Patients
Data of pediatric SAA patients treated with IST combined with rATG and CSA were analyzed retrospectively from March 2013 to July 2020 at Hematology Oncology Center, Beijing Children’s Hospital, Capital Medical University, National Center for Children’s Health.
Inclusion criteria were: <18 years of age; previously untreated SAA. Patients with a diagnosis of an inherited bone marrow failure syndrome were excluded, as well as subjects with a human leukocyte antigen matched sibling who underwent transplantation.
The collected data included age, sex, clinical classification, previous therapy and IST, hematological response, chromosome karyotype and status at the final follow-up, etc. Every pediatric case underwent bone marrow aspirate and biopsy, cytogenetics, fluorescence in situ hybridization (FISH), clonal analysis for paroxysmal nocturnal hemoglobinuria (PNH), telomere flow-FISH analysis and immunological analyses. Follow up was performed for a minimum of 1 year or until death, and was completed in July 2021.