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.