Predictive Value of T Cell Receptor Repertoire Profiling for Immunosuppressive Therapy in Severe Aplastic Anemia

Increasing evidence supports the hypothesis of autologous immune attack in severe aplastic anemia(SAA):the pre-dominant role of activated cytotoxic T cells(CTL)expressing γ-interferon in inhibiting the growth of bone marrow(BM)cells,putative autoantigens,and oligoclonal expansion of CD8+T cells.1 For SAA patients,the definitive therapies are immunosuppressive therapy(IST)or he-matopoietic stem transplantation(HSCT);IST is most widely applied in the clinic because of the lack of HLA-matched sibling or unrelated donors,patients'age,and the cost of HSCT.2,3 However,only about 60％of SAA patients are responders after receiving IST,and less than 10％ach-ieve complete remission(CR)2,3;effective biomarkers for the efficacy prediction of IST in SAA patients are lacking.3 Our previous publications have demonstrated that T cell receptor(TCR)repertoire profiling has been identified as a biomarker for predicting the clinical outcomes and efficacy of patients.4,5 However,systematic evaluation of the pre-dictive value of the TCR repertoire for SAA patients during IST is still little known.