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Free keywords:
IMMUNE-RESPONSES; PATHOPHYSIOLOGY; RECONSTITUTION; SURVIVAL; DISEASEaplastic anemia; T-cell receptor V-beta chain repertoire; children;
Abstract:
One of the major obstacles of immunosuppressive therapy (IST) in
children with severe aplastic anemia (SAA) comes from the often
months-long unpredictability of bone-marrow (BM) recovery. In this
prospective study in children with newly diagnosed very severe AA (n =
10), who were enrolled in the therapy study SAA-BFM 94, we found a
dramatically reduced diversity of both CD4+ and CD8+ BM cells, as scored
by comprehensive V-beta chain T-cell receptor (TCR) analysis. Strongly
skewed TCR V-beta pattern was highly predictive for good or at least
partial treatment response (n = 6, CD8+ complexity scoring median 35.5,
range 24-73). In contrast, IST in patients with rather moderate
reduction of TCR V-beta diversity (n = 4, CD8+ complexity scoring median
109.5, range 82-124) always failed (P = 0.0095). If confirmed in a
larger series of patients, TCR V-beta repertoire in BM may help to
assign children with SAA up-front either to IST or to allogeneic
stem-cell transplantation. Blood Cancer Journal (2011) 1, e8;
doi:10.1038/bcj.2011.6; published online 4 March 2011
