Analysis of clinical characteristics and prognosis of non-severe aplastic anemia children with chromosomal abnormalities

Abstract

Objective: To analyze the clinical characteristics and prognosis of non-severe aplastic anemia (NSAA) with chromosomal abnormalities in children. Method: A retrospective analysis of 304 cases with NSAA with successful karyotyping from 2001 to 2014 in the Institute of Hematology & Blood Disease Hospital was carried out. The treatment response, condition of blood transfusion were analyzed using χ2 test, the cumulative survival was estimated by the Kaplan-Meier method. Result: Out of 304 patients, 28 patients had chromosomal abnormalities with trisomy 8 (7 cases, 25.0%), abnormalities in chromosome 7 (5 cases, 17.9%), and other types (16 cases, 57.1%). There were no significant differences in the treatment response(40.9% (9/22)vs. 58.6%(119/203), χ2=2.539, P=0.111), the rate of getting rid of blood transfusion(54.5%(6/11) vs. 65.0%(39/60), χ2=6.455, P=0.086), five-year progression-free survival (49.2% vs.70.8%, χ2=0.849, P=0.357), and five-year cumulative survival (79.1% vs. 92.8%, χ2=0.330, P=0.556) between the patients with or without chromosomal abnormalities. There were significant differences in the rate of disease progression(41.7%(10/24) vs. 22.3%(48/215), χ2=4.394, P=0.045), the incidence of myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) (20.8%(5/24)vs. 0.9%(2/215), χ2=30.082, P=0.000)and the five-year cumulative incidence of MDS or AML(33.4% vs. 0.8%, χ2=17.798, P=0.000)between children with and without chromosomal abnormalities. Conclusion: The incidence of chromosomal abnormalities in children with NSAA is 9.2%. The clinical features and treatment response are similar, but children with chromosomal abnormalities have a poorer prognosis, and have higher risk of progressing to MDS or AML.