among pediatric patients with aplastic anemia (AA) and explore PNH-status, T-regulatory and NK-cell frequency as potential markers of clinical response.


Data were retrospectively analyzed from twenty-six patients diagnosed with AA. PNH populations, T- and NK-subsets were determined via flow cytometry.


At diagnosis, 9/23 patients with severe AA (SAA) versus 1/3 with moderate AA (MAA) were PNH(pos) . Among PNH(pos) patients treated with ATG based immunosuppression, 2/6 had a complete response (CR), while 4/6 had a partial response (PR), similarly 2/6 PNH(neg) patients had a CR and 4/6 had a PR. Lymphocyte subset immunophenotyping revealed that T-regulatory cells represented 7.2% of total lymphocytes at diagnosis. Their frequency varied with disease severity (5.5% for SAA and 14.1% for MAA) and response (8.9% for CR and 1.5% for PR), generally increasing following therapy with IST (14.6%). The NK cell frequency was not substantially different based on disease severity or response.


Neither PNH cell populations, nor NK cell frequency corresponded with disease severity or response. T-regulatory cell frequency, although not statistically significant given the small sample size, corresponded with both severity and response, indicating potential utility as a prognostic tool.

[Indexed for MEDLINE]

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