Diagnosis and treatment of acquired aplastic anaemia in adults: 142 cases from a multicentre, prospective cohort study in Shanghai, China.

To improve diagnosis and therapy for aplastic anaemia (AA) in Shanghai, clinical and laboratory data for patients with AA (n = 142) and hypocellular myelodysplastic syndrome (MDS; n = 22) were comparatively analysed (follow-up 2 - 6 years). Red blood cell distribution width and absolute lymphocyte and reticulocyte counts were significantly different between the two groups. AA was diagnosed in 54.2% of patients using a single bone marrow aspirate smear plus peripheral haemogram results, and in 95.1% using an additional bone marrow biopsy; 4.9% required multiple-site bone marrow examination. Clonal chromosomal abnormalities occurred in 3.9% and 31.8% of patients with AA and MDS, respectively. In patients with severe AA, 12.0% received antithymocyte globulin (ATG) + cyclosporin A (CSA; effectiveness rate 77.8%; 5-year survival 74.1%), 45.3% received CSA + androgen therapy (effectiveness rate 58.8%; 5-year survival 76.5%) and 26.7% received androgen monotherapy (effectiveness rate 25.0%). Multivariate analysis of prognostic factors indicated that therapy regimen and blood platelet count affected survival. Peripheral blood smears, bone marrow spicule classification and biopsy are important diagnostic factors. Standardization of evidence-based therapy and promotion of ATG + CSA would improve general therapeutic effects in AA.