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

Research output: Contribution to journalArticle

14 Citations (Scopus)

Abstract

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

Original languageEnglish (US)
Pages (from-to)455-460
Number of pages6
JournalPediatric Blood and Cancer
Volume60
Issue number3
DOIs
StatePublished - Mar 2013

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Aplastic Anemia
Immune System Diseases
Biomarkers
Pediatrics
Natural Killer Cells
Regulatory T-Lymphocytes
Therapeutics
Immunophenotyping
Lymphocyte Subsets
Sample Size
Immunosuppression
Population
Flow Cytometry
Lymphocytes

Keywords

  • Aplastic anemia
  • Immunosuppression
  • PNH clones

ASJC Scopus subject areas

  • Oncology
  • Pediatrics, Perinatology, and Child Health
  • Hematology

Cite this

Immune markers of disease severity and treatment response in pediatric acquired aplastic anemia. / Sutton, Kathryn S.; Shereck, Evan; Nemecek, Eneida; Kurre, Peter.

In: Pediatric Blood and Cancer, Vol. 60, No. 3, 03.2013, p. 455-460.

Research output: Contribution to journalArticle

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abstract = "Background: 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. Methods: Data were retrospectively analyzed from twenty-six patients diagnosed with AA. PNH populations, T- and NK-subsets were determined via flow cytometry. Results: 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. Conclusions: 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.",
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