Conflict of interest: Nothing to report.
Immune markers of disease severity and treatment response in pediatric acquired aplastic anemia†
Article first published online: 18 JUL 2012
Copyright © 2012 Wiley Periodicals, Inc.
Pediatric Blood & Cancer
Volume 60, Issue 3, pages 455–460, March 2013
How to Cite
Sutton, K. S., Shereck, E. B., Nemecek, E. R. and Kurre, P. (2013), Immune markers of disease severity and treatment response in pediatric acquired aplastic anemia. Pediatr. Blood Cancer, 60: 455–460. doi: 10.1002/pbc.24247
- Issue published online: 15 JAN 2013
- Article first published online: 18 JUL 2012
- Manuscript Accepted: 7 JUN 2012
- Manuscript Received: 3 APR 2012
- aplastic anemia;
- PNH clones
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 PNHpos. Among PNHpos patients treated with ATG based immunosuppression, 2/6 had a complete response (CR), while 4/6 had a partial response (PR), similarly 2/6 PNHneg 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. Pediatr Blood Cancer 2013; 60: 455–460. © 2012 Wiley Periodicals, Inc.