Progress of diagnosis and management of non-severe aplastic anemia in children / 中华实用儿科临床杂志

ABSTRACT

Acquired aplastic anemia (AA) is a rare heterogeneous disease characterized by pancytopenia and hypoplastic bone marrow.The differential diagnosis should always take in account inherited forms of AA,like Fanconi anemia(FA),dyskeratosis-congenita(DC),and Shwachman-Diamond syndrome (SDS).Patient with transfusion-dependent non-severe aplastic anemia(NSAA),with severe AA (SAA) and very severe AA(VSAA),if an human leukocyte antigen (HLA) matched family donor(MFD) is found,then hematopoietic stem cell transplantation (HSCT) using bone marrow (BM) stem cells is the treatment of choice.If a MFD is not available,the immunosuppressive therapy (IST) with the combination of antithymocyte globulin (ATG) plus cyclosporin (CsA) still represents the first line choice.For transfusion-independent NSAA patients,most hematologists suggests no intervention,however,some studies indicate the patients with transfusion-independent NSAA may benefit from IST,and the rate of progression to SAA and transfusion-dependent NSAA is lower than other observation groups.So a multicenter randomized clinical trial is needed.